AveXis, Inc. (NASDAQ:AVXS) and REGENXBIO Inc. (NASDAQ:RGNX) today
announced an exclusive worldwide license agreement for AveXis to
develop and commercialize gene therapy treatments using REGENXBIO’s
NAV AAV9 vector to treat two 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.
Under the terms of the license agreement, REGENXBIO
will receive an upfront payment upon execution, ongoing fees,
milestone payments and royalties on net sales of products
incorporating the NAV AAV9 vector.
“Building on our experience and the success we have
seen to date with the use of REGENXBIO’s NAV AAV9 vector in our
spinal muscular atrophy clinical trials, this new license agreement
reflects progress on executing our corporate strategy and our
vision of becoming the leader in the treatment of rare and
life-threatening neurological genetic diseases,” said Sean Nolan,
President and Chief Executive Officer of AveXis. “While we remain
intensely focused on the development and commercialization of
AVXS-101 for the treatment of spinal muscular atrophy, we are
excited by the potential for gene therapy to address the needs of
patients with RTT and ALS – two devastating diseases for which
there are no cures and insufficient existing
treatments.”
“This license agreement for our NAV AAV9 vector
highlights the strength of our relationship with our existing NAV
Technology Licensee, AveXis, and our commitment to bringing
important new NAV-based gene therapies to patients with severe
diseases with significant unmet medical need,” said Kenneth T.
Mills, President and Chief Executive Officer of REGENXBIO. "As a
leader in AAV-based gene therapy, REGENXBIO continues to
selectively and strategically license our NAV Technology Platform
for specific vector and indication combinations in a way that
allows us to maintain our focus on internal product development
while at the same time advancing the overall field by expanding the
pipeline of NAV-based gene therapies.”
Preclinical data demonstrating promising efficacy
and safety of gene therapy treatments for RTT and ALS using NAV
AAV9, generated by AveXis’ Chief Scientific Officer Dr. Brian
Kaspar at Nationwide Children's Hospital, has been licensed by
AveXis. AveXis intends to move forward with initiating IND-enabling
studies in both RTT and ALS and plans to provide more details
on these programs in the second half of 2017.
About Rett SyndromeRett syndrome
(RTT) is a devastating, rare neurological disorder characterized by
slowed growth, loss of normal movement and coordination and loss of
communication skills. RTT is caused by an X-linked dominant
mutation in the methyl CpG binding protein 2 (MECP2) gene, which
results in problems with the protein production critical for brain
development. Rett Syndrome occurs in approximately one of every
10,000 female births and usually begins to show signs and symptoms
in infants between six and 18 months of age. Current treatments
only offer symptomatic relief and do not target the genetic cause
of the disease, leaving a significant unmet need.
About Genetic Amyotrophic Lateral
SclerosisAmyotrophic lateral sclerosis (ALS) is a
progressive neurodegenerative disease that affects nerve cells in
the brain and the spinal cord. Familial or inherited forms of ALS
reflect five to 10 percent of ALS cases, or approximately one to
two thousand people in the U.S., and can be caused by mutations in
several genes known to be associated with ALS. Approximately 20
percent of these cases are caused by mutations in the gene that
produces the copper zinc superoxide dismutase 1 (SOD1) enzyme,
which leads to a progressive degeneration of motor neurons
affecting movement and muscle control. ALS usually occurs in people
between the ages of 40 and 70. Current treatments only offer modest
benefits and do not target the genetic cause of the disease,
leaving a significant unmet need
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, recently completed a Phase 1 clinical trial
for the treatment of SMA Type 1. For additional information, please
visit www.avexis.com.
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.
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’ research, development
and regulatory plans for its programs for treatment of RTT and ALS,
its expectations regarding initiation of IND-enabling studies for
these programs and timing of providing an update on these programs.
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" section of AveXis’ Annual
Report on Form 10-K for the year ended December 31, 2016, filed
with the SEC on March 16, 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 Forward-Looking
StatementsThis press release contains “forward-looking
statements,” within the meaning of the Private Securities
Litigation Reform Act of 1995, regarding, among other things,
REGENXBIO’s research, development and regulatory plans in
connection with its NAV Technology Platform and gene therapy
treatments. Such forward-looking statements are based on current
expectations and involve inherent risks and uncertainties,
including factors that could cause actual results to differ
materially from those projected by such forward-looking statements.
All of REGENXBIO’s development timelines could be subject to
adjustment depending on recruitment rate, regulatory agency review
and other factors that could delay the initiation and completion of
clinical trials. Meaningful factors which could cause actual
results to differ include, but are not limited to, 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
ability to obtain and maintain regulatory approval of REGENXBIO’s
product candidates, and the labeling for any approved products; the
scope, progress, expansion, and costs of developing and
commercializing REGENXBIO’s product candidates; REGENXBIO’s ability
to establish and maintain development partnerships; REGENXBIO’s
expenses and revenue; regulatory developments in the United States
and foreign countries; the sufficiency of REGENXBIO’s cash
resources and needs for additional financing; and other factors
discussed in 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 Quarterly Report on Form 10-Q for the quarter
ended March 31, 2017, which are on file with the Securities and
Exchange Commission (SEC) and available at www.sec.gov. In addition
to the risks described above and in REGENXBIO’s filings with the
SEC, other unknown or unpredictable factors also could affect
REGENXBIO’s results. There can be no assurance that the actual
results or developments anticipated by REGENXBIO will be realized
or, even if substantially realized, that they will have the
expected consequences to, or effects on, REGENXBIO. 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. REGENXBIO cautions investors not
to rely too heavily on the forward-looking statements REGENXBIO
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). REGENXBIO 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.
AVEXIS CONTACTS:
Media Inquiries:
Lauren Barbiero
W2O Group
646-564-2156
lbarbiero@w2ogroup.com
Investor Inquiries:
Jim Goff
AveXis, Inc.
650-862-4134
jgoff@avexis.com
REGENXBIO CONTACTS:
Investors
Heather Savelle, 646-395-3734
heather@argotpartners.com
Media
Adam Pawluk, 202-591-4063
apawluk@jpa.com
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