Sarepta Therapeutics Provides Program Update for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Mus...
September 09 2020 - 5:19PM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced that it has
completed a Type C ‘written response only’ meeting with the Office
of Tissues and Advanced Therapies (OTAT), part of the Center for
Biologics Evaluation and Research (CBER) at the U.S. Food and Drug
Administration (FDA), to obtain OTAT’s concurrence on the
commencement of its next clinical trial for SRP-9001 using
commercial process material. SRP-9001
(AAVrh74.MHCK7.micro-dystrophin) is Sarepta’s investigational gene
transfer therapy for the treatment of Duchenne muscular dystrophy.
Among other items, OTAT has requested that
Sarepta utilize an additional potency assay for release of SRP-9001
commercial process material prior to dosing in a clinical study.
Sarepta has several existing assays and data that it believes could
be employed in response to OTAT’s request. However, additional
dialogue with the Agency is required to determine the acceptability
of the potency assay approach.
“We look forward to working with OTAT to
potentially satisfy their requests and to obtain clarity on the
timing of the commencement of our commercial supply study. We will
provide further updates as we are able,” said Doug Ingram,
president and chief executive officer, Sarepta Therapeutics. “Every
day, thousands of children degenerate from the irreversible damage
caused by Duchenne muscular dystrophy. It is for that reason that
we will work relentlessly with the Division to satisfy any requests
of OTAT and continue the advancement of a potentially
transformative therapy for these patients.”
About SRP-9001
(AAVrh74.MHCK7.micro-dystrophin)SRP-9001 is an
investigational gene transfer therapy intended to deliver the
micro-dystrophin-encoding gene to muscle tissue for the targeted
production of the micro-dystrophin protein. Sarepta is responsible
for global development and manufacturing for SRP-9001 and plans to
commercialize SRP-9001 in the United States. In December 2019, the
Company announced a licensing agreement granting Roche the
exclusive right to launch and commercialize SRP-9001 outside the
United States. Sarepta has exclusive rights to the micro-dystrophin
gene therapy program initially developed at the Abigail Wexner
Research Institute at Nationwide Children’s Hospital.
About Sarepta Therapeutics
At Sarepta, we are leading a revolution in precision genetic
medicine and every day is an opportunity to change the lives of
people living with rare disease. The Company has built an
impressive position in Duchenne muscular dystrophy (DMD) and in
gene therapies for limb-girdle muscular dystrophies (LGMDs),
mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and
other CNS-related disorders, with more than 40 programs in various
stages of development. The Company’s programs and research focus
span several therapeutic modalities, including RNA, gene therapy
and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Sarepta Forward-Looking
Statements
This 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 Sarepta’s belief that its existing
assays and data could be employed in response to OTAT’s request;
the acceptability of Sarepta’s potency assay approach by the FDA;
our plan to work with OTAT to potentially satisfy their requests
and to obtain clarity on the timing of the commencement of our
commercial supply study; and the potential of SRP-9001 to be a
transformative therapy for DMD patients.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Known risk factors include, among others: delays in the
commencement of Sarepta’s next clinical study for SRP-9001 could
delay, prevent or limit our ability to gain regulatory approval for
SRP-9001; any inability to complete successfully clinical
development could result in additional costs to Sarepta or impair
Sarepta’s ability to generate revenues from product sales,
regulatory and commercialization milestones and royalties; SRP-9001
may not result in a viable treatment suitable for commercialization
due to a variety of reasons, including the results of future
research may not be consistent with past positive results or may
fail to meet regulatory approval requirements for the safety and
efficacy of product candidates; Sarepta may not be able to execute
on its business plans and goals, including meeting its expected or
planned regulatory milestones and timelines, clinical development
plans, and bringing its product candidates to market, due to a
variety of reasons, many of which may be outside of Sarepta’s
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 Sarepta’s
product candidates and the COVID-19 pandemic; 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,
2019, 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 Sarepta which you are encouraged to review.
Any of the foregoing risks could materially and
adversely affect Sarepta’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.
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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