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 REGENXBIO’s 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 REGENXBIO’s 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
REGENXBIO’s 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.”
About SMA
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. REGENXBIO’s 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 company’s 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, REGENXBIO’s 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 REGENXBIO’s expectations
and predictions is subject to a number of risks and uncertainties,
including the timing of enrollment, commencement and completion of
REGENXBIO’s 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 “Management’s Discussion and Analysis of Financial
Condition and Results of Operations” sections of REGENXBIO’s Annual
Report on Form 10-K for the year ended December 31, 2016 and
comparable “risk factors” sections of REGENXBIO’s 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
SEC’s 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 “Management’s
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.
REGENXBIO CONTACTS:
Investors Natalie Wildenradt, 646-681-8192
natalie@argotpartners.com
Media Adam Pawluk, 202-591-4063
apawluk@jpa.com
AVEXIS CONTACTS:
Media Inquiries:Lauren BarbieroW2O
Group646-564-2156lbarbiero@w2ogroup.com Investor
Inquiries:Jim GoffAveXis,
Inc.650-862-4134jgoff@avexis.com
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