UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
 
  
FORM 10-Q
 
 
(Mark One)
 
ý       QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
  For the quarterly period ended June 30, 2018
  or
o          TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the transition period from               to            
Commission file number 001-37418
 
Axovant Sciences Ltd.
(Exact name of registrant as specified in its charter)
 
Bermuda
 
98-1333697
(State or other jurisdiction of
incorporation or organization)
 
(I.R.S. Employer
Identification No.)
 
 
 
Suite 1, 3rd Floor
11-12 St. James's Square
London SW1Y 4LB, United Kingdom
 
Not Applicable
(Address of principal executive offices)
 
(Zip Code)
Registrant’s telephone number, including area code: +44 203 318 9708
 
 

(former name, former address and former fiscal year, if changed since last report)
 
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.   Yes   ý    No   o
Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if any, every Interactive Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit and post such files).   Yes   ý    No   o
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of "large accelerated filer", "accelerated filer", "smaller reporting company," and "emerging growth company" in Rule 12b-2 of the Exchange Act. (Check one):
Large accelerated filer
o
Accelerated filer
x
Non-accelerated filer
o   (Do not check if a smaller reporting company)
Smaller reporting company
o
 
 
Emerging growth company
x
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. x

 Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act).   Yes   o    No   ý
 The number of shares outstanding of the Registrant’s common shares, $0.00001 par value per share, on August 3, 2018 , was 122,080,788 .






AXOVANT SCIENCES LTD.
QUARTERLY REPORT ON FORM 10-Q
FOR THE QUARTER ENDED JUNE 30, 2018
 
TABLE OF CONTENTS
 
 
Page
 
 
 
 
 


2



PART I.                                                   FINANCIAL INFORMATION

Item 1.                                                          Financial Statements (Unaudited)

AXOVANT SCIENCES LTD.
Condensed Consolidated Balance Sheets
(Unaudited, in thousands, except share and per share data)


 
 

 June 30, 2018
 
March 31, 2018
Assets
 

 
 

Current assets:
 

 
 

Cash
$
92,934

 
$
154,337

Prepaid expenses and other current assets
7,448

 
2,174

Income tax receivable
1,616

 
1,751

Total current assets
101,998

 
158,262

 
 
 
 
Other non-current assets
3,784

 

Property and equipment, net
1,802

 
2,524

 
 
 
 
Total assets
$
107,584

 
$
160,786

 
 
 
 
Liabilities and Shareholders’ Equity
 

 
 

Current liabilities:
 

 
 

Accounts payable
$
1,431

 
$
3,949

Due to RSL, RSI and RSG
2,156

 
1,011

Accrued expenses
25,133

 
31,862

Current portion of long-term debt
14,791

 
9,753

Total current liabilities
43,511

 
46,575

 
 
 
 
   Long-term debt
38,247

 
42,925


 
 
 
Total liabilities
81,758

 
89,500

 
 
 
 
Commitments and contingencies (Note 11)


 


 
 
 
 
Shareholders’ equity:
 

 
 

Common shares, par value $0.00001 per share, 1,000,000,000 shares authorized, 107,795,074 and 107,788,074 issued and outstanding at June 30, 2018 and March 31, 2018, respectively
1

 
1

Additional paid-in capital
633,979

 
628,110

Accumulated deficit
(608,839
)
 
(556,951
)
Accumulated other comprehensive income
685

 
126

Total shareholders’ equity
25,826

 
71,286

Total liabilities and shareholders’ equity
$
107,584

 
$
160,786

 
The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

3



AXOVANT SCIENCES LTD.
Condensed Consolidated Statements of Operations
(Unaudited, in thousands, except share and per share amounts)

 
Three Months Ended June 30,
 
2018

2017
Operating expenses:
 
 
 
Research and development expenses (1)
 
 
 
(includes total share-based compensation expense of $2,517 and $6,256 for the three months ended June 30, 2018 and 2017, respectively)
$
37,418


$
43,712

General and administrative expenses (2)
 
 
 
(includes total share-based compensation expense of $3,342 and $9,344 for the three months ended June 30, 2018 and 2017, respectively)
11,754


21,518

Total operating expenses
49,172


65,230

Other expenses:
 
 
 
Interest expense
1,970

 
1,874

Other expense (income)
668

 
(357
)
Loss before income tax expense
(51,810
)

(66,747
)
Income tax expense
78


2,519

Net loss
$
(51,888
)

$
(69,266
)
Net loss per common share — basic and diluted
$
(0.48
)

$
(0.65
)
Weighted average common shares outstanding — basic and diluted
107,789,920


106,400,912

 
(1) Includes $2,619 and $3,001 of total costs allocated from RSL, RSI and RSG for the three months ended June 30, 2018 and June 30, 2017, respectively.

(2) Includes $1,302 and $1,873 of total costs allocated from RSL, RSI and RSG for the three months ended June 30, 2018 and June 30, 2017, respectively.

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.








4



AXOVANT SCIENCES LTD.
Condensed Consolidated Statements of Comprehensive Loss
(Unaudited, in thousands)


 
Three Months Ended June 30,
 
2018
 
2017
 
 
 
 
Net loss
$
(51,888
)
 
$
(69,266
)
Other comprehensive income (loss):
 
 
 
Foreign currency translation adjustment
559

 
(349
)
  Total other comprehensive income (loss)
559

 
(349
)
Comprehensive loss
$
(51,329
)
 
$
(69,615
)

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.


5



AXOVANT SCIENCES LTD.
Condensed Consolidated Statement of Shareholders’ Equity
(Unaudited, in thousands, except share data)

 
Common Shares
 
Additional  Paid-in Capital
 
Accumulated
Deficit
 
Accumulated Other Comprehensive Income
 
Total
Shareholders’
Equity
 
Shares
 
Amount
 
 
 
 
 
 
 
 
 
 
 
 
Balance at March 31, 2018
107,788,074

 
$
1

 
$
628,110

 
$
(556,951
)
 
$
126

 
$
71,286

Exercise of stock options
7,000

 

 
10

 

 

 
10

Share-based compensation expense

 

 
5,369

 

 

 
5,369

Capital contribution — share-based compensation expense

 

 
490

 

 

 
490

Foreign currency translation adjustment

 

 

 

 
559

 
559

Net loss

 

 

 
(51,888
)
 

 
(51,888
)
Balance at June 30, 2018
107,795,074

 
$
1

 
$
633,979

 
$
(608,839
)
 
$
685

 
$
25,826

 

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.


6



AXOVANT SCIENCES LTD.
Condensed Consolidated Statements of Cash Flows
(Unaudited, in thousands)

 
 
Three Months Ended June 30,
 
2018
 
2017
Cash flows from operating activities:
 

 
 
Net loss
$
(51,888
)
 
$
(69,266
)
Adjustments to reconcile net loss to net cash used in operating activities:
 

 
 
Foreign currency translation adjustment
559

 
(349
)
Share-based compensation
5,859

 
15,600

Depreciation and non-cash amortization
1,034

 
335

Deferred tax assets

 
2,709

Changes in operating assets and liabilities:
 

 
 
Prepaid expenses and other current assets
(5,274
)
 
(1,170
)
Other non-current assets
(3,784
)
 

Accounts payable
(2,518
)
 
(4
)
Due to RSL, RSI and RSG
1,193

 
1,547

Accrued expenses
(6,729
)
 
3,245

Income tax receivable
135

 
(566
)
Net cash used in operating activities
(61,413
)
 
(47,919
)
Cash flows from investing activities:
 

 
 
    Purchases of property and equipment

 
(2,171
)
Net cash used in investing activities

 
(2,171
)
Cash flows from financing activities:
 

 
 
    Exercise of stock options
10

 
747

    Cash proceeds from issuance of common shares, net of costs

 
134,628

Net cash provided by financing activities
10

 
135,375

Net change in cash
(61,403
)
 
85,285

Cash—beginning of period
154,337

 
212,573

Cash—end of period
$
92,934

 
$
297,858

 

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

7



AXOVANT SCIENCES LTD.
Notes to Condensed Consolidated Financial Statements (Unaudited)

Note 1—Description of Business
 
Axovant Sciences Ltd., together with its wholly owned subsidiaries (the "Company"), is a clinical-stage biopharmaceutical company focused on the acquisition, development and commercialization of novel therapeutics in the fields of neurology and psychiatry. The Company is developing a pipeline of clinical and nonclinical product candidates that focuses on the various aspects of debilitating neurological diseases such as Parkinson's disease, oculopharyngeal muscular dystrophy, Lewy body dementia ("LBD") and other indications in the fields of neurology and psychiatry.

The Company is an exempted limited company incorporated under the laws of Bermuda in October 2014 under the name Roivant Neurosciences Ltd. The Company changed its name to Axovant Sciences Ltd. in March 2015 . The Company has six wholly owned subsidiaries. Axovant Holdings Limited ("AHL"), a direct wholly owned subsidiary of Axovant Sciences Ltd., was incorporated in England and Wales in August 2016; Axovant Sciences, Inc. ("ASI"), a direct wholly owned subsidiary of AHL, was incorporated in Delaware in February 2015; Axovant Sciences GmbH ("ASG"), a direct wholly owned subsidiary of AHL, was organized in Switzerland in August 2016; Axovant Sciences America, Inc. ("ASA"), a direct wholly owned subsidiary of AHL, was incorporated in Delaware in July 2017; and Axovant Treasury Holdings, Inc. ("ATH"), a direct wholly owned subsidiary of ASL and Axovant Treasury, Inc. ("ATI"), a direct wholly owned subsidiary of ATH, were each incorporated in Delaware in March 2018. ASG holds all of the Company's intellectual property rights and is the principal operating company for conducting the Company’s business.
The Company's near-term focus is to develop its gene therapy product candidates AXO-Lenti-PD, a potential one-time treatment for Parkinson's disease, and AXO-AAV-OPMD, a potential one-time treatment for oculopharyngeal muscular dystrophy ("OPMD"). The Company intends to begin a Phase 1/2 study of AXO-Lenti-PD in advanced Parkinson's disease patients before the end of 2018 and a placebo-controlled study of AXO-AAV-OPMD in OPMD patients in 2019. Prior to the recent in-licensing of AXO-Lenti-PD in June 2018 and AXO-AAV-OPMD in July 2018, the Company's primary focus had been on developing nelotanserin, a selective inverse agonist of the 5-HT 2A receptor, and intepirdine, an antagonist of the 5-HT 6 receptor. In January 2018, the Company announced the results of a pilot Phase 2 study of nelotanserin in patients with LBD that experience visual hallucinations. The Company plans to make a determination of the overall development strategy for nelotanserin once it has reviewed topline data in the second half of 2018 from its currently ongoing Phase 2 study of nelotanserin in REM Sleep Behavior Disorder ("RBD") in LBD patients, and has completed its ongoing comprehensive clinical, regulatory and commercial review. In addition, the Company will determine its development plans for RVT-104, a combination of rivastigmine and a peripheral muscarinic receptor antagonist, as a potential treatment for patients with Alzheimer's disease or dementia with Lewy bodies ("DLB"), which is a sub-type of LBD, once it has completed its ongoing comprehensive clinical, regulatory and commercial review in the context of its recent acquisition of AXO-Lenti-PD, AXO-AAV-OPMD and any other newly acquired product candidates.

From its inception, the Company has devoted substantially all of its efforts to organizing and staffing the Company, raising capital, acquiring product candidates and advancing its product candidates into clinical development. The Company has determined that it has one operating and reporting segment as it allocates resources and assesses financial performance on a consolidated basis. The Company does not expect to generate revenue unless and until it successfully completes development and obtains regulatory approval for one of its product candidates. The Company believes it currently has sufficient funds to meet its financial needs for at least the next 12 months. The Company will be required to obtain further funding through public or private offerings of its share capital, debt financing, collaboration and licensing arrangements or other sources. Adequate additional funding may not be available to the Company on acceptable terms, or at all.



8



Note 2—Summary of Significant Accounting Policies
 
(A) Basis of Presentation:
 
The Company’s fiscal year ends on March 31, and its fiscal quarters end on June 30, September 30 and December 31.
 
The accompanying interim unaudited condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States ("U.S. GAAP") for interim financial information and with the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. These interim unaudited condensed consolidated financial statements should be read in conjunction with the Company’s audited consolidated financial statements and notes thereto included in the Company’s Annual Report on Form 10-K for the fiscal year ended March 31, 2018 the ("Annual Report"), filed with the Securities and Exchange Commission ("SEC") on June 11, 2018. In the opinion of management, all adjustments (consisting of normal recurring adjustments) considered necessary to present fairly the financial position of the Company and its results of operations and cash flows for the interim periods presented have been included. Operating results for the three months ended June 30, 2018 are not necessarily indicative of the results that may be expected for the year ending March 31, 2019, for any other interim period, or for any other future year.
 
Any reference in these notes to applicable guidance is meant to refer to the authoritative U.S. GAAP as found in the Accounting Standards Codification ("ASC") and Accounting Standards Update ("ASU") issued by the Financial Accounting Standards Board ("FASB"). The unaudited condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. The Company has no unconsolidated subsidiaries. All intercompany balances and transactions have been eliminated in consolidation.
 
There have been no significant changes in the Company’s accounting policies from those disclosed in its Annual Report.
 
(B) Use of Estimates:
 
The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. The Company regularly evaluates estimates and assumptions related to the assets, liabilities, costs and expenses (including compensation expense) allocated to the Company under its services agreements with Roivant Sciences, Inc. ("RSI") and Roivant Sciences GmbH ("RSG"), each a wholly owned subsidiary of the Company’s parent company, RSL, as well as contingent liabilities, share-based compensation and research and development costs. Specifically, the Company estimates the grant date fair value of stock option awards with only time-based vesting requirements using a Black-Scholes valuation model, and uses a Monte Carlo Simulation method under the income approach to estimate the grant date fair value of stock option awards with market-based performance conditions. The Company bases its estimates and assumptions on historical experience and on various other factors that it believes to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results could differ from those estimates.
 
(C) Net Loss per Common Share:
 
Basic net loss per common share is computed by dividing the net loss applicable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net loss per common share is computed by dividing the net loss applicable to common shareholders by the diluted weighted-average number of common shares outstanding during the period calculated in accordance with the treasury stock method. Stock options to purchase 14.9 million common shares were not included in the calculation of diluted weighted-average common shares outstanding for the three months ended June 30, 2018 because they were anti-dilutive given the net loss of the Company. Stock options and a warrant which, combined, would enable the purchase of an aggregate of 14.5 million common shares were not included in the calculation of diluted weighted-average common shares outstanding for the three months ended June 30, 2017 because they were anti-dilutive given the net loss of the Company.


9



(D) Fair Value Measurements:

The Company utilizes fair value measurement guidance prescribed by accounting standards to value its financial instruments. The guidance establishes a fair value hierarchy for instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and the Company’s own assumptions (unobservable inputs). Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are inputs that reflect the Company’s assumptions about the inputs that market participants would use in pricing the asset or liability and are developed based on the best information available in the circumstances.

Fair value is defined as the exchange price, or exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. As a basis for considering market participant assumptions in fair value measurements, the guidance establishes a three-tier fair value hierarchy that distinguishes among the following:

Level 1-Valuations are based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access.
Level 2-Valuations are based on quoted prices for similar assets or liabilities in active markets, quoted prices for identical or similar assets or liabilities in markets that are not active and models for which all significant inputs are observable, either directly or indirectly.
Level 3-Valuations are based on inputs that are unobservable (supported by little or no market activity) and significant to the overall fair value measurement.

To the extent the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.
The Company’s financial instruments include cash, accounts payable and long-term debt. Cash and accounts payable are stated at their respective historical carrying amounts, which approximate fair value due to their short-term nature. The carrying value of the Company’s debt was $53.0 million as of June 30, 2018 and approximates fair value based on current interest rates for similar types of borrowings and is in Level 2 of the fair value hierarchy. See Note 5 for the actual book carrying value of the Company's long-term debt at June 30, 2018 .
(E) Recent Accounting Pronouncements:

In February 2016, the FASB issued ASU No. 2016-02, ‘‘ Leases (Topic 842) ’’ (‘‘ASU No. 2016-02’’), which is a comprehensive new lease standard that amends various aspects of existing accounting guidance for leases. The core principle of ASU No. 2016-02 will require lessees to present the assets and liabilities that arise from leases on their balance sheets. ASU No. 2016-02 is effective for annual periods beginning after December 15, 2018, and interim periods within fiscal years beginning after December 15, 2018. Early adoption is permitted. The Company is currently evaluating the provisions of ASU No. 2016-02 and its impact on our consolidated financial statements and related disclosures.

In January 2017, the FASB issued ASU 2017-01, ‘‘ Business Combinations (Topic 805): Clarifying the Definition of a Business ’’ (‘‘ASU No. 2017-01’’), which clarifies the definition of a business with the objective of adding guidance to assist entities with evaluating whether transactions should be accounted for as acquisitions (or disposals) of assets or businesses. The Company adopted the provisions of ASU No. 2017-01 on April 1, 2018 on a prospective basis. The impact on the Company's consolidated financial statements and disclosures will depend on the facts and circumstances of any specific future transactions. See Note 3 for further information regarding the impact of the adoption of ASU No. 2017-01 on its license agreement executed during the three months ended June 30, 2018.


10



In February 2018, the FASB issued ASU No. 2018-02, " Income Statement-Reporting Comprehensive Income (Topic 220): Reclassification of Certain Tax Effects from Accumulated Other Comprehensive Income" ("ASU No. 2018-02"). ASU No. 2018-02 allows companies to reclassify stranded tax effects resulting from the Tax Cuts and Jobs Act, from accumulated other comprehensive (loss) income to retained earnings. ASU No. 2018-02 is effective for interim and annual reporting periods beginning after December 15, 2018 and early adoption is permitted. The Company expects to adopt the provisions of ASU No. 2018-02 for the fiscal year beginning April 1, 2019. As the Company has not yet completed its final review of the impact of ASU No. 2018-02 but expects to by March 31, 2019, the Company has not determined whether the adoption of this guidance will have a material impact on its consolidated financial statements or disclosures. 

In March 2018, the FASB issued ASU No. 2018-05, " Income Taxes (Topic 740): Amendments to SEC Paragraphs Pursuant to SEC Staff Accounting Bulletin No. 118 ," ("ASU No. 2018-05"). ASU No. 2018-05 amends certain SEC material in Topic 740 for the income tax accounting implications of the Tax Cuts and Jobs Act. ASU No. 2018-05 was effective immediately. The Company evaluated the impact of the Tax Cuts and Jobs Act as well as the guidance of Staff Accounting Bulletin 118 and incorporated the changes into the determination of a reasonable estimate of deferred taxes and appropriate disclosures in the notes to the Company’s consolidated financial statements. The Company will continue to evaluate the impact this tax reform legislation may have on our results of operations, financial position, cash flows and related disclosures.

In June 2018, the FASB issued ASU No. 2018-07, " Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting," (ASU No. 2018-07) . ASU No. 2018-07 requires equity-classified share-based payment awards issued to nonemployees to be measured on the grant date, rather than remeasuring the awards through the performance completion date as previously required. Additionally, for nonemployee awards with performance conditions, compensation cost associated with the award is to be recognized when achievement of the performance condition is probable, rather than upon achievement of the performance condition. Further, the requirement to reassess the liability or equity classification for nonemployee awards upon vesting is eliminated, except for awards in the form of convertible instruments. ASU No. 2018-07 also clarifies that any share-based payment awards issued to customers should be evaluated under ASC 606, Revenue from Contracts with Customers . ASU No. 2018-07 is effective for fiscal years beginning after December 15, 2018, including interim periods within that fiscal year, with early adoption permitted after the adoption of ASU No. 2014-09. The Company expects to adopt the provisions of ASU No. 2018-07 for the fiscal year beginning April 1, 2019. As the Company has not yet completed its final review of the impact of ASU No. 2018-07 but expects to by March 31, 2019, the Company has not determined whether the adoption of this guidance will have a material impact on its consolidated financial statements or disclosures. 


Note 3—License Agreement

On June 5, 2018, the Company, through its wholly owned subsidiary, ASG, entered into an exclusive license agreement (the "Oxford BioMedica Agreement") with Oxford BioMedica (UK) Ltd. ("Oxford BioMedica"), pursuant to which the Company received a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Oxford BioMedica to develop and commercialize AXO-Lenti-PD and related gene therapy products for all diseases and conditions. In June 2018, as consideration for the license, the Company made an upfront nonrefundable payment to Oxford BioMedica of $30.0 million , $5.0 million of which will be applied as a credit against the process development work and clinical supply that Oxford BioMedica will provide to the Company. Under the terms of the Oxford BioMedica Agreement, the Company could be obligated to make payments to Oxford BioMedica totaling up to $55.0 million upon the achievement of specified development milestones and $757.5 million upon the achievement of specified regulatory and sales milestones. The Company will also be obligated to pay Oxford BioMedica a tiered royalty from 7% to 10% , based on yearly aggregate net sales of the underlying gene therapy products, subject to specified reductions upon the occurrence of certain events as set forth in the Oxford BioMedica Agreement. These royalties are required to be paid, on a product-by-product and country-by-country basis, until the latest to occur of the expiration of the last to expire valid claim of a licensed patent covering such product in such country, the expiration of regulatory exclusivity for such product in such country, or 10 years after the first commercial sale of such product in such country.

The Company is solely responsible, at its expense, for all activities related to the development and commercialization of the gene therapy products underlying the Oxford BioMedica Agreement. Pursuant to the Oxford BioMedica Agreement, the Company is required to use commercially reasonable efforts to develop, obtain regulatory approval of, and commercialize a gene therapy product underlying the Oxford BioMedica Agreement in the United States and at least one major market country in Europe. In addition, the Company is required to meet certain diligence milestones and to include at least one U.S.-based clinical trial site in a pivotal study of a gene therapy product underlying the Oxford BioMedica Agreement. If the Company fails to meet any of these specified development milestones, it may cure such failure by paying Oxford BioMedica certain fees, which range from $0.5 million to $1.0 million .

11




The Company has evaluated the Oxford BioMedica Agreement and has determined that the acquired set of assets and activities did not meet the definition of a business and thus the transaction was not considered a business combination. The Company determined that the IPR&D had not reached technological feasibility and therefore has no alternative future use. Accordingly, the Company recorded $25.0 million of the initial payment under the license agreement as research and development expense in the Company's unaudited condensed consolidated statements of operations during the three months ended June 30, 2018. As the remaining $5.0 million of the initial payment under the licensing agreement represents a nonrefundable payment for process development work and clinical supplies that Oxford BioMedica will provide over the term of the license agreement, the Company capitalized $1.25 million within prepaid expenses and other current assets and $3.75 million within other non-current assets in its unaudited condensed consolidated balance sheet as of June 30, 2018, which will be recorded to research and development expense as the process development work and clinical supplies are provided by Oxford BioMedica.

Note 4—Accrued Expenses

As of June 30, 2018 and March 31, 2018 , the Company’s accrued expenses consisted of the following (in thousands):

 
June 30, 2018
 
March 31, 2018
 
 
 
 
Research and development expenses
$
18,831

 
$
21,855

Salaries, bonuses, and other compensation expenses
2,722

 
7,718

Legal expenses
2,043

 
779

Other expenses
1,537

 
1,510

Total accrued expenses
$
25,133

 
$
31,862


Note 5—Long-term Debt

On February 2, 2017, the Company and its subsidiaries, AHL, ASG and ASI, entered into a loan and security agreement (as amended on May 24 and September 22, 2017, the "Loan Agreement") with Hercules Capital, Inc., ("Hercules"), under which the Company, AHL and ASG (the "Borrowers") borrowed an aggregate of $55.0 million (the "Term Loan"). Subsequently, the Company has added its subsidiary ASA as a Borrower in July 2017 and its subsidiaries ATH and ATI as Borrowers in April 2018. Pursuant to the Loan Agreement, ASI has issued a guaranty of the Borrowers’ obligations under the Loan Agreement. The Term Loan bears interest at a variable per annum rate calculated for any day as the greater of either (i) the prime rate plus 6.80% , and (ii) 10.55% . The Term Loan has a scheduled maturity date of March 1, 2021. The Borrowers are obligated to make monthly payments of accrued interest under the Loan Agreement until September 1, 2018, followed by monthly installments of principal and interest beginning October 1, 2018 through March 1, 2021. In connection with the Loan Agreement, the Borrowers and ASI, as guarantor, granted Hercules a first position lien on substantially all of their respective assets, excluding intellectual property. Prepayment of the Term Loan is subject to penalty.

On May 24, 2017, the Loan Agreement was amended such that, commencing July 1, 2017, the required minimum amount of unrestricted cash is equal to the lesser of (i) $35.0 million (the "Applicable Amount") plus certain aged accounts payable amounts (as further defined in the Loan Agreement) and (ii) the outstanding amount of debt under the Loan Agreement plus certain aged accounts payable (as further defined in the Loan Agreement), provided that the Applicable Amount may be lowered to $30 million upon the achievement of certain clinical milestones as set forth in the Loan Agreement.

The Loan Agreement also includes customary events of default. Upon the occurrence of an event of default, a default interest rate of an additional 5.0% may be applied to the outstanding principal balance, and Hercules may declare all outstanding obligations immediately due and payable and take such other actions as set forth in the Loan Agreement. At no time has the Company been in default under the provisions of the Loan Agreement. In addition, for so long as the Term Loan remains outstanding, the Company shall be required to use its commercially reasonable efforts to afford Hercules the opportunity to participate in future underwritten equity offerings of the Company’s common shares up to a total of $3.0 million .

In connection with the Loan Agreement, the Company issued a warrant to Hercules, exercisable for an aggregate of 274,086 of the Company’s common shares at an exercise price of $12.04 per share (the "Warrant"). In August 2017, Hercules exercised the Warrant on a cashless basis and received a net issuance of 129,827 of the Company's common shares. The Company has accounted for the Warrant as an equity instrument since it was indexed to the Company’s common shares and met the criteria for classification in shareholders’ equity. The relative fair value of the Warrant on the date of issuance was approximately $2.3 million and was treated as a discount to the debt. This amount will be amortized to interest expense under the effective interest

12



method over the life of the Term Loan, which is a period of 48 months. The Company estimated the value of the Warrant using the Black-Scholes model. The key assumptions used to value the Warrant were as follows:
Exercise price
 
$
12.04

Share price on date of issuance
 
$
11.96

Volatility
 
77.6
%
Risk-free interest rate
 
2.27
%
Expected dividend yield
 
%
Contractual term (in years)
 
7


In addition, at the closing of the Term Loan, the Company paid transaction costs of $1.5 million , which were recorded as a discount on the debt and will be amortized to interest expense using the effective interest method over the life of the Term Loan, which is a period of 48 months.
Outstanding debt obligations are as follows (in thousands):
 
 
June 30, 2018
 
March 31, 2018
 
 
 
 
 
Principal amount
 
$
55,000

 
$
55,000

Less: unamortized discount and debt issuance costs
 
(1,962
)
 
(2,322
)
Loan payable less unamortized discount and debt issuance costs
 
53,038

 
52,678

Less: current portion of long-term debt
 
(14,791
)
 
(9,753
)
Long-term loan payable, net of current maturities
 
$
38,247

 
$
42,925



Note 6—Related Party Transactions
 
(A) Services Agreements:

In 2015, the Company and ASI entered into a services agreement with RSI (the ‘‘Services Agreement’’) under which RSI has agreed to provide certain administrative and research and development services to the Company. The Company and ASI amended and restated the Services Agreement with RSI on October 13, 2015 effective for the fiscal year commencing April 1, 2015. Under the Services Agreement, as amended and restated, the Company pays or reimburses RSI for any expenses it, or third parties acting on its behalf, incurs for the Company. For any general and administrative and research and development activities performed by RSI employees, RSI charges back the employee compensation expense plus a pre-determined mark-up. Employee compensation expense, inclusive of base salary and fringe benefits, is determined based upon the relative percentage of time utilized on Company matters. All other costs are billed back at cost. The accompanying interim unaudited condensed consolidated financial statements include third-party expenses that have been paid by RSI and RSL.

In February 2017, the Company and ASI amended and restated the Services Agreement, effective as of December 13, 2016, to add ASG as a services recipient. In addition, in February 2017, ASG entered into a separate services agreement with RSG, effective as of December 13, 2016, for the provision of services by RSG to ASG in relation to the identification of potential product candidates and project management of clinical trials, as well as other services related to development, administrative and financial activities.

Under the Services Agreements, for the three months ended June 30, 2018 and 2017 , the Company incurred expenses of $3.4 million and $2.7 million , respectively, inclusive of the pre-determined mark-up.


13



(B) Family Relationships:
 
Geetha Ramaswamy, MD, the former Vice President, Medical and Scientific Strategy of ASI and an employee of RSI, is the mother of Vivek Ramaswamy, the Chairman of the Company's Board of Directors and former Chief Executive Officer of the Company. Sarah Friedhoff, formerly Senior Business Operations and Research and Development Specialist of ASI, is the daughter of Lawrence Friedhoff, MD, PhD, formerly the Chief Development Officer of ASI and currently Chief of Research & Development of RSI. Shankar Ramaswamy, MD, the Senior Vice President, Business Development of ASI, and a former employee of RSI, is the brother of Vivek Ramaswamy. Lawrence Friedhoff, MD, PhD, Geetha Ramaswamy, MD and Sarah Friedhoff were no longer employed by ASI beginning in October 2017.

Salary expenses for Shankar Ramaswamy, MD were $75,000 and $66,950 for the three months ended June 30, 2018 and 2017, respectively. Salary expenses for Geetha Ramaswamy, MD were $ 66,950 for the three months ended June 30, 2017 . Salary expenses for Sarah Friedhoff were $ 19,313 for the three months ended June 30, 2017 .

Note 7—Shareholders' Equity

In April 2017, the Company issued and sold 7,753,505 common shares, including 1,011,326 common shares sold pursuant to the exercise in full of the underwriters’ option to purchase additional shares, at an offering price of $18.54 per common share for gross proceeds of $143.7 million . The net proceeds to the Company were $134.6 million , after deducting underwriting discounts and commissions and offering expenses paid by the Company.

During the three months ended March 31, 2018, RSL incurred $0.3 million of expenses on behalf of the Company. This amount was treated as a capital contribution.

On June 5, 2018, the Company entered into a share purchase agreement with RSL, its majority shareholder, pursuant to which the Company agreed to issue and sell to RSL 14,285,714 of its common shares at a purchase price of $ 1.75 per share, which was the closing price per share of the Company's common shares on the Nasdaq Global Select Market on June 5, 2018. In July 2018, the Company received $25.0 million of net proceeds from RSL upon the closing of this private placement.

On June 22, 2018, the Company entered into a sales agreement with Cowen and Company, LLC ("Cowen") to sell the Company's common shares having an aggregate offering price of up to $ 75.0 million from time to time through an at-the-market equity offering program under which Cowen is acting as the Company's agent. Cowen is entitled to compensation for its services in an amount up to 3% of the gross proceeds of any of the Company's common shares sold under the sales agreement. As of June 30, 2018, $ 75.0 million of the Company's common shares remained available for sale under the sales agreement.

Note 8—Share-Based Compensation

In April 2017, the number of common shares authorized for issuance under the Company's 2015 Equity Incentive Plan increased automatically to an aggregate of approximately 16.5 million common shares in accordance with the terms of the 2015 Equity Incentive Plan. In June 2017, the Company's Board of Directors amended and restated the 2015 Equity Incentive Plan (the "2015 Plan") to, among other things, increase the number of common shares authorized for issuance thereunder to approximately 20.5 million common shares. The 2015 Plan became effective upon shareholder approval in August 2017. In April 2018, the number of common shares authorized for issuance under the 2015 Plan increased automatically to approximately 24.8 million common shares in accordance with the terms of the 2015 Plan. At June 30, 2018 , a total of 9.2 million common shares were available for future grant under the 2015 Plan, and options to purchase approximately 14.9 million common shares were outstanding under the 2015 Plan, with a weighted average exercise price of $4.95 per share.

(A) Stock Options Granted to Employees and Directors:

During the three months ended June 30, 2018 and 2017 , the Company granted options to its employees and directors under the 2015 Plan to purchase a total of 0.7 million and 7.1 million common shares, respectively. The stock options granted during the three months ended June 30, 2018 includes approximately 0.3 million common shares with market-based performance conditions to an employee with an exercise price of $3.79 per share, a contractual term of 10 years, and a corresponding estimated grant date fair value of $0.6 million . As of June 30, 2018, stock options with market-based performance conditions to purchase 1.3 million common shares were outstanding with a weighted-average exercise price of $1.91 per share. The market-based performance options vest based on exceeding certain closing prices of the Company's common stock. As of June 30, 2018, stock options with market-based performance conditions to purchase approximately 0.4 million common shares with a weighted-average exercise price of $1.46 per share were vested, which occurred during the three months ended June 30, 2018.


14



The Company recorded total share-based compensation expense related to stock options issued to Company employees and directors of $4.6 million and $12.2 million , respectively, for the three months ended June 30, 2018 and 2017. At June 30, 2018 , total unrecognized compensation expense related to non-vested options was $33.8 million , which is expected to be recognized over the remaining weighted-average service period of 2.35 years .
 
(B) Share-Based Compensation for Related Parties:

(1) Stock Options Granted to Non-Employees:
 
During the three months ended June 30, 2018 and 2017 , the Company granted options to purchase a total of 1,000,000 and 209,600 common shares, respectively, to employees of RSI and other consultants as compensation for support services provided to the Company. The fair value of the stock options granted to RSI employees and other consultants is accounted for by the Company in accordance with the authoritative guidance for non-employee equity awards and is remeasured on each valuation date until performance is complete using the Black-Scholes pricing model.
 
Each award is subject to a specified vesting schedule. Compensation expense will be recognized by the Company over the required service period to earn each award. The Company recorded $0.4 million and $1.0 million of share-based compensation expense for the three months ended June 30, 2018 and 2017, respectively. The share-based compensation was recorded as research and development and general and administrative expense in the accompanying unaudited condensed consolidated statements of operations. The total remaining unrecognized compensation cost related to the non-vested stock options amounted to $1.7 million as of June 30, 2018 , which is expected to be recognized over the remaining weighted-average service period of 2.50 years. 

(2) Share-Based Compensation Allocated to the Company by RSL:

The Company incurs share-based compensation expense for RSL common share awards and RSL options issued by RSL to RSL, RSG and RSI employees. Share-based compensation expense is allocated to the Company by RSL based upon the relative percentage of time utilized by RSL, RSG and RSI employees on Company matters.

The RSL common share awards are fair valued on the date of grant and that fair value is recognized over the requisite service period. Significant judgment and estimates were used to estimate the fair value of these awards, as they are not publicly traded. RSL common share awards are subject to specified vesting schedules and requirements (a mix of time-based, performance-based and corporate event-based, including targets for RSL’s post-IPO market capitalization and future financing events). The Company estimated the fair value of each RSL option on the date of grant using the Black-Scholes closed-form option-pricing model.

The Company recorded share-based compensation expense of $0.5 million and $2.1 million for the three months ended June 30, 2018 and 2017 , respectively, in relation to the RSL common share awards and options issued by RSL to RSG and RSI employees.

(3) Share-Based Compensation for Family Members:

The Company recorded aggregate share-based compensation expense of $0.8 million and $1.4 million for the three months ended June 30, 2018 and 2017 , respectively, in connection with options vesting for Geetha Ramaswamy, MD, Shankar Ramaswamy, MD and Sarah Friedhoff.

Shankar Ramaswamy, MD, while previously employed by RSI, was also granted RSL common shares. The Company recorded share-based compensation expense of $0.1 million and $0.1 million for the three months ended June 30, 2018 and 2017, respectively, related to the RSL common share awards held by Shankar Ramaswamy, which the Company has recorded as research and development expense in the accompanying unaudited condensed consolidated statements of operations. At June 30, 2018 , total unrecognized compensation expense related to these non-vested RSL common share awards was $7,401 and is expected to be recognized over the remaining weighted-average service period of 0.02 years .

Note 9 - Restructuring

In October 2017, the Company initiated and committed to the first of two corporate realignments to focus its efforts and resources on the Company's ongoing and future programs that included a reduction in its workforce and a transfer of certain employees to affiliates. The second realignment was initiated and committed to in February 2018. The Company completed the reduction in headcount from these actions in the fourth quarter of fiscal 2018.

15




During the three months ended June 30, 2018 , the Company made cash expenditures of approximately $1.0 million for one-time severance and related costs in connection with the corporate realignments completed in the prior year.

The impacted employees are eligible to receive severance payments in specified amounts, health benefits and outplacement services. The Company has recorded these charges in research and development and general and administrative expenses in the accompanying condensed consolidated statements of operations based on responsibilities of the impacted employees.

 
Balance as of
 
 
 
 
 
 
 
Balance as of
 
March 31, 2018
 
Expenses, net
 
Cash
 
Noncash
 
June 30, 2018
 
 
 
 
 
(in thousands)
 
 
 
 
Employee severance and
 
 
 
 
 
 
 
 
 
other personnel benefits
$
2,460

 
$

 
$
(978
)
 
$

 
$
1,482



Note 10—Income Taxes
 
The Company is not subject to taxation under the laws of Bermuda since it was organized as a Bermuda Exempted Limited Company, for which there is no current tax regime. The Company’s provision for income taxes is primarily federal, state and local income taxes in the United States. The Company assesses the realizability of its deferred tax assets at each balance sheet date based on available positive and negative evidence in order to determine the amount which is more likely than not to be realized and records a valuation allowance as necessary. The Company's effective tax rates of (0.2)% and (3.8)% for the three months ended June 30, 2018 and 2017, respectively, differ from the Bermuda federal statutory rate of 0% primarily due to the U.S. permanent unfavorable tax differences, stock compensation deductions and a valuation allowance that effectively eliminates the Company's net deferred tax assets.

On December 22, 2017, the President of the United States signed into law an Act to provide for reconciliation pursuant to Titles II and V of the concurrent resolution on the budget for fiscal year 2018 (commonly known as the ‘‘Tax Cuts and Jobs Act’’), which introduced a comprehensive set of tax reforms. The Tax Cuts and Jobs Act significantly revises U.S. tax law by, among other provisions, lowering the U.S. federal statutory income tax rate from 35% to 21% and eliminating or reducing certain income tax deductions.

The effects of changes in tax laws are required to be recognized in the period in which the legislation is enacted. However, due to the complexity and significance of the Tax Cuts and Jobs Act’s provisions, the SEC staff issued Staff Accounting Bulletin No. 118 ("SAB 118"), which allows companies to record the tax effects of the Tax Cuts and Jobs Act on a provisional basis based on a reasonable estimate, and then, if necessary, subsequently adjust such amounts during a limited measurement period as more information becomes available. The measurement period ends when a company has obtained, prepared, and analyzed the information necessary to finalize its accounting, but cannot extend beyond one year from enactment.

The Tax Cuts and Jobs Act did not have a material impact on our financial statements since our deferred temporary differences are fully offset by a valuation allowance and the Company does not have any offshore earnings from which to record the mandatory transition tax. However, given the significant complexity of the Tax Cuts and Jobs Act, anticipated guidance from the U.S. Treasury about implementing the Tax Cuts and Jobs Act, and the potential for additional guidance from the SEC or the FASB related to the Tax Cuts and Jobs Act, these estimates may be adjusted during the measurement period. The Company's provisional amounts for income taxes were based on the Company’s present interpretations of the Tax Cuts and Jobs Act and current available information, including assumptions and expectations about future events, such as its projected financial performance, and are subject to further refinement as additional information becomes available (including potential new or interpretative guidance issued by the FASB or the Internal Revenue Service and other tax agencies) and further analyses are completed. The Company continues to analyze the changes in certain income tax deductions and gather additional data to compute the full impacts on the Company’s deferred and current tax assets and liabilities.


16



Note 11—Commitments and Contingencies
 
As of June 30, 2018, the Company had entered into commitments under a license agreement with Oxford BioMedica (UK), a development, marketing, and supply agreement with Arena Pharmaceuticals GmbH ("Arena"), a Loan Agreement with Hercules, an amended services agreement with RSI, a separate service agreement with RSG (Refer to Note 6(A)) and a license agreement with Qaam Pharmaceuticals LLC. In addition, the Company has entered into services agreements with third parties for pharmaceutical manufacturing and research activities. Expenditures to contract research organizations and contract manufacturing organizations represent significant costs in clinical development. Subject to required notice periods and the Company's obligations under binding purchase orders, the Company can elect to discontinue the work under these agreements at any time. The Company expects to enter into other commitments as the business further develops.

During the three months ended June 30, 2018 , there were no material changes outside the ordinary course of business to the specified contractual obligations set forth in the contractual obligations table included in the Annual Report.

Note 12—Subsequent Events

Benitec Biopharma License and Collaboration Agreement

On July 8, 2018, ASG entered into a license and collaboration agreement (the "Benitec Agreement") with Benitec Biopharma Limited ("Benitec"). Pursuant to the Benitec Agreement, the Company received a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Benitec to develop and commercialize investigational gene therapy AXO-AAV-OPMD and related gene therapy products (collectively, now the "AXO-AAV-OPMD Program") for all diseases and conditions.

Under the Benitec Agreement, the Company will also collaborate with Benitec on five additional research plans as part of the "Collaboration Programs" for other genetic neurological disorders using Benitec technologies. The Company will receive a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Benitec to develop and commercialize products arising from each Collaboration Program.

Under the terms of the Benitec Agreement, the Company made an upfront nonrefundable payment of $10.0 million . In addition, the Company will be obligated to make payments to Benitec totaling up to (i) for the AXO-AAV-OPMD Program, $67.5 million upon the achievement of specified development and regulatory milestones and $120.0 million upon the achievement of specified sales milestones, and (ii) for each Collaboration Program, $33.5 million upon the achievement of specified development and regulatory milestones and $60.0 million upon the achievement of specified sales milestones.

Benitec will receive 30% of net profits of world-wide sales of products from the AXO-AAV-OPMD Program, subject to an agreed minimum amount for such payments. This profit-sharing payment will be made for so long as the Company or its affiliates or sublicensees commercialize such products. The Company will also pay Benitec a tiered royalty based on yearly aggregate net sales of products arising from each Collaboration Program, subject to specified reductions upon the occurrence of certain events as set forth in the Benitec Agreement. These royalties are required to be paid, on a product-by-product and country-by-country basis, until the latest to occur of the expiration of the last to expire valid claim of a licensed patent covering such product in such country, the expiration of regulatory exclusivity for such product in such country, or ten years after the first commercial sale of such product in such country.

Under the Benitec Agreement, Benitec will perform certain development and manufacturing activities for the AXO-AAV-OPMD Program and research activities for each Collaboration Program, and the Company will reimburse Benitec for its costs incurred, in accordance with an agreed-upon research and development plan and budget. The Company is solely responsible, at its expense, for all other activities related to the research, development and commercialization of products from the AXO-AAV-OPMD Program and the Collaboration Programs.

Closing of RSL Private Placement Financing

On July 9, 2018, the Company received $25.0 million of net proceeds from RSL in exchange for the issuance and sale of 14,285,714 of the Company's common shares to RSL at a purchase price of $1.75 per common share, which was the closing price per share of the Company's common shares on the Nasdaq Global Select Market on June 5, 2018, the date of the share purchase agreement.

17



Item 2.                                                          Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following discussion and analysis of our financial condition, results of operations and cash flows should be read in conjunction with (1) the unaudited interim condensed consolidated financial statements and the related notes thereto included elsewhere in this Quarterly Report on Form 10-Q, and (2) the audited consolidated financial statements and notes thereto and management’s discussion and analysis of financial condition and results of operations for the fiscal year ended March 31, 2018 included in our Annual Report on Form 10-K, filed with the Securities and Exchange Commission (the "SEC") on June 11, 2018. Unless the context requires otherwise, references in this report to "Axovant", the "Company," "we," "us," and "our" refer to Axovant Sciences Ltd. and its subsidiaries.
This Quarterly Report on Form 10-Q contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended (the "Securities Act"), and Section 21E of the Securities Exchange Act of 1934, as amended (the "Exchange Act").  These statements are often identified by the use of words such as "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "project," "will," "would" or the negative or plural of these words or similar expressions or variations, although not all forward-looking statements contain these identifying words.  We cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. The forward-looking statements appearing in a number of places in this Quarterly Report on Form 10-Q include, but are not limited to, statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things:

the success and timing of our ongoing development and commercialization of AXO-Lenti-PD, AXO-AAV-OPMD, nelotanserin and RVT-104;
our relationship under our license agreement with Oxford BioMedica (UK) Ltd. and Benitec Biopharma Limited;
the success of our interactions with international regulatory authorities;
the anticipated start dates, durations and completion dates of our ongoing and future nonclinical studies and clinical trials;
the anticipated designs of our future clinical studies;
anticipated future regulatory submissions and the timing of, and our ability to, obtain and maintain regulatory approval for our product candidates;
the rate and degree of market acceptance and clinical utility of any approved product candidate;
our ability to identify and in-license or acquire additional product candidates;
our commercialization, marketing and manufacturing capabilities and strategy;
continued service of our key scientific or management personnel;
our ability to obtain, maintain and enforce intellectual property rights for our product candidates;
our anticipated future cash position;
our estimates regarding our results of operations, financial condition, liquidity, capital requirements, prospects, growth and strategies;
the success of competing drugs that are or may become available;
our stated objective of becoming the leading biopharmaceutical company focused on neurology and psychiatry.

We have based these forward-looking statements largely on our current expectations and projections about future events, including the responses we expect from the U.S. Food and Drug Administration (the "FDA") and other regulatory authorities and financial trends that we believe may affect our financial condition, results of operations, business strategy, nonclinical studies and clinical trials and financial needs. Such forward-looking statements are subject to a number of risks, uncertainties, assumptions and other factors known and unknown that could cause actual results and the timing of certain events to differ materially from future results expressed or implied by the forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those identified herein, and those discussed in the section titled "Risk Factors" set forth in Part II, Item 1A of this Quarterly Report on Form 10-Q and in our other filings with the SEC. These risks are not exhaustive. You should not rely upon forward-looking statements as predictions of future events. Furthermore, such forward-looking statements speak only as of the date of this report. New risk factors emerge from time to time and it is not possible for our management to predict all risk factors, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Except as required by law, we undertake no obligation to update any forward-looking statements to reflect events or circumstances after the date of such statements.

Overview

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We are a clinical-stage biopharmaceutical company focused on the acquisition, development and commercialization of novel therapeutics in the fields of neurology and psychiatry. We are developing a pipeline of clinical and nonclinical product candidates that focuses on the various aspects of debilitating neurological diseases such as Parkinson's disease, oculopharyngeal muscular dystrophy, Lewy body dementia ("LBD"), and other indications in the fields of neurology and psychiatry. Our goal is to be the leading biopharmaceutical company focused on the fields of neurology and psychiatry.
Our near-term focus is to develop our gene therapy product candidates AXO-Lenti-PD, a potential one-time treatment for Parkinson's disease, and AXO-AAV-OPMD, a potential one-time treatment for oculopharyngeal muscular dystrophy ("OPMD"). We intend to begin a Phase 1/2 study of AXO-Lenti-PD in advanced Parkinson's disease patients before the end of 2018 and a placebo-controlled study of AXO-AAV-OPMD in OPMD patients in 2019. Prior to the recent in-licensing of AXO-Lenti-PD in June 2018 and AXO-AAV-OPMD in July 2018, our primary focus had been on developing nelotanserin, a selective inverse agonist of the 5-HT 2A receptor, and intepirdine, an antagonist of the 5-HT6 receptor. In January 2018, we announced the results of a pilot Phase 2 study of nelotanserin in patients with LBD that experience visual hallucinations. We plan to make a determination of the overall development strategy for nelotanserin once we have reviewed topline data in the second half of 2018 from our currently ongoing Phase 2 study of nelotanserin in REM Sleep Behavior Disorder ("RBD") in LBD patients, and have completed our ongoing comprehensive clinical, regulatory and commercial review. In addition, we will determine our development plans for RVT-104, a combination of rivastigmine and a peripheral muscarinic receptor antagonist, as a potential treatment for patients with Alzheimer's disease or dementia with Lewy bodies ("DLB"), which is a sub-type of LBD, once we have completed our ongoing comprehensive clinical, regulatory and commercial review in the context of our recent acquisition of AXO-Lenti-PD, AXO-AAV-OPMD and any other newly acquired product candidates.
In January 2018, we announced the discontinuation of our development of intepirdine following our announcement that neither the Phase 2b HEADWAY clinical trial of intepirdine in patients with DLB nor the pilot Phase 2 Gait and Balance clinical trial of intepirdine in patients with dementia and gait impairment met their respective primary endpoints, and the September 2017 announcement that our Phase 3 MINDSET clinical trial of intepirdine in patients with mild-to-moderate Alzheimer's disease did not meet its co-primary efficacy endpoints. Following the announcement of Phase 3 MINDSET clinical trial results, we also discontinued further development of RVT-103 which had been intended for use in combination with intepirdine. We remain committed to identifying, developing and commercializing other novel treatments for unmet needs in neurology and psychiatry. We are continuing to actively explore opportunities to acquire or in-license additional products, product candidates and technologies to further build our pipeline.
We were founded in October 2014 and our operations to date have been limited to organizing and staffing our company, raising capital, acquiring our product candidates and advancing our product candidates into clinical development. To date, we have not generated any revenue and we have financed our operations primarily through the public and private offerings of our equity securities. As of June 30, 2018, we had $92.9 million of cash. In July 2018, we received $25.0 million of net proceeds from the issuance and sale of our common shares in a private placement to RSL. We recorded net losses of $51.9 million and $69.3 million for the three months ended June 30, 2018 and 2017, respectively, and $221.6 million for the year ended March 31, 2018. We have determined that we have one operating and reporting segment.

Our Product Pipeline

The following table summarizes the status of our development programs to which Axovant Sciences GmbH, our wholly owned subsidiary, holds global commercial rights:


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Compound
Clinical Indication
Development Stage
 
 
 
Gene Therapy Programs
 
 
AXO-Lenti-PD
Parkinson's disease
Phase 1/2
 
 
 
AXO-AAV-OPMD
Oculopharyngeal muscular dystrophy
Preclinical
 
 
 
AXO-AAV-ALS
Amyotrophic lateral sclerosis
Research
 
 
 
AXO-AAV-FTD
Frontotemporal dementia
Research
 
 
 
Four additional AXO-AAV
Undisclosed
Research
     Collaboration Programs
 
 
 
 
 
Small Molecule Programs
 
 
Nelotanserin
Visual hallucinations in LBD
Phase 2 Pilot Study Completed
 
REM sleep behavior disorder in LBD
Phase 2 Ongoing
 
 
 
RVT-104
Alzheimer's disease and DLB
Proof of Concept
 
 
 

Gene Therapy Programs
AXO-Lenti-PD
Overview
AXO-Lenti-PD (previously known as OXB-102) is an in vivo lentiviral gene therapy investigational product candidate currently being developed for the one-time treatment of Parkinson’s disease. We licensed the worldwide development and commercialization rights to AXO-Lenti-PD and its predecessor product candidate ProSavin from Oxford BioMedica (UK) Ltd. ("Oxford BioMedica"), under an exclusive license agreement (the "Oxford BioMedica Agreement") entered into in June 2018.
AXO-Lenti-PD delivers a construct of three genes that encode the critical enzymes required for the biochemical synthesis of endogenous dopamine from tyrosine. The three enzymes are: Tyrosine Hydroxylase (or TH, the enzyme that converts tyrosine to L-dopa), Cyclohydrolase 1 (or CH1, the rate-limiting enzyme for synthesis of Tetrahydrobiopterin, or BH4, a critical cofactor for production of L-dopa), and Aromatic L-Amino Acid Decarboxylase (or AADC, the enzyme that converts L-dopa to dopamine). AXO-Lenti-PD is delivered by a one-time MRI-guided stereotactic infusion into the putamen. We believe that delivery of all three of these genes will enable the continuous, tonic, endogenous synthesis of dopamine in non-dopaminergic cells. Dopamine deficiency plays a central role in Parkinson's disease and we believe that restoring dopamine synthesis capability in patients will offer lasting improvement in the symptoms of Parkinson's disease. Oxford BioMedica previously conducted a Phase 1/2 clinical study with ProSavin (also known as OXB-101), an earlier version of this product candidate. In this clinical trial, ProSavin was observed to have a favorable long-term safety profile and demonstrated effects on motor function, supporting proof-of-concept. AXO-Lenti-PD delivers an optimized transgene construct relative to ProSavin.
Parkinson's Disease Overview
Parkinson's disease is a chronic neurodegenerative disorder that primarily results in progressive and debilitating motor symptoms. It is estimated that up to one million people in the United States and 7 million to 10 million people worldwide suffer from Parkinson's disease. It typically develops between the ages of 55 and 65 years and affects approximately 1% of people over the age of 60 years. The underlying factors that result in the development of Parkinson's disease are largely unknown. However, Parkinson's disease is a neurodegenerative disease that results in reduced levels of the neurotransmitter dopamine in the striatum, a region in the brain. Dopamine is essential for movement, and low levels of dopamine in patients with Parkinson's disease are believed to result in the typical motor symptoms of the disease, including hypo- and bradykinesia, rigidity, tremor, and postural instability.

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The treatment of Parkinson's disease is currently limited to symptomatic treatments, as no therapies have proven effective in altering the course of the disease or addressing the underlying pathophysiological processes. The mainstay of treatment typically involves the daily administration of oral L-dopa, the precursor to dopamine. While L-dopa is effective in controlling motor symptoms early in the disease, progressive loss of dopaminergic neurons and chronic L-dopa therapy are believed to contribute to the " wearing off" of L-dopa's efficacy in the more advanced stages of the disease. Patients become increasingly less responsive to oral L-dopa therapy and require higher doses to manage their symptoms. More advanced Parkinson's disease patients often begin to experience " on-off" motor fluctuations, characterized by unpredictable "OFF periods" of reduced mobility and increased rigidity and tremor. In addition, abnormal and involuntary movements known as dyskinesias may occur at higher L-dopa blood levels. Approximately 10% of patients per year develop " on-off " motor fluctuations after starting L-dopa therapy.
As Parkinson's disease progresses, other therapies can be given in combination with L-dopa and include dopamine receptor agonists and inhibitors of enzymes related to dopamine metabolism, such as monoamine oxidase B (MAO-B) and catechol O-methyl transferase (COMT). These therapies aim to further improve overall dopaminergic function. Patient-friendly treatment options for motor fluctuations in advanced Parkinson's disease are limited. Subcutaneous injections of the dopamine agonist apomorphine are used for the acute treatment of OFF episodes. Duopa/Duodopa is an enteral suspension of L-dopa and the peripheral AADC inhibitor carbidopa that is continuously administered over the course of the day through a surgically-placed percutaneous endoscopic gastrostomy with jejunal ("PEG-J") tube to reduce fluctuations in L-dopa blood levels. Deep-Brain Stimulation ("DBS"), a procedure in which electrodes are surgically placed in the basal ganglia, either in the subthalamic nucleus or internal globus pallidus, is another option in advanced Parkinson's disease. Through an impulse generator, electrical stimuli are delivered to the brain to modulate neural signals within these target regions. It remains unclear exactly how DBS improves the symptoms of Parkinson's disease. Both Duopa/Duodopa and DBS require indwelling hardware -- a PEG-J tube, or electrodes, leads and impulse generator -- respectively.
Predecessor Product Candidate: ProSavin (OXB-101)
ProSavin, the predecessor therapy candidate to AXO-Lenti-PD, delivered the same three genes (AADC, TH, and CH1) as AXO-Lenti-PD in the same lentiviral vector, but in a different payload configuration. AXO-Lenti-PD was the result of multifactorial experimentation to modify the payload configuration to improve endogenous dopamine production. The initial Phase 1/2 clinical trial of ProSavin was completed in 2012 and long-term follow-up is ongoing.
Nonclinical Studies for ProSavin
Nonclinical studies in non-human primate models of Parkinson's disease demonstrated that ProSavin can safely restore striatal dopamine production to approximately 50% and correct motor deficits without associated dyskinesias (p-value < 0.05). ProSavin was observed to improve Parkinson's disease symptoms and clinical disease severity in the same non-human primate model, with a durable response seen up to 12 months (p-value < 0.05 at all time points beyond week 4). One of the ProSavin treated non-human primates was continued on the study and exhibited a sustained motor improvement until the study was concluded at 44 months. Nonclinical study data did not reveal adverse reactions nor findings with potential impact on patient safety and provided pertinent data on the optimal method of delivery in the clinic. ProSavin was also observed to be well tolerated when co-administered with L-dopa and apomorphine, indicating that it can be used in conjunction with these commonly used Parkinson's disease medications.
In summary, these experiments were determined to demonstrate the long-term safety of therapeutic doses of ProSavin as well as significant efficacy to improve measures of movement and reduce dyskinesias in animal models. These results supported the initiation of clinical trials for ProSavin.

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Phase 1/2 Clinical Trial of ProSavin
ProSavin was evaluated for safety and efficacy in a Phase 1/2 study in patients with advanced Parkinson's disease by Oxford BioMedica. In this study, ProSavin was observed to be safe and well-tolerated with sustained improvements on motor function as measured by the Unified Parkinson's Disease Rating Scale ("UPDRS") Part III (motor) score in the state "OFF" levodopa medication, which we refer to as UPDRS Part III "OFF." The Phase 1/2 clinical trial was conducted at sites in the United Kingdom and France on a total of 15 patients with advanced Parkinson's disease. Three dose levels of ProSavin were assessed in four patient cohorts: dose level one (1.9 × 10 7 transducing units ("TU"); cohort 1); dose level two (4.0 × 10 7 TU; cohorts 2a and 2b); and dose level three (1 × 10 8 TU; cohort 3). Cohorts 2b and 3 underwent a modified delivery method to increase the rate of delivery of the viral vector. The primary endpoints were the number and severity of adverse events as well as the UPDRS Part III "OFF" scores at 6 months after gene therapy administration. No serious adverse events related to ProSavin or the surgical procedure were reported. Reported adverse events ("AEs") were generally mild and related to either Parkinson's disease progression or L-dopa-induced dyskinesias that were ameliorated with reduction of L-dopa administration. The most common AEs in the first 12 months were dyskinesia (n=11 subjects), " on-off " motor fluctuations (n=9), headache (n=4), and akinesia (n=3).
Across all patients, mean UPDRS Part III "OFF" scores were significantly improved at six months (33% reduction, p-value=0.0001) and 12 months (31% reduction, p-value=0.0001). Sustained improvement was seen through four years of follow-up and the long-term follow-up study is still ongoing (10 years exposure in the earliest subject). Clinical data from this study were published in The Lancet in 2014.
Second-Generation Product Candidate: AXO-Lenti-PD
AXO-Lenti-PD is a re-engineered gene therapy product candidate that was selected following multifactorial experimentation to modify the payload configuration of ProSavin to further improve dopamine production. The modifications included a different ordering of the genes, the fusion of TH and CH1 with a flexible linker, and the removal of a genetic control element between TH and AADC. We believe these changes lead to more balanced stoichiometry of gene expression and colocalization of enzymatic activity. The targeted net result is improved dopamine production in transfected cells.
Nonclinical studies for AXO-Lenti-PD
In vitro experiments with AXO-Lenti-PD demonstrated up to 10-fold increases in dopamine + L-dopa production over ProSavin. Functionally, in non-human primate models, AXO-Lenti-PD demonstrated a similar level of improvement in spontaneous locomotor activity compared to ProSavin at approximately 1/5 th  of the dose. We believe these data demonstrate that AXO-Lenti-PD has greater potency compared to ProSavin in terms of dopamine production, enzymatic activity and functional improvement in animal models of Parkinson's disease.
Planned Phase 1/2 Clinical Study of AXO-Lenti-PD
We plan to initiate a Phase 1/2 clinical study of AXO-Lenti-PD in patients with advanced Parkinson's disease before the end of 2018 with clinical data expected to be available in 2019. The planned study design consists of two parts:

Part A is a non-randomized dose-escalation of multiple potential dose levels.

Part B is a double-blind design with patients randomized either to an active group receiving the optimal dose as determined in Part A, or a control group receiving an imitation "sham" surgical procedure.
The study will evaluate the safety and tolerability of AXO-Lenti-PD as well as assess efficacy using clinical measures of motor function, such as UPDRS Part III, and biomarkers. Sufficient gene therapy product is currently available to initiate the planned Phase 1/2 clinical study.

AXO-AAV Programs
Silence-and-Replace Technology
The Silence-and-Replace technology platform is designed to produce a long-term restoration of normal gene function and is achieved by combining RNA interference (silence) with gene therapy (replace) in a single administration of a single viral vector construct. This approach may be applicable to various genetic diseases, particularly autosomal dominant genetic disorders caused by nucleotide repeat expansion.

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Multiple neurological and muscular diseases are associated with erroneous expression of a mutated gene. RNA interference ("RNAi") has shown potential to silence the expression of disease-associated genes. Commonly-used RNAi approaches, in which small interfering RNA ("siRNA") is introduced directly into the cell, achieve only transient gene silencing and are limited by the requirement for repeated administration and variable concentrations of siRNA over time. To provide lasting gene silencing, the Silence-and-Replace technology employs ddRNAi, in which viral vectors deliver a DNA construct that produces short hairpin RNAs ("shRNAs"), which are processed by the cell into siRNAs, which then silence the mutated genes.
In an autosomal dominant genetic disorder, particularly one caused by nucleotide repeat expansion, silencing of the mutant gene can also lead to silencing of the wild type gene, which may be required for normal function. The Silence-and-Replace strategy is designed to address this potential issue by delivering a functional copy of the gene that is re-engineered to be resistant to knockdown. The gene that encodes the functional protein may be contained within the same viral vector as the ddRNAi construct.

AXO-AAV-OPMD Program
Overview
The AXO-AAV-OPMD Program is an investigational gene therapy being developed as a one-time treatment for OPMD, which we licensed from Benitec Biopharma Limited ("Benitec") in July 2018. The Program utilizes an AAV vector to deliver a Silence-and-Replace construct to silence the mutant poly-A binding protein N1 (" PABPN1" ) gene that causes OPMD and replace it with a functional copy of the PABPN1 gene. This Silence-and-Replace approach aims to knock down the expression of both the wild-type and mutant PABPN1 gene through ddRNAi, while at the same time expressing a re-engineered copy of the PABPN1 gene coding for the functional PABPN1 protein which is resistant to knockdown. The gene therapy will be delivered in a single administration directly into target muscle tissue to provide long-term correction of muscle pathology and restoration of function. 

Oculopharyngeal Muscular Dystrophy Overview
 
OPMD is a muscular disease that is inherited through a primarily autosomal dominant pattern. OPMD is estimated to affect approximately 15,000 people in North America and Europe. The disease generally presents in patients between the ages of 40 and 70 years old and is characterized primarily by progressive difficulty swallowing, eyelid drooping, and weakness of the proximal extremities. Swallowing difficulties can have life-threating consequences, including malnutrition and aspiration pneumonia. As the disease progresses, the swallowing difficulties become more severe and other muscles may become involved. There are no products approved for the treatment of OPMD and therefore, treatment options available to patients are limited. OPMD is caused by mutations in the gene coding for PABPN1, a ubiquitously expressed protein that regulates the processing of messenger RNAs. The normal PABPN1 protein contains ten copies of the amino acid alanine, which forms a polyalanine tract. In OPMD, the mutated PABPN1 gene has an expansion of alanine-encoding trinucleotide repeats, resulting in an abnormally long polyalanine tract. The protein that forms from the mutated gene is prone to aggregating into insoluble nuclear inclusion bodies which leads to muscle cell pathology and disease progression.

Nonclinical studies for AXO-AAV-OPMD

Data from mouse models of OPMD showed gene therapy from the AXO-AAV-OPMD Program provided up to 86% inhibition of PABPN1 gene expression, while restoring functional PABPN1 transgene expression up to 63% of normal levels. The A17 mouse model is a well-validated in vivo model that is designed to exhibit many of the key pathological features of OPMD patients. The levels of gene silencing and expression achieved in this model coincided with decreased muscle pathology and a restoration of muscle force and muscle weight to near wild-type levels.

Planned Clinical Study for AXO-AAV-OPMD Program

We expect to initiate a placebo-controlled clinical study for the investigational AXO-AAV-OPMD Program in 2019. The FDA and European Commission have granted Orphan Drug Designation to the AXO-AAV-OPMD Program for the treatment of OPMD.


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Additional Collaboration Programs
Under our license and collaboration agreement with Benitec (the "Benitec Agreement"), we will pursue five additional investigational gene therapy research plans as part of collaboration programs focused on genetic neurological disorders utilizing Benitec’s technologies. We plan to initiate a research plan to develop gene therapy products targeting the C9orf72 gene, which is associated with ALS and frontotemporal dementia ("FTD"). In addition, we plan to initiate four other research plans focused on undisclosed genetic neurological disorders.
 
ALS and FTD are neurological disorders that have been linked to hexanucleotide repeats in the C9orf72 gene.  Thirty to forty percent of familial ALS cases are associated with C9orf72 gene mutations and these patients have a progressive muscle weakness resulting from the death of motor neurons in the spinal cord and brain.  Patients with FTD associated with C9orf72 gene mutations have a progressive brain disorder that affects personality, behavior, language and movement.  While the exact role of C9orf72 gene mutation is unknown, both expression of the mutated C9orf72 gene and lack of functional C9orf72 gene are believed to be implicated. We believe Silence-and-Replace gene therapy is a promising approach for the restoration of normal C9orf72 gene function and has the potential to deliver lasting benefits for ALS and FTD patients.

Small Molecule Programs
Nelotanserin
Overview
In October 2015, we acquired from our majority shareholder, Roivant Sciences Ltd. ("RSL"), the global rights to nelotanserin, a selective inverse agonist of the 5-HT 2A receptor. To date, we have been investigating and developing nelotanserin to address visual hallucinations and RBD in patients with LBD. In January 2018, we reported results for a pilot Phase 2 Visual Hallucination study of nelotanserin in patients with LBD. Nelotanserin was generally well tolerated but did not show any statistical trends of improvement on prespecified analyses of various scales to assess visual hallucinations. However, we did observe positive trends on UPDRS Part III in the primary efficacy population (3.12 point improvement, p=0.075 unadjusted) which will also be assessed in the ongoing RBD study. We expect data from the Phase 2 RBD study in the second half of calendar 2018. We plan to make a determination of the overall development strategy for nelotanserin once we have reviewed the topline results from this study, and completed our ongoing comprehensive clinical, regulatory and commercial review.

Nelotanserin for REM Sleep Behavior Disorder in Lewy Body Dementia
Medical Need
RBD is a common clinical feature of LBD, and is a condition where patients lose normal sleep paralysis resulting in the physical acting out of their dreams, impacting their quality of life and endangering themselves and their bed partners. While off-label treatment of RBD with benzodiazepines is common, this class of drugs is associated with severe side effects in patients with dementia, including sedation, worsening of cognition and increased risk of falls. We believe that there is a need for new therapeutic options that can reduce the frequency of RBD without sedating patients or worsening cognition in patients with dementia.

Clinical Development
In March 2016, we initiated a four-week, double-blind, randomized, placebo-controlled Phase 2 study in patients with DLB and Parkinson's disease dementia suffering from RBD. This study will utilize objective measures of efficacy as assessed in a sleep-lab setting. Due to challenges with recruitment for this study, we elected to close enrollment prior to reaching our enrollment target. Because of this smaller than planned enrollment, the study may not qualify as pivotal. We expect to receive top-line results for this study in the second half of calendar year 2018. Patients completing the double-blind portion of this study were eligible to enroll in an open label extension study of nelotanserin.

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RVT-104
In August 2016, we and Qaam Pharmaceuticals LLC entered into an exclusive license agreement under which we in-licensed the rights to develop and commercialize RVT-104, a product candidate that combines rivastigmine, a cholinesterase inhibitor, with a peripherally acting quaternary amine muscarinic receptor antagonist, as a potential treatment for patients with Alzheimer's disease or DLB. Cholinesterase inhibitors are the standard of care in both Alzheimer's disease and DLB. Despite their widespread use, many patients cannot tolerate the cholinesterase inhibitors because of their cholinergic side effects such as nausea, vomiting and diarrhea. We believe that drugs that can mitigate these cholinergic side effects will allow more patients to receive optimal cholinesterase inhibitor therapy as well as potentially allow for dosing with higher than currently approved doses. We will make a decision about development plans for this program after an internal portfolio review in the context of AXO-Lenti-PD, AXO-AAV-OPMD and any other newly acquired product candidates.
Our Key Agreements
Oxford BioMedica License Agreement
On June 5, 2018, we, through our wholly owned subsidiary, ASG, entered into the Oxford BioMedica Agreement, pursuant to which we received a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Oxford BioMedica to develop and commercialize AXO-Lenti-PD and related gene therapy products for all diseases and conditions. In June 2018, as partial consideration for the license, we made an upfront payment to Oxford BioMedica of $30.0 million, $5.0 million of which will be applied as a credit against the process development work and clinical supply that Oxford BioMedica will provide to us. Under the terms of the Oxford BioMedica Agreement, we could be obligated to make payments to Oxford BioMedica totaling up to $55.0 million upon the achievement of specified development milestones and $757.5 million upon the achievement of specified regulatory and sales milestones. We will also be obligated to pay Oxford BioMedica a tiered royalty from 7% to 10%, based on yearly aggregate net sales of the Gene Therapy Products, subject to specified reductions upon the occurrence of certain events as set forth in the Oxford BioMedica Agreement. These royalties are required to be paid, on a product-by-product and country-by-country basis, until the latest to occur of the expiration of the last to expire valid claim of a licensed patent covering such product in such country, the expiration of regulatory exclusivity for such product in such country, or 10 years after the first commercial sale of such product in such country.
We are solely responsible, at our expense, for all activities related to the development and commercialization of the Gene Therapy Products. Pursuant to the Oxford BioMedica Agreement, we are required to use commercially reasonable efforts to develop, obtain regulatory approval of, and commercialize a Gene Therapy Product in the United States and at least one major market country in Europe. In addition, we are required to meet certain diligence milestones and to include at least one U.S.-based clinical trial site in a pivotal study of a Gene Therapy Product. If we fail to meet any of these specified development milestones, we may cure such failure by paying Oxford BioMedica certain fees, which range from $0.5 million to $1.0 million.
Benitec Biopharma License and Collaboration Agreement
On July 8, 2018, we, through our wholly owned subsidiary, ASG, entered into the Benitec Agreement, pursuant to which we received a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Benitec to develop and commercialize investigational gene therapy AXO-AAV-OPMD and related gene therapy products (collectively, the "AXO-AAV-OPMD Program") for all diseases and conditions.
Under the Benitec Agreement, we will also collaborate with Benitec on five additional research plans ("Collaboration Programs") for other genetic neurological disorders using Benitec technologies. We will receive a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Benitec to develop and commercialize products arising from each Collaboration Program.

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Under the terms of the Benitec Agreement, we made an upfront payment of $10.0 million. In addition, we will be obligated to make payments to Benitec totaling up to (i) for the AXO-AAV-OPMD Program, $67.5 million upon the achievement of specified development and regulatory milestones and $120.0 million upon the achievement of specified sales milestones, and (ii) for each Collaboration Program, $33.5 million upon the achievement of specified development and regulatory milestones and $60.0 million upon the achievement of specified sales milestones. Benitec will receive 30% of net profits of our world-wide sales of products from the AXO-AAV-OPMD Program, subject to an agreed minimum amount for such payments. This profit-sharing payment will be made for so long as we or our affiliates or sublicensees commercialize such products. We will also pay Benitec a tiered royalty based on yearly aggregate net sales of products arising from each Collaboration Program, subject to specified reductions upon the occurrence of certain events as set forth in the Benitec Agreement. These royalties are required to be paid, on a product-by-product and country-by-country basis, until the latest to occur of the expiration of the last to expire valid claim of a licensed patent covering such product in such country, the expiration of regulatory exclusivity for such product in such country, or ten years after the first commercial sale of such product in such country.
Under the Benitec Agreement, Benitec will perform certain research activities for each Collaboration Program and development and manufacturing activities for the AXO-AAV-OPMD Program, and we will reimburse Benitec for its costs incurred, in accordance with an agreed-upon research and development plan and budget. We are solely responsible, at our expense, for all other activities related to the research, development and commercialization of products from the Collaboration Programs and the AXO-AAV-OPMD Program.
Arena Development Agreement for Nelotanserin
In October 2015, we exercised an option to acquire global rights, title, interest and obligations in and to nelotanserin from our parent company, RSL. In May 2015, RSL entered into a development, marketing and supply agreement for nelotanserin (the "Arena Development Agreement") with Arena Pharmaceuticals GmbH ("Arena"), and we entered into a Waiver and Option Agreement with RSL. Upon the exercise of our option, we assumed RSL’s rights and obligations under the Arena Development Agreement, as amended on October 18, 2017. In January 2018, we were notified by Arena that it has assigned all of its rights and obligations under the Arena Development Agreement to an affiliate, 125 Royalty Inc. Under the Waiver and Option Agreement, we recorded $5.3 million as research and development expense which was 110% of the payments made to Arena by RSL, and the costs incurred by RSL in connection with the development of nelotanserin. We will be responsible for future contingent payments under the Arena Development Agreement, including up to $4.0 million in potential development milestone payments, up to $37.5 million in potential regulatory milestone payments and up to $60.0 million in potential commercial milestone payments. Under the Arena Development Agreement, we are also obligated to purchase all commercial supplies of nelotanserin from Arena at a fixed price equal to 15% of net sales of nelotanserin.
The Arena Development Agreement will remain in effect until terminated: (1) by the parties’ mutual agreement; (2) for any reason by us upon 90 days’ written notice to Arena; (3) by either party upon written notice for the other party’s material breach or insolvency event if such party fails to cure such breach or the insolvency event is not dismissed within the specified cure period; or (4) by Arena if we or our affiliates participate in a challenge to certain Arena patents.
Services Agreements with Roivant Sciences, Inc. and Roivant Sciences GmbH
In October 2014, we and our wholly owned subsidiary, Axovant Sciences, Inc. ("ASI") entered into a services agreement with Roivant Sciences, Inc. ("RSI"), a wholly owned subsidiary of RSL, pursuant to which RSI provides us with services in relation to the identification of potential product candidates and project management of clinical trials, as well as other services related to our development, administrative and financial functions. In February 2017, in connection with the contribution and assignment of all of our intellectual property rights to ASG, we amended and restated this services agreement effective as of December 13, 2016, as a result of which ASG was added as a recipient of services from RSI. In addition, ASG also entered into a separate services agreement with Roivant Sciences GmbH ("RSG"), a wholly owned subsidiary of RSL, effective as of December 13, 2016, for the provision of services by RSG to ASG in relation to the identification of potential product candidates and project management of clinical trials, as well as other services related to development, administrative and financial activities. Under the terms of both services agreements, we are obligated to pay or reimburse RSI and RSG for the costs they, or third parties acting on their behalf, incur in providing services to us or ASG, including administrative and support services as well as research and development services. In addition, we are obligated to pay RSI and RSG for their services at a pre-determined mark-up on the costs incurred directly by RSI and RSG in connection with any general and administrative and research and development services provided directly by RSI and RSG.
Under the services agreement in effect as of December 31, 2016, we incurred expenses of $3.4 million and $2.7 million for the three months ended June 30, 2018 and 2017 , respectively, inclusive of the mark-up. We have recorded these charges as research and development expense and general and administrative expense in our condensed consolidated statements of operations.

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Venture Debt Financing from Hercules Capital, Inc.
On February 2, 2017, we and our wholly owned subsidiaries, Axovant Holdings Limited ("AHL"), ASG and ASI entered into a loan and security agreement, as amended on May 24, 2017 and September 22, 2017 (the ‘‘Loan Agreement’") with Hercules Capital, Inc. ("Hercules") under which we, AHL and ASG (collectively the "Borrowers") borrowed an aggregate of $55.0 million (the "Term Loan"). ASI issued a guaranty of the Borrowers’ obligations under the Loan Agreement. At the closing of the Term Loan, the Borrowers paid Hercules a facility charge of $550,000. Subsequently, we added our subsidiary ASA as a Borrower in July 2017 and our subsidiaries ATH and ATI as Borrowers in April 2018. The Term Loan bears interest at a variable per annum rate calculated for any day as the greater of either (i) the prime rate plus 6.80%, and (ii) 10.55%. The Term Loan has a scheduled maturity date of March 1, 2021. The Borrowers are obligated to make monthly payments of accrued interest under the Loan Agreement until September 1, 2018, followed by monthly installments of principal and interest through March 1, 2021. In connection with the Loan Agreement, the Borrowers and ASI, as guarantor, granted Hercules a first position lien on substantially all of their respective assets, excluding intellectual property. Prepayment of the Term Loan is subject to penalty.
The Loan Agreement includes customary affirmative and restrictive covenants and representations and warranties, including a minimum cash covenant, a covenant against the occurrence of a "change in control," financial reporting obligations, and certain limitations on indebtedness, liens (including a negative pledge on intellectual property and other assets), investments, distributions (including dividends), collateral, transfers, mergers or acquisitions, taxes, corporate changes, and deposit accounts. The Loan Agreement also includes customary events of default, including payment defaults, breaches of covenants following any applicable cure period, the occurrence of certain events that could reasonably be expected to have a "material adverse effect" as set forth in the Loan Agreement, cross acceleration to the debt and certain events relating to bankruptcy or insolvency. Upon the occurrence of an event of default, a default interest rate of an additional 5.0% may be applied to the outstanding principal balance, and Hercules may declare all outstanding obligations immediately due and payable and take such other actions as set forth in the Loan Agreement.
In addition, for so long as the Term Loan remains outstanding, we are required to use commercially reasonable efforts to afford Hercules the opportunity to participate in future underwritten equity offerings of our common shares up to a specified amount.
In connection with the entry into the Loan Agreement, we issued a warrant to Hercules which was exercisable for an aggregate of 274,086 of our common shares at an exercise price of $12.04 per share. In August 2017, Hercules exercised the warrant on a cashless basis and received a net issuance of 129,827 of our common shares.

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Financial Operations Overview
Revenue
We have not generated any revenue from the sale of any products, and we do not expect to generate any revenue unless and until we obtain regulatory approval of and begin to commercialize one of our product candidates in development.
Research and Development Expense
Since our inception, our operations have primarily been focused on organizing and staffing our company, raising capital, acquiring, and preparing for and advancing our product candidates, intepirdine, nelotanserin, RVT-103, RVT-104 and AXO-Lenti-PD, into clinical development. Our research and development expenses include program-specific costs, as well as unallocated internal costs.
Program-specific costs include:
direct third-party costs, which include expenses incurred under agreements with contract research organizations and contract manufacturing organizations, the cost of consultants who assist with the development of our product candidates on a program-specific basis, investigator grants, sponsored research, manufacturing costs in connection with producing materials for use in conducting nonclinical and clinical studies, and any other third-party expenses directly attributable to the development of our product candidates; and
upfront payments for the purchase of in-process research and development, which include costs incurred under the Oxford BioMedica Agreement and the Arena Development Agreement.
Unallocated internal costs include:
share-based compensation expense for research and development personnel, including expense related to RSL common share awards and RSL options issued by RSL to RSI and RSG employees;
personnel-related expenses, which include employee-related expenses, such as salaries, benefits and travel expenses, for research and development personnel;
costs allocated to us under our services agreements with RSI and RSG; and
other expenses, which includes the cost of consultants who assist with our research and development but are not allocated to a specific program.

Research and development activities will continue to be central to our business model. We expect to continue to incur research and development expense as we continue our development program for nelotanserin in LBD. However, due to the termination of the MINDSET, HEADWAY and Gait and Balance trials of intepirdine, we expect our overall research and development expense to decrease significantly until such time as we undertake additional development programs, including in relation to the AXO-Lenti-PD program, the AXO-AAV-OPMD program, the Collaboration Programs with Benitec and additional product candidates we may in-license or acquire as we pursue our updated business plan. We also expect our share-based compensation and other employee-related expenses for our research and development personnel to increase as a result of the transfer of certain activities from RSI and RSG in July 2018, offset by a reduction in costs allocated to us under our services agreements with RSI and RSG.

Product candidates in later stages of clinical development, such as nelotanserin, generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. The duration, costs and timing of clinical trials of our products in development and any other product candidates will depend on a variety of factors that include, but are not limited to, the following:
the number of trials required for approval;
the per patient trial costs;
the number of patients who participate in the trials;
the number of sites included in the trials;
the countries in which the trials are conducted;
the length of time required to enroll eligible patients;

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the number of doses that patients receive;
the drop-out or discontinuation rates of patients;
the potential additional safety monitoring or other studies requested by regulatory agencies;
the duration of patient follow-up;
the timing and receipt of regulatory approvals; and
the efficacy and safety profile of the product candidates.
In addition, the probability of success of our products in development and any other product candidate will depend on numerous factors, including competition, manufacturing capability and commercial viability. We may never succeed in achieving regulatory approval of our product candidates for any indication in any country. As a result of the uncertainties discussed above, we are unable to determine in advance the duration and completion costs of any clinical trial we conduct, or when and to what extent we will generate revenue from the commercialization and sale of our products in development or other product candidates, if at all.
General and Administrative Expense
General and administrative expenses consist primarily of share-based compensation, legal and accounting fees, consulting services, services received under the services agreements with RSI and RSG and employee-related expenses, such as salaries, benefits and travel expenses, for general and administrative personnel.
We anticipate that our general and administrative expenses will decrease, primarily as the result of a reduction in share-based compensation and other employee-related expenses for our general and administrative personnel due to the recent reduction in headcount, partially offset by the decision to build out internal administration and finance functions at Axovant and reduce our utilization of RSI services.



29



Results of Operations for the Three Months Ended June 30, 2018 and 2017
The following table summarizes our results of operations for the three months ended June 30, 2018 and 2017 (in thousands):

 

Three Months Ended June 30,
 
 
 
2018
 
2017
 
Change
Operating expenses:
 
Research and development expenses
 
 
 
 
 
(includes total share-based compensation expense of $2,517 and $6,256 for the three months ended June 30, 2018 and 2017, respectively)
$
37,418

 
$
43,712

 
$
(6,294
)
General and administrative expenses
 
 
 
 
 
(includes total share-based compensation expense of $3,342 and $9,344 for the three months ended June 30, 2018 and 2017, respectively)
11,754

 
21,518

 
(9,764
)
Total operating expenses
$
49,172

 
$
65,230

 
$
(16,058
)

Research and Development Expenses
For the three months ended June 30, 2018 and 2017, our research and development expenses consisted of the following (in thousands):
 

Three Months Ended June 30,
 
 
 
2018
 
2017
 
Change
Program-specific costs:
 
 
 
 
 
   AXO-Lenti-PD
$
25,132

 
$

 
$
25,132

   Intepirdine
1,297

 
26,807

 
(25,510
)
   Nelotanserin
3,354

 
3,214

 
140

   RVT-103
1

 
243

 
(242
)
   RVT-104
(76
)
 
349

 
(425
)
Unallocated internal costs:
 
 
 
 

   Share-based compensation
2,517

 
6,256

 
(3,739
)
   Personnel-related
1,536

 
3,841

 
(2,305
)
   Services agreements
2,245

 
1,092

 
1,153

   Other
1,412

 
1,910

 
(498
)
Total research and development expenses
$
37,418

 
$
43,712

 
$
(6,294
)
Research and development expenses were $37.4 million for the three months ended June 30, 2018 and consisted primarily of $25.1 million related to AXO-Lenti-PD including the $25.0 million license fee paid to Oxford BioMedica, $3.4 million related to the nelotanserin clinical study, share-based compensation expense of $2.5 million , employee salaries and benefits of $1.5 million and $1.3 million related to intepirdine clinical studies and related wind down activities. The share-based compensation expense for the three months ended June 30, 2018 included $0.4 million related to the RSL common share awards and RSL options issued by RSL to RSI employees.
Research and development expenses were $43.7 million for the three months ended June 30, 2017 and consisted primarily of program-specific costs of $30.6 million , share-based compensation of $6.3 million and personnel-related expenses of $3.8 million . The share-based compensation expense included $1.8 million related to the RSL common share awards and RSL options issued by RSL to RSI employees.

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Research and development expenses decreased by $6.3 million , to $37.4 million , in the three months ended June 30, 2018 compared to the three months ended June 30, 2017 , as intepirdine costs decreased by $25.5 million due to the discontinuation of our intepirdine program, offset by the $25.0 million license fee paid to Oxford BioMedica for AXO-Lenti-PD in June 2018. Share-based compensation expense decreased by $3.7 million , primarily due to decreased headcount related to our previously announced reduction in workforce and personnel-related expenses decreased by $2.3 million primarily due to decreased headcount.
General and Administrative Expenses
General and administrative expenses were $11.8 million for the three months ended June 30, 2018 and consisted primarily of share-based compensation expense of $3.3 million , legal and professional fees of $3.0 million , employee salaries and related benefits of $1.9 million , and $1.2 million of direct and indirect costs allocated to us under the services agreements with RSI and RSG. The share-based compensation expense for the three months ended June 30, 2018 included share-based compensation expense of $0.1 million for RSL common share awards and RSL options issued to RSI employees.
General and administrative expenses were $21.5 million for the three months ended June 30, 2017 and consisted primarily of share-based compensation expense of $9.3 million , employee salaries and related benefits of $4.7 million , legal and professional fees of $1.7 million , and $1.6 million of direct and indirect costs allocated to us under the services agreement with RSI. The share-based compensation expense for the three months ended June 30, 2017 included $0.3 million for RSL common share awards and RSL options issued to RSI employees.
General and administrative expenses decreased by $9.8 million , to $11.8 million , in the three months ended June 30, 2018 compared to the three months ended June 30, 2017 , primarily due to decreases in share based compensation expense of $6.0 million and personnel-related expenses of $2.8 million due to decreased headcount and a decrease in marketing expenses of $2.6 million due to negative clinical trial results, partially offset by an increase of $1.3 million in professional fees due to business development activities, public filings and financings.
Interest Expense
Interest expense was $2.0 million for the three months ended June 30, 2018 , consisting of interest paid and the amortization of debt discount related to the Loan Agreement.
Interest expense for the three months ended June 30, 2017 was $1.9 million , consisting of interest paid and the amortization of debt discount related to the Loan Agreement with Hercules.
Income Tax Expense
Income tax expense decreased by $2.4 million , to $0.1 million , in the three months ended June 30, 2018 compared to the three months ended June 30, 2017 , which was due to a valuation allowance recorded to offset our net deferred tax assets related to net operating losses during the three months ended June 30, 2017 , which did not recur during the three months ended June 30, 2018 .
Liquidity and Capital Resources
Overview
In April 2017, we raised net proceeds of approximately $134.6 million , after deducting underwriting discounts and commissions and offering expenses paid by us, from the sale of 7,753,505 common shares in a follow-on public offering. As of June 30, 2018 , we had cash totaling $92.9 million .
On June 22, 2018, we entered into a sales agreement with Cowen and Company, LLC ("Cowen") to sell our common shares having an aggregate offering price of up to $75.0 million from time to time through an at-the-market equity offering program under which Cowen is acting as our agent. Cowen is entitled to compensation for its services in an amount up to 3% of the gross proceeds of any of our common shares sold under the sales agreement. As of June 30, 2018, $75.0 million of our common shares remained available for sale under the sales agreement.
On July 9, 2018, we received $25.0 million of net proceeds from RSL in exchange for the issuance and sale of 14,285,714 of our common shares to RSL at a purchase price of $1.75 per share, which was the closing price per share of our common shares on the Nasdaq Global Select Market on June 5, 2018, the date of the share purchase agreement.

31



Loan and Security Agreement with Hercules Capital, Inc.
On February 2, 2017, we and our wholly owned subsidiaries, AHL, ASG and ASI, entered into the Loan Agreement with Hercules. Pursuant to the Loan Agreement, we, AHL and ASG, as the Borrowers, borrowed an aggregate of $55.0 million. ASI issued a guaranty of the Borrowers’ obligations under the Loan Agreement, and at the closing, we paid Hercules a facility charge of $550,000. Subsequently, we added our subsidiary ASA as a Borrower in July 2017 and our subsidiaries ATH and ATI as Borrowers in April 2018.
The Term Loan bears interest at a variable per annum rate calculated for any day as the greater of either (i) the prime rate plus 6.80%, and (ii) 10.55%. The Term Loan has a scheduled maturity date of March 1, 2021. The borrowers are obligated to make monthly payments of accrued interest under the Loan Agreement until September 1, 2018, followed by monthly installments of principal and interest through March 1, 2021. The borrowers’ obligations under the Loan Agreement are secured by a first position lien on substantially all of their and ASI’s respective assets, other than intellectual property. If we prepay the loan prior to March 1, 2021, we will be obligated to pay Hercules a prepayment charge, based on a percentage of the then-outstanding principal balance, equal to 3.0% if the prepayment occurs within the first 18 months following February 2, 2017, 2.0% if the prepayment occurs after 18 months but prior to 36 months following February 2, 2017, and 1.0% if the prepayment occurs thereafter.
The Loan Agreement includes customary affirmative and restrictive covenants and representations and warranties, including a minimum cash covenant, a covenant against the occurrence of a "change in control," financial reporting obligations, and certain limitations on indebtedness, liens (including a negative pledge on intellectual property and other assets), investments, distributions (including dividends), collateral, transfers, mergers or acquisitions, taxes, corporate changes, and deposit accounts. The Loan Agreement also includes customary events of default, including payment defaults, breaches of covenants following any applicable cure period, the occurrence of certain events that could reasonably be expected to have a "material adverse effect" as set forth in the Loan Agreement, cross acceleration to the debt and certain events relating to bankruptcy or insolvency. Upon the occurrence of an event of default, a default interest rate of an additional 5.0% may be applied to the outstanding principal balance, and Hercules may declare all outstanding obligations immediately due and payable and take such other actions as set forth in the Loan Agreement. In addition, for so long as the Term Loan remains outstanding, we are required to use commercially reasonable efforts to afford Hercules the opportunity to participate in future underwritten equity offerings of our common shares up to a total of $3.0 million .
In connection with the entry into the Loan Agreement, we issued a warrant to Hercules which was exercisable for an aggregate of 274,086 of our common shares at an exercise price of $12.04 per share. In August 2017, Hercules exercised the warrant on a cashless basis and received a net issuance of 129,827 of our common shares.
For the three months ended June 30, 2018 , we used $61.4 million and $0.0 million of cash in our operating and investing activities, respectively. We have incurred and expect to continue to incur significant and increasing operating losses at least for the next several years. We do not expect to generate revenue unless and until after we successfully complete development and obtain regulatory approval for one of our products in development. Our cash utilization may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our planned clinical trials and our expenditures on other research and development activities and activities related to potential commercialization. We anticipate that we will continue to incur significant expenses as we:

initiate clinical development of AXO-Lenti-PD for advanced Parkinson's disease, including our planned Phase 1/2 trial before the end of 2018;
initiate clinical development of AXO-AAV-OPMD for the treatment of OPMD, including our planned placebo-controlled clinical study in 2019;
continue the clinical development of nelotanserin for LBD and other potential indications;
potentially continue the clinical development of RVT-104, a combination of a peripheral muscarinic receptor antagonist and high-dose rivastigmine;

continue open-label extension studies for patients completing our nelotanserin phase 2 studies;
seek to identify, acquire, develop and commercialize additional product candidates;
integrate acquired technologies into a comprehensive regulatory and product development strategy;
achieve milestones under our agreements with third parties that will require us to make substantial payments to those parties;
maintain, expand and protect our intellectual property portfolio;

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hire and retain scientific, clinical, regulatory, manufacturing, quality control, commercial and administrative personnel;
add operational, financial and management information systems and personnel, including personnel to support our drug development efforts;
seek regulatory approvals for any product candidates that successfully complete clinical trials;
scale up external manufacturing capabilities to commercialize our product candidates;
establish a sales, marketing and distribution infrastructure for drug candidates for which we may obtain regulatory approval; and
operate as a public company.
Our primary use of cash is to fund the research and development of our product candidates. We believe that our existing cash resources will be sufficient to enable us to fund our operating expenses and capital expenditure requirements for at least the next 12 months. We have based our estimates on assumptions that may prove to be incorrect, and we could use our available capital resources sooner than we currently expect. Our existing funds will not be sufficient to enable us to complete all necessary development and to commercially launch all of our products. Accordingly, we may be required to obtain further funding through public or private equity offerings, debt financing, collaboration and licensing arrangements or other sources. Adequate additional funding may not be available to us on acceptable terms, or at all. If we are unable to raise capital in sufficient amounts or on terms acceptable to us, we may have to significantly delay, scale back or discontinue the development or commercialization of one or more of our product candidates or potentially discontinue operations.
Until such time, if ever, as we can generate substantial revenue from sales of our products in development, we expect to finance our cash needs through a combination of equity offerings, debt financings and potential collaboration, license or development agreements. We do not currently have any committed external source of funds. To the extent that we raise additional capital through the sale of equity or convertible debt securities, our shareholders’ ownership interests will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect our shareholders’ rights. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may be required to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or to grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds when needed, we may be required to delay, limit, reduce or terminate our drug development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Cash Flows
The following table sets forth a summary of our cash flows for the three months ended June 30, 2018 and 2017 (in thousands):
 
Three Months Ended June 30,
 
2018
 
2017
Net cash used in operating activities
$
(61,413
)
 
$
(47,919
)
Net cash used in investing activities

 
(2,171
)
Net cash provided by financing activities
10

 
135,375

Operating Activities  
Cash flows from operating activities consist of net loss adjusted for non-cash items, including depreciation and amortization and share-based compensation expense, as well as the effect of changes in working capital and other activities.

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For the three months ended June 30, 2018 , net cash used in operating activities was $61.4 million and was primarily attributable to a net loss of $51.9 million , which includes costs incurred for research and development activities, including CRO fees, manufacturing, regulatory and other clinical trial costs and our general and administrative expenses, a decrease of $6.7 million in accrued expenses, an increase of $5.3 million in prepaid assets and other current assets, and an increase of $3.8 million in other non-current assets, which were partially offset by $5.9 million of non-cash share-based compensation expense. For the three months ended June 30, 2017 , net cash used in operating activities was $47.9 million and was primarily attributable to a net loss of $69.3 million , which includes costs incurred for research and development activities, including CRO fees, manufacturing, regulatory and other clinical trial costs, and our general and administrative expenses, partially offset by $15.6 million of non-cash share-based compensation expense, as well as an increase of $3.2 million in accrued liabilities and a decrease of $2.7 million in deferred tax assets.
Investing Activities  
For the three months ended June 30, 2018 , no cash was used in investing activities. For the three months ended June 30, 2017, net cash used in investing activities was $2.2 million , consisting of purchases of furniture and equipment.
Financing Activities
For the three months ended June 30, 2018 , net cash provided by financing activities was approximately $10,000 and consisted of proceeds from the exercise of stock options. For the three months ended June 30, 2017 , net cash used in financing activities was $135.4 million , which consisted primarily of net proceeds of $134.6 million received from the sale of 7,753,505 common shares in a follow-on public offering.
Contractual Obligations
Our contractual obligations did not materially change outside the ordinary course of our business during the three months ended June 30, 2018, as compared to those disclosed in our Annual Report on Form 10-K for the year ended March 31, 2018, except that, in June 2018, we entered into the Oxford BioMedica Agreement and, in July 2018, we entered into the Benitec Agreement. See “-Our Key Agreements” above for additional information regarding these agreements and our commitments thereunder.

Off-Balance Sheet Arrangements
We did not have during the periods presented, and we do not currently have, any off-balance sheet arrangements, as defined under the SEC’s rules. Accordingly, our operating results, financial condition and cash flows are not subject to off-balance sheet risks.
Critical Accounting Policies and Significant Judgments and Estimates
Our unaudited condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States ("U.S. GAAP"). The preparation of these financial statements requires us to make estimates, judgments and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities as of the dates of the balance sheets and the reported amounts of expenses during the reporting periods. In accordance with U.S. GAAP, we evaluate our estimates and judgments on an ongoing basis. Significant estimates include assumptions used in the determination of some of our costs incurred under the services agreements with RSI and RSG, which costs are charged to research and development and general and administrative expense, as well as assumptions used to estimate the fair value of our common shares. We base our estimates on historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.
We define our critical accounting policies as those under U.S. GAAP that require us to make subjective estimates and judgments about matters that are uncertain and are likely to have a material impact on our financial condition and results of operations, as well as the specific manner in which we apply those principles.
We believe the estimates and judgments involved in our contingent payment liabilities, research and development accruals, share-based compensation and income taxes have the greatest potential impact on our unaudited condensed consolidated financial statements, and consider these to be our critical accounting policies and estimates.
Our significant accounting policies are more fully described in Note 2 to our unaudited condensed consolidated financial statements in this Quarterly Report on Form 10-Q and Note 2 to our consolidated financial statements in our Annual Report. There have been no material changes to our critical accounting policies and significant judgments and estimates as compared to the critical accounting policies and significant judgments and estimates described in our Annual Report.
Recent Accounting Pronouncements


34



In February 2016, the Financial Accounting Standards Board ("FASB") issued ASU No. 2016-02, ‘‘ Leases (Topic 842) ’’ (‘‘ASU No. 2016-02’’), which is a comprehensive new lease standard that amends various aspects of existing accounting guidance for leases. The core principle of ASU No. 2016-02 will require lessees to present the assets and liabilities that arise from leases on their balance sheets. ASU No. 2016-02 is effective for annual periods beginning after December 15, 2018, and interim periods within fiscal years beginning after December 15, 2018. Early adoption is permitted. We are currently evaluating the provisions of ASU No. 2016-02 and its impact on our consolidated financial statements and related disclosures.

In January 2017, the FASB issued ASU 2017-01, ‘‘ Business Combinations (Topic 805): Clarifying the Definition of a Business ’’ (‘‘ASU No. 2017-01’’), which clarifies the definition of a business with the objective of adding guidance to assist entities with evaluating whether transactions should be accounted for as acquisitions (or disposals) of assets or businesses. We adopted the provisions of ASU No. 2017-01 on April 1, 2018 on a prospective basis. The impact on our consolidated financial statements and disclosures will depend on the facts and circumstances of any specific future transactions. Refer to Note 3, “License Agreement,” in the accompanying notes to the unaudited condensed consolidated financial statements for further information regarding the impact of the adoption of ASU No. 2017-01 on the license agreement executed during the three months ended June 30, 2018.

In February 2018, the FASB issued ASU No. 2018-02, " Income Statement-Reporting Comprehensive Income (Topic 220): Reclassification of Certain Tax Effects from Accumulated Other Comprehensive Income" ("ASU No. 2018-02"). ASU No. 2018-02 allows companies to reclassify stranded tax effects resulting from the Tax Cuts and Jobs Act, from accumulated other comprehensive (loss) income to retained earnings. ASU No. 2018-02 is effective for interim and annual reporting periods beginning after December 15, 2018 and early adoption is permitted. We expect to adopt the provisions of ASU No. 2018-02 for the fiscal year beginning April 1, 2019. As we have not yet completed our final review of the impact of ASU No. 2018-02 but expect to by March 31, 2019, we have not determined whether the adoption of this guidance will have a material impact on our consolidated financial statements or disclosures.

In March 2018, the FASB issued ASU No. 2018-05, " Income Taxes (Topic 740): Amendments to SEC Paragraphs Pursuant to SEC Staff Accounting Bulletin No. 118 ," ("ASU No. 2018-05"). ASU No. 2018-05 amends certain SEC material in Topic 740 for the income tax accounting implications of the Tax Cuts and Jobs Act. ASU No. 2018-05 was effective immediately. We evaluated the impact of the Tax Cuts and Jobs Act as well as the guidance of Staff Accounting Bulletin 118 and incorporated the changes into the determination of a reasonable estimate of deferred taxes and appropriate disclosures in the notes to our consolidated financial statements. We will continue to evaluate the impact this tax reform legislation may have on our results of operations, financial position, cash flows and related disclosures.

In June 2018, the FASB issued ASU No. 2018-07, " Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting," (ASU No. 2018-07) . ASU No. 2018-07 requires equity-classified share-based payment awards issued to nonemployees to be measured on the grant date, rather than remeasuring the awards through the performance completion date as previously required. Additionally, for nonemployee awards with performance conditions, compensation cost associated with the award is to be recognized when achievement of the performance condition is probable, rather than upon achievement of the performance condition. Further, the requirement to reassess the liability or equity classification for nonemployee awards upon vesting is eliminated, except for awards in the form of convertible instruments. ASU No. 2018-07 also clarifies that any share-based payment awards issued to customers should be evaluated under ASC 606, Revenue from Contracts with Customers . ASU No. 2018-07 is effective for fiscal years beginning after December 15, 2018, including interim periods within that fiscal year, with early adoption permitted after the adoption of ASU No. 2014-09. We expect to adopt the provisions of ASU No. 2018-07 for the fiscal year beginning April 1, 2019. As we have not yet completed our final review of the impact of ASU No. 2018-07 but expect to by March 31, 2019, we have not determined whether the adoption of this guidance will have a material impact on our consolidated financial statements or disclosures. 

Item 3.        Quantitative and Qualitative Disclosures About Market Risk
Market risk is the potential loss arising from adverse changes in market rates and market prices such as interest rates, foreign currency exchange rates, and changes in the market value of equity instruments. As of June 30, 2018 , we had cash of $92.9 million , consisting of non-interest-bearing deposits denominated in the U.S. dollar and Swiss franc. We also have long-term debt that bears interest at a prime-based variable rate. A 10% change in this interest rate would have an approximate $0.6 million impact on our annual interest expense. We do not believe we are currently exposed to any material market risk.


35



Item 4.        Controls and Procedures
Evaluation of Disclosure Controls and Procedures
Under the supervision of our principal executive officer and principal financial officer, we evaluated the effectiveness of our disclosure controls and procedures as of June 30, 2018 , the end of the period covered by this Quarterly Report on Form 10-Q. The term "disclosure controls and procedures" (as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act of 1934, as amended (the "Exchange Act"), means controls and other procedures of a company that are designed to provide reasonable assurance that information required to be disclosed by us in the reports that we file or submit under the Exchange Act is recorded, processed, summarized and reported within the time periods specified in the SEC’s rules and forms.
Disclosure controls and procedures include, without limitation, controls and procedures designed to provide reasonable assurance that information required to be disclosed by us in the reports that we file or submit under the Exchange Act is accumulated and communicated to our management, including our principal executive officer and principal financial officer, as appropriate, to allow for timely decisions regarding required disclosure. Based on this evaluation, our principal executive officer and principal financial officer concluded that our disclosure controls and procedures were effective as of June 30, 2018 at the reasonable assurance level.
Changes in Internal Control over Financial Reporting
There was no change in our internal control over financial reporting identified in connection with the evaluation required by Rule 13a-15(d) and 15d-15(d) of the Exchange Act that occurred during the period covered by this Quarterly Report on Form 10-Q that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.
Inherent Limitations on Effectiveness of Controls
Our management, including our principal executive officer and principal financial officer, does not expect that our disclosure controls and procedures or our internal controls will prevent all error and all fraud. A control system, no matter how well conceived and operated, can provide only reasonable, not absolute, assurance that the objectives of the control system are met. Further, the design of a control system must reflect the fact that there are resource constraints, and the benefits of controls must be considered relative to their costs. Because of the inherent limitations in all control systems, no evaluation of controls can provide absolute assurance that all control issues and instances of fraud, if any, within Axovant Sciences Ltd. have been detected.


36



PART II: OTHER INFORMATION

Item 1.                                                          Legal Proceedings
From time to time, we may become involved in legal proceedings relating to claims arising from the ordinary course of business. We are not currently a party to any material legal proceedings, and we are not aware of any pending or threatened legal proceeding against us that we believe could have an adverse effect on our business, operating results or financial condition.

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Item 1A.                                                 Risk Factors
You should carefully consider the following risk factors, in addition to the other information contained in this Quarterly Report on Form 10-Q, including the section of this report titled "Management’s Discussion and Analysis of Financial Condition and Results of Operations" and our unaudited condensed consolidated financial statements and related notes. If any of the events described in the following risk factors and the risks described elsewhere in this report occurs, our business, operating results and financial condition could be seriously harmed, and the trading price of our common shares could decline. This Quarterly Report on Form 10-Q also contains forward-looking statements that involve risks and uncertainties. Our actual results could differ materially from those anticipated in the forward-looking statements as a result of factors that are described below and elsewhere in this report.
  Risks Related to Our Business, Financial Position and Capital Requirements
  We have a limited operating history and have never generated any product revenues.
We are a clinical-stage biopharmaceutical company with a limited operating history. We were formed in October 2014, and our operations to date have been limited to organizing and staffing our company, raising capital, acquiring drug development programs and preparing for and advancing our existing and former product candidates into clinical development. We have not yet demonstrated an ability to successfully complete a large-scale, pivotal clinical trial, obtain marketing approval, manufacture a commercial-scale product, or arrange for a third-party to do so on our behalf, or conduct sales and marketing activities necessary for successful product commercialization. Consequently, we have no meaningful operations upon which to evaluate our business and predictions about our future success or viability may not be as accurate as they could be if we had a longer operating history or a history of successfully developing and commercializing pharmaceutical products.
In addition, we may encounter unforeseen expenses, difficulties, complications, delays and other known and unknown factors. The failure of our Phase 3 MINDSET trial, Phase 2b HEADWAY trial and Phase 2 Gait and Balance trial for intepirdine has required us to reevaluate our future development plans for our product candidates, as well as our business plan more broadly. We may never be successful in developing or commercializing any of our product candidates, including our newly licensed product candidates AXO-Lenti-PD and AXO-AAV-OPMD, which remain in early stages of clinical development. If successful in developing and obtaining marketing approval of one of our product candidates, we would need to transition from a company with a product development focus to a company capable of supporting commercial activities. We may not be successful in such a transition.
Our ability to generate revenue and become profitable depends upon our ability to successfully complete the development of our product candidates and other assets in the fields of neurology and psychiatry and to obtain the necessary regulatory approvals for their commercialization. We have never been profitable, have not generated any revenue from product sales, and have no products approved for commercial sale.
Even if we receive regulatory approval for our product candidates, we do not know when those candidates will generate revenue, if at all. Our ability to generate product revenue depends on a number of factors, including our ability to:
successfully commence and complete clinical trials and obtain regulatory approval for the marketing of our product candidates, including AXO-Lenti-PD and AXO-AAV-OPMD;
set an acceptable price for our product candidates and obtain coverage and adequate reimbursement from third-party payers;
establish effective sales, marketing and distribution systems for our product candidates;
add operational, financial and management information systems and personnel, including personnel to support our clinical, manufacturing and planned future commercialization efforts and operations as a public company;
initiate and continue relationships with third-party manufacturers, including Oxford BioMedica for AXO-Lenti-PD, and have commercial quantities of our product candidates manufactured at acceptable cost and quality levels;
attract and retain an experienced management and advisory team;
achieve broad market acceptance of our products in the medical community and with third-party payers and consumers;
launch commercial sales of our products, whether alone or in collaboration with others; and
maintain, expand and protect our intellectual property portfolio.


38



Because of the numerous risks and uncertainties associated with product development, we are unable to predict the timing or amount of increased expenses, or when, or if, we will be able to achieve or maintain profitability. Our expenses could increase beyond expectations if we are required by the FDA, European Medicines Agency ("EMA"), Japan’s Pharmaceutical and Medical Devices Agency ("PMDA"), or comparable regulatory authorities in other countries, to perform studies or clinical trials in addition to those that we currently anticipate. Even if our product candidates are approved for commercial sale, we anticipate incurring significant costs associated with their commercial launch. If we cannot successfully execute any one of the foregoing, our business may not succeed, and your investment will be adversely affected.
We are in the process of implementing a business plan that may continue to evolve as we integrate our newly licensed product candidates AXO-Lenti-PD and AXO-AAV-OPMD and await relevant clinical data for nelotanserin. Our business plan may lead to the initiation of one or more development programs or the execution of one or more transactions that you do not agree with or that you do not perceive as favorable to your investment.
In early 2018, we began a process to review our strategic alternatives, including identifying potential business development opportunities, following the discontinuation of further development of intepirdine in January 2018. Also beginning in early 2018, we undertook a reassessment of our development plans for nelotanserin in various indications and RVT-104, which included an internal portfolio review of RVT-104 in the context of any newly acquired clinical assets. We plan to make a determination of the overall development strategy for nelotanserin once we have reviewed final data from our currently ongoing Phase 2 study of nelotanserin in REM Sleep Behavior Disorder and completed our ongoing comprehensive clinical, regulatory and commercial review.
In June 2018, we announced that we received from Oxford BioMedica a worldwide exclusive license to develop and commercialize AXO-Lenti-PD and its predecessor product candidate ProSavin and related gene therapy products, and in July 2018, we announced that we received from Benitec a worldwide exclusive license to develop and commercialize investigational gene therapy AXO-AAV-OPMD and related gene therapy products. We initially plan to pursue a strategy to leverage our clinical experience and expertise in neurology and psychiatry to pursue the clinical development and regulatory approval of AXO-Lenti-PD and AXO-AAV-OPMD while evaluating overall development strategy for nelotanserin and RVT-104. In addition, we continue to be actively engaged in extensive business development efforts to identify, evaluate and potentially obtain rights to and develop additional therapeutic and gene therapy product candidates that would complement our strategic goals and leverage our competitive advantages.
This business plan requires us to be successful in a number of challenging, uncertain and risky activities, including pursuing development of AXO-Lenti-PD and AXO-AAV-OPMD in indications for which we have limited or no human clinical data, identifying promising new assets for development that are available for acquisition or in-license and that fit our strategic focus and, if so identified, negotiating and executing an acquisition or in-license agreement for one or more of those programs on favorable terms, converting early stage gene therapy research efforts into clinical development opportunities, building internal or outsourced gene therapy capabilities and designing and executing a nonclinical and/or clinical development program for any newly acquired product candidates. We may not be successful at one or more of the activities required for us to execute this business plan. We are also continuing to consider other alternatives, including mergers or other transactions involving our company as a whole or other collaboration transactions. We cannot be sure when or if this process will result in any type of transaction. Even if we pursue a transaction, such transaction may not be consistent with our shareholders’ expectations or may not ultimately be favorable for our shareholders, either in the shorter or longer term.
Our growth prospects and the future value of our company are primarily dependent on the progress of our ongoing and planned clinical development programs for AXO-Lenti-PD and AXO-AAV-OPMD as well as the outcome of our ongoing business development efforts and pipeline expansion activities, together with the amount of our remaining available cash. The development of AXO-Lenti-PD and AXO-AAV-OPMD and the outcome of our ongoing business development efforts and pipeline expansion activities are highly uncertain.
We have only very limited data from small, uncontrolled clinical trials, performed by or on behalf of Oxford BioMedica, regarding the safety and tolerability of ProSavin, as the predecessor product candidate to AXO-Lenti-PD, in patients with advanced Parkinson’s disease, as well as nonclinical in vitro experiments with AXO-Lenti-PD. Prior ProSavin trials were not powered to demonstrate the efficacy of the therapy with statistical significance. Given the information above, these trials could be underpowered to demonstrate a potential clinical benefit for AXO-Lenti-PD in these indications. In addition, we have no prior clinical data regarding the safety, tolerability and efficacy of AXO-AAV-OPMD or any additional gene therapy product under the Benitec Agreement.

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We expect to continue to reassess and make changes to our existing development programs and pipeline expansion strategy. Our future plans for our AXO-Lenti-PD and AXO-AAV-OPMD development programs may be affected by the results of competitors’ clinical trials of product candidates addressing Parkinson’s disease and OPMD, respectively. Our future plans for additional gene therapy products under the Benitec Agreement may also be affected by the results of competitors’ clinical trials of product candidates addressing our target indications, including, for example, ALS and FTD, in the case of our first planned additional Benitec collaboration gene therapy product. Our plans for our business development efforts and pipeline expansion activities may be affected by the results of competitors' ongoing research and development efforts. We may modify, expand or terminate some or all of our development programs, clinical trials or collaborative research programs at any time as a result of new competitive information or as the result of changes to our product pipeline or business development strategy.
We expect that our remaining cash balances will continue to decline as we pursue these development programs, pursue our collaborative research programs, pursue our business development activities and until such time, if any, as we receive additional funding, and the value of our shareholders’ investment may decline as a result.
We expect to incur significant losses for the foreseeable future and may never achieve or maintain profitability.
Investment in pharmaceutical and biological product development is highly speculative because it entails substantial upfront capital expenditures and significant risk that a product candidate will fail to gain regulatory approval or become commercially viable. We have never generated any revenues, and we cannot estimate with precision the extent of our future losses. We do not currently have any products that are available for commercial sale and we may never generate revenue from selling products or achieve profitability. We expect to continue to incur substantial and increasing losses through the projected commercialization of our product candidates. Our product candidates have not been approved for marketing in the United States or any other jurisdiction, and we may never receive any such approvals. In addition, we discontinued further development of our product candidate intepirdine, which was our most progressed product candidate in clinical development at that time. While we have recently in-licensed our new product candidates AXO-Lenti-PD and AXO-AAV-OPMD, these product candidates remain in early stages of clinical development. As a result, we are uncertain when or if we will achieve profitability and, if so, whether we will be able to sustain it. Our ability to produce revenue and achieve profitability is dependent on our ability to complete the development of our product candidates, obtain necessary regulatory approvals, and have our product candidates manufactured and successfully marketed and commercialized. We cannot assure you that we will be profitable even if we successfully commercialize our product candidates. If we do successfully obtain regulatory approval to market our product candidates, our revenues will be dependent, in part, upon, among other things, the size of the markets in the territories for which we gain regulatory approval, the number of competitors in such markets, the accepted price for our product candidates and whether we own the commercial rights for that territory. If the indication approved by regulatory authorities is narrower than we expect, or the treatment population is narrowed by competition, physician choice or treatment guidelines, we may not generate significant revenue from sales of our product candidates, even if approved. Even if we do achieve profitability, we may not be able to sustain or increase profitability on a quarterly or annual basis. Failure to become and remain profitable may adversely affect the market price of our common shares and our ability to raise capital and continue operations.
We expect our research and development expenses to be significant as we develop: AXO-Lenti-PD for the potential treatment of Parkinson's disease; AXO-AAV-OPMD for the potential treatment of OPMD; our additional gene therapy products under the Benitec Agreement for the potential treatment of ALS, FTD and other undisclosed indications; nelotanserin for the potential treatment of multiple aspects of LBD; and RVT-104 for the potential treatment of Alzheimer's disease and DLB. In addition, if we obtain regulatory approval for any of our product candidates, we expect to incur increased sales and marketing expenses. As a result, we expect to continue to incur significant and increasing operating losses and negative cash flows for the foreseeable future. These losses have had and will continue to have an adverse effect on our financial position and working capital.
We may not be successful in our efforts to identify and acquire additional product candidates.
Part of our strategy involves the business development activities of identifying and acquiring novel product candidates, and this aspect of our business has become more important as a result of our discontinuation of our intepirdine program. The process by which we identify product candidates may fail to yield product candidates for clinical development for a number of reasons, including those discussed in these risk factors and also:
the process by which we identify and decide to acquire product candidates may not be successful;
potential product candidates may, on further study, be shown to have harmful side effects or other characteristics that indicate that they are unlikely to be products that will receive marketing approval and achieve market acceptance; or
potential product candidates may not be effective in treating their targeted diseases.

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The process of identifying and acquiring product candidates is highly competitive, and our ability to compete successfully is impacted by the fact that many of the companies with which we compete for these candidates have significantly greater experience, development and commercialization capabilities, name recognition and financial and human resources than we do. Further, our business development efforts are led by our senior executive officers and other management team members and would be significantly impaired if we were to lose the services of any of these executives. The time and resources spent on business development activities may also distract management’s attention from our other development and business activities. Even if we are successful in identifying and acquiring additional product candidates, we may choose to focus our efforts and resources on a potential product candidate that ultimately proves to be unsuccessful. If we are unable to identify and acquire suitable product candidates for clinical development, this would adversely impact our business strategy and our financial position and share price.
We are heavily dependent on the success of AXO-Lenti-PD and AXO-AAV-OPMD, our gene therapy product candidates, which are still in early stages of clinical development, and if either product candidate does not receive regulatory approval or is not successfully commercialized, our business may be harmed.
We currently have no products that are approved for commercial sale and may never be able to develop marketable drug products. We expect that a substantial portion of our efforts and expenditures over the next few years will be devoted to AXO-Lenti-PD and AXO-AAV-OPMD. Accordingly, our business currently depends heavily on the successful development, regulatory approval and commercialization of either product candidate. We cannot be certain that any of our product candidates will receive regulatory approval or be successfully commercialized even if we receive regulatory approval. The research, testing, manufacturing, labeling, approval, sale, marketing and distribution of drug products and biological products are and will remain subject to extensive regulation by the FDA, the EMA, the PMDA and other comparable regulatory authorities that each have differing regulations. We are not permitted to market our product candidates in the United States or in any foreign countries until they receive the requisite approvals from the FDA or comparable regulatory authorities in other countries. We have not submitted marketing applications to the FDA or foreign regulatory authorities and do not expect to be in a position to do so for the foreseeable future. Obtaining marketing approval is an extensive, lengthy, expensive and inherently uncertain process, and regulatory authorities may delay, limit or deny approval of our product candidates for many reasons, including:
we may not be able to demonstrate that a product candidate is safe and effective as a treatment for our targeted indications to the satisfaction of the applicable regulatory authorities;
our BLA, NDA or other key regulatory filings may be delayed or rejected due to issues, including those related to the FDA’s Pharmaceutical Quality/CMC guidance, timing of results from supporting studies, database lock, and data conversion, cleaning, and transfer;
the regulatory authorities may require additional nonclinical studies or registrational studies of the product candidate in Parkinson’s disease or other indications, which would increase our costs and prolong our development;
the results of our clinical trials may not meet the level of statistical or clinical significance required for marketing approval;
the regulatory authorities may disagree with the number, design, size, conduct or implementation of our clinical trials;
the contract research organizations ("CROs") that we retain to conduct clinical trials may take actions outside of our control, or otherwise commit errors or breaches of protocols, that materially adversely impact our clinical trials;
the regulatory authorities may not find the data from nonclinical studies and clinical trials sufficient to demonstrate that the clinical and other benefits of the product candidate outweigh its safety risks;
the regulatory authorities may disagree with our interpretation of data from our nonclinical studies and clinical trials or may require that we conduct additional studies;
the regulatory authorities may not accept data generated at our clinical trial sites;
the regulatory authorities may require, as a condition of approval, limitations on approved labeling or distribution and use restrictions;
the FDA may require development of a risk evaluation and mitigation strategy ("REMS") as a condition of approval;
the regulatory authorities may identify deficiencies in the manufacturing processes or facilities of our third-party manufacturers, including Oxford BioMedica, which is expected to be our sole and exclusive supplier of AXO-Lenti-PD until the process is validated or BLA submission for AXO-Lenti-PD, as well as Arena, our sole and exclusive supplier for nelotanserin, or any manufacturer that Arena may engage to manufacture nelotanserin on its behalf; or
the regulatory authorities may change their approval policies or adopt new regulations.

The terms of our credit facility place restrictions on our operating and financial flexibility.
In February 2017, we and our subsidiaries entered into the Loan Agreement with Hercules. The Loan Agreement is secured by substantially all of our property and that of our subsidiaries that are parties to the Loan Agreement, other than intellectual property.

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The Loan Agreement subjects us and our subsidiaries to various affirmative and restrictive covenants, including a minimum cash covenant that ceases to apply if we achieve certain clinical development milestones as set forth in the Loan Agreement, a covenant against the occurrence of a "change in control," financial reporting obligations, and certain limitations on the incurrence of indebtedness, liens (including a negative pledge on intellectual property and other assets), investments, distributions (including dividends), collateral, transfers, mergers or acquisitions, taxes, corporate changes, and deposit accounts. Compliance with these covenants may limit our flexibility in operating our business and our ability to take actions that might be advantageous to us and our shareholders.
Additionally, we may be required to repay the entire amount of outstanding indebtedness under the Loan Agreement in cash if we fail to stay in compliance with our covenants or suffer some other event of default under the Loan Agreement. Under the Loan Agreement, an event of default will occur if, among other things: we fail to make payments under the Loan Agreement; we breach any of our covenants under the Loan Agreement, subject to specified cure periods with respect to certain breaches; there occurs an event that has a material adverse effect on (i) our business, operations, properties, assets or financial condition, (ii) our ability to perform or satisfy our obligations under the Loan Agreement as they become due or Hercules’s ability to enforce its rights or remedies with respect to our obligations under the Loan Agreement, or (iii) the collateral or liens securing our obligations under the Loan Agreement; we or our assets become subject to certain legal proceedings, such as bankruptcy proceedings; we are unable to pay our debts as they become due; or we default on contracts with third parties which would permit Hercules to accelerate the maturity of such indebtedness or that could have a material adverse effect on us. We may not have enough available cash or be able to raise additional funds through equity or debt financings to repay such indebtedness at the time any such event of default occurs. In that case, we may be required to delay, limit, reduce or terminate our clinical development efforts or grant to others rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves. Hercules could also exercise its rights as collateral agent to take possession and dispose of the collateral securing the loan for its benefit, which collateral includes all of our property other than our intellectual property. Our business, financial condition and results of operations could be substantially harmed as a result of any of these events.
We will require additional capital to fund our operations, and if we fail to obtain necessary financing, we may not be able to complete the development and commercialization of our product candidates.
We expect to spend substantial amounts to complete the development of, seek regulatory approvals for and commercialize our product candidates. These expenditures will include costs payable to Oxford BioMedica under the Oxford BioMedica Agreement, Benitec under the Benitec Agreement, as well as Arena under the Arena Development Agreement. Under the terms of these agreements, we are obligated to make significant cash payments upon the achievement of specified development, regulatory and sales performance milestones, as well as payments in connection with the sale of resulting products and the manufacture and supply of our product candidates for commercial purposes. For example, we are required to pay Benitec 30% of the net profits from worldwide sales of approved and commercialized products arising from the AXO-AAV-OPMD Program, subject to an agreed minimum for such payments.
We will require additional capital to complete the development and potential commercialization of our product candidates, particularly AXO-Lenti-PD and AXO-AAV-OPMD, which remain in early stages of clinical development. If we are unable to raise capital when needed or on acceptable terms, we could be forced to delay, reduce or eliminate our development program or any future commercialization efforts. In addition, attempting to secure additional financing may divert the time and attention of our management from day-to-day activities and harm our product candidate development efforts.
We believe our existing cash resources will be sufficient to meet our financial needs for at least the next 12 months. This estimate is based on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect. Because the length of time and activities associated with successful development of our product candidates is highly uncertain, we are unable to estimate the actual funds we will require for development and any approved marketing and commercialization activities. Our future funding requirements, both near and long-term, will depend on many factors, including, but not limited to:
the progress, timing, costs and results of our clinical trials of our product candidates;
the outcome, timing and cost of meeting regulatory requirements established by the FDA, the EMA, or the PMDA, and other comparable foreign regulatory authorities;
the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights;
the cost of defending potential intellectual property disputes, including patent infringement actions brought by third parties against us or our product candidates or any future product candidates;
the effect of competing technological and market developments;
the cost and timing of completion of commercial-scale manufacturing activities;
the cost of establishing sales, marketing and distribution capabilities for our product candidates in regions where we choose to commercialize our products on our own; and

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the initiation, progress, timing and results of our commercialization of our product candidates, if approved for commercial sale.

We cannot be certain that additional funding will be available on acceptable terms, or at all. If we are unable to raise additional capital in sufficient amounts or on terms acceptable to us, we may have to significantly delay, scale back or discontinue the development or commercialization of our product candidates or potentially discontinue operations.
We may be required to make significant payments to third parties under the agreements pursuant to which we acquired our product candidates.
In October 2015, we acquired the rights to nelotanserin and assumed the obligations under the Arena Development Agreement; in August 2016, we entered into a license agreement with Qaam for RVT-104; in June 2018, we entered into a license agreement with Oxford BioMedica for AXO-Lenti-PD; and in July 2018, we entered into a license agreement with Benitec for AXO-AAV-OPMD and five research collaboration programs. Under these agreements, we are subject to significant obligations, including payment obligations upon achievement of specified milestones and payments based on product sales, as well as other material obligations. For example, under our agreement with Oxford Biomedica, we could be obligated to make payments totaling up to $55.0 million upon the achievement of specified development milestones and $757.5 million upon the achievement of specified regulatory and sales milestones. In addition, we will also be obligated to pay Oxford BioMedica a tiered royalty percentage ranging from 7% to 10% based on yearly aggregate net sales of the Gene Therapy Products licensed under the agreement. Under the Benitec Agreement, we will be obligated to make payments to Benitec totaling up to (i) for the AXO-AAV-OPMD Program, $67.5 million upon the achievement of specified development and regulatory milestones and $120.0 million upon the achievement of specified sales milestones, and (ii) for each collaboration program under the Benitec Agreement, $33.5 million upon the achievement of specified development and regulatory milestones and $60.0 million upon the achievement of specified sales milestones. In addition, Benitec will receive 30% of net profits of worldwide sales of approved and commercialized products arising from the AXO-AAV-OPMD Program, subject to an agreed minimum amount for such payments, and a tiered royalty based on yearly aggregate net sales of products arising from each Collaboration Program, subject to specified reductions upon the occurrence of certain events. If these payments become due under the terms of the agreements, we may not have sufficient funds available to meet our obligations, in which case our development efforts would be substantially harmed. Further, failure to make these payments or to meet our other material obligations may result in our counterparties pursuing remedies under those agreements that could adversely affect our operations.
Raising additional funds by issuing securities may cause dilution to existing shareholders and raising funds through lending and licensing arrangements may restrict our operations or require us to relinquish proprietary rights.
We expect that significant additional capital will be needed in the future to continue our planned operations. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, strategic alliances and license and development agreements in connection with any collaborations. We do not have any committed external source of funds. To the extent that we raise additional capital by issuing equity securities, including pursuant to our "shelf" registration statement filed with the SEC, our existing shareholders’ ownership may experience substantial dilution, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a common shareholder. Additional debt financing or preferred equity financing, if available, may involve agreements that include covenants further limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends.
If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise develop and market ourselves.

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We currently rely on our affiliates RSI and RSG to provide various administrative, research and development and other services.
To operate our business, we rely in part on services provided by RSI or RSG, wholly owned subsidiaries of RSL, pursuant to the Services Agreements we have with these entities. Personnel and support staff who provide services to us under these Services Agreements are not required to treat management and administration of our business as their primary responsibility or act exclusively for us, and we do not expect them to do so. Under the Services Agreements, RSI and RSG have the discretion to determine who, among their employees, will perform services for us. RSI and RSG have limited financing and accounting and other resources. If either RSI or RSG fails to perform its obligations in accordance with the terms of the Services Agreements or to effectively manage services provided to us, the operations of our business may be adversely affected.
In addition, we expect that the level of support we receive from RSI and RSG will decrease in the near term as RSL seeks to decrease and decentralize the amount of services it provides to its affiliated companies. As a result, we will be required to replace many of these services, including research and development, administrative and financial functions, with our own internally developed teams or substitute third-party service providers. We primarily intend to develop these capabilities internally and expect to incur increased costs as we hire and train additional personnel. If we are unable to develop these capabilities or we fail to do so in a timely and effective manner, the operations of our business would be adversely affected.
We may not be able to manage our business effectively if we are unable to attract and retain key personnel. In addition, if we are unable to effectively transition and integrate our new executive officers and solidify and implement our updated business strategy, our business and financial performance could be adversely affected.
We may not be able to attract or retain qualified management and commercial, scientific and clinical personnel due to the intense competition for qualified personnel among biotechnology, pharmaceutical and other businesses. If we are not able to attract and retain necessary personnel to accomplish our business objectives, we may experience constraints that will significantly impede the achievement of our development objectives, our ability to raise additional capital and our ability to implement our business strategy.
Several members of our senior management team are relatively new to Axovant. Our financial performance will depend in significant part on our senior management team and key employees, including new members of management with expertise in the gene therapy development field. In addition, recent corporate restructurings may have impacted employee morale and led, and may continue to lead, to higher rates of voluntary attrition compared to prior years. We are highly dependent on the skills and leadership of our management team. Our senior management and key employees may terminate their position with us at any time.
If we lose one or more members of our senior management team or key employees, our ability to successfully implement our business strategy could be seriously harmed. Replacing these individuals may be difficult, cause disruption, and may take an extended period of time because of the limited number of individuals in our industry with the breadth of skills and experience required to develop, gain regulatory approval of and commercialize products successfully. Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain or motivate additional key personnel. We do not maintain "key person" insurance for any of our executives or other employees.
We will need to expand our organization, and we may experience difficulties in managing this growth, which could disrupt our operations.
We expect to hire, either directly or through ASI, ASG or ASA, additional employees for our managerial, clinical, scientific and engineering, operational, sales and marketing teams. We may have operational difficulties in connection with identifying, hiring and integrating new personnel. Future growth would impose significant additional responsibilities on our management, including the need to identify, recruit, maintain, motivate and integrate additional employees, consultants and contractors. Also, our management may need to divert a disproportionate amount of its attention away from our day-to-day activities, including development of product candidates, and devote a substantial amount of time to managing these growth activities. If our management is unable to effectively manage our growth, our expenses may increase more than expected, our ability to generate and grow revenues could be reduced, and we may not be able to implement our business strategy. We may not be able to effectively manage the expansion of our operations across our entities, which may result in weaknesses in our infrastructure, give rise to operational mistakes, loss of business opportunities, loss of employees and reduced productivity among remaining employees. Our future financial performance and our ability to commercialize our product candidates and compete effectively will depend, in part, on our ability to effectively manage any future growth.

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Many of the other pharmaceutical companies that we compete against for qualified personnel and consultants have greater financial and other resources, different risk profiles and a longer history in the industry than we do. They also may provide more diverse opportunities and better chances for career advancement. Some of these characteristics may be more appealing to high-quality candidates and consultants than what we have to offer. If we are unable to continue to attract and retain high-quality personnel and consultants, the rate and success at which we can discover and develop product candidates and our business will be limited.
Our employees, independent contractors, principal investigators, consultants, commercial collaborators, service providers and other vendors, or those of our affiliates, may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements, which could have an adverse effect on our results of operations.
Our employees and contractors, including principal investigators, consultants, commercial collaborators, manufacturers, service providers and other vendors, or those of our affiliates, may engage in fraudulent or other illegal activity. Misconduct by these parties could include intentional, reckless and/or negligent conduct or other unauthorized activities that violate the laws and regulations, including those of the FDA and other similar regulatory bodies that require the reporting of true, complete and accurate information; manufacturing standards; federal, state and foreign healthcare fraud and abuse laws and data privacy; or laws that require the true, complete and accurate reporting of financial information or data. In particular, sales, marketing and other business arrangements in the healthcare industry are subject to extensive laws intended to prevent fraud, kickbacks, self-dealing, bribery, corruption, antitrust violations, and other abusive practices. These laws may restrict or prohibit a wide range of business activities, including research, manufacturing, distribution, pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements. Activities subject to these laws also involve the improper use or misrepresentation of information obtained in the course of clinical trials, the creation of fraudulent data in nonclinical studies or clinical trials or illegal misappropriation of drug product, which could result in regulatory sanctions and serious harm to our reputation. It is not always possible to identify and deter employee or third-party misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to be in compliance with such laws or regulations. Additionally, we are subject to the risk that a person or government agency could allege such fraud or other misconduct, even if none occurred. If our employees, independent contractors, principal investigators, consultants, commercial collaborators, service providers or other vendors, or those of our affiliates, are alleged or found to be in violation of any such regulatory standards or requirements, or become subject to a corporate integrity agreement or similar agreement and curtailment of our operations, it could have a significant impact on our business and financial results, including the imposition of significant civil, criminal and administrative penalties, damages, monetary fines, suspension or delay in clinical trials, possible exclusion from participation in Medicare, Medicaid and other federal healthcare programs, FDA debarment, contractual damages, reputational harm, diminished profits and future earnings, additional reporting requirements and oversight, any of which could adversely affect our ability to operate our business and our results of operations.
International expansion of our business exposes us to business, regulatory, political, operational, financial and economic risks associated with doing business in various jurisdictions globally.
Our business strategy incorporates international expansion, including establishing and maintaining operations and certain key functions in various jurisdictions around the world and establishing and maintaining relationships with distributors and manufacturers globally. Doing business internationally involves a number of risks, including:
multiple, conflicting and changing laws and regulations such as tax laws, export and import restrictions, employment laws, anti-bribery and anti-corruption laws, regulatory requirements and other governmental approvals, permits and licenses;
failure by us or our distributors to obtain appropriate licenses or regulatory approvals for the sale or use of our product candidates, if approved, in various countries;
difficulties in managing foreign operations;
complexities associated with managing multiple payer-reimbursement regimes or self-pay systems;
financial risks, such as longer payment cycles, difficulty enforcing contracts and collecting accounts receivable and exposure to foreign currency exchange rate fluctuations;
reduced protection for intellectual property rights;
reduced protection of contractual rights in the event of bankruptcy or insolvency of the other contracting party;
natural disasters, political and economic instability, including wars, terrorism and political unrest, outbreak of disease, boycotts, curtailment of trade and other business restrictions;

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failure to comply with foreign laws, regulations, standards and regulatory guidance governing the collection, use, disclosure, retention, security and transfer of personal data, including the European Union General Data Privacy Regulation ("GDPR"), which introduces strict requirements for processing personal data of individuals within the European Union; and
failure to comply with the United Kingdom Bribery Act 2010 ("U.K. Bribery Act") and similar anti-bribery and anti-corruption laws in other jurisdictions, and the Foreign Corrupt Practices Act, including its books and records provisions and its anti-bribery provisions, including by failing to maintain accurate information and control over sales and distributors’ activities.

Any of these risks, if encountered, could significantly harm our future international expansion and operations and, consequently, negatively impact our financial condition, results of operations and cash flows.
Our business and operations would suffer in the event of system failures, security breaches or cyber-attacks.
Our computer systems, as well as those of various third parties on which we rely, including those of RSL and its affiliates and our CROs and other contractors, consultants, and law and accounting firms, may sustain damage from computer viruses, unauthorized access, data breaches, phishing attacks, cybercriminals, natural disasters, terrorism, war and telecommunication and electrical failures. We rely on our third-party providers to implement effective security measures and identify and correct for any such failures, deficiencies or breaches. The risk of a security breach or disruption, particularly through cyber-attacks or cyber intrusion, including by computer hackers, foreign governments, and cyber terrorists, has generally increased as the number, intensity and sophistication of attempted attacks and intrusions from around the world have increased. We have experienced phishing attacks in the past, which have not had a material impact on our operations, however, we may in the future experience material system failures or security breaches that could cause interruptions in our operations, or result in a material disruption of our drug development programs. For example, the loss of nonclinical or clinical trial data from completed, ongoing or planned trials could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. To the extent that any disruption or security breach were to result in a loss of or damage to our data or applications, or inappropriate disclosure of personal, confidential or proprietary information, we could incur liability and the further development of our product candidates could be delayed.
The failure to successfully implement a new enterprise resource planning system, or maintain our current system, could adversely impact our business and results of operations.
We may implement a company-wide enterprise resource planning ("ERP") system to upgrade certain existing business, operational, and financial processes, upon which we rely. However, until such time, we will be reliant on financial systems supported by RSI and RSG. ERP implementations are complex and time-consuming projects that require transformations of business and financial processes in order to reap the benefits of the ERP system; any such transformation involves risk inherent in the conversion to a new computer system, including loss of information and potential disruption to normal operations. Additionally, if the ERP system is not effectively implemented as planned, or the system does not operate as intended, the effectiveness of our internal controls over financial reporting could be adversely affected or our ability to assess those controls adequately could be delayed. Significant delays in documenting, reviewing and testing our internal control could cause us to fail to comply with our SEC reporting obligations related to our management's assessment of our internal control over financial reporting. In addition, if we experience interruptions in service or operational difficulties and are unable to effectively manage our business during or following the implementation of the ERP system, our business and results of operations could be harmed.
Potential product liability lawsuits against us could cause us to incur substantial liabilities and limit commercialization of any products that we may develop.
The use of our product candidates in clinical trials and the sale of any products for which we obtain marketing approval exposes us to the risk of product liability claims. Product liability claims might be brought against us by consumers, health care providers, pharmaceutical companies or others selling or otherwise coming into contact with our products. On occasion, large judgments have been awarded in class action lawsuits based on drugs that had unanticipated adverse effects. If we are not successful in defending ourselves against product liability claims, we could incur substantial liability and costs. In addition, regardless of merit or eventual outcome, product liability claims may result in:
impairment of our business reputation and significant negative media attention;
withdrawal of participants from our clinical trials;
significant costs to defend related litigation;
distraction of management’s attention from our primary business;
substantial monetary awards to patients or other claimants;
inability to commercialize our product candidates or any future product candidate;

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product recalls, withdrawals or labeling, marketing or promotional restrictions;
decreased demand for our product candidates or any future product candidate, if approved for commercial sale; and
loss of revenue.

The product liability insurance we currently carry, and any additional product liability insurance coverage we acquire in the future, may not be sufficient to reimburse us for any expenses or losses we may suffer. Moreover, insurance coverage is becoming increasingly expensive, and in the future, we may not be able to maintain insurance coverage at a reasonable cost or in sufficient amounts to protect us against losses due to liability. A successful product liability claim or series of claims brought against us could cause our share price to decline and, if judgments exceed our insurance coverage, could adversely affect our results of operations and business, including preventing or limiting the commercialization of any product candidates we develop.
If we fail to comply with applicable U.S. and foreign privacy and data protection laws and regulations, we may be subject to liabilities that adversely affect our business, operations and financial performance.
Our information security systems are subject to laws and regulations requiring that we take measures to protect the privacy and security of certain information we gather and use in our business. For example, HIPAA, and its implementing regulations impose, among other requirements, certain regulatory and contractual requirements regarding the privacy and security of personal health information. In addition to HIPAA, numerous other federal and state laws, including, without limitation, state security breach notification laws, state health information privacy laws and federal and state consumer protection laws, govern the collection, use, and storage of personal information.
We may also be subject to or affected by foreign laws and regulation, including regulatory guidance, governing the collection, use, disclosure, security, transfer and storage of personal data, such as information that we collect about patients and healthcare providers in connection with clinical trials and our other operations in the U.S. and abroad. The global legislative and regulatory landscape for privacy and data protection continues to evolve, and implementation standards and enforcement practices are likely to remain uncertain for the foreseeable future. This evolution may create uncertainty in our business, result in liability or impose additional costs on us. The cost of compliance with these laws, regulations and standards is high and is likely to increase in the future.
It is possible that each of these privacy laws may be interpreted and applied in a manner that is inconsistent with our practices. Any failure or perceived failure by us to comply with federal, state, or foreign laws or self-regulatory standards could result in negative publicity, diversion of management time and effort and proceedings against us by governmental entities or others. In many jurisdictions, enforcement actions and consequences for noncompliance are rising. As we continue to expand into other foreign countries and jurisdictions, we may be subject to additional laws and regulations that may affect how we conduct business.


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Risks Related to Clinical Development, Regulatory Approval and Commercialization
Clinical trials are very expensive, time-consuming, difficult to design and implement and involve an uncertain outcome.
Our product candidates are still in development and will require extensive clinical testing before we are prepared to submit an application for marketing approval to regulatory authorities. We cannot predict with any certainty if or when we might submit any such application for regulatory approval for our product candidates or whether any such application will be approved by the applicable regulatory authority in our target markets. Human clinical trials are very expensive and difficult to design and implement, in part because they are subject to rigorous regulatory requirements. For instance, regulatory authorities may not agree with our proposed endpoints for any clinical trials of our product candidates, which may delay the commencement of our clinical trials. The clinical trial process is also time-consuming. We estimate that clinical trials of our product candidates will take at least several years to complete. Furthermore, failure can occur at any stage of the trials, and we could encounter problems that cause us to abandon or repeat clinical trials. Product candidates in later stages of clinical trials may fail to show the desired safety and efficacy traits despite having progressed through nonclinical studies and initial clinical trials, and the results of smaller nonclinical or early clinical trials therefore may not be predictive of the results of large scale or later-stage clinical programs. For example, in January 2018, we announced that intepirdine did not meet its primary efficacy endpoints in the Phase 2B HEADWAY and pilot Phase 2 Gait and Balance studies. In light of the data from these studies, we discontinued our intepirdine program. Likewise, there can be no assurance that the results of studies conducted by collaborators or other third parties will be viewed favorably or are indicative of our own future study results. A number of companies in the biopharmaceutical industry, and especially in the neurology field, have suffered significant setbacks in advanced clinical trials due to lack of efficacy or adverse safety profiles, notwithstanding promising results in earlier trials, and in the regulatory approval process. For example, in August 2017, Acorda Therapeutics received a refusal to file letter from the FDA regarding its NDA for INBRIJA, an investigational treatment for symptoms of OFF episodes in patients with Parkinson’s disease taking a carbidopa/levodopa regimen.
Our gene therapy product candidates, AXO-Lenti-PD and AXO-AAV-OPMD, are in early stages of development. The outcome of nonclinical testing and early clinical trials may not be predictive of the success of later stage clinical trials, interim results of a clinical trial do not necessarily predict final results and results from one completed clinical trial may not be replicated in a subsequent clinical trial with a similar study design. The Phase 1/2 clinical trial of ProSavin conducted by Oxford BioMedica was conducted with a small patient population and was not blinded or placebo-controlled, making it difficult to predict whether the favorable results that we observed in such trials will be repeated in larger and more advanced clinical trials.
The commencement and completion of clinical trials may be delayed by several factors, including:
failure to obtain regulatory approval to commence a trial;
unforeseen safety issues;
determination of dosing issues;
lack of effectiveness during clinical trials;
inability to reach agreement on acceptable terms with prospective CROs and clinical trial sites;
slower than expected rates of patient recruitment or failure to recruit suitable patients to participate in a trial;
changes in or modifications to clinical trial design;
failure to manufacture or obtain supply of sufficient quantities of a drug candidate or placebo or failure to obtain sufficient quantities of concomitant medication for use in clinical trials;
inability or unwillingness of medical investigators to follow our clinical and other applicable protocols;
inability to monitor patients adequately during or after treatment;
failure to establish sufficient number of clinical trial sites; or
clinical sites or others deviating from trial protocol, inappropriately unblinding results, or dropping out of a trial.

Further, by way of example, we, a regulatory agency or an institutional review board ("IRB") at a clinical trial site may suspend our clinical trials at any time if it appears that we or our collaborators are failing to conduct a trial in accordance with regulatory requirements, including the FDA’s current Good Clinical Practice ("GCP") regulations, that we are exposing participants to unacceptable health risks, or if the FDA finds deficiencies in our IND submissions or the conduct of these trials. Therefore, we cannot predict with any certainty the schedule for commencement and completion of clinical trials. If we experience delays in the commencement or completion of our clinical trials, or if we terminate a clinical trial prior to completion, the commercial prospects of our product candidates could be harmed, and our ability to generate revenues may be delayed. In addition, any delays in our clinical trials could increase our costs, cause a drop in our share price, slow down the approval process and jeopardize our ability to commence product sales and generate revenues. Any of these occurrences may harm our business, financial condition and results of operations.

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Moreover, principal investigators for our clinical trials may serve as scientific advisors or consultants to us from time to time and receive compensation in connection with such services. Under certain circumstances, we may be required to report some of these relationships to the applicable regulatory agency, which may conclude that a financial relationship between us and a principal investigator has created a conflict of interest or otherwise affected interpretation of the study. The applicable regulatory agency may therefore question the integrity of the data generated at the applicable clinical trial site and the utility of the clinical trial itself may be jeopardized. This could result in a delay in approval, or rejection, of our marketing applications by the applicable regulatory agency and may ultimately lead to the denial of marketing approval of one or more of our product candidates.
In addition, we acquired worldwide rights to our product candidates and were not involved in their development prior to such acquisitions. Any difficulties we experience in transitioning and integrating such product candidates into our operations may result in delays in clinical trials as well as problems in our development efforts and regulatory filings, particularly if we do not receive all of the necessary drug products, information, reports and data from third parties in a timely manner. More particularly, we have had no involvement with or control over the nonclinical and clinical development of our product candidates prior to acquiring the rights to them. We are dependent on predecessors including Oxford BioMedica, Benitec and Arena, having conducted such research and development in accordance with the applicable protocols, legal, regulatory and scientific standards, having accurately reported the results of all clinical trials and other research conducted prior to our acquisition of the product candidates, having correctly collected and interpreted the data from these trials and other research and having supplied us with complete information, data sets and reports required to adequately demonstrate the results reported through the date of our acquisition of these assets. In addition, we have limited data regarding the safety, tolerability and efficacy of AXO-Lenti-PD for the treatment of Parkinson’s disease and AXO-AAV-OPMD for the treatment of OPMD, and we have not previously conducted development activities for a biological product candidate. Problems related to predecessors including Oxford BioMedica, Benitec and Arena as well as our limited available data for AXO-Lenti-PD in the treatment of Parkinson’s disease and AXO-AAV-OPMD in the treatment of OPMD could result in increased costs and delays in the development of our product candidates, which could adversely affect our ability to generate future revenues.
Enrollment and retention of patients in clinical trials is an expensive and time-consuming process and could be made more difficult or rendered impossible by multiple factors outside our control.
We may encounter delays in enrolling, or be unable to enroll, a sufficient number of patients to complete any of our clinical trials, and even once enrolled we may be unable to retain a sufficient number of patients to complete any of our trials. Patient enrollment and retention in clinical trials depends on many factors, including the size of the patient population, the nature of the trial protocol, the effectiveness of our patient recruitment efforts, the existing body of safety and efficacy data with respect to the study drug, the perceived risks and benefits of gene therapy approaches for the treatment of neurological diseases, the number and nature of competing treatments and ongoing clinical trials of competing drugs for the same indication, including more traditional approaches for the treatment of Parkinson’s disease or OPMD, perceived risk of the delivery procedure, patients with pre-existing conditions that preclude their participation in any trial, the proximity of patients to clinical sites and the eligibility criteria for the study. Furthermore, the negative results we have reported in clinical trials to date and any other negative results we may report in clinical trials of any of our product candidates in the future may make it difficult or impossible to recruit and retain patients in other clinical trials of those product candidates. Similarly, negative results reported by our competitors about their drug candidates may negatively affect patient recruitment in our clinical trials. Delays or failures in planned patient enrollment or retention may result in increased costs, program delays or both, which could have a harmful effect on our ability to develop our product candidates or could render further development impossible. In addition, we expect to rely on CROs and clinical trial sites to ensure proper and timely conduct of our future clinical trials and, while we intend to enter into agreements governing their services, we will be limited in our ability to compel their actual performance.
We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.
Drug development, particularly in the gene therapy field, is highly competitive and subject to rapid and significant technological advancements. As a significant unmet medical need exists in the neurology field, particularly for the treatment of Parkinson's disease and Alzheimer’s disease, there are several large and small pharmaceutical companies focused on delivering therapeutics for the treatment of these diseases. Further, it is likely that additional drugs will become available in the future for the treatment of our target indications.

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We consider our most direct competitor with respect to AXO-Lenti-PD to be Voyager Therapeutics, which is developing VY-AADC, a gene therapy product candidate for the treatment of advanced Parkinson’s disease. VY-AADC delivers the AADC gene, one of the three genes contained in AXO-Lenti-PD, via an Adeno-Associated Virus ("AAV virus-based vector"). Voyager anticipates entering a Phase 3 study in mid-2018. Agilis Biotherapeutics, to be acquired by PTC Therapeutics, is developing AGIL-AADC, another AAV virus-based vector gene therapy that delivers the AADC gene, for the treatment of AADC deficiency, a rare disorder that involves loss of AADC gene function. In addition, DBS (Deep Brain Stimulation) is approved for treating Parkinson’s disease and is marketed by multiple device manufacturers, including Medtronic, Abbott and Boston Scientific. DBS treatment involves permanent placement of hardware in the brain via stereotactic neurosurgery and may require follow-up adjustments or even invasive device replacements. Another surgical approach is Abbvie’s Duopa which is delivered via a port implanted in the abdominal wall. Further efforts are also underway to develop new improved formulations of L-dopa, including Acorda’s Inbrija and Mitsubishi Tanabe’s ND0612. Adjunct therapies are also being developed or have recently been approved to supplement L-dopa therapy, including Sunovion’s sublingual apopmorphine and Adamas Pharmaceuticals’ GoCovri. Several companies are also trying to develop other disease modifying therapies that could prevent the progression of Parkinson’s disease. Examples of these early stage efforts include Denali Therapeutics’ LRRK2 inhibitors and anti-alpha synuclein antibodies from Prothena/Roche and Biogen, as well as Prevail Therapeutics’ pipeline of AAV-based therapeutics targeting lysosomal dysfunction.
We consider our most direct competitor with respect to AXO-AAV-OPMD to be BioBlast Pharma, which is developing trehalose, an investigational drug product thought to stabilize mutant proteins and increase autophagy, for the treatment of OPMD and other indications. In addition, there are surgical approaches to address the symptoms of OPMD, such as cricopharyngeal myotomy.
We consider our most direct competitor with respect to nelotanserin to be Acadia Pharmaceuticals, which is marketing and developing pimavanserin, a 5-HT2A receptor inverse agonist that received FDA approval in April 2016 for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis. We believe the FDA approval of pimavanserin adds further validation to the therapeutic relevance of 5-HT2A as a potential target for the treatment of visual hallucinations.
Many of our existing or potential competitors have substantially greater financial, technical and human resources than we do and significantly greater experience in the discovery and development of product candidates, as well as in obtaining regulatory approvals of those product candidates in the United States and in foreign countries. Our current and potential future competitors also have significantly more experience commercializing drugs, particularly gene therapy and other biological products, that have been approved for marketing. Mergers and acquisitions in the pharmaceutical and biotechnology industries could result in even more resources being concentrated among a small number of our competitors.
We will face competition from other drugs or from other non-drug products currently approved or that will be approved in the future in the neurology field, including for the treatment of Parkinson’s disease. Therefore, our ability to compete successfully will depend largely on our ability to:
develop and commercialize drugs that are superior to other products in the market;
demonstrate through our clinical trials that our product candidates are differentiated from existing and future therapies;
attract qualified scientific, product development and commercial personnel;
obtain patent or other proprietary protection for our medicines;
obtain required regulatory approvals;
obtain coverage and adequate reimbursement from, and negotiate competitive pricing with, third-party payers; and
successfully collaborate with pharmaceutical companies in the discovery, development and commercialization of new medicines.

The availability of our competitors’ products could limit the demand, and the price we are able to charge, for any product candidate we develop. The inability to compete with existing or subsequently introduced drugs would have an adverse impact on our business, financial condition and prospects.
Established pharmaceutical companies may invest heavily to accelerate discovery and development of novel compounds or to in-license novel compounds that could make our product candidates less competitive. In addition, any new product that competes with an approved product must demonstrate compelling advantages in efficacy, convenience, tolerability and safety in order to overcome price competition and to be commercially successful. Accordingly, our competitors may succeed in obtaining patent protection, discovering, developing, receiving regulatory and marketing approval for or commercializing drugs before we do, which would have an adverse impact on our business and results of operations.

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If we are not able to obtain required regulatory approvals, we will not be able to commercialize our product candidates, and our ability to generate revenue will be materially impaired.
The activities associated with the development and commercialization of our product candidates, including their design, research, testing, manufacture, safety, efficacy, recordkeeping, labeling, packaging, storage, approval, advertising, promotion, sale and distribution, are subject to comprehensive regulation by the FDA and other regulatory agencies in the United States and by the EMA, the PMDA and similar regulatory authorities outside the United States. Failure to obtain marketing approval for our product candidates will prevent us from commercializing them.
We have not received approval from regulatory authorities to market any product candidate in any jurisdiction, and we will need to complete pivotal clinical trials successfully for our product candidates before we can submit any application for regulatory approval. It is possible that our product candidates in the future will never obtain the appropriate regulatory approvals necessary for us to commence product sales.
We expect to rely on third-party CROs and consultants to assist us in filing and supporting the applications necessary to gain marketing approvals. Securing marketing approval requires the submission of extensive nonclinical and clinical data and supporting information for our product candidates to regulatory authorities for each therapeutic indication to establish safety and efficacy of the product candidate for that indication. Securing marketing approval also requires the submission of information about the product manufacturing process to, and inspection of manufacturing facilities by, the regulatory authorities. Delays or errors in the submission of applications for marketing approval or issues, including those related to gathering the appropriate data and the inspection process, may ultimately delay or affect our ability to obtain regulatory approval, commercialize our product candidates and generate product revenues.
Our product candidates may cause adverse effects or have other properties that could delay or prevent their regulatory approval or limit the scope of any approved label or market acceptance.
Adverse events caused by our product candidates or that of adjuncts could cause us, other reviewing entities, clinical trial sites or regulatory authorities to interrupt, delay or halt clinical trials and could result in the denial of regulatory approval. If an unacceptable frequency or severity of adverse events are reported in our clinical trials for our product candidates or any future product candidates, our ability to obtain regulatory approval for such product candidates may be negatively impacted. .The laws and regulations governing controlled substances could limit commercialization of our product candidates, and failure to comply with those laws and regulations could also result in adverse regulatory, legal, and operational consequences.
In particular, there have been several significant adverse side effects in gene therapy treatments in the past, including reported cases of leukemia in trials using earlier generation viral vectors. Gene therapy is still a relatively new approach to disease treatment and additional adverse side effects could develop. Possible adverse side effects that could occur with treatment with gene therapy products include an immunologic reaction early after administration which could substantially limit the effectiveness of the treatment or represent safety risks for patients. Another traditional safety concern for gene therapies using viral vectors has been the possibility of insertional mutagenesis by the vectors, leading to malignant transformation of transduced cells. Additionally, in previous clinical trials involving AAV vectors for gene therapy, some subjects experienced the development of a positive ELISPOT test associated with T-cell responses, which is of unclear clinical translatability.
There is also the potential risk of delayed adverse events following exposure to gene therapy products due to persistent biologic activity of the genetic material or other components of products used to carry the genetic material. Possible adverse side effects that may occur with treatment with gene therapy products include an immunologic reaction early after administration that could substantially limit the effectiveness of the treatment or represent safety risks for patients. Many times, side effects are only detectable after investigational products are tested in larger scale, pivotal clinical trials or, in some cases, after they are made available to patients on a commercial scale after approval.
In addition to side effects that may be caused by AXO-Lenti-PD, AXO-AAV-OPMD and our other product candidates, the administration process or related procedures also can cause adverse side effects. For example, integration of AAV DNA into the host cell's genome has been reported to occur. Further, our AAV delivery system for AXO-AAV-OPMD has not been validated in human clinical trials previously, and if such delivery system does not meet the safety criteria or cannot provide the desired efficacy results, then we may be forced to suspend or terminate our development of AXO-AAV-OPMD.
If additional clinical experience indicates that AXO-Lenti-PD, AXO-AAV-OPMD or any other product candidate has side effects or causes serious or life-threatening side effects, the development of the product candidate may fail or be delayed, or, if the product candidate has received regulatory approval, such approval may be revoked or limited.
Furthermore, if any of our products are approved and then cause serious or unexpected side effects, a number of potentially significant negative consequences could result, including:
regulatory authorities may withdraw their approval of the product or require a REMS to impose restrictions on its distribution or other risk management measures;

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regulatory authorities may require the addition of labeling statements, such as warnings or contraindications;
we may be required to change the way the product is administered or to conduct additional clinical trials;
we could be sued and held liable for harm caused to patients;
we could elect to discontinue the sale of our product; and
our reputation may suffer.

Any of these events could prevent us from achieving or maintaining market acceptance of the affected product candidate and could substantially increase the costs of commercializing our product candidates.
Our lentiviral-based gene therapy product candidate AXO-Lenti-PD and our AAV-based gene therapy product candidate AXO-AAV-OPMD are based on novel gene transfer technology, which makes it difficult to predict the time and cost of product candidate development and of subsequently obtaining regulatory approval.
We expect to concentrate our research and development efforts in gene therapy on AXO-Lenti-PD and AXO-AAV-OPMD. The use of gene therapy in the treatment of Parkinson’s disease and OPMD is novel. There can be no assurance that we will not experience problems or delays in developing new product candidates and that such problems or delays will not cause unanticipated costs, or that any such development problems can be solved. We may also experience delays in developing a sustainable, reproducible and scalable manufacturing process or transferring that process for our gene therapy product candidates from their current manufacturers, which may prevent us from completing our clinical studies or commercializing our products on a timely or profitable basis, if at all.
In addition, the clinical trial requirements of the FDA and other foreign regulatory authorities and the criteria these regulators use to determine the safety and efficacy of a product candidate vary substantially according to the type, complexity, novelty and intended use and market of such product candidates. The regulatory approval process for novel product candidates such as ours can be more expensive and take longer than for other, better known or more extensively studied product candidates. To date, only a limited number of gene therapies have received marketing authorization from the FDA or foreign regulatory authorities. Until August 2017, the FDA had never approved a gene therapy product. Since that time, it has only approved a small number of product candidates, including Kymriah by Novartis International AG, for pediatric and young adult patients with a form of acute lymphoblastic leukemia, Yescarta by Kite Pharma, Inc., for adult patients with certain forms of non-Hodgkin lymphoma, and Luxturna by Spark Therapeutics, Inc., for patients with an inherited form of vision loss. It is difficult to determine how long it will take or how much it will cost to obtain regulatory approvals for our product candidates in either the United States, or other major markets or how long it will take to commercialize our product candidates, if any are approved. Approvals by foreign regulatory authorities may not be indicative of what the FDA may require for approval, and vice versa.
Regulatory requirements governing gene therapy products have changed frequently and may continue to change in the future. The FDA has established the Office of Tissues and Advanced Therapies within its Center for Biologics Evaluation and Research ("CBER") to consolidate the review of gene therapy and related products, and has established the Cellular, Tissue and Gene Therapies Advisory Committee to advise the CBER in its review. Gene therapy clinical trials conducted at institutions that receive funding for recombinant DNA research from the NIH, also are potentially subject to review by the NIH Office of Science Policy’s Recombinant DNA Advisory Committee (the "RAC") in limited circumstances. Although the FDA decides whether individual gene therapy protocols may proceed, the RAC public review process, if undertaken, can delay the initiation of a clinical trial, even if the FDA has reviewed the trial design and details and authorized its initiation. Conversely, the FDA can put an investigational new drug application ("IND") on clinical hold even if the RAC has provided a favorable review or an exemption from in-depth, public review. If we were to engage an NIH-funded institution, to conduct a clinical trial, that institution’s institutional biosafety committee ("IBC") as well as its institutional review board ("IRB") would need to review the proposed clinical trial to assess the safety of the trial and may determine that RAC review is needed. In addition, adverse developments in clinical trials of gene therapy products conducted by others may cause the FDA or other oversight bodies to change the requirements for approval of any of our product candidates. Similarly, foreign regulatory authorities may issue new guidelines concerning the development and marketing authorization for gene therapy medicinal products and require that we comply with these new guidelines.
The FDA, NIH and the European Medicines Agency ("EMA") have each expressed interest in further regulating biotechnology, including gene therapy and genetic testing. For example, the EMA advocates a risk-based approach to the development of a gene therapy product. Agencies at both the federal and state level in the United States, as well as the U.S. Congressional committees and other governments or governing agencies, have also expressed interest in further regulating the biotechnology industry. Such action may delay or prevent commercialization of some or all of our product candidates.

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These regulatory review committees and advisory groups and any new guidelines they promulgate may lengthen the regulatory review process, require us to perform additional studies, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of these product candidates or lead to significant post-approval limitations or restrictions. As we advance our product candidates, we will be required to consult with these regulatory and advisory groups and comply with applicable guidelines. If we fail to do so, we may be required to delay or discontinue development of certain of our product candidates. These additional processes may result in a review and approval process that is longer than we otherwise would have expected. Delay or failure to obtain, or unexpected costs in obtaining, the regulatory approval necessary to bring a potential product to market could decrease our ability to generate sufficient product revenue, and our business, financial condition, results of operations and prospects would be materially and adversely affected.
The FDA recently announced that it is preparing to release a series of draft guidance regarding potential accelerated approval endpoints for certain gene therapy products and other clinical and manufacturing issues related to gene therapy products. We cannot be certain when such guidance will be issued or whether any such guidance will address accelerated approval endpoints or other clinical or manufacturing issues that will be relevant to or have an impact on our gene therapy candidates or the duration or expense of any applicable regulatory review processes.
Even if we obtain FDA approval for our product candidates in the United States, we may never obtain approval for or commercialize them in any other jurisdiction, which would limit our ability to realize their full market potential.
In order to market any products in any particular jurisdiction, we must establish and comply with numerous and varying regulatory requirements on a country-by-country basis regarding safety and efficacy. Approval by the FDA in the United States does not ensure approval by regulatory authorities in other countries or jurisdictions. In addition, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries, and regulatory approval in one country does not guarantee regulatory approval in any other country. Approval processes vary among countries and can involve additional product testing and validation and additional administrative review periods. Seeking foreign regulatory approval could result in difficulties and costs for us and require additional nonclinical studies or clinical trials which could be costly and time consuming. Regulatory requirements can vary widely from country to country and could delay or prevent the introduction of our products in those countries. We do not have any product candidates approved for sale in any jurisdiction, including in international markets, and we do not have experience in obtaining regulatory approval in international markets. If we fail to comply with regulatory requirements in international markets or to obtain and maintain required approvals, or if regulatory approvals in international markets are delayed, our target market will be reduced and our ability to realize the full market potential of any product we develop will be unrealized.
Even if we obtain regulatory approval for our product candidates, we will still face extensive regulatory requirements and our products may face future development and regulatory difficulties.
Any product candidate for which we obtain marketing approval, along with the manufacturing processes, post-approval clinical data, labeling, packaging, distribution, adverse event reporting, storage, recordkeeping, export, import, advertising and promotional activities for such product, among other things, will be subject to extensive and ongoing requirements of and review by the FDA, the EMA, the PMDA and other comparable foreign regulatory authorities. These requirements include submissions of safety and other post-marketing information and reports, establishment registration and drug listing requirements, continued compliance with current good manufacturing practices, or cGMP, requirements relating to manufacturing, quality control, quality assurance and corresponding maintenance of records and documents, requirements regarding the distribution of samples to physicians and recordkeeping and current GCP requirements for any clinical trials that we conduct post-approval. Even if marketing approval of a product candidate is granted, the approval may be subject to limitations on the indicated uses for which the product may be marketed or to the conditions of approval, including any requirement to implement a REMS. If any of our product candidates receives marketing approval, the accompanying labels for such products may limit the approved use of the drug, which could limit sales.

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Regulatory authorities may also impose requirements for costly post-marketing studies or clinical trials and surveillance to monitor the safety or efficacy of the product. For example, the holder of an approved NDA or BLA is obligated to monitor and report adverse events and any failure of a product to meet the specifications in the NDA or BLA. The FDA typically advises that patients treated with gene therapy undergo follow-up observations for potential adverse events for a 15-year period. The holder of an approved NDA or BLA also must submit new or supplemental applications and obtain FDA approval for certain changes to the approved product, product labeling or manufacturing process. These authorities closely regulate the post-approval marketing and promotion of drugs to ensure drugs are marketed only for the approved indications and in accordance with the provisions of the approved labeling. We will be subject to stringent restrictions on manufacturers’ communications regarding off-label use and if we do not market our products for their approved indications, we may be subject to enforcement action for off-label marketing.  Violations of the DCA or PHSA in the United States, and other comparable regulations in foreign jurisdictions, relating to the promotion of prescription drugs may lead to enforcement actions and investigations alleging violations of U.S. federal and state health care fraud and abuse laws, as well as state consumer protection laws and comparable laws in foreign jurisdictions.
In addition, later discovery of previously unknown adverse events or other problems with our products, manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may yield various results, including:
restrictions on manufacturing such products;
restrictions on the labeling or marketing of such products;
restrictions on product marketing, distribution or use;
requirements to conduct post-marketing studies or clinical trials;
warning or untitled letters;
withdrawal of the products from the market;
recall of products;
fines, restitution or disgorgement of profits or revenues;
suspension or withdrawal of marketing approvals;
refusal to permit the import or export of such products;
product seizure; or
injunctions or the imposition of civil or criminal penalties.

Government regulations may change, and additional government regulations may be enacted, either of which could prevent, limit or delay regulatory approval of our product candidates or any future product candidate. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval that we may have obtained.
Even if our product candidates receive marketing approval, they may fail to achieve market acceptance by physicians, patients, third-party payers or others in the medical community necessary for commercial success.
Even if our product candidates receive marketing approval, they may nonetheless fail to gain sufficient market acceptance by physicians, patients, third-party payers and others in the medical community, including due to the novelty of gene therapy products in general. If they do not achieve an adequate level of acceptance, we may not generate significant product revenues and become profitable. The degree of market acceptance for our product candidates, if approved for commercial sale, will depend on a number of factors, including but not limited to:
the efficacy and potential advantages compared to alternative treatments;
the effectiveness of sales and marketing efforts;
the cost of treatment in relation to alternative treatments, including any similar generic treatments;
our ability to offer our products for sale at competitive prices;
the convenience and ease of administration compared to alternative treatments;
the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;
the ethical, social and legal concerns about gene therapy;
the strength of marketing and distribution support;
the availability of third-party coverage and adequate reimbursement;
the prevalence and severity of any side effects; and
any restrictions on the use of our product together with other medications.

We expect sales of our product candidates, if approved, to generate substantially all of our product revenues for the foreseeable future. The failure of any of our product candidates, if approved, to find market acceptance would harm our business and could require us to seek additional financing.

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Our gene therapy approach for our gene therapy product candidates, AXO-Lenti-PD and AXO-AAV-OPMD, utilizes lentiviral and AAV vectors, respectively, derived from plasmids that encode viral proteins, which may be perceived as unsafe or may result in unforeseen adverse events. Negative public opinion and increased regulatory scrutiny of gene therapy and genetic research may damage public perception of our product candidates or adversely affect our ability to conduct our business or obtain regulatory approvals for our product candidates.
Gene therapy remains a novel technology, with only a limited number of gene therapy products approved to date. Public perception may be influenced by claims that gene therapy is unsafe, unethical or immoral, and gene therapy may not gain the acceptance of the public or the medical community. In particular, our success will depend upon the comfort level of physicians to prescribe our product candidates, including AXO-Lenti-PD (if approved) and AXO-AAV-OPMD (if approved), in lieu of, or in addition to, existing or standard treatments they are already familiar with and for which greater clinical data may be available. More restrictive government regulations or negative public opinion would have a negative effect on our business or financial condition and may delay or impair the development and commercialization of AXO-Lenti-PD or AXO-AAV-OPMD. Earlier gene therapy trials led to several well-publicized adverse events, including cases of leukemia and death seen in such trials using earlier generation vectors. For example, a public backlash developed against gene therapy following the death of a patient in 1999 during a gene therapy trial of research subjects with ornithine transcarbamylase ("OTC") deficiency, a rare disorder in which the liver lacks a functional copy of the OTC gene. The death of the trial subject was due to complications of adenovirus vector administration. In addition, there is the potential risk of delayed adverse events following exposure to gene therapy products due to persistent biological activity of the genetic material or other components of products used to carry the genetic material. Adverse events in our clinical studies, even if not ultimately attributable to our product candidates (such as the many adverse events that typically arise from the conditioning process), or adverse events in other lentiviral gene therapy trials, and the resulting publicity, could result in increased governmental regulation, unfavorable public perception, potential regulatory delays in the testing or approval of our potential product candidates, stricter labeling requirements for those product candidates that are approved and a decrease in demand for any such product candidates.
We may not be able to benefit from orphan drug designation for AXO-AAV-OPMD.
The FDA and European Commission granted AXO-AAV-OPMD orphan drug designation for the treatment of OPMD in 2018 and 2017, respectively. The designation of AXO-AAV-OPMD as an orphan drug does not guarantee that any regulatory agency will accelerate regulatory review of, or ultimately approve, that product candidate, nor does it limit the ability of any regulatory agency to grant orphan drug designation to product candidates of other companies that treat the same indications as our product candidate prior to our product candidate receiving exclusive marketing approval.
We may lose orphan drug exclusivity if the FDA or European Commission determines that the request for designation was materially defective or if we cannot assure sufficient quantity of the applicable drug to meet the needs of patients with OPMD.
Even if we maintain orphan drug exclusivity for AXO-AAV-OPMD or obtain orphan drug exclusivity for our other product candidate, the exclusivity may not effectively protect the product candidate from competition because regulatory authorities still may authorize different drugs for the same condition.
If we are unable to establish sales, marketing and distribution capabilities either on our own or in collaboration with third parties, we may not be successful in commercializing our product candidates, even if approved.
We do not have a full infrastructure for the sales, marketing or distribution of our product candidates should they be approved, and the cost of establishing and maintaining such an organization may exceed the cost-effectiveness of doing so. In order to market any product that may be approved, we must build our sales, distribution, marketing, managerial and other non-technical capabilities or make arrangements with third parties to perform these services and obtain requisite licenses. To achieve commercial success for any product for which we have obtained marketing approval, we will need a sales and marketing organization.
We plan to commercialize our product candidates in the United States, the European Union, Japan and other major markets. If our product candidates are approved for marketing, we may build a focused sales, distribution and marketing infrastructure to market them. There are significant expenses and risks involved with establishing our own sales, marketing and distribution capabilities, including our ability to hire, retain and appropriately incentivize qualified individuals, generate sufficient sales leads, provide adequate training to sales and marketing personnel, and effectively manage a geographically dispersed sales and marketing team. Any failure or delay in the development of our internal sales, marketing and distribution capabilities, and any failure to obtain and maintain the requisite licenses, could delay any product launch, which would adversely impact the commercialization of our product candidates. 
Factors that may inhibit our efforts to commercialize our products on our own include:
our inability to recruit, train and retain adequate numbers of effective sales and marketing personnel;
the inability of sales personnel to obtain access to physicians or attain adequate numbers of physicians to prescribe any drugs; and

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unforeseen costs and expenses associated with creating an independent sales and marketing organization.

If we are unable to build our own sales force or negotiate a collaborative relationship for the commercialization of our product candidates, we may be forced to delay the potential commercialization of such products or reduce the scope of our sales or marketing activities for our product candidates. If we elect to increase our expenditures to fund commercialization activities ourselves, we will need to obtain additional capital, which may not be available to us on acceptable terms, or at all. If we do not have sufficient funds, we will not be able to bring our product candidates to market or generate product revenue. We could enter into arrangements with collaborative partners or otherwise at an earlier stage than otherwise would be ideal and we may be required to relinquish rights to one or more of our product candidates or otherwise agree to terms unfavorable to us, any of which may have an adverse effect on our business, operating results and prospects.
If we obtain approval to commercialize any products outside of the United States, a variety of risks associated with international operations could materially adversely affect our business.
If our product candidates are approved for commercialization, we may enter into agreements with third parties to market them in certain jurisdictions outside the United States. We expect that we will be subject to additional risks related to international operations or entering into international business relationships, including:
different regulatory requirements for drug approvals and rules governing drug commercialization in foreign countries;
reduced protection for intellectual property rights;
unexpected changes in tariffs, trade barriers and regulatory requirements;
economic weakness, including inflation, or political instability in particular foreign economies and markets;
compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
foreign reimbursement, pricing and insurance regimes;
foreign taxes;
foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, and other obligations incident to doing business in another country;
workforce uncertainty in countries where labor unrest is more common than in the United States;
potential noncompliance with the U.S. Foreign Corrupt Practices Act, the U.K. Bribery Act and similar anti-bribery and anti-corruption laws in other jurisdictions;
production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and
business interruptions resulting from geopolitical actions, including war and terrorism, or natural disasters including earthquakes, typhoons, floods and fires.

Our current and future relationships with investigators, health care professionals, consultants, third-party payers, and customers will be subject to applicable healthcare regulatory laws, which could expose us to penalties.
Our business operations and current and future arrangements with investigators, healthcare professionals, consultants, third-party payers and customers may expose us to broadly applicable fraud and abuse and other healthcare laws and regulations. These laws may regulate the business or financial arrangements and relationships through which we conduct our operations, including how we research, market, sell and distribute our products for which we obtain marketing approval. Such laws include:
the federal Anti-Kickback Statute prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, lease, order or recommendation of, any good, facility, item or service, for which payment may be made, in whole or in part, under a federal healthcare program such as Medicare and Medicaid. A person or entity does not need to have actual knowledge of the federal Anti-Kickback Statute or specific intent to violate it to have committed a violation; in addition, the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the civil False Claims Act;
the federal false claims laws, including the civil False Claims Act, impose criminal and civil penalties, including through civil whistleblower or qui tam actions, against individuals or entities for knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent, knowingly making, using or causing to be made or used, a false record or statement material to a false or fraudulent claim, or knowingly making, or causing to be made, a false statement to avoid, decrease or conceal an obligation to pay money to the federal government;

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the federal Health Insurance Portability and Accountability Act of 1996 ("HIPAA") imposes criminal and civil liability for, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program or making false or fraudulent statements relating to healthcare matters. Similar to the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it to have committed a violation;
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act and its implementing regulations, also imposes obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information on health plans, health care clearing houses, and most providers and their business associates, defined as independent contractors or agents of covered entities that create, receive or obtain protected health information in connection with providing a service for or on behalf of a covered entity;
a number of federal, state and foreign laws, regulations, guidance and standards that impose requirements regarding the protection of health or other personal data that are applicable to or affect our operations;
the federal Physician Payments Sunshine Act, which requires certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to the government information related to payments or other "transfers of value" made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors) and teaching hospitals, and requires applicable manufacturers and group purchasing organizations to report annually to the government ownership and investment interests held by the physicians described above and their immediate family members and payments or other "transfers of value" to such physician owners (covered manufacturers are required to submit reports to the government by the 90th day of each calendar year); and
analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, may apply to our business practices, including but not limited to, research, distribution, sales, and marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payers, including private insurers, or otherwise restrict payments that may be made to healthcare providers and other potential referral sources; and state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures, as well as state and local laws that require the registration of pharmaceutical sales representatives; and state and foreign laws governing the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.

Efforts to ensure that our current and future business arrangements with third parties will comply with applicable healthcare laws and regulations will involve substantial costs. It is possible that governmental authorities will conclude that our business practices do not comply with current or future statutes, regulations, agency guidance or case law involving applicable healthcare laws. If our operations are found to be in violation of any of these or any other health regulatory laws that may apply to us, we may be subject to significant penalties, including the imposition of significant civil, criminal and administrative penalties, damages, monetary fines, disgorgement, individual imprisonment, possible exclusion from participation in Medicare, Medicaid and other federal healthcare programs, contractual damages, reputational harm, diminished profits and future earnings, additional reporting requirements and oversight if we become subject to a corporate integrity agreement or similar agreement, and curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our results of operations. Even the mere issuance of a subpoena or the fact of an investigation alone, regardless of the merit, may result in negative publicity, a drop in our share price, and other harm to our business, financial condition and results of operations.
Defending against any such actions can be costly, time-consuming and may require significant financial and personnel resources. Therefore, even if we are successful in defending against any such actions that may be brought against us, our business may be impaired.
Recently enacted and future legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates and affect the prices we may obtain.
In the United States and some foreign jurisdictions, there have been a number of legislative and regulatory changes and proposed changes regarding the healthcare system that could, among other things, prevent or delay marketing approval of our product candidates, restrict or regulate post-approval activities and affect our ability to profitably sell any products for which we obtain marketing approval.

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For example, in March 2010, the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act, collectively the Affordable Care Act, was enacted to broaden access to health insurance, reduce or constrain the growth of healthcare spending, enhance remedies against fraud and abuse, add new transparency requirements for health care and health insurance industries, impose new taxes and fees on the health industry and impose additional health policy reforms. The law has continued the downward pressure on pharmaceutical pricing, especially under the Medicare program, and increased the industry’s regulatory burdens and operating costs. Among the provisions of the Affordable Care Act of importance to our potential drug candidates are the following:
an annual, nondeductible fee payable by any entity that manufactures or imports specified branded prescription drugs and biologic agents;
an increase in the statutory minimum rebates a manufacturer must pay under the Medicaid Drug Rebate Program;
a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected;
a new Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 50% (and 70% commencing January 1, 2019) point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries under their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D;
extension of manufacturers’ Medicaid rebate liability to individuals enrolled in Medicaid managed care organizations;
expansion of eligibility criteria for Medicaid programs in certain states;
expansion of the entities eligible for discounts under the Public Health Service pharmaceutical pricing program;
a new requirement to annually report drug samples that manufacturers and distributors provide to physicians; and
a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research.

We cannot predict the full impact of the Affordable Care Act on pharmaceutical companies, as many of the reforms require the promulgation of detailed regulations implementing the statutory provisions, some of which has not yet fully occurred. For example, in January 2016, the Centers for Medicare and Medicaid Services issued a final rule regarding the Medicaid Drug Rebate Program, effective April 1, 2016, that, among other things, revises the manner in which the "average manufacturer price" is to be calculated by manufacturers participating in the program and implements certain amendments to the Medicaid rebate statute created under the Affordable Care Act. Further, there have been judicial and Congressional challenges to certain aspects of the Affordable Care Act, and we expect there will be additional challenges and amendments to the Affordable Care Act in the future. Since January 2017, the President of the United States has signed two Executive Orders and other directives designed to delay the implementation of certain provisions of the Affordable Care Act or otherwise circumvent some of the requirements for health insurance mandated by the Affordable Care Act. The Tax Cuts and Jobs Act of 2017 includes a provision repealing, effective January 1, 2019, the tax-based shared responsibility payment imposed by the Affordable Care Act on certain individuals who fail to maintain qualifying health coverage for all or part of a year that is commonly referred to as the "individual mandate." Congress will likely consider other legislation to replace elements of the Affordable Care Act. The Affordable Care Act is likely to continue the downward pressure on pharmaceutical pricing and may also increase our regulatory burdens and operating costs. We continue to evaluate the effect that the Affordable Care Act and its possible repeal and replacement has on our business.
Other legislative changes have been proposed and adopted since the Affordable Care Act was enacted. For example, in August 2011, President Obama signed into law the Budget Control Act of 2011, which, among other things, created the Joint Select Committee on Deficit Reduction to recommend to Congress proposals in spending reductions. The Joint Select Committee did not achieve a targeted deficit reduction of at least $1.2 trillion for the years 2013 through 2021, triggering the legislation’s automatic reduction to several government programs. This included further reductions to Medicare payments to providers of 2% per fiscal year, which went into effect in April 2013 and, due to subsequent legislative amendments to the statute, will stay in effect through 2025 unless additional Congressional action is taken. Additionally, in January 2013, the American Taxpayer Relief Act of 2012 was signed into law, which, among other things, reduced Medicare payments to several providers and increased the statute of limitations period in which the government may recover overpayments to providers from three to five years.

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Further, there have been several recent U.S. Congressional inquiries and proposed federal and state legislation designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the out-of-pocket cost of prescription drugs and reform government program reimbursement methodologies for drugs. Such scrutiny has resulted in several recent Congressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to pharmaceutical product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for products. At the federal level, the current administration’s budget proposal for fiscal year 2019 contains further drug price control measures that could be enacted during the 2019 budget process or in other future legislation, including, for example, measures to permit Medicare Part D plans to negotiate the price of certain drugs under Medicare Part B, to allow some states to negotiate drug prices under Medicaid, and to eliminate cost sharing for generic drugs for low-income patients. Additionally, on May 11, 2018, the President of the United States laid out his administration’s "Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs" to reduce the cost of prescription drugs while preserving innovation and cures. The Department of Health and Human Services ("HHS") has already started the process of soliciting feedback on some of these measures and, at the same time, is immediately implementing others under its existing authority. Although some of these and other proposals will require authorization through additional legislation to become effective, Congress and the U.S. presidential administration have each indicated that they will continue to seek new legislative and/or administrative measures to control drug costs. At the state level, legislatures have become increasingly aggressive in passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
Moreover, the Drug Supply Chain Security Act, which was enacted in 2012 as part of the Food and Drug Administration Safety and Innovation Act, imposes new obligations on manufacturers of pharmaceutical products related to product tracking and tracing. Legislative and regulatory proposals have been made to expand post-approval requirements and restrict sales and promotional activities for pharmaceutical products. We are not sure whether additional legislative changes will be enacted, or whether the current regulations, guidance or interpretations will be changed, or what the impact of such changes on our business, if any, may be. In addition, increased scrutiny by the U.S. Congress of the FDA’s approval process may significantly delay or prevent marketing approval, as well as subject us to more stringent product labeling and post-marketing testing and other requirements.
We expect that additional state and federal healthcare reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could result in reduced demand for our product candidates or additional pricing pressures.
Coverage and adequate reimbursement may not be available for our product candidates, which could make it difficult for us to sell our products profitably, if approved.
Market acceptance and sales of any approved product candidates that we develop will depend in part on the extent to which coverage and adequate reimbursement for these products and related treatments will be available from third-party payers, including government health administration authorities and private health insurers. In the United States, no uniform policy of coverage and reimbursement for products exists among third-party payers. Third-party payers decide which drugs or therapies they will pay for and establish reimbursement levels. One payer’s determination to provide coverage for a product does not assure that other payers will also provide coverage, and adequate reimbursement, for the product. Additionally, a third-party payer’s decision to provide coverage for a drug or therapy does not imply that an adequate reimbursement rate will be approved. Each plan determines whether or not it will provide coverage for a drug or therapy, what amount it will pay the manufacturer for the drug or therapy, on what tier of its formulary the drug or therapy will be placed, and whether to require step therapy. The position of a drug on a formulary generally determines the co-payment that a patient will need to make to obtain the drug and can strongly influence the adoption of a drug by patients and physicians. Patients who are prescribed treatments for their conditions and providers prescribing such services generally rely on third-party payers to reimburse all or part of the associated healthcare costs. Patients are unlikely to use our products unless coverage is provided, and reimbursement is adequate to cover a significant portion of the cost of our products.

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The process for determining whether a third-party payer will provide coverage for a product may be separate from the process for setting the price of a product or for establishing the reimbursement rate that such a payer will pay for the product. Even if we do obtain adequate levels of reimbursement, third-party payers, such as government or private healthcare insurers, carefully review and increasingly question the coverage of, and challenge the prices charged for, products. A primary trend in the U.S. healthcare industry and elsewhere is cost containment. Increasingly, third-party payers are requiring that pharmaceutical companies provide them with predetermined discounts from list prices and are challenging the prices charged for products. We may also be required to conduct expensive pharmacoeconomic studies to justify the coverage and the amount of reimbursement for particular medications. We cannot be sure that coverage and reimbursement will be available for any product that we commercialize and, if reimbursement is available, what the level of reimbursement will be. Inadequate coverage or reimbursement may impact the demand for, or the price of, any product for which we obtain marketing approval. If coverage and adequate reimbursement are not available, or are available only to limited levels, we may not be able to successfully commercialize any product candidates that we develop.


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Risks Related to Our Dependence on Third Parties
We do not have our own manufacturing capabilities and will rely on third parties to produce clinical and commercial supplies of our product candidates.
We are building teams with drug formulation and manufacturing expertise but do not own or operate, nor do we expect to own or operate, facilities for product manufacturing, storage and distribution, or testing. In addition to the technical challenges of drug product formulation and scale-up and environmental compliance aspects of chemical and biologics manufacturing, our vendors of manufacturing services will need to comply with U.S. and foreign regulatory authority licensure and GMP quality requirements. These obligations are enforced by periodic inspection and audit by regulatory authorities, and any adverse findings or violations discovered on such inspections could distract our vendors and be costly and time consuming to remediate, potentially impacting their supply of clinical and future commercial products to us.
Under the Oxford BioMedica Agreement, Oxford BioMedica will manufacture and supply the AXO-Lenti-PD in accordance with separate clinical and commercial supply agreements, which will be negotiated between us and Oxford BioMedica. The Oxford BioMedica Agreement contains certain key provisions of the clinical and commercial supply agreements, including pricing structure and our ability to transfer the technology to another manufacturer at any time following the completion of formal process characterization, process validation or BLA submission. Further, the process for manufacturing gene therapy products such as AXO-Lenti-PD is more complex than those required for most chemical pharmaceuticals. Moreover, unlike chemical pharmaceuticals, the physical and chemical properties of a gene therapy product such as ours generally cannot be fully characterized. Although we may establish our own manufacturing facility or use that of a third-party contract manufacturer to support a commercial launch of AXO-Lenti-PD, if approved, the timeframe for us to obtain approval for such facility or qualify such third-party contract manufacturer and ensure that all processes, methods and equipment are compliant with GMP requirements is uncertain. As a result, we will heavily depend on Oxford BioMedica and its key personnel to manufacture sufficient quantities of AXO-Lenti-PD drug product for future clinical trials as well as in commercial quantities if such product candidate receives regulatory approval.
Under the Benitec Agreement, Benitec will be responsible for certain development and manufacturing activities for the AXO-AAV-OPMD Program, and we will reimburse Benitec for its costs incurred, in accordance with an agreed-upon development plan and budget. Benitec is responsible for completing the GMP manufacturing processes necessary to initiate clinical trials of AXO-AAV-OPMD. If Benitec fails to complete these processes in a timely manner, our clinical development of AXO-AAV-OPMD may be delayed.
Under the Arena Development Agreement, subject to specified exceptions, Arena remains the sole and exclusive commercial manufacturer of nelotanserin, and we will depend on Arena to manufacture sufficient quantities of nelotanserin if nelotanserin is approved for commercial sale. Subject to Arena's approval, we have the right to contract with third parties for the manufacture of nelotanserin for development purposes only. Arena has sold their manufacturing facility and will be reliant on third parties to supply finished drug product for commercial sale. We and Arena are reliant on third-party suppliers for the active pharmaceutical ingredient in nelotanserin, and we and Arena have an agreement in place for the supply of active pharmaceutical ingredient. If we are unable to maintain a relationship with this or other third-party contractors, or if Arena is unable to manufacture or otherwise supply nelotanserin finished product to us, whether as a result of its own inability to obtain active pharmaceutical ingredient or finished drug product or otherwise, we could experience delays in our development and commercial efforts. In January 2018, we were notified by Arena that it has assigned all of its rights and obligations under the Arena Development Agreement to an affiliate, 125 Royalty Inc.
Further, our reliance on third-party manufacturers entails risks to which we would not be subject if we manufactured product candidates ourselves, including:
failure to satisfy their contractual duties or obligations;
inability to meet our product specifications and quality requirements consistently;
delay or inability to procure or expand sufficient manufacturing capacity;
manufacturing and/or product quality issues related to manufacturing development and scale-up;
costs and validation of new equipment and facilities required for scale-up;
failure to comply with applicable laws, regulations and standards, including cGMP and similar foreign standards;
deficient or improper record-keeping;
contractual restrictions on our ability to engage additional or alternative manufacturers;
inability to negotiate manufacturing agreements with third parties under commercially reasonable terms;
termination or nonrenewal of manufacturing agreements with third parties in a manner or at a time that is costly or damaging to us;

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reliance on a limited number of sources, and in some cases, single sources for product components, such that if we are unable to secure a sufficient supply of these product components, we will be unable to manufacture and sell our product candidates or any future product candidate in a timely fashion, in sufficient quantities or under acceptable terms;
lack of access or licenses to proprietary manufacturing methods used by third-party manufacturers to make our product candidates;
lack of qualified backup suppliers for those components that are currently purchased from a sole or single source supplier;
operations of our third-party manufacturers or suppliers could be disrupted by conditions unrelated to our business or operations, including the bankruptcy of the manufacturer or supplier or regulatory sanctions related to the manufacture of our or other company’s products;
carrier disruptions or increased costs that are beyond our control; and
failure to deliver our products under specified storage conditions and in a timely manner.
Our product candidates AXO-Lenti-PD and AXO-AAV-OPMD are manufactured using technically complex processes requiring specialized facilities, highly specific raw materials and other production constraints. The complexity of these processes, as well as strict government standards for the manufacture and storage of our product candidates, subjects us to manufacturing risks for this product candidate. If supply from a manufacturing facility is interrupted, there could be a significant disruption in supply of our product candidates. Further, under the terms of our supply and manufacturing agreements, including those we will enter into with Oxford BioMedica, we may be limited in entering into arrangements with third parties for the manufacture and supply of AXO-Lenti-PD or AXO-AAV-OPMD. Even if we were able to engage other manufacturers or suppliers, we may not be able to enter into arrangements with on favorable terms or at all. Use of new third-party manufacturers could increase the risk of delays in production or insufficient supplies of our product candidates as we transfer our manufacturing technology to these manufacturers and as they gain experience manufacturing our product candidates.
Any of these events affecting our product candidates or those of adjuncts could lead to clinical trial delays, cost overruns, delay or failure to obtain regulatory approval or impact our ability to successfully commercialize our products, as well as potential product liability litigation, product recalls or product withdrawals. Some of these events could be the basis for FDA action, including injunction, recall, seizure, or total or partial suspension of production.
We intend to rely on third parties to conduct, supervise and monitor our nonclinical studies and our clinical trials, and if those third parties perform in an unsatisfactory manner, it may harm our business.
We intend to rely on CROs and nonclinical and clinical trial sites to ensure the proper and timely conduct of our nonclinical studies and our clinical trials, and we expect to have limited influence over their actual performance. In addition, pursuant to the Oxford BioMedica Agreement and the Benitec Agreement, we may rely on Oxford BioMedica and Benitec employees for certain services in connection with the transition of AXO-Lenti-PD and AXO-AAV-OPMD, respectively. We will not have complete control over those employees or their execution of services provided to us under the Oxford BioMedica Agreement or the Benitec Agreement.
We intend to rely upon CROs to monitor and manage data for our clinical programs, as well as the execution of future nonclinical studies. We expect to control only certain aspects of our CROs’ activities. Nevertheless, we will be responsible for ensuring that each of our studies is conducted in accordance with the applicable protocol, legal, regulatory and scientific standards and our reliance on the CROs does not relieve us of our regulatory responsibilities.
We and our CROs will be required to comply with Good laboratory practices or GLPs and GCPs, which are regulations and guidelines enforced by the FDA and are also required by the competent authorities of the member states of the European Economic Area and comparable foreign regulatory authorities in the form of International Council for Harmonization guidelines for any of our product candidates that are in nonclinical and clinical development. The regulatory authorities enforce GCPs through periodic inspections of trial sponsors, principal investigators and clinical trial sites. If we or our CROs fail to comply with GCPs, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may reject our marketing applications or require us to perform additional clinical trials before approving our marketing applications. Accordingly, if we or our CROs fail to comply with these regulations or other applicable laws, regulations or standards, or fail to recruit a sufficient number of subjects, we may be required to repeat clinical trials, which would delay the relevant regulatory approval process. Failure by our CROs to properly execute study protocols in accordance with applicable law could also create product liability and healthcare regulatory risks for us as sponsors of those studies.

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Our CROs will not be our employees, and we will not control whether or not they devote sufficient time and resources to our clinical and nonclinical programs. These CROs may also have relationships with other commercial entities, including our competitors, for whom they may also be conducting clinical trials, or other drug development activities which could harm our competitive position. We face the risk of potential unauthorized disclosure or misappropriation of our intellectual property by CROs, which may reduce our trade secret and intellectual property protection and allow our potential competitors to access and exploit our proprietary technology. If our CROs do not successfully carry out their contractual duties or obligations, fail to meet expected deadlines, or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our (or their own) clinical protocols or regulatory requirements or for any other reasons, our clinical trials may be extended, delayed or terminated, and we may not be able to obtain regulatory approval for, or successfully commercialize, any product candidate that we develop. As a result, our financial results and the commercial prospects for any product candidate that we develop could be harmed, our costs could increase, and our ability to generate revenues could be delayed.
If our relationships with these CROs terminate, we may not be able to enter into arrangements with alternative CROs or do so on commercially reasonable terms or in a timely manner. Switching or adding additional CROs involves substantial cost and requires management time and focus. In addition, there is a natural transition period when a new CRO commences work. As a result, delays occur, which can materially impact our ability to meet our desired clinical development timelines. Though we intend to carefully manage our relationships with our CROs, there can be no assurance that we will not encounter challenges or delays in the future or that these delays or challenges will not have an adverse impact on our business, financial condition and prospects.
Certain intellectual property of Oxford BioMedica relating to AXO-Lenti-PD and other gene therapy products that we have licensed from Oxford BioMedica are subject to a lien under Oxford BioMedica’s debt agreements. The foreclosure on such intellectual property or exercise of other remedies available to the lenders under such debt agreements could materially adversely affect our rights under the Oxford BioMedica Agreement and our future prospects.
Certain intellectual property and other intangible assets of Oxford BioMedica, excluding the Gene Therapy Product-specific intellectual property licensed under the Oxford BioMedica Agreement, are encumbered by an existing loan agreement between Oxford BioMedica and certain of its lenders. There can be no assurance that Oxford BioMedica will remain in compliance with its obligations under the loan agreement. In the event of foreclosure or exercise of other remedies by the lenders under such agreement on the assets (including such intellectual property) pledged to such lenders, our ability to use and develop AXO-Lenti-PD and other gene therapy product candidates under the license may be materially adversely affected, and we may be required to negotiate with third-party lenders with whom we do not have a prior relationship.
We may seek to enter into collaborations in the future with other third parties. If we are unable to enter into such collaborations, or if these collaborations are not successful, our business could be adversely affected.
We will seek to enter into additional collaborations in the future, including sales, marketing, distribution, development, licensing, and/or broader collaboration agreements. Our likely collaborators include large and mid-size pharmaceutical companies, regional and national pharmaceutical companies, biotechnology companies, and medical device manufacturers. However, we may not be able to enter into additional collaborations on favorable terms or at all. Our ability to generate revenues from our collaborations will depend on our and our collaborators’ abilities to successfully perform the functions assigned to each of us in these arrangements. In addition, our collaborators have the ability to abandon research or development projects and terminate applicable agreements. Moreover, an unsuccessful outcome in any clinical trial for which our collaborator is responsible could be harmful to the public perception and prospects of our existing product candidate pipeline.
Our relationship with any future collaborations may pose several risks, including the following:
collaborators have significant discretion in determining the amount and timing of the efforts and resources that they will apply to these collaborations;
collaborators may not perform their obligations as expected;
the nonclinical studies and clinical trials conducted as part of these collaborations may not be successful;
collaborators may not pursue development and commercialization of any product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs based on nonclinical study or clinical trial results, changes in the collaborators’ strategic focus or available funding or external factors, such as an acquisition, that divert resources or create competing priorities;
collaborators may delay nonclinical studies and clinical trials, provide insufficient funding for nonclinical studies and clinical trials, stop a nonclinical study or clinical trial or abandon a product candidate, repeat or conduct new nonclinical studies or clinical trials or require a new formulation of a product candidate for nonclinical studies or clinical trials;
we may not have access to, or may be restricted from disclosing, certain information regarding product candidates being developed or commercialized under a collaboration and, consequently, may have limited ability to inform our stockholders about the status of such product candidates;

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collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours;
product candidates developed in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates;
a collaborator with marketing and distribution rights to one or more of our product candidates that achieve regulatory approval may not commit sufficient resources to the marketing and distribution of any such product candidate;
disagreements with collaborators, including disagreements over proprietary rights, contract interpretation or the preferred course of development of any product candidates, may cause delays or termination of the research, development or commercialization of such product candidates, may lead to additional responsibilities for us with respect to such product candidates or may result in litigation or arbitration, any of which would be time consuming and expensive;
collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation;
disputes may arise with respect to the ownership or inventorship of intellectual property developed pursuant to our collaborations;
collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability;
the terms of our collaboration agreement may restrict us from entering into certain relationships with other third parties, thereby limiting our options; and
collaborations may be terminated for the convenience of the collaborator and, if terminated, we could be required to raise additional capital to pursue further development or commercialization of the applicable product candidates.

We will face significant competition in seeking appropriate collaborators, and the negotiation process is time-consuming and complex. Our ability to reach a definitive collaboration agreement with any future collaborators will depend, among other things, upon our assessment of the collaborator’s resources and expertise, the terms and conditions of the proposed collaboration and the proposed collaborator’s evaluation of several factors. Those factors may include the design or results of clinical trials, the likelihood of approval by the FDA or similar regulatory authorities outside the United States, the potential market for the subject product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing products, the existence of uncertainty with respect to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the challenge, and industry and market conditions generally. We may also be restricted under future license agreements from entering into agreements on certain terms with potential collaborators.


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Risks Related to Our Intellectual Property
If we are unable to obtain and maintain patent protection for our technology and products or if the scope of the patent protection obtained is not sufficiently broad, we may not be able to compete effectively in our markets.
We rely, and will continue to rely, upon a combination of patents, trademarks, trade secret protection and confidentiality agreements with employees, consultants, collaborators, advisors and other third parties to protect the intellectual property related to our current and future drug development programs and product candidates. Our success depends in large part on our ability to obtain and maintain patent protection in the United States and other countries for our current product candidates and any future product candidates. We seek to protect our proprietary position by filing patent applications in the United States and abroad related to our current and future product development programs and product candidates. The patent prosecution process is expensive and time-consuming, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner.
The patent applications we have in-licensed cannot be enforced against third parties practicing the technology claimed in such applications unless and until a patent issues from such application(s). The licensed patent applications may fail to result in issued patents with claims that cover AXO-AAV-OPMD or other gene therapy product candidates in the United States or in foreign countries. As a result, our in-licensed patent portfolio alone may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.
It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection. The patent applications that we own or in-license may fail to result in issued patents with claims that cover our current product candidates or any future product candidates in the United States or in other foreign countries. We may also inadvertently make statements to regulatory agencies during the regulatory approval process that may be inconsistent with positions that have been taken during prosecution of our patents which may result in such patents being narrowed, invalidated, or held unenforceable. The patents and patent applications that we own or in-license may fail to result in issued patents with claims that cover our current product candidates or any future product candidates in the United States or in other foreign countries.
The patent rights that we own or have licensed relating to our product candidates may be limited in ways that may affect our ability to exclude third parties from competing against us if we obtain regulatory approval to market these product candidates. For our current product candidates or future product candidates for which we do not hold or do not obtain composition of matter patents, competitors who obtain the requisite regulatory approval can offer products with the same composition as our products so long as the competitors do not infringe any method patents that we may hold. Method use patents only protect the product when used or sold for the specified method. However, this type of patent protection does not limit a competitor from making and marketing a product that is identical to our product that is labeled for an indication that is outside of the patented method, or for which there is a substantial use in commerce outside the patented method.
There is no assurance that all of the potentially relevant prior art relating to our patents and patent applications has been found, which can prevent a patent from issuing from a pending patent application or be used to invalidate a patent. The patent examination process may require us to narrow our claims, which may limit the scope of patent protection that we may obtain. Even if patents do successfully issue based on our owned or in-licensed applications and even if such patents cover our current or future product candidates, third parties may challenge their validity, enforceability or scope, which may result in such patents being narrowed, invalidated, or held unenforceable. Any successful opposition to these patents or any other patents owned by or licensed to us in the future could deprive us of rights necessary for the successful commercialization of any current or future product candidates, if approved. Further, if we encounter delays in regulatory approvals, the period of time during which we could market a product candidate under patent protection could be reduced.
If the patent applications we hold or have in-licensed with respect to our development programs and product candidates fail to issue, if their breadth or strength of protection is threatened, or if they fail to provide meaningful exclusivity for our current or future product candidates, it could dissuade companies from collaborating with us to develop product candidates and threaten our ability to commercialize future drugs. Our owned or in-licensed pending applications cannot be enforced against third parties practicing the technology claimed in such applications unless and until a patent issues from such applications. Any such outcome could have an adverse effect on our business.

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The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions and has in recent years been the subject of much litigation. In addition, the laws of foreign countries may not protect our rights to the same extent as the laws of the United States. For example, European patent law restricts the patentability of methods of treatment of the human body more than United States law does. Publications of discoveries in scientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18 months after filing, or in some cases not at all. Therefore, we cannot know with certainty whether we were the first to make the inventions claimed in our owned or licensed patents or pending patent applications, or whether we were the first to file for patent protection of such inventions. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights are highly uncertain. Our pending and future patent applications may not result in patents being issued which protect our technology or products, in whole or in part, or which effectively prevent others from commercializing competitive technologies and products. Changes in either the patent laws or interpretation of the patent laws in the United States and other countries may diminish the value of our patents or narrow the scope of our patent protection.
Patent reform legislation could increase uncertainties and costs surrounding the prosecution of our owned and in-licensed patent applications and the enforcement or defense of our owned or in-licensed issued patents. On September 16, 2011, the Leahy-Smith America Invents Act (the "Leahy-Smith Act") was signed into law. The Leahy-Smith Act made a number of significant changes to United States patent laws. These include provisions that affect the way patent applications are prosecuted and challenged at the U.S. Patent and Trademark Office (USPTO), and may also affect patent litigation The USPTO has developed and continues to develop new regulations and procedures to govern administration of the Leahy-Smith Act, and many of the substantive changes to patent law associated with the Leahy-Smith Act, and in particular, the first to file provisions, only became effective on March 16, 2013. Accordingly, it is not clear what, if any, impact the Leahy-Smith Act, subsequent rulemaking, and judicial interpretation of the Leahy-Smith Act and regulations with have on the operation of our business. However, the Leahy-Smith Act and its implementation could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement and/or defense of our issued patents, all of which could have an adverse effect on our business and financial condition.
Moreover, we may be subject to a third-party pre-issuance submission of prior art to the USPTO, or become involved in opposition, derivation, reexamination, inter partes review, post-grant review or interference proceedings challenging our patent rights or the patent rights of others. An adverse determination in any such submission, proceeding or litigation could reduce the scope of, or invalidate, our patent rights, allow third parties to commercialize our technology or products and compete directly with us, without payment to us, or result in our inability to manufacture or commercialize products without infringing third-party patent rights. 
Even if our patent applications that we own or license issue as patents, they may not issue in a form that will provide us with any meaningful protection, prevent competitors from competing with us or otherwise provide us with any competitive advantage. Our competitors may be able to circumvent our patents by developing similar or alternative technologies or products in a non-infringing manner. In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future product candidates.
The issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our owned and licensed patents may be challenged in the courts or patent offices in the United States and abroad. Such challenges may result in loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology and products, or limit the duration of the patent protection of our technology and products. 
The inventorship and/or ownership rights for patents we own or in-license may be challenged by third parties. Such challenges could result in loss of exclusive rights to such patents, which could limit our ability to stop others from using or commercializing similar or identical technology and products or require us to obtain a license from such third parties on commercially reasonable terms to secure exclusive rights, or our business could be harmed. If any such challenges to inventorship and/or ownership were asserted, there is no assurance that a court would find in our favor or that, if we choose to seek a license, such license would be available to us on acceptable terms or at all. 
Moreover, patents have a limited lifespan. In the United States, the natural expiration of a patent is generally 20 years after it is filed. Various extensions may be available; however, the life of a patent, and the protection it affords, is limited. Without patent protection for our current or future product candidates, we may be open to competition from biosimilar or generic versions of such products. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, our owned and licensed patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.

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If we do not obtain protection under the Hatch-Waxman Amendments by extending the patent term and obtain data exclusivity for our product candidates, our business may be materially harmed.
Our commercial success will largely depend on our ability to obtain and maintain patent and other intellectual property in the United States and other countries with respect to our proprietary technology, product candidates and our target indications. Given the amount of time required for the development, testing and regulatory review of new drug candidates, patents protecting our drug candidates might expire before or shortly after such candidates begin to be commercialized. We expect to seek extensions of patent terms in the United States and, if available, in other countries where we are prosecuting patents.
Depending upon the timing, duration and specifics of FDA marketing approval of our product candidates, one or more of our U.S. patents may be eligible for limited patent term restoration under the Drug Price Competition and Patent Term Restoration Act of 1984, referred to as the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent restoration term of up to five years beyond the normal expiration of the patent as compensation for patent term lost during development and the FDA regulatory review process, which is limited to the approved indication (or any additional indications approved during the period of extension). This extension is limited to only one patent that covers the approved product, the approved use of the product, or a method of manufacturing the product. However, the applicable authorities, including the FDA and the USPTO in the United States, and any equivalent regulatory authority in other countries, may not agree with our assessment of whether such extensions are available, and may refuse to grant extensions to our patents, or may grant more limited extensions than we request. We may not be granted an extension because of, for example, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable requirements. Moreover, the applicable time period or the scope of patent protection afforded could be less than we request.
If we are unable to extend the expiration date of our existing patents or obtain new patents with longer expiry dates, our competitors may be able to take advantage of our investment in development and clinical trials by referencing our clinical and nonclinical data to obtain approval of competing products following our patent expiration and launch their product earlier than might otherwise be the case.
The validity, scope and enforceability of any patents that cover our drug product candidates, including those listed in the Orange Book, can be challenged by third parties.
One or more third parties may challenge the current patents, or patents that may issue in the future, within our portfolio, including those covering nelotanserin, which could result in the invalidation of, or render unenforceable, some or all of the relevant patent claims or a finding of non-infringement. For example, if one of our drug product candidates is approved by the FDA and a third-party files an Abbreviated New Drug Application ("ANDA") for a generic drug containing nelotanserin, and relies in whole or in part on studies conducted by or for us, the third-party will be required to certify to the FDA that either: (1) there is no patent information listed in the FDA’s Orange Book with respect to our NDA for the applicable approved drug candidate; (2) the patents listed in the Orange Book have expired; (3) the listed patents have not expired, but will expire on a particular date and approval is sought after patent expiration; or (4) the listed patents are invalid or will not be infringed by the manufacture, use or sale of the third-party’s generic drug. A certification that the new drug will not infringe the Orange Book-listed patents for the applicable approved drug candidate, or that such patents are invalid, is called a paragraph IV certification. If the third-party submits a paragraph IV certification to the FDA, a notice of the paragraph IV certification must also be sent to us once the third-party’s ANDA is accepted for filing by the FDA. We may then initiate a lawsuit to defend the patents identified in the notice. The filing of a patent infringement lawsuit within 45 days of receipt of the notice automatically prevents the FDA from approving the third-party’s ANDA until the earliest of 30 months or the date on which the patent expires, the lawsuit is settled, or the court reaches a decision in the infringement lawsuit in favor of the third-party. If we do not file a patent infringement lawsuit within the required 45-day period, the third-party’s ANDA will not be subject to the 30-month stay of FDA approval.
Moreover, a third party may challenge the current patents, or patents that may issue in the future, within our portfolio, including those covering nelotanserin, which could result in the invalidation of some or all of the patents that might otherwise be eligible for listing in the Orange Book for one of our drug product candidates. If a third-party successfully challenges all of the patents that might otherwise be eligible for listing in the Orange Book for one of our drug products prior to FDA approval of our drug product candidate, we will not be entitled to the 30-month stay of FDA approval upon the filing of an ANDA for a generic drug containing, for example, nelotanserin, and relies in whole or in part on studies conducted by or for us.
Litigation or other proceedings to enforce or defend intellectual property rights are often very complex in nature, may be very expensive and time-consuming, may divert our management’s attention from our core business, and may result in unfavorable results that could limit our ability to prevent third parties from competing with our drug candidates.

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The validity, scope and enforceability of any patents that cover our biologic product candidates can be challenged by third parties.
For biologics, such as AXO-Lenti-PD and AXO-AAV-OPMD, the Biologics Price Competition and Innovation Act (BPCIA) provides a mechanism for one or more third parties to seek FDA approval to manufacture or sell a biosimilar or interchangeable versions of brand name biological products. Due to the large size and complexity of biological products, as compared to small molecules, a biosimilar must be "highly similar" to the reference product with "no clinically meaningful differences between the two." The BPCIA does not require reference product sponsors to list patents in an Orange Book and does not include an automatic 30-month stay of FDA approval upon the timely filing of a lawsuit. The BPCIA, however, does require a formal pre-litigation process which includes the exchange of information between a biosimilar applicant and a reference biologic sponsor that includes the identification of relevant patents and each parties’ basis for infringement and invalidity. After the exchange of this information, we may then initiate a lawsuit within 30 days to defend the patents identified in the exchange. If the biosimilar applicant successfully challenges the asserted patent claims it could result in the invalidation of, or render unenforceable, some or all of the relevant patent claims or result in a finding of non-infringement.
Litigation or other proceedings to enforce or defend intellectual property rights are often very complex in nature, may be very expensive and time-consuming, may divert our management’s attention from our core business, and may result in unfavorable results that could limit our ability to prevent third parties from competing with our drug candidates
Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
Periodic maintenance fees on any issued patent are due to be paid to the USPTO and other foreign patent agencies in several stages over the lifetime of the patent. The USPTO and various foreign national or international patent agencies require compliance with a number of procedural, documentary, fee payment and other similar provisions during the patent application process. While an inadvertent lapse can in many cases be cured by payment of a late fee or by other means in accordance with the applicable rules, there are situations in which noncompliance can result in abandonment or lapse of the patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. Non-compliance events that could result in abandonment or lapse of patent rights include, but are not limited to, failure to timely file national and regional stage patent applications based on our international patent application, failure to respond to official actions within prescribed time limits, non-payment of fees and failure to properly legalize and submit formal documents. If we or our licensors fail to maintain the patents and patent applications covering our current or future product candidates, our competitors might be able to enter the market, which would have an adverse effect on our business.
We may need to license intellectual property from third parties, and such licenses may not be available or may not be available on commercially reasonable terms.
A third-party may hold intellectual property, including patent rights that are important or necessary to the development of our current or future product candidates. It may be necessary for us to use the patented or proprietary technology of one or more third parties to commercialize our product candidates, in which case we would be required to obtain a license from these third parties on commercially reasonable terms, or our business could be harmed, possibly materially. If any such patents were to be asserted against us, there is no assurance that a court would find in our favor or that, if we choose or are required to seek a license, a license to any of these patents would be available to us on acceptable terms or at all.
It may be necessary to use a patented or proprietary AAV related technology of one or more third parties to manufacture and commercialize AXO-AAV-OPMD. If we are unable to obtain licenses from such third parties when needed or on commercially reasonable terms, our ability to commercialize AXO-AAV-OPMD, if approved, would likely be delayed.

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Third-party claims or litigation alleging infringement of patents or other proprietary rights or seeking to invalidate patents or other proprietary rights may delay or prevent the development and commercialization of our product candidates.
Our commercial success depends in part on our avoiding infringement and other violations of the patents and proprietary rights of third parties. There is a substantial amount of litigation, both within and outside the United States, involving patent and other intellectual property rights in the biotechnology and pharmaceutical industries, including patent infringement lawsuits, interferences, derivation and administrative law proceedings, inter partes review, and post-grant review before the USPTO, as well as oppositions and similar processes in foreign jurisdictions. Numerous U.S. and foreign issued patents and pending patent applications, which are owned by third parties, exist in the fields in which we and our collaborators are developing product candidates. As the biotechnology and pharmaceutical industries expand and more patents are issued, and as we gain greater visibility and market exposure as a public company, the risk increases that our product candidates or other business activities may be subject to claims of infringement of the patent and other proprietary rights of third parties. Third parties may assert that we are infringing their patents or employing their proprietary technology without authorization.
There may be third-party patents or patent applications with claims to compositions, materials, formulations, methods of manufacture or methods for treatment related to our current or future product candidates. Because patent applications can take many years to issue, there may be currently pending patent applications that may later result in issued patents that our current or future product candidates may infringe. In addition, third parties may obtain patents in the future and claim that use of our technologies infringes upon these patents. If any third-party patents were held by a court of competent jurisdiction to cover the manufacturing process of any of our product candidates, any molecules formed during the manufacturing process, any final product itself or the intended method of treatment of the product, including combination therapy, the holders of any such patents may be able to block our ability to commercialize such product candidate unless we obtained a license under the applicable patents, or until such patents expire. 
A license may not be available on commercially reasonable terms or at all.  In addition, we may be subject to claims that we are infringing other intellectual property rights, such as trademarks or copyrights, or misappropriating the trade secrets of others, and to the extent that our employees, consultants or contractors use intellectual property or proprietary information owned by others in their work for us, disputes may arise as to the rights in related or resulting know-how and inventions.
Parties making claims against us may obtain injunctive or other equitable relief, which could effectively block our ability to further develop and commercialize one or more of our product candidates. Defense of these claims, regardless of their merit, would involve substantial litigation expense and would be a substantial diversion of employee resources from our business.  In the event of a successful infringement or other intellectual property claim against us, we may have to pay substantial damages, including treble damages and attorneys’ fees for willful infringement, obtain one or more licenses from third parties, pay royalties or redesign our affected products, which may be impossible or require substantial time and monetary expenditure.  We cannot predict whether any such license would be available at all or whether it would be available on commercially reasonable terms. Furthermore, even in the absence of litigation, we may need to obtain licenses from third parties to advance our research or allow commercialization of our product candidates, and we have done so from time to time. We may fail to obtain any of these licenses at a reasonable cost or on reasonable terms, if at all.  In that event, we would be unable to further develop and commercialize one or more of our product candidates, which could harm our business significantly. Claims that we have misappropriated the confidential information or trade secrets of third parties could have a similar negative impact on our business.
We cannot provide any assurances that third-party patents do not exist which might be enforced against our product candidates, resulting in either an injunction prohibiting our sales, or, with respect to our sales, an obligation on our part to pay royalties or other forms of compensation to third parties.
We are aware of a third-party patent, as well as third-party patent applications, directed to administering a combination of cholinesterase inhibitor with a peripheral muscarinic receptor antagonist that could adversely affect the potential commercialization of RVT-104. While we do not believe that any such claims that would cover the potential commercialization of RVT-104 are valid or enforceable, we may be incorrect in this belief.
We are also aware of a third-party patent application directed to methods for producing a recombinant lentiviral vector that could adversely affect the potential commercialization of AXO-Lenti-PD. While we do not believe that any such claims that would cover the methods of making AXO-Lenti-PD are patentable, we may be incorrect in this belief.

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If we breach any of our license or collaboration agreements, it could compromise our development and commercialization efforts for our product candidates.
We have licensed rights to intellectual property from third parties in order to commercialize our product candidates, and, in the case of AXO-Lenti-PD, intend to enter into commercial supply and manufacturing agreements with Oxford BioMedica. In particular, our product candidate AXO-Lenti-PD is dependent on the Oxford BioMedica Agreement. Pursuant to such agreement, we received from Oxford BioMedica a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Oxford BioMedica to develop and commercialize AXO-Lenti-PD and related gene therapy products for all diseases and conditions. We also received from Oxford BioMedica an exclusive option to obtain a worldwide license to other patents and know-how controlled by Oxford BioMedica related to certain technology processes. Under the terms of the Oxford BioMedica Agreement, we and Oxford BioMedica have each agreed to customary non-compete restrictions limiting our respective abilities to develop certain directly-competing gene therapy products. We are solely responsible, at our expense, for all activities related to the development and commercialization of the Gene Therapy Products under the license. We must provide Oxford BioMedica with regular forecasts and updates with respect to our development and commercialization activities. We are required to use commercially reasonable efforts to develop, obtain regulatory approval of, and commercialize a Gene Therapy Product in the United States and at least one major market country in Europe. We are required to meet certain diligence milestones relating to clinical site selection, obtaining regulatory advice for a Gene Therapy Product, and inclusion of at least one U.S. based clinical site in a pivotal study of a Gene Therapy Product.
On July 8, 2018, we entered into the Benitec Agreement with Benitec. In particular, our product candidate AXO-AAV-OPMD is dependent on the Benitec Agreement. Pursuant to such agreement, we received from Benitec a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Benitec to develop and commercialize AXO-AAV-OPMD and related gene therapy products. Under the Benitec Agreement, Benitec also agreed to collaborate on five additional research plans for other genetic neurological disorders using Benitec technologies. We will receive a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Benitec to develop and commercialize products arising from each collaboration program. We are required to use commercially reasonable efforts to develop and to seek regulatory approval for at least one collaboration product candidate from each collaboration program in the United States and major market countries in Europe. In addition, we are required to use commercially reasonable efforts to develop and to seek regulatory approval for at least one AXO-AVV-OPMD product candidate for OPMD in each of the United States, Canada, France, and Israel. We shall be solely responsible for the cost for the development, manufacture, and commercialization of AXO-AAV-OPMD product candidates and collaboration product candidates.
Disputes may arise between us and any of these counterparties regarding intellectual property rights that are subject to such agreements, including, but not limited to:
the scope of rights granted under the agreement and other interpretation-related issues;
whether and the extent to which our technology and processes infringe on intellectual property of the licensor that is not subject to the agreement;
our right to sublicense patent and other rights to third parties;
our diligence obligations with respect to the use of the licensed technology in relation to our development and commercialization of our product candidates, and what activities satisfy those diligence obligations;
the ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners;
our right to transfer or assign our license; and
the effects of termination.
These or other disputes over intellectual property that we have licensed (or will license or acquire in the future) may prevent or impair our ability to maintain our current arrangements on acceptable terms or may impair the value of the arrangement to us. Any such dispute could have an adverse effect on our business.

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If we materially breach or fail to perform any provision under these license and collaboration agreements, including failure to make payments to a licensor or collaborator when due for royalties and failure to use commercially reasonable efforts to develop and commercialize our product candidates, such as AXO-Lenti-PD or AXO-AAV-OPMD, such licensors and collaborators have the right to terminate our agreement, and upon the effective date of such termination, our right to practice the licensed patent rights and other intellectual property would end. Any uncured, material breach under the agreements could result in our loss of rights to practice the patent rights and other intellectual property licensed to us under the agreements. 
We may become involved in lawsuits to protect or enforce our patents, the patents of our licensors or our other intellectual property rights, which could be expensive, time consuming and unsuccessful.
Competitors may infringe or otherwise violate our patents, the patents of our licensors or our other intellectual property rights.  To counter infringement or unauthorized use, we may be required to file legal claims, which can be expensive and time-consuming. In addition, in an infringement proceeding, a court may decide that a patent of ours or our licensors is not valid or is unenforceable or may refuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the technology in question. An adverse result in any litigation or defense proceedings could put one or more of our patents at risk of being invalidated or interpreted narrowly and could put our patent applications at risk of not issuing. The initiation of a claim against a third-party may also cause the third-party to bring counter claims against us such as claims asserting that our patents are invalid or unenforceable. 
In patent litigation in the United States, defendant counterclaims alleging invalidity or unenforceability are commonplace. Grounds for a validity challenge could be an alleged failure to meet any of several statutory requirements, including lack of novelty, obviousness, non-enablement, or lack of statutory subject matter. Grounds for an unenforceability assertion could be an allegation that someone connected with prosecution of the patent withheld relevant material information from the USPTO, or made a materially misleading statement, during prosecution. Third parties may also raise similar validity claims before the USPTO in post-grant proceedings such as ex parte reexaminations, inter partes review, or post-grant review, or oppositions or similar proceedings outside the United States, in parallel with litigation or even outside the context of litigation. The outcome following legal assertions of invalidity and unenforceability is unpredictable. We cannot be certain that there is no invalidating prior art, of which we and the patent examiner were unaware during prosecution. For the patents and patent applications that we have licensed, we may have limited or no right to participate in the defense of any licensed patents against challenge by a third-party. If a defendant were to prevail on a legal assertion of invalidity or unenforceability, we would lose at least part, and perhaps all, of any future patent protection on our current or future product candidates. Such a loss of patent protection could harm our business.
We may not be able to detect or prevent, alone or with our licensors, misappropriation of our intellectual property rights, particularly in countries where the laws may not protect those rights as fully as in the United States. Any litigation or other proceedings to enforce our intellectual property rights may fail, and even if successful, may result in substantial costs and distract our management and other employees.
Even if we establish infringement, the court may decide not to grant an injuction against further infringing activity and instead award only monetary damages, which may or may not be an adequate remedy. Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation. There could also be public announcements of the results of hearings, motions or other interim proceedings or developments. If securities analysts or investors perceive these results to be negative, it could have an adverse effect on the price of our common shares.
Because of the expense and uncertainty of litigation, we may not be in a position to enforce our intellectual property rights against third parties.
Because of the expense and uncertainty of litigation, we may conclude that even if a third-party is infringing our issued patent, any patents that may be issued as a result of our pending or future patent applications or other intellectual property rights, the risk-adjusted cost of bringing and enforcing such a claim or action may be too high or not in the best interest of our company or our shareholders. In such cases, we may decide that the more prudent course of action is to simply monitor the situation or initiate or seek some other non-litigious action or solution.

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Changes in U.S. patent law or the patent law of other countries or jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our products.
The United States has recently enacted and implemented wide-ranging patent reform legislation. The U.S. Supreme Court has ruled on several patent cases in recent years, either narrowing the scope of patent protection available in certain circumstances or weakening the rights of patent owners in certain situations. In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of patents, once obtained. Depending on actions by the U.S. Congress, the federal courts, and the USPTO, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce patents that we have licensed or that we might obtain in the future. Similarly, changes in patent law and regulations in other countries or jurisdictions or changes in the governmental bodies that enforce them or changes in how the relevant governmental authority enforces patent laws or regulations may weaken our ability to obtain new patents or to enforce patents that we have licensed or that we may obtain in the future.
The United States federal government retains certain rights in inventions produced with its financial assistance under the Bayh-Dole Act. The federal government retains a "nonexclusive, nontransferable, irrevocable, paid-up license" for its own benefit. The Bayh-Dole Act also provides federal agencies with "march-in rights". March-in rights allow the government, in specified circumstances, to require the contractor or successors in title to the patent to grant a "nonexclusive, partially exclusive, or exclusive license" to a "responsible applicant or applicants." If the patent owner refuses to do so, the government may grant the license itself.
We may not be able to protect our intellectual property rights throughout the world, which could impair our business.
Filing, prosecuting and defending patents covering our current and future product candidates throughout the world would be prohibitively expensive. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and, further, may export otherwise infringing products to territories where we may obtain patent protection, but where patent enforcement is not as strong as that in the United States. These products may compete with our products in jurisdictions where we do not have any issued or licensed patents and any future patent claims or other intellectual property rights may not be effective or sufficient to prevent them from so competing.
Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions. The legal systems of some countries do not favor the enforcement of patents and other intellectual property protection, which could make it difficult for us to stop the infringement of our patents generally. Proceedings to enforce our patent rights in foreign jurisdictions could result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be commercially meaningful.
Many countries, including European Union countries, India, Japan and China, have compulsory licensing laws under which a patent owner may be compelled under specified circumstances to grant licenses to third parties. In those countries, we may have limited remedies if patents are infringed or if we are compelled to grant a license to a third-party, which could materially diminish the value of those patents. This could limit our potential revenue opportunities. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a significant commercial advantage from the intellectual property that we develop or license.

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Our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.
Because we expect to rely on third parties to manufacture our product candidates, and we expect to continue to collaborate with third parties on the development of our current and future product candidates, we must, at times, share trade secrets with them. We also conduct joint research and development programs that may require us to share trade secrets under the terms of our collaboration or similar agreements. We seek to protect our proprietary technology in part by entering into confidentiality agreements and, if applicable, material transfer agreements, consulting agreements or other similar agreements with our advisors, employees, third-party contractors and consultants prior to beginning research or disclosing proprietary information. These agreements typically limit the rights of the third parties to use or disclose our confidential information, including our trade secrets. Despite the contractual provisions employed when working with third parties, the need to share trade secrets and other confidential information increases the risk that such trade secrets become known by our competitors, are inadvertently incorporated into the technology of others, or are disclosed or used in violation of these agreements. Any disclosure, either intentional or unintentional, by our employees, the employees of third parties with whom we share our facilities or third-party consultants and vendors that we engage to perform research, clinical trials or manufacturing activities, or misappropriation by third parties (such as through a cybersecurity breach) of our trade secrets or proprietary information could enable competitors to duplicate or surpass our technological achievements, thus eroding our competitive position in our market. Further, adequate remedies may not exist in the event of unauthorized use or disclosure. Given that our proprietary position is based, in part, on our know-how and trade secrets, a competitor’s discovery of our trade secrets or other unauthorized use or disclosure would impair our competitive position and may have an adverse effect on our business and results of operations.
In addition, these agreements typically restrict the ability of our advisors, employees, third-party contractors and consultants to publish data potentially relating to our trade secrets, although our agreements may contain certain limited publication rights. Policing unauthorized use of our or our licensors' intellectual property is difficult, expensive and time-consuming, and we may be unable to determine the extent of any unauthorized use. Moreover, enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable. In addition, some courts inside and outside the United States are less willing or unwilling to protect trade secrets. Despite our efforts to protect our trade secrets, our competitors may discover our trade secrets, either through breach of our agreements with third parties, independent development or publication of information by any of our third-party collaborators. A competitor's discovery of our trade secrets would impair our competitive position and have an adverse impact on our business.
We may be subject to claims that our employees, consultants or independent contractors have wrongfully used or disclosed confidential information of their former employers or other third parties.
We employ individuals who were previously employed at other biotechnology or pharmaceutical companies. Although we seek to protect our ownership of intellectual property rights by ensuring that our agreements with our employees, collaborators, and other third parties with whom we do business include provisions requiring such parties to assign rights in inventions to us, we may be subject to claims that we or our employees, consultants or independent contractors have inadvertently or otherwise used or disclosed confidential information of our employees’ former employers or other third parties. We may also be subject to claims that former employers or other third parties have an ownership interest in our patents. Litigation may be necessary to defend against these claims. There is no guarantee of success in defending these claims, and if we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights, such as exclusive ownership of, or right to use, valuable intellectual property. Even if we are successful, litigation could result in substantial cost and be a distraction to our management and other employees. Moreover, any such litigation or the threat thereof may adversely affect our reputation, our ability to form strategic alliances or sublicense our rights to collaborators, engage with scientific advisors or hire employees or consultants, each of which would have an adverse effect on our business, results of operations and financial condition.
In addition, while it is our policy to require our employees and contractors who may be involved in the development of intellectual property to execute agreements assigning such intellectual property to us, we may be unsuccessful in executing such an agreement with each party who in fact develops intellectual property that we regard as our own. Our and their assignment agreements may not be self-executing or may be breached, and we may be forced to bring claims against third parties, or defend claims they may bring against us, to determine the ownership of what we regard as our intellectual property.
If we or our licensors fail in prosecuting or defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel. Even if we and our licensors are successful in prosecuting or defending against such claims, litigation could result in substantial costs and be a distraction to management.

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Intellectual property litigation could cause us to spend substantial resources and distract our personnel from their normal responsibilities.
Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses and could distract our technical and management personnel from their normal responsibilities. In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments and if securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of our common shares. Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing or distribution activities. We may not have sufficient financial or other resources to conduct such litigation or proceedings adequately. Some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources. Accordingly, despite our efforts, we may not be able to prevent third parties from infringing upon or misappropriating our intellectual property. In addition, the uncertainties associated with litigation could compromise our ability to raise the funds necessary to initiate or continue our clinical trials and internal research programs, or in-license needed technology or other product candidates. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could compromise our ability to compete in the marketplace, including compromising our ability to raise the funds necessary to continue our clinical trials, continue our research programs, license necessary technology from third parties, or enter into development collaborations that would help us commercialize our drug candidates, if approved.
If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
In addition to seeking patents for our current and future drug candidates, we also rely on trade secrets, including unpatented know-how, technology and other proprietary information, to maintain our competitive position. We seek to protect our trade secrets, in part, by entering into non-disclosure and confidentiality agreements with parties who have access to them, such as our employees, corporate collaborators, outside scientific collaborators, contract manufacturers, consultants, advisors and other third parties. We also enter into confidentiality and invention or patent assignment agreements with our employees and consultants. Despite these efforts, any of these parties may breach the agreements and disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for such breaches. Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable. In addition, some courts inside and outside the United States are less willing or unwilling to protect trade secrets. If any of our trade secrets were to be lawfully obtained or independently developed by a competitor, we would have no right to prevent them, or those to whom they communicate it, from using that technology or information to compete with us. If any of our trade secrets were to be disclosed to or independently developed by a competitor, our competitive position would be harmed.
Any trademarks we have obtained or may obtain may be infringed or successfully challenged, resulting in harm to our business.
We expect to rely on trademarks as one means to distinguish any of our drug candidates that are approved for marketing from the products of our competitors. Once we select new trademarks and apply to register them, our trademark applications may not be approved. Third parties may oppose or attempt to cancel our trademark applications or trademarks, or otherwise challenge our use of the trademarks. In the event that our trademarks are successfully challenged, we could be forced to rebrand our drugs, which could result in loss of brand recognition and could require us to devote resources to advertising and marketing new brands. Our competitors may infringe our trademarks and we may not have adequate resources to enforce our trademarks.
If we attempt to enforce our trademarks and assert trademark infringement claims, a court may determine that the marks we have asserted are invalid or unenforceable, or that the party against whom we have asserted trademark infringement has superior rights to the marks in question. In this case, we could ultimately be forced to cease use of such trademarks.
Intellectual property rights do not necessarily address all potential threats to our competitive advantage.
The degree of future protection afforded by our intellectual property rights is uncertain because intellectual property rights have limitations, and may not adequately protect our business, or permit us to maintain our competitive advantage. The following examples are illustrative:
others may be able to make products that are the same as or similar to our product candidates, but that are not covered by the claims of the patents or other intellectual property rights that we own that we have exclusively licensed and have the right to enforce;
we, our licensor or any collaborators might not have been the first to make the inventions covered by the issued patents or pending patent applications that we own;

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we or our licensor might not have been the first to file patent applications covering certain of our inventions;
others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our intellectual property rights;
it is possible that our pending patent applications will not lead to issued patents;
issued patents that we own may not provide us with any competitive advantages, or may be held invalid or unenforceable as a result of legal challenges;
our competitors might conduct research and development activities in the United States and other countries that provide a safe harbor from patent infringement claims for certain research and development activities, as well as in countries where we do not have patent rights, and then use the information learned from such activities to develop competitive products for sale in our major commercial markets; and
we may not develop additional proprietary technologies that are patentable.


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Risks Related to Our Common Shares
An active trading market for our common shares may not be sustained.
Although our common shares are listed on the Nasdaq Global Select Market ("Nasdaq"), we cannot assure you that an active trading market for our common shares will continue to develop or be sustained. In addition, as a result of RSL owning 69.6% of our common shares as of June 30, 2018, or 73.1% of our common shares after giving effect to the RSL private placement in July 2018, trading in our common shares may be less liquid than the shares of companies with broader public ownership. If an active market for our common shares is not sustained, you may not be able to sell your shares quickly or at the market price. An inactive market may also impair our ability to raise capital to continue to fund operations by selling common shares and may impair our ability to acquire other companies or technologies by using our common shares as consideration.
The market price of our common shares has been and is likely to continue to be highly volatile, and you may lose some or all of your investment.
The market price of our common shares has been and is likely to continue to be highly volatile and may be subject to wide fluctuations in response to a variety of factors, including the following:
any additional delays in the commencement, enrollment and ultimate completion of our clinical trials;
results of clinical trials of our product candidates or those of our competitors, such as our announcement of the failure of our Phase 2B HEADWAY clinical trial of intepirdine in patients with DLB and the pilot Phase 2 Gait and Balance clinical trial of intepirdine in patients with dementia and gait impairment to meet their respective primary endpoints and the September 2017 announcement that our Phase 3 MINDSET clinical trial of intepirdine in patients with mild-to-moderate Alzheimer's disease did not meet its co-primary efficacy endpoints;
any delay in filing applications for marketing approval of AXO-Lenti-PD or AXO-AAV-OPMD, and any adverse development or perceived adverse development with respect to applicable regulatory authorities’ review of those applications;
failure to successfully develop and commercialize AXO-Lenti-PD, AXO-AAV-OPMD or any other of our current or future product candidates;
failure to maintain our relationship with Oxford BioMedica or Benitec or comply with the terms of the Oxford BioMedica Agreement or the Benitec Agreement;
inability to obtain additional funding;
regulatory or legal developments in the United States and other countries applicable to our product candidates, including gene therapies;
adverse regulatory decisions or statements;
changes in the structure of healthcare payment systems;
inability to obtain adequate product supply for our current product candidates or any future product candidate, or the inability to do so at acceptable prices;
introduction of new products, services or technologies by our competitors;
failure to meet or exceed financial projections we provide to the public;
failure to meet or exceed the estimates and projections of the investment community;
changes in the market valuations of similar companies;
market conditions in the pharmaceutical and biotechnology sectors, and the issuance of new or changed securities analysts’ reports or recommendations;
announcements of significant acquisitions, strategic partnerships, joint ventures or capital commitments by us or our competitors;
significant lawsuits, including patent or shareholder litigation, and disputes or other developments relating to our proprietary rights, including patents, litigation matters and our ability to obtain patent protection for our technologies;
additions or departures of key scientific or management personnel;
short sales of our common shares;
sales of our common shares by us or our shareholders in the future;
negative coverage in the media or analyst reports, whether accurate or not;
issuance of subpoenas or investigative demands, or the public fact of an investigation by a government agency, whether meritorious or not;
trading volume of our common shares;
general economic, industry and market conditions; and
the other factors described in this "Risk Factors" section.


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In addition, the stock markets have experienced extreme price and volume fluctuations that have affected and continue to affect the market prices of equity securities of many companies. These fluctuations have often been unrelated or disproportionate to the operating performance of those companies. Broad market and industry factors, as well as general economic, political, regulatory and market conditions, may negatively affect the market price of our common shares, regardless of our actual operating performance.
Volatility in our share price could subject us to securities class action litigation.
In the past, securities class action litigation has often been brought against a company following a decline in the market price of its securities and/or the discontinuation of development of a product candidate due to adverse clinical circumstances or results. This risk is especially relevant for us because pharmaceutical companies have experienced significant share price volatility in recent years. If we face such litigation, it could result in substantial costs and a diversion of management’s attention and resources, which could harm our business.
We are a "controlled company" within the meaning of the applicable rules of the Nasdaq and, as a result, qualify for exemptions from certain corporate governance requirements.  If we rely on these exemptions, you will not have the same protections afforded to shareholders of companies that are subject to such requirements.
RSL controls a majority of the voting power of our outstanding common shares. As a result, we are a "controlled company" within the meaning of the Nasdaq corporate governance requirements. Under these rules, a company of which more than 50% of the voting power for the election of directors is held by an individual, group or another company is a "controlled company" and may elect not to comply with certain corporate governance requirements, including the requirements:
that a majority of its board of directors consists of independent directors;
for an annual performance evaluation of the nominating and corporate governance and compensation committees;
to require director nominees to be selected, or recommended for the board of directors’ selection, either by independent directors constituting a majority of the Board’s independent directors in a vote in which only independent directors participate or a nominations committee comprised solely of independent directors; and
to have a compensation committee that is composed entirely of independent directors with a written charter addressing the committee’s purpose and responsibility.

We have elected to use certain of these exemptions and we may continue to use all or some of these exemptions in the future. As a result, you may not have the same protections afforded to shareholders of companies that are subject to all of the Nasdaq corporate governance requirements.
RSL owns a significant percentage of our common shares and is able to exert significant control over matters subject to shareholder approval.
Based on common shares outstanding as of June 30, 2018, RSL beneficially owns approximately 69.6% of the voting power of our outstanding common shares, or 73.1% of our common shares after giving effect to the RSL private placement in July 2018, and has the ability to substantially influence us through this ownership position. For example, RSL and its shareholders may be able to control elections of directors, issuance of equity, including to our employees under equity incentive plans, amendments of our organizational documents, or approval of any merger, sale of assets, or other major corporate transaction. RSL’s interests may not always coincide with our corporate interests or the interests of other shareholders, and it may act in a manner with which you may not agree or that may not be in the best interests of our other shareholders. Further, RSL is a privately held company whose ownership and governance structure is not transparent to our other shareholders. There may be changes to the management or ownership of RSL that could impact RSL’s interests in a way that may not coincide with our corporate interests or the interests of other shareholders. So long as RSL continues to own a significant amount of our equity, it will continue to be able to strongly influence or effectively control our decisions.
Our organizational and ownership structure may create significant conflicts of interests.
Our organizational and ownership structure involves a number of relationships that may give rise to certain conflicts of interest between us and minority holders of our common shares, on the one hand, and RSL and its shareholders, on the other hand. Certain of our directors and employees have equity interests in RSL and, accordingly, their interests may be aligned with RSL’s interests, which may not always coincide with our corporate interests or the interests of our other shareholders. Further, our other shareholders may not have visibility into the RSL ownership of any of our directors or officers, which may change at any time through acquisition, disposition, dilution, or otherwise. Any change in our directors’ or officers’ RSL ownership could impact the interests of those holders.

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In addition, we are party to certain related party agreements with RSL, RSI and RSG. These entities and their shareholders, including certain of our directors and employees, may have interests which differ from our interests or those of the minority holders of our common shares. For example, we are party to an information sharing and cooperation agreement with RSL pursuant to which RSL has granted us a right of first review on any potential dementia-related product or investment opportunity that RSL may consider pursuing. It is possible that we could fail to pursue a product candidate under this agreement and that product candidate is then successfully developed and commercialized by RSL or one of its other subsidiaries or affiliates. Any material transaction between us and RSL, RSI or RSG is subject to our related party transaction policy, which requires prior approval of such transaction by our Audit Committee. To the extent we fail to appropriately deal with any such conflicts of interests, it could negatively impact our reputation and ability to raise additional funds and the willingness of counterparties to do business with us, all of which could have an adverse effect on our business, financial condition, results of operations and cash flows.
If securities or industry analysts cease to publish research or reports about our business, or publish negative reports about our business, our share price and trading volume could decline.
The trading market for our common shares depends, in part, on the research and reports that securities or industry analysts publish about us or our business. We do not have any control over these analysts. If our financial performance fails to meet analyst estimates or one or more of the analysts who cover us downgrade our common shares or change their opinion of our common shares, our share price would likely decline. If one or more of these analysts cease coverage of our company or fail to regularly publish reports on us, we could lose visibility in the financial markets, which could cause our share price or trading volume to decline.
Because we do not anticipate paying any cash dividends on our common shares in the foreseeable future, capital appreciation, if any, would be your sole source of gain.
We have never declared or paid any cash dividends on our common shares. We currently anticipate that we will retain future earnings for the development, operation and expansion of our business and do not anticipate declaring or paying any cash dividends for the foreseeable future. We are also subject to Bermuda legal constraints that may affect our ability to pay dividends on our common shares and make other payments. Additionally, our ability to pay dividends is currently restricted by the terms of the Loan Agreement. As a result, capital appreciation, if any, of our common shares would be your sole source of gain on an investment in our common shares for the foreseeable future.
Future sales of our common shares, or the perception that such sales may occur, could depress our share price, even if our business is doing well.
Sales of a substantial number of our common shares in the public market, or the perception by investors that our shareholders intend to sell substantial amounts of our common shares in the public market, could depress the market price of our common shares, even if our business is doing well. Such a decrease in our share price could in turn impair our ability to raise capital through the sale of additional equity securities.
All of the shares sold in our initial public offering ("IPO") and our follow-on offering described below, as well as shares issued upon the exercise of options granted to persons other than our officers and directors, are freely transferable without restrictions or further registration under the Securities Act of 1933, as amended (the "Securities Act"). As of June 30, 2018, 75,000,000 of our outstanding common shares, or 89,285,714 of our common shares after giving effect to the RSL private placement in July 2018, representing a majority of our common shares, were held by RSL. If RSL or any of our executive officers or directors were to sell our common shares, or if the market perceived that RSL or any of our executive officers or directors intend to sell our common shares, it could negatively affect our share price. Prior to RSL’s corporate reorganization and recapitalization in December 2015, any decision by RSL to sell or otherwise dispose of our shares required the unanimous agreement of all of the directors of RSL, including Vivek Ramaswamy, our director and former principal executive officer. Subsequent to RSL’s corporate reorganization and recapitalization in December 2015, any such decision no longer requires a unanimous vote of RSL’s directors, meaning that all or a portion of the shares of our common stock held by RSL may be sold without Vivek Ramaswamy’s consent. However, any such sales must still be made in compliance with the Securities Act and the rules and regulations thereunder, which could limit the number of our shares that RSL could sell in any 90-day period.

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We have filed registration statements on Form S-8 under the Securities Act to register the common shares that may be issued under our equity incentive plans from time to time. Shares registered under these registration statements are available for sale in the public market subject to vesting arrangements and exercise of options, as well as Rule 144 in the case of our affiliates. We also filed a "shelf" registration statement on Form S-3 under the Securities Act in December 2016, allowing us, from time to time, to offer up to $750 million of any combination of registered common shares, preferred shares, debt securities and warrants. In April 2017, we offered and sold approximately $134.6 million of our common shares, net of underwriting discounts and commissions and offering expenses, pursuant to this registration statement.
We have incurred and will continue to incur substantial costs as a result of operating as a public company, and our management has been and will be required to continue to devote substantial time to compliance with our public company responsibilities and corporate governance practices.
As a public company, we have incurred and will continue to incur significant legal, accounting and other expenses. The Sarbanes-Oxley Act of 2002, the Dodd-Frank Wall Street Reform and Consumer Protection Act, the listing requirements of Nasdaq and other applicable securities rules and regulations impose various requirements on public companies. Our management and other personnel devote a substantial amount of time to compliance with these requirements. Moreover, changing rules and regulations may increase our legal and financial compliance costs and make some activities more time-consuming and costly. If, notwithstanding our efforts to comply with new or changing laws, regulations and standards, we fail to comply, regulatory authorities may initiate legal proceedings against us, and our business may be harmed.
Further, failure to comply with these laws, regulations and standards may make it more difficult and more expensive for us to obtain directors’ and officers’ liability insurance, which could make it more difficult for us to attract and retain qualified members of our Board of Directors or members of senior management.
If we are unable to maintain proper and effective internal controls over financial reporting and disclosure controls and procedures, investor confidence in our company and, as a result, the value of our common shares, may be adversely affected.
Effective internal controls over financial reporting are necessary for us to provide reliable financial reports and to protect from fraudulent, illegal or unauthorized transactions. Effective disclosure controls and procedures enable us to make timely and accurate disclosure of financial and non-financial information that we are required to disclose. If we cannot provide effective controls and reliable financial reports and other disclosures, our business and operating results could be harmed. We have in the past discovered, and may in the future discover, areas of our internal controls over financial reporting or disclosure controls and procedures that, even if effective, could be improved. For example, with respect to disclosure controls and procedures, in January 2018, we issued a press release disclosing clinical trial results that included an erroneous statistical value. We issued a correction the next day, and we are taking steps to further enhance controls over our clinical data disclosure process. We are required, pursuant to Section 404 of the Sarbanes-Oxley Act, to furnish a report by management on the effectiveness of our internal control over financial reporting as of the end of each fiscal year. Our independent registered public accounting firm will not be required to attest to the effectiveness of our internal control over financial reporting until our first annual report required to be filed with the SEC following the later of the date we are deemed to be a "large accelerated filer," as defined in the Exchange Act, or the date we are no longer an "emerging growth company," as defined in the JOBS Act.
If material weaknesses or control deficiencies occur or our disclosure controls and procedures are ineffective in the future, we may be unable to report our financial results or make other disclosures accurately on a timely basis, which could cause our reported financial results or other disclosures to be materially misstated and result in the loss of investor confidence and cause the market price of our common shares to decline.
We are an emerging growth company, and we cannot be certain if the reduced reporting requirements applicable to emerging growth companies will make our common shares less attractive to investors.
We are an "emerging growth company," as defined in the JOBS Act. For as long as we continue to be an emerging growth company, we may take advantage of exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies, including exemption from compliance with the auditor attestation requirements of Section 404, reduced disclosure obligations regarding executive compensation and exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and shareholder approval of any golden parachute payments not previously approved. We will remain an emerging growth company until the earliest of (1) March 31, 2021, (2) the last day of the fiscal year in which we have total annual gross revenue of at least $1.07 billion, (3) the date on which we are deemed to be a "large accelerated filer," which means the market value of our common shares that are held by non-affiliates exceeds $700.0 million as of the prior September 30, the end of our second fiscal quarter, and (4) the date on which we have issued more than $1.0 billion in non-convertible debt during the prior three-year period.

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In addition, under the JOBS Act, emerging growth companies can delay adopting new or revised accounting standards until such time as those standards apply to private companies. We have irrevocably elected not to avail ourselves of this exemption from new or revised accounting standards and, therefore, we will be subject to the same new or revised accounting standards as other public companies that are not emerging growth companies.
Even after we no longer qualify as an emerging growth company, we may still qualify as a "smaller reporting company" which would allow us to take advantage of many of the same exemptions from disclosure requirements including exemption from compliance with the auditor attestation requirements of Section 404 and reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements.
We cannot predict if investors will find our common shares less attractive because we may rely on these exemptions. If some investors find our common shares less attractive as a result, there may be a less active trading market for our common shares and our share price may be more volatile.
We are a Bermuda company and it may be difficult for you to enforce judgments against us or our directors and executive officers.
We are a Bermuda exempted company. As a result, the rights of our shareholders are governed by Bermuda law and our memorandum of association and bye-laws. The rights of shareholders under Bermuda law may differ from the rights of shareholders of companies incorporated in another jurisdiction. It may be difficult for investors to enforce in the United States judgments obtained in U.S. courts against us based on the civil liability provisions of the U.S. securities laws. It is doubtful whether courts in Bermuda will enforce judgments obtained in other jurisdictions, including the United States, against us or our directors or officers under the securities laws of those jurisdictions or entertain actions in Bermuda against us or our directors or officers under the securities laws of other jurisdictions. 
Bermuda law differs from the laws in effect in the United States and may afford less protection to our shareholders.
We are organized under the laws of Bermuda. As a result, our corporate affairs are governed by the Bermuda Companies Act 1981, as amended (the "Companies Act"), which differs in some material respects from laws typically applicable to U.S. corporations and shareholders, including the provisions relating to interested directors, amalgamations, mergers and acquisitions, takeovers, shareholder lawsuits and indemnification of directors. Generally, the duties of directors and officers of a Bermuda company are owed to the company only. Shareholders of Bermuda companies typically do not have rights to take action against directors or officers of the company and may only do so in limited circumstances. Shareholder class actions are not available under Bermuda law. The circumstances in which shareholder derivative actions may be available under Bermuda law are substantially more proscribed and less clear than they would be to shareholders of U.S. corporations. The Bermuda courts, however, would ordinarily be expected to permit a shareholder to commence an action in the name of a company to remedy a wrong to the company where the act complained of is alleged to be beyond the corporate power of the company or illegal, or would result in the violation of the company’s memorandum of association or bye-laws. Furthermore, consideration would be given by a Bermuda court to acts that are alleged to constitute a fraud against the minority shareholders or, for instance, where an act requires the approval of a greater percentage of the company’s shareholders than those who actually approved it.
When the affairs of a company are being conducted in a manner that is oppressive or prejudicial to the interests of some shareholders, one or more shareholders may apply to the Supreme Court of Bermuda, which may make such order as it sees fit, including an order regulating the conduct of the company’s affairs in the future or ordering the purchase of the shares of any shareholders by other shareholders or by the company. Additionally, under our bye-laws and as permitted by Bermuda law, each shareholder has waived any claim or right of action against our directors or officers for any action taken by directors or officers in the performance of their duties, except for actions involving fraud or dishonesty. In addition, the rights of our shareholders and the fiduciary responsibilities of our directors under Bermuda law are not as clearly established as under statutes or judicial precedent in existence in jurisdictions in the United States, particularly the State of Delaware. Therefore, our shareholders may have more difficulty protecting their interests than would shareholders of a corporation incorporated in a jurisdiction within the United States.

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There are regulatory limitations on the ownership and transfer of our common shares.
Common shares may be offered or sold in Bermuda only in compliance with the provisions of the Companies Act and the Bermuda Investment Business Act 2003, which regulates the sale of securities in Bermuda. In addition, the Bermuda Monetary Authority must approve all issues and transfers of shares of a Bermuda exempted company. However, the Bermuda Monetary Authority has, pursuant to its statement of June 1, 2005, given its general permission under the Exchange Control Act 1972 and related regulations for the issue and free transfer of our common shares to and among persons who are non-residents of Bermuda for exchange control purposes as long as the shares are listed on an appointed stock exchange, which includes Nasdaq. Specific permission of the Bermuda Monetary Authority has also been obtained dated June 8, 2015 to the issue and transfer of our shares, options, warrants, depositary receipts, rights, loan notes, debt instruments and our other securities to persons resident and non-resident of Bermuda for exchange control purposes while our shares are listed on an appointed stock exchange.  The general permission and the specific permission would cease to apply if we were to cease to be listed on Nasdaq or any other appointed stock exchange.
Our bye-laws enable our board of directors to issue preference shares, which may discourage a change of control.
Our bye-laws contain provisions that enable our board of directors to determine the powers, preferences, and rights of our preference shares and to issue the preference shares without shareholder approval.
This could discourage, delay or prevent a transaction involving a change in control of our company and may prevent our shareholders from receiving the benefit from any premium to the market price of our common shares offered by a bidder in a takeover context. Even in the absence of a takeover attempt, the existence of this provision may adversely affect the prevailing market price of our common shares if it is viewed as discouraging takeover attempts in the future.
We may reduce the voting power of your common shares without your consent.
Under our amended and restated bye-laws, in the event that any U.S. person holds, directly, indirectly or constructively, 9.5% or more of the total voting power of our issued share capital, excluding any U.S. person that held, directly, indirectly or constructively, 9.5% or more of the total voting power of issued share capital immediately prior to the closing of our IPO, the aggregate votes conferred by the common shares held by such person (or by any person through which such U.S. person indirectly or constructively holds shares) will be reduced by our Board of Directors to the extent necessary such that the common shares held, directly, indirectly or constructively, by such U.S. person will constitute less than 9.5% of the voting power of all issued and outstanding shares. RSL, certain of its affiliates, and Vivek Ramaswamy, our founder and former principal executive officer, will not be subject to these provisions. Further, our Board of Directors may determine that shares shall carry different or no voting rights as it reasonably determines, based on the advice of counsel, to be appropriate to (1) avoid the existence of any U.S. person who holds 9.5% or more of the total voting power of our issued share capital or (2) avoid adverse tax, legal or regulatory consequences to us, any subsidiary of ours or any holder of our common shares or its affiliates.
These provisions may discourage potential investors from acquiring a stake or making a significant investment in our company as well as discourage a takeover attempt, which may prevent our shareholders from receiving the benefit of any such transactions as well as adversely affect the prevailing market price of our common shares if viewed as discouraging takeover attempts in the future.
We may become subject to unanticipated tax liabilities and higher effective tax rates.
We are organized under the laws of Bermuda, where we are not subject to any income or withholding taxes. We are centrally managed and controlled in the United Kingdom, and under current U.K. tax law, a company which is centrally managed and controlled in the United Kingdom is regarded as resident in the United Kingdom for taxation purposes. We may also become subject to income, withholding or other taxes in certain jurisdictions by reason of our activities and operations, and it is also possible that taxing authorities in any such jurisdictions could assert that we are subject to greater taxation than we currently anticipate. Any such additional tax liability could adversely affect our results of operations. For example, ASG is our principal operating company for conducting our business and is the entity that holds our intellectual property rights, including AXO-Lenti-PD and nelotanserin. The establishment of this Swiss entity as our principal operating company and the transfer of our intellectual property rights to this entity may result in a higher overall effective tax rate.

81



The intended tax effects of our corporate structure and intercompany arrangements depend on the application of the tax laws of various jurisdictions and on how we operate our business.
We and RSL, our principal shareholder, are incorporated under the laws of Bermuda. We currently have subsidiaries in the United Kingdom, Switzerland and the United States. If we succeed in growing our business, we expect to conduct increased operations through our subsidiaries in various countries and tax jurisdictions in part through intercompany service agreements between us, our majority shareholder, RSL, and our subsidiaries. In that case, our corporate structure and intercompany transactions, including the manner in which we develop and use our intellectual property, will be organized so that we can achieve our business objectives in a tax-efficient manner and in compliance with applicable transfer pricing rules and regulations. If two or more affiliated companies are located in different countries or tax jurisdictions, the tax laws and regulations of each country generally will require that transfer prices be the same as those between unrelated companies dealing at arm's length and that appropriate documentation be maintained to support the transfer prices. While we believe that we operate in compliance with applicable transfer pricing laws and intend to continue to do so, our transfer pricing procedures are not binding on applicable tax authorities. If tax authorities in any of these countries were to successfully challenge our transfer prices as not reflecting arm’s length transactions, they could require us to adjust our transfer prices and thereby reallocate our income to reflect these revised transfer prices, which could result in a higher tax liability to us. In addition, if the country from which the income is reallocated does not agree with the reallocation, both countries could tax the same income, potentially resulting in double taxation. If tax authorities were to allocate income to a higher tax jurisdiction, subject our income to double taxation or assess interest and penalties, it would increase our consolidated tax liability, which could adversely affect our financial condition, results of operations and cash flows.
Significant judgment is required in evaluating our tax positions and determining our provision for income taxes. During the ordinary course of business, there are many transactions and calculations for which the ultimate tax determination is uncertain. For example, our effective tax rates could be adversely affected by changes in foreign currency exchange rates or by changes in the relevant tax, accounting, and other laws, regulations, principles, and interpretations. As we intend to operate in numerous countries and taxing jurisdictions, the application of tax laws can be subject to diverging and sometimes conflicting interpretations by tax authorities of these jurisdictions. It is not uncommon for taxing authorities in different countries to have conflicting views, for instance, with respect to, among other things, the manner in which the arm’s length standard is applied for transfer pricing purposes, or with respect to the valuation of intellectual property. In addition, tax laws are dynamic and subject to change as new laws are passed and new interpretations of the law are issued or applied. For example, on December 22, 2017, an Act to provide for reconciliation pursuant to titles II and V of the concurrent resolution on the budget for fiscal year 2018 (commonly known as the Tax Cuts and Jobs Act) was enacted in the United States, which introduced a comprehensive set of tax reforms. We continue to assess the impact of such tax reform legislation on our business and may determine that changes to our structure, practice or tax positions are necessary in light of the Tax Cuts and Jobs Act. Certain impacts of this legislation have been taken into account, including the reduction of the U.S. corporate income tax rate from the previous 35 percent to 21 percent. The Tax Cuts and Jobs Act, in conjunction with the tax laws of other jurisdictions in which we operate, however, may require consideration of changes to our structure and the manner in which we conduct our business. Such changes may nevertheless be ineffective in avoiding an increase in our consolidated tax liability, which could adversely affect our financial condition, results of operations and cash flows.
Changes in our effective tax rate may reduce our net income in future periods.
Our tax position could be adversely impacted by changes in tax rates, tax laws, tax practice, tax treaties or tax regulations or changes in the interpretation thereof by the tax authorities in Europe (including the United Kingdom and Switzerland), the United States, Bermuda, and other jurisdictions as well as being affected by certain changes currently proposed by the Organisation for Economic Co-operation and Development and their action plan on Base Erosion and Profit Shifting.  Such changes may become more likely as a result of recent economic trends in the jurisdictions in which we operate, particularly if such trends continue. If such a situation was to arise, it could adversely impact our tax position and our effective tax rate. Failure to manage the risks associated with such changes, or misinterpretation of the laws providing such changes, could result in costly audits, interest, penalties, and reputational damage, which could adversely affect our business, results of our operations, and our financial condition.
Our actual effective tax rate may vary from our expectation and that variance may be material. A number of factors may increase our future effective tax rates, including: (1) the jurisdictions in which profits are determined to be earned and taxed; (2) the resolution of issues arising from any future tax audits with various tax authorities; (3) changes in the valuation of our deferred tax assets and liabilities; (4) increases in expenses not deductible for tax purposes, including transaction costs and impairments of goodwill in connection with acquisitions; (5) changes in the taxation of share-based compensation; (6) changes in tax laws or the interpretation of such tax laws, and changes in generally accepted accounting principles; and (7) challenges to the transfer pricing policies related to our structure.

82



U.S. holders of our common shares may suffer adverse tax consequences if we are characterized as a passive foreign investment company.
Generally, if, for any taxable year, at least 75% of our gross income is passive income, or at least 50% of the value of our assets is attributable to assets that produce passive income or are held for the production of passive income, including cash, we would be characterized as a passive foreign investment company ("PFIC") for U.S. federal income tax purposes. For purposes of these tests, passive income includes dividends, interest, and gains from the sale or exchange of investment property and rents and royalties other than rents and royalties which are received from unrelated parties in connection with the active conduct of a trade or business. Additionally, a look-through rule generally applies with respect to 25% or more owned subsidiaries. If we are characterized as a PFIC, U.S. holders of our common shares may suffer adverse tax consequences, including having gains realized on the sale of our common shares treated as ordinary income rather than capital gain, the loss of the preferential tax rate applicable to dividends received on our common shares by individuals who are U.S. holders, and having interest charges apply to distributions by us and the proceeds of sales of our common shares.
Our status as a PFIC will depend on the nature and composition of our income and the nature, composition and value of our assets from time to time. The 50% passive asset test described above is generally based on the fair market value of each asset, with the value of goodwill and going concern value determined in large part by reference to the market value of our common shares, which may be volatile. Our status may also depend, in part, on how quickly we utilize the cash proceeds from our IPO and subsequent financings in our business. With respect to the taxable year that ended on March 31, 2018, we believe that we were not a PFIC, however, with respect to foreseeable future taxable years, because the PFIC tests are based upon the value of our assets, including any goodwill and going concern value, and the nature and composition of our income and assets, which cannot be known at this time, we cannot predict whether we will or will not be classified as a PFIC. Because the determination of whether we are a PFIC for any taxable year is a fact-intensive determination made annually after the end of each taxable year, and because certain aspects of the PFIC rules are uncertain, we cannot provide any assurances regarding our PFIC status for the current or future taxable years.
In our current taxable year ending March 31, 2019, we expect to implement structures and arrangements intended to mitigate the possibility that we will be classified as a PFIC. The failure or inability to implement such structures or arrangements may have an adverse impact on the determination of whether we are classified as a PFIC.
U.S. holders that own 10 percent or more of the vote or value of our common shares may suffer adverse tax consequences because we and/or any of our non-U.S. subsidiaries are expected to be characterized as a controlled foreign corporation ("CFC"), under Section 957(a) of the U.S. Internal Revenue Code of 1986, as amended ("the Code").
A non-U.S. corporation is considered a CFC if more than 50 percent of (1) the total combined voting power of all classes of stock of such corporation entitled to vote, or (2) the total value of the stock of such corporation, is owned, or is considered as owned by applying certain constructive ownership rules, by United States shareholders (U.S. persons who own stock representing 10% or more of the vote or, for taxable years of non-U.S. corporations beginning after December 31, 2017 and for taxable years of shareholders with or within which such taxable years of non-U.S. corporations end, 10% or more of the value) on any day during the taxable year of such non-U.S. corporation. Certain United States shareholders of a CFC generally are required to include currently in gross income such U.S. shareholders’ share of the CFC’s "Subpart F income", a portion of the CFC’s earnings to the extent the CFC holds certain U.S. property, and a portion of the CFC's "global intangible low-taxed income" (as defined under Section 951A of the Code). Such United States shareholders are subject to current U.S. federal income tax with respect to such items, even if the CFC has not made an actual distribution to such shareholders. "Subpart F income" includes, among other things, certain passive income (such as income from dividends, interests, royalties, rents and annuities or gain from the sale of property that produces such types of income) and certain sales and services income arising in connection with transactions between the CFC and a person related to the CFC. "Global intangible low-taxed income" may include most of the remainder of a CFC’s income over a deemed return on its tangible assets.
As a result of certain changes in the U.S. tax law introduced by the Tax Cuts and Jobs Act, we believe that we and our non-U.S. subsidiaries are classified as CFCs in the current taxable year. For U.S. holders who hold 10% or more of the vote or value of our common shares, this may result in adverse U.S. federal income tax consequences, such as current U.S. taxation of Subpart F income and of any such shareholder’s share of our accumulated non-U.S. earnings and profits (regardless of whether we make any distributions), taxation of amounts treated as global intangible low-taxed income under Section 951A of the Code with respect to such shareholder, and being subject to certain reporting requirements with the U.S. Internal Revenue Service. Any such U.S. holder who is an individual generally would not be allowed certain tax deductions or foreign tax credits that would be allowed to a U.S. corporation. If you are a U.S. holder who holds 10% or more of the vote or value of our common shares, you should consult your own tax advisors regarding the U.S. tax consequences of acquiring, owning, or disposing our common shares and the impact of the Tax Cuts and Jobs Act, especially the changes to the rules relating to CFCs.

83



Item 2.          Unregistered Sales of Equity Securities and Use of Proceeds
None.

Item 3.          Defaults Upon Senior Securities.
None.

Item 4.          Mine Safety Disclosures.
Not applicable.

Item 5.          Other Information.
None.

Item 6.                 Exhibits.



84



 
 
 
Incorporated by Reference
Exhibit
Number
 
Description of Document
Schedule/Form
File No.
Exhibit
Filing Date
 
 
 
 
 
 
 
3.1
 
S-1
333-204073
3.1
05/11/2015
 
 
 
 
 
 
 
3.2
 
S-1
333-204073
3.2
05/11/2015
 
 
 
 
 
 
 
3.3
 
8-K
001-37418
3.1
12/21/2017
 
 
 
 
 
 
 
10.1*
 
 
 
 
 
 
 
 
 
 
 
 
10.2*
 
 
 
 
 
 
 
 
 
 
 
 
10.3*+
 
 
 
 
 
 
 
 
 
 
 
 
10.4
 
8-K
001-37418
1.1
06/22/2018
 
 
 
 
 
 
 
31.1*
 
 
 
 
 
 
 
 
 
 
 
 
31.2*
 
 
 
 
 
 
 
 
 
 
 
 
32.1*#
 
 
 
 
 
 
 
 
 
 
 
 
32.2*#
 
 
 
 
 
 
 
 
 
 
 
 
101.INS*
 
XBRL Instance Document
 
 
 
 
 
 
 
 
 
 
 
101.SCH*
 
XBRL Taxonomy Extension Schema
 
 
 
 
 
 
 
 
 
 
 
101.CAL*
 
XBRL Taxonomy Extension Calculation Linkbase
 
 
 
 
 
 
 
 
 
 
 
101.DEF*
 
XBRL Taxonomy Extension Definition Linkbase
 
 
 
 
 
 
 
 
 
 
 
101.LAB*
 
XBRL Taxonomy Extension Label Linkbase
 
 
 
 
 
 
 
 
 
 
 
101.PRE*
 
XBRL Taxonomy Extension Presentation Linkbase
 
 
 
 
 
*    Filed herewith.
+    Portions of this exhibit (indicated by asterisks) have been omitted pursuant to a request for confidential treatment and have been separately filed with the Securities and Exchange Commission.
#    These certifications are being furnished solely to accompany this Quarterly Report on Form 10-Q pursuant to 18 U.S.C. Section 1350, and are not being filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, and are not to be incorporated by reference into any filing of the Registrant, whether made before or after the date hereof, regardless of any general incorporation language in such filing.

85



SIGNATURES
 Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
 
 
 
AXOVANT SCIENCES LTD.
 
 
 
 
 
 
 
 
 
 
By:
/s/ Gregory Weinhoff
Date
August 7, 2018
 
Gregory Weinhoff
(Duly Authorized Officer and Principal Financial Officer)
 
 
 
 
 
 
 
 
 




86

Exhibit 10.1
Execution Version







AMENDED AND RESTATED INFORMATION SHARING AND COOPERATION AGREEMENT

by and among AXOVANT SCIENCES LTD.,
AND

ROIVANT SCIENCES LTD.


Dated as of June 5, 2018



TABLE OF CONTENTS
 
 
Page
Article 1 DEFINITIONS AND INTERPRETATION
 
Section 1.01
Definitions.
1

Section 1.02
Additional Defined Terms
8

Section 1.03
Other Definitional and Interpretive Matters
9

Article 2 RIGHT OF FIRST REVIEW
 
Section 2.01
.
10

Article 3 FINANCIAL REPORTING AND DISCLOSURE COVENANTS
 
Section 3.01
Financial Reporting and Controls
10

Section 3.02
Private Company Information Rights
20

Article 4 COMPLIANCE COVENANTS
 
Section 4.01
Compliance
20

Article 5 EXCHANGE OF INFORMATION; CONFIDENTIALITY
 
Section 5.01
Privilege
26

Section 5.02
Ownership of Information.
26

Section 5.03
Record Retention
26

Section 5.04
Limitation of Liability
26

Section 5.05
Confidentiality
26

Section 5.06
Protective Arrangements
29

Section 5.07
Preservation of Legal Privileges
29

Article 6 TAX MATTERS.
 
Section 6.01
PFIC
30

Section 6.02
QEF Information
31

Article 7 DISPUTE RESOLUTION
 
Section 7.01
Limitation on Monetary Damages Equitable Remedies.
31

Section 7.02
Disputes.
31

Section 7.03
Escalation; Mediation.
32

Section 7.04
Binding Arbitration.
33

Article 8 FURTHER ASSURANCES
 
Section 8.01
Further Assurances.
35

Article 9 MISCELLANEOUS
 
Section 9.01
Counterparts; Entire Agreement; Conflicting Agreements.
36

Section 9.02
No Construction Against Drafter.
37

Section 9.03
Governing law.
37

Section 9.04
Assignability
37

Section 9.05
Notices
37

Section 9.06
Severability
40

Section 9.07
Force Majeure
41

Section 9.08
Headings.
41

Section 9.09
Termination; Survival.
41

Section 9.10
Waivers of Default.
41

Section 9.11
Specific Performance
41

Section 9.12
Amendments
42

Section 9.13
Waiver of Jury Trial
42




Section 9.14
Limitation on Monetary Damages
43

Section 9.15
Indemnity and Expenses
43

Section 9.16
Maintenance of Insurance
44

Section 9.17
No Third-Party Beneficiaries
45

Section 9.18
Expenses
45





AMENDED AND RESTATED
INFORMATION SHARING AND COOPERATION AGREEMENT

This    AMENDED    AND    RESTATED    INFORMATION    SHARING    AND COOPERATION AGREEMENT, dated as of June 5, 2018 (this “Agreement”), is by and between Roivant Sciences Ltd., a Bermuda exempted limited company (“Roivant”), and Axovant Sciences Ltd., a Bermuda exempted limited company (the “Company”), (with each of the Company and Roivant, a “Party” and together, the “Parties”).

RECITALS

WHEREAS, Roivant is the beneficial owner of a majority of the issued and outstanding stock of the Company;

WHEREAS, the Parties entered into that certain Information Sharing and Cooperation Agreement(the “ Original Agreement ”), dated March 18, 2015 (the “ Original Effective Date ”), in connection with the Company’s initial public offering; and

WHEREAS, the Parties wish to amend and restate the Original Agreement in its entirety, effective as of the Closing Date (as defined in that certain Share Purchase Agreement, dated as of June 5, 2018, by and between Roivant and the Company) (the “ Effective Date ”).

NOW, THEREFORE, in consideration of the covenants and agreements contained herein, the parties hereto agree as follows:

ARTICLE 1
DEFINITIONS AND INTERPRETATION
Section 1.01     Definitions .    The following terms, as used herein, have the following
meanings:

Affiliate ” means, with respect to any Person, any other Person that, directly or indirectly through one or more intermediaries, controls, is controlled by or is under common control with such Person, including any general partner, managing member, officer or director of such Person or any venture capital, private equity or other investment fund or account now or hereafter existing that is

1


controlled by one or more general partners or managing members of, or shares the same management company or investment advisor with, such Person.

Applicable Money Laundering Laws ” means Laws applying to the Company and, in the case of each Subsidiary of the Company, the Laws applying to such Subsidiary, prohibiting money laundering.

Board ” means the Board of Directors of the Company.

Business Day ” means any day other than a Saturday or Sunday on which banks are open for business in New York, New York, London, United Kingdom, and Bermuda.


2


Bye-laws ” means the Amended and Restated Bye-laws of the Company, as the same may be amended from time to time.

Common Shares ” means the common shares of the Company.

Compliance Officer ” means, with respect to any Person, the individual on the senior management of such Person who has been delegated the responsibility for ensuring compliance with all Specified Laws.

Compliance Program ” means a quality and regulatory compliance program, overseen by the Compliance Oversight Committee, for ensuring compliance by the Company and its Subsidiaries with the Specified Laws.

Control ” means the possession, directly or indirectly, of the power to direct or cause the direction of the management and policies of a person, whether through the ownership of voting securities, by contract or otherwise, with “Controlled” having a correlative meaning.

Data Privacy and Cybersecurity Rules and Regulations ” means, collectively, all of the following to the extent relating to data privacy, data protection or cybersecurity (including the collection, storage, use, maintenance, access, disclosure, processing, security, transfer, aggregation, confidentiality, integrity and availability) of Personal Information, and confidential, proprietary and/or business information: (i) all Laws, encompassing U.S. state and federal, regional and international data privacy and cybersecurity laws, regulations and guidance including but not limited to the Health Insurance Portability and Accountability Act, the Gramm-Leach- Bliley Act, the Federal Information Security Management Act, the Fair Credit Reporting Act, the Fair and Accurate Credit Transaction Act, the Federal Trade Commission Act, the Privacy Act of 1974, the CAN-SPAM Act, the Telephone Consumer Protection Act, the Telemarketing and Consumer Fraud and Abuse Prevention Act, Children’s Online Privacy Protection Act, the EU Data Protection Directive, the EU General Data Protection Regulation, the Canadian Personal Information Protection and Electronic Documents Act, the Swiss Federal Act on Data Protections, and U.S. state data privacy, cybersecurity and data breach notification laws, (ii) the Company’s own rules, policies, procedures and public statements (including all data protection and privacy policies and related notices, (iii) industry-recognized privacy and cybersecurity standards (such as NAI, ISO 27001, COBIT, NIST, HIPAA, PCI-DSS, ITAR, etc.), and (iv) contracts into which the Company has entered or by which it is otherwise bound.


3


Equity Securities ” means, without duplication, (a) the Common Shares, (b) any other class of equity security or equity-linked security issued by the Company or any corporate successor thereto and (c) any other securities convertible into or exchangeable or exercisable for, or options, warrants or other rights to acquire, Common Shares, or any other equity or equity-linked securities issued by the Company or any corporate successor thereto.

Exchange Act ” means the U.S. Securities Exchange Act of 1934, as amended, and the rules and regulations promulgated from time to time thereunder.

FCPA ” means the U.S. Foreign Corrupt Practices Act of 1977, as amended. “ FDA ” means the U.S. Food and Drug Administration.


4


GAAP ” means United States generally accepted accounting principles.

Government Official ” means (a) an officer or employee of any national, regional, local or other component of government, (b) a director, officer or employee of any entity in which a government or any component of a government possesses a majority or controlling interest; (c) a candidate for public office; (d) a political party or political party official; (e) an officer or employee of a public international organization (e.g., the European Commission or World Bank); and (f) any individual who is acting in an official capacity for any government, component of a government, political party or public international organization, even if such individual is acting in that capacity temporarily and without compensation.

Health-Related Requirements ” means (a) the federal Laws applicable to the activities of a pharmaceutical or biological product manufacturer, including but not limited to federal health care program and FDA requirements relating to research; development; interactions with health care professionals, patient advocacy or assistance organizations, charitable organizations, and professional societies; data integrity and security; labeling; marketing; sale; distribution; import; export; product pricing and reimbursement; Quality Management Systems; price, safety, and other reporting obligations; safety monitoring; or exclusion and debarment (collectively, “ manufacturer activities ”); (b) the U.S. anti-corruption Laws (e.g., the FCPA) applicable to manufacturer activities occurring outside the United States; and (c) non-U.S. laws that are equivalent to the requirements set forth in clauses (a) and (b) of this definition (e.g., the UKBA and any other applicable Laws prohibiting bribery and corruption).

IPO ” means the underwritten initial public offering of Equity Securities (it being understood that an IPO shall not include a registration effected solely to implement an employee benefit plan, a merger or other business combination or a registration on Form S-4, Form S-8 or any substantially equivalent or successor form thereto).

Law ” means any national, federal, state, provincial, local or foreign law, statute, code, ordinance, rule, regulation, order, judgment, writ, stipulation, award, injunction, decree or arbitration award or finding.

Local ABAC Laws ” means local Laws applying to the Company and, in the case of each Subsidiary of the Company, the Laws applying to such Subsidiary, prohibiting bribery and corruption.


5


Person ” means an individual, company, corporation, limited liability company, partnership, association, joint stock company, trust, joint venture, unincorporated organization or other entity or organization, including a governmental authority.

Quality Management Systems ” means those systems supporting the development and manufacture of pharmaceutical drug substances (i.e., active pharmaceutical ingredients (APIs)) and drug products, including biotechnology and biological products, throughout the product lifecycle.

Regulatory and Governance Requirements ” means all (a) ethics, conduct, conflict, insider trading and other internal policies and guidelines applicable generally to Roivant or any of its Representatives and (b) applicable regulatory, internal controls (including internal controls with


6


respect to financial reporting and remediation of any deficiencies), audit, compliance, record keeping, document retention, financial reporting, tax and legal requirements applicable to Roivant or any of its Representatives, in each case, as amended or updated from time to time.

Reportable Event ” means any event that may (a) represent a substantial deviation from applicable policies, procedures, systems or controls regarding Specified Laws; or (b) represent a violation of any Specified Law that could have a material compliance, regulatory, legal financial, reputational or safety impact on the Company, its Affiliates, and its or their stakeholders or patients, in each case, as reasonably determined by the Company.

Representatives ” means, with respect to a Person, such Person’s directors, officers, employees, agents, legal counsel, financial advisors and other representatives.

Sanctions ” means economic or financial sanctions or trade embargoes, including
(a) United Nations sanctions imposed pursuant to any United Nations Security Council Resolution;
(b) U.S. sanctions administered by the Office of Foreign Assets Control of the U.S. Department of the Treasury, the U.S. Department of State, the U.S. Department of Commerce or any other U.S. government authority or department; (c) EU restrictive measures implemented pursuant to any EU Council or Commission Regulation or Decision adopted pursuant to a Common Position in furtherance of the EU’s Common Foreign and Security Policy; (d) UK sanctions adopted by the Terrorist Asset-Freezing etc. Act 2010 or other legislation and statutory instruments enacted pursuant to the United Nations Act 1946 or the European Communities Act 1972 or enacted by or pursuant to other Laws; and (e) any other trade, economic or financial sanctions Laws, embargoes or restrictive measures administered, enacted or enforced by any authority, government or official institution as applicable to Company and each of its Subsidiaries or any transaction in which Company or each Subsidiary of the Company is engaged.

Sarbanes-Oxley Act ” means the Sarbanes-Oxley Act of 2002, as amended, and the rules and regulations promulgated from time to time thereunder.

SEC ” means the U.S. Securities and Exchange Commission.

Securities Act ” means the U.S. Securities Act of 1933, as amended, and the rules and regulations promulgated from time to time thereunder.


7


Specified Laws ” means (a) the UKBA or FCPA; (b) applicable trade, economic or financial sanctions Laws, embargoes or other restrictive measures, including (i) Local ABAC Laws, (ii) Applicable Money Laundering Laws, (iii) Sanctions and (iv) applicable Laws prohibiting fraud, tax evasion, insider dealing and market manipulation; (c) applicable Health- Related Requirements; (d) applicable securities Laws, including the Exchange Act, the Sarbanes- Oxley Act and the Securities Act; and (e) applicable Data Privacy and Cybersecurity Rules and Regulations (f) all other Laws of any jurisdiction that are similar to the Laws described in the foregoing clauses (a) – (e).

Subsidiary ” means, with respect to any specified Person, any other Person (a) Controlled by such first Person or (b) of which at least a majority of the securities or ownership interests having by their terms ordinary voting power to elect a majority of the board of directors or other


8


persons performing similar functions is directly or indirectly owned or controlled by such first Person and/or by one or more of its Subsidiaries.

UKBA ” means the UK Bribery Act 2010, as amended.

Section 1.02     Additional Defined Terms . Each of the following terms is defined in the Section set forth opposite such term:

TERM
SECTION
 
 
Acceptance Notice
2.01
Agreement
Preamble
Annual Financial Statements
3.01(c)
Company
Preamble
Dementia Opportunity
2.01
Effective Date
Recitals
Expert Councils
4.01(e)
Indemnified Liabilities
9.15(a)(i)
Indemnitees
9.15(a)(i)
Offer Notice
2.01
Original Agreement
Recitals
Original Effective Date
Recitals
Policies
4.01(a)
Privilege
5.01
Quarterly Financial Statements
3.01(b)(i)
Roivant
Preamble

Section 1.03 Other Definitional and Interpretive Matters . Unless otherwise expressly provided herein, for purposes of this Agreement, the following rules of interpretation shall apply:

(a)      Calculation of Time . When calculating the period before which, within which or after which any act is to be done or step taken pursuant to this Agreement, the date that is the reference date in calculating such period shall be excluded. If the last day of such period is not a Business Day, the period in question shall end on the next succeeding Business Day.

(b)      Dollars . Any reference in this Agreement to “$” means U.S. dollars.


9


(c)      Annexes/Exhibits/Schedules . The Annexes, Exhibits and Schedule to this Agreement are hereby incorporated and made a part hereof and are an integral part of this Agreement. Any capitalized terms used in any Annex, Exhibit or Schedule but not otherwise defined therein shall be defined as set forth in this Agreement.

(d)      Gender and Number . Any reference in this Agreement to gender shall include all genders, and words imparting the singular number only shall include the plural and vice versa.

(e)      Herein . The words “herein,” “hereof” and “hereunder” refer to this Agreement as a whole and not merely to a subdivision in which such words appear unless the context otherwise requires.


10


(f)      Other . The words “include,” “includes” and “including” when used herein shall be deemed in each case to be followed by the words “without limitation.” The word “extent” in the phrase “to the extent” means the degree to which a subject or other thing extends, and such phrase does not mean simply “if.”

ARTICLE 2

RIGHT OF FIRST REVIEW


Section 2.01 As consideration for entering into this Agreement, Roivant agrees not to pursue any potential dementia-related product or investment opportunity (a “ Dementia Opportunity ”) without first notifying the Company (the “ Offer Notice ”) of its intention to do so, such Offer Notice to include reasonable details of such opportunity to the extent known or otherwise made available to Roivant, and offering the Company the opportunity to evaluate and independently pursue the Dementia Opportunity. The Company shall have 30 days after receipt of the Offer Notice to inform Roivant of its intent to pursue in good faith the Dementia Opportunity subject to the Offering Notice (the “ Acceptance Notice ”) in which case Roivant shall not pursue such Dementia Opportunity and shall refer such Dementia Opportunity to the Company. If the Company has not entered into a definitive agreement with respect to the Dementia Opportunity described in the Offer Notice within 60 days of the Acceptance Notice, Roivant shall be free to pursue such Dementia Opportunity.

ARTICLE 3

FINANCIAL REPORTING AND DISCLOSURE COVENANTS

Section 3.01 Financial Reporting and Controls . The Parties agree that they will comply with the requirements set forth in this Section 3.01 , (A) with respect to Sections 3.01 (d), (e), (h), (i), (j), (k), (l), (m) and (n) from and after the Effective Date, and (B) with respect to Sections 3.01 (a), (b), (c), (f), and (g), from and after such time that Roivant (i) notifies the Company that it is actively engaging in the preparation of a registration statement to be filed under the Securities Act for an initial public offering of its securities or (ii) has a class of securities registered under Section 13(a) or 15(d) of the Exchange Act and Roivant is required (x) by GAAP to consolidate the results of operations and financial position of the Company, (y) to account for its investment in the Company under the equity method of accounting (determined in accordance with GAAP and consistent with SEC reporting requirements) or (z) to otherwise include separate financial statements of the Company in its filings with the SEC pursuant to any rule of the SEC.

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(a) Disclosure of Financial Controls . In connection with the filing of Roivant’s annual and quarterly reports under the Exchange Act or any investigations of prior periods, the Company shall cause its principal executive officer and principal financial officer to provide to Roivant and its Representatives (A) on a timely basis, if this provision is applicable by virtue of Section 3.01(B)(ii) (x) and (B) on a timely basis and if reasonably requested by Roivant, if this provision is applicable by virtue of Sections 3.01(B)(i) or (ii) (y) or (z), (1) certifications to Roivant corresponding to those required under Sections 302 and 906 of the Sarbanes-Oxley Act, (2) any certificate that may be reasonably necessary for Roivant to satisfy the requirements applicable to


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it under Section 404 of the Sarbanes-Oxley Act, (3) any certificates or other written information that the Company’s principal executive officer or principal financial officer received as support for the certificates provided to Roivant and (4) a reasonable opportunity to discuss with the Company’s principal financial officer and other appropriate officers and employees of the Company any issues reasonably related to the foregoing.

(b) Quarterly Financial Statements .

(i)      As soon as reasonably practicable and no later than 15 days before the date by which Roivant is required to file a quarterly report on Form 10-Q if this provision is applicable by virtue of Section 3.01(B) (ii)(x) above or 10 days before the date by which Roivant is required to file a quarterly report on Form 10-Q if this provision is applicable by virtue of Section 3.01(B)(i) or (ii) (y) or (z) above, the Company will deliver to Roivant and its Representatives reasonably complete drafts of (A) the consolidated financial statements of the Company (and notes thereto) for the quarterly periods and for the period from the beginning of the current fiscal year to the end of such quarter, setting forth in each case in comparative form for each such fiscal quarter of the Company the consolidated figures (and notes thereto) for the corresponding quarter and periods of the previous fiscal year prepared in accordance with Article 10 of Regulation S-X and GAAP and (B) a discussion and analysis by management of the Company’s financial condition and results of operations for such fiscal period, including, without limitation, an explanation of any material period-to-period change and any off-balance sheet transactions, prepared in accordance with Item 303(b) of Regulation S-K. The information set forth in (A) and (B) above is referred to in this Agreement as the “ Quarterly Financial Statements ”. As soon as reasonably possible and no later than 5 days before the date by which Roivant is required to file a quarterly report on Form 10-Q, the Company will deliver to Roivant and its Representatives the final form of the Quarterly Financial Statements; provided , however , that the Company may continue to revise such Quarterly Financial Statements prior to its filing thereof in order to make corrections, updates and changes, which corrections, updates and changes, if substantive, will be delivered by the Company to Roivant as soon as reasonably possible. At Roivant’s request, the Company’s Representatives will consult and discuss with Roivant’s Representatives any such corrections, updates and changes. To the extent that the fiscal year of Roivant is not the same as the fiscal year of the Company or Roivant is not subject to reporting obligations under Section 13(a) or 15(d) of the Exchange Act, the obligation to deliver Quarterly Financial Statements before the date by which Roivant is required to file its quarterly report on Form 10-Q shall be determined based on the date by which the Company is required to file its quarterly report on Form 10-Q.

(ii)      As soon as reasonably practicable and no later than 45 days after the end of its fiscal year, the Company will deliver to Roivant and its Representatives its consolidated

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financial statements (and notes thereto) for the last quarter of its fiscal year, setting forth in each case in comparative form for such fiscal quarter of the Company the consolidated figures (and notes thereto) for the corresponding quarter of the previous fiscal year prepared in accordance with Article 10 of Regulation S-X and GAAP; provided , however , that the Company may continue to revise such financial statements in order to make corrections, updates and changes in connection with the preparation of its audited annual


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financial statements, which corrections, updates and changes, if substantive, will be delivered by the Company to Roivant as soon as reasonably possible.

(c) Annual Financial Statements . As soon as reasonably practicable and no later than 45 days after the end of its fiscal year if this provision is applicable by virtue of Section 3.01(B)(ii) (x) above or 55 days after the end of its fiscal year if this provision is applicable by virtue of Section 3.01(B)(i) or (ii) (y) or (z) above, the Company will deliver to Roivant and its Representatives reasonably complete drafts of (i) the consolidated financial statements of the Company (and notes thereto) for such year, setting forth in each case in comparative form the consolidated figures (and notes thereto) for the previous fiscal years, prepared in accordance with Article 10 of Regulation S-X and GAAP and (ii) a discussion and analysis by management of the Company’s financial condition and results of operations for such year, including, without limitation, an explanation of any material period-to-period changes and any off-balance sheet transactions, prepared in accordance with Item 303(a) and 305 of Regulation S-K. The information set forth in (i) and (ii) above is referred to in this Agreement as the “ Annual Financial Statements ”. As soon as reasonably possible and no later than 15 days before the date by which Roivant is required to file its annual report on Form 10-K if this provision is applicable by virtue of Section 3.01(B)(ii) (x) above or 10 days before the date by which Roivant is required to file its annual report on Form 10-K if this provision is applicable by virtue of Section 3.01(B)(ii) (y) or (z) above, the Company will deliver to Roivant and its Representatives the final form of the Annual Financial Statements and an opinion on the Annual Financial Statements by the Company’s independent registered public accountants (the “ Company Auditors ”); provided , however , that the Company may, if necessary, continue to revise such Annual Financial Statements prior to the filing thereof in order to make corrections, updates and changes, which corrections, updates and changes, if substantive, will be delivered by the Company to Roivant as soon as reasonably possible. At Roivant’s request, the Company’s Representatives will consult and discuss with Roivant’s Representatives any such corrections, updates and changes. To the extent that the fiscal year of Roivant is not the same as the fiscal year of the Company or Roivant is not subject to reporting obligations under Section 13(a) or 15(d) of the Exchange Act, the obligation to deliver Annual Financial Statements before the date by which Roivant is required to file its annual report on Form 10-K shall be determined based on the date by which the Company is required to file its quarterly report on Form 10-K.

(d) Supplemental Information . Roivant may reasonably request, and within a reasonable period of time agreed to by Roivant and the Company following such request, the Company shall, at Roivant’s sole cost and expense, make available to Roivant and its Representatives other supplemental information on a monthly, quarterly or annual basis necessary or advisable in order to satisfy Roivant’s financial reporting requirements pursuant to 3.01(B)(ii) above, and other Regulatory and Governance Requirements, the form, substance and timing of such supplemental information to be agreed by Roivant and the Company in advance.


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(e) Conformance of Financial Statements . Subject to the other terms in this Agreement, the Company shall not make or adopt any significant changes to its accounting estimates or accounting policies and principles from those in effect on the Effective Date to the extent that such changes would significantly impact Roivant’s financial statements. Notwithstanding the previous sentence, nothing in this Agreement shall prevent the Company making those changes to its accounting estimates or accounting policies and principles if such changes are required by GAAP


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or which the audit committee of the Company determines are necessary or appropriate for the proper presentation of the Company’s financial statements; provided , however , that the Company shall first consult with Roivant.

(f) Press Releases and Similar Information . The Company and Roivant will consult with each other as to the timing of their annual and quarterly earnings releases and any interim financial guidance for a current or future period and, to the extent reasonably possible. If the Company and Roivant are unable to agree as to such timing, then Roivant and the Company shall each make reasonable efforts to issue their respective annual and quarterly earnings releases at approximately the same time on the same date, which will include for these purposes during the same period of time beginning after the close of market on one day and ending just prior to the opening of market on the next day. Roivant and the Company agree to consult with each other as to the timing of their respective earnings release conference calls.

(g) Cooperation on Filings . The Company agrees to provide to Roivant and its Representatives, and to instruct the Company Auditors to provide to Roivant and its Representatives, all material information with respect to the Company that Roivant reasonably requires in connection with the preparation by Roivant of its Quarterly Reports on Form 10-Q, Annual Reports to Shareholders, Annual Reports on Form 10-K, any Current Reports on Form 8- K and any registration statements, or other filings made by Roivant with the SEC, any national securities exchange or otherwise made publicly available with respect to the disclosures pertaining to the Company (collectively, the “ Roivant Public Filings ”). The Company and Roivant agree to reasonably cooperate with each other with respect to the requesting and furnishing of such required information in order to enable Roivant to file all Roivant Public Filings within the deadlines as required by applicable law. The Company will cause the Company Auditors (as defined below) to consent to any reference to them as experts in any Roivant Public Filings required under any law, rule or regulation. In addition, Roivant shall provide to the Company necessary and appropriate information that the Company reasonably requires, to the extent Roivant has such information and the Company does not, in connection with required filings made by the Company to a reasonably applicable governmental authority.

(h) Access to the Company Auditors . The Company will authorize the Company Auditors to make reasonably available to Roivant’s auditors both the personnel who performed, or are performing, the annual audit and quarterly reviews of the Company and work papers related to the annual audit and quarterly reviews of the Company, in all cases within a reasonable time prior to Roivant’s auditors opinion date, so that Roivant’s auditors are able to perform the procedures they consider necessary to take responsibility for the work of the Company Auditors as it relates to Roivant’s auditors report on Roivant’s statements.


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(i) Access to Records . If Roivant determines in good faith that there may be a material inaccuracy in the Company’s financial statements or deficiency or inadequacy in the Company’s internal accounting controls or operations that could reasonably be expected to materially impact Roivant’s financial statements, and at Roivant’s request, the Company will provide Roivant’s internal auditors with reasonable access to the Company’s books and records so that Roivant may conduct reasonable audits relating to the financial statements provided by the Company under this Agreement, as well as to the internal accounting controls and operations of the Company.


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(j) Tax Information . Each Party shall make available to the other and its Representatives all information relating to such Party or any of its Subsidiaries necessary or appropriate to enable the other Party to prepare its federal, state, local and foreign income tax returns; provided that Roivant and its Representatives shall have no obligation to provide information about (A) its directors or investors or (B) its Subsidiaries other than Axovant and Axovant’s subsidiaries. Such information shall be prepared by the Party making it available at its sole cost and expense, and each Party shall make such information available to the other Party and its Representatives with reasonable promptness in light of the timing applicable to the purpose for which such information is to be used.

(k) Compliance Inspection Rights . Without limiting the generality of the requirements of clause (m) of this Section 3.01 , the Company shall provide Roivant and its Representatives with the right to visit and inspect any of the offices and properties of the Company and its Subsidiaries and inspect the books and records of the Company and its Subsidiaries and controlled Affiliates as they relate to Specified Laws, as well as to review and make copies of correspondence and other documents, however sent or received, possessed by the Company and/or the Company’s Subsidiaries and controlled Affiliates pertaining to compliance with the Policies and Specified Laws, at such times as a Reportable Event has been communicated to the Company’s Board of Directors for the purpose of verifying and evaluating the Company’s and its Subsidiaries’ compliance with the Company’s Compliance Program, and to make appropriate officers and directors of the Company and its Subsidiaries available at such times as reasonably requested by Roivant for consultation with Roivant and its Representatives with respect to matters relating to the Compliance Program.

(l) Shareholder Information Rights . The Company shall, and shall cause each of its Subsidiaries to, promptly upon request, at its sole cost and expense, (A) make available to Roivant and its Representatives such information, documents and other materials, whether current, historical or prospective, produced by, relating to or otherwise in the possession of the Company or any of its Subsidiaries as Roivant may from time to time reasonably request and (B) give Roivant and its Representatives (x) the right to examine and make copies of or extracts from the any records of the Company or any of its Subsidiaries for any reasonable purpose, (y) reasonable access to the Company’s and its Subsidiaries’ offices, properties, and employees and (z) the reasonable opportunity to discuss any matters with the Company’s and its Subsidiaries’ senior management, in the case of each of clauses (A) and (B) in connection with any proper purpose.

(m) Provision of Information . The Company shall provide, or cause to be provided, to Roivant, as soon as reasonably practicable after request therefor, confirmation as to whether the Company is in possession of information that would reasonably be considered to be material nonpublic information with respect to the Company under applicable U.S. securities laws, and sufficient additional information as is necessary, in the reasonable judgment of Roivant and its

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counsel, to determine whether such information is material with respect to Roivant under applicable U.S. securities laws.

(n) Fiscal Year. The Company shall not change its fiscal year without the prior written consent of Roivant.


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Section 3.02 Private Company Information Rights . If the Company is not subject to the requirements of Section 3.01 (a), (b), (c), (f), and (g), then the Company shall make available to Roivant:

(i)      consolidated annual financial statements, audited by an accounting firm of international standing and reputation, as soon as practicable, and in any event within 75 Business Days after the end of each fiscal year;

(ii)      unaudited consolidated quarterly financial statements, as soon as practicable, and in any event within 40 Business Days after the end of each fiscal quarter;

(iii)      an annual budget, as soon as practicable, and in any event at least 20 Business Days prior to the beginning of a fiscal year;

(iv)      the Company’s, and each of its Subsidiaries’, capitalization table from time to time, and in any event at least once quarterly within 10 Business Days after the end of each fiscal quarter;

Notwithstanding the foregoing, documents required to be delivered under Section 3.02(i) and (ii), to the extent any such documents are included in materials otherwise filed with the SEC shall be deemed to have been delivered on the date on which the Company files such documents with the SEC and such documents are publicly available on the SEC’s EDGAR filing system or any successor thereto.
ARTICLE 4
COMPLIANCE COVENANTS
Section 4.01     Compliance . The Company shall observe the following requirements:

(a) Adoption of Policies . The Company shall adopt, implement and maintain at all times policies with respect to the Specified Laws as Roivant may from time to time direct, including with respect to regulatory, Quality Management Systems standards, internal controls (including with respect to financial reporting and remediation of deficiencies), audit, compliance, record keeping, document retention, financial reporting, tax and legal requirements (collectively, the “ Policies ”), that, in each case, are (x) applicable to the Company and its Subsidiaries and its and their respective Representatives, (y) consistent with, and no less restrictive than, the corresponding policies established by Roivant, as they may be updated from time to time, and (z) are otherwise satisfactory to Roivant. For the avoidance of doubt, to the extent that Roivant has not established a corresponding

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policy with respect to a requirement that the Company wishes to establish, which the Company must confirm with Roivant, the Company’s Policy will govern unless and until Roivant establishes a corresponding policy. After such time, the Company’s Policy may continue to govern only if it is no less restrictive than Roivant’s policy. In establishing its corresponding policies, Roivant shall in good faith consider any timely and reasonable requests or inputs from the Company. The Company shall provide Roivant with a copy of each of its Policies upon finalization and shall promptly notify the Company of any updates, amendments or changes thereto.


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(b) Compliance Committee . Absent a waiver by Roivant, the board of directors of the company shall at all times after the date which is ninety (90) days after the Effective Date have a Compliance Oversight Committee (the “ Compliance Oversight Committee ”), whose composition, meetings and proceedings shall be subject to the requirements for any other committee of the Board pursuant to the Bye-laws, to oversee the Company’s and its Subsidiaries’ Compliance Program. In administering the Compliance Program, the Compliance Oversight Committee shall:

(i)      appoint an individual on the senior management team of the Company (the “ Compliance Officer ”) who will be responsible for the management and administration of the Compliance Program; provided that, until the Compliance Oversight Committee shall have appointed a Compliance Officer, the principal executive officer of the Company shall perform the duties of the Compliance Officer;

(ii)      cause the Company and its Subsidiaries to implement a training and education plan to ensure that the Company’s employees receive adequate training regarding the Compliance Program; and

(iii)      cause the Company to establish an internal reporting procedure that includes a confidential hot line mechanism to enable directors, officers, employees and agents of the Company and its Subsidiaries to report to the Compliance Officer (and/or such other person who is not in the reporting individual’s chain of command as the Compliance Oversight Committee may from time to time designate) any identified issues or questions associated with the Company’s policies, conduct, practices or procedures related to the Specified Laws.

(c) Compliance Officer . In administering the Compliance Program, the Compliance Officer shall:

(i)      make periodic reports (but in any event at least quarterly) regarding the status of the Compliance Program directly to the Compliance Oversight Committee;

(ii)      make reports regarding compliance matters directly to the Board at any time he or she considers appropriate;

(iii)      annually certify to the Compliance Oversight Committee (together with the principal executive officer of the Company, if the principal executive officer is not acting as Compliance Officer) that to the best of his or her knowledge and after reasonable due diligence, except as otherwise described in the report, the Company and its Subsidiaries and

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their respective directors, officers, employees and agents are each in compliance with all Specified Laws applicable to the Company and its Affiliates; provided that, if either the Compliance Officer or the principal executive officer of the Company is unable to provide such a certification, he or she shall provide an explanation directly to the Compliance Oversight Committee of the reasons why he or she is unable to provide such certification; and

(iv)      notify the Compliance Oversight Committee of (1) any actual or threatened investigation, regulatory or legal proceeding involving the Specified Laws or (2) any


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Reportable Event, in each case within 48 hours after discovery of the underlying facts or as soon thereafter as practicable.

(d)
Compliance with Law .

(i)      The Company shall not, and shall cause its Subsidiaries and its and their respective directors, officers, employees and agents not to, directly or indirectly, make, offer, promise or authorize any payment or transfer of any money or anything of value to or for the benefit of a Government Official or individual employed by another entity in the private sector that would violate either the UKBA or the FCPA or engage in any conduct that would reasonably be expected to be deemed to violate the UKBA or the FCPA in any material respect.

(ii)      The Company shall not, and shall cause its Subsidiaries and its and their respective directors, officers and employees not to, directly or indirectly, make, offer, promise or authorize any payment or transfer of any money or anything of value to or for the benefit of a Government Official or individual employed by another entity in the private sector that would violate Local ABAC Laws or engage in any conduct that would reasonably be expected to be deemed to violate Local ABAC Laws, Applicable Money Laundering Laws, Sanctions or applicable Laws prohibiting fraud, tax evasion, insider dealing and market manipulation in any material respect.

(iii)      The Company shall, and shall cause each of its direct and indirect Subsidiaries to, keep and maintain books and records reflecting accurately and in reasonable detail transactions involving the Company and its direct and indirect Subsidiaries and to implement financial controls giving reasonable assurance that payments will be made by or on behalf of the Company and its direct and indirect Subsidiaries only in accordance with management instructions.

(iv)      The Company shall, and shall cause its Subsidiaries and its and their respective directors, officers and employees to, otherwise comply in all material respects with all Specified Laws.

(e) Participation on Expert Councils . To facilitate collaboration and best practices amongst Roivant, the Company and other Affiliates of Roivant and the Company, Roivant may from time to time sponsor expert councils (the “ Expert Councils ”). The Company shall participate in such Expert Councils by appointing an individual with relevant expertise to each such Expert

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Council. No such Expert Council shall be convened on more than a semi-annual basis, unless urgent circumstances dictate otherwise. The objectives of the Expert Councils shall include facilitating discussion on changes in the applicable field of expertise, trends in the field and experiences, best practices and issues of a general nature. All proceedings of an Expert Council shall be subject to the terms of confidentiality set forth in Article 5 .


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ARTICLE 5

EXCHANGE OF INFORMATION; CONFIDENTIALITY

Section 5.01 Privilege . In the event that a Party reasonably determines that the provision of information pursuant to this Agreement would violate any law or bona fide contractual restriction, or result in the waiver of any Privilege, the Parties shall take all commercially reasonable measures to permit the compliance with the provision of information obligations in a manner that avoids any such harm or consequence, which shall include, but not be limited to, compliance with Sections 5.05 , 5.06 and 5.07 hereof. For purposes of this Agreement, the term “ Privilege ” shall mean information and advice that has been previously developed but is legally protected from disclosure under legal privileges, such as the attorney-client privilege, work product exemption or similar concept of legal protection

Section 5.02 Ownership of Information . Any information owned by a Party that is provided to the other Party pursuant to the terms of this Agreement shall be deemed to remain the property of such Party. Unless expressly set forth in this Agreement, nothing contained in this Agreement shall be construed as granting or conferring any right, title or interest (whether by license or otherwise) in, to, or under any such information.

Section 5.03 Record Retention . To facilitate the provision of information pursuant to this Agreement, the Company agrees to retain all information in its possession or control in accordance with its document retention policies, as such policies may be reasonably amended or revised after the Effective Date. The Company shall provide Roivant with reasonable notice of any material amendment or revision to its retention policies after the Effective Date. The Company shall not materially amend or revise its retention policy in effect at the time of its IPO for a period of three years after the IPO.

Section 5.04 Limitation of Liability . Each Party shall have no liability to the other Party in the event that any information exchanged or provided pursuant to this Agreement is found to be inaccurate or the requested information is not provided, in the absence of willful misconduct by, or gross negligence of, such Party. Each Party shall not have any liability to the other Party if any information is destroyed in compliance with its document retention policies.

Section 5.05     Confidentiality .


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(a) Subject to Section 5.07 . Each Party (the “ Receiving Party ”) agrees to hold, and to cause its Representatives, including for the avoidance of doubt underwriters or other parties providing financing to such Party, to hold in strict confidence, with at least the same degree of care that applies to its confidential and proprietary information pursuant to its policies in effect as of the Effective Date, all information with respect to the other Party (the “ Providing Party ”) that is accessible to it, in its possession (including information in its possession prior to the Original Effective Date) or furnished by the Providing Party or its Representative, or accessible to, in the possession of, or furnished to the Receiving Party or its Representatives pursuant to this Agreement or otherwise, except, in each case, to the extent that such information (i) is or becomes part of the public domain through no breach of this Agreement by the Receiving Party or its Representatives,
(ii) was independently developed following the Original Effective Date by the Receiving Party or


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its Representatives who have not accessed or otherwise received the applicable information; provided that such independent development can be demonstrated by competent, contemporaneous written records of the Receiving Party, (iii) became or becomes available to the Receiving Party following the Original Effective Date on a non-confidential basis from a third Party who is not bound directly or indirectly by a duty of confidentiality to the Providing Party; or
(iv) is provided by the Providing Party or its Representatives and appropriately disclosed by the Receiving Party or its Representative in accordance with the terms of Article 4 . The Parties acknowledge that they may have in their possession confidential or proprietary information of third Parties that was received under confidentiality or non-disclosure agreements with such third Party. The Parties will hold in strict confidence the confidential and proprietary information of third Parties to which they have access in accordance with the terms of any such agreements.

(b) Notwithstanding anything herein to the contrary, Roivant and its Representative shall be permitted to:

(i)      use the Company’s trademark in its written materials when referencing the Company;

(ii)      disclose confidential information (x) to Roivant’s attorneys, accountants, consultants and other professionals who are subject to a duty or undertaking of confidentiality to the extent necessary to obtain their services in connection with monitoring its investment in the Company or enforcing any of its rights under this Agreement or any other agreements with the Company; (y) to any Affiliate, partner, member, prospective member, or wholly-owned subsidiary of Roivant; provided that Roivant informs such person that such information is confidential and such person is under an obligation to maintain the confidentiality of such information; (z) to any prospective purchaser of any Equity Securities from Roivant, if such prospective purchaser agrees to be bound by confidentiality provisions at least as restrictive as this Section 5.05 and also agrees to customary standstill agreement with respect to the Company’s securities until such time as such confidential information is publicly disclosed;

(iii)      provide confidential information regarding the Company (including but not limited to historical financial and other information) to persons who have a legitimate reason to know such information and who are under an obligation to keep such information confidential, including the Roivant’s Representatives, investors and potential investors, and

(iv)      publish non-confidential information of the Company (including but not limited to historical financial and other information).

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(c) Notwithstanding anything to the contrary in this Article 5 , the Receiving Party shall have no right to use any information disclosed by the Providing Party unless otherwise provided for in this Agreement or specifically provided for in any other agreement between the Parties.

Section 5.06 Protective Arrangements . In the event that the Receiving Party either determines on the advice of its counsel that it is required to disclose any information pursuant to applicable Law (including the rules and regulations of the SEC in connection with any proposed


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registration of the Receiving Party’s securities under the Securities Act or the Exchange Act, or pursuant to the requirements of any national securities exchange) or receives any request or demand from any governmental or regulatory authority to disclose or provide information of the Providing Party that is subject to the confidentiality provisions hereof, the Receiving Party shall, to the extent permitted by Law and except in connection with a general regulatory examination unrelated to the Providing Party, notify the Providing Party prior to disclosing or providing such information and shall reasonably cooperate at the expense of the Receiving Party in seeking any reasonable protective arrangements (including by seeking confidential treatment of such information) requested by the Providing Party. Subject to the foregoing, the Receiving Party may thereafter disclose or provide information to the extent required by such Law or requested or required by such governmental authority; provided , however , that the Receiving Party provides the Providing Party, to the extent legally permissible and except in connection with a general regulatory examination unrelated to the Providing Party, upon request with a copy of the information so disclosed.

Section 5.07     Preservation of Legal Privileges .

(a) The Parties recognize that they possess and will possess Privileged information. Each Party recognizes that they shall be jointly entitled to the Privilege with respect to such Privileged information and that each shall be entitled to maintain, preserve and assert for its own benefit all such information and advice, but the Parties shall ensure that such information is maintained so as to protect the Privileges with respect to the other Party’s interest. To that end, no Party will knowingly waive or compromise any Privilege associated with such information and advice without the prior written consent of the other Party, which shall not be unreasonably withheld. In the event that Privileged information is required to be disclosed to any arbitrator or mediator in connection with a dispute between the Parties, such disclosure shall not be deemed a waiver of Privilege with respect to such information, and any Party receiving it in connection with a proceeding shall be informed of its nature and shall be required to safeguard and protect it.

(b) Upon receipt by either Party of any subpoena, discovery or other request that may call for the production or disclosure of information that is the subject of a Privilege, or if a Party obtains knowledge that any current or former employee of a Party has received any subpoena, discovery or other request that may call for the production or disclosure of such information, such Party shall provide the other Party a reasonable opportunity to review the information and to assert any rights it may have under this Section 5.07 or otherwise to prevent the production or disclosure of such information. Absent receipt of written consent from the other Party to the production or disclosure of information that may be covered by a Privilege, each Party agrees that it will not produce or disclose any information that may be covered by a Privilege unless a court of competent jurisdiction has entered a final, nonappealable order finding that the information is not entitled to protection under any applicable Privilege.


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ARTICLE 6
TAX MATTERS
Section 6.01     PFIC . For so long as Roivant owns Equity Securities, the Company will use reasonable best efforts to avoid, in respect of any taxable year, being treated as a passive


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foreign investment company (“ PFIC ”) within the meaning of Section 1297 of the Code, including, but not limited to, causing any of its subsidiaries to file an election pursuant to Treasury Regulation Section 301.7701-3. No later than 75 days after the end of each taxable year, the Company shall deliver to Roivant an analysis as to whether the Company believes that it will be treated as a PFIC in respect of such taxable year. Such analysis may be prepared by the Company, but in preparing such analysis the Company shall consult with its internationally recognized tax advisors.

Section 6.02 QEF Information . For so long as Roivant owns Equity Securities, the Company shall use reasonable best efforts to provide, and shall cause each of its subsidiaries to use reasonable best efforts to provide, to Roivant all information that may be necessary to allow Roivant, and any direct or indirect owners of Roivant, to evaluate the analysis referenced in Section
6.01 and to fulfill their U.S. tax filing and reporting obligations. The Company shall provide, and shall cause each of its subsidiaries to provide, such information to Roivant, and any direct or indirect owners of Roivant, as may reasonably be required to timely file and maintain a “qualified electing fund” election (as defined in Section 1295(a) of the Code) with respect to any such entity.

ARTICLE 7
DISPUTE RESOLUTION
Section 7.01     Limitation on Monetary Damages Equitable Remedies . Subject to Section
9.15      , the Company and Roivant hereby agree that neither Party shall have any liability for monetary damages for any breach of this Agreement so long as such Party used commercially reasonable efforts to comply with the obligation such Party breached and continues thereafter to use commercially reasonable efforts to remedy such breach. In addition to other remedies provided by applicable law, the Company and Roivant may each enforce the provisions of this Agreement through such legal or equitable remedies as a court of competent jurisdiction shall allow without the necessity of proving actual damages or bad faith, and the Party subject to a claim under this Agreement hereby waives any claim or defense that such Party has an adequate remedy at law, and waives any requirement for the securing or posting of any bond in connection with such equitable remedy.

Section 7.02 Disputes . The procedures for discussion, negotiation and mediation set forth in this Article 7 shall apply to all disputes, controversies or claims (whether arising in contract, tort or otherwise) that may arise out of or relate to, or arise under or in connection with, this Agreement or the transactions contemplated hereby or thereby (including all actions taken in furtherance of the transactions contemplated hereby on the Effective Date). the Company hereby agrees that its members of the board of directors or senior management that are not affiliated with Roivant shall lead all discussions, negotiations and mediations that occur pursuant to this Article 7.


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Section 7.03     Escalation; Mediation .

(a)      It is the intent of the Parties to use their respective commercially reasonable efforts to resolve expeditiously any dispute, controversy or claim between or among them with respect to the matters covered by this Agreement. In furtherance of the foregoing, any Party involved in a dispute, controversy or claim with respect to such matters may deliver a notice (an “ Escalation


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Notice ”) demanding an in person meeting involving representatives of the Parties at a senior level of management of the Parties (or if the Parties agree, of the appropriate strategic business unit or division within such entity). A copy of any such Escalation Notice shall be given to the general counsel, or like officer or official, of each Party involved in the dispute, controversy or claim (which copy shall state that it is an Escalation Notice pursuant to this Agreement). Any agenda, location or procedures for such discussions or negotiations between the Parties may be established by the Parties from time to time; provided , however , that the Parties shall use their commercially reasonable efforts to meet within 30 days of the Escalation Notice.

(b)      If the Parties are not able to resolve the dispute, controversy or claim through the escalation process referred to in clause (a) above within 90 days of delivery of the Escalation Notice, then the matter shall be referred to mediation; provided that such period of time may be extended upon mutual written consent of the Parties. The Parties shall retain a mediator to aid the Parties in their discussions and negotiations by informally providing advice to the Parties. Any opinion expressed by the mediator shall be strictly advisory and shall not be binding on the Parties, nor shall any opinion expressed by the mediator be admissible in any other proceeding. The mediator may be chosen from a list of mediators previously selected by the Parties or by other agreement of the Parties. Costs of the mediation shall be borne equally by the Parties involved in the matter, except that each Party shall be responsible for its own expenses. Mediation shall be a prerequisite to the commencement of any action by either Party.

Section 7.04     Binding Arbitration .

(a) If, after complying with the provisions set forth in Section 7.03 above the Parties are unable to reach resolution to any dispute, controversy or claim between the Parties, any such dispute, controversy or claim between the Parties, including any claim arising out of, in connection with, or in relation to the interpretation, performance, breach, or termination of this Agreement, shall be resolved exclusively and finally by confidential binding arbitration. The seat, or legal place, of arbitration shall be New York, New York. The language of the arbitration shall be English. The arbitration shall be administered by the International Centre for Dispute Resolution in accordance with its International Arbitration Rules in force when the Notice of Arbitration is submitted in accordance with such Rules. Each Party shall select one person to act as arbitrator and the two selected shall select a third arbitrator, who shall act as president of the panel. Where there are multiple claimants or multiple respondents, the multiple claimants, jointly, and the multiple respondents, jointly, shall select the party-appointed arbitrators. Except as may be required by law, to comply with a legal duty, or to pursue a legal right, neither a party nor an arbitrator may disclose the existence, content, or results of any arbitration hereunder without the prior written consent of both parties. Nothing herein shall prevent either Party from seeking provisional measures from any court of competent jurisdiction, and any such request shall not be deemed incompatible with the agreement to arbitrate or a waiver of the right to arbitrate. Each Party shall consent, for purposes of provisional

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measures or the enforcement of any arbitral award, to the non-exclusive jurisdiction of the state and federal courts located in New York, New York, and each Party shall not assert that such courts constitute forum non-conveniens. The award shall be final and binding on the parties. Judgment on the award may be entered in any court of competent jurisdiction.


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(b) Unless otherwise agreed in writing, the Parties will continue to provide service and honor all other commitments under this Agreement during the course of dispute resolution pursuant to the provisions of this Article 7, except to the extent such commitments are the subject of such dispute, controversy or claim.

ARTICLE 8
FURTHER ASSURANCES
Section 8.01     Further Assurances .

(a) In addition to the actions specifically provided for elsewhere in this Agreement, each of the Parties hereto will cooperate with each other and shall use their commercially reasonable efforts to take, or cause to be taken, all actions, and to do, or cause to be done, all things, reasonably necessary, proper or advisable under applicable laws, regulations and agreements to consummate and make effective the transactions contemplated by this Agreement.

(b) Without limiting the foregoing each Party hereto shall cooperate with the other Party, and without any further consideration, but at the expense of the requesting Party, to execute and deliver, or use its commercially reasonable efforts to cause to be executed and delivered, all instruments, including instruments of conveyance, assignment and transfer and to make all filings with, and to obtain all consents, approvals or authorizations of, any governmental authority or any other person or entity under any permit, license, agreement, indenture, order, decree, financial assurance (including letter of credit) or other instrument, and to take all such other actions as such Party may reasonably be requested to take by such other Party hereto from time to time, consistent with the terms of this Agreement.

(c) Nothing in this Agreement shall be construed to restrict or limit any right, responsibility or authority of either of Parties hereto or their respective, independent registered public accountants, audit committee or board of directors in violation of any law, legal requirement or listing standard applicable to such Party, whether existing today or hereafter. In the event either Party hereto reasonably determines that any provision in this Agreement does or will so limit any right, responsibility or authority of such Party or such Party’s independent registered public accountants, audit committee or board of directors, then the Parties hereto agree to attempt to negotiate in good faith any changes necessary or advisable to this Agreement to avoid or prevent such violation.

ARTICLE 9
MISCELLANEOUS

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Section 9.01     Counterparts; Entire Agreement; Conflicting Agreements .


(a) This Agreement may be executed in one or more counterparts all of which shall be considered one and the same agreement, and shall become effective when one or more counterparts have been signed by each of the Parties and delivered to the other Party. Execution of this


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Agreement or any other documents pursuant to this Agreement by facsimile or other electronic transmission (including in PDF form) shall be deemed to be, and shall have the same effect as, executed by an original signature.

(b) This Agreement contains the entire agreement of the Parties with respect to the subject matter hereof, supersede all previous agreements, negotiations, discussions, writings, understandings, commitments and conversations with respect to such subject matter and there are no agreements or understandings between the Parties with respect to such subject matter other than those set forth or referred to herein or therein.

(c) In the event and to the extent that there shall be a conflict between the provisions of this Agreement and the provisions of any other agreement between the Parties, the other agreement shall control with respect to the subject matter thereof, and this Agreement shall control with respect to all other matters.

Section 9.02 No Construction Against Drafter . The Parties acknowledge that this Agreement and all the terms and conditions herein have been fully reviewed and negotiated by the Parties and their respective attorneys. Having acknowledged the foregoing, the Parties agree that any principle of construction or rule of law that provides that, in the event of any inconsistency or ambiguity, an agreement shall be construed against the drafter of the agreement shall have no application to the terms and conditions of this Agreement.

Section 9.03 Governing law . This Agreement shall be governed by and construed and interpreted in accordance with the laws of the State of New York, without regard to the conflict of laws principles thereof that would result in the application of any law other than the laws of the State of New York.

Section 9.04 Assignability. This Agreement shall be binding upon and inure to the benefit of the Parties and their respective heirs, successors, legal representatives and permitted assigns; provided, however, that no Party may assign its respective rights or delegate its respective obligations under this Agreement without the express prior written consent of the other Party.

Section 9.05 Notices . All notices and other communications required or permitted hereunder to be given to a party to this Agreement shall be in person or in writing transmitted via facsimile or email or mailed by registered or certified mail, postage prepaid, or otherwise delivered by hand or by messenger, addressed to such party’s address as set forth below or at such other address as the party shall have furnished to each other party in writing in accordance with this provision.

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If to Roivant, to:

Roivant Sciences Ltd.
Suite 1, 3rd Floor
11-12 St. James’s Square
London SW1Y 4LB, United Kingdom Attention: Corporate Secretary


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Facsimile: +1 (441) 292 4720 Em ail: info@roivant.com
with a copy sent concurrently to: Roivant Sciences, Inc.
320 West 37th Street, 6th Floor New York, NY 10018
Attention: Allen Waxman, General Counsel Em ail: allen.waxm an@roivant.com

If to the Company to:

Axovant Sciences Ltd.
Suite 1, 3rd Floor
11-12 St. James’s Square
London SW1Y 4LB, United Kingdom Attention: Corporate Secretary Facsimile: +1 (441) 292 4720
Em ail: marianne.romeo@roivant.com with a copy sent concurrently to:
Axovant Sciences, Inc.
11 Times Square, 33rd Floor New York, NY 10036
Attention: Stephen Mohr, General Counsel Facsimile: (646) 767-5434
Em ail: stephen.mohr@axovant.com

Any notice sent in accordance with this Section 9.05 shall be effective (i) if mailed, 7 Business Days after mailing, (ii) if sent by messenger, upon delivery, and (iii) if sent via facsimile or email, upon transmission and electronic confirmation of receipt or (if transmitted and received on a non-

39


Business Day) on the first Business Day following transmission and electronic confirmation of receipt ( provided , however , that any notice of change of address shall only be valid upon receipt).

Section 9.06 Severability . If any provision of this Agreement or the application thereof to any person or circumstance is determined by a court of competent jurisdiction to be invalid, void or unenforceable, the remaining provisions hereof or the application of such provision to persons or circumstances or in jurisdictions other than those as to which it has been held invalid or unenforceable, shall remain in full force and effect and shall in no way be affected, impaired or invalidated thereby, so long as the economic or legal substance of the transactions contemplated hereby is not affected in any manner adverse to any Party. Upon such determination, the Parties shall negotiate in good faith in an effort to agree upon such a suitable and equitable provision to effect the original intent of the Parties.


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Section 9.07 Force Majeure . No Party shall be deemed in default of this Agreement to the extent that any delay or failure in the performance of its obligations under this Agreement results from any cause beyond its reasonable control and without its fault or negligence, such as acts of God, acts of civil or military authority, embargoes, epidemics, war, riots, insurrections, fires, explosions, earthquakes, floods, unusually severe weather conditions or labor problems. In the event of any such excused delay, the time for performance shall be extended for a period equal to the time lost by reason of the delay.

Section 9.08 Headings . The article, section and paragraph headings contained in this Agreement are for reference purposes only and shall not affect in any way the meaning or interpretation of this Agreement.

Section 9.09 Termination; Survival . This Agreement and all covenants and obligations herein shall terminate at such time as Roivant is no longer required by GAAP reporting requirements to consolidate the results of operations and financial position of the Company, account for its investment in the Company under the equity method of accounting (determined in accordance with GAAP and consistent with SEC reporting requirements) or otherwise include separate financial statements of the Company in its filings with the SEC pursuant to any rule of the SEC; provided that (i) the Parties may terminate this Agreement at any time upon mutual written consent and (ii) Roivant may terminate this Agreement upon written notice to the Company in the event of a bankruptcy, liquidation, dissolution or winding-up of the Company. Notwithstanding any termination of this Agreement pursuant to this Section 9.09 , the obligations of the Parties hereto pursuant to Sections 5.01, 5.02 , 5.04 , 5.05 , 5.06 , 5.07 , Article 7 , Article 8 and Article 9 (other than Section 9.16 (Maintenance of Insurance)) shall survive until the expiration of the applicable statute of limitations.

Section 9.10 Waivers of Default . Waiver by any Party of any default by the other Party of any provision of this Agreement shall not be deemed a waiver by the waiving Party of any subsequent or other default, nor shall it prejudice the rights of the other Party.

Section 9.11 Specific Performance . The Parties acknowledge that money damages may not be an adequate remedy for violations of this Agreement .In the event of any actual or threatened default in, or breach of, any of the terms, conditions and provisions of this Agreement, the Party or Parties who are or are to be thereby aggrieved shall have the right to specific performance and injunctive or other equitable relief of its rights under this Agreement, in addition to any and all other rights and remedies at law or in equity, and all such rights and remedies shall be cumulative..


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Section 9.12 Amendments . No provisions of this Agreement shall be deemed waived, amended, supplemented or modified by any Party, unless such waiver, amendment, supplement or modification is in writing and signed by an authorized representative of the Party against whom it is sought to enforce such waiver, amendment, supplement or modification.

Section 9.13 Waiver of Jury Trial . SUBJECT TO ARTICLE 7 AND SECTIONS 9.10 AND 9.11 HEREIN, EACH OF THE PARTIES HEREBY WAIVES TO THE FULLEST EXTENT PERMITTED BY APPLICABLE LAW ANY RIGHT IT MAY HAVE TO A TRIAL BY JURY WITH RESPECT TO ANY COURT PROCEEDING DIRECTLY OR INDIRECTLY ARISING OUT OF AND PERMITTED UNDER OR IN CONNECTION WITH THIS


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AGREEMENT OR THE TRANSACTIONS CONTEMPLATED BY THIS AGREEMENT. EACH OF THE PARTIES HEREBY (A) CERTIFIES THAT NO REPRESENTATIVE, AGENT OR ATTORNEY OF THE OTHER PARTY HAS REPRESENTED, EXPRESSLY OR OTHERWISE, THAT SUCH OTHER PARTY WOULD NOT, IN THE EVENT OF LITIGATION, SEEK TO ENFORCE THE FOREGOING WAIVER AND (B) ACKNOWLEDGES THAT IT HAS BEEN INDUCED TO ENTER INTO THIS AGREEMENT AND THE TRANSACTIONS CONTEMPLATED BY THIS AGREEMENT, AS APPLICABLE, BY, AMONG OTHER THINGS, THE MUTUAL WAIVERS AND CERTIFICATIONS IN THIS SECTION 9.14.

Section 9.14 Limitation on Monetary Damages . The Parties hereto hereby agree that no Party shall have any liability for monetary damages for any breach of this Agreement so long as such Party used commercially reasonable efforts to comply with the obligation such Party breached and continues thereafter to use commercially reasonable efforts to remedy such breach.

Section 9.15     Indemnity and Expenses .

(a)
Indemnity .

(i)      The Company shall indemnify and hold harmless Roivant and its respective partners, shareholders, members, Affiliates, directors, officers, fiduciaries, managers, controlling Persons, employees, agents, counsel and other representatives and each of the partners, shareholders, members, Affiliates, directors, officers, fiduciaries, managers, controlling Persons, employees, agents, counsel and other representatives of each of the foregoing (collectively, the “ Indemnitees ”) from and against any and all actions, causes of action, suits, claims, liabilities, losses, damages and costs and out-of-pocket expenses in connection therewith (including reasonable attorneys’ and accountants’ fees and expenses) incurred by the Indemnitees or any of them on or after the Effective Date (collectively, the “ Indemnified Liabilities ”) as a result of, arising out of or in any way relating to (i) Roivant’s status as a holder of Equity Securities and (ii) the operations of the Company or any of its Subsidiaries; provided that the foregoing indemnification rights shall not be available with respect to any such Indemnified Liabilities arising on account of an Indemnitee’s gross negligence or willful misconduct; provided , further , that, if and to the extent that the foregoing undertaking may be unavailable or unenforceable for any reason, the Company shall make the maximum contribution to the payment and satisfaction of each of the Indemnified Liabilities which is permissible under applicable Law.


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(ii)      Each Party shall indemnify and hold harmless the other Party and its Indemnitees for any breach of Section 5.05 hereof due to the gross negligence or willful misconduct of such Party or its Representatives.

Section 9.16 Maintenance of Insurance . The Company shall, and shall cause its Subsidiaries to, (a) maintain Roivant as additional named insured on each of its and its Subsidiaries’ insurance policies and arrangements during the term of this Agreement and (b) maintain insurance coverage reasonably sufficient to meet its indemnification obligations under this Agreement.


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Section 9.17 No Third-Party Beneficiaries . This Agreement is not intended, nor shall it be deemed, to confer any rights or remedies on any person other than the Parties hereto and their respective successors and assigns. This Agreement does not create any third-party beneficiary hereto and the Company and Roivant are the only parties entitled to commence any action, proceeding or claim under this Agreement.

Section 9.18 Expenses . Each Party is responsible for its own fees, costs and expenses incurred in connection with this Agreement and the activities contemplated hereby; provided , further , to the extent that the observation of the covenants and performance of the obligations set forth in Article 3 result in additional significant financial expenses to the Company, upon the Company’s request, the Parties will discuss potential reimbursement by Roivant with respect to such additional financial expenses incurred by the Company.


[ Signature Pages Follow ]

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IN WITNESS WHEREOF, the parties hereto, intending to be legally bound, have caused this Agreement to be executed as of the date first written above.

COMPANY:
 
 
 
AXOVANT SCIENCES LTD.
 
 
 
By:
/s/ Gregory Weinhoff
Name:
Gregory Weinhoff
Title:
Principal Financial Officer

SHAREHOLDER:
 
 
 
ROIVANT SCIENCES LTD.
 
 
 
By:
/s/ Marianne Romeo
Name:
Marianne Romeo
Title:
Head, Global Transactions & Risk Management













[Signature Page to Information Sharing and Cooperation Agreement ]

46


IN WITNESS WHEREOF, the parties hereto, intending to be legally bound, have caused this Agreement to be executed as of the date first written above.

COMPANY:
 
 
 
AXOVANT SCIENCES LTD.
 
 
 
By:
/s/ Gregory Weinhoff
Name:
Gregory Weinhoff
Title:
Principal Financial Officer

SHAREHOLDER:
 
 
 
ROIVANT SCIENCES LTD.
 
 
 
By:
/s/ Marianne Romeo
Name:
Marianne Romeo
Title:
Head, Global Transactions & Risk Management













[Signature Page to Information Sharing and Cooperation Agreement ]

47
Exhibit 10.2


SHARE PURCHASE AGREEMENT
by and between
ROIVANT SCIENCES LTD.
and
AXOVANT SCIENCES LTD.
Dated as of June 5, 2018









SHARE PURCHASE AGREEMENT
THIS SHARE PURCHASE AGREEMENT (“ Agreement ”), dated as of June 5, 2018, is made by and between ROIVANT SCIENCES LTD. (the “ Investor ”), a Bermuda exempted company, with its principal place of business at Suite 1, 3 rd Floor, 11-12 St. James’s Square, London SW1Y 4LB, United Kingdom, and AXOVANT SCIENCES LTD. (the “ Company ”), a Bermuda exempted company, with its principal place of business at Suite 1, 3 rd Floor, 11-12 St. James’s Square, London SW1Y 4LB, United Kingdom. The Investor and the Company are collectively referred to as “the Parties .”
WHEREAS, the Investor wishes to purchase from the Company, and the Company wishes to issue and sell to the Investor, 14,285,714 common shares, par value $0.00001 per share ( “ Common Shares ”), for an aggregate amount of $24,999,999.50, at the Price Per Share (as defined below), under the terms and conditions of this Agreement; and
 
WHEREAS, each of the Parties wishes to set forth in this Agreement certain terms and conditions regarding, among other things, the Investor’s ownership of the Common Shares being purchased hereby.
    
NOW, THEREFORE, in consideration of the following mutual promises and obligations, and for good and valuable consideration the adequacy and sufficiency of which are hereby acknowledged, the Parties agree as follows:


ARTICLE I
DEFINITIONS
When used in this Agreement, the following terms shall have the respective meanings specified therefor below:
Business Day ” shall mean any day other than a Saturday or Sunday or a day on which banks located in New York, New York or Bermuda are authorized or required by law to close.
Governmental Entity ” shall mean any federal, state, municipal, local, provincial, regional Governmental Entity in the United States or other political subdivision thereof or in any foreign country and any Person exercising executive, legislative, judicial regulatory or administrative functions of or pertaining to government.
Information Sharing Agreement ” shall mean that certain Amended and Restated Information Sharing and Cooperation Agreement in substantially the form attached as Exhibit A hereto.
Legal Restraint ” shall mean any temporary restraining order, preliminary or permanent injunction or other judgment or order issued by or under the authority of any Governmental Entity.
Material Adverse Effect ” shall mean any events, occurrences or circumstances which give rise to or would reasonably be expected to give rise to, individually or in the aggregate, a material adverse effect on (i) the business, properties, financial condition, results of operations or prospects of the Company, or (ii) the ability of the Company to comply with its obligations under this Agreement and the related transaction documents.
Person ” shall mean and include an individual, a part-ner-ship, a joint venture, a corporation, a limited liability company, a limited liability partnership, a trust, an incor-porated organization and a Governmental Entity.
SEC ” shall mean the U.S. Securities Exchange and Commission or any successor agency thereto.
Securities Act ” shall mean the U.S. Securities Act of 1933, as amended.

ARTICLE II

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PURCHASE AND SALE OF COMMON SHARES
Section 2.1 Issuance of Common Shares. Subject to the terms and conditions hereof, on the date hereof, the Company agrees to issue and sell to the Investor, and the Investor agrees to purchase, 14,285,714 Common Shares (the “ Purchased Shares ”) for an aggregate purchase price of $24,999,999.50 (the “ Purchase Price ”) representing a per share price of $1.75 (the “ Price Per Share ”).
Section 2.2      Closing . The purchase and sale of the Purchased Shares (the “ Closing ”) shall occur on the second (2nd) full Business Day following the satisfaction or waiver of each closing condition set forth under Article V hereto (the “ Closing Date ”) at the offices of Cooley LLP, 3175 Hanover Street, Palo Alto, CA 94304, or such other place as shall be determined by agreement between the Company and the Investor.
Section 2.3      Delivery . (a) At or prior to the Closing, the Company shall deliver or cause to be delivered the following to the Investor:
(i) an electronic copy of a share certificate, registered in the Investor’s name, representing the Purchased Shares, or a copy of an account statement issued by the Company’s transfer agent, evidencing a book entry notification for the Purchased Shares in the name of the Investor;
(ii) a certificate dated the Closing Date and executed by an authorized officer of the Company that each of the conditions set forth in Sections 5.1(a) , 5.2(a) , 5.2(b) and 5.2(c) are satisfied in all respects; and
(iii) a counterpart to the Information Sharing Agreement, duly executed by the Company.

(b) At the Closing, the Investor shall deliver the following to the Company:
     (i) payment of the Purchase Price by wire transfer of immediately available funds to an account specified by the Company at least one (1) Business Day prior to the Closing; and
(ii) a counterpart to the Information Sharing Agreement, duly executed by the Investor.



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ARTICLE III
REPRESENTATIONS AND WARRANTIES OF THE COMPANY
The Company hereby represents and warrants to the Investor as follows:
Section 3.1 Organization, Good Standing and Qualification . The Company is a corporation duly organized, validly existing and in good standing under the laws of Bermuda. The Company has all requisite corporate power and corporate authority to own and operate its properties and assets, to carry on its business as now conducted, to enter into this Agreement and any related transaction documents, to issue and sell the Purchased Shares and to carry out the other transactions contemplated under this Agreement and any related transaction documents. The Company’s Certificate of Incorporation, Memorandum of Association and Second Amended and Restated Bye-laws as filed with the SEC (together, the “ Organizational Documents ”) are true, correct and complete copies of said documents as in effect on the date hereof.
Section 3.2 Authorization . All corporate action on the part of the Company, its directors and shareholders necessary for the authorization, execution and delivery of this Agreement and the related transaction documents, the performance of all obligations of the Company thereunder, including the authorization, issuance and delivery of the Purchased Shares, has been taken. This Agreement and the related transaction documents have been duly executed and delivered by the Company and constitute valid and legally binding obligations of the Company, enforceable against the Company in accordance with their respective terms (except as such enforceability may be limited by applicable bankruptcy, insolvency, reorganization, moratorium or other laws of general application relating to or affecting enforcement of creditors’ rights).
Section 3.3 No Conflicts . The execution, delivery and performance of this Agreement and the related transaction documents and compliance with the provisions thereof by the Company, does not and shall not: (a) violate any provision of law, statute, ordinance, rule or regulation or any ruling, writ, injunction, order, judgment or decree of any Governmental Entity, (b) conflict with or result in any breach of any of the terms, conditions or provisions of, or constitute (whether or not with due notice or lapse of time, or both) a default (or give rise to any right of termination, cancellation or acceleration) or result in the creation of any lien, security interest, charge or encumbrance upon any of the properties or assets of the Company pursuant to any agreement, document, instrument, contract, understanding, arrangement, note, indenture, mortgage, lease or permit to which the Company is a party, or under which the Company or any of its assets is bound or affected, or (c) violate or conflict with any of the provisions of the Organizational Documents; except, in the case of subsections (a) and (b) as would not have a Material Adverse Effect.
Section 3.4 Valid Issuance of Purchased Shares . When issued, sold and delivered in accordance with the terms hereof for the consideration expressed herein, the Purchased Shares shall be validly issued and outstanding, fully paid and nonassessable, free from any encumbrances or restrictions on transfer, including preemptive rights, rights of first refusal or other similar rights, other than restrictions on transfer under this Agreement and the related transaction documents and under federal and state securities laws.
Section 3.5 Governmental Consents . No consent, approval, order or authorization of, or registration, qualification, designation, declaration or filing with, any Governmental Entity is required in connection with the consummation of the transactions contemplated by this Agreement and the related transaction documents, except for registration or qualification, or taking such action to secure exemption from such registration or qualification, of the Purchased Shares under applicable state or federal securities laws, which actions shall be taken, by and at the expense of the Company, on a timely basis as may be required.
Section 3.6 Offering . Subject to the accuracy of the Investor’s representations set forth in Sections 4.3 and 4.4 , the offer, sale and issuance of the Purchased Shares to be issued in conformity with the terms of this Agreement constitute transactions which are exempt from the registration requirements of the Securities Act and from all applicable state registration or qualification requirements, other than those with which the Company has complied.
Section 3.7 Brokers’ or Finders’ Fees . No broker, finder, investment banker or other Person is entitled to any brokerage, finder’s or other fee or commission in connection with the transactions contemplated by this Agreement.

ARTICLE IV

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REPRESENTATIONS AND WARRANTIES OF THE INVESTOR
     The Investor hereby represents and warrants as follows:
Section 4.1 Organization; Good Standing . The Investor is a corporation duly organized, validly existing and in good standing under the laws of Bermuda. The Investor has all requisite corporate power and corporate authority to enter into this Agreement and the related transaction documents, to purchase the Purchased Shares and to carry out the other transactions contemplated under this Agreement and the related transaction documents.
Section 4.2 Authorization . All corporate action on the part of the Investor, and its directors and shareholders necessary for the authorization, execution and delivery of this Agreement and the related transaction documents, the performance of all obligations of the Investor thereunder, including the subscription and purchase of the Purchased Shares, has been taken. This Agreement has been duly executed and delivered by the Investor and constitutes a valid and legally binding obligation of the Investor, enforceable against the Investor in accordance with its terms (except as such enforceability may be limited by applicable bankruptcy, insolvency, reorganization, moratorium or other laws of general application relating to or affecting enforcement of creditors’ rights).
Section 4.3 Purchase Entirely for Own Account . The Purchased Shares shall be acquired for investment for the Investor’s own account, not as a nominee or agent, and not with a view to the resale or distribution of any part thereof, and the Investor has no present intention of selling, granting any participation, or otherwise distributing the Purchased Shares. The Investor does not have any contract, undertaking, agreement or arrangement with any Person to sell, transfer or grant participation to such Person any of the Purchased Shares.
Section 4.4 Investment Experience and Accredited Investor Status . The Investor is knowledgeable, sophisticated and experienced in making, and is qualified to make, decisions with respect to investments in shares representing an investment decision like that involved in the purchase of the Purchased Shares. The Investor is an “accredited investor” (as defined in Rule 501 of Regulation D under the Securities Act).
Section 4.5 Restricted Stock . The Investor understands that the Purchased Shares, when issued, shall be restricted securities under the federal securities laws inasmuch as they are being acquired from the Company in a transaction not involving a public offering and that under such laws and applicable regulations such securities may be resold without registration under the Securities Act only in certain limited circumstances. It is understood that the certificates representing the Purchased Shares shall bear the following legend:

“These securities have not been registered under the Securities Act of 1933. They may not be sold, offered for sale, pledged or hypothecated in the absence of a registration statement in effect with respect to the securities under the Securities Act or an opinion of that such registration is not required or unless sold pursuant to Rule 144 of the Securities Act or a registration statement thereunder.”
Section 4.6 No General Solicitation. The Investor understands and acknowledges that neither the Company nor any other person offered to sell the Purchased Shares to it by means of any form of “general solicitation” (as such term is used in Regulation D under the Securities Act) or advertising, including but not limited to: (A) any advertisement, article, notice or other communication published in any newspaper, magazine or similar media or broadcast over television or radio, or (B) any seminar or meeting whose attendees were invited by any general solicitation or general advertising.

ARTICLE V

CONDITIONS TO CLOSING
 
Section 5.1     Conditions to the Obligations of the Company and the Investor. The respective obligations of the Company and the Investor hereunder to consummate the Closing are subject to fulfillment, prior to or at the Closing, of the following conditions (compliance with which or the occurrence of which may be waived in whole or in part by either the Company or the Investor in writing with respect to fulfillment of conditions to its own obligations to the extent permitted by applicable law):

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    (a)     No Legal Restraint shall be in effect preventing the consummation of the transactions contemplated by this Agreement and any related transaction documents.
(b)     The Company shall have prepared and sent an “information statement” (the “ Information Statement ”) as defined in Rule 14c-1 under the Securities Exchange Act of 1934, as amended (the “ Exchange Act ”), containing the information specified in Schedule 14C under the Exchange Act in all material respects, to its shareholders in a manner consistent with the requirements set forth under Rule 14c-2(d) under the Exchange Act.
(c)    A period of twenty (20) calendar days shall have elapsed following the date on which the Information Statement is sent to the Company’s shareholders in accordance with clause (b) of this Section 5.1.
(d)    The Purchased Shares shall have been duly listed on the Nasdaq Global Select Market, subject to notice of issuance.
Section 5.2     Conditions to the Obligations of the Investor. The obligations of the Investor hereunder to consummate the Closing are subject to fulfillment, prior to or at the Closing, of the following conditions (compliance with which or the occurrence of which may be waived in whole or in part by the Investor in writing):
(a)    The Company shall have performed and complied in all material respects with all the terms, provisions and conditions of this Agreement to be complied with and performed by the Company at or before the Closing.
(b)    Since the date of this Agreement, there shall have been no event or occurrence that would have a Material Adverse Effect.
(c) Each of the representations and warranties of the Company shall be true and correct in all material respects as of the Closing as if made as of the Closing, other than those representations and warranties qualified by materiality or Material Adverse Effect which shall be true and correct in all respects.
(d)     The Company shall have delivered or caused to be delivered to the Investor the items set forth in Section 2.3(a).

Section 5.3 Conditions to the Obligations of the Company. The obligations of the Company hereunder to consummate the Closing are subject to fulfillment, prior to or at such Closing, of the following conditions (compliance with which or the occurrence of which may be waived in whole or in part by the Company in writing):
 
    (a)     Each of the representations and warranties of the Investor shall be true and correct in all material respects on and as of the Closing as if made as of the Closing, other than representations and warranties made as of a specified date, which shall be true and correct as of the date so specified.

(b)     The Investor shall have performed and complied in all material respects with all the terms, provisions and conditions of this Agreement to be complied with and performed by the Investor at or before such Closing.
(c)     The Investor shall have delivered or caused to be delivered to the Company the items set forth in Section 2.3(b).
         Section 5.4 Frustration of Closing Conditions. Neither the Company nor the Investor may rely on the failure of any condition set forth in this Article V to be satisfied if such failure was caused by (i) such party’s failure to act in good faith or to use its reasonable best efforts to cause the Closing to occur, or (ii) such party’s own representations and warranties not being true and correct.

ARTICLE VI
MISCELLANEOUS

5



Section 6.1      Further Assurances. The parties agree to take such reasonable steps and execute such other and further documents as may be necessary or appropriate to cause the terms and conditions contained herein to be carried into effect.
Section 6.2     Successors and Assigns. Except as otherwise expressly provided herein, the terms and conditions of this Agreement shall inure to the benefit of and be binding upon the respective successors and assigns of the parties. This Agreement and the rights and duties of the Company set forth herein may not be assigned, in whole or in part, by the Company.
Section 6.3      Entire Agreement. This Agreement (including exhibits hereto) contains the complete understanding of the parties with respect to the subject matter hereof and thereof and super-sedes all prior understandings and writings relating to the subject matter hereof and thereof. The exhibits to this Agreement are incorporated into and form an integral part hereof.
Section 6.4      Governing Law; Submission to Jurisdiction. This Agreement shall be governed by and construed in accordance with the laws of the State of New York, without regard to conflict of laws principles. Each of the parties irrevocably submits to the exclusive jurisdiction of any state or federal court sitting in the County of New York, in the State of New York, for the purposes of any suit, action or other proceeding arising out of this Agreement or any transaction contemplated hereby. To the fullest extent they may effectively do so under applicable law, the parties hereto irrevocably waive and agree not to assert, by way of motion, as a defense or otherwise, any claim that they are not subject to the jurisdiction of any such court, any objection that they may now or hereafter have to the laying of the venue of any such suit, action or proceeding brought in any such court and any claim that any such suit, action or proceeding brought in any such court has been brought in an inconvenient forum.
EACH OF THE PARTIES HERETO HEREBY WAIVES TO THE FULLEST EXTENT PERMITTED BY APPLICABLE LAW ANY RIGHT IT MAY HAVE TO A TRIAL BY JURY WITH RESPECT TO ANY LITIGATION DIRECTLY OR INDIRECTLY ARISING OUT OF, UNDER OR IN CONNECTION WITH THIS AGREEMENT. EACH OF THE PARTIES HERETO HEREBY (A) CERTIFIES THAT NO REPRESENTATIVE, AGENT OR ATTORNEY OF THE OTHER PARTY HAS REPRESENTED, EXPRESSLY OR OTHERWISE, THAT SUCH OTHER PARTY WOULD NOT, IN THE EVENT OF LITIGATION, SEEK TO ENFORCE THE FOREGOING WAIVER AND (B) ACKNOWLEDGES THAT IT HAS BEEN INDUCED TO ENTER INTO THIS AGREEMENT AND THE TRANSACTIONS CONTEMPLATED BY THIS AGREEMENT, AS APPLICABLE, BY, AMONG OTHER THINGS, THE MUTUAL WAIVERS AND CERTIFICATIONS IN THIS SECTION.

6




Section 6.5     Counterparts. This Agreement may be executed in counterparts, each of which shall be deemed an original but which together shall constitute one and the same instrument.
Section 6.6     Titles and Subtitles. The titles and subtitles used in this Agreement are used for convenience only and are not to be considered in construing or interpreting this Agreement.
Section 6.7     Notices. All notices, instructions and other communications hereunder or in connection herewith shall be in writing, shall be sent to the address of the relevant party set forth below and shall be (a) delivered personally, (b) sent by registered or certified mail, return receipt requested, postage prepaid, (c) sent via a reputable nation-wide overnight courier service, or (d) sent by email or facsimile transmission, with a confirmation copy to be sent by registered or certified mail, return receipt requested, postage prepaid. Any such notice, instruction or communication shall be deemed to have been delivered upon receipt if delivered by hand, three (3) Business Days after it is sent by registered or certified mail, return receipt requested, postage prepaid, one (1) Business Day after it is sent via a reputable nationwide overnight courier service, or when transmitted and receipt is confirmed, if transmitted by facsimile or email (if such transmission is on a Business Day; or otherwise, on the next Business Day following such transmission). Either party may change its address by giving notice to the other party in the manner provided above.
To the Company:
Axovant Sciences Ltd.
 
Suite 1, 3 rd  Floor
11-12 St. James’s Square
London SW1Y 4LB, United Kingdom

Attention: Gregory Weinhoff, Principal Financial Officer
Email: gregory.weinhoff@axovant.com
With a copy (which
Cooley LLP
shall not constitute
notice to the Company):
3175 Hanover Street
Palo Alto, CA 94304
 
 
 
 Attention:Frank Rahmani
(650) 843-5753
Email: rahmaniff@cooley.com

John T. McKenna
(650) 843-5059
Email: jmckenna@cooley.com
To the Investor:
Roivant Sciences Ltd.
 
Suite 1, 3 rd  Floor
11-12 St. James’s Square
London SW1Y 4LB, United Kingdom
 
Attention: Allen Waxman, General Counsel
Email: allen.waxman@roivant.com  
With a copy to (which
White & Case LLP
shall not constitute
1221 Avenue of the Americas
notice to the Investor):
New York, New York 10020
 
Attention: Chang-Do Gong
                  (212) 819-7808
Email: cgong@whitecase.com

7



Section 6.8 Expenses. Each party shall pay its own fees and expenses with respect to this Agreement. If any action at law or in equity is necessary to enforce or interpret the terms of this Agreement or the Articles, the prevailing party shall be entitled to reasonable attorney’s fees, costs and necessary disbursements in addition to any other relief to which such party may be entitled.
Section 6.9 Amendments and Waivers. Any term of this Agreement may be amended and the observance of any term of this Agreement may be waived (either generally or in a particular instance and either retroactively or prospectively), only with the written consent of the Company and the Investor.
Section 6.10 Severability. If, under applicable laws, any provision hereof is invalid or unenforceable, or otherwise directly or indirectly affects the validity of any other material provision(s) of this Agreement (“ Severed Clause ”), then, it is mutually agreed that this Agreement shall endure except for the Severed Clause. The parties shall consult and use their reasonable best efforts to agree upon a valid and enforceable provision which shall be a reasonable substitute for such Severed Clause in light of the intent of this Agreement.
[Remainder of page intentionally blank.]

IN WITNESS WHEREOF, the parties have executed and delivered this Agreement as of the date first above written.
ROIVANT SCIENCES LTD.
By:     /s/ Marianne L. Romeo             
Name:     Marianne L. Romeo
Title:    Head, Global Transactions & Risk Management
AXOVANT SCIENCES LTD.
By:     /s/ Gregory Weinhoff             
Name:     Gregory Weinhoff
Title:     Principal Financial Officer


8



[***] = Portions of this exhibit have been omitted and filed separately with the Securities and Exchange Commission. Confidential treatment requested under 17 C.F.R. Section 240.24b-2.









Exhibit 10.3

EXECUTION VERSION



Uick




OXFORD BIOMEDICA (UK) LTD


AND


AXOVANT SCIENCES GMBH













LICENCE AGREEMENT










This Licence Agreement (the “Agreement”) is made on June 5, 2018 (the “Effective Date”) BETWEEN
(1)
OXFORD BIOMEDICA (UK) LTD, a company incorporated in England and registered under number 03028927, whose registered office is at Windrush Court, Transport Way,
Oxford, OX4 6LT (“BioMedica”); and

(2)
AXOVANT SCIENCES GMBH, a company organized and existing under the laws of Switzerland, having its principal place of business at Viaduktstrasse 8, 4051 Basel, Switzerland (the “Licensee”).

BACKGROUND

(A)
BioMedica has special expertise and longstanding experience in the field of lentiviral vector gene therapy products, including pre-clinical and clinical development, manufacture, testing and product release.

(B)
BioMedica has developed and controls certain intellectual property rights relating to lentiviral vectors for dopamine replacement therapy, including OXB-102, Prosavin and alternative vectors, and certain intellectual property rights useful for the production and use of lentiviral vectors in human cells.

(C)
The Licensee wishes to take, and BioMedica wishes to grant, an exclusive licence under intellectual property rights of BioMedica to develop and commercialise certain products, and to engage BioMedica to perform certain development work relating to the manufacture of such products on the terms of this Agreement.

OPERATIVE PROVISIONS

1.
DEFINITIONS

1.1
In this Agreement, the following terms have the meanings set forth in this Clause 1.1 (Definitions):

(a)
“Accompanying Innovative Product” shall have the meaning set out in Schedule 5 (Net Sales).

(b)
“Accounting Standards” shall mean internationally recognised accounting principles (including IFRS, US GAAP, and the like), in each case, as generally and consistently applied by the applicable Selling Entity.

(c)
“Affiliate” shall mean:

(i)
with respect to the Licensee, any entity or any other person that controls, is controlled by, or is under common control with, the Licensee, but excluding Roivant Sciences Ltd., a company organized under the laws of Bermuda (“Roivant”), and any entity or any other person controlled by, or under common control with, Roivant other than through the intermediary of Axovant Sciences Ltd., and for the avoidance of doubt, the Licensee’s Affiliates as of the Effective Date are Axovant Holdings Limited, a company organized under the laws of England and Wales,


2





Axovant Sciences, Inc., a company organized under the laws of the State of Delaware, Axovant Sciences America, Inc., a company organized under the laws of the State of Delaware, Axovant Treasury Holdings, Inc., a company organized under the laws of the State of Delaware, Axovant Treasury, Inc., a company organized under the laws of the State of Delaware, and Axovant Sciences Ltd.;

(ii)
with respect to BioMedica, any entity or other person that controls, is controlled by, or is under common control with, BioMedica; and

(iii)
with respect to any Third Party, any entity or other person that controls, is controlled by, or is under common control with, such Third Party;

but in each case only for so long as such control exists; and in this context, “control”, and with correlative meanings, “controlled by” and “under common control with”, shall have the meaning of Control set out in Clause 1.1(gg) (Control).

(d)
“Alternative Product” shall mean in relation to a Product:

(i)
any gene therapy product:

(A)
designed to produce proteins having the enzymatic or cofactor activities of all of the transgenes delivered by the Product; and

(B)
delivering no other therapeutic moiety; or

(ii)
a Biosimilar Product.

(e)
“Annual Net Sales” shall mean the [***].

(f)
“Applicable Laws” shall mean the applicable provisions of any and all national, supranational, regional, state and local laws (including common law or other binding law), statutes, regulations, codes, directives, injunctions, orders, permits of or from any court, arbitrator, Regulatory Authority or governmental agency or authority having jurisdiction over or related to the subject item, together with all applicable current international regulatory requirements and standards, such as Good Manufacturing Practices, Good Clinical Practices and Good Laboratory Practices and comparable foreign regulatory standards.

(g)
“Axovant Sciences Ltd.” shall mean Axovant Sciences Ltd., a company organized under the laws of Bermuda.

(h)
“Backup Product” shall mean any lentiviral-based vector product for delivering:

(i)
transgenes, which transgenes encode proteins having the enzymatic or cofactor activities of either one or two (but not three) of the following: AADC (aromatic L-amino acid decarboxylase), TH (tyrosine hydroxylase), or CH1 (GTP cyclohydrolase 1); and

(ii)
no other therapeutic moiety;


3





and in each case is Covered by a Licensed Patent or the research, development or Manufacture of which used or uses the Licensed Know-How.

(i)
“BioMedica Competing Product” shall mean:

(i)
any product for delivering transgenes, in a lentiviral-based vector, which transgenes encode proteins having the enzymatic or cofactor activities of any one or two or three of the following: AADC (aromatic L-amino acid decarboxylase), TH (tyrosine hydroxylase), or CH1 (GTP cyclohydrolase 1), in each case either alone or in combination with other therapeutic moieties, but excluding a BioMedica Reserved Product, and

(ii)
any product for delivering transgenes, in a lentiviral-based vector, which transgenes encode proteins having the enzymatic or cofactor activities of all three (3) of the following: AADC (aromatic L-amino acid decarboxylase), TH (tyrosine hydroxylase), and CH1 (GTP cyclohydrolase 1), and any other therapeutic moiety where the therapeutic effect of such product is the increased production of dopamine.

(j)
“BioMedica Indemnitees” shall have the meaning set forth in Clause 14.2 (Licensee Indemnification).

(k)
“BioMedica IP” shall mean:

(i)
the Patents that Cover the development, manufacture, use, or commercialization of the Products, including those Patents identified in Part 1 and Part 2 of Schedule 1 (the “Licensed Patents”);

(ii)
the Know-How that is necessary or reasonably useful for the research, development, use, offering for sale, sale, import, and export of Products, but excluding Know-How for the Manufacture of Products (which is described in Clause 1.1(k)(iii) (BioMedica IP)); and

(iii)
the Know-How that is reasonably useful (that exists as of the date of transfer pursuant to Clause 7.3 (Technology Transfer)) or that is necessary (that exists at any time during the Term) to practice the process transferred to the Licensee or its designee pursuant to Clause 7.3 (Technology Transfer) for Manufacture of Products;

(together 1.1(k)(ii) (BioMedica IP) and 1.1(k)(iii) (BioMedica IP) being the “Licensed Know-How”);

in each case, to the extent Controlled by BioMedica as of the Effective Date or during the Term, but excluding the BioMedica TRiP Patents and BioMedica TRiP Know-How (subject to Clause 2.2(a) (TRiP and WPRE Option)) and the WPRE Patent (subject to Clause 2.2(a) (TRiP and WPRE Option)).

(l)
“BioMedica Qualified Person” shall mean the person with legal responsibility to certify that each batch of medicinal product for human or veterinary use within the European Union has been manufactured, tested in accordance with all Applicable Laws and all safety and quality requirements of the clinical trial


4





authorisation or market authorisation prior to use in a Clinical Trial or prior to release for sale and placing on the market. The detailed duties of the qualified person for medicinal products for human use are set out in Article 51 of EU Directive 2001/83/EC and for medicinal products for veterinary use are set out in Article 55 of EU Directive 2001/82/EC.

(m)
“BioMedica Reserved Product” shall mean any lentiviral-based vector product for delivering:

(i)
transgenes, which transgenes encode proteins having the enzymatic or cofactor activities of all three (3) of: AADC (aromatic L-amino acid decarboxylase), TH (tyrosine hydroxylase), and CH1 (GTP cyclohydrolase 1); and

(ii)
any other therapeutic moiety;

except to the extent that the therapeutic effect of such product is the increased production of dopamine.

(n)
“BioMedica TRiP Know-How” shall mean Know-How Controlled by BioMedica as of the Effective Date or generated or acquired by BioMedica after the Effective Date, in each case, with respect to the TRiP System.

(o)
“BioMedica TRiP Patents” shall mean the Patents Controlled by BioMedica that Cover the TRiP System and shall include the Patents listed in Part 3 of Schedule 1 which may be updated by BioMedica from time to time with additional Patents.

(p)
“Biosimilar Product” means a pharmaceutical product that, with respect to a Product, (i) has been licensed as a biosimilar or interchangeable product by the FDA pursuant to Section 351(k) of the Public Health Service Act (42 U.S.C. 262(k)), as may be amended, or any subsequent or superseding law, statute, or regulation, (ii) has been licensed as a similar biological medical product by the EMA pursuant to Directive 2001/83/EC, as may be amended, or any subsequent or superseding law, statute, or regulation, or (iii) has otherwise achieved analogous Regulatory Approval from another applicable Regulatory Authority.

(q)
“BLA” shall mean (i) a Biologics License Application (as defined in Title 21 of the United States Code of Federal Regulations, as amended from time to time) or equivalent submission filed with the FDA seeking Regulatory Approval, or (ii) any equivalent application to a Regulatory Authority in any other country, including a marketing authorisation application (“MAA”) filed with the EMA.

(r)
“Business Day” shall mean any day except a Saturday, Sunday or any other day when commercial banks in London, England, New York City, New York or Basel, Switzerland are authorised to close.

(s)
“Calendar Quarter” shall mean each respective period of three (3) consecutive months ending on March 31, June 30, September 30, and December 31.

(t)
“Calendar Year” shall mean any period of twelve consecutive months beginning on 1 January and ending on 31 December.


5





(u)
“Change of Control of BioMedica” shall mean the occurrence of any of the following:

(i)
any reorganization, recapitalization, consolidation, amalgamation or merger (or similar transaction or series of related transactions) of Oxford BioMedica plc, or any sale or exchange of outstanding shares (or similar transaction or series of related transactions) of Oxford BioMedica plc, and in each case as a result of such transaction, any person or group of persons who are connected persons of each other or who are acting in concert owns, directly or indirectly, shares representing more than [***] of the voting power of Oxford BioMedica plc or such surviving entity;

(ii)
as a result of a tender or exchange offer, open market purchases, privately negotiated purchases, consolidation, merger or otherwise, any person or group of persons who are connected persons of each other or who are acting in concert owns, directly or indirectly, shares representing more than [***] of the voting power of Oxford BioMedica plc;

(iii)
Oxford BioMedica plc ceases to control BioMedica;

(iv)
the sale, transfer or other disposition (including by way of merger or consolidation) of all or substantially all of the business and assets of Oxford BioMedica plc and its Affiliates, taken as a whole, to one or more Third Parties whether through a single transaction or a series of transactions; or

(v)
the shareholders of Oxford BioMedica plc. adopt a plan or proposal for liquidation or dissolution of Oxford BioMedica plc.

(v)
“Change of Control of the Licensee” shall mean the occurrence of any of the following:

(i)
any reorganization, recapitalization, consolidation, amalgamation or merger (or similar transaction or series of related transactions) of Axovant Sciences Ltd., or any sale or exchange of outstanding shares (or similar transaction or series of related transactions) of Axovant Sciences Ltd., and in each case as a result of such transaction Roivant ceases to own, directly or indirectly, shares representing more than [***] of the voting power of Axovant Sciences Ltd. or the surviving entity of such transaction or series of related transactions, in each case without regard to whether Axovant Sciences Ltd. is the surviving entity and, in each case, any person or group of persons who are connected persons of each other or who are acting in concert, in each case other than Roivant, owns, directly or indirectly, shares representing more than [***] of the voting power of Axovant Sciences Ltd. or such surviving entity;

(ii)
as a result of a tender or exchange offer, open market purchases, privately negotiated purchases, consolidation, merger or otherwise, any person or group of persons who are connected persons of each other or


6





who are acting in concert, in each case other than Roivant, owns, directly or indirectly, shares representing more than [***] of the voting power of Axovant Sciences Ltd.;

(iii)
Axovant Sciences Ltd. ceases to control the Licensee, other than in connection with a Group Reorganisation;

(iv)
the sale, transfer or other disposition (including by way of merger or consolidation) of all or substantially all of the business and assets of Axovant Sciences Ltd. and its Affiliates, taken as a whole, to one or more Third Parties (other than Roivant or its Affiliates) whether through a single transaction or a series of transactions; or

(v)
the shareholders of Axovant Sciences Ltd. adopt a plan or proposal for liquidation or dissolution of Axovant Sciences Ltd.

(w)
“Change Proposal” shall have the meaning set out in Clause 5.1(c) (Process Development Services).

(x)
“Charge” shall have the meaning set out in Clause 16.1(a)(ii) (Term Loan Agreement).

(y)
“Claim” shall have the meaning set out in Clause 14.2 (Licensee Indemnification).

(z)
“Clinical Project Team” shall have the meaning set out in Clause 3.1 (Clinical Project Team).

(aa) “Clinical Supply Agreement” shall have the meaning set out in Clause 7.1(b) (Manufacture and Supply of Product).
(bb)    “Clinical Trials” shall mean any clinical testing of Products in human subjects.
(cc)    “Combination Product” shall have the meaning set out in Schedule 5 (Net
Sales).

(dd) “Commercially Reasonable Efforts” shall mean, with respect to the Licensee’s obligations under this Agreement with respect to a Product, the use of such efforts and employment of such resources:

(i)
as would normally be used or employed by a reasonable Third Party company having, together with its Affiliates, similar financial and other resources as the Licensee together with its Affiliates;

(ii)
for a product of similar market potential at a similar stage of its product life as the relevant Product;

(iii)
when utilizing sound and reasonable scientific, medical, and business judgment to develop and commercialise such product in a timely manner;

(iv)
taking into account relative safety and efficacy, product profile, the competitiveness of the marketplace and the market potential of such


7





product, the nature and extent of market exclusivity, including patent coverage and regulatory data protection, price and reimbursement status, and all relevant factors;

(v)
but in all cases without taking into account the fact that an alternative competing product is being developed or commercialised by or on behalf of such reasonable Third Party company or one of its Affiliates or sublicensees.

Commercially Reasonable Efforts shall be determined on a market-by-market basis, and it is anticipated that the level of effort will change over time, reflecting changes in the status of the product and the markets involved.

(ee) “Commercial Supply Agreement” shall have the meaning set out in Clause 7.1(b) (Manufacture and Supply of Product).

(ff) “Control” or “Controlled” shall mean with respect to any Know-How or other intellectual property right, the possession (whether directly or indirectly and whether by ownership or license, other than pursuant to this Agreement) by a Party of the right to grant to the other Party access or a license as provided herein under such Know-How or other intellectual property right without violating the terms of any agreement or other arrangements with any Third Party or misappropriating the proprietary or trade secret information of a Third Party.

(gg) “Control” for the purposes of Clause 1.1(c) (Affiliates) and Clause 2.5 (Non- Compete) shall mean ownership of [***] or more of the shares of stock entitled to vote for the election of directors, in the case of a corporation, or [***] or more of the equity interest in the case of any other type of legal entity, status as a general partner in any partnership, or any other arrangement whereby the person or entity controls or has the right to control the board of directors or equivalent governing body of a corporation or other entity, or the ability to cause the direction of the management or policies of a corporation or other entity. In the case of entities organised under the laws of certain countries, the maximum percentage ownership permitted by law for a foreign investor may be less than [***], and in such case such lower percentage shall be substituted in the preceding sentence, provided that such foreign investor has the power to direct the management and policies of such entity.

(hh) “Confidential Information” shall mean all information of a confidential or proprietary nature which is disclosed directly or indirectly by one Party (the “Disclosing Party”) or its Affiliates, to the other Party (the “Receiving Party”) or its Affiliates at any time during the Term, without regard to the form or manner in which such information is disclosed or obtained (including information disclosed orally or in documentary or electronic form or by way of model, or obtained by observation), and without limiting the foregoing, in addition shall include:

(i)
the existence and terms of this Agreement for which both Parties shall be deemed to be the Receiving Party;


8





(ii)
information disclosed under the confidentiality agreement between the Parties originally executed 15 th November 2017, amended on 7 th December 2017 (the “CDA”), which, with effect from the Effective Date, shall be deemed to be Confidential Information of the relevant Party under this Agreement and subject to the terms of this Agreement in place of the terms of the CDA with this Agreement superseding the CDA; and

(iii)
the Know-How within the BioMedica IP for which BioMedica shall be deemed to be the Disclosing Party and the Licensee deemed to be the Receiving Party.

(ii) “Cover” shall mean, with respect to a claim of a Patent and a Product, that such claim would be infringed, absent a license, by the manufacture, use, keeping, offer for sale, sale or importation of such Product in such country (and for the purposes of this definition, any pending claim of a patent application shall be deemed to be issued and in-force having claims in the form in which they are then pending).

(jj)    “Divest” shall have the meaning set out in Clause 2.5 (Non-Compete).

(kk) “Dollars” shall mean United States dollars and “$” shall be interpreted accordingly.

(ll) “Diligence Milestones” shall have the meaning set out in Clause 6.6 (Diligence Milestones).
(mm)    “EMA” shall mean the European Medicines Agency, or any successor thereto.
(nn)    “European Commission” means the authority within the European Union that
has the legal authority to grant Regulatory Approvals in the European Union
based on input received from the EMA or other competent Regulatory Authorities.

(oo) “Exclusivity Period” shall have the meaning set out in Clause 2.6(c)(i) (BioMedica Reserved Product).

(pp) “Existing Inventory” shall have the meaning set out in Clause 7.2 (Manufacture and Supply of Product).

(qq) “FDA” shall mean the United States Food and Drug Administration, or any successor thereto.

(rr) “Fees” shall have the meaning set out in Clause 5.5(a) (Process Development Service Costs).

(ss) “Field” shall mean all uses, including the treatment, prevention and diagnosis of any and all human and animal diseases, disorders and conditions.

(tt) “First Commercial Sale” shall mean, with respect to a Product, the first sale to a Third Party of such Product in a given regulatory jurisdiction after Regulatory Approval has been obtained in such jurisdiction for such Product.


9





(uu) “Fiscal Quarter” means each of the following three (3)-month periods during each Fiscal Year: January 1 through March 31; April 1 through June 30; July 1 through September 30; and October 1 through December 31; provided, that the first Fiscal Quarter shall commence on the Effective Date and end on June 30, 2018.

(vv) “Fiscal Year” means with respect to the Licensee, the twelve (12)-month period from April 1 through March 31 provided, that the Licensee may change its Fiscal Year on [***] prior written notice to BioMedica and BioMedica shall, in such instance, make such appropriate adjustments to the reporting and other payment terms under this Agreement to accommodate such change.

(ww) “FTE” shall mean the equivalent of the work of a full-time employee of BioMedica for a [***] period.

(xx)
“FTE Rate” shall mean a rate of £[***] per FTE per year, to be pro-rated on an hourly basis of £[***] per FTE per hour, assuming [***] hours per year for an FTE.
(yy)    “GBP” means British pounds sterling, and “£” shall be interpreted accordingly.
(zz)    “General Process Development Results” shall have the meaning set out in
Clause 5.4 (Process Development Results).

(aaa) “Group Reorganisation” means any arrangement (by scheme of arrangement, share exchange, or otherwise) under which the shares in Axovant Sciences Ltd. are acquired by a new body corporate in terms that the shareholders of the body corporate and their respective shareholdings and percentage equity interests in that new body corporate after that acquisition are the same or substantially the same as they were immediately prior to that acquisition.

(bbb) “IND” shall mean an investigational new drug application or equivalent application filed with a Regulatory Authority in a given country, which application is required to commence human clinical trials in such country.

(ccc) “Indemnified Party” shall have the meaning set out in Clause 14.4 (Indemnification Procedure).

(ddd) “Indemnifying Party” shall have the meaning set out in Clause 14.4 (Indemnification Procedure).

(eee) “Insolvency Event” in relation to a Party or entity shall mean any of the following events:

(i)
that Party ceasing or threatening to cease to carry on business;

(ii)
that Party being deemed by a competent authority to be unable or admitting inability to pay its debts;

(iii)
a moratorium is declared by a competent authority in respect of any indebtedness of that Party;


10





(iv)
that Party giving notice to any of its creditors that it has suspended or is about to suspend payment of any of its debts;

(v)
that Party commencing negotiations with one or more of its creditors with a view to rescheduling any of its indebtedness by reason of financial difficulties;

(vi)
an arrangement, composition, or assignment with or for the benefit of its creditors is entered into or proposed by or in relation to that Party;

(vii)
a receiver, administrative receiver, liquidator, or compulsory manager taking possession of or being appointed over the whole or any material part of the assets of that Party;

(viii)
any expropriation, attachment, sequestration, distress, execution or other analogous process is levied or enforced on or affects the whole or any material part of the assets of that Party (and is not discharged within [***]);

(ix)
enforcement of any mortgage, charge, pledge, lien or other security interest securing any obligation of any person or any other agreement or arrangement having a similar effect over any assets of that Party;

(x)
that Party or its directors or the holder of a qualifying floating charge giving notice of his, their or its intention to appoint an administrator;

(xi)
that Party or its directors or any of its creditors or the holder of a qualifying floating charge making an application to the court for the appointment of an administrator;

(xii)
an administrator being appointed of that Party;

(xiii)
the winding-up, dissolution, administration or reorganisation (by way of voluntary arrangement, scheme of arrangement or otherwise) with respect to that Party; provided that this does not apply to any winding-up petition that is frivolous or vexatious and is discharged, stayed or dismissed within [***] of commencement;

(xiv)
that Party being struck off the register of companies; or

(xv)
the happening in relation to that Party of an event analogous to any of the above in any jurisdiction in which it is incorporated or resident or in which it carries on business or has assets.

(fff) “Inventions” means all inventions, whether or not patentable, that are discovered, made, conceived, or conceived and reduced to practice in the course of activities contemplated by this Agreement other than in the course of the Process Development Services.

(ggg) “Joint Inventions” shall have the meaning set out in Clause 11.1(a) (Ownership).


11





(hhh)    “Joint Patents” shall have the meaning set out in Clause 11.1(a) (Ownership).

(iii)
“Know-How” shall mean unpatented technical and other information which is not generally known, including information comprising or relating to discoveries, inventions, data, designs, formulae, methods, models, research plans, procedures, designs for experiments and tests and results of experimentation and testing (including results of research or development), processes (including manufacturing processes, specification and techniques), laboratory records, chemical, pharmacological, toxicological, pre-clinical, clinical, analytical and quality control data, trial data, case report forms, data analyses, reports or summaries and information contained in submissions to and information from ethical committees and regulatory authorities.

(jjj) “Lender Agent” shall have the meaning set out in Clause 16.1 (Term Loan Agreement).

(kkk) “Licensed Know-How” shall have the meaning set forth in Clause 1.1(k) (BioMedica IP).

(lll) “Licensed Patents” shall have the meaning set forth in Clause 1.1(k) (BioMedica IP).

(mmm) “Licensee Competing Product” shall mean any viral vector gene therapy product (other than the Product) for intraputamental delivery of transgenes:

(i)
in any vector other than a lentiviral-based vector; and

(ii)
which transgenes encode proteins having the enzymatic or cofactor activities of either one or two or three of the following: AADC (aromatic L-amino acid decarboxylase), TH (tyrosine hydroxylase), or CH1 (GTP cyclohydrolase 1).

(nnn) “Licensee Indemnitees” shall have the meaning set forth in Clause 14.3 (BioMedica Indemnification).

(ooo) “Loan Agreement” shall have the meaning set out in Clause 16.1 (Term Loan Agreement).

(ppp) “Major Markets” shall mean the United States, United Kingdom, Germany, France, Italy, Spain, and Japan.

(qqq) “Manufacture” shall mean all activities related to the production, manufacture, processing, filling, finishing, packaging, labelling, inspection, receiving, holding and shipping of the Products, or any constituents or packaging materials with respect thereto, or any intermediate of any of the foregoing, including process and cost optimization, process qualification and validation, release, testing, quality assurance and quality control. When used as a verb, “Manufacture” shall mean to engage in Manufacturing.

(rrr) “Market Penetration” shall mean, with respect to a Product and one or more Alternative Product(s) sold in any given country, the number of units of all Alternative Product(s) sold in such country during any period of three (3)


12





consecutive Calendar Quarters, expressed as a percentage of the total number of units of such Product sold in such country during the [***] Calendar Quarters immediately preceding the launch of any Alternative Product(s) in such country, in each case as reported by the IQVIA script data or, in the absence of available IQVIA script data, according to an alternative source generally utilised in the pharmaceutical industry as agreed by the Parties.

(sss)    “Material Issue” shall mean:

(i)
a material finding regarding the safety or efficacy of the Product; or

(ii)
a material development regarding the commercial viability of the Product;

in each case, which a reasonable Third Party would consider to be sufficient to cease all further development and/or commercialisation of the Product.

(ttt) “Net Receipts” shall mean [***]:

(i)
[***];

(ii)
[***];


13






(iii)
[***];

[***].

(uuu)    “Net Sales” shall have the meaning set out in Schedule 5 (Net Sales).

(vvv) “Oaktree” shall have the meaning set out in Clause 16.1 (Term Loan Agreement).

(www) “Ongoing Clinical Trials” shall have the meaning set out in Clause 4.1 (Transfer of Know-How and Ongoing Clinical Trials).

(xxx)
“Original Product” shall mean any lentiviral-based vector product for delivering:

(i)
transgenes, which transgenes encode proteins having the enzymatic or cofactor activities of all of AADC (aromatic L-amino acid decarboxylase),
TH (tyrosine hydroxylase), and CH1 (GTP cyclohydrolase 1); and

(ii)
no other therapeutic moiety;

including, as at the Effective Date, Prosavin and OXB-102.

(yyy) “OXB-102” shall mean the product that is the subject of the OXB-102 Clinical Trial.

(zzz) “OXB-102 Clinical Trial” shall mean clinical study numbered, as of the Effective Date, OXB-102-01 with study title “A Phase I/II Safety and Dose Evaluation Study of OXB-102 in Patients with Bilateral Idiopathic Parkinson’s Disease”.

(aaaa) “OXB Project Personnel” shall have the meaning set out in Clause 6.2 (Engagement of BioMedica in Clinical Trials).

(bbbb) “Oxford BioMedica plc” shall mean the public limited company formed under the laws of England and Wales.


14





(cccc)
“Paediatric Priority Review Voucher” shall mean a priority review voucher awarded by the FDA to the sponsor of a rare paediatric disease product application pursuant to Section 529 of the United States Federal Food, Drug, and Cosmetic Act (21 U.S.C. § 301 et seq.), as amended, and the rules and regulations promulgated thereunder, or an equivalent voucher under a superseding law.

(dddd)
“Paediatric Priority Review Voucher Receipts” shall mean any payments received by the Licensee or its Affiliates pursuant to one or more agreements relating to the transfer by the Licensee or its Affiliates to a Third Party of a Paediatric Priority Review Voucher.

(eeee)
“Party” shall mean BioMedica or the Licensee and, when used in the plural, shall mean BioMedica and the Licensee.

(ffff)
“Patents” shall mean (i) pending patent applications, issued patents, utility models and designs; (ii) all reissues, extensions (including patent term adjustments and supplementary protection certificates), substitutions, confirmations, registrations, re-registrations, re-examinations, continuations, continuations-in-part, divisions, and patents of addition, with respect to the foregoing; and (iii) extensions, renewals or restorations of any of the foregoing by existing or future extension, renewal or restoration mechanisms, including supplementary protection certificates or the equivalent thereof.

(gggg) “Phase 1 Clinical Trial” shall mean, in reference to a clinical trial of a Product,
(i) a trial that would satisfy the requirements for a Phase 1 study as defined in 21 C.F.R. § 312.21(a) or (ii) a Phase 1 study as defined in the ICH E8 Guideline (or, in either case, any amended or successor regulation or guideline).

(hhhh)
“Phase 1/2 Clinical Trial” shall mean, in reference to a clinical trial of a Product, that such trial combines both a Phase 1 Clinical Trial and a Phase 2 Clinical Trial of such Product into a single protocol, where the Phase 1 Clinical Trial portion is performed first to (i) establish initial safety, tolerability, pharmacokinetic and pharmacodynamic information for a Product or (ii) determine an optimal dose of such Product in subjects, and the Phase 2 Clinical Trial portion is performed second to further evaluate safety and efficacy of such Product in subjects treated with a selected dose.

(iiii)
“Phase 2 Clinical Trial” shall mean, in reference to a clinical trial of a Product,
(i)      a trial that would satisfy the requirements for a Phase 2 study as defined in 21 C.F.R. § 312.21(b) or (ii) a Phase 2 study as defined in the ICH E8 Guideline (or, in either case, any amended or successor regulation or guideline).

(jjjj)
“Pivotal Clinical Trial” shall mean a controlled clinical study in humans of the efficacy and safety of a product, that is (1) prospectively designed to, or (2) for the purposes of Clause 8.2 (Development and Regulatory Milestone Payments) is subsequently determined to, demonstrate statistically whether such product is effective and safe for use in humans in the indication being investigated in a manner sufficient to submit a BLA to obtain Regulatory Approval to market such product, in each case as evidenced by (i) an agreement with or statement from the FDA on a Special Protocol Assessment or equivalent in another country, or


15





(ii)      other guidance or minutes issued by the FDA for such registration trial or equivalent in another country.

(kkkk) “Plasmids” means the plasmids required for Good Manufacturing Practice Manufacture of Products.

(llll) “Pricing and Reimbursement Approval” means, with respect to a Product in a particular country or region, the approval, agreement, determination or decision of at least one applicable decision making body establishing the price or level of reimbursement for such Product.

(mmmm) “Process Development Project Team” shall have the meaning set out in Clause 3.3 (Process Development Project Team).

(nnnn) “Process Development Services” has the meaning set out in Clause 5.1(a) (Process Development Services).

(oooo) “Process Development Results” shall mean all results, data, information and inventions (and all intellectual property rights in the same) first identified, developed, generated or created in the course of performance of the Process Development Services.

(pppp) “Product” shall mean:

(i)
an Original Product; or

(ii)
pursuant to Clause 2.1(c) (Grant of Licence; Right of Reference), any Backup Product;

and in each case is Covered by a Licensed Patent or the research, development or Manufacture of which used or uses the Licensed Know-How.
(qqqq) “Product Marks” shall have the meaning set out in Clause 6.8 (Trademarks).
(rrrr)    “Product-Specific Patents” shall mean the Patents listed in Part 2 of Schedule
1 .

(ssss) “Product-Specific Process Development Results” shall have the meaning set out in Clause 5.4 (Process Development Results).
(tttt)    “Prosavin” shall mean the product the subject of the Prosavin Clinical Trial.
(uuuu) “Prosavin Clinical Trial” shall mean clinical study number PS1/001/09 with
study title “A multicentre, open-label extension study to determine the long term
safety, tolerability and efficacy of ProSavin® in patients with bilateral, idiopathic Parkinson’s disease”.

(vvvv) “Records” shall have the meaning set out in Clause 5.3 (Record Keeping and Reporting).

(wwww) “Regulatory Authority” shall mean any local or national agency, authority, department, inspectorate, minister, ministry official, or public or


16





statutory person (whether autonomous or not) of any government of any country having jurisdiction over any of the Parties or the development, Manufacture, sale, or use of medicinal products, including, where applicable, the EMA, FDA, or any similar health regulatory authority in any country.

(xxxx) “Regulatory Approval” shall mean, with respect to Products in a particular country or region, all approvals from the applicable Regulatory Authorities necessary and sufficient for the marketing and sale of a Product in such country or region.

(yyyy) “Regulatory Exclusivity” shall mean any exclusive marketing rights or data exclusivity rights conferred by any Regulatory Authority with respect to a Product other than Patents, including rights conferred in the U.S. under the Hatch- Waxman Act or the FDA Modernization Act of 1997, under national implementations of Article 10 of Directive 2001/83/EC or rights similar thereto in any other jurisdiction.
(zzzz) “Regulatory Filings” shall have the meaning set out in Clause 6.3 (Regulatory).
(aaaaa) “Royalty Report” shall have the meaning set out in Clause 9.2 (Royalty
Reports).

(bbbbb)
“Royalty Term” shall mean, in relation to a Product sold in a country, the period of time from the First Commercial Sale by a Selling Entity of such Product in such country until the latest of (i) the date upon which there is no Valid Claim in such country that Covers such Product (provided that in the event that a pending claim within the BioMedica IP is subsequently granted following expiry of the Royalty Term under this paragraph, the Royalty Term shall come back into force to the extent that such claim is a Valid Claim that Covers such Product); (ii) the expiry of any Regulatory Exclusivity in respect of such Product in such country; and (iii) ten (10) years after such First Commercial Sale of such Product in such country.

(ccccc) “Scientific Advisory Board” shall have the meaning set out in Clause 3.6 (Scientific Advisory Board).

(ddddd)
“Selling Entity” shall mean the Licensee, its Affiliates, or Sublicensees, as applicable.

(eeeee)
“Senior Officers” shall mean, for the Licensee, the President, and for BioMedica, the Chief Executive Officer.

(fffff) “Start Date” shall have the meaning set out in Clause 2.6(b) (BioMedica Reserved Product).

(ggggg)
“Sublicensee” shall mean a Third Party to whom the Licensee, its Affiliates, or its or their Sublicensee has granted rights under the BioMedica IP, including through multiple tiers.

(hhhhh)
“Tax” or “Taxes” means any and all federal, provincial, territorial, state, municipal, local, foreign or other taxes, imposts, rates, levies, assessments and other charges in the nature of a tax, including without limitation all income,


17





goods and services, transfer, value added, sales, use, stamp, documentary, and withholding taxes, and all customs and import duties, together with all interest, penalties, and additions thereto imposed with respect to such amounts, in each case whether disputed or not.

(iiiii) “Third Party” shall mean any entity other than BioMedica or the Licensee or an Affiliate of BioMedica or the Licensee.
(jjjjj)    “Term” shall have the meaning set out in Clause 15.1 (Term).
(kkkkk) “Transfer Plan” shall have the meaning set out in Clause 4.1 (Transfer of Know-
How and Ongoing Clinical Trials).

(lllll) “TRiP Option” shall have the meaning set out in Clause 2.2(a) (TRiP and WPRE Option).

(mmmmm) “TRiP System” shall mean the heterologous translation control system described in PCT/GB2014/053813 for repressing translation of one or more nucleotides of interest.

(nnnnn)    “US” shall mean the United States of America.

(ooooo)
“Valid Claim” shall mean a claim of (i) an unexpired and issued patent within the BioMedica IP that has not been disclaimed, revoked, or held invalid, unpatentable or unenforceable by an administrative agency, court or other government agency of competent jurisdiction in a final and non-appealable decision (or a decision unappealed within the time limit allowed for appeal), and which has not been admitted to be invalid or unenforceable through reissue, re- examination or disclaimer or otherwise; or (ii) a pending patent application within the BioMedica IP which has not been finally rejected by a patent office or other governmental agency of competent jurisdiction in an unappealable decision or a decision that is un-appealed within the time allowed for appeal and has not been pending for more than five (5) years from the date of filing of such pending patent application.

(ppppp)
“Value Added Tax” or “VAT” means the tax imposed by Council Directive 2006/112/EC of the European Community and any national legislation implementing that directive together with legislation supplemental thereto and in particular, in relation to the United Kingdom, the tax imposed by the Value Added Tax Act of 1994 or other tax of a similar nature imposed elsewhere instead of or in addition to value added tax; and outside the European Union (and including the United Kingdom in the event that the United Kingdom ceases to be a member of the European Union during the Term), any tax corresponding to, or substantially similar to, the common system of value added tax referred to in this definition, excluding any Tax imposed on or with respect to the income of any of BioMedica or its Affiliates.

(qqqqq)
“Work Package” shall mean any written statement setting out a programme of process development work to be performed under this Agreement and containing the information set out in Schedule 3 and such other information as may be necessary for the performance of such programme of work.


18





(rrrrr) “WPRE Option” shall have the meaning set out in Clause 2.2(a) (TRiP and WPRE Option).

(sssss) “WPRE Patent” shall mean any patent that claims priority to International Application Number PCT/US1998/019441.

1.2
Interpretation. In this Agreement:

(a)
unless otherwise specified, references to clauses and schedules are to the clauses and schedules of this Agreement;

(b)
the words “include”, “including” and “in particular” are to be construed as being by way of illustration or emphasis only and are not to be construed so as to limit the generality of any words preceding them;

(c)
the words “other” and “otherwise” are not to be construed as being limited by any words preceding them;

(d)
headings are used for convenience only and do not affect its interpretation; and

(e)
a reference to the singular includes a reference to the plural and vice versa and a reference to any gender includes a reference to all other genders.

2.
GRANT OF LICENCE

2.1
Grant of Licence; Right of Reference

(a)
BioMedica hereby grants to the Licensee, subject to the terms and conditions set forth in this Agreement, a worldwide, exclusive (even as to BioMedica, except as expressly set forth herein) license, with the right to grant sublicenses (including through one or multiple tiers) under the BioMedica IP to research, develop, Manufacture and have Manufactured (subject to Clause 7 (Manufacture) with respect to Manufacture of Products), use, offer for sale, sell, have sold, import, and export the Products in the Field.

(b)
BioMedica hereby grants to the Licensee, its Affiliates, and Sublicensees, subject to the terms and conditions set forth in this Agreement, a “Right of Reference” as that term is defined in 21 C.F.R. § 314.3(b) (or any other similar provision under Applicable Law outside the US) to all regulatory documents, dossiers and filings that relate to the Product or any lentiviral vector gene therapy product manufactured by BioMedica, provided that:

(i)
such right shall apply only to such regulatory documents, dossiers or filings to which BioMedica is able to grant such “Right of Reference” without violating the terms of any agreement or other arrangements with any Third Party or misappropriating the proprietary or trade secret information of a Third Party; and

(ii)
such right shall be for the sole purpose of enabling the Licensee its Affiliates, and Sublicensees to file, obtain approval and maintain any Regulatory Filings with respect to the Products or exercise any of its license rights to Products.


19





BioMedica shall provide a signed statement to that effect, if requested by the Licensee in accordance with Applicable Law.

(c)
If the Licensee considers that a Material Issue has arisen, the Licensee shall immediately notify BioMedica in writing providing all available details of the Material Issue. Following such notification, the Parties shall discuss the nature of the Material Issue in good faith. If, following such discussion the Licensee maintains its opinion that there is a Material Issue, then the definition of Product shall be deemed to mean only a Backup Product and shall not include an Original Product.

(d)
BioMedica and its Affiliates shall not, alone or in collaboration with any Third Party, clinically develop, seek Regulatory Approval for, Manufacture (or have Manufactured), use, sell (or have sold), market, promote, import, export, or otherwise commercialize (or grant any rights in or to) an Original Product or a Backup Product during the Term.

(e)
BioMedica shall, from time to time but at least annually on the anniversary of the Effective Date, update Part 1 and Part 2 of Schedule 1 to include any additional Patents Controlled by BioMedica that Cover the development, manufacture, use, or commercialization of the Products, excluding the BioMedica TRiP Patents unless and until the Licensee exercises its option pursuant to Clause 2.2(a) (TRiP and WPRE Option) and the WPRE Patent unless and until the Licensee exercises its option pursuant to Clause 2.2(b) (TRiP and WPRE Option).

2.2
TRiP and WPRE Option.

(a)
BioMedica hereby grants to the Licensee an exclusive option to obtain a worldwide, exclusive licence, with the right to grant sublicenses (including through multiple tiers) under the BioMedica TRiP Patents and the BioMedica TRiP Know-How solely to research, develop, Manufacture and have Manufactured (subject to Clause 7 (Manufacture) with respect to Manufacture of Products) use, offer for sale, sell, have sold, import, and export the Products in the Field (“TRiP Option”). The Licensee may exercise the TRiP Option by written notice to BioMedica. Upon exercise of the TRiP Option, the licence under the BioMedica TRiP Patents and the BioMedica TRiP Know-How shall be deemed part of the definition of “BioMedica IP”.

(b)
BioMedica hereby grants to the Licensee an exclusive option to obtain a worldwide, non-exclusive licence, without the right to grant sublicenses under the WPRE Patent solely to research, develop, Manufacture and have Manufactured (subject to Clause 7 (Manufacture) with respect to Manufacture of Products) use, offer for sale, sell, have sold, import, and export the Products in the Field (“WPRE Option”). The Licensee may exercise the WPRE Option by written notice to BioMedica. Upon exercise of the WPRE Option, the licence under the WPRE Patent shall be deemed part of the definition of “Licensed Patents”.

2.3
Retained Rights


20





Notwithstanding anything in this Agreement to the contrary, BioMedica retains a worldwide, non-exclusive, sub-licensable (including through one or multiple tiers) right under the BioMedica IP:

(a)
to the extent necessary to give effect to and perform the obligations of BioMedica and its Affiliates under the agreement between L’Assistance Publique – Hopitaux de Paris, Le Commissariat a L’Energie Atomique et aux Energies Alternatives (CEA), and Oxford BioMedica (UK) Limited, dated 7 March 2017; and

(b)
to the extent necessary to perform its obligations under this Agreement and any other written agreement between the Parties.

2.4
Sublicensing

(a)
The Licensee may grant sublicenses through one or multiple tiers, under any or all of the rights granted in Clause 2.1 (Grant of License; Right of Reference), to its Affiliates and to Third Parties. The Licensee shall, and shall procure that each of its Affiliates and each Sublicensee shall, notify BioMedica of the identity of any Sublicensee in writing not more than [***] following the grant of rights under the BioMedica IP to such Sublicensee.

(b)
All sublicenses of the rights granted under this Agreement or any sublicense must be in writing and:

(i)
include legally binding provisions at least as stringent as those contained in this Clause 2.4 (Sublicensing) and Clause 12 (Confidentiality);

(ii)
oblige the Sublicensee to report in writing to the Licensee achievement of any milestone event referred to in Clause 8.2 (Development and Regulatory Milestone Payments);

(iii)
oblige the Sublicensee to keep, at its normal place of business, accurate and up-to-date records and books of account in sufficient detail for the determination of Net Sales by the Sublicensee (through multiple tiers). Such books and records shall be retained by the Sublicensee (through multiple tiers) for three (3) years from the date on which they are generated, and shall be maintained in accordance with applicable Accounting Standards;

(iv)
provide that such sublicense shall automatically terminate upon termination of this Agreement; provided that, if the Sublicensee is not in material breach of any of its obligations under such sublicense, at the written request of the Sublicensee to BioMedica made within [***] following the termination of this Agreement, BioMedica shall enter into a direct licence with the Sublicensee on substantially the same terms as this Agreement but limited to the scope granted by the Licensee (or Sublicensee) to the Sublicensee, provided that:

(A)
BioMedica shall not be required to undertake obligations in addition to those required by this Agreement;


21





(B)
the financial terms of such direct licence shall be replaced by the applicable financial terms provided under this Agreement; and

(C)
BioMedica receives, in relation to the Sublicensee, at least those rights that it received in relation to the Licensee under this Agreement.

(c)
The Licensee shall remain liable to BioMedica in respect of any acts or omissions of any Affiliate or Sublicensee (through multiple tiers) that would, if effected by the Licensee, constitute a breach of this Agreement.

2.5
Non-Compete

(a)
BioMedica and its Affiliates shall not, alone or in collaboration with any Third Party, clinically develop, seek Regulatory Approval for, manufacture (or have manufactured), use, sell (or have sold), market, promote, import, export or otherwise commercialise a BioMedica Competing Product during the Term.

(b)
Notwithstanding the restrictions set out in Clause 2.5(a) (Non-Compete):

(i)
in the event that BioMedica or any of its Affiliates (A) acquires, by way of acquisition of the rights in, or acquisition of Control of any Third Party having rights in; or (B) assigns this Agreement, and all of its rights and obligations under this Agreement to a Third Party which itself or one of whose Affiliates is clinically developing, seeking Regulatory Approval for, manufacturing (or having manufactured), using, selling (or having sold), marketing, promoting, importing, exporting or otherwise commercialising; a BioMedica Competing Product, BioMedica shall (or shall procure that its respective applicable Affiliate) divest the BioMedica Competing Product, or cease to develop, seek Regulatory Approval for, manufacture (or have manufactured), use, sell (or have sold), market, promote, import, export or otherwise commercialise the BioMedica Competing Product, as soon as reasonably practicable, and in any case within twelve (12) months, after the completion of the acquisition. “Divest” as used in this Clause 2.5(b) (Non-Compete) shall mean the sale or transfer or exclusive license of rights to the BioMedica Competing Product to a Third Party without receiving a continuing share of profit, royalty payment, or other economic interest in the success of such BioMedica Competing Product;

(ii)
in the event of a Change of Control of BioMedica:

(A)
if, at the time of completion of such Change of Control of BioMedica, the Third Party acquirer has rights in a BioMedica Competing Product, neither the Third Party acquirer nor any of its Affiliates existing immediately prior to such transaction will have any obligations under this Clause 2.5 (Non-Compete) with respect to such BioMedica Competing Product, provided that such Third Party acquirer shall not have access to, and shall not refer to, rely upon, or use in any manner, the BioMedica IP or any intellectual property or Confidential Information of the Licensee


22





for the continued conduct of such BioMedica Competing Product; and

(B)
such Third Party acquirer or any of its Affiliates (excluding BioMedica and its Affiliates immediately before such Change of Control of BioMedica) may commence development, manufacture, using, selling, marketing, promoting, importing, exporting, and otherwise commercialising any BioMedica Competing Product, provided that each entity that is responsible for such BioMedica Competing Product shall not have access to, and shall not refer to, rely upon, or use in any manner, the BioMedica IP or any intellectual property or Confidential Information of the Licensee for the continued conduct of such BioMedica Competing Product.

(c)
The Licensee and its Affiliates shall not, alone or in collaboration with any Third Party, clinically develop, seek Regulatory Approval for, manufacture (or have manufactured), use, sell (or have sold), market, promote, import, export or otherwise commercialise a Licensee Competing Product during the Term.

(d)
Notwithstanding the restrictions set out in Clause 2.5(c) (Non-Compete);

(i)
in the event that the Licensee or any of its Affiliates (A) acquires, by way of acquisition of the rights in or acquisition of Control of any Third Party having rights in; or (B) assigns this Agreement, and all of its rights and obligations under this Agreement to a Third Party which itself or one of whose Affiliates is clinically developing, seeking Regulatory Approval for, manufacturing (or having manufactured), using, selling (or having sold), marketing, promoting, importing, exporting or otherwise commercialising; a Licensee Competing Product, the Licensee shall (or shall procure that its respective applicable Affiliate) divest the Licensee Competing Product, or cease to develop, seek Regulatory Approval for, manufacture (or have manufactured), use, sell (or have sold), market, promote, import, export or otherwise commercialise the Licensee Competing Product, as soon as reasonably practicable, and in any case within twelve (12) months, after the completion of the acquisition. “Divest” as used in this Clause 2.5(d) (Non-Compete) shall mean the sale or transfer or exclusive license of rights to the Licensee Competing Product to a Third Party without receiving a continuing share of profit, royalty payment, or other economic interest in the success of such Licensee Competing Product;

(ii)
in the event of a Change of Control of the Licensee:

(A)
if, at the time of completion of such Change of Control of the Licensee, the Third Party acquirer has rights in a Licensee Competing Product, neither the Third Party acquirer nor any of its Affiliates existing immediately prior to such transaction will have any obligations under this Clause 2.5 (Non-Compete) with respect to such Licensee Competing Product, provided that such Third Party acquirer shall not have access to, and shall not refer to, rely upon, or use in any manner, the BioMedica IP;


23





(B)
such Third Party acquirer or any of its Affiliates (excluding the Licensee and its Affiliates immediately before such Change of Control of the Licensee) may commence development, manufacture, using, selling, marketing, promoting, importing, exporting, and otherwise commercialising any Licensee Competing Product provided each entity that is responsible for such Licensee Competing Product shall not have access to, and shall not refer to, rely upon, or use in any manner, the BioMedica IP.

(e)
Each of the Parties recognises that the restrictions contained in this Clause 2.5 (Non-Compete) are properly required for the adequate protection of the Parties’ rights hereunder, and agree that if any provision in this Clause 2.5 (Non- Compete) is determined by any court to be unenforceable by reason of its extending for too great a period of time or over too great a geographic area, or by reason of its being too extensive in any other respect, such restrictions shall be interpreted to extend only for the longest period of time and over the greatest geographic area, and to otherwise have the broadest application as shall be enforceable.

2.6
BioMedica Reserved Product

(a)
If BioMedica commences development of a BioMedica Reserved Product, BioMedica may not grant rights to any Third Party to develop or commercialise such BioMedica Reserved Product until it has followed the procedure set out in this Clause 2.6 (BioMedica Reserved Product).

(b)
BioMedica shall notify the Licensee in writing promptly after the earlier of the date on which BioMedica (i) initiates substantial discussions with, or receives a term sheet from, a Third Party regarding the grant of a licence to such Third Party in respect of the BioMedica Reserved Product; or (ii) obtains final results of the first clinical trial of such BioMedica Reserved Product. If subsequently requested in writing by the Licensee, BioMedica shall provide to the Licensee the full, then-existing pre-clinical and clinical data in relation to the BioMedica Reserved Product within the Control of BioMedica (the date of such provision being the “Start Date”), provided that such Know-How shall be kept confidential by the Licensee pursuant to Clause 12 (Confidentiality), used only to for the purpose of determining whether to enter into an agreement with BioMedica in relation to the BioMedica Reserved Product, and disclosed only to those employees of the Licensee and its Affiliates who need to know the same for such purpose.

(c)
Within [***] after the Start Date, the Licensee may notify BioMedica in writing that it wishes to negotiate in good faith a licence in respect of the BioMedica Reserved Product. If the Licensee issues such notice:

(i)
the Parties shall negotiate in good faith, during the period expiring [***] after the Licensee’s notice to BioMedica that it wishes to negotiate a licence in respect of the BioMedica Reserved Product (“Exclusivity Period”), the grant of such licence by BioMedica to the Licensee; and


24





(ii)
during the Exclusivity Period (or the end of such negotiations without entering into a definitive agreement, if earlier), BioMedica shall not grant to any Third Party any licence in respect of the BioMedica Reserved Product.

(d)
If the Parties do not enter into a definitive agreement for the BioMedica Reserved Product during the Exclusivity Period, then BioMedica shall not, within a period of twelve (12) months thereafter, grant to any Third Party a licence in respect of the BioMedica Reserved Product on terms which are materially more favourable to such Third Party than the terms offered to the Licensee when taking into account only (i) the scope and exclusivity of rights granted, and (ii) the monetary payments due to BioMedica or its Affiliates.

2.7
Compliance with Applicable Laws

In the course of performing its obligations or exercising its rights pursuant to this Agreement, each Party:

(a)
shall comply with, and shall cause its and its Affiliates’ and Sublicensees’ employees and contractors to comply with, all Applicable Laws; and

(b)
shall not employ or use the services of any person that has been debarred for example as under clause 306(a) or 306(b) of the United States Federal Food, Drug, and Cosmetic Act, as amended. If either Party becomes aware of the debarment or disqualification or threatened debarment or disqualification of any person providing services to such Party, including the Party itself or its Affiliates or Sublicensees, that directly or indirectly relate to activities contemplated by this Agreement, such Party shall immediately notify the other Party in writing and such Party shall cease employing, contracting with, or retaining any such person to perform any such services.

3.
MANAGEMENT

3.1
Clinical Project Team

(a)
Within [***] after the Effective Date, the Parties shall establish a Clinical Project Team (“Clinical Project Team”) with each Party designating and notifying the other Party of its initial members to serve on the Clinical Project Team.

(b)
The Clinical Project Team will remain in place until completion of the transfer of the Clinical Trials and, unless otherwise requested in writing by the Licensee for a reasonable extension period, will be disbanded at the end of such period.

(c)
The Clinical Project Team shall consist of personnel of BioMedica and the Licensee (or its Affiliate), in each case, with appropriate background and expertise to contribute to the Clinical Project Team. Each Party may change its members on the Clinical Project Team from time to time upon written notice to the other Party.

(d)
Either Party may, from time to time, invite additional representatives or consultants, who are not Clinical Project Team members but who have


25





knowledge and experience necessary to assist the Clinical Project Team, to attend Clinical Project Team meetings, subject to (i) approval of the other Party (not to be unreasonably withheld, conditioned, or delayed) and (ii) such representatives and consultants being bound by confidentiality obligations at least as stringent as those in this Agreement.

(e)
The Licensee shall appoint one (1) of its members to chair Clinical Project Team meetings, ensure the orderly conduct of Clinical Project Team meetings, and ensure that written minutes of each Clinical Project Team meeting are taken and issued to each of the Parties.

(f)
The Clinical Project Team shall meet as often as required. Such meetings may be conducted by telephone, videoconference or in person as determined by the chairperson. Each Party may call for meetings of the Clinical Project Team with reasonable prior notice (it being agreed that at least [***] shall constitute reasonable notice), to discuss matters within the purview of the Clinical Project Team.

3.2
Role of Clinical Project Team

The Clinical Project Team shall, subject to Clause 3.5 (Limits), be responsible for:

(a)
planning and overseeing the conduct of the Ongoing Clinical Trials until transfer and the transfer of the Ongoing Clinical Trials to the Licensee;

(b)
providing a forum for, and facilitation of, communications between the Parties with respect to the Clinical Trials;

(c)
providing a forum for updates regarding safety, regulatory and other similar information as required to be provided by BioMedica under this Agreement; and

(d)
agreeing, by consensus of both Parties, the period of time for which the Licensee shall utilise, pursuant to Clause 6.2 (Engagement of Experienced BioMedica Personnel), the services of the OXB Project Personnel.

3.3
Process Development Project Team

(a)
Within [***] after the Effective Date, the Parties shall establish a Process Development Project Team (“Process Development Project Team”) with each Party designating and notifying the other Party of its initial members to serve on the Process Development Project Team.

(b)
The Process Development Project Team will remain in place until completion of the last Work Package and, unless otherwise requested in writing by the Licensee for a reasonable extension period, will be disbanded at the end of such period.

(c)
The Process Development Project Team shall consist of personnel of BioMedica and the Licensee (or its Affiliate), in each case, with appropriate background and expertise to contribute to the Process Development Project Team. Each Party may change its members on the Process Development Project Team from time to time upon written notice to the other Party.


26





(d)
Either Party may, from time to time, invite additional representatives or consultants, who are not Process Development Project Team members but who have knowledge and experience necessary to assist the Process Development Project Team, to attend Process Development Project Team meetings, subject to (i) approval of the other Party (not to be unreasonably withheld, conditioned, or delayed) and (ii) such representatives and consultants being bound by confidentiality obligations at least as stringent as those in this Agreement.

(e)
The Licensee shall appoint one (1) of its members to chair Process Development Project Team meetings, ensure the orderly conduct of Process Development Project Team meetings, and ensure that written minutes of each Process Development Project Team meeting are taken and issued to each of the Parties.

(f)
The Process Development Project Team shall meet as often as required. Such meetings may be conducted by telephone, videoconference or in person as determined by the chairperson. Each Party may call for meetings of the Process Development Project Team with reasonable prior notice (it being agreed that at least [***] shall constitute reasonable notice), to discuss matters within the purview of the Process Development Project Team.

3.4
Role of Process Development Project Team

The Process Development Project Team shall, subject to Clause 3.5 (Limits), be responsible for:

(a)
preparing descriptions of Work Packages, which must be reasonably, mutually agreed to in good faith in writing and executed by an authorised representative of each Party in order to become effective;

(b)
discussing amendments to Work Packages and making recommendations to authorised representatives of each Party regarding such amendments;

(c)
providing a forum for, and facilitation of, communications between the Parties with respect to the Work Packages and the results of Work Packages; and

(d)
discussing the Work Packages and overseeing and monitoring their performance.

3.5
Limits

Notwithstanding any provision to the contrary, neither the Clinical Project Team nor the Process Development Project Team shall have any power to make any decisions, amend or modify the provisions of this Agreement or any Work Package, or to waive compliance with this Agreement and each Party shall retain the rights, powers and discretion granted to it under this Agreement and no such rights, powers or discretion shall be delegated to or vested in the Clinical Project Team or the Process Development Project Team.

3.6
Scientific Advisory Board

The Licensee shall establish a scientific advisory committee (the “Scientific Advisory Board”) to provide advice regarding the clinical and scientific aspects of the development of the


27





Product. The Licensee shall invite up to two (2) appropriately qualified individuals nominated by BioMedica to take part in each meeting of the Scientific Advisory Board. BioMedica shall use reasonable efforts to ensure that such individuals are reasonably available for participation in such Scientific Advisory Board.

3.7
Costs of Governance

The costs incurred by each Party in connection with attendance at meetings of the Clinical Project Team, Process Development Project Team and Scientific Advisory Board shall be borne solely by such Party.

4.
TRANSITION AND REPORTING ACTIVITIES

4.1
Transfer of Know-How and Ongoing Clinical Trials

(a)
BioMedica shall transfer the Licensed Know-How (excluding the Licensed Know- How referred to in Clause 1.1(k)(iii) (BioMedica IP)) and the Ongoing Clinical Trials to the Licensee pursuant to this Clause 4.1 (Transfer of Know-How and Ongoing Clinical Trials).

(b)
The Parties acknowledge and agree that as of Effective Date, BioMedica is conducting the Prosavin Clinical Trial and preparing for the initiation of the OXB- 102 Clinical Trial (the “Ongoing Clinical Trials”), and has entered into various agreements with Third Parties in connection with the conduct of such Ongoing Clinical Trials.

(c)
Within [***] after the Effective Date, BioMedica shall make available for download by the Licensee all Licensed Know-How (excluding the Licensed Know-How referred to in Clause 1.1(k)(iii) (BioMedica IP)) which immediately before the Effective Date was made available to the Licensee to view in the electronic due diligence data room.

(d)
Without prejudice to Clause 4.1(c) (Transfer of Know-How and Ongoing Clinical Trials), BioMedica shall transfer all Licensed Know-How (excluding the Licensed Know-How referred to in Clause 1.1(k)(iii) (BioMedica IP)) and responsibility for the Ongoing Clinical Trials to the Licensee as soon as practicable after the Effective Date in accordance with this Clause 4.1 (Transfer of Know-How and Ongoing Clinical Trials), and in order to execute such transfer:

(i)
within [***] after the Effective Date, the Clinical Project Team shall prepare and submit to authorised representatives of the Parties for execution, a transfer plan and budget (“Transfer Plan”) regarding the activities to be performed by the Parties in connection with the transfer of such Licensed Know-How and all responsibility for all Ongoing Clinical Trials from BioMedica to the Licensee;

(ii)
such Transfer Plan shall include the responsibilities of each of the Parties in relation to:

(A)
transfer of all pre-clinical and clinical data Controlled by BioMedica that was generated in the course of the Ongoing Clinical Trials relating specifically to the Products in the Field and that is necessary or


28





reasonably useful for the research, development, use, sale, import, or export of the Products in the Field, including all clinical study reports (including the final clinical study report), results and other data in each case related to the Ongoing Clinical Trials;

(B)
transfer of the applicable INDs for such Ongoing Clinical Trials from BioMedica to the Licensee;

(C)
transfer or assignment, as applicable and to the extent possible, of BioMedica’s rights and obligations under contracts with Third Parties relating to the Ongoing Clinical Trials;

(D)
transfer of all Regulatory Filings that are necessary to conduct the Ongoing Clinical Trials;

(E)
transfer physical possession of the Existing Inventory to, at the Licensee’s discretion, either (1) the Licensee, (2) the site of an Ongoing Clinical Trial, or (3) any contractor of the Licensee; and

(F)
transfer or assignment, as applicable and to the extent possible, of BioMedica’s rights and obligations under contracts with Third Parties relating to the Existing Inventory; and

(iii)
following execution of such Transfer Plan by authorised representatives of the Parties, each of the Parties shall perform their obligations under such Plan to expeditiously transfer of all responsibility for all Ongoing Clinical Trials from BioMedica to the Licensee, and BioMedica shall provide the Licensee with reasonable access during normal business hours (upon reasonable advance notice), to BioMedica personnel either in-person at BioMedica’s facility or by teleconference as required for the performance of such activities by BioMedica.

(e)
With effect from the date on which all responsibility for an Ongoing Clinical Trial is transferred from BioMedica to the Licensee, all legal and regulatory responsibility for such Ongoing Clinical Trial shall transfer to the Licensee.

(f)
The Licensee shall reimburse BioMedica for reasonable time spent by BioMedica in connection with preparation and performance by BioMedica of the Transfer Plan at the FTE Rate (except to the extent that such obligations are performed by the OXB Project Personnel pursuant to Clause 6.2 (Engagement of Experienced BioMedica Personnel), in which case the time spent by such OXB Project Personnel shall be paid for by the Licensee in accordance with Clause 6.2 (Engagement of Experienced BioMedica Personnel)). BioMedica shall provide reasonably detailed invoices to the Licensee for such time spent by BioMedica, and the Licensee shall pay the undisputed amount invoiced within [***] after the date of any such invoice.

(g)
The Licensee shall reimburse BioMedica, within [***] after issue of a reasonably detailed invoice by BioMedica to the Licensee, for amounts that BioMedica reasonably incurs after the Effective Date with respect to the Ongoing Clinical Trials.


29





4.2
Safety Data Exchange

The Parties shall, promptly after the Effective Date, enter into such safety data exchange agreements as appropriate to cover the exchange of safety data relating to the Product and the Ongoing Clinical Trials.

4.3
Interim Performance of Ongoing Clinical Trials

(a)
Notwithstanding Clause 6.1 (Development) and Clause 6.3 (Regulatory), until completion of the transfer of the Ongoing Clinical Trials in accordance with Clause 4.1 (Transfer of Know-How and Ongoing Clinical Trials), BioMedica shall:

(i)
be the sponsor of the Ongoing Clinical Trials and shall conduct the Ongoing Clinical Trials in accordance with the applicable clinical trial protocols. BioMedica shall consult with the Licensee in relation to all aspects of the conduct of the Ongoing Clinical Trial and shall implement all decisions and directions of the Licensee which are consistent with the protocols and Applicable Law, provided that BioMedica shall be entitled to take any steps it deems necessary in connection with patient safety in connection with the Ongoing Clinical Trials;

(ii)
remain the primary contact for Regulatory Authorities with respect to the Ongoing Clinical Trials;

(iii)
keep the Licensee reasonably informed as to any interactions with any Regulatory Authorities relating to the Ongoing Clinical Trials, shall provide any Regulatory Filings and material correspondence with any Regulatory Authorities relating to the Ongoing Clinical Trials to the Licensee for the Licensee’s review and comment; and

(iv)
remain responsible for all safety reporting obligations to the Regulatory Authorities with respect to the Ongoing Clinical Trials; provided that, BioMedica shall keep the Licensee reasonably informed as to any such matters reported.

(b)
BioMedica shall issue invoices to the Licensee in respect of the following costs and expenses incurred by BioMedica in connection with BioMedica’s interim conduct of the Ongoing Clinical Trials and transfer of the Ongoing Clinical Trials to the Licensee, in each case after the Effective Date:

(i)
except to the extent that such obligations are performed by the OXB Project Personnel pursuant to Clause 6.2 (Engagement of Experienced BioMedica Personnel), fees for the time spent by BioMedica after the Effective Date in connection with conduct of the Ongoing Clinical Trials at the FTE Rate;

(ii)
all reasonable, out-of-pocket expenses incurred by BioMedica after the Effective Date in connection with conduct of the Ongoing Clinical Trials provided that, BioMedica shall include adequate supporting documentation for such expenses together with any invoices; and


30





(iii)
the reasonable cost of all devices, consumables and the like used in connection with performance of the Ongoing Clinical Trials after the Effective Date; and

such invoices shall be itemized and include a reasonably detailed description of the work actually performed, and the Licensee shall pay such invoices within [***] after the date of any such invoice.

5.
BIOMEDICA PROCESS DEVELOPMENT

5.1
Process Development Services

(a)
Promptly after the Effective Date the Process Development Project Team shall prepare, and submit to authorised representatives of the Parties for execution, one or more Work Packages pursuant to which BioMedica shall conduct process development activities in relation to the Manufacture of the Products (the “Process Development Services”). Each Work Package shall:

(i)
become binding only once it is recorded in writing and is signed by authorised representatives of both Parties;

(ii)
set out a fixed price for the performance by BioMedica of the activities described and results required (as applicable) under such Work Package to the extent relating to the development of a serum-free suspension process;

(iii)
set out reasonable estimates on the basis of time (at the FTE Rate) and materials for the performance by BioMedica of additional activities described (other than the development of a serum-free suspension process) and results required (as applicable) under such Work Package(s);

(iv)
describe the items to be provided by BioMedica to the Licensee under such Work Package(s), including items specifically designated or characterized as deliverables in a Work Package.

(b)
In the event of any conflict between this Agreement and a Work Package, this Agreement shall control unless the Work Package expressly refers to the Parties’ intent to alter the terms of this Agreement with respect to that Work Package.

(c)
Upon the receipt of a proposal from the Licensee to change the terms of a Work Package (a “Change Proposal”), the Parties shall discuss such Change Proposal in good faith. Changes to a Work Package will not be effective unless mutually agreed in writing by the Parties.

5.2
Performance of Work Packages

(a)
BioMedica shall perform the activities set out in the Work Packages in accordance with the provisions of such Work Package, this Agreement, and all Applicable Laws.


31





(b)
BioMedica shall perform such activities in a professional manner, in conformance with that level of care and skill ordinarily exercised by other professionals in the biopharmaceutical industry in similar circumstances.

(c)
The Licensee shall promptly provide to BioMedica all materials, information, advice, approvals and other assistance reasonably required by BioMedica in connection with its performance of the Work Packages. BioMedica shall not be liable for any breach of this Agreement arising from any delay or failure of the Licensee to provide the same.

(d)
BioMedica may subcontract the performance of its obligations under the Work Packages to any Affiliate or Third Party provided that BioMedica remains responsible for (i) the work allocated to such subcontractors to the same extent it would if it had done such work itself, (ii) monitoring the performance of such subcontractors and (iii) any action or inaction by any such subcontractor that if taken or not taken by BioMedica would constitute a breach of this Agreement.

(e)
BioMedica shall use reasonable efforts to comply with any timelines, milestones, schedules, or target dates for completing the services or any portion thereof as set forth in a Work Package; provided that the Parties acknowledge that unless specified in a Work Package, the nature of the work to be conducted under the Work Packages(s) is of an experimental nature and as such, any timelines set out in any Work Package are good faith estimates only; provided further that BioMedica recognizes the importance of the Process Development Services to the Licensee and agrees to allocate to the performance of such services an equivalent level of prioritization as it allocates to similar activities BioMedica performs for Third Parties. BioMedica shall promptly inform the Process Development Project Team upon becoming aware of any unforeseen results, problems, difficulties, delays, or the like with regard to the activities under the Work Packages and the estimated duration of any delay.

5.3
Record Keeping and Reporting

(a)
BioMedica shall create and maintain written records of the data and other information generated or recorded in the performance of the Process Development Services (the “Records”) in sufficient detail and in a good scientific manner, including all Process Development Results. During the course of conducting the Process Development Services, BioMedica shall, at the Licensee’s request and expense, provide the Licensee with copies of the Records.

(b)
BioMedica shall provide updates on the status and results of the Work Packages to the Process Development Project Team and shall provide reports outlining the progress of the Process Development Services in accordance with the applicable Work Package.

5.4
Process Development Results

(a)
Notwithstanding Clause 11.1 (Ownership), the Licensee owns all right, title and interest in and to the Process Development Results that solely and specifically relate to a Product such that they cannot reasonably be used in connection with the manufacture of any other product (the “Product-Specific Process Development Results”). BioMedica


32





hereby assigns and transfers to the Licensee all of its right, title and interest in and to the Product-Specific Process Development Results. BioMedica shall take, and shall cause its employees and contractors to take, all further acts reasonably required to evidence such assignment and transfer to the Licensee, at the Licensee’s reasonable expense. The Licensee may, at its expense, to prepare, file, prosecute and maintain any patent applications and patents claiming the Product-Specific Process Development Results provided that such patent applications do not include any BioMedica IP.

(b)
Notwithstanding Clause 11.1 (Ownership), BioMedica owns all right, title and interest in and to the Process Development Results that are not Product-Specific Process Development Results (the “General Process Development Results”). BioMedica hereby grants to the Licensee a non-exclusive, perpetual, irrevocable, worldwide, royalty-free, sublicenseable (through one or multiple tiers) license under all General Process Development Results to research, develop, Manufacture and have Manufactured (subject to Clause 7 (Manufacture) with respect to Manufacture of Products), use, offer for sale, sell, have sold, import, and export (i) the Products in the Field and (ii) any other products that BioMedica manufactures for the Licensee or its Affiliates, including pursuant to separate agreements.

5.5
Process Development Services Costs

(a)
Subject to the terms and conditions of this Agreement, the Licensee shall pay BioMedica the fees specified in each Work Package (“Fees”) as BioMedica’s sole and complete compensation for all Process Development Services and Process Development Results.

(b)
BioMedica shall issue invoices to the Licensee in respect of the Fees associated with each Work Package in accordance with the terms of each Work Package. BioMedica may not submit for payment any invoice for services that the Licensee has not consented to pursuant to an executed Work Package or Change Proposal.

(c)
Except as otherwise stated in the Work Package and subject to Clause 9.3 (Payment), such invoices shall be exclusive of VAT or other applicable taxes and the Licensee shall pay such invoices within [***] after receipt of such invoice.

(d)
Notwithstanding the foregoing, the Licensee is entitled to, and BioMedica shall issue to the Licensee, a credit in the amount of [***] (being a portion of the upfront payment received by BioMedica), such credit to be applied to (i) amounts that would otherwise have become due in accordance with the Work Packages (and representing the reimbursement of BioMedica’s costs of salaries, equipment and overheads in providing the Process Development Services) and (ii) the cost of procuring at least one (or more if such credit has not been fully used) clinical batches of Product that is Manufactured using the manufacturing process developed pursuant to such Work Packages, excluding the cost of Plasmids. BioMedica shall include on each invoice raised with respect to such amounts the amount of the credit used, the total amount of such credit remaining and a description of the work actually performed in sufficient detail to permit reasonable


33





comparison to the description of activities agreed to be completed under the applicable Work Package. To the extent that the amount of the Work Packages and the cost of procuring one clinical batch of Product that is Manufactured using the manufacturing process developed pursuant to such Work Packages exceed such [***] credit, BioMedica shall be responsible for such costs and expenses and shall not invoice the Licensee for such amounts. Notwithstanding the foregoing, BioMedica shall issue invoices to the Licensee for all Plasmids used by BioMedica in the course of Manufacture of the Product, and the Licensee shall pay such invoices within [***] after receipt of such invoice.

6.
DEVELOPMENT, REGULATORY, AND COMMERCIALISATION

6.1
Development

(a)
Following completion of the transfer of the Ongoing Clinical Trials pursuant to Clause 4.1 (Transfer of Know-How and Ongoing Clinical Trials), the Licensee shall have sole discretion (subject to the provisions of this Agreement including Clause 6.2 (Engagement of Experienced BioMedica Personnel)), control and responsibility, at the Licensee’s sole cost, to conduct development of Products (including all clinical trials, formulation studies, and regulatory activities) that is necessary for or otherwise supports obtaining and maintaining Regulatory Approval for Products in the Field.

(b)
The Licensee may perform its development activities under this Agreement through one or more subcontractors, provided that the Licensee remains responsible for (i) the work allocated to such subcontractors to the same extent it would if it had done such work itself, (ii) monitoring the performance of such subcontractors and (iii) any action or inaction by any such subcontractor that if taken or not taken by the Licensee would constitute a breach of this Agreement.

(c)
As soon as practicable and no later than [***] after the Effective Date, the Licensee shall provide to BioMedica an initial plan in reasonable detail of any development activities and regulatory strategy anticipated to be conducted to obtain Regulatory Approval and Pricing and Reimbursement Approval (as applicable) of the Product in each Major Market, including good faith estimates of dates for reaching each Development Milestone and Regulatory Milestone as set out in Clause 8.2 (Development and Regulatory Milestone Payments). Promptly after the expiry of each six (6) month period thereafter, the Licensee shall provide to BioMedica an update of such development plan, including an update of the good faith estimates of dates for reaching each remaining Development Milestone and Regulatory Milestone as set out in Clause 8.2 (Development and Regulatory Milestone Payments), together with a summary in reasonable detail of all development activities conducted by the Licensee in the preceding [***] including, as applicable, the status of any Clinical Trials of the Product. The Parties acknowledge that such statements shall be used by BioMedica in the assessment of the performance by the Licensee of its diligence obligations under this Agreement and may be disclosed by BioMedica to any Third Party to whom BioMedica has reporting obligations with respect to the Product, in each case under written obligations of confidentiality and non- use at least as stringent as those herein.


34





6.2
Engagement of Experienced BioMedica Personnel

(a)
With effect from the Effective Date, the Licensee shall, in connection with (i) the conduct of the Transfer Plan, and (ii) performance of the Ongoing Clinical Trials, use reasonable efforts to utilise the services of up to six (6) FTEs that are engaged by BioMedica at the time of such performance as employees, consultants, or contractors (“OXB Project Personnel”) for the longer of (A) one
(1) year after the Effective Date and (B) the period of time determined by the Clinical Project Team; provided, that the Licensee may cease utilising the services of any OXB Project Personnel who fail to meet the Licensee’s reasonable performance standards, as the Licensee applies to its own employees, consultants, and contractors. Such FTEs shall be made up of the time commitment set out in Schedule 2 by the individuals described in Schedule 2 , which individuals have the relevant background knowledge and experience relating to the Products and the Ongoing Clinical Trials. The OXB Project Personnel shall transfer such Licensed Know-How as is in their possession to the Licensee in the course of conducting the Transfer Plan. If any of the OXB Project Personnel are unable or unwilling to continue as OXB Project Personnel or terminates his or her employment relationship with BioMedica, BioMedica shall promptly notify the Licensee in writing, and shall use reasonable efforts to find a suitable replacement to serve as OXB Project Personnel, such replacement subject to the Licensee’s prior written consent, such consent not to be unreasonably withheld, conditioned, or delayed.

(b)
BioMedica shall issue invoices to the Licensee in respect of fees for the time spent by the OXB Project Personnel in connection with the conduct of the Transfer Plan, and performance of the Ongoing Clinical Trials at the FTE Rate, provided that in the event that the Licensee does not utilise the full time commitment of the relevant individuals as set out in Schedule 2 for each year of the period of time described in Clause 6.2(a) (Engagement of Experienced BioMedica Personnel), then so long as such individuals are able and willing to serve as OXB Project Personnel and meeting the Licensee’s reasonable performance standards, then BioMedica shall be entitled to invoice for the full time commitment so specified. The Licensee shall pay such invoices within [***] after receipt of the same.

(c)
In order to protect the legitimate business interests of BioMedica, the Licensee shall not (and shall procure that its Affiliates do not) during the term of the Ongoing Clinical Trials and for a period of [***] following completion of the transfer of the Ongoing Clinical Trials (i) attempt to solicit or entice away, or (ii) solicit or entice away, from the employment or service of BioMedica, any OXB Project Personnel other than by means of a public advertising campaign not specifically targeted at such individuals; provided that, it is understood and agreed that an offer of employment to any OXB Project Personnel that results from an unsolicited inquiry by such person will not be deemed to be a violation of this provision.

6.3
Regulatory

Following completion of the transfer of the Ongoing Clinical Trials pursuant to Clause 4.1 (Transfer of Know-How and Ongoing Clinical Trials), the Licensee shall have sole discretion (subject to the provisions of this Agreement), control and responsibility, at the Licensee's sole

35





cost, to draft, prepare, submit and file, all INDs, BLAs, MAAs and other regulatory documents, dossiers and filings (collectively, the “Regulatory Filings”) with respect to the Products. All such Regulatory Filings shall be in the name of, and be owned solely by, the Licensee. BioMedica shall provide the Licensee, in a reasonably timely manner, but subject to the Licensee reimbursing BioMedica for all internal costs (at the FTE Rate) and out-of-pocket costs incurred by BioMedica in connection therewith, with such assistance as may be reasonably requested by the Licensee for the Licensee’s preparation of those portions of each Regulatory Filing and responses to requests from Regulatory Authorities that are specifically related to Manufacture of the Products, including provision of Licensed Know-How necessary for inclusion in such Regulatory Filings.

6.4
Commercialization

(a)
Subject to the terms and conditions of this Agreement, the Licensee has the sole and exclusive control over all matters relating to the commercialization of Products in the Field. Without limiting the foregoing, the Licensee has the sole and exclusive right and control for the following with respect to Products in the Field: (a) developing and executing a commercial launch and pre-launch plan,
(b)      negotiating with applicable Regulatory Authorities regarding the price and reimbursement status of Products; (c) marketing and promotion; (d) booking sales and distribution and performance of related services; (e) handling all aspects of order processing, invoicing and collection, inventory and receivables;
(f) providing customer support, including handling medical queries, and performing other related functions; and (g) conforming its practices and procedures to Applicable Laws relating to the marketing, detailing and promotion of Products in the Field. As between the Parties, the Licensee shall bear all of its costs and expenses incurred in connection with such commercialization activities.

(b) At least [***] before First Commercial Sale, the Licensee shall provide to BioMedica an initial plan of launch and marketing activities for the Major Markets with respect to the Product. Promptly after each anniversary of the Effective Date, the Licensee shall provide to BioMedica an update of such plan, together with a high-level summary of the commercialisation activities conducted by the Licensee in the past [***]. The Parties acknowledge that such statements shall be used by BioMedica in the assessment of the performance by the Licensee of its diligence obligations under this Agreement and may be disclosed by BioMedica to any Third Party to whom BioMedica has reporting obligations with respect to the Product, in each case under written obligations of confidentiality and non-use at least as stringent as those herein.

6.5
Diligence

Without regard to whether or not the Licensee achieves the Diligence Milestones, the Licensee shall use Commercially Reasonable Efforts to:

(a)
develop and seek Regulatory Approval and Pricing and Reimbursement Approval (to the extent applicable) for at least one Product in (i) the United States and (ii) at least one of the United Kingdom, Germany, France, Spain and Italy; and


36





(b)
commercialise each Product in each country for which the Licensee obtains Regulatory Approval.

6.6
Diligence Milestones

Notwithstanding the generality of Clause 6.5 (Diligence), the Licensee shall or shall procure that its Affiliates or a Sublicensee shall:

(a)
[***];

(b)
[***]; and

(c)
[***];

(collectively, the “Diligence Milestones”).

6.7
Failure to Meet Diligence Milestones

If the Licensee fails to achieve a Diligence Milestone set forth in Clause 6.6 (Diligence Milestones), then the Licensee shall pay to BioMedica (within [***] after receipt of an invoice from BioMedica) the amounts set forth in Clauses 6.7(a)–(c) (Failure to Meet Diligence Milestones). Upon receipt of such payment, BioMedica shall not be permitted to declare a material breach of Clause 6.6 (Diligence Milestones) for the Licensee’s failure to achieve such Diligence Milestone (provided for clarity that this clause shall not limit or exclude any right or remedy of BioMedica for breach of Clause 6.5 (Diligence)).

(a)
in the event that the Diligence Milestone set forth in Clause 6.6(a) (Diligence Milestones) is not achieved: [***] upon the first anniversary of the Effective Date and each anniversary of the Effective Date thereafter until such time as such Diligence Milestone is met;

(b)
in the event that the Diligence Milestone set forth in Clause 6.6(b) (Diligence Milestones) is not achieved: [***] upon the second anniversary of the Effective Date and each anniversary of the Effective Date thereafter until such time as such Diligence Milestone is met; and

(c)
in the event that the Diligence Milestone set forth in Clause 6.6(c) (Diligence Milestones) is not achieved: [***], provided that it is understood and agreed that [***];


37





provided, that Licensee shall not be required to pay any of the foregoing amounts of the Licensee’s failure to achieve a diligence milestone is directly attributable to a failure of BioMedica to supply the Licensee with Product.

6.8
Trademarks

The Parties shall negotiate in good faith to enter into a trademark agreement under which BioMedica shall grant a licence under certain trademarks Controlled by BioMedica that are related to the Products. The Licensee may brand Products using trademarks, logos, and trade names it determines appropriate for such Products, which may vary by region or within a region (the “Product Marks”); provided, that the Licensee shall display and use (and shall procure that its Affiliates and Sublicensees display and use) trademarks of BioMedica included in the trademark agreement in relation to the Product in accordance with Applicable Law and as agreed in the trademark agreement. The Licensee shall own all rights in the Product Marks (other than those licensed from BioMedica under the trademark agreement) and shall register and maintain the Product Marks that it determines reasonably necessary, at the Licensee’s cost and expense.

7.
MANUFACTURE

7.1
Manufacture and Supply of Product

(a)
BioMedica shall Manufacture the Products for use by the Licensee in the development and commercialization of the Products, subject to and in accordance with the terms of this Agreement and subject to and in accordance with the separate clinical and commercial supply agreements to be negotiated by the Parties as set forth below.

(b)
Within [***] after the Effective Date, the Parties shall negotiate in good faith a clinical supply agreement governing supply of Product by BioMedica for clinical use in accordance with the terms of Schedule 7 – Part 1 . Such clinical supply agreement (the “Clinical Supply Agreement”) shall have the terms set forth on Schedule 7 – Part 1 and such other terms and conditions customary for a clinical supply agreement. Upon request by the Licensee, the Parties shall negotiate in good faith a commercial manufacturing and supply agreement (the “Commercial Supply Agreement”) on commercially reasonable terms, including the terms set forth on Schedule 7 – Part 2 .

7.2
Existing Inventory

(a)
The Parties acknowledge that BioMedica holds batches of Product as of the Effective Date as described in Schedule 6 (the “Existing Inventory”). The Parties acknowledge that as at the Effective Date, the Existing Inventory has not been released by the BioMedica Qualified Person for use in Clinical Trials. BioMedica shall take all commercially reasonable steps to procure the testing, fill, finish and release by the BioMedica Qualified Person of all Existing Inventory, provided that the Parties acknowledge that it cannot be determined at the Effective Date whether or not the Existing Inventory will be released by the BioMedica Qualified Person for use in Clinical Trials. If any of the Existing Inventory is not released by the BioMedica Qualified Person for use in Clinical Trials or does not meet specifications that would enable use in Clinical Trials, then BioMedica shall replace such Existing Inventory with Product that is released by the BioMedica


38





Qualified Person for use in Clinical Trials at its sole cost and expense. The Licensee shall reimburse BioMedica, within [***] after issue of invoices raised by BioMedica, for all costs incurred by BioMedica after the Effective Date in connection with stability studies of the Existing Inventory (or stability studies of such replacement Product provided by BioMedica) and quality control testing necessary for the release of the Existing Inventory by the BioMedica Qualified Person.

(b)
The Parties agree that:

(i)
to the extent that the Existing Inventory is in the possession of BioMedica or its contractors, BioMedica shall store such Existing Inventory in accordance with its current procedures and use commercially reasonable efforts to preserve the integrity of the Existing Inventory pending physical transfer in accordance with the Transfer Plan provided that in the event of any loss or damage to such Existing Inventory after the date of release of the same by the BioMedica Qualified Person for use in Clinical Trials, BioMedica shall not have any liability to the Licensee for such loss;

(ii)
BioMedica shall keep the Licensee regularly informed, through the Clinical Project Team or otherwise, regarding developments in connection with the release by the BioMedica Qualified Person of the Existing Inventory for use in Clinical Trials and the anticipated date of such release, and shall take reasonable steps at the request and expense of the Licensee to assist and facilitate the Licensee to enter into appropriate agreements with the Third Party contractors identified in Schedule 6 ; and

(iii)
title to and risk of loss in the Existing Inventory shall pass to the Licensee, without cost or charge to the Licensee, when the Existing Inventory is released by the BioMedica Qualified Person for use in Clinical Trials.

(c)
With respect to Existing Inventory, as at the date on which such Existing Inventory is released by the BioMedica Qualified Person for use in the Ongoing Clinical Trials, BioMedica warrants that such Existing Inventory has been manufactured:

(i)
in accordance with the specifications that have been provided by BioMedica to the Licensee in the electronic due diligence data room as of the Effective Date, which such specifications are hereby incorporated by reference into this Agreement; and

(ii)
in accordance with Good Manufacturing Practice as defined in European legislation.

7.3
Technology Transfer

(a)
At any time following the completion of formal process characterization studies that enable process definition, process validation or BLA submission in respect of a Product, the Licensee may request that BioMedica perform a one-time-only complete technology transfer sufficient for the Manufacture of the Products by the Licensee, its Affiliate or


39





its Sublicensee or contractor. Following such request, BioMedica shall (1) provide to the Licensee a transfer of all Know-How Controlled by BioMedica that is necessary or reasonably useful to enable the Manufacture of Product by the Licensee and has not otherwise already been transferred under this Agreement, (2) provide to the Licensee a transfer of (i) twenty (20) vials of the master cell bank and twenty (20) vials of the working cell bank, in each case for the production of OXB-102 from the serum-free suspension process; provided that if the technology transfer occurs before payment of the milestone for confirmation of OXB-102 titer from the serum-free suspension process, BioMedica shall also provide to the Licensee sufficient vials of the master cell bank and working cell bank negotiated in good faith, in each case from the serum-containing cell factory process, and (ii) any other tangible materials necessary or reasonably useful to enable the Manufacture of Product by the Licensee and has not otherwise already been transferred under this Agreement, and (3) make BioMedica’s personnel available on a reasonable basis to support and consult with the Licensee with respect to such Manufacture, in each case in accordance with this Clause 7.3 (Technology Transfer) and pursuant to a technology transfer plan and budget agreed to by the Parties in good faith, which will set out the responsibilities of each of the Parties with respect to such technology transfer. In addition:

(i)
in the event that BioMedica has or will contract with Third Parties with respect to the Manufacture of Product, BioMedica will, upon Licensee’s request, grant to the Licensee reasonable access and permission to engage in substantive discussions with such Third Parties and reasonably assist the Licensee in such discussions, and will execute, acknowledge, and deliver such further instruments, and do all further similar acts, as may be necessary or appropriate to carry out the purposes and intent of such technology transfer;

(ii)
such technology transfer shall be completed as promptly as possible and in accordance with generally accepted industry technology transfer standards; and

(iii)
such transfer budget shall provide for the Licensee to pay to BioMedica the then current BioMedica FTE rate for time spent in connection with preparation and performance of the technology transfer plan, together with pre-approved travel and subsistence costs incurred by BioMedica personnel in connection with these activities. The Licensee shall reimburse BioMedica, within [***] after issue of invoices raised by BioMedica, for such time spent by BioMedica pursuant to such transfer plan and budget.

(b)
Notwithstanding Clause 7.3(a) (Technology Transfer), BioMedica shall not be obliged to transfer the Licensed Know-How relating to Manufacture of Products to an Affiliate of the Licensee or a Third Party (and the Licensee may not transfer the Licensed Know-How relating to Manufacture of Products to an Affiliate of the Licensee or a Third Party) unless:

(i)
such Affiliate, Sublicensee or contractor has entered into a legally binding confidentiality agreement with BioMedica on terms at least as stringent as the terms set forth herein; and


40





(ii)
such Affiliate or Third Party is prohibited from further sublicensing or transferring any part of the process to Manufacture of the Product, unless such sublicensing or transferring is to qualify a second supplier to Manufacture the Product; provided that it is understood and agreed that only one (1) Affiliate or Third Party is permitted to be qualified as a second supplier at any time.

The Licensee shall not sublicense its rights to Manufacture the Products except to a Third Party in compliance with the provisions of this Clause 7.3 (Technology Transfer).

(c)
In connection with a transfer of the Licensed Know-How relating to Manufacture of Products to a second supplier pursuant to Clause 7.3(b) (Technology Transfer), BioMedica shall use good faith efforts to make BioMedica personnel available on a reasonable basis to support and consult with Licensee, an Affiliate, or a Third Party, as applicable, but shall have no obligation to effect such a transfer. Licensee hereby acknowledges that, over time, BioMedica resources with the relevant knowledge to provide such support and consultation will diminish. Licensee will reimburse BioMedica within [***] after issuance of invoices by BioMedica for its reasonable and documented costs in connection with such support and consultation.

8.
MONETARY OBLIGATIONS

8.1
License Fee

In partial consideration of the rights granted by BioMedica to the Licensee hereunder, within ten (10) days after receipt of an invoice issued by BioMedica on or promptly after the Effective Date, the Licensee shall pay to BioMedica Thirty Million Dollars ($30,000,000).

8.2
Development and Regulatory Milestone Payments

The Licensee shall promptly (no later than [***] thereafter) notify BioMedica in writing upon first achievement of each of the milestone events set out below, in relation to a Product, by the Licensee, its Affiliate or any Sublicensee. After receipt of such notification from the Licensee, BioMedica shall issue to the Licensee an invoice for the appropriate milestone payment set out below in connection with such milestone event.


41






Development Milestone Event
Development Milestone Payment
[***]
$[***]
[***]
$[***]
[***]
$[***]
[***]
$[***]
[***]
$[***]
[***]
$[***]
[***]
$[***]



Regulatory Milestone Event
Regulatory Milestone Payment
[***]
$[***]
[***]
$[***]
[***]
$[***]
[***]



$[***]
[***]

$[***]

Each milestone payment set forth in this Clause 8.2 (Development and Regulatory Milestone Payments) is payable one time only, regardless of the number of times the corresponding event is achieved by one or more of the same or different Products. In no event will the total amounts payable by the Licensee to BioMedica under this Clause 8.2 (Development and Regulatory Milestone Payments) exceed [***].


42





8.3
Commercial Milestone Payments

The Licensee shall notify BioMedica in writing upon first achievement of the following Annual Net Sales in the Royalty Report for the period during which such Annual Net Sales were achieved. After receipt of such notification from the Licensee, BioMedica shall issue to the Licensee an invoice for the appropriate milestone payment set out below in connection with such Annual Net Sales event:

Annual Net Sales Milestone
Milestone Payment
Annual Net Sales of [***] or more
$[***]
Annual Net Sales of [***]or more
$[***]
Annual Net Sales of [***]or more
$[***]

Each milestone payment is payable one time only, regardless of the number of times the corresponding event is achieved by one or more of the same or different Products. In no event will the total amounts payable by the Licensee to BioMedica under this Clause 8.3 (Commercial Milestone Payments) exceed [***].

8.4
Royalties

(a)
The Licensee shall pay BioMedica royalties as set forth below on Annual Net Sales in respect of all Product sold during the Royalty Term, as calculated by multiplying the applicable royalty rate set forth below by the corresponding amount of Annual Net Sales in such Fiscal Year:

(i)
[***] on that portion of Annual Net Sales that is less than [***];

(ii)
[***] on that portion of Annual Net Sales that is equal to or greater than [***] but less than [***];

(iii)
[***] on that portion of Annual Net Sales that is equal to or greater than [***] but less than [***]; and

(iv)
[***] on that portion of Annual Net Sales of Product that is equal to or greater than [***].

For the purposes of the above: Annual Net Sales shall be measured from the beginning of each Fiscal Year, and the royalty rates set forth above shall be re- set at the beginning of each Fiscal Year.

(b)
If, at any time during the Royalty Term for a Product in a country for which there is no Valid Claim that Covers such Product in such country, one or more Third Parties who are not Sublicensees sell in such country an Alternative Product to such Product and such


43





Alternative Product achieves Market Penetration of at least [***] in such country, then the royalties due in respect of the Net Sales of such Product in such country shall thereafter be reduced to [***] of the amount that would otherwise have been due in respect of such Net Sales.

(c)
The Licensee may deduct from any royalty payments to BioMedica under Clause 8.4(a) (Royalties) in respect of a given Fiscal Quarter [***] of any licensee fees, milestone payments, and royalties paid by the Licensee to a Third Party during such Fiscal Quarter or the immediately preceding Fiscal Quarter in consideration for a license under such Third Party’s Patents or Know- How that are necessary for (i) sale of an Original Product or (ii) Manufacture of an Original Product using the unmodified and unenhanced manufacturing process transferred by BioMedica pursuant to Clause 7.3 (Technology Transfer).

(d)
In no circumstances will the royalties payable to BioMedica pursuant to this Clause 8.4 (Royalties) in respect of any Fiscal Quarter be reduced, as a result of Clauses 8.4(b) (Royalties) or 8.4(c) (Royalties), below [***] of the royalties otherwise payable under this Clause 8.4 (Royalties).

8.5
Net Receipts

The Licensee shall promptly (no later than [***] thereafter) notify BioMedica in writing upon receipt of any Net Receipts. After receipt of such notification from the Licensee, BioMedica shall issue to the Licensee an invoice for an amount equal to [***] of the Net Receipts. The Licensee shall pay such invoice within [***] after receipt of such invoice.

8.6
Paediatric Priority Review Voucher

In the event that (i) pursuant to Clause 2.1(c) (Grant of License; Right of Reference), the term “Product” is amended to have the meaning of “Backup Product”; and (ii) the Licensee is issued a Paediatric Priority Review Voucher; and (iii) the Licensee or its Affiliates transfers such Paediatric Priority Review Voucher to any Third Party, then the Licensee shall promptly (no later than [***] thereafter) notify BioMedica in writing upon receipt of any Paediatric Priority Review Voucher Receipts. After receipt of such notification from the Licensee, BioMedica shall issue to the Licensee an invoice for an amount equal to [***] of the Paediatric Priority Review Voucher Receipts. The Licensee shall pay such invoices within [***] after receipt of such invoice.

8.7
Third Party Payments

BioMedica shall be solely responsible for all payments, if any, due with respect to the Products pursuant to any agreement BioMedica has in place with a Third Party as of the Effective Date.


44





9.
INVOICES, PAYMENTS AND REPORTS

9.1
Invoices and Estimates

(a)
Within [***] after the end of each month during the Term, BioMedica shall provide the Licensee with a good faith estimate of the amount for which it anticipates that it will invoice the Licensee for any amounts for which BioMedica is required to invoice the Licensee under this Agreement in such month.

(b)
BioMedica shall include a reasonably detailed description of the work performed or other basis for any amount for which it issues an invoice to the Licensee under this Agreement.

9.2
Milestone Payments

The Licensee shall make the payments referred to in Clause 8.2 (Development and Regulatory Milestone Payments), Clause 8.3 (Commercial Milestone Payments), and Clause 8.5 (Net Receipts), and Clause 8.6 (Paediatric Priority Review Voucher) within [***] after receipt of an invoice from BioMedica, provided that each milestone payment shall be due only once.

9.3
Royalty Reports

(a)
Not later than (i) [***] following the end of each Fiscal Quarter (excluding the last Fiscal Quarter of a Fiscal Year and the Fiscal Quarter in which the expiry or termination of this Agreement takes effect) or (ii) [***] after the end of the last Fiscal Quarter of a Fiscal Year and the Fiscal Quarter in which the expiry or termination of this Agreement takes effect, the Licensee shall submit to BioMedica a written report (“Royalty Report”) detailing:

(i)
on a country-by-country basis:

(A)
the quantity of Product sold by each Selling Entity; and

(B)
the Net Sales of Products during such Fiscal Quarter by each Selling Entity in GBP and the currency in which the Net Sales were recorded showing the conversion rates used; and

(ii)
the Net Receipts received or obtained in such Fiscal Quarter and the Sublicensee to which they relate.

(b)
Following receipt of the Royalty Report, BioMedica shall issue an invoice to the Licensee for the royalties due under this Agreement for the reported Fiscal Quarter. The Licensee shall pay such invoice within [***] after receipt of such invoice; provided, that the Licensee shall pay such invoice within [***] after receipt of any invoice issued based on the Royalty Report for the last Fiscal Quarter of a Fiscal Year.

9.4
Payment

(a)
All sums due to BioMedica under this Agreement:


45





(i)
are exclusive of Value Added Tax, which where applicable will be paid by the Licensee in addition. BioMedica shall provide to the Licensee all customary receipts for payment of such taxes and cooperate with the Licensee in making applications for and securing any available exemptions or reductions of VAT reasonably available; notwithstanding the foregoing, the Parties acknowledge and agree that (1) on the basis of Applicable Law in force as at the date of this Agreement and subject to any changes in relevant circumstances of the Licensee or any Affiliate during the term of this Agreement, there is no current expectation that BioMedica will be required to charge Value Added Tax with respect to the transactions under this Agreement; and (2) each Party will not take any position or otherwise take any action that is inconsistent with the position that, for United Kingdom Tax purposes, Value Added Tax is inapplicable to the transactions under this Agreement, unless there is a relevant change in Applicable Law or relevant change in circumstances of the Licensee or any Affiliate, or unless otherwise required by a final determination of a court of competent jurisdiction;


(ii)
shall be paid in GBP, in cash by transferring an amount in aggregate to the following account:

Account name: [***]
Account number:    [***]
Sort code:    [***]
IBAN:    [***]
SWIFT:    [***]

(b)
When conversion of payments from any foreign currency is required, the Licensee shall convert such amounts into GBP using the average exchange rate applicable at the invoice date using the OANDA forex currency converter, or other reputable currency converter agreed between the Parties from time to time.

(c)
If laws or regulations require withholding by the Licensee of any Taxes imposed upon BioMedica on account of any royalties and payments paid under this Agreement, such Taxes shall be deducted by the Licensee as required by law from such remittable royalty and payment and shall be paid by the Licensee to the proper tax authorities. To the extent that amounts are so withheld and paid to the proper taxing authority, such amounts shall be treated for all purposes of this Agreement as having been paid to BioMedica. Official receipts of payment of any withholding Tax shall be secured and sent to BioMedica as evidence of such payment. The parties shall cooperate to ensure that any withholding Taxes imposed are reduced as far as possible under Applicable Law (including any relevant Tax treaty), which shall include providing assistance with the completion of any required forms. In addition, the Parties shall cooperate in accordance with Applicable Law to minimize indirect Taxes (such as Value Added Tax, transfer Tax, sales Tax, consumption Tax and other similar Taxes) in connection with this Agreement, provided that where any such Taxes are Taxes that are or may be imposed outside the United Kingdom, the Licensee shall indemnify BioMedica to its reasonable satisfaction against any material and reasonable out of pocket costs and expenses in taking any action in cooperation requested by the Licensee.


46





(d)
If any undisputed payment due is not paid by the due date, BioMedica may charge interest on any outstanding amount of such payment on a daily basis at the lower of (i) the maximum rate permitted under Applicable Law and (ii) a rate equivalent to [***] per annum above the base rate of the Bank of England then in force in London.

(e)
Notwithstanding anything else in this Section 9.4, each Party shall be solely responsible for the payment of all Taxes imposed on its share of income (however denominated) or gain arising directly or indirectly from the activities of the Parties under this Agreement, except to the extent any such Taxes are deducted or withheld and taken into account pursuant to the definition of “Net Receipts”.

10.
RECORDS AND AUDIT

10.1
Records

Each Party and its Affiliates shall keep, at their normal place of business, accurate and up-to-date records and books of account in sufficient detail for the determination of amounts payable by the Licensee under this Agreement. Such books and records shall be retained by each Party and its Affiliates for three (3) years following the Fiscal Year to which they pertain, in the case of the Licensee, or the Calendar Year to which they pertain, in the case of BioMedica, and shall be maintained in accordance with applicable Accounting Standards.

10.2
Audit

(a)
At the request and expense (except as provided below) of a Party wishing to conduct an audit (the “Auditing Party”) of the other Party’s records and books (the “Other Party”), the Other Party and its Affiliates shall permit an independent, certified public accountant appointed by the Auditing Party and reasonably acceptable to the Other Party, during normal business hours and upon reasonable prior written notice to the Other Party, once per Fiscal Year, to examine (i) in the case of an audit by BioMedica of the Licensee or its Affiliates, those records and all other material documents of the Licensee or its Affiliates relating to or relevant to calculation of the amounts due to BioMedica hereunder; and (ii) in the case of an audit by the Licensee of BioMedica or its Affiliates, those timesheets and records of pass-through costs in relation to the Process Development Services, transfer of the Licensed Know-How, Ongoing Clinical Trials, and technology transfer; in each case in respect of any period within three (3) years following the Fiscal Year (in the case of the Licensee) or Calendar Year (in the case of BioMedica) to which such records or documents pertain. The results of any such examination shall be made available to both Parties. In the event of an audit of BioMedica, the amounts incurred by BioMedica or its Affiliates shall be reconciled if a disparity is determined. If, as a result of any such examination of the Licensee or its Affiliates, it is the opinion of the accountant that the amounts paid to BioMedica were more or less than the amount which should have been paid, then the Licensee shall make all payments required to eliminate any such discrepancy within [***] after BioMedica’s written demand therefor, and BioMedica shall credit any excess amounts paid against future payments due from the Licensee (and if no further payments are due, shall be refunded by BioMedica at the request of the


47





Licensee). In addition, (A) if such underpaid amount is in excess of [***] of the amount that actually should have been paid by the Licensee under this Agreement for any applicable Fiscal Year, then the Licensee shall reimburse BioMedica for the reasonable cost of such audit; or (B) if such overpaid amount is in excess of [***] of the amount that actually should have been paid by the Licensee under this Agreement for any applicable Fiscal Year, then BioMedica shall reimburse the Licensee for the reasonable cost of such audit.

(b)
At the request and expense (except as provided below) of BioMedica, the Licensee shall perform an audit, through an independent, certified public accountant appointed by BioMedica and reasonably acceptable to the Licensee, of those records and all other material documents of any Sublicensee relating to or relevant to calculation of the amounts due to BioMedica hereunder in respect of any period within three (3) years after the date to which such records or documents relate. The results of any such examination shall be made available to both Parties. If, as a result of any such examination, it is the opinion of the accountant that the amounts paid to BioMedica were (i) less than the amount which should have been paid, then the Licensee shall make all payments required to eliminate any such discrepancy within [***] after BioMedica’s demand therefor, or (ii) more than the amount which should have been paid, then such additional amounts shall be credited against future payments until such discrepancy is eliminated. In addition, if such underpaid amount is in excess of [***] of the amount that actually should have been paid by the Licensee, then the Licensee shall reimburse BioMedica for the reasonable cost of such audit.

11.
INTELLECTUAL PROPERTY

11.1
Ownership

(a)
In respect of any Inventions, inventorship as between the Parties will be determined in accordance with the rules of inventorship of the jurisdiction in which such Inventions were invented. Each Party solely owns any Inventions made solely by its own employees, agents, or independent contractors, together with all intellectual property rights therein. The Parties jointly own any Inventions that are made jointly by employees, agents, or independent contractors of each Party, together with all intellectual property rights therein (“Joint Inventions”). All Patents claiming Joint Inventions will be referred to as “Joint Patents”.

(b)
Each Party may practice and exploit the Joint Inventions and Joint Patents without the duty of accounting or seeking consent from the other Party and, subject to the terms of this Agreement, including Clause 2.5 (Non-Compete), each Party hereby grants to the other a non-exclusive, worldwide, fully paid up, perpetual, irrevocable, sublicensable (through multiple tiers), transferable licence under such Joint Inventions and Joint Patents. Each Party shall use reasonable efforts to promptly disclose to the other Party all Joint Inventions, including any invention disclosures, or other similar documents, submitted to it by its employees, agents or independent contractors describing inventions that are Joint Inventions, and all information relating to such Joint Inventions to the extent necessary or useful for the preparation, filing and maintenance of any Patent with respect to such Joint Invention.


48





(c)
For clarity, Process Development Results are excluded from the definition of Inventions and the provisions of this Clause 11 (Ownership), and ownership of such Process Development Results are addressed in Clause 5.4 (Process Development Results).

(d)
To the extent applicable, each Party shall cause all employees, independent contractors, consultants and others who perform activities for such Party under this Agreement to be under an obligation to assign (or, if such Party is unable to cause such person or entity to agree to such assignment obligation despite such Party using reasonable efforts to negotiate such assignment obligation, provide a license under) their rights in and to any Inventions and all intellectual property rights therein to such Party, except where Applicable Law requires otherwise and except in the case of governmental, not-for-profit and public institutions which have standard policies against such an assignment (in which case a Party shall obtain a suitable license, or right to obtain such a license).

(e)
Each Party shall cooperate fully with the other Party in the preparation, filing, prosecution and maintenance of Licensed Patents and Joint Patents under Clause 11.2 (Filing, prosecution and maintenance of Joint Patents) and Clause 11.3 (Filing, prosecution and maintenance of Licensed Patents), respectively. Such cooperation includes (i) executing all papers and instruments, or requiring its employees or contractors, to execute such papers and instruments, so as enable the other Party to apply for and to prosecute patent applications in any country as permitted by Clause 11.2 (Filing, prosecution and maintenance of Joint Patents) and Clause 11.3 (Filing, prosecution and maintenance of Licensed Patents), and (ii) promptly informing the other Party of any matters coming to such Party’s attention that may affect the preparation, filing, prosecution or maintenance of any such patent applications.

11.2
Filing, prosecution and maintenance of Joint Patents

(a)
Subject to Clause 11.2(b) (Filing, prosecution and maintenance of Joint Patents), as between the Parties, the Licensee has the first right, but not the obligation, to prepare, file, prosecute, maintain and defend the Joint Patents at the Licensee’s cost and expense. The Licensee shall consult with BioMedica with respect to the filing and prosecution of the Joint Patents. The Licensee shall provide BioMedica with drafts of any proposed filings to allow BioMedica to review and comment before such filings are due. BioMedica may provide comments on such of those documents to be submitted to any patent office, within [***] after receiving the same from the Licensee. The Licensee shall keep BioMedica reasonably informed on the prosecution status of all Joint Patents.

(b)
If the Licensee wishes to discontinue the prosecution or maintenance of any Joint Patents, BioMedica shall have the right but not the obligation to assume responsibility for the prosecution and maintenance of any Joint Patents at BioMedica’s sole expense.

11.3
Filing, prosecution and maintenance of Licensed Patents

(a)
Subject to Clause 11.3(b) (Filing, prosecution and maintenance of Licensed Patents), BioMedica may, in its discretion, prepare, file, prosecute, maintain and defend the


49





Licensed Patents at BioMedica’s cost. The Licensee shall not take any such actions in connection with any Licensed Patents without the prior written consent of BioMedica. BioMedica shall consult with the Licensee with respect to the filing and prosecution of the Product-Specific Patents. The Product-Specific Patents as of the Effective Date are set forth in Part 2 of Schedule 1 , which BioMedica shall update from time to time, but at least annually on the anniversary of the Effective Date. BioMedica shall provide the Licensee with drafts of any proposed filings with respect to the Product-Specific Patents to allow the Licensee to review and comment before such filings are due. The Licensee may provide comments on such of those documents to be submitted to any patent office, within [***] after receiving the same from BioMedica. BioMedica shall update the Licensee and keep the Licensee reasonably informed on the prosecution status of all Product-Specific Patents.

(b)
If BioMedica wishes to discontinue the prosecution or maintenance of any Product-Specific Patents, the Licensee shall have the right but not the obligation to assume responsibility for the prosecution and maintenance of any such Product-Specific Patents at the Licensee’s sole expense.

(c)
BioMedica and the Licensee shall discuss in good faith whether and on which Licensed Patents to seek patent term extension or supplemental patent protection, including supplemental protection certificates. BioMedica and the Licensee shall cooperate in good faith in connection with all such activities. Notwithstanding the foregoing, if the Parties cannot agree, BioMedica will determine whether and on which Licensed Patents BioMedica will seek patent term extensions or supplemental patent protection in relation to the relevant Product and the Licensee shall not make any such applications.

11.4
Enforcement of Joint Patents

(a)
If either Party becomes aware of any actual or suspected infringement in the Field of any Joint Patent, it will notify the other Party in writing to that effect and include evidence of the alleged infringement by such Third Party.

(b)
The Licensee shall have the first right, but not the obligation, to initiate an action against a Third Party infringer of any Joint Patent at its cost and expense. Should the Licensee bring an action against a Third Party infringer of a Joint Patent,
(i)      the Licensee shall keep BioMedica reasonably informed regarding the progress of any litigation and settlement discussion with any alleged infringer,
(ii)      the Licensee shall copy BioMedica on all documents and correspondence other than routine procedural documents and correspondences, (iii) BioMedica shall have the right to provide ongoing comments on documents prior to submission and advice regarding its position and interests in such action, which advice and comments will be considered in good faith by the Licensee, and (iv) the Licensee shall not enter into any settlement, consent judgment or other voluntary disposition of any such action without the consent of BioMedica, such consent not to be unreasonably withheld or delayed. If the Licensee elects to pursue such action, BioMedica may be represented in such action by attorneys of its own choice at its own expense. Upon the Licensee’s request, BioMedica shall timely commence or join in any such litigation to establish standing and cooperate with the Licensee in such litigation; provided that, if BioMedica is so involuntarily joined in any such proceedings, the Licensee shall be responsible for costs and expenses reasonably


50





incurred by BioMedica in connection with such proceedings.

(c)
If the Licensee elects, by notice to BioMedica, not to initiate an action against a Third Party infringer of any Joint Patent or to discontinue such an action, BioMedica shall have the right to initiate (or continue) such an action, at BioMedica’s expense. The Licensee shall have the right to consult with BioMedica with respect to such action and to participate and be represented by independent counsel in any such action initiated by BioMedica, at the Licensee’s expense. BioMedica shall not settle any such action if such settlement would adversely affect or limit the licences granted to the Licensee hereunder without the prior written consent of the Licensee, such consent not to be unreasonably withheld, conditioned, or delayed.

11.5
Enforcement of Licensed Patents

(a)
If either Party becomes aware of any actual or suspected infringement in the Field, of any Product-Specific Patents, it will notify the other Party in writing to that effect and include evidence of the alleged infringement by such Third Party.

(b)
BioMedica shall have the first right, but not the obligation, to initiate an action against a Third Party infringer of Licensed Patents. Should BioMedica bring an action against a Third Party infringer of any Product-Specific Patents, (i) the Licensee shall have the right to consult with BioMedica and to participate and be represented by independent counsel in any action initiated by BioMedica at the Licensee’s expense, (ii) BioMedica shall keep the Licensee reasonably informed regarding the progress of any litigation and settlement discussion with any alleged infringer, (iii) BioMedica shall copy the Licensee on all documents and correspondence other than routine procedural documents and correspondences, and (iv) the Licensee shall have the right to provide ongoing comments on documents prior to submission and advice regarding its position and interests in such action, which timely advice and comments will be considered in good faith by BioMedica. BioMedica shall retain the sole control of any infringement action filed against any Third Party infringer pursuant to this Clause 11.5(b) (Enforcement of Licensed Patents) and shall bear all costs and expenses in connection therewith. BioMedica shall not settle any such action if such settlement would adversely affect or limit the licences granted to the Licensee hereunder without the prior written consent of the Licensee, such consent not to be unreasonably withheld or delayed.

(c)
If BioMedica elects, by notice to the Licensee, not to initiate an action against a Third Party infringer of any Product-Specific Patents or to discontinue such an action, but not in any other circumstances, the Licensee shall have the right to initiate (or continue) such an action (if required by Applicable Law, in the name of and on behalf of, BioMedica), at the Licensee’s expense. Should the Licensee bring an action against a Third Party infringer of a Product-Specific Patent, (i) the Licensee shall keep BioMedica reasonably informed regarding the progress of any litigation and settlement discussion with any alleged infringer, (ii) the Licensee shall copy BioMedica on all documents and correspondence other than routine procedural documents and correspondences, (iii) BioMedica shall have the right to provide ongoing comments on documents prior to submission and advice regarding its position and interests in such action, which timely


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advice and comments will be considered in good faith by the Licensee, and (iv) the Licensee shall not enter into any settlement, consent judgment or other voluntary disposition of any such action without the consent of BioMedica, such consent not to be unreasonably withheld or delayed. If the Licensee elects to pursue such action, BioMedica may be represented in such action by attorneys of its own choice at its own expense. Upon the Licensee’s request, BioMedica shall timely join in any such litigation to establish standing and cooperate with the Licensee in such litigation; provided that, if BioMedica is so involuntarily joined in any such proceedings, the Licensee shall be responsible for costs and expenses reasonably incurred by BioMedica in connection with such proceedings.

11.6
Enforcement Generally

(a)
Any recoveries obtained by either Party as a result of any proceeding against a Third Party infringer under Clause 11.4 (Enforcement of Joint Patents) or Clause 11.5 (Enforcement of Licensed Patents) shall first be used to reimburse each Party for all litigation costs paid by that Party in connection with such litigation on a pro rata basis and second, any remainder after the reimbursement of litigation costs shall be split between the Parties in proportion to the relative contribution of each Party to the total litigation costs.

(b)
Either Party shall, at the request and expense of the enforcing Party in respect of the Licensed Patents or Joint Patents, as applicable, cooperate to the fullest extent reasonably possible and will lend its name to such actions if required by law in order for the enforcing Party to bring such action. This clause will not be construed to require either Party to undertake any activities, including legal discovery, at the request of any Third Party, except as may be required by lawful process of a court of competent jurisdiction.

11.7
Third Party Infringement Suit

Each Party shall notify the other Party promptly in writing of any claim of, or action for, infringement of any Patents owned or controlled by a Third Party which is threatened, made or brought against either Party by reason of either Party’s performance of its obligations under this Agreement. At the request of either Party in receipt of such claim or action, the Parties shall consider whether to enter into a common interest agreement wherein the Parties agree to their shared, mutual interest in the outcome of such potential dispute, and if the Parties agree to do so, the Parties shall promptly meet to consider the claim or assertion and the appropriate course of action. Each Party may represent itself in any litigation to which it is a party, at its own expense, unless otherwise agreed upon by the Parties or as otherwise set forth in this Agreement.

12.
CONFIDENTIALITY

12.1
Duty of Confidence

Notwithstanding Clause 12.2 (Representatives), each Receiving Party shall:

(a)
keep the Confidential Information of the Disclosing Party secret and confidential at all times;


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(b)
not disclose or permit the disclosure of any Confidential Information of the Disclosing Party, in whole, in part, or in summary, to any person, except as expressly permitted by this Agreement or any other agreement between the Parties;

(c)
not use the Confidential Information of the Disclosing Party or permit it to be used, in whole or in part, for any purpose other than performance of the obligations and enjoyment of the rights granted under this Agreement or any other agreement between the Parties; and

(d)
inform the Disclosing Party immediately if it becomes aware of the possession, use or knowledge of any of the Confidential Information of the Disclosing Party by an unauthorised person, and to provide any assistance in relation to such unauthorised possession, use or knowledge that the Disclosing Party may require.

12.2
Representatives

The Receiving Party may permit access to the Confidential Information of the Disclosing Party only to those of its Affiliates, directors, officers, employees, consultants and permitted sublicensees (or Sublicensees, in the case of the Licensee) who:

(a)
reasonably require such access for the performance of the obligations or enjoyment of the rights granted under this Agreement;

(b)
have been informed of the confidential nature of such Confidential Information, the Disclosing Party’s interest in such Confidential Information, and the provisions of this Clause 12 (Confidentiality) ; and

(c)
have entered into legally binding confidentiality obligations to the Receiving Party on terms that are no less onerous than those set out in this Agreement, and which extend to such Confidential Information.

The Receiving Party shall ensure that all those who have access to the Confidential Information of the Disclosing Party by or on behalf of the Receiving Party comply with the provisions of this Clause 12 (Confidentiality). Notwithstanding any other provision of this Agreement, the Receiving Party shall be liable to the Disclosing Party for any acts or omissions of any such person, that would, if effected by the Receiving Party, constitute a breach of this Clause 12 (Confidentiality).

12.3
Exceptions

The Receiving Party’s obligations under Clause 12.1 (Duty of Confidence) shall not apply to any Confidential Information of the Disclosing Party that the Receiving Party can prove by means of reasonable written evidence:

(a)
was known to the Receiving Party prior to disclosure by the Disclosing Party;

(b)
is or becomes publicly known other than as a result of breach of this Agreement by the Receiving Party or by anyone to whom the Receiving Party disclosed the Confidential Information of the Disclosing Party;


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(c)
is received by the Receiving Party from a Third Party lawfully entitled to make the disclosure without restrictions on such Third Party’s rights to disclose or use; or

(d)
is developed by or on behalf of the Receiving Party without any direct or indirect access to, or use of, the Confidential Information of the Disclosing Party;

except that the above exceptions do not extend to circumstances where the Confidential Information is specific, does not fall within the above exceptions, and is embraced by more general information which does fall within the above exceptions.

12.4
Required Disclosures

The Receiving Party will not be in breach of its obligations under this Agreement to the extent that it is required to disclose Confidential Information of the Disclosing Party by Applicable Law, including the rules of any recognized stock exchange, provided that, before making such a disclosure, the Receiving Party shall, to the extent it is legally permitted to do so:

(a)
give reasonable advance notice to the Disclosing Party of the proposed disclosure

(b)
use efforts to secure confidential treatment of such Confidential Information at least as diligent as such Party would use to protect its own Confidential Information, but in no event less than reasonable efforts;

(c)
take all reasonable action to avoid disclosure of Confidential Information hereunder; and

(d)
take into account reasonable requests of the Disclosing Party in relation to such disclosure.

Any information disclosed pursuant to this Clause 12.4 (Required Disclosures) that receives confidential treatment from the Third Party to whom it was disclosed (e.g., portions redacted from a material agreement filed with a securities exchange governmental authority) shall remain Confidential Information and subject to the restrictions set forth in this Agreement, including the foregoing provisions of this Clause12.4 (Required Disclosures).

12.5
Additional Disclosures

In addition to disclosures allowed under Clause 12.4 (Required Disclosures):

(a)
The Licensee may disclose Confidential Information of BioMedica to the extent such disclosure is necessary in connection with sublicensing (or potential sublicensing) the rights granted under this Agreement, provided that, such disclosure is made only under obligations of confidence and non-use at least as stringent as set out in this Agreement and any such sub-licensing is in accordance with this Agreement.

(b)
BioMedica may disclose, to any licensor or assignor of intellectual property rights to BioMedica, financial Confidential Information of the Licensee provided to


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BioMedica under this Agreement to the extent required and for the specific purpose of enabling BioMedica to comply with its contractual royalty reporting obligations to any such licensor or assignor of intellectual property rights to BioMedica; provided that, any such disclosure is made only under obligations of confidence and non-use at least as stringent as set out in this Agreement.

(c)
Each Party may disclose Confidential Information of the other Party in connection with the filing, prosecuting, or maintaining of Patents as permitted by this Agreement and for Regulatory Filings for a Product, provide that the Licensee shall use efforts to secure confidential treatment of Licensed Know- how in Regulatory Filings at least as diligent as the Licensee would use to protect its own Confidential Information, but in no event less than reasonable efforts.

(d)
Each Party and its Affiliates may disclose the existence of this Agreement and the material terms hereof to potential and actual investors, acquirers, licensees, sublicensees, and other financial and commercial partners of such Party or its Affiliates in connection with:

(i)
the raising of finance,

(ii)
evaluating or carrying out a license or collaboration,

(iii)
the sale of any equity interest in such Party or its Affiliates, or

(iv)
the sale of the business or relevant part of the business of the Party or its Affiliates;

in each case under written obligations of confidentiality and non-use at least as stringent as those herein (and with respect to potential and actual investors and financial partners only, except for the period of confidentiality, which shall be consistent with industry standards).

12.6
Return and Destruction of Confidential Information

At the Disclosing Party’s written request on expiration or termination of this Agreement, the Receiving Party shall:

(a)
promptly destroy or erase all Confidential Information of the Disclosing Party that the Receiving Party has received under this Agreement including any copies made and permanently delete all electronic copies of any such Confidential Information from the Receiving Party’s computer systems; and

(b)
make no further use of any such Confidential Information.

The Receiving Party may, however, keep one copy of the Confidential Information of the Disclosing Party in its legal files solely for the purpose of enabling it to comply with the provisions of this Agreement, and the Receiving Party shall not be required to remove such Confidential Information of the Disclosing Party from its back-up or archive electronic records including its electronic laboratory notebook and laboratory information management systems.


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12.7
Press Releases and Publicity

Neither Party shall make, nor permit any of its representatives to make, any public announcement, whether oral or written, concerning this Agreement or make any use of the name, symbol, trade mark, trade name or logo of the other Party or its Affiliates without the prior written consent of the other Party (such consent not to be unreasonably withheld, conditioned, or delayed); provided, however, that (a) notwithstanding any other provision of this Agreement each Party shall be permitted to make an announcement in the agreed form set out in Schedule 8 and otherwise repeat the information contained therein (or contained in any previous announcement consented to by the other Party), and (b) each Party shall be permitted to make required disclosures pursuant to Section 12.4 (Required Disclosures), and in each case such activities shall not constitute a breach of this Agreement.

12.8
Publications

The Licensee may freely publish and give presentations on its development and commercialization of the Products subject to the remainder of this Clause 12.8 (Publications). BioMedica may only publish or give presentations with respect to any Product to the extent identified in Part 4 of Schedule 1 or with the Licensee’s prior written consent, to be given in the Licensee’s sole discretion, provided that this Clause 12.8 (Publications) shall not prevent BioMedica from publishing or giving presentations in respect of information which has already been published or otherwise disclosed publicly. In the case of publication by the Licensee, or an approved publication by BioMedica, the following procedures shall apply: The non-publishing party may review and comment on any material proposed for disclosure or publication by the publishing party with respect to a Product, whether by oral presentation, manuscript, or abstract. The publishing party shall deliver a complete copy of the material to the non-publishing party at least [***] prior to submitting such material to a publisher or initiating any other disclosure. The non-publishing party shall review any such material and give its comments to the publishing party within [***] of the receipt of such material. The publishing party shall comply with the non-publishing party’s request to delete references to its Confidential Information in any such material and shall delay any submission for publication or other public disclosure for a period of up to an additional [***] for the purpose of preparing and filing appropriate patent applications.

12.9
Prior Confidentiality Agreement.

As of the Effective Date, the terms of this Clause 12 (Confidentiality) shall supersede any prior non-disclosure, secrecy or confidentiality agreement between the Parties (or their Affiliates) relating to the subject of this Agreement, including the CDA. Any information disclosed pursuant to any such prior agreement shall be deemed Confidential Information for purposes of this Agreement.


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13.
REPRESENTATIONS AND WARRANTIES

13.1
Mutual Warranties

Each Party warrants to the other as of the Effective Date that:

(a)
it is duly organised, validly existing, and in good standing under the laws of its jurisdiction of formation;

(b)
it has full corporate power and authority to execute, deliver, and perform this Agreement, and has taken all corporate action required by law and its organisational documents to authorise the execution and delivery of this Agreement and the consummation of the transactions contemplated by this Agreement;

(c)
all consents, approvals and authorisations from all governmental authorities or other Third Parties required to be obtained by such Party in connection with this Agreement have been obtained; and

(d)
the execution and delivery of this Agreement and all other instruments and documents required to be executed pursuant to this Agreement do not and shall not (i) conflict with or result in a breach of any provision of its organisational documents, (ii) result in a breach of any agreement to which it is a party; or (iii) violate any Applicable Law.

13.2
BioMedica Warranties

BioMedica warrants to the Licensee as of the Effective Date that:

(a)
except as identified in Schedule 1 , there are no claims, demands, challenges, oppositions, nullity actions, interferences, inter-partes reexaminations, inter- partes reviews, post-grant reviews, derivation proceedings, litigation, arbitration or other proceedings asserted in writing against BioMedica or its Affiliates (and BioMedica has no knowledge of any claim or any of the foregoing) in respect of (i) the BioMedica IP (other than matters raised in the ordinary course of patent prosecution), including any claim or any of the foregoing alleging that such BioMedica IP is invalid or unenforceable or violates, infringes, constitutes misappropriation or otherwise conflicts or interferes with, or would violate, infringe or otherwise conflict or interfere with, any intellectual property or proprietary right of any Third Party; or (ii) Prosavin or OXB-102;

(b)
as far as it is aware, BioMedica has filed and prosecuted the Licensed Patents in good faith and complied with all duties of disclosure with respect thereto;

(c)
as far as it is aware, BioMedica has not committed any act, or omitted to commit any act, that may cause the Licensed Patents to expire prematurely or be declared invalid or unenforceable;

(d)
as far as it is aware, all application, registration, maintenance and renewal fees in respect of the Licensed Patents as of the date of execution of this Agreement have been paid and all necessary documents and certificates have been filed with the relevant agencies for the purpose of maintaining such patents;


57





(e)
BioMedica has the right to grant the licences and rights granted under this Agreement;

(f)
BioMedica has not granted any Third Party rights that would otherwise interfere or be inconsistent with the rights granted hereunder;

(g)
as of the Effective Date, the Patents listed in Part 1 and Part 2 of Schedule 1 are all the Patents that BioMedica Controls that Cover the Products;

(h)
as far as it is aware, no Third Party is infringing or threatening to infringe (i) any Product-Specific Patents, or (ii) any other Licensed Patents in relation to Product;

(i)
as far as it is aware, BioMedica has independently developed all Know-How within the BioMedica IP or otherwise has a valid right to use and to grant the licenses and rights hereunder in and to such Know-How;

(j)
BioMedica has no outstanding submissions for publication related to the Products or the Ongoing Clinical Trials except as described in Schedule 1 ; and

(k)
that certain Nonexclusive Agreement by and between The Board of Trustees of the Leland Stanford Junior University Stanford Agreement and Oxford BioMedica (UK) Limited dated as of June 24, 2003 (the “Stanford Agreement”) is in full force and effect, no notice has been delivered of any breach under the Stanford Agreement and no rights granted herein are inconsistent with the Stanford Agreement. As of the Effective Date, BioMedica is in the process of amending and restating the Stanford Agreement, and BioMedica has provided to the Licensee a copy of the Stanford Agreement and current drafts of the amendment of the Stanford Agreement. BioMedica acknowledges that it shall be liable for obtaining any amendment to the Stanford Agreement that may be necessary to enable BioMedica to carry out its obligations under this Agreement, but it is acknowledged and agreed by the Parties that BioMedica is not permitted to sublicense its rights under the Stanford Agreement and that such liability shall not extend to obtaining an amendment to permit such a sublicense.

13.3
Licensee Warranties

The Licensee warrants to BioMedica as of the Effective Date that:

(a)
there is no action, suit, proceeding, inquiry or investigation brought by or before any governmental entity now pending or, to the knowledge of the Licensee, threatened, against or affecting the Licensee or any of its Affiliates, which would reasonably be expected, individually or in the aggregate, materially and adversely affect the performance by the Licensee of its obligations hereunder; and the aggregate of all pending legal or governmental proceedings to which the Licensee or any of its Affiliates is a party or of which any of their respective properties or assets is the subject, including ordinary routine litigation incidental to the business, if determined adversely to the Licensee, would not reasonably be expected to materially and adversely affect the performance by the Licensee of its obligations hereunder; and


58





(b)
each of the Licensee and its Affiliates is insured by recognized, financially sound and reputable institutions with policies in such amounts and with such deductibles and covering such risks as are generally deemed adequate and customary for their businesses, including directors’ and officers’ liability insurance and policies covering real and personal property owned or leased by the Licensee and its Affiliates against theft, damage, destruction and acts of vandalism and policies covering the Licensee and its Affiliates for product liability claims and clinical trial liability claims; and the Licensee has no reason to believe that it or its Affiliates will not be able (i) to renew its existing insurance coverage as and when such policies expire or (ii) to obtain comparable coverage from similar institutions as may be necessary or appropriate to conduct its business as now conducted and at a cost that would not reasonably be expected to materially and adversely affect the performance by the Licensee of its obligations hereunder; and neither the Licensee nor any of its Affiliates has been denied any insurance coverage which it has sought or for which it has applied.

13.4
No Other Warranties.

Each of the Parties acknowledges that, in entering into this Agreement, it does not do so in reliance on any representation, warranty, or other provision except as expressly provided in this Agreement.
EXCEPT AS EXPRESSLY SET FORTH IN THIS AGREEMENT, EACH PARTY EXPRESSLY DISCLAIMS ANY AND ALL WARRANTIES OF ANY KIND, EXPRESS OR IMPLIED, INCLUDING THE WARRANTIES OF DESIGN, MERCHANTABILITY, FITNESS FOR A PARTICULAR PURPOSE, NONINFRINGEMENT OF THE INTELLECTUAL PROPERTY RIGHTS OF THIRD PARTIES, OR ARISING FROM A COURSE OF DEALING, USAGE OR TRADE PRACTICES, IN ALL CASES WITH RESPECT THERETO. EACH OF THE PARTIES ACKNOWLEDGES THAT, IN ENTERING INTO THIS AGREEMENT, IT DOES NOT DO SO IN RELIANCE ON ANY REPRESENTATION, WARRANTY, OR OTHER PROVISION EXCEPT AS EXPRESSLY PROVIDED IN THIS AGREEMENT.

14.
LIABILITY, INDEMNIFICATION AND INSURANCE

14.1
Liability

(a)
Each Party shall be liable for any acts or omissions of its Affiliates or any Sublicensees that would, if effected by such Party, constitute a breach of this Agreement.

(b)
EXCEPT WITH RESPECT TO BREACHES OF CLAUSE 12 (CONFIDENTIALITY) AND TO THE PARTIES’ INDEMNIFICATION OBLIGATIONS IN CLAUSE 14.2 (LICENSEE INDEMNIFICATION) AND CLAUSE 14.3 (BIOMEDICA INDEMNIFICATION), IN NO EVENT SHALL EITHER PARTY BE LIABLE TO THE OTHER PARTY FOR SPECIAL, INDIRECT, INCIDENTAL, PUNITIVE OR CONSEQUENTIAL DAMAGES, OR FOR LOST PROFITS (BUT ONLY TO THE EXTENT THAT SUCH LOST PROFITS ARE SPECIAL, INDIRECT, INCIDENTAL, PUNITIVE OR CONSEQUENTIAL DAMAGES), UNDER ANY THEORY OR CLAIM WHETHER IN CONTRACT, TORT, NEGLIGENCE, STRICT LIABILITY OR OTHER


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LEGAL OR EQUITABLE THEORY OR CLAIM ARISING OUT OF OR RELATING TO THIS AGREEMENT.

(c)
Nothing in this Agreement shall exclude or limit, or purport to exclude or limit, a Party’s liability in the case of:

(i)
breach of Clause 12 (Confidentiality);

(ii)
fraud or fraudulent misrepresentation;

(iii)
death or personal injury resulting from its negligence;

(iv)
negligence or intentional misconduct; or

(v)
any other matter in respect of which it would be unlawful to exclude or restrict liability.

14.2
Licensee Indemnification

(a)
The Licensee shall indemnify BioMedica and its Affiliates, and their respective officers, directors, employees, contractors and agents (the “BioMedica Indemnitees”), from and against any and all losses, liabilities, damages, reasonable legal costs and other reasonable expenses of any nature whatsoever suffered or incurred in connection with any Third Party demands, claims, actions, or proceedings (each, a “Claim”) against a BioMedica Indemnitee arising out of (i) the research, development, use, manufacture, offer for sale, sale, import or export of any Product by or on behalf of the Licensee or its Affiliates or any Sublicensees (except to the extent that such Claim arises out of a breach by BioMedica of Clause 7.2(c) (Existing Inventory) or the supply by BioMedica of defective product in breach of any clinical or commercial supply agreement, with such concepts being defined and interpreted in accordance with such supply agreement) other than the Manufacture of Product by or on behalf of BioMedica; (ii) the negligence or wilful misconduct on the part of the Licensee Indemnitees; or (iii) implementation by BioMedica of any written instructions or directions of the Licensee in connection with the Ongoing Clinical Trials; (iv) the breach by the Licensee of any warranty or covenant made by the Licensee in this Agreement; except in each case except to the extent that such Claim arises out of the negligence of, wilful misconduct of, or breach of this Agreement by the BioMedica Indemnitees.

(b)
The Licensee shall indemnify the BioMedica Indemnitees for all costs incurred by the BioMedica Indemnitees in connection with provision, after the Effective Date, of clinical intervention or compensation to patients in the Ongoing Clinical Trials who may suffer injury or death or personal injury in connection with such Ongoing Clinical Trials after responsibility for such Ongoing Clinical Trials has been transferred to Licensee pursuant to Clause 4.1 (Transfer of Know-How and Ongoing Clinical Trials), except to the extent that such intervention or compensation is rendered necessary due to the negligence or wilful misconduct on the part of a BioMedica Indemnitee or failure by a BioMedica Indemnitee to perform the Ongoing Clinical trials in accordance with a specific written instruction of the Licensee.


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14.3
BioMedica Indemnification

(a)
BioMedica shall indemnify the Licensee and its Affiliates, and their respective officers, directors, employees, contractors, and agents (the “Licensee Indemnitees”), from and against any and all losses, liabilities, damages, reasonable legal costs and other reasonable expenses of any nature whatsoever suffered or incurred in connection with any Claim against a Licensee Indemnitee arising out of (i) the negligence or wilful misconduct on the part of the BioMedica Indemnitees; (ii) the development or use of the Products by BioMedica or its Affiliates or sublicensees prior to the Effective Date or (iii) the breach by BioMedica of any warranty or covenant made by BioMedica in this Agreement; except in each case to the extent that such Claim arises out of the negligence of, wilful misconduct of, or breach of this Agreement by the Licensee Indemnitees.

(b)
BioMedica shall indemnify the Licensee Indemnitees for all costs incurred by the Licensee Indemnitees in connection with provision of clinical intervention or compensation to patients who were treated with the Product prior to the Effective Date in any Ongoing Clinical Trial of the Product who may suffer injury or death or personal injury in connection with such Clinical Trials.

14.4
Indemnification Procedure

Where a Party (the “Indemnified Party”) seeks indemnification from the other Party (the “Indemnifying Party”) under this Clause 14.4 (Indemnification Procedure):

(a)
the Indemnified Party shall provide prompt written notice to the Indemnifying Party of the assertion or commencement of any Claims;

(b)
the Indemnifying Party shall have the right to assume (with its own counsel and at its own costs) sole control of the defence or settlement of the same and shall not be liable for any settlement made by the Indemnified Party without the Indemnifying Party’s prior written consent;

(c)
the Indemnified Party shall:

(i)
promptly provide all assistance and information reasonably required by the Indemnifying Party;

(ii)
not make any admission of liability, conclude any agreement or make any compromise or settlement with any person in relation to such Claim without the prior written consent of the Indemnifying Party; and

(iii)
have the right to participate in (but not control) the defence of the Claim and to retain its own counsel in connection with such Claim at its own expense.

14.5
Mitigation of Loss

Each Indemnified Party will take and will ensure that its Affiliates take all such reasonable steps and action as are necessary or as the Indemnifying Party may reasonably require in order to mitigate any losses, liabilities, damages, legal costs and other expenses (or potential losses,


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liabilities, damages, costs and expenses) in connection with indemnified Claims. Nothing in this Agreement shall or shall be deemed to relieve any Party of any common law or other duty to mitigate any losses incurred by it.

14.6
Insurance

Each Party shall obtain and maintain such types and amounts of insurance including self-insurance as is normal and customary for it to cover its indemnification obligations under this Agreement, and will upon request by the other Party, provide such other Party with a certificate of insurance in that regard, along with any amendments and revisions thereto.

15.
TERM AND TERMINATION

15.1
Term

This Agreement shall commence as of the Effective Date and, unless sooner terminated as provided hereunder, shall expire on a country-by-country basis upon the expiration of the Royalty Term with respect to such country (the “Term”). Upon expiration of this Agreement pursuant to this Clause 15.1 (Term), the licenses granted by BioMedica to the Licensee under Clause 2.1 (Grant of Licence; Right of Reference) shall become fully paid-up, royalty-free, and irrevocable.

15.2
Termination

(a)
The Licensee may terminate the Agreement at any time for convenience upon (i) two (2) months’ prior written notice prior to the First Commercial Sale of a Product; or (ii) [***] written notice to BioMedica after the First Commercial Sale of a Product.

(b)
Material Breach

(i)
Allegation and Cure. If either Party is in material breach of any obligation hereunder (including failure by the Licensee to make a payment due under this Agreement), the non-breaching Party may give written notice in good faith to the alleged breaching Party specifying the claimed particulars of such breach. If such alleged breach is not cured within [***] after such notice (or [***] with respect to any failure to make any payment due under this Agreement), the non- breaching Party shall have the right thereafter to terminate this Agreement pursuant to Clause 15.2(b)(iii) (Right to Terminate); provided, however, that if such breach is capable of being cured but cannot be cured within such [***] period, the breaching Party shall have the right to cure such breach during an additional period as is reasonable in the circumstances by initiating actions to cure such breach during such [***] period and diligently pursuing such actions.

(ii)
Disputes. If either Party disputes any aspect of this Clause 15.2(b) (Material Breach) (including, for example, (x) the alleged breaching Party disputes in good faith the existence or materiality of such breach; or (y) the non-breaching Party disputes in good faith the diligent pursuit of actions to cure such breach), then


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the Parties shall seek to resolve such disagreement pursuant to Clause 16.15 (Dispute Resolution; Arbitration).

(iii)
Right to Terminate.

(A)
Agreement of Material Breach. If the Parties agree in writing that there has been a material breach of an obligation and the relevant cure period in Clause 15.2(b)(i) (Allegation and Cure) has expired without cure, then the non-breaching Party shall have the right thereafter to terminate this Agreement immediately by giving written notice to the breaching Party.

(B)
Determination of Material Breach. Subject to sub-clauses (C), (D), and (E) below, if the tribunal under Clause 16.15 (Dispute Resolution; Arbitration) finally determines that there has been a material breach of any obligation under this Agreement and the relevant cure period in Clause 15.2(b)(i) (Allegation and Cure) has expired without cure, the non-breaching Party shall have the right thereafter to terminate this Agreement immediately by giving written notice to the breaching Party within [***] following such final determination of the tribunal; provided, for clarity, that the Licensee may cure any such breach prior to determination by the tribunal that there has been a material breach.

(C)
Cure of Material Breaches other than Diligence. For a material breach of any obligation under this Agreement other than Clause 6.5 (Diligence), the breaching Party shall have the right to cure such breach within [***] following such final determination of the tribunal and the non-breaching Party may not terminate if such breach is cured in such period.

(D)
Cure of Material Breach of Diligence Prior to First Commercial Sale. For a material breach of a diligence obligation under Clause 6.5 (Diligence) prior to First Commercial Sale of a Product in the United States or any country in Europe, Licensee shall have no right to cure such breach following such final determination of the tribunal.

(E)
Cure of Material Breach of Diligence After First Commercial Sale. For the first material breach of a diligence obligation under Clause 6.5 (Diligence) after First Commercial Sale of a Product in the United States or any country in Europe, the Licensee may cure such breach within a reasonable period of time (as is determined by the tribunal) following the final determination by the tribunal of such breach and Licensor may not terminate this Agreement for so long as Licensee is diligently pursuing actions to cure such breach. For any subsequent material breach of a diligence obligation under Clause 6.5 (Diligence) after First Commercial Sale of a Product in the United States or any country in Europe, the Licensee shall not have the right to cure such breach


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following the final determination by the tribunal of such a breach.

(c)
Either Party may terminate this Agreement upon notice to the other Party if an Insolvency Event occurs in relation to the other Party and such Insolvency Event is not dismissed or remedied within [***] after its occurrence. Unless otherwise prohibited by Applicable Laws, in any event when a Party first becomes aware of the likely occurrence of any Insolvency Event in regard to that Party, it shall promptly so notify the other Party in sufficient time to give the other Party sufficient notice to protect its interests under this Agreement.

15.3
Consequences of Termination

(a)
In the event of termination of this Agreement, such termination shall be deemed to be a termination of all outstanding Work Packages.

(b)
In the event of termination of this Agreement, the Licensee shall, as directed by BioMedica, wind-down any ongoing Clinical Trials with respect to the Product in an orderly fashion and in compliance with all Applicable Laws.

(c)
In the case of termination of this Agreement:

(i)
all rights and licences granted hereunder to the Licensee or its Affiliates shall terminate except that the Licensee and its Affiliates shall have the right to sell any Product in its or their possession or control (or the subject of a binding non-cancellable order with a Third Party manufacturer) for a period not to exceed [***] from the date of termination, subject to payment of any applicable royalties under this Agreement;

(ii)
the Licensee shall consent to the cancellation of any formal licence granted to it, or of any registration of it in any register, in relation to any BioMedica IP;

(iii)
subject to Clause 2.4(b)(iv) (Sublicensing), any sub-licences granted by the Licensee or its Affiliates pursuant to this Agreement shall terminate;

(iv)
to the extent the Licensee owns or holds any right, title or interest in any Product Marks under which any Product has been or is being marketed or sold, the Licensee shall assign such Product Marks to BioMedica (it being understood that the foregoing will not include any trademarks or internet domain names that contain the corporate or business names of the Licensee or its Affiliates);

(v)
the Licensee shall and shall procure that its Affiliates and Sublicensees shall, (1) promptly assign and transfer to BioMedica or its nominee, at no cost (except as set out below) to BioMedica all Regulatory Filings and Regulatory Approvals held by the Licensee or its Affiliates or Sublicensees that solely and specifically relate to the Product and shall provide BioMedica with all information, and execute all documents, reasonably necessary to transfer such Regulatory Filings and Regulatory Approvals to BioMedica, provided that BioMedica shall reimburse the Licensee, at the then current Licensee FTE rate, for time spent in


64





connection with such transfer to BioMedica, and (2) grant BioMedica a “Right of Reference” as that term is defined in 21 C.F.R. § 314.3(b) (or any other similar provision under Applicable Law outside the US) to all Regulatory Filings and Regulatory Approvals held by the Licensee or its Affiliates or Sublicensees that are necessary for BioMedica’s research, development, manufacture, use, sale, import and export of the Products but that do not solely and specifically relate to the Product;

(vi)
the Licensee shall and shall procure that its Affiliates and Sublicensees shall grant to BioMedica an exclusive licence under (1) all Patents that Cover the Product as then being developed or commercialised by the Licensee and (2) the Know-How that is necessary or reasonably useful for the research, development, manufacture, use, sale, import and export of the Products, in each case that are Controlled by the Licensee, its Affiliates, and Sublicensees as of the effective date of termination, solely to research, develop, manufacture, have manufactured, use, sell, have sold, import and export such Products; and

(vii)
the Licensee shall and shall procure that its Affiliates and Sublicensees shall, promptly transfer to BioMedica or its nominee a copy of all Know- How licensed under Clause 15.3(c)(vi) (Termination), provided that BioMedica shall reimburse the Licensee, at the then-current Licensee FTE rate, for time spent in connection with such transfer to BioMedica.

(d)
The following Clauses shall survive any expiration or termination of this Agreement for the period of time specified therein (or, if no such period is specified, indefinitely): Clause 1 (Definitions) to the extent defined terms are contained in surviving Clauses; Clause 8 (Monetary Obligations) with respect to those payments that accrued prior to the effective date of termination or expiration or pursuant to Clause 15.3(c)(i) (Consequences of Termination); Clause 9 (Payments and Reports); Clause 10.1 (Records) with respect to those records created pursuant to the Agreement; Clause 10.2 (Audit); Clause 11.1 (Ownership) with respect to Inventions, Joint Inventions and Joint Patents; Clause 11.2 (Filing, prosecution and maintenance of Joint Patents); Clause 12 (Confidentiality); Clause 13.4 (No Other Warranties); Clause 14 (Liability, Indemnification, and Insurance); Clause 15.3 (Consequences of Termination); Clause 15.4 (Termination Not Sole Remedy); Clause 16.5 (Notice and Other Communications); Clause 16.9 (Entire Agreement); Clause 16.11 (Waiver of Rights); Clause 16.12 (Unenforceable Provisions); Clause 16.14 (Governing Law); Clause 16.15 (Dispute Resolution; Jurisdiction);Clause16.16 (Equitable Relief); and Clause 16.17 (Cumulative Remedies).

15.4
Termination Not Sole Remedy

A Party’s right of termination under this Agreement, and the exercise of any such right, shall be without prejudice to any other right or remedy (including any right to claim damages) that such Party may have in the event of a breach of contract or other default by the other Party.


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16.
GENERAL

16.1
Term Loan Agreement.

(a)
The Parties agree and acknowledge:

(i)
that BioMedica obtained external financing pursuant to a Term Loan Agreement dated 29 June 2017 between BioMedica as borrower, Oxford BioMedica plc, as guarantor, Cortland Capital Market Services LLC, as administrative agent, OCM Strategic Credit Investments S.à.r.l., OCM Strategic Credit Investments 2 S.à.r.l., OCM Luxembourg SC Fund A S.à.r.l., and OCM Luxembourg SC Fund B S.à.r.l. as lenders (“Oaktree”) (the “Loan Agreement”);

(ii)
that the extension of credit pursuant to the Loan Agreement is secured by all or substantially all of BioMedica’s assets pursuant to a debenture governed by English law granted in favour of Cortland Capital Market Services LLC as agent for the lender (the “Lender Agent”), excluding the Product-Specific Patents, but which may include, inter alia, a charge over Oxford BioMedica’s right, title and interest in and to this Agreement (the “Charge”); and

(iii)
that notwithstanding the granting of the Charge, BioMedica shall remain liable under this Agreement to perform all of its obligations under this Agreement and shall remain entitled to exercise all its rights, powers and discretions under this Agreement unless and until the Licensee receives notice from Oaktree or the Lender Agent to the contrary informing the Licensee that an Event of Default (as defined in the Loan Agreement) has occurred, when all such rights, powers and discretions shall be exercisable by, and notices shall be given to, the Lender Agent or as it directs.

(b)
Pursuant to the terms of the Loan Agreement and the Charge, the Licensee hereby consents:

(i)
to the grant of the Charge;

(ii)
upon an Event of Default, to the assumption by the Lender Agent (or other nominee of the lenders under the Loan Agreement) of BioMedica’s rights under this Agreement; and

(iii)
to BioMedica’s disclosure (subject to the confidentiality terms set out in the Loan Agreement, provided that, such confidentiality terms are at least as protective to the Licensee’s Confidential Information as the terms set forth in this Agreement) to the Lender Agent and Oaktree of this Agreement and any non-technical information exchanged between the Parties, solely to the extent required in order for BioMedica to comply with its obligations to the Lender Agent and Oaktree under the Charge.

(iv)
the Licensee will sign and return two (2) original copies of the receipt of notice set out at Schedule 4 to BioMedica within [***] of the Effective Date.


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16.2
Force Majeure

Neither Party shall have any liability or be deemed to be in breach of this Agreement for any delays or failures in performance of this Agreement that result from circumstances beyond the reasonable control of that Party and which circumstances are not reasonably foreseeable. The Party affected by such circumstances shall promptly notify the other Party in writing when such circumstances cause a delay or failure in performance and use its reasonable endeavours to avoid or remove the causes of non-performance and shall continue performance as expeditiously as possible as soon as such causes have been removed.

16.3
Compliance with Law

Each Party shall perform its obligations under this Agreement in accordance with all Applicable Laws. No Party shall, or shall be required to, undertake any activity under or in connection with this Agreement which violates, or which it reasonably believes, in good faith, may violate, any Applicable Law.

16.4
Further Action

Each Party agrees, without the necessity of any further consideration, to execute, acknowledge, and deliver such further instruments, and do all further similar acts, as may be necessary or appropriate to carry out the purposes and intent of this Agreement.

16.5
Notices and Other Communications

Any notice to be given under this Agreement must be in writing, and be delivered to the other Party by courier or other recorded delivery post (with an advance copy by email) and will be deemed to be received on the date of delivery. Until changed by notice given in accordance with this Clause 16.5 (Notice and Other Communications), all notices should be addressed as follows:



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For BioMedica:
For the Licensee:
Attn: Company Secretary
Attn: President and Chief Commercial Officer
Address: Oxford BioMedica (UK) Limited
Address: Axovant Sciences GmbH
Windrush Court, Transport Way
Viaduktstrasse 8
Oxford, OX4 6LT, United Kingdom
4051 Basel, Switzerland
With a copy to: IP@oxfordbiomedica.co.uk
With a copy to: legal@axovant.com


16.6
Amendment

This Agreement may only be amended in writing signed by duly authorised representatives of the Parties.

16.7
Assignment

Neither Party may assign or otherwise transfer any of its rights nor obligations under this Agreement without the other Party’s prior written consent (which consent shall not be


68





unreasonably withheld or delayed), except that either Party may assign this Agreement, including all its rights and obligations under this Agreement, without the consent of the other Party, to (i) an Affiliate, or (ii) an entity acquiring all or substantially all of the assets or business of the assigning Party to which this Agreement relates (other than by way of acquisition of Control of a Party or its Affiliates, which for clarity, does not require an assignment of this Agreement), provided that, in all cases:

(a)
the assigning Party shall provide the non-assigning Party with prompt written notice of any such assignment; and

(b)
the permitted assignee shall assume the obligations of the assigning Party hereunder in writing.

Notwithstanding the foregoing sentence, either Party may grant or permit any lien to any person or entity in connection with a financing for the Licensee or its Affiliates from time to time, together with an assignment if legally required under such lien, provided that the Licensee is not relieved of any of its obligations hereunder.

16.8
Third Party Rights

The provisions of this Agreement are for the sole benefit of the Parties and their successors and permitted assigns, and they shall not be construed as conferring any rights to any Third Party except as otherwise expressly provided in Clause 14 (Liability, Indemnification and Insurance). Except as expressly provided in Clause 14 (Liability, Indemnification and Insurance), no person who is not a Party to this Agreement nor any Affiliate of the Licensee, shall have the right to enforce any term of this Agreement which expressly or by implication confers a benefit on that person without the express prior agreement in writing of the Parties.

16.9
Entire Agreement

This Agreement constitutes the entire agreements between the Parties relating to its subject matter and in relation to such subject matter supersedes all earlier understandings and agreements between the Parties.

16.10
Relationship

Nothing in this Agreement creates, implies or evidences any contract of employment or any partnership or joint venture between the Parties, or authorises either Party to act as agent for the other. Moreover, each Party agrees not to construe this Agreement, or any of the transactions contemplated hereby, as a partnership for any tax purposes. Each Party shall act solely as an independent contractor, and nothing in this Agreement shall be construed to give any Party the power or authority to act for, bind, or commit the other.

16.11
Waiver of Rights

No failure or delay by a Party to exercise any right or remedy provided under this Agreement or by law or to insist upon compliance with any term or condition of this Agreement will constitute a waiver of that (or any other) right or remedy or excuse a similar subsequent failure to perform any such term or condition by the other Party. No waiver shall be effective unless it has been given in writing and signed by the Party giving such waiver. No single or partial exercise of such


69





right or remedy will preclude or restrict the further exercise of that (or any other) right or remedy.

16.12
Unenforceable Provisions

If the whole or any part of any provision of this Agreement is unenforceable in any jurisdiction, then this Agreement shall be construed as if such provision were not contained herein and the remainder of this Agreement shall continue in full force and effect. The Parties shall use reasonable efforts to substitute for the invalid or unenforceable provision a valid and enforceable provision which conforms as nearly as possible to the original intent of the Parties.Thevalidity and enforceability of that provision in any other jurisdiction will not be affected.

16.13
Counterparts

This Agreement may be executed in any number of counterparts, each of which is an original but all of which together will constitute one document.

16.14
Governing Law

This Agreement and any dispute arising out of or relating to this Agreement is governed by and construed in accordance with English law, independent of any choice of law principles that may otherwise apply.

16.15
Dispute Resolution; Arbitration

Any dispute, controversy or claim initiated by either Party arising out of, resulting from or relating to this Agreement, or the performance by either Party of its obligations under this Agreement (a “Dispute”), shall be referred initially to the Senior Officers for resolution. The Party alleging a Dispute shall provide written notice specifying the claimed particulars of such Dispute to the Senior Officers of the other Party. The Party receiving such notice shall provide a written response within [***] of receipt. If the Senior Officers are unable to resolve the Dispute within [***] of the original notice, then the Dispute, whether before or after termination of this Agreement, shall be referred to and finally resolved by arbitration under the LCIA Rules, which Rules are deemed to be incorporated by reference into this Clause 16.15 (Dispute Resolution; Arbitration). The number of arbitrators shall be three (3). The seat, or legal place, of arbitration shall be London. The language to be used in the arbitral proceedings shall be English. Each Party shall nominate one (1) arbitrator. The Parties shall request that the LCIA appoints the third arbitrator as the presiding arbitrator. When appointing arbitrators, the Parties and the LCIA will seek to appoint arbitrators whose availability enables compliance with the timetable set out in this Clause 16.15 (Dispute Resolution; Arbitration). Given the expedited nature of the arbitration, the arbitral tribunal shall have the authority to order limited production of documents relevant and material to the issues in dispute, upon the showing by either party of a justifiable need for such document(s). Except as may be required by law, neither a party nor its representatives may disclose the existence, content, or results of any arbitration hereunder without the prior written consent of both parties. The Parties each agree that they shall take all possible steps to: (a) comply with the timetable set out in this Clause 16.15 (Dispute Resolution; Arbitration); (b) assist the Tribunal in complying with the timetable set out in this Clause16.15 (Dispute Resolution; Arbitration); and (c) convene the final hearing as soon as possible. The final award shall be made by the arbitral tribunal within


70





[***] from the appointment of the presiding arbitrator, unless the arbitral tribunal concludes that the interest of justice requires that such limit be extended by a reasonable amount of time. The arbitration award shall be final and binding on the parties. Judgment upon the award may be entered by any court having jurisdiction thereof. Subject to the right of the tribunal to order otherwise, the prevailing party shall be entitled to recover its reasonable costs, including administrative fees and expenses, arbitrators’ fees and expenses, and fees and expenses of legal representation, incurred in the arbitration proceedings. Notwithstanding the foregoing, the Parties agree that if the tribunal determines that the Licensee was in material breach of its diligence obligations under Clause 6.5 (Diligence) at the date on which a dispute was referred to arbitration but that the Licensee cured such breach before the tribunal hands down the final award, the tribunal will order the Licensee to pay BioMedica's reasonable costs, including administrative fees and expenses, arbitrators’ fees and expenses, and fees and expenses of legal representation, incurred in the arbitration proceedings.

16.16
Interim or Provisional Relief

Nothing contained in this Agreement shall prevent either Party from seeking interim or provisional relief from a court of competent jurisdiction, including a preliminary injunction or other interim measures either prior to or during any arbitration, if necessary to protect the interests of such Party or to preserve the status quo. Such an action may be filed and maintained notwithstanding any ongoing discussions between the Parties, and any such request shall not be deemed incompatible with the agreement to arbitrate or a waiver of any right under this Agreement.

16.17
Cumulative Remedies

No remedy referred to in this Agreement is intended to be exclusive, but each shall be cumulative and in addition to any other remedy referred to in this Agreement or otherwise available under law.

16.18
Performance by Affiliates

Each Party may perform some or all of its obligations under this Agreement through its Affiliates and may exercise some or all of its rights under this Agreement through its Affiliates; provided, that each Party shall remain responsible and be the guarantor of the performance by its Affiliates and shall cause its Affiliates to comply with the provisions of this Agreement in connection with such performance or exercise. Each Party shall prohibit all of its Affiliates from taking any action that such Party is prohibited from taking under this Agreement as if such Affiliates were parties to this Agreement.

[Signature page follows]


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[Intentionally Blank]


71





[Intentionally Blank]

72





This Agreement has been entered into on the date shown on the first page.

OXFORD BIOMEDICA (UK) LIMITED

Signature:
/s/ John Dawson
Name:
John Dawson
Title:
CEO


73





This Agreement has been entered into on the date shown on the first page.

AXOVANT SCIENCES GMBH

Signature:
/s/ Mark Altmeyer
Name:
Mark Altmeyer
Title:
President



74





SCHEDULE 1
PATENTS and PUBLICATIONS


[***]







75










76





SCHEDULE 2
OXB Project Personnel

Team Member
Role
% FTE dedicated to AXON
[***]
Clinical Lead
[***]%
 
[***]
Senior Clinical Study Manager
[***]%
 
[***]
Senior Clinical Study Manager
[***]%
 
[***]
Regulatory Lead
[***]%
 
[***]
Pharmacovigilance
[***]%
 
[***]
Medical Monitor
[***]%
 
[***]
Project Manager
[***]%
 
To be confirmed
Quality Assurance
[***]%
 
To be confirmed
Senior Clinical Study Administrator
[***]%
 


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SCHEDULE 3
TEMPLATE WORK PACKAGE

Work Package [●]

This Work Package (the “Work Package”) is made on [●] (“Work Package Effective Date”) BETWEEN
(1)
OXFORD BIOMEDICA (UK) LIMITED, a company incorporated in England and registered under number 03028927, whose registered office is at Medawar Centre,
Robert Robinson Avenue, Oxford Science Park, Oxford, OX4 4GA (“BioMedica”); and
(2)
AXOVANT SCIENCES GMBH, a corporation organized and existing under the laws of Switzerland, having its principal place of business at Viaduktstrasse 8, 4051 Basel, Switzerland (the “Licensee”).


BACKGROUND

BioMedica and the Licensee have entered into a Licence Agreement dated June 5, 2018 (the “Agreement”) under which they agreed to perform certain activities on the terms of that Agreement. Unless otherwise defined in this Work Package, capitalized terms used in this Work Package shall have the meaning given to them in the Agreement.

1.
SUBSTANTIVE PROVISIONS

Work Package reference
Work Package [●]
Version number
 
Agreement to which this Work Package relates
License Agreement dated June 5, 2018 between Oxford BioMedica and the Licensee
Title of Work Package
 
Scope of Work Package (brief overview)
insert ‘See above’ if title provides sufficient overview
Expected start date
 
Estimated duration of Work Package
 
Estimated costs of Work Package
time and material work packages to include estimated resource costs, estimated FTE days, estimated materials and external costs, total estimated amount for services and materials, and payment schedule; fixed cost work packages to include detailed payment schedule
 
Materials to be provided by BioMedica
 
Materials to be provided by the Licensee
 
Information to be provided by the Licensee
 
Deliverables
 


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Approved subcontractors (if any)
2.
DETAILED DESCRIPTION OF ACTIVITIES TO BE COMPLETED UNDER WORK
PACKAGE:

2.1
[work plan]

3.
TERM AND TERMINATION

3.1
Unless terminated earlier in accordance with the terms of the Agreement or this Clause 3, this Work Package shall continue until all obligations under this Work Package have been performed.

3.2
The Licensee may terminate this Work Package for any reason by giving not less than [***] prior written notice to BioMedica and upon expiry of such notice, this Work Package shall be terminated and the parties shall proceed in accordance with Clause 3.4 of this Work Package.

3.3
If either Party is in breach of any material obligation under this Work Package (including failure by the Licensee to make an payment due in connection with this Work Package), the non-breaching Party may give written notice to the breaching Party specifying the claimed particulars of such breach, and in the event such breach is not cured within [***] after such notice (or [***] with respect to any failure to make any payment due under this Work Package), the non-breaching Party shall have the right thereafter to terminate this Work Package immediately by giving written notice to the breaching Party; provided, however, that if such breach is capable of being cured but cannot be cured within such cure period and the breaching Party initiates actions to cure such breach within such period and thereafter diligently pursues such actions, the breaching Party shall have such additional period as is reasonable in the circumstances to cure such breach.

3.4
In the event of termination of this Work Package:

(a)
the Parties shall cease all work related to this Work Package and have no further obligations to perform activities under this Work Package, save that the Parties shall carry out those limited activities as are necessary for an orderly wind-down of the relevant Work Package;

(b)
the Licensee shall pay to BioMedica following receipt of a reasonably detailed invoice all undisputed amounts due and all approved expenses actually incurred by BioMedica in relation to the performance of this Work Package prior to the effective date of termination and orderly wind-down activities (provided that BioMedica shall pay costs of wind-down activities if termination is due to BioMedica’s uncured material breach under Clause 3.3 of this Work Package).

4.
[ADDITIONAL TERMS]

[insert any additional terms or variation to the Agreement]


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Agreed by:

OXFORD BIOMEDICA (UK) LIMITED

Signature:              Date:     

Name:         

Title:         



AXOVANT SCIENCES GMBH
Signature:              Date:     

Name:         

Title:         


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SCHEDULE 4
NOTICE OF CHARGE

To:
Axovant Sciences GmbH Viaduktstrasse 8
4051 Basel, Switzerland
Attention: President and Chief Commercial Officer
With a copy to:
The Legal Department and Ryan Morick Cortland Capital Market Services LLC
225 W. Washington Street 9 th Floor
Chicago, Illinois 60606 and
The Legal Department and Edgar Lee
OCM Strategic Credit Investments S.à r.l. OCM Strategic Credit Investments 2 S.à r.l. OCM Luxembourg SC Fund A S.à r.l.
OCM Luxembourg SC Fund B S.à r.l. 26A Boulevard Royal, 7th Floor,
L-2449 Luxembourg
Facsimile: +352 26 63 25 47 00
E-mail: amkumar@oaktreecapital.com and
The Legal Department and Edgar Lee Oaktree Capital Management, L.P.
333 South Grand Ave., 28th Floor Los Angeles, CA 90071
Facsimile: (213) 830-6293
Email: amkumar@oaktreecapital.com and
Ari Blaut
Sullivan & Cromwell LLP 125 Broad Street
New York NY 10004
Facsimile: (212) 291 9219
E-mail: blauta@sullcrom.com

Date: June 5, 2018

To whom it may concern


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We, Oxford BioMedica (UK) Limited (the “Company”) HEREBY GIVE NOTICE that by a first fixed charge contained in a mortgage debenture (the “Debenture”) dated 29 June 2017 and made between the Company and Cortland Capital Market Services LLC (the “Administrative Agent”) the Company charged to the Administrative Agent all of its present and future right, title and interest in and to the licence agreement effective June 5, 2018 made between Oxford BioMedica (UK) Limited and Axovant Sciences GmbH (the “Agreement”), including, but not limited to, the right to demand and receive all moneys whatsoever payable to or for the benefit of the Company under or arising from the Agreement, all remedies provided for in the Agreement or available at law or in equity in relation to the Agreement, the right to compel performance of the Agreement and all other rights, interests and benefits whatsoever accruing to or for the benefit of the Company arising from the Agreement.

All moneys payable by you to the Company pursuant to the Agreement shall be paid to the Company’s account as set out in the Agreement unless and until you receive notice from the Administrative Agent to the contrary informing you that an Event of Default, as defined in the Debenture, has occurred, in which event you should make all future payments as directed by the Administrative Agent.

Notwithstanding the charge referred to above or the making of any payment by you to the Administrative Agent pursuant to it, the Company shall remain liable under the Agreement to perform all the obligations assumed by it under the Agreement and neither the Administrative Agent nor any receiver nor any delegate appointed by the Administrative Agent or any such receiver shall be at any time under any obligation or liability to you under or in respect of the Agreement. The Company shall also remain entitled to exercise all its rights, powers and discretions under the Agreement and you should continue to give notices under the Agreement to the Company in each case unless and until you receive notice from the Administrative Agent to the contrary informing you that an Event of Default has occurred, when all such rights, powers and discretions shall be exercisable by, and notices shall be given to, the Administrative Agent or as it directs.

The Company confirms that:

(i)
in the event of any conflict between communications received from it and from the Administrative Agent, the communication from the Administrative Agent shall prevail;

(ii)
none of the instructions, authorisations or confirmations in this Notice of Charge (the “Notice”) can be revoked or varied in any way except with the Administrative Agent’s specific written consent; and

(iii)
any written notice or instructions given to you by the Administrative Agent in accordance with this Notice shall be conclusive.

Kindly acknowledge receipt of this Notice and confirm your agreement to it by signing the enclosed form of acknowledgement and returning it to the Administrative Agent at 225 W. Washington Street, 9 th Floor Chicago, Illinois 60606 for the attention of Ryan Morick.

This Notice, and any non-contractual obligations arising out of, or in connection with it is governed by English law.


82






To:    Cortland Capital Market Services LLC
Address:
225 W. Washington Street,
9 th Floor
Chicago, Illinois 60606
Attention    The Legal Department and Ryan Morick With a copy to:    The Legal Department and Edgar Lee
OCM Strategic Credit Investments S.à r.l.
OCM Strategic Credit Investments 2 S.à r.l.
OCM Luxembourg SC Fund A S.à r.l.
OCM Luxembourg SC Fund B S.à r.l. 26A Boulevard Royal, 7th Floor,
L-2449 Luxembourg
Facsimile: +352 26 63 25 47 00
E-mail: amkumar@oaktreecapital.com
and
The Legal Department and Edgar Lee
Oaktree Capital Management, L.P. (together with “OCM”, the “Lenders”)
333 South Grand Ave., 28th Floor Los Angeles, CA 90071 Facsimile: (213) 830-6293
Email: amkumar@oaktreecapital.com
and
Ari Blaut
Sullivan & Cromwell LLP 125 Broad Street
New York NY 10004
Facsimile: (212) 291 9219
E-mail: blauta@sullcrom.com


Date: Dear Sirs
We acknowledge the Charge in respect of the licence agreement dated June 5, 2018 by and between Oxford BioMedica (UK) Limited and Axovant Sciences GmbH (the “Agreement”) as set out in the Agreement and the Notice of Charge dated June 5, 2018 (the “Notice”). Terms and expressions defined in the Notice shall have the same meanings when used in this acknowledgment.

We agree to and confirm that:


83





(a)
we will pay all moneys hereafter becoming due to Oxford BioMedica (UK) Limited (“Oxford BioMedica”) in respect of the Agreement as directed in the Notice and accept and will comply with the terms of the Notice;

(b)
we will send to you copies of any notices which we may give to Oxford BioMedica under the Agreement at the same time as we send them to Oxford BioMedica;

(c)
we have not received notice of any other charge, assignment or other Third Party right or interest whatsoever in, of, over, or affecting, Oxford BioMedica’s interests in the Agreement or any other notice relating to the Agreement; and

(d)
this acknowledgement is freely assignable or transferable by you, by any subsequent assignee, transferee or successor in title in accordance with the terms of the Agreement (“Subsequent Party”) and by any receiver appointed by you or by any Subsequent Party pursuant to the Debenture.


Yours faithfully,


AXOVANT SCIENCES GMBH


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SCHEDULE 5
Net Sales

“Net Sales” shall mean the [***]:

(a)
[***];

(b)
[***];

(b)
[***];

(c)
[***];

(d)
[***];

(e)
[***];

(f)
[***];

(g)
[***];


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(h)
[***]; and

(i)
[***];

[***].

Net Sales shall be determined from the books and records of each Selling Entity maintained in accordance with GAAP or IFRS, as applicable, consistently applied. Notwithstanding anything else contained in this Schedule 5 , in the event of an inconsistency between “Net Sales” calculated pursuant to this Schedule 5 and “net sales” publicly reported by a Selling Entity in financial statements required by Applicable Law (including, for Licensee and its Affiliates, in periodic reports filed with U.S. Securities and Exchange Commission) for any period, the “net sales” in such publicly reported financial statements shall be deemed to be the “Net Sales” for such period for purposes of this Agreement.

[***].

[***].

[***].

[***].

In the case of rebates, discounts and other forms of reimbursements on portfolio product offerings, all rebates, discounts and other forms of reimbursements shall be allocated among products on the basis on which such rebates, discounts and other forms of reimbursements were actually granted or, if such basis cannot be determined, in accordance with the applicable Selling Entity’s existing allocation method, consistently applied to all relevant products; provided that, any such allocation is fair and reasonable and in compliance with generally accepted accounting principles and Applicable Laws, including any price reporting laws, rules and regulations.

[***].

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If the Parties cannot agree on the mean selling prices of the Product and Accompanying Innovative Product for the purpose of this paragraph, or the fair market value as referred to in this paragraph, the Parties shall refer the matter to a panel of three (3) independent experts in the field (“Expert Panel”) for a determination. Each of BioMedica and Licensee shall select one independent expert each within [***] after either Party notifies the other in writing that it wishes to proceed to expert determination and such experts will select a third independent expert within [***] thereafter. The Expert Panel will determine the mean selling prices of the Product and Accompanying Innovative Product or the fair market value as referred to in this paragraph. The Expert Panel will meet in London, England so that each Party may present its position. The Expert Panel will, within [***] after the conclusion of the meeting, issue a written determination describing the findings and conclusions of the Expert Panel. Each Party shall bear its own counsel fees, costs, and disbursements arising out of the Expert Panel described in this Schedule 5 , and shall pay an equal share of the fees and costs of the experts and all other general fees related to the Expert Panel.


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SCHEDULE 6

[***]





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SCHEDULE 7
Clinical Supply Agreement Terms Outline


The clinical supply price shall not exceed the cGMP Batch price for current 200L transient serum-free suspension process remains at [***]. 1
Work that is not on a batch basis, such as process characterisation, will be invoiced at the FTE Rate.
The Licensee to have the right to technology transfer of the manufacturing process if there is a prolonged failure by BioMedica to supply Product (to be defined in such agreement, but which shall include, (i) an objective, backward-looking standard, such as repeated failure to supply the Licensee with quantities ordered and (ii) a subjective, forward-looking standard, such as reasonably likely failure as a result of catastrophe or other circumstances beyond the reasonable control of BioMedica), without prejudice to the rights under Clause 7.3 (Technology Transfer) of this Licence Agreement.
BioMedica reserves the right to review the FTE Rates and cGMP batch prices on an annual basis from 2019 onwards.
Quality Agreement to be put in place between BioMedica Quality personnel and the Licensee Quality personnel to regulate matters in relation to the supply agreement.
The Licensee to provide [***] batch forecast with [***] binding period. BioMedica agrees to maintain the capacity and availability to supply the Licensee with quantities of Product consistent with such forecasts.


Commercial Supply Agreement Terms Outline
The price for the Product for use by Licensee for commercial purposes will be on a batch-by-batch basis that is consistent with, and in no event less favourable to Licensee than, BioMedica’s other reasonable, normal, and customary manufacturing costing methodology it uses with Third Parties.
















1 This includes, facility, personnel, consumables, and fill-finish but excludes Plasmid costs.


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SCHEDULE 8

Agreed Form Press Releases

Attached.


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OXFORDBIOMEDIC_2.JPG      AXOVANTOXFORDBIOMEDIC_IMAGE3.JPG



AXOVANTOXFORDBIOMEDIC_IMAGE4.JPG


Call in details for analysts

Oxford BioMedica and Axovant Sciences enter into a $842.5 Million Exclusive Worldwide Licence Agreement for OXB-102 for the Treatment of Parkinson’s Disease
Oxford, UK– 06 June 2018: Oxford BioMedica plc (LSE:OXB) (“Oxford BioMedica” or “the Group”), a leading gene and cell therapy group, today announces that it has entered into an exclusive worldwide licensing agreement with Axovant Sciences (NASDAQ: AXON) to develop and commercialise OXB-102, a gene therapy developed by Oxford BioMedica for Parkinson’s disease utilising the LentiVector ® platform.

Under the terms of the agreement, Oxford BioMedica will receive a $30 million upfront payment (approximately £22 million) including $5 million as pre-payment for manufacturing activities related to OXB-102, now renamed, AXO-Lenti-PD. Oxford BioMedica is also eligible to receive $55 million upon the achievement of specified development milestones and $757.5 million upon the achievement of specified regulatory and sales milestone, with 7% to 10% tiered royalties on net sales of AXO-Lenti-PD.

Axovant Sciences, a clinical-stage biopharmaceutical company dedicated to advancing treatments for patients with life-altering neurologic conditions and a member of the Roivant family of companies, will fund all clinical development costs and manufacturing process development and scale-up activities for AXO-Lenti-PD. The agreement also allows for both parties to put in place a clinical and commercial supply agreement for GMP manufacturing of AXO-Lenti-PD at Oxford BioMedica. Roivant remains committed to Axovant’s success and are excited about the scientific potential of AXO-Lenti-PD. Axovant will be able to harness the full Roivant drug development platform to ensure its rapid development.

Axovant Sciences expects to initiate a Phase I/II dose escalation study of AXO-Lenti-PD in patients with advanced Parkinson’s disease by the end of 2018.

John Dawson, Chief Executive Officer of Oxford BioMedica, said: “We are delighted to sign this significant agreement which not only underlines our LentiVector ® enabled platform and product development strategy but further demonstrates Oxford BioMedica’s ability to build multiple partnerships with leaders in their respective therapeutics fields. This agreement with Axovant also successfully demonstrates Oxford BioMedica’s pre-stated strategy to externalise product development beyond the end of the pre-clinical phase.

“Axovant’s expertise and focus on neurological disorders, which includes Parkinson’s disease, makes them an ideal development and commercialisation partner for this programme. Coupled with


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strong support from parent company Roivant, we believe Axovant is perfectly positioned to bring AXO-Lenti-PD to the market as quickly as possible to treat patients with Parkinson’s, a disease which still has a high unmet need.”

Commenting on the announcement, Pavan Cheruvu, Chief Executive Officer of Axovant Sciences, said: “Axovant, together with our parent company Roivant, remains committed to developing innovative treatments for serious degenerative conditions such as Parkinson’s disease, and we are excited to partner with Oxford BioMedica, a recognised global leader in cell and gene therapy. OXB-102 is a potentially best-in-class gene therapy with the potential to transform Parkinson’s disease treatment. This is an area of significant unmet medical need and a major market opportunity. Advancing this high-quality candidate is a key priority for the team at Axovant and we very much look forward to working with Oxford BioMedica.”

-Ends-

For further information, please contact:


Oxford BioMedica plc:
John Dawson, Chief Executive Officer
Stuart Paynter, Chief Financial Officer

Tel: +44 (0)1865 783 000

Financial and corporate communications enquiries:
Consilium Strategic Communications
Mary-Jane Elliott/Matthew Neal/Laura Thornton

Tel: +44 (0)20 3709 5700


Peel Hunt (Joint Corporate Brokers):
James Steel
Christopher Golden

Tel: +44 (0)20 7418 8900

WG Partners (Joint Corporate Brokers):
David Wilson
Claes Spang

Tel: +44 (0)20 3705 9321



The information contained within this announcement is deemed by the Company to constitute inside information as stipulated under the Market Abuse Regulation (EU) No. 596/2014. Upon the publication of this announcement via the Regulatory Information Service, this inside information is now considered to be in the public domain.




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Notes to editors

About Oxford BioMedica ®
Oxford BioMedica (LSE:OXB) is a leading gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford BioMedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector ® ), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. The Group has also entered into a number of partnerships, including with Novartis, Bioverativ, Sanofi, GSK, Orchard Therapeutics, GC LabCell and Immune Design, through which it has long-term economic interests in other


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potential gene and cell therapy products. Oxford BioMedica is based across several locations in Oxfordshire, UK and employs more than 320 people. Further information is available at www.oxfordbiomedica.co.uk .

About OXB-102 (AXO-Lenti-PD)
OXB-102, now known as AXO-Lenti-PD, is a lentiviral gene therapy with a clinically validated mechanism of action for the delivery of the three genes that encode the enzymes required for endogenous dopamine synthesis. Unlike current drug treatment, in which efficacy diminishes with long-term use, AXO-Lenti-PD is designed to provide patient benefit for multiple years following a single administration. AXO-Lenti-PD is a second-generation vector construct of OXB-101, also known as ProSavin ® , with greater potency and increased transgenic expression observed in preclinical studies. Oxford BioMedica has successfully completed a Phase I/II study for OXB-101, which met its primary endpoint. The results, which were published in Lancet in 2014, demonstrate favourable safety and tolerability and a statistically significant improvement of motor function as measured by the UPDRS Part III score at 6 and 12 months following a one-time administration. This improvement was sustained in most patients for up to four years despite the progressively degenerative nature of Parkinson’s disease.

About Axovant Sciences
Axovant is a clinical-stage biopharmaceutical company dedicated to advancing treatments for patients with life-altering neurologic conditions. Axovant is committed to developing a pipeline of product candidates, and ultimately commercializing them, by identifying and developing novel treatments for unmet needs in neurology. For more information, visit www.axovant.com .

About Roivant Sciences
Roivant Sciences is a global biopharmaceutical company focused on reducing the time and cost of the drug development process to improve the lives of patients and their families. Roivant partners with innovative biopharmaceutical companies and academic institutions to ensure that important medicines are rapidly delivered to patients.

The Roivant family of companies includes Myovant (women’s health and prostate cancer), Axovant (neurology), Urovant (urology), Enzyvant (rare diseases), Dermavant (dermatology), Genevant (RNA therapeutics), Metavant (cardiometabolic diseases), Datavant (healthcare data), and Arbutus (hepatitis B). Today there are 24 investigational drugs in 11 therapeutic areas being tested in over 50 clinical trials and over 300 nonclinical studies across the Roivant family of companies.

For more information, please visit www.roivant.com


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DRAFT – CONFIDENTIAL
Axovant Licenses Investigational Gene Therapy for Parkinson’s Disease from Oxford BioMedica and Announces Key Leadership Team Addition
Exclusive worldwide license to lentiviral vector gene therapy constitutes the first transaction of Axovant’s 2018 pipeline expansion
Fraser Wright, Co-Founder and former Chief Technology Officer of Spark Therapeutics, to join Axovant as CTO for gene therapy programs
Axovant will receive $25 million equity financing from Roivant Sciences to support clinical development of AXO-Lenti-PD and additional business development
Conference call / webcast today at 8:00am Eastern Time

BASEL, Switzerland, June 6, 2018 (GLOBE NEWSWIRE) -- Axovant Sciences (NASDAQ:AXON) today announced that it has licensed the exclusive worldwide rights to develop and commercialize OXB-102, now AXO-Lenti-PD, from Oxford BioMedica. AXO-Lenti-PD is an investigational gene therapy for Parkinson’s disease that delivers three genes encoding a critical set of enzymes required for dopamine synthesis in the brain. Oxford BioMedica is a world leader in lentiviral vector product development and manufacturing, and will be the clinical and commercial supplier of AXO-Lenti-PD. Axovant expects to initiate a Phase 1/2 dose escalation study of AXO-Lenti-PD in patients with advanced Parkinson’s disease by the end of 2018.

Under the terms of the license agreement with Oxford BioMedica, Axovant obtained rights to AXO- Lenti-PD, as well as its predecessor product ProSavin®, for an initial payment of $30 million in cash, $5 million of which will be applied as a credit against the process development work and clinical supply that Oxford BioMedica will provide to Axovant. Oxford BioMedica is also eligible to receive additional development, regulatory and commercial milestone payments potentially in excess of $812 million, and tiered royalties on net sales of AXO-Lenti-PD, if approved. Roivant has agreed to purchase $25 million of Axovant common shares, which will support the clinical development of AXO-Lenti-PD and additional business development activities.

Fraser Wright, PhD, will join Axovant as Chief Technology Officer overseeing the company’s gene therapy initiatives. Dr. Wright is the Co-Founder and former Chief Technology Officer of Spark Therapeutics and has over 20 years of leadership experience in the development of novel vector- based biologic products. At Spark he oversaw process development and clinical-stage manufacturing for LUXTURNA™. Prior to Spark, he was the founding Scientific Director of the Clinical Vector Core Laboratory at The Children’s Hospital of Philadelphia, where he directed clinical core staff in gene therapy investigational product development, manufacture, and quality control testing for ten first-in-human viral vector investigational products including LUXTURNA™ and KYMRIAH®. He was also previously the Director of Development and Clinical Manufacturing at Avigen. Dr. Wright was formerly a Research Professor of Pathology and Laboratory Medicine at the University of Pennsylvania School of Medicine, and he is the lead inventor on numerous issued patents during his time in that role. He received his BSc and PhD in biochemistry from the University of Toronto, where he was also an assistant professor of biochemistry and medicine.

Pavan Cheruvu, MD, Chief Executive Officer of Axovant, stated, “Axovant remains committed to developing innovative treatments for serious neurodegenerative conditions such as Parkinson’s disease, and we are excited to partner with Oxford BioMedica, a recognized global leader in cell and gene therapy. We are also pleased to welcome Fraser to our leadership team. He brings over two decades of experience in gene therapy manufacturing, and will be committed to building world- class gene therapy capabilities at Axovant. We will continue to pursue promising new therapeutic approaches based on transformative science, and will further expand our pipeline with high-quality


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assets like AXO-Lenti-PD. This is part of our long-term goal of building Axovant into a leader in the development and commercialization of innovative new medicines for neurological indications.”

“This is an exciting time to join Axovant, and I look forward to the opportunity to work closely with Oxford BioMedica and help build gene therapy capabilities at Axovant,” said Dr. Wright. “AXO-Lenti- PD is a strong foundation for Axovant’s new pipeline, and I am excited to begin preparing the Phase 1/2 clinical study in advanced Parkinson’s disease later this year.”

Commenting on the announcement, John Dawson, Chief Executive Officer of Oxford BioMedica said: “We are delighted to sign this significant agreement which not only underlines our LentiVector®-enabled platform and product development strategy but further demonstrates Oxford BioMedica’s ability to build multiple partnerships with leaders in their respective therapeutics fields. We believe Axovant’s expertise and focus on neurological disorders, which includes Parkinson’s disease, makes them an ideal development and commercialisation partner for this programme. Coupled with strong support and financial resources from parent company Roivant, we believe Axovant is well positioned to advance the development of AXO-Lenti-PD for the treatment of patients with Parkinson’s, a disease which still has a high unmet need.”

Teleconference/Webcast Details
To participate in the live conference call today, June 6, at 8:00 a.m. EDT, please dial 1-833- 652-5918 from the U.S. and Canada or +1 409-767-9227 internationally, and use the passcode 8289429.
The live call is being webcast and can be accessed on the “Events and Presentations” page of the “Investors” section of the Company’s website at http://investors.axovant.com. A replay of the webcast will be available for 30 days following the live event.

About AXO-Lenti-PD
AXO-Lenti-PD, formerly OXB-102, is an investigational gene therapy for Parkinson’s disease that delivers three genes encoding a critical set of enzymes required for dopamine synthesis in the brain and is designed to provide patient benefit for multiple years following a single administration. AXO- Lenti-PD is a next-generation gene therapy with a modified payload configuration of the predecessor product, ProSavin®, to further improve endogenous dopamine production. Oxford BioMedica has successfully completed a Phase 1/2 study for ProSavin, which met its primary endpoint. The results, which were published in The Lancet in 2014, demonstrate favorable safety and tolerability and a statistically significant improvement of motor function as measured by the UPDRS Part III score at 6 and 12 months. This improvement was sustained in most patients for up to four years despite the progressively degenerative nature of Parkinson’s disease.

About Parkinson’s Disease
Parkinson’s disease is caused by degeneration of nerve cells in a portion of the brain called the substantia nigra which leads to a reduction in dopamine. Low dopamine causes nerve cells to activate without normal control. Characteristic Parkinson’s disease symptoms include tremor, limb rigidity, slow physical movement, and gait and balance issues. Approximately one million Americans live with Parkinson's disease, with 60,000 diagnosed each year. The combined direct and indirect cost of Parkinson’s disease, including treatment, Social Security payments, and lost income, is estimated to be nearly $25 billion per year in the United States alone.

About Axovant Sciences
Axovant is a clinical-stage biopharmaceutical company dedicated to advancing innovative treatments for patients with serious neurologic and neuropsychiatric conditions, and turning promising therapies into lasting solutions for patients. Axovant is committed to developing and commercializing a pipeline of product candidates


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by identifying and developing novel treatments for unmet needs in neurology and psychiatry.
About Oxford BioMedica
Oxford BioMedica (LSE:OXB) is a leading gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford BioMedica and its subsidiaries (the "Group") have built a sector-leading lentiviral vector delivery platform (LentiVector®), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. The Group has also entered into a number of partnerships, including with Novartis to manufacture Kymriah®, Bioverativ, Sanofi, GSK, Orchard Therapeutics, GC LabCell and Immune Design, through which it has long-term economic interests in other potential gene and cell therapy products. Oxford BioMedica is based across several locations in Oxfordshire, UK and employs more than 320 people.
About Roivant Sciences
Roivant Sciences is a global biopharmaceutical company focused on reducing the time and cost of the drug development process to improve the lives of patients and their families. Roivant partners with innovative biopharmaceutical companies and academic institutions to ensure that important medicines are rapidly delivered to patients.
Forward-Looking Statements and Information
This press release contains forward-looking statements, including statements regarding Axovant’s plans to advance the development of AXO-Lenti-PD and expand its pipeline. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the ability to identify and in- license or acquire product candidates, and the success, cost and timing of Axovant’s product development activities and any planned clinical trials. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Axovant’s business in general, see the “Risk Factors” section of Axovant’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on February 9, 2018, and other filings that Axovant makes with the SEC from time to time. These forward-looking statements are based on information available to Axovant as of the date of this press release and speak only as of the date of this release. Axovant disclaims any obligation to update these forward-looking statements, except as may be required by law.
Financial details regarding this transaction will be provided in Axovant’s Form 8-K to be filed with the SEC. All trademarks are property of their respective owners.

Contacts: Investors
Tricia Truehart (631) 892-7014
investors@axovant.com

Media


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Paul Davis
(646) 495-5310
media@axovant.com


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SCHEDULE 9


[***]




99



Exhibit 31.1
CERTIFICATION
I, Pavan Cheruvu, certify that:
1. I have reviewed this Quarterly Report on Form 10-Q of Axovant Sciences Ltd.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) for the registrant and have:
a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
b)     Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

c)     Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and

d)     Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and

5. The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
Date: August 7, 2018
By:
/s/ Pavan Cheruvu
 
 
Pavan Cheruvu
 
 
Principal Executive Officer





Exhibit 31.2
  CERTIFICATION
I, Gregory Weinhoff, certify that:
1. I have reviewed this Quarterly Report on Form 10-Q of Axovant Sciences Ltd.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) for the registrant and have:
a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
5. The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
Date: August 7, 2018
By:
/s/ Gregory Weinhoff
 
 
Gregory Weinhoff
 
 
Principal Financial Officer





Exhibit 32.1
CERTIFICATION PURSUANT TO 18 U.S.C. SECTION 1350,
AS ADOPTED PURSUANT TO
SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002  
In connection with the Quarterly Report on Form 10-Q of Axovant Sciences Ltd. (the “Company”) for the period ended June 30, 2018 as filed with the Securities and Exchange Commission on the date hereof (the “Report”), the undersigned, Pavan Cheruvu, Principal Executive Officer of the Company, hereby certifies, pursuant to 18 U.S.C. Section 1350, that to his knowledge: 
(1) the Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
(2) the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of the Company.
Date: August 7, 2018
By:
/s/ Pavan Cheruvu
 
 
Pavan Cheruvu
 
 
Principal Executive Officer


A signed original of this written statement required by Section 906 has been provided to the Company and will be retained by the Company and furnished to the SEC or its staff upon request.





Exhibit 32.2
CERTIFICATION PURSUANT TO 18 U.S.C. SECTION 1350,
AS ADOPTED PURSUANT TO
SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002
In connection with the Quarterly Report on Form 10-Q of Axovant Sciences Ltd. (the “Company”) for the period ended June 30, 2018 as filed with the Securities and Exchange Commission on the date hereof (the “Report”), the undersigned, Gregory Weinhoff, Principal Financial Officer of the Company, hereby certifies, pursuant to 18 U.S.C. Section 1350, that to his knowledge:
(1) the Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
(2) the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of the Company.
Date: August 7, 2018
By:
/s/ Gregory Weinhoff
 
 
Gregory Weinhoff
 
 
Principal Financial Officer

A signed original of this written statement required by Section 906 has been provided to the Company and will be retained by the Company and furnished to the SEC or its staff upon request.