– Sarepta anticipates filing for accelerated approval of
SRP-9003 in 2025
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in
precision genetic medicine for rare diseases, today announced that
enrollment and dosing is complete in EMERGENE (Study SRP-9003-301),
a Phase 3 clinical trial of SRP-9003 (bidridistrogene
xeboparvovec). SRP-9003 is an investigational gene therapy for the
treatment of limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E/R4),
or beta-sarcoglycanopathy. EMERGENE is a global study, and the
primary endpoint is the biomarker expression of beta-sarcoglycan
protein, the absence of which is the sole cause of LGMD2E/R4.
“The completion of enrollment in the EMERGENE study marks a
significant milestone to bring a potentially disease-modifying
treatment to individuals living with LGMD2E, an ultra-rare form of
LGMD with no treatments beyond symptom management. We are committed
to securing approval of SRP-9003 as quickly as possible and are now
closer to reaching that goal for patients and their families,” said
Louise Rodino-Klapac, Ph.D., executive vice president, chief
scientific officer and head of research and development, Sarepta
Therapeutics. “The design of EMERGENE is an important precedent
that informs development plans for Sarepta’s other LGMD pipeline
programs, including our LGMD2D program which we just initiated and
our LGMD2C program which we expect to initiate in the first quarter
of 2025, and serves as a pathfinder for heterogenous neuromuscular
gene therapies more broadly.”
Data from EMERGENE are expected in the first half of 2025.
Assuming a positive pre-Biologics License Application (BLA) meeting
and supportive data from EMERGENE, Sarepta anticipates submitting a
BLA to the U.S. Food and Drug Administration seeking accelerated
approval for SRP-9003 in 2025.
About Study SRP-9003-301 (EMERGENE)
EMERGENE, Study 9003-301 is a Phase 3, multinational, open-label
study of SRP-9003 for the treatment of LGMD2E/R4 in ambulatory and
non-ambulatory participants, ages 4 and older. EMERGENE’s primary
endpoint is expression of beta-sarcoglycan 60 days after dosing.
Secondary outcomes and endpoints include functional measures
through month 60 and safety.
About SRP-9003 (bidridistrogene xeboparvovec)
SRP-9003 (bidridistrogene xeboparvovec) is an investigational
gene therapy that uses the AAVrh74 vector, which is designed to be
systemically and robustly delivered to skeletal, diaphragm and
cardiac muscle, making it an ideal candidate to treat neuromuscular
diseases. SRP-9003 is intended to deliver a full-length
beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for
its ability to robustly express in the heart1,2,3 which is
critically important for patients with limb-girdle muscular
dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy
and LGMDR4, many of whom die from pulmonary or cardiac
complications.
About Limb-girdle Muscular Dystrophy
Limb-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.
Patients with LGMD Type 2E/R4 (beta-sarcoglycanopathy) typically
begin showing neuromuscular symptoms such as difficulty running,
jumping and climbing stairs before age 10. The disease, which is an
autosomal recessive subtype of LGMD, frequently progresses to loss
of ambulation in the teen years and often leads to early
mortality.
About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic
medicine for rare diseases that devastate lives and cut futures
short. We hold leadership positions in Duchenne muscular dystrophy
(DMD) and limb-girdle muscular dystrophies (LGMDs), and we
currently have more than 40 programs in various stages of
development. Our vast pipeline is driven by our multi-platform
Precision Genetic Medicine Engine in gene therapy, RNA and gene
editing. For more information, please visit www.sarepta.com or
follow us on LinkedIn, X (formerly Twitter), Instagram and
Facebook.
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.
Forward-Looking Statements
This press release contains “forward-looking statements.” Any
statements that are not statements of historical fact may be deemed
to be forward-looking statements. Words such as “believe,”
“anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,”
“look,” “potential,” “possible” and similar expressions are
intended to identify forward-looking statements. These
forward-looking statements include, without limitation, statements
relating to our future operations, technologies and scientific
approaches, business plans, priorities, research and development
programs, the potential benefits of SRP-9003, including SRP-9003
serving as a pathfinder for certain gene therapy more broadly, and
expected plans and milestones, including data from EMERGENE in the
first half of 2025 and potentially submitting a BLA in 2025, and
initiating our LGMD2C program in the first quarter of 2025.
Actual results could materially differ from those stated or
implied by these forward-looking statements as a result of such
risks and uncertainties. Known risk factors include the following:
our data may not be sufficient for obtaining regulatory approval;
success in preclinical and clinical trials, especially if based on
a small patient sample, does not ensure that later clinical trials
will be successful, and the results of future research may not be
consistent with past positive results or with advisory committee
recommendations, or may fail to meet regulatory approval
requirements for the safety and efficacy of product candidates; we
may not be able to comply with all FDA requests in a timely manner
or at all; the possible impact of regulations and regulatory
decisions by the FDA and other regulatory agencies on our business;
the commencement and completion of our clinical trials and
announcement of results may be delayed or prevented for a number of
reasons, including, among others, denial by the regulatory agencies
of permission to proceed with our clinical trials, or placement of
a clinical trial on hold, challenges in identifying, recruiting,
enrolling and retaining patients to participate in clinical trials
and inadequate quantity or quality of supplies of a product
candidate or other materials necessary to conduct clinical trials;
different methodologies, assumptions and applications we use to
assess particular safety or efficacy parameters may yield different
statistical results, and even if we believe the data collected from
clinical trials of our product candidates are positive, these data
may not be sufficient to support approval by the FDA or other
global regulatory authorities; we may not be able to execute on our
business plans, including meeting our expected or planned
regulatory milestones and timelines, research and clinical
development plans, and bringing our product candidates to market,
for various reasons, many of which may be outside of our control,
including possible limitations of company financial and other
resources, manufacturing limitations that may not be anticipated or
resolved for in a timely manner, regulatory, court or agency
decisions, such as decisions by the United States Patent and
Trademark Office with respect to patents that cover our product
candidates; and those risks identified under the heading “Risk
Factors” in our most recent Annual Report on Form 10-K for the year
ended December 31, 2023, and 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 the 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, except as required by law.
Source: Sarepta Therapeutics, Inc.
References:
- Pozsgai ER, et al. Systemic AAV-Mediated b-Sarcoglycan Delivery
Targeting Cardiac and Skeletal Muscle Ameliorates Histological and
Functional Deficits in LGMD2E Mice. Mol. Ther. 2017 Apr
5;25(4):855-869.
- Mendell JR, et al. Assessment of Systemic Delivery of
rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular
Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun
15;77(9):1-10.
- Salva MZ, et al. Design of tissue-specific regulatory cassettes
for high-level rAAV-mediated expression in skeletal and cardiac
muscle. Mol Ther. 2007;15(2):320-329.
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version on businesswire.com: https://www.businesswire.com/news/home/20241218625378/en/
Investor Contact: Ian Estepan, 617-274-4052
iestepan@sarepta.com
Media Contact: Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com
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