AveXis Enters into Licensing Agreement with Genethon
March 13 2018 - 4:01PM
AveXis, Inc. (NASDAQ:AVXS) and Genethon today announced they have
entered into an exclusive, worldwide license agreement for in vivo
gene therapy delivery of AAV9 vector into the central nervous
system (CNS) for the treatment of spinal muscular atrophy (SMA).
“Adding to our robust intellectual property
estate, this agreement further strengthens our position by
providing freedom to operate when using intravenous or intrathecal
routes of administration to deliver the AAV9 vector into the CNS
for the treatment of SMA,” said Sean Nolan, President and Chief
Executive Officer of AveXis. “With our proprietary gene therapy,
AVXS-101, currently being evaluated in patients with SMA in ongoing
clinical trials in the U.S., and soon in Europe, we are pleased to
have this exclusive worldwide agreement in place.”
Under the terms of the agreement, Genethon
granted AveXis a license to patents in the U.S., Europe and Japan,
for the AAV9 SMN product and in vivo gene therapy delivery of AAV9
vector into the CNS using intrathecal or intravenous routes of
administration for the treatment of SMA.
“Genethon is pleased to enter into this
agreement with AveXis and to contribute to the efforts for the
development of treatments for SMA patients who have urgent medical
needs,” said Frédéric Revah, Chief Executive Officer of Genethon.
“It demonstrates Genethon’s capability to develop effective
first-in-class technologies and the excellence of our translational
research driven by the commitment to treat patients living with
rare diseases.”
About SMASMA 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 and is the leading genetic
cause of infant mortality.
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 2 typically presents between
six and 18 months of age, and those affected will never walk
without support and most will never stand without support. SMA Type
2 results in mortality in more than 30 percent of patients by the
age of 25.
About AVXS-101AveXis’ initial
product candidate, AVXS-101, is its proprietary gene therapy
currently in development for the one-time treatment of SMA Types 1
and 2, designed to address the monogenic root cause of SMA and
prevent further muscle degeneration by addressing the defective
and/or loss of the primary SMN gene. AVXS-101 also targets motor
neurons, providing rapid onset of effect and crossing the blood
brain barrier to allow effective targeting of both central and
systemic features.
About AveXis, Inc.AveXis, Inc.
is a clinical-stage gene therapy company, dedicated to developing
and commercializing novel treatments for patients suffering from
rare and life-threatening neurological genetic diseases. Our
initial product candidate, AVXS-101, is our proprietary gene
therapy currently in development for the treatment of spinal
muscular atrophy, or SMA, Type 1, the leading genetic cause of
infant mortality, and SMA Type 2. The U.S. Food and Drug
Administration, or FDA, has granted AVXS-101 Orphan Drug
Designation for the treatment of all types of SMA and Breakthrough
Therapy Designation, as well as Fast Track Designation for the
treatment of SMA Type 1. In addition to developing AVXS-101 to
treat SMA Type 1 and Type 2, we also plan to develop other novel
treatments for rare neurological diseases, including Rett syndrome
and a genetic form of amyotrophic lateral sclerosis caused by
mutations in the superoxide dismutase 1 (SOD1) gene.
About GenethonCreated by the
AFM-Telethon, the French Muscular Dystrophy Association (AFM),
Genethon, located in Evry, France, is a non-profit R&D
organization dedicated to the development of biotherapies for
orphan genetic diseases, from the research to clinical validation.
Genethon is specialized in the discovery and development of gene
therapy drugs and has multiple ongoing programs at clinical,
preclinical and research stage for neuromuscular, blood, immune
system, and liver diseases.
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’ freedom to operate afforded by the license agreement with
Genethon and AveXis’ research, development and regulatory plans for
AVXS-101. 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,
2017, filed with the SEC on February 28, 2018. 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.
Media Inquiries:Lauren BarbieroW2O
Group646-564-2156lbarbiero@w2ogroup.com Investor Inquiries:Jim
GoffAveXis, Inc.650-862-4134jgoff@avexis.com
Genethon Press Contact:Stéphanie
Bardon+330169471278sbardon@afm-telethon.fr
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