|
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
|
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
Delaware
|
47-2608175
|
|
(State or other jurisdiction of incorporation or organization)
|
(IRS Employer Identification No.)
|
Large accelerated filer
|
Accelerated filer
|
Non-accelerated filer
|
Smaller reporting company
|
(Do not check if a smaller reporting company)
|
|
Emerging growth company
|
PART I
|
FINANCIAL INFORMATION
|
||||
Item 1.
|
Financial Statements (unaudited):
|
||||
Condensed Consolidated Balance Sheets as of March 31, 2018 and December 31, 2017 (as revised)
|
3
|
||||
Condensed Consolidated Statements of Operations for the three months ended March 31, 2018 and 2017 (as revised)
|
4
|
||||
Condensed Consolidated Statements of Comprehensive Loss for the three months ended March 31, 2018 and 2017 (as revised)
|
5
|
||||
Condensed Consolidated Statements of Cash Flows for the three months ended March 31, 2018 and 2017 (as revised)
|
6
|
||||
Notes to Condensed Consolidated Financial Statements
|
7
|
||||
Item 2.
|
Management's Discussion and Analysis of Financial Condition and Results of Operations:
|
||||
Overview
|
16
|
||||
Results of Operations
|
20
|
||||
Liquidity and Capital Resources
|
22
|
||||
Item 3.
|
Quantitative and Qualitative Disclosures about Market Risk
|
23
|
|||
Item 4.
|
Controls and Procedures
|
24
|
|||
PART II
|
OTHER INFORMATION
|
||||
Item 1.
|
Legal Proceedings
|
25
|
|||
Item 1A
|
Risk Factors
|
25
|
|||
Item 2.
|
Unregistered Sales of Equity Securities and Use of Proceeds
|
47
|
|||
Item 3.
|
Defaults Upon Senior Securities
|
47
|
|||
Item 4.
|
Mine Safety Disclosures
|
47
|
|||
Item 5.
|
Other Information
|
47
|
|||
Item 6.
|
Exhibits
|
48
|
|||
Signatures
|
|
49
|
|
March 31,
2018
|
December 31,
2017
|
||||||
(as revised)
|
||||||||
ASSETS
|
||||||||
Current assets:
|
||||||||
Cash and cash equivalents
|
$
|
69,907
|
$
|
58,367
|
||||
Short-term investments
|
175,028
|
201,763
|
||||||
Contracts receivable
|
-
|
5,413
|
||||||
Other current assets
|
5,628
|
1,302
|
||||||
Total current assets
|
250,563
|
266,845
|
||||||
Property, plant and equipment, net
|
50
|
77
|
||||||
Licenses, net
|
1,191
|
1,221
|
||||||
Deposits and other assets
|
661
|
661
|
||||||
Total assets
|
$
|
252,465
|
$
|
268,804
|
||||
LIABILITIES AND STOCKHOLDERS' EQUITY
|
||||||||
Current liabilities:
|
||||||||
Accounts payable
|
$
|
2,578
|
$
|
2,381
|
||||
Payable to Ionis Pharmaceuticals, Inc.
|
27,737
|
14,365
|
||||||
Accrued compensation
|
3,773
|
4,083
|
||||||
Accrued liabilities
|
13,413
|
7,570
|
||||||
Current portion of deferred revenue
|
48,866
|
58,192
|
||||||
Other current liabilities
|
1,929
|
1,875
|
||||||
Total current liabilities
|
98,296
|
88,466
|
||||||
Long-term portion of deferred rent
|
10
|
12
|
||||||
Long-term portion of deferred revenue
|
7,859
|
12,501
|
||||||
Total liabilities
|
106,165
|
100,979
|
||||||
Stockholders' equity:
|
||||||||
Common stock, $0.001 par value; 100,000,000 shares authorized at March 31, 2018 and December 31, 2017; 66,803,803 and 66,541,629 shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively
|
67
|
67
|
||||||
Additional paid-in capital
|
472,549
|
464,430
|
||||||
Accumulated other comprehensive loss
|
(468
|
)
|
(451
|
)
|
||||
Accumulated deficit
|
(325,848
|
)
|
(296,221
|
)
|
||||
Total stockholders' equity
|
146,300
|
167,825
|
||||||
Total liabilities and stockholders' equity
|
$
|
252,465
|
$
|
268,804
|
Three Months Ended
March 31,
|
||||||||
|
2018
|
2017
|
||||||
(as revised)
|
||||||||
Revenue:
|
||||||||
Research and development revenue under collaborative agreement
|
$
|
17,108
|
$
|
6,094
|
||||
Total revenue
|
17,108
|
6,094
|
||||||
Expenses:
|
||||||||
Research and development
|
27,970
|
64,794
|
||||||
General and administrative
|
19,465
|
4,676
|
||||||
Total operating expenses
|
47,435
|
69,470
|
||||||
Loss from operations
|
(30,327
|
)
|
(63,376
|
)
|
||||
Other income (expense):
|
||||||||
Investment income
|
868
|
61
|
||||||
Interest expense
|
—
|
(541
|
)
|
|||||
Other expense
|
(168
|
)
|
—
|
|||||
Loss before income tax expense
|
(29,627
|
)
|
(63,856
|
)
|
||||
Income tax expense
|
—
|
—
|
||||||
Net loss
|
$
|
(29,627
|
)
|
$
|
(63,856
|
)
|
||
Net loss per share of preferred stock, basic and diluted
|
$
|
—
|
$
|
(2.21
|
)
|
|||
Weighted-average shares of preferred stock outstanding, basic and diluted
|
—
|
28,884,540
|
||||||
Net loss per share of common stock, basic and diluted
|
$
|
(0.44
|
)
|
$
|
—
|
|||
Weighted-average shares of common stock outstanding, basic and diluted
|
66,616,337
|
—
|
Three Months Ended
March 31,
|
||||||||
|
2018
|
2017
|
||||||
(as revised)
|
||||||||
Net loss
|
$
|
(29,627
|
)
|
$
|
(63,856
|
)
|
||
Unrealized gains (losses) on investments, net of tax
|
(45
|
)
|
(28
|
)
|
||||
Currency translation adjustment
|
28
|
6
|
||||||
Comprehensive loss
|
$
|
(29,644
|
)
|
$
|
(63,878
|
)
|
Three Months Ended
March 31,
|
||||||||
2018
|
2017
|
|||||||
(as revised)
|
||||||||
Operating activities:
|
||||||||
Net loss
|
$
|
(29,627
|
)
|
$
|
(63,856
|
)
|
||
Adjustments to reconcile net loss to net cash (used in) provided by operating activities:
|
||||||||
Depreciation
|
27
|
29
|
||||||
Amortization of licenses
|
30
|
30
|
||||||
Amortization of premium on investments, net
|
149
|
24
|
||||||
Non-cash interest expense for line of credit with Ionis Pharmaceuticals, Inc.
|
—
|
541
|
||||||
Stock-based compensation expense
|
6,384
|
3,180
|
||||||
Changes in operating assets and liabilities:
|
||||||||
Contracts receivable
|
5,413
|
—
|
||||||
Other current and long-term assets
|
(4,326
|
)
|
(1,915
|
)
|
||||
Prepaid amounts to Ionis Pharmaceuticals, Inc.
|
—
|
(3,005
|
)
|
|||||
Accounts payable
|
197
|
(459
|
)
|
|||||
Payable to Ionis Pharmaceuticals, Inc.
|
13,372
|
(9,355
|
)
|
|||||
Accrued compensation
|
(310
|
)
|
(1,658
|
)
|
||||
Deferred rent
|
(10
|
)
|
(9
|
)
|
||||
Accrued liabilities
|
5,843
|
331
|
||||||
Income taxes payable
|
63
|
(9
|
)
|
|||||
Deferred revenue
|
(13,968
|
)
|
102,301
|
|||||
Net cash (used in) provided by operating activities
|
(16,763
|
)
|
26,170
|
|||||
Investing activities:
|
||||||||
Purchases of short-term investments
|
(9,906
|
)
|
(49,465
|
)
|
||||
Proceeds from sale of short-term investments
|
36,447
|
2,750
|
||||||
Net cash (used in) provided by investing activities
|
26,541
|
(46,715
|
)
|
|||||
Financing activities:
|
||||||||
Proceeds from exercise of common stock options and employee stock purchase
plan issuances
|
1,724
|
—
|
||||||
Proceeds from line of credit from Ionis Pharmaceuticals, Inc.
|
—
|
91,000
|
||||||
Offering costs paid
|
—
|
(459
|
)
|
|||||
Net cash provided by financing activities
|
1,724
|
90,541
|
||||||
Effect of exchange rates on cash
|
38
|
5
|
||||||
Net increase in cash and cash equivalents
|
11,540
|
70,001
|
||||||
Cash and cash equivalents at beginning of period
|
58,367
|
7,857
|
||||||
Cash and cash equivalents at end of period
|
$
|
69,907
|
$
|
77,858
|
||||
Supplemental disclosures of non-cash financing activities:
|
||||||||
Unpaid deferred offering costs
|
$
|
450
|
$
|
319
|
1. |
Basis of Presentation and Organization
|
2. |
Summary of Significant Accounting Policies
|
December 31, 2017
(in thousands)
|
||||||||||||
|
As revised under Topic 606
|
As originally reported
under Topic 605
|
Effect
of change
|
|||||||||
Current portion of deferred revenue
|
$
|
58,192
|
$
|
50,579
|
$
|
7,613
|
||||||
Long
‑
term portion of deferred revenue
|
12,501
|
8,306
|
4,195
|
|||||||||
Accumulated deficit
|
$
|
(296,221
|
)
|
$
|
(284,413
|
)
|
$
|
(11,808
|
)
|
Three Months Ended
March 31, 2017
(in thousands, except per share data)
|
||||||||||||
|
As revised
under Topic 606
|
As originally reported
under Topic 605
|
Effect
of change
|
|||||||||
Research and development revenue under collaborative agreements
|
$
|
6,094
|
$
|
9,597
|
$
|
(3,503
|
)
|
|||||
Loss from operations
|
(63,376
|
)
|
(59,873
|
)
|
(3,503
|
)
|
||||||
Net loss
|
(63,856
|
)
|
(60,353
|
)
|
(3,503
|
)
|
||||||
Net loss per share of preferred stock, basic and diluted
|
$
|
(2.21
|
)
|
$
|
(2.09
|
)
|
$
|
(0.12
|
)
|
Three Months Ended
March 31, 2017
(in thousands)
|
||||||||||||
|
As revised under Topic 606
|
As originally reported
under Topic 605
|
Effect
of change
|
|||||||||
Net loss
|
$
|
(63,856
|
)
|
$
|
(60,353
|
)
|
$
|
(3,503
|
)
|
|||
Adjustments to reconcile net loss to net cash provided by operating activities:
|
||||||||||||
Deferred revenue
|
102,301
|
98,798
|
3,503
|
|||||||||
Cash and cash equivalents at beginning of period
|
7,857
|
7,857
|
—
|
|||||||||
Cash and cash equivalents at end of period
|
$
|
77,858
|
$
|
77,858
|
$
|
—
|
3. |
Investments and Fair Value Measurements
|
Gross Unrealized
|
Estimated
|
|||||||||||||||
March 31, 2018
|
Cost
|
Gains
|
Losses
|
Fair Value
|
||||||||||||
Available-for-sale securities:
|
||||||||||||||||
Corporate debt securities
|
$
|
101,180
|
$
|
—
|
$
|
(220
|
)
|
$
|
100,960
|
|||||||
Debt securities issued by U.S. government agencies
|
68,782
|
—
|
(135
|
)
|
68,647
|
|||||||||||
Total securities with a maturity of one year or less
|
169,962
|
—
|
(355
|
)
|
169,607
|
|||||||||||
Corporate debt securities
|
2,919
|
—
|
(23
|
)
|
2,896
|
|||||||||||
Debt securities issued by U.S. government agencies
|
2,529
|
—
|
(4
|
)
|
2,525
|
|||||||||||
Total securities with a maturity of one to two years
|
5,448
|
—
|
(27
|
)
|
5,421
|
|||||||||||
Total available-for-sale securities
|
$
|
175,410
|
$
|
—
|
$
|
(382
|
)
|
$
|
175,028
|
Gross Unrealized
|
Estimated
|
|||||||||||||||
December 31, 2017
|
Cost
|
Gains
|
Losses
|
Fair Value
|
||||||||||||
Available-for-sale securities:
|
||||||||||||||||
Corporate debt securities
|
$
|
132,434
|
$
|
—
|
$
|
(206
|
)
|
$
|
132,228
|
|||||||
Debt securities issued by U.S. government agencies
|
38,135
|
—
|
(59
|
)
|
38,076
|
|||||||||||
Total securities with a maturity of one year or less
|
170,569
|
—
|
(265
|
)
|
170,304
|
|||||||||||
Corporate debt securities
|
8,267
|
—
|
(35
|
)
|
8,232
|
|||||||||||
Debt securities issued by U.S. government agencies
|
23,264
|
—
|
(37
|
)
|
23,227
|
|||||||||||
Total securities with a maturity of one to two years
|
31,531
|
—
|
(72
|
)
|
31,459
|
|||||||||||
Total available-for-sale securities
|
$
|
202,100
|
$
|
—
|
$
|
(337
|
)
|
$
|
201,763
|
|
At
March 31, 2018
|
Quoted Prices
in Active Markets
(Level 1)
|
Significant Other
Observable Inputs
(Level 2)
|
|||||||||
Cash equivalents (1)
|
$
|
65,963
|
$
|
65,963
|
$
|
—
|
||||||
Corporate debt securities (2)
|
103,856
|
—
|
103,856
|
|||||||||
Debt securities issued by U.S. government agencies (2)
|
71,172
|
—
|
71,172
|
|||||||||
Total
|
$
|
240,991
|
$
|
65,963
|
$
|
175,028
|
|
At
December 31, 2017
|
Quoted Prices
in Active Markets
(Level 1)
|
Significant Other
Observable Inputs
(Level 2)
|
|||||||||
Cash equivalents (1)
|
$
|
48,430
|
$
|
48,430
|
$
|
—
|
||||||
Corporate debt securities (2)
|
140,460
|
—
|
140,460
|
|||||||||
Debt securities issued by U.S. government agencies (2)
|
61,303
|
—
|
61,303
|
|||||||||
Total
|
$
|
250,193
|
$
|
48,430
|
$
|
201,763
|
(1) |
Included in cash and cash equivalents on our condensed consolidated balance sheets.
|
(2) |
Included in short-term investments on our condensed consolidated balance sheets.
|
4. |
Strategic Collaboration with Novartis
|
● |
Development activities for AKCEA-APO(a)-L
Rx
;
|
● |
Development activities for AKCEA-APOCIII-L
Rx
;
|
● |
API for AKCEA-APO(a)-L
Rx
; and
|
● |
API for AKCEA-APOCIII-L
Rx
.
|
● |
$75.0 million from the upfront payment we received;
|
● |
$28.4 million for the premium paid by Novartis, which represents the excess of the fair value Ionis received from Novartis' purchase of Ionis' stock at a premium in the first quarter of 2017; and
|
● |
$5.0 million for the premium Novartis would have paid to purchase Ionis' stock if we did not complete our IPO within 15 months of the inception of the agreement.
|
● |
$64.0 million for development services for AKCEA-APO(a)-L
Rx
;
|
● |
$40.1 million for development services for AKCEA-APOCIII-L
Rx
;
|
● |
$1.5 million for the delivery of AKCEA-APO(a)-L
Rx
API; and
|
● |
$2.8 million for the delivery of AKCEA-APOCIII-L
Rx
API.
|
● |
We will satisfy the development services performance obligation for AKCEA-APO(a)-L
Rx
as the research and development services are performed. We determined that the period of performance of the research and development services was two years, or through December 2018. We recognize revenue related to research and development services performed using an input method by calculating costs incurred at each period end relative to total costs expected to be incurred;
|
● |
We will satisfy the development services performance obligation for AKCEA-APOCIII-L
Rx
as the research and development services are performed. We determined that the period of performance of the research and development services was two and a half years, or through June 2019. We recognize revenue related to research and development services performed using an input method by calculating costs incurred at each period end relative to total costs expected to be incurred;
|
● |
We will recognize the amount attributed to the AKCEA-APO(a)-L
Rx
API supply when we deliver API to Novartis; and
|
● |
We recognized the amount attributed to the AKCEA-APOCIII-L
Rx
API supply when we delivered API to Novartis in 2017.
|
5. |
Development, Commercialization and License Agreement and Services Agreement with Ionis
|
● |
investor relations services,
|
● |
human resources and personnel services,
|
● |
risk management and insurance services,
|
● |
tax related services,
|
● |
corporate record keeping services,
|
● |
financial and accounting services,
|
● |
credit services, and
|
● |
COO/CFO/CBO oversight.
|
Three Months Ended
March 31,
|
||||||||
|
2018
|
2017
|
||||||
Services performed by Ionis
|
$
|
1,945
|
$
|
2,957
|
||||
Active pharmaceutical ingredient manufactured by Ionis
|
5,229
|
3,083
|
||||||
Sublicensing expenses
|
—
|
48,394
|
||||||
Out-of-pocket expenses paid by Ionis
|
6,236
|
8,868
|
||||||
Total expenses generated by transactions with Ionis
|
13,410
|
63,302
|
||||||
Payable balance to Ionis at the beginning of the period
|
14,365
|
24,355
|
||||||
Prepaid amounts to Ionis
|
—
|
3,005
|
||||||
Less: total amounts paid to Ionis during the period
|
(38
|
)
|
(42,268
|
)
|
||||
Less: non-cash sublicensing expenses
|
—
|
(33,394
|
)
|
|||||
Total amount payable to Ionis at period end
|
$
|
27,737
|
$
|
15,000
|
Three Months Ended
March 31,
|
||||||||
|
2018
|
2017
|
||||||
Research and development expenses
|
$
|
2,314
|
$
|
1,600
|
||||
General and administrative expenses
|
4,070
|
1,580
|
||||||
Total
|
$
|
6,384
|
$
|
3,180
|
7. |
Accumulated Other Comprehensive Loss
|
|
2018
|
|||
Balance, as of December 31, 2017
|
$
|
(451
|
)
|
|
Unrealized gains (losses) on investments, net of tax (1)
|
(45
|
)
|
||
Currency translation adjustment
|
28
|
|||
Net other comprehensive income (loss)
|
(17
|
)
|
||
Balance, as of March 31, 2018
|
$
|
(468
|
)
|
(1) |
There was no tax benefit for other comprehensive income (loss) for the three months ended March 31, 2018.
|
8. |
Basic and Diluted Net Loss Per Share
|
Three Months Ended
March 31,
|
||||||||
|
2018
|
2017
|
||||||
Losses allocated to preferred shares
|
$
|
—
|
$
|
(63,856
|
)
|
|||
Weighted-average preferred shares outstanding
|
—
|
28,884,540
|
||||||
Basic loss per preferred share
|
$
|
—
|
$
|
(2.21
|
)
|
|||
Losses allocated to common shares
|
$
|
(29,627
|
)
|
$
|
—
|
|||
Weighted-average common shares outstanding
|
66,616,337
|
—
|
||||||
Basic loss per common share
|
$
|
(0.44
|
)
|
$
|
—
|
● |
Options to purchase common stock;
|
● |
Unvested restricted stock units; and
|
● |
Employee Stock Purchase Plan, or ESPP.
|
9. |
Initial Public Offering
|
● |
$132.3 million from the sale of 17,968,750 shares of our common stock in our IPO of which $25 million was invested by Ionis; and
|
● |
$50.0 million from the purchase of 6,250,000 shares by Novartis in a concurrent private placement.
|
● |
the conversion of all outstanding shares of Series A convertible preferred stock into 28,884,540 shares of our common stock; and
|
● |
the conversion of $106.0 million of outstanding principal plus accrued interest from the line of credit into 13,438,339 shares of common stock.
|
10. |
Subsequent Events
|
● |
commercialize inotersen following receipt of regulatory approval and perform certain other non-commercial activities with respect to inotersen, in each case, in accordance with a global strategic plan;
|
● |
partner on the completion of all pivotal studies, of a follow-on drug to inotersen, AKCEA-TTR-L
Rx
and perform other non-commercial activities with respect to AKCEA-TTR-L
Rx
;
|
● |
commercialize AKCEA-TTR-L
Rx
, following receipt of regulatory approval in accordance with a global strategic plan;
|
● |
share in profits and losses with respect to inotersen and AKCEA-TTR-L
Rx
;
|
● |
manufacture (including through a third party) each product following receipt of regulatory approval for such product; and
|
● |
sublicense the development and commercialization of either product to third parties or affiliates, with the consent of Ionis.
|
ITEM 2 |
MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
|
● |
Announcement and closing of the partnership with Ionis to commercialize the TTR franchise.
|
● |
Addition of Sarah Boyce to Akcea as president and a member of our board of directors.
|
● |
Initiation of the volanesorsen global Early Access Program including initiation of the Early Access to Medicines Scheme, or EAMS, in the UK for the treatment FCS.
|
● |
Presentation of multiple data sets at the International Symposium on Amyloidosis, or ISA, including the NEURO-TTR and Open Label Extension Studies for TEGSEDI and the preclinical data set for AKCEA-TTR-L
Rx
.
|
● |
Presentation of AKCEA-APOCIII-L
Rx
Phase 1/2 results at the American College of Cardiology, or ACC, Annual Scientific Session and Expo.
|
● |
Convening of the first FCS global connection summit where the patient leaders named the first Friday in November as FCS Awareness Day.
|
December 31, 2017
(in thousands)
|
||||||||||||
|
As revised under Topic 606
|
As originally reported
under Topic 605
|
Effect
of change
|
|||||||||
Current portion of deferred revenue
|
$
|
58,192
|
$
|
50,579
|
$
|
7,613
|
||||||
Long
‑
term portion of deferred revenue
|
12,501
|
8,306
|
4,195
|
|||||||||
Accumulated deficit
|
$
|
(296,221
|
)
|
$
|
(284,413
|
)
|
$
|
(11,808
|
)
|
Three Months Ended
March 31, 2017
(in thousands, except per share data)
|
||||||||||||
|
As revised
under Topic 606
|
As originally reported
under Topic 605
|
Effect
of change
|
|||||||||
Research and development revenue under collaborative agreements
|
$
|
6,094
|
$
|
9,597
|
$
|
(3,503
|
)
|
|||||
Loss from operations
|
(63,376
|
)
|
(59,873
|
)
|
(3,503
|
)
|
||||||
Net loss
|
(63,856
|
)
|
(60,353
|
)
|
(3,503
|
)
|
||||||
Net loss per share of preferred stock, basic and diluted
|
$
|
(2.21
|
)
|
$
|
(2.09
|
)
|
$
|
(0.12
|
)
|
Three Months Ended
March 31, 2017
(in thousands)
|
||||||||||||
|
As revised under Topic 606
|
As originally reported
under Topic 605
|
Effect
of change
|
|||||||||
Net loss
|
$
|
(63,856
|
)
|
$
|
(60,353
|
)
|
$
|
(3,503
|
)
|
|||
Adjustments to reconcile net loss to net cash provided by operating activities:
|
||||||||||||
Deferred revenues
|
102,301
|
98,798
|
3,503
|
|||||||||
Cash and cash equivalents at beginning of period
|
7,857
|
7,857
|
—
|
|||||||||
Cash and cash equivalents at end of period
|
$
|
77,858
|
$
|
77,858
|
$
|
—
|
Three Months Ended
March 31,
|
||||||||
|
2018
|
2017
|
||||||
External volanesorsen expenses
|
$
|
6,559
|
$
|
5,603
|
||||
Other external research and development project expenses
|
13,126
|
4,910
|
||||||
Sublicensing expenses
|
—
|
48,394
|
||||||
Research and development personnel and overhead expenses
|
5,971
|
4,287
|
||||||
Total research and development expenses, excluding non-cash stock-based compensation expense
|
25,656
|
63,194
|
||||||
Non-cash stock-based compensation expense
|
2,314
|
1,600
|
||||||
Total research and development expenses
|
$
|
27,970
|
$
|
64,794
|
Three Months Ended
March 31,
|
||||||||
|
2018
|
2017
|
||||||
General and administrative support expenses
|
$
|
5,487
|
$
|
1,578
|
||||
Pre-commercialization expenses for volanesorsen
|
9,909
|
1,518
|
||||||
Total general and administrative expenses, excluding non-cash stock-based compensation expense
|
15,396
|
3,096
|
||||||
Non-cash stock-based compensation expense
|
4,069
|
1,580
|
||||||
Total general and administrative expenses
|
$
|
19,465
|
$
|
4,676
|
● |
the design, initiation, progress, size, timing, costs and results of our clinical and nonclinical studies;
|
● |
the outcome, timing and cost of regulatory approvals by the FDA and comparable foreign regulatory authorities, including the potential for the FDA or comparable foreign regulatory authorities to require that we perform more studies than, or evaluate clinical endpoints other than, those that we currently expect;
|
● |
the number and characteristics of drugs that we may pursue;
|
● |
our need to expand our development activities, including our need and ability to hire additional employees;
|
● |
the effect of competing technological and market developments;
|
● |
the cost of establishing sales, marketing, manufacturing and distribution capabilities for our drugs;
|
● |
our strategic collaborators' success in developing and commercializing our drugs;
|
● |
our need to add infrastructure, implement internal systems and hire additional employees to operate as a public company; and
|
● |
the revenue, if any, generated from commercial sales of our drugs for which we receive marketing authorization, which may be affected by market conditions, including obtaining coverage and adequate reimbursement of our drugs from third-party payors, including government programs and managed care organizations, and competition within the therapeutic class to which our drugs are assigned.
|
ITEM 3. |
QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
|
ITEM 4. |
CONTROLS AND PROCEDURES
|
ITEM 1. |
LEGAL PROCEEDINGS
|
ITEM 1A. |
RISK FACTORS
|
● |
completing clinical development of volanesorsen and inotersen for additional indications and nonclinical and clinical development of AKCEA-APO(a)-L
Rx
, AKCEA-TTR-L
Rx
, AKCEA-ANGPTL3-L
Rx
and AKCEA-APOCIII-L
Rx
;
|
● |
seeking and obtaining regulatory and marketing authorization for our drugs, including volanesorsen, inotersen, AKCEA-APO(a)-L
Rx
and our other drugs in development;
|
● |
establishing and maintaining supply and manufacturing relationships with third parties that can provide the amount and quality of products and services we need to continue to develop and, if approved, commercialize volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
and our other drugs in development;
|
● |
launching and commercializing volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
by establishing a sales, marketing and distribution infrastructure;
|
● |
launching and co-commercializing AKCEA-APO(a)-L
Rx
and AKCEA-APOCIII-L
Rx
through our collaboration with Novartis Pharma AG, or Novartis, under terms that we plan to negotiate with Novartis in the future;
|
● |
educating physicians about our target patient populations, including patients with familial chylomicronemia syndrome, or FCS, and patients with familial partial lipodystrophy, or FPL;
|
● |
obtaining market acceptance of volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
and our other drugs in development as viable treatment options;
|
● |
obtaining and maintaining adequate coverage and reimbursement from third-party payors for volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
and our other drugs in development;
|
● |
addressing any competing technological and market developments;
|
● |
implementing additional internal systems and infrastructure, as needed, to ultimately operate without reliance on Ionis;
|
● |
negotiating favorable terms in any partnership, licensing or other arrangements into which we may enter;
|
● |
maintaining, protecting and expanding our portfolio of intellectual property rights, including patents, product trademarks and know-how;
|
● |
developing and commercializing volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
and our other drugs in development without infringing others' intellectual property rights; and
|
● |
attracting, hiring and retaining qualified personnel.
|
● |
the clinical study may produce negative or inconclusive results;
|
● |
regulators may require that we hold, suspend or terminate clinical research for noncompliance with regulatory requirements;
|
● |
we, our partners, the FDA or foreign regulatory authorities could suspend or terminate a clinical study due to adverse side effects of a drug on people in the study;
|
● |
we or our partners may decide, or regulators may require us, to conduct additional preclinical testing or clinical studies;
|
● |
we or our partners may not identify, recruit and train suitable clinical investigators at a sufficient number of study sites;
|
● |
the institutional review board for a prospective site might withhold or delay its approval for the study;
|
● |
enrollment in our clinical studies may be slower than we anticipate;
|
● |
patients who enroll in the clinical study may later drop out due to adverse events, a perception they are not benefiting from participating in the study, fatigue with the clinical study process or personal issues;
|
● |
a clinical study site may deviate from the protocol for the study;
|
● |
the cost of our clinical studies may be greater than we anticipate;
|
● |
we or our partners may require additional capital to fund the clinical study; and
|
● |
the supply or quality of our drugs or other materials necessary to conduct the clinical studies may be insufficient, inadequate or delayed.
|
● |
such authorities may disagree with the design or implementation of our clinical studies;
|
● |
we or our partners may be unable to demonstrate to the satisfaction of the FDA or other regulatory authorities that a drug is safe and effective for any indication;
|
● |
such authorities may not accept clinical data from studies conducted at clinical facilities that have deficient clinical practices or that are in countries where the standard of care is potentially different from the United States;
|
● |
we or our partners may be unable to demonstrate that our drug's clinical and other benefits outweigh its safety risks to support approval;
|
● |
such authorities may disagree with the interpretation of data from preclinical or clinical studies;
|
● |
such authorities may find deficiencies in the manufacturing processes or facilities of third-party manufacturers who manufacture clinical and commercial supplies for our drugs; and
|
● |
the approval policies or regulations of such authorities or their prior guidance to us or our partners during clinical development may significantly change in a manner rendering our clinical data insufficient for approval.
|
● |
restrictions on our ability to conduct clinical studies, including full or partial clinical holds on ongoing or planned clinical studies;
|
● |
restrictions on such products' manufacturing processes;
|
● |
changes to the product label;
|
● |
restrictions on the marketing of a product;
|
● |
restrictions on product distribution;
|
● |
requirements to conduct post-marketing clinical studies;
|
● |
Untitled or Warning Letters;
|
● |
withdrawal of the products from the market;
|
● |
refusal to approve pending applications or supplements to approved applications that we submit;
|
● |
recall of products;
|
● |
fines, restitution or disgorgement of profits or revenue;
|
● |
suspension or withdrawal of regulatory approvals;
|
● |
refusal to permit the import or export of our products;
|
● |
product seizure;
|
● |
injunctions; or
|
● |
imposition of civil or criminal penalties.
|
● |
not provide us with accurate or timely information regarding their inventories, the number of patients who are using our drugs or complaints regarding our drugs;
|
● |
not effectively sell or support volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
or our other drugs;
|
● |
reduce or discontinue their efforts to sell or support volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
or our other drugs;
|
● |
not devote the resources necessary to sell volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
or our other drugs in the volumes and within the time frames that we expect;
|
● |
not satisfy financial obligations to us or others; or
|
● |
cease operations.
|
● |
receipt and scope of marketing authorizations;
|
● |
establishment and demonstration in the medical and patient community of the efficacy and safety of our drugs and their potential advantages over competing products;
|
● |
cost and effectiveness of our drugs compared to other available therapies;
|
● |
patient convenience of the dosing regimen for our drugs; and
|
● |
reimbursement by government and third-party payors.
|
● |
safer than our drugs;
|
● |
more effective than our drugs;
|
● |
priced lower than our drugs;
|
● |
reimbursed more favorably by government and other third-party payors than our drugs; or
|
● |
more convenient to use than our drugs.
|
● |
criminal and civil sanctions, including fines and civil monetary penalties;
|
● |
the possibility of exclusion from federal healthcare programs, including Medicare and Medicaid; and
|
● |
corporate integrity agreements, which could impose rigorous operational and monitoring requirements on us.
|
● |
conduct the cardiovascular outcome studies that are likely to be required for approval of AKCEA-APO(a)-L
Rx
and AKCEA-APOCIII-L
Rx;
|
● |
seek and obtain regulatory approvals for AKCEA-APO(a)-L
Rx
and AKCEA-APOCIII-L
Rx
; and
|
● |
globally commercialize AKCEA-APO(a)-L
Rx
and AKCEA-APOCIII-L
Rx
.
|
● |
any determination with respect to our business strategy and policies, including the appointment and removal of officers and directors;
|
● |
any determinations with respect to mergers, business combinations or disposition of assets;
|
● |
our financing and dividend policy;
|
● |
compensation and benefit programs and other human resources policy decisions;
|
● |
termination of, changes to or determinations under our existing license agreements and services agreement with Ionis;
|
● |
changes to any other agreements that may adversely affect us; and
|
● |
determinations with respect to our tax returns.
|
● |
A board of directors having a majority of independent directors;
|
● |
A compensation committee composed entirely of independent directors that approves the compensation payable to the company's chief executive officer and other executive officers; and
|
● |
A nominating committee composed entirely of independent directors that nominates candidates for election to the board of directors, or that recommends such candidates for nomination by the board of directors (or obligating the listed company to cause a majority of the board's independent directors to exercise this oversight of director nominations).
|
● |
a Joint Steering Committee, or JSC, having equal membership from us and Ionis, sets the development strategy for our drugs by mutual agreement. A Regulatory Sub-committee, established by the JSC and having equal membership from our company and Ionis, will set the regulatory strategy for each of our drugs by mutual agreement. If the JSC or the Regulatory Sub-committee cannot come to a mutual agreement, then this could delay our ability to develop and commercialize inotersen and AKCEA-TTR-L
Rx
. In the event of a disagreement at the JSC, Ionis has final decision-making authority on decisions relating to development matters, Akcea has final decision making authority on decision relating to commercial matters, and the holder of the regulatory approvals for a product in a country has final decision making authority for regulatory affairs;
|
● |
we will need Ionis' consent prior to granting any sublicense to a third party for inotersen or AKCEA-TTR-LRx. If Ionis does not grant such consent with respect to a sublicense, then we would not be able to enter into such arrangement, which could delay or prevent our ability to develop and commercialize inotersen and AKCEA-TTR-L
Rx
;
|
● |
we will need to obtain Ionis' approval to in-license a product, acquire a product or acquire another company, until the time Ionis ceases to hold at least 50% of our outstanding capital stock;
|
● |
we only have the right to lead the prosecution and enforcement of certain of the patent rights licensed to us under our inotersen licensing agreement, so called product-specific patent rights. Ionis will control the prosecution and enforcement of other patent rights licensed to us, and they may do so in a manner that does not advance or is inconsistent with our interests; and
|
● |
although our agreements with Ionis prohibit Ionis from developing and commercializing drugs that modulate TTR via the binding of such drug to the RNA that encodes TTR using the technology licensed to us under our inotersen licensing agreement, Ionis is free to pursue other products that treat the same indications that would be treated by inotersen and AKCEA-TTR-L
Rx
.
|
● |
a Joint Steering Committee, or JSC, comprising two senior members from our company and two senior members from Ionis, sets the development strategy for our drugs by mutual agreement. A Regulatory Sub-committee, established by the JSC and having equal membership from our company and Ionis, will set the regulatory strategy for each of our drugs by mutual agreement. If the JSC or the Regulatory Sub-committee cannot come to a mutual agreement, it could delay our ability to develop and commercialize volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
and our other drugs in development;
|
● |
we will need to mutually agree with Ionis on the terms of any additional sublicense to a third party for volanesorsen and our other drugs in development. If we cannot mutually agree on terms for a sublicense to a third party or if Ionis does not agree to a sublicense at all, it could delay or prevent our ability to develop and commercialize volanesorsen and our other drugs in development;
|
● |
we will need to obtain Ionis' approval to in-license a product, acquire a product or acquire another company, until the time Ionis ceases to hold at least 50% of our outstanding capital stock; and
|
● |
there is nothing in our agreements with Ionis to prevent Ionis from developing and commercializing drugs targeting RNAs that are not apoC-III, Apo(a) or ANGPTL3 to pursue the same indications we are pursuing with our drugs.
|
● |
issued patent claims to the specific antisense sequence and chemical composition of volanesorsen in the United States, Australia, and Europe;
|
● |
issued patent claims in the United States and Australia drawn to the use of antisense compounds complementary to an active region of human apoC-III messenger ribonucleic acid, including the site targeted by volanesorsen;
|
● |
additional patent applications designed to protect the volanesorsen composition in Canada; and
|
● |
additional methods of use in jurisdictions worldwide for volanesorsen.
|
● |
others may make compounds that are similar to our drugs but that are not covered by the claims of the patents that we own or have exclusively licensed;
|
● |
we, or our license partners or current or future strategic partners, might not have been the first to make the inventions covered by the issued patent or pending patent application that we own or have exclusively licensed;
|
● |
we, or our license partners or current or future strategic partners, might not have been the first to file patent applications covering our inventions;
|
● |
others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our intellectual property rights;
|
● |
our pending licensed patent applications or those that we own in the future may not lead to issued patents;
|
● |
issued patents that we hold rights to may be held invalid or unenforceable, including as a result of legal challenges by our competitors;
|
● |
our competitors might conduct research and development activities 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;
|
● |
we may not develop additional proprietary technologies that are patentable; and
|
● |
the patents of others may have an adverse effect on our business.
|
● |
manage our clinical studies and the regulatory process effectively;
|
● |
manage the manufacturing of our drugs for clinical and commercial use;
|
● |
integrate current and additional management, administrative, financial and sales and marketing personnel;
|
● |
develop a marketing and sales infrastructure;
|
● |
hire new personnel necessary to effectively commercialize volanesorsen and our other drugs in development;
|
● |
develop our administrative, accounting and management information systems and controls; and
|
● |
hire and train additional qualified personnel.
|
● |
interruption of our development, manufacturing and distribution efforts;
|
● |
injury to our employees and others;
|
● |
environmental damage resulting in costly clean up; and
|
● |
liabilities under federal, state and local laws and regulations governing health and human safety, as well as the use, storage, handling and disposal of these materials and resultant waste products.
|
● |
compliance with differing or unexpected regulatory requirements for our drugs and foreign employees;
|
● |
complexities associated with managing multiple payor reimbursement regimes, government payors or patient self-pay systems;
|
● |
difficulties in staffing and managing foreign operations;
|
● |
in certain circumstances, increased dependence on the commercialization efforts and regulatory compliance of third-party distributors or strategic partners;
|
● |
foreign government taxes, regulations and permit requirements;
|
● |
U.S. and foreign government tariffs, trade restrictions, price and exchange controls and other regulatory requirements;
|
● |
anti-corruption laws, including the Foreign Corrupt Practices Act, or the FCPA, and its equivalent in foreign jurisdictions;
|
● |
economic weakness, including inflation, natural disasters, war, events of terrorism or political instability in particular foreign countries;
|
● |
fluctuations in currency exchange rates, which could result in increased operating expenses and reduced revenue, and other obligations related to doing business in another country;
|
● |
compliance with tax, employment, privacy, immigration and labor laws, regulations and restrictions for employees living or traveling abroad;
|
● |
workforce uncertainty in countries where labor unrest is more common than in the United States; and
|
● |
changes in diplomatic and trade relationships.
|
● |
our failure to effectively develop and commercialize volanesorsen and our other drugs in development;
|
● |
Novartis' failure to exercise its option and/or effectively develop and commercialize AKCEA-APO(a)-L
Rx
and AKCEA-APOCIII-L
Rx
to the extent it exercises its option to license those drugs from us;
|
● |
changes in the market's expectations about our operating results;
|
● |
adverse results or delays in preclinical or clinical studies;
|
● |
our decision to initiate a clinical study, not to initiate a clinical study or to terminate an existing clinical study;
|
● |
adverse regulatory decisions, including failure to receive regulatory approval for volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
and our other drugs in development;
|
● |
success or failure of competitive products or antisense drugs more generally;
|
● |
adverse developments concerning our manufacturers or our strategic partnerships;
|
● |
inability to obtain adequate product supply for any drug for clinical studies or commercial sale or inability to do so at acceptable prices;
|
● |
the termination of a strategic partnership or the inability to establish additional strategic partnerships;
|
● |
unanticipated serious safety concerns related to the use of volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
and our other drugs in development;
|
● |
adverse safety or other clinical results, such as those that have occurred in the past or that may occur in the future, related to drugs being developed by Ionis or other companies that are or may be perceived to be similar to our drugs;
|
● |
our ability to effectively manage our growth;
|
● |
the size and growth, if any, of the targeted market;
|
● |
our operating results do not meet the expectation of securities analysts or investors in a particular period;
|
● |
actual or anticipated fluctuations in our quarterly financial results or the quarterly financial results of companies perceived to be similar to us;
|
● |
securities analysts do not publish reports about us or our business or publish negative reports;
|
● |
changes in financial estimates and recommendations by securities analysts concerning our company, our market opportunity, or the biotechnology and pharmaceutical industries in general;
|
● |
operating and stock price performance of other companies that investors deem comparable to us;
|
● |
overall performance of the equity markets;
|
● |
announcements by us or our competitors of acquisitions, new drugs or programs, significant contracts, commercial relationships or capital commitments;
|
● |
our and our strategic partners' ability to successfully market volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
and our other drugs in development;
|
● |
changes in laws and regulations affecting our business, including but not limited to clinical study requirements for approvals;
|
● |
disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain and maintain patent protection for volanesorsen, inotersen, AKCEA-APO(a)-L
Rx,
AKCEA-TTR-L
Rx
and our other drugs in development;
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commencement of, or involvement in, litigation involving our company, our general industry, or both;
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changes in our capital structure, such as future issuances of securities or the incurrence of additional debt;
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the volume of shares of our common stock available for public sale;
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additions or departures of key scientific or management personnel;
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any major change in our board or management;
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changes in accounting practices;
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ineffectiveness of our internal control over financial reporting;
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significant changes in our relationship with Ionis;
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sales of substantial amounts of common stock by our directors, executive officers or significant stockholders or the perception that such sales could occur; and
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general economic and political conditions such as recessions, interest rates, fuel prices, elections, drug pricing policies, international currency fluctuations and acts of war or terrorism.
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authorize "blank check" preferred stock, which could be issued by our board of directors without stockholder approval and may contain voting, liquidation, dividend and other rights superior to our common stock;
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specify that only board of directors or holders of greater than 10% of our common stock can call special meetings of our stockholders;
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prohibit stockholder action by written consent once Ionis no longer holds a majority of our voting power;
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establish an advance notice procedure for stockholder approvals to be brought before an annual meeting of our stockholders, including proposed nominations of persons for election to our board of directors;
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provide that a majority of directors then in office, even though less than a quorum, may fill vacancies on our board of directors;
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specify that no stockholder is permitted to cumulate votes at any election of directors;
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expressly authorize our board of directors to modify, alter or repeal our amended and restated bylaws; and
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require supermajority votes of the holders of our common stock to amend specified provisions of our amended and restated certificate of incorporation and amended and restated bylaws.
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derivative action or proceeding brought on our behalf;
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action asserting a claim of breach of a fiduciary duty owed by any of our directors, officers or other employees to us or our stockholders;
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action asserting a claim against us arising pursuant to any provision of the Delaware General Corporation Law, our amended and restated certificate of incorporation or our amended and restated bylaws; or
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other action asserting a claim against us that is governed by the internal affairs doctrine.
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ITEM 2. |
UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS
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ITEM 3. |
DEFAULT UPON SENIOR SECURITIES
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ITEM 4. |
MINE SAFETY DISCLOSURES
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ITEM 5. |
OTHER INFORMATION
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ITEM 6. |
EXHIBITS
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(1) |
Previously filed as Exhibit 3.2 to the Registrant's Current Report on Form 8-K (File No. 001-38137), filed with the Securities and Exchange Commission on July 19, 2017, and incorporated herein by reference.
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(2) |
Previously filed as Exhibit 4.1 to the Registrant's Current Report on Form 8-K (File No. 001-38137), filed with the Securities and Exchange Commission on March 15, 2018, and incorporated herein by reference.
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(3) |
Previously filed as Exhibit 10.1 to the Registrant's Current Report on Form 8-K (File No. 001-38137), filed with the Securities and Exchange Commission on March 15, 2018, and incorporated herein by reference.
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(4) |
Previously filed as Exhibit 10.2 to the Registrant's Current Report on Form 8-K (File No. 001-38137), filed with the Securities and Exchange Commission on March 15, 2018, and incorporated herein by reference.
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(5) |
Previously filed as Exhibit 10.3 to the Registrant's Current Report on Form 8-K (File No. 001-38137), filed with the Securities and Exchange Commission on March 15, 2018, and incorporated herein by reference.
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(6) |
Previously filed as Exhibit 10.4 to the Registrant's Current Report on Form 8-K (File No. 001-38137), filed with the Securities and Exchange Commission on March 15, 2018, and incorporated herein by reference.
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Signatures
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Title
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Date
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/s/ PAULA SOTEROPOULOS
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Chief Executive Officer
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Paula Soteropoulos
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(on behalf of the Registrant and in her capacity as principal executive officer)
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May 4, 2018
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/s/ MICHAEL MACLEAN
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Chief Financial Officer
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Michael MacLean
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(on behalf of the Registrant and in his capacity as principal financial and accounting officer)
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May 4, 2018
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By:
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/s/ Paula Soteropoulos
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Name:
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Paula Soteropoulos
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Title:
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Chief Executive Officer
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AKCEA THERAPEUTICS, INC.
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By:
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/s/ Elizabeth L. Hougen
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Name:
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Elizabeth L. Hougen
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Title:
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Chief Financial Officer
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1. |
General
.
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2. |
Administration
.
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3. |
Shares Subject to the Plan
.
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4. |
Eligibility
.
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5. |
Option Provisions
.
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6. |
Provisions of Stock Awards other than Options
.
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7. |
Covenants of the Company
.
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8. |
Miscellaneous
.
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9. |
Adjustments upon Changes in Common Stock; Other Corporate Events
.
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10. |
Termination or Suspension of the Plan
.
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11. |
Effective Date of Plan
.
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12. |
Choice of Law
.
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13.
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Definitions
. As used in the Plan, the following definitions shall apply to the capitalized terms indicated below:
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1. |
I have reviewed this Quarterly Report on Form 10-Q of Akcea Therapeutics, Inc.;
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2. |
Based on my knowledge, this quarterly 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 quarterly report;
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3. |
Based on my knowledge, the condensed consolidated financial statements, and other financial information included in this quarterly report, fairly present in all material respects the financial condition, condensed consolidated results of operations and condensed consolidated cash flows of the registrant as of, and for, the periods presented in this quarterly report;
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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)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
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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;
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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;
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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
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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
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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 registrant’s board of directors (or persons performing the equivalent functions):
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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
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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.
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Dated: May 4, 2018
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/s/ PAULA SOTEROPOULOS
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Paula Soteropoulos
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Chief Executive Officer
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1. |
I have reviewed this Quarterly Report on Form 10-Q of Akcea Therapeutics, Inc.;
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2. |
Based on my knowledge, this quarterly 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 quarterly report;
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3. |
Based on my knowledge, the condensed consolidated financial statements, and other financial information included in this quarterly report, fairly present in all material respects the financial condition, condensed consolidated results of operations and condensed consolidated cash flows of the registrant as of, and for, the periods presented in this quarterly report;
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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)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
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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;
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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;
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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
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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
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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 registrant’s board of directors (or persons performing the equivalent functions):
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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
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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.
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Dated: May 4, 2018
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/s/ MICHAEL MACLEAN
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Michael MacLean
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Chief Financial Officer
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1. |
The Company’s Quarterly Report on Form 10-Q for the period ended March 31, 2018, to which this Certification is attached as Exhibit 32.1 (the “Periodic Report”), fully complies with the requirements of Section 13(a) or Section 15(d) of the Securities Exchange Act of 1934, as amended; and
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2. |
The information contained in the Periodic Report fairly presents, in all material respects, the financial condition of the Company at the end of the period covered by the Periodic Report and the results of operations of the Company for the period covered by the Periodic Report.
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Dated: May 4, 2018
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/s/ PAULA SOTEROPOULOS
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/s/ MICHAEL MACLEAN
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Paula Soteropoulos
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Michael MacLean
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Chief Executive Officer
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Chief Financial Officer
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