Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced it has recently
signed an agreement with the Research Institute at Nationwide
Children’s Hospital (Nationwide Children’s) giving Sarepta the
exclusive option to a Nationwide Children’s gene therapy candidate,
calpain 3 (CAPN-3), to treat Limb-girdle muscular dystrophy type 2A
(LGMD2A).
LGMDs represent a group of distinct genetic
neuromuscular diseases with a generally common set of symptoms,
including progressive, debilitating weakness and wasting that
begins in muscles around the hips and shoulders before progressing
to muscles in the arms and legs. Many LGMD sub-types are seriously
life-limiting and often life-ending diseases. Also known as
calpainopathy, LGMD2A is caused by mutations in the CAPN-3 gene and
is the most common type of LGMD, accounting for almost a third of
cases.
Like Sarepta’s micro-dystrophin and five other
LGMD programs, the LGMD2A program employs the AAVrh74 vector,
designed to systematically and robustly deliver treatment to
skeletal muscle, including the diaphragm, without promiscuously
crossing the blood brain barrier, making it an ideal candidate to
treat muscle disease.
The CAPN-3 program is currently in pre-clinical
trials. The program is led by Zarife Sahenk, M.D., Ph.D., an
attending neurologist at Nationwide Children's, Director of
Clinical and Experimental Neuromuscular Pathology at The Research
Institute at Nationwide Children's and Professor of Pediatrics,
Pathology and Neurology at The Ohio State University College of
Medicine.
“We are pleased to expand and deepen our working
relationship with Nationwide Children’s and Dr. Sahenk, with whom
we are already working on a gene therapy candidate to treat
Charcot-Marie-Tooth. With six LGMD gene therapy programs now in our
portfolio, our commitment and investment in research for this group
of neuromuscular diseases is unparalleled,” said Doug Ingram,
Sarepta’s president and chief executive officer. “Recent positive
early results from our LGMD2E program support expanding our
development strategy to LGMD2A, as both programs utilize AAVrh74
vector, address sub-populations of LGMD, and address a
well-characterized disease by directly replacing the missing
protein which is the cause of the disease by transducing the native
protein. We continue to fuel our gene therapy development engine
aimed at building an enduring model that delivers potentially
transformative therapies to treat genetically based diseases.”
“LGMD2A is the most common form of limb-girdle
muscular dystrophy and its relentless progression causes patients
to lose the ability to walk in early adulthood,” said Dr. Zahenk.
“Our preclinical work suggests that a gene therapy approach has the
potential to help those living with LGMD2A and we look forward
to collaborating with Sarepta to advance this program in the
clinic.”
About Limb-Girdle Muscular
DystrophyLimb-girdle muscular dystrophies are genetic
diseases that cause progressive, debilitating weakness and wasting
that begins in muscles around the hips and shoulders before
progressing to muscles in the arms and legs. Sarepta’s six LGMD
gene therapy programs in development now include LGMD2E, LGMD2D,
LGMD2C, LGMD2B, LGMD2L and LGMD2A.
About Sarepta
TherapeuticsSarepta is at the forefront of precision
genetic medicine, having built an impressive and competitive
position in Duchenne muscular dystrophy (DMD) and more recently in
gene therapies for Limb-girdle muscular dystrophy diseases (LGMD),
Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related
disorders, totaling over 20 therapies in various stages of
development. The Company’s programs and research focus span several
therapeutic modalities, including RNA, gene therapy and gene
editing. Sarepta is fueled by an audacious but important mission:
to profoundly improve and extend the lives of patients with rare
genetic-based diseases. For more information, please visit
www.sarepta.com.
About Nationwide Children’s
HospitalNamed to the Top 10 Honor Roll on U.S. News
& World Report’s 2018-19 list of “Best
Children’s Hospitals,” Nationwide Children’s Hospital is one of
America’s largest not-for-profit freestanding pediatric
health care systems providing wellness, preventive,
diagnostic, treatment and rehabilitative care for infants, children
and adolescents, as well as adult patients with congenital disease.
Nationwide Children’s has a staff of more than 13,000 providing
state-of-the-art pediatric care during more than 1.4 million
patient visits annually. As home to the Department of Pediatrics of
The Ohio State University College of Medicine, Nationwide
Children’s physicians train the next generation of pediatricians
and pediatric specialists. The Research Institute at Nationwide
Children’s Hospital is one of the Top 10 National Institutes of
Health-funded freestanding pediatric research facilities. More
information is available at NationwideChildrens.org.
Forward-Looking StatementsThis
press release contains "forward-looking statements." Any statements
contained in this press release that are not statements of
historical fact may be deemed to be forward-looking statements.
Words such as "believes," "anticipates," "plans," "expects,"
"will," "intends," "potential," "possible" and similar expressions
are intended to identify forward-looking statements. These
forward-looking statements include statements regarding the
potential benefits of the agreement with Nationwide Children’s
giving Sarepta the exclusive option to a Nationwide Children’s gene
therapy candidate, calpain 3 (CAPN-3), to treat LGMD2A; the design
of the AAVrh74 vector to systematically and robustly deliver
treatment to cardiac and skeletal muscle, including the diaphragm,
without promiscuously crossing the blood brain barrier, making it
an ideal candidate to treat muscle disease; the potential of our
gene therapy development engine to deliver transformative therapies
to treat genetically based diseases; the potential of a gene
therapy approach to help those living with LGMD2A; and Sarepta’s
mission to profoundly improve and extend the lives of patients with
rare genetic-based diseases.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Known risk factors include, among others: the expected benefits and
opportunities related to the agreement with Nationwide Children’s
may not be realized or may take longer to realize than expected due
to challenges and uncertainties inherent in product research and
development; in particular, the agreement may not result in any
viable treatments suitable for commercialization due to a variety
of reasons, including any inability of the parties to perform their
commitments and obligations under the agreement, the results of
research may not be consistent with past results or may not be
positive or may otherwise fail to meet regulatory approval
requirements for the safety and efficacy of product candidates,
possible limitations of Company financial and other resources,
manufacturing limitations that may not be anticipated or resolved
for in a timely manner, and regulatory, court or agency decisions,
such as decisions by the United States Patent and Trademark Office
with respect to patents that cover Sarepta’s product candidates;
and even if commercialization of any products under the agreement
is achieved, this may not result in any significant revenues to the
parties; if the actual number of patients suffering from LGMD2A is
smaller than estimated, Sarepta’s revenue and ability to achieve
profitability may be adversely affected; and those risks identified
under the heading “Risk Factors” in Sarepta’s most recent Annual
Report on Form 10-K for the year ended December 31, 2018 and most
recent Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission (SEC) as well as other SEC filings made by the
Company which you are encouraged to review.
Any of the foregoing risks could materially and
adversely affect the Company’s business, results of operations and
the trading price of Sarepta’s common stock. For a detailed
description of risks and uncertainties Sarepta faces, you are
encouraged to review Sarepta's 2018 Annual Report on Form 10-K and
most recent Quarterly Report on Form 10-Q filed with the SEC as
well as other SEC filings made by Sarepta. We caution investors not
to place considerable reliance on the forward-looking statements
contained in this press release. Sarepta does not undertake any
obligation to publicly update its forward-looking statements based
on events or circumstances after the date hereof.
Internet Posting of
Information
We routinely post information that may be
important to investors in the 'For Investors' section of our
website at www.sarepta.com. We encourage investors and
potential investors to consult our website regularly for important
information about us.
Source: Sarepta Therapeutics, Inc.
Sarepta Therapeutics, Inc.
Investors: Ian Estepan, 617-274-4052iestepan@sarepta.com
Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com
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