SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): January 8, 2018
(Exact Name of Registrant as Specified in its Charter)
(State or other jurisdiction
9600 Blackwell Road, Suite 210
|(Address of principal executive offices)||(Zip Code)|
(Registrants telephone number, including area code)
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
|☐||Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)|
|☐||Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)|
|☐||Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))|
|☐||Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))|
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ☒
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. ☒
|Item 1.01.||Entry into a Material Definitive Agreement.|
Amendment to License Agreement with AveXis
On January 8, 2018, REGENXBIO Inc. (the Company) entered into a First Amendment (the Amendment) to the License Agreement dated March 21, 2014 (the License Agreement) between the Company and AveXis, Inc. (AveXis). Under the Amendment, the Company granted AveXis an exclusive, worldwide commercial license, with rights to sublicense, to any recombinant adeno-associated virus (AAV) vector in the Companys intellectual property portfolio during the term of the License Agreement for the treatment of spinal muscular atrophy (SMA) in humans by in vivo gene therapy. Under the original License Agreement, the Company had granted AveXis a license relating only to the AAV9 vector.
Additionally, the Amendment modifies the terms and conditions of the License Agreement relating to assignment. Under the amended assignment provision, AveXis is permitted to transfer the License Agreement without the Companys consent in connection with a change of control of AveXis, subject to the transferee or successor agreeing in writing to be bound by the terms of the License Agreement and the payment to the Company of certain fees due upon such change of control, as described below. Under the original License Agreement, any assignment by AveXis without the Companys prior written consent had been prohibited. The Amendment also provides that, solely upon the written request of AveXis, the Company may, in its sole discretion after receipt of such request, provide certain collaboration services to AveXis regarding the development and commercialization of gene therapy product candidates for the treatment of SMA.
Pursuant to the Amendment, in consideration for the additional rights granted thereunder, AveXis paid to the Company a fee of $80.0 million upon entry into the Amendment. In addition, AveXis will pay to the Company (i) $30.0 million on the first anniversary of the effective date of the Amendment, (ii) $30.0 million on the second anniversary of the effective date of the Amendment and (iii) potential commercial milestone payments of up to $120 million. In the event of a change of control of AveXis, to the extent that any fee described in (i) or (ii) above, or the first $40 million of milestone payments described in (iii) above, has not yet been paid to the Company, AveXis will be obliged to pay any such unpaid fee to the Company upon the change of control. Additionally, for any product developed for the treatment of SMA using an AAV vector, other than AAV9, REGENXBIO will receive a low double-digit royalty on net sales.
A copy of the Amendment will be filed as an exhibit to the Companys Annual Report on Form 10-K for the year ended December 31, 2017. The Company intends to seek confidential treatment for certain portions of the Amendment.
|Item 7.01.||Regulation FD Disclosure.|
On January 8, 2018, the Company and AveXis issued a press release announcing their entry into the Amendment. A copy of the press release is furnished herewith as Exhibit 99.1 and is incorporated herein by reference.
The information in Item 7.01 of this Current Report on Form 8-K and Exhibit 99.1 attached hereto shall not be deemed filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the Exchange Act), or otherwise subject to liability under that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
|Item 9.01.||Financial Statements and Exhibits.|
|99.1||Press release dated January 8, 2018 relating to the entry into the Amendment by the Company and AveXis.|
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 hereunto duly authorized.
|Date: January 9, 2018||By:||
/s/ Patrick J. Christmas II
|Patrick J. Christmas II|
|Senior Vice President, General Counsel|
REGENXBIO and AveXis Announce Expansion of Relationship through Amended License Agreement for the
Development and Commercialization of Treatments for Spinal Muscular Atrophy
|||AveXis acquires exclusive rights to entire NAV Technology Platform for the development of treatments for SMA|
|||Amended agreement permits assignment by AveXis upon a change of control without REGENXBIOs consent|
|||REGENXBIO could receive up to $260 million, including $140 million in guaranteed upfront and annual payments|
ROCKVILLE, Md. and CHICAGO, Ill., January 8, 2018 (GLOBE NEWSWIRE) REGENXBIO Inc. (Nasdaq: RGNX) and AveXis, Inc. (Nasdaq: AVXS) today announced that they have entered into an amendment which expands upon the exclusive, worldwide license agreement they entered into in March 2014 (2014 License Agreement) for the development and commercialization of products to treat spinal muscular atrophy (SMA). AveXis initial proprietary gene therapy candidate, AVXS-101, is in a pivotal trial for the treatment of SMA Type 1, and a Phase 1 trial for SMA Type 2. AVXS-101 uses REGENXBIOs NAV AAV9 vector.
An expanded relationship further aligns REGENXBIO with AveXis in their commitment to develop and commercialize novel gene therapy treatments for SMA, and serves as additional validation of our NAV Technology Platform and its potential utility in this therapeutic area, said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. We are encouraged by the innovation and progress of AVXS-101 and are pleased to broaden our relationship with AveXis by providing deeper technology access to AveXis as it advances its mission towards commercialization.
Under the terms of the amendment, REGENXBIO granted AveXis exclusive, worldwide rights to all vectors in REGENXBIOs NAV Technology Platform for the treatment of SMA in addition to adding and amending certain terms of the 2014 License Agreement, including the modification of the assignment provision to now permit assignment in the event of a change of control by AveXis without REGENXBIOs consent. REGENXBIO will receive, in addition to the payments and royalties owed under the original 2014 License Agreement, an upfront payment of $80 million, an additional payment of $30 million after one year and an additional payment of $30 million after two years, and REGENXBIO is eligible to receive potential commercial milestone payments of up to $120 million. For any product developed for the treatment of SMA using the NAV AAV9 vector, REGENXBIO will continue to receive mid-single to low double-digit royalties on net sales as defined in the original 2014 License Agreement, and for any product developed for the treatment of SMA using a NAV vector, other than NAV AAV9, REGENXBIO will receive a low double-digit royalty on net sales.
We have been very encouraged by the clinical data demonstrated to date by AVXS-101 utilizing the NAV Technology Platform from REGENXBIO in infants with SMA Type 1 and are optimistic that we may also see robust results in our recently initiated Phase 1 trial in SMA Type 2, said Sean Nolan, President and Chief Executive Officer of AveXis. We believe this expanded partnership with REGENXBIO, which provides exclusive rights to the entire REGENXBIO NAV Technology Platform, will further strengthen our leadership position in gene therapy treatments for this devastating disease.
SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births.
The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients. SMA Type 1 is the leading genetic cause of infant mortality.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIOs NAV ® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
About AveXis, Inc.
AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The companys initial proprietary gene therapy candidate, AVXS-101, is in the pivotal phase of study for the treatment of SMA Type 1, and a Phase 1 trial for SMA Type 2. The company also intends to expand the study of gene therapy into two additional rare neurological monogenic disorders: Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.
REGENXBIO Forward-Looking Statements
This press release includes forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIOs collaboration with AveXis to develop and commercialize products to treat SMA, and future potential milestone and royalty payments by AveXis to REGENXBIO. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIOs expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion of REGENXBIOs clinical trials; the timing and success of preclinical studies and clinical trials conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the Risk Factors and Managements Discussion and Analysis of Financial Condition and Results of Operations sections of REGENXBIOs Annual Report on Form 10-K for the year ended December 31, 2016 and comparable risk factors sections of REGENXBIOs Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SECs website at www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
AveXis Forward-Looking Statements
This press release contains forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, AveXis continued collaboration with REGENXBIO to develop and commercialize products to treat SMA, future potential milestone and royalty payments by AveXis to REGENXBIO, AveXis research, development and regulatory plans for AVXS-101, including and the potential of AVXS-101 to positively impact quality of life and alter the course of disease in patients with SMA Type 1. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, the scope, progress, expansion, and costs of developing and commercializing AveXis product candidates; regulatory developments in the U.S. and EU, as well as other factors discussed in the Risk Factors and the Managements Discussion and Analysis of Financial Condition and Results of Operations sections of AveXis Annual Report on Form 10-K for the year ended December 31, 2016, filed with the SEC on March 16, 2017, and AveXis Quarterly Report on Form 10-Q for the quarter ended September 30, 2017, filed with the SEC on November 9, 2017. In addition to the risks described above and in the Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect AveXis results. There can be no assurance that the actual results or developments anticipated by AveXis will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, AveXis. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved.
All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. AveXis cautions investors not to rely too heavily on the forward-looking statements AveXis makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). AveXis undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Natalie Wildenradt, 646-681-8192
Adam Pawluk, 202-591-4063