REGENXBIO Inc. (NASDAQ:RGNX) and AveXis, Inc. (NASDAQ:AVXS) 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.
“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.”
“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.”
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 Syndrome
Rett 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 Sclerosis
Amyotrophic 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 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, recently completed a Phase 1
clinical trial for the treatment of SMA Type 1. For additional
information, please visit www.avexis.com.
REGENXBIO 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, 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 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 CONTACTS:
Investors
Heather Savelle, 646-395-3734
heather@argotpartners.com
Media
Adam Pawluk, 202-591-4063
apawluk@jpa.com
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
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