Sarepta Therapeutics to Commence Dosing of the Next Study with Commercial Process Material for the SRP-9001 Gene Therapy Prog...
November 05 2020 - 4:02PM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced important
updates related to the development plan for SRP-9001
(AAVrh74.MHCK7.micro-dystrophin), the Company’s investigational
gene transfer therapy for the treatment of Duchenne muscular
dystrophy.
In light of the risks that the ongoing COVID-19
pandemic posed to the execution of a large, multi-center trial,
Sarepta proposed a new clinical study in the September 2020 Type C
meeting to support the Company’s clinical and regulatory efforts --
Study SRP-9001-103 (Study 103). Study 103 is an open-label study in
up to 10 patients evaluating the safety and expression of
commercial process material for SRP-9001 and is intended to
accelerate Sarepta’s validation of commercial process
material.
In the Type C meeting written response, 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), requested, among other things, an
additional potency assay to be used for the release of SRP-9001
commercial process material. Subsequent to the written feedback,
Sarepta and OTAT have engaged in informal and productive dialog and
as a result of those discussions, Sarepta anticipates commencing
dosing in Study 103 this year.
“Our ability to rapidly address outstanding
issues regarding our potency assay approach and commence Study 103
is an important milestone for SRP-9001 and for Duchenne muscular
dystrophy patients who daily degenerate from this cruel disease,”
said Doug Ingram, president and CEO, Sarepta. “Time and delay are
not on the side of patients with Duchenne. This rapid resolution
would not have occurred without the willingness of OTAT to engage
with us informally and to discuss collaboratively how we could
allow the program to advance. I want to thank the professionals at
OTAT for their willingness, in midst of many competing priorities
and in the face of a pandemic, to work with us to find an
appropriate solution that will allow us to dose patients this year
with our commercial process material of SRP-9001. We look forward
to commencing Study 103 this year and having a data read-out from
our ongoing blinded, placebo-controlled trial, Study 102, in early
2021.”
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 of 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 upon approval.
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
TherapeuticsAt 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 and competitive 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.
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 the expectation to commence
clinical dosing with commercial-process material for SRP-9001
(study 103) before the end of 2020; the potential of Study 103 to
accelerate Sarepta’s validation of commercial-process material; and
the expectation to have a data read-out from our ongoing blinded,
placebo-controlled trial (Study 102), in early 2021.
These forward-looking statements involve risks
and uncertainties, many of which are beyond our control. Known risk
factors include, among others: success in pre-clinical trials and
clinical trials, especially if based on a small patient sample,
does not ensure that later clinical trials will be successful; 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;
our data for SRP-9001 may not be sufficient for obtaining
regulatory approval; we may not be able to execute on our business
plans and goals, including meeting our expected or planned
regulatory milestones and timelines, clinical development plans,
and bringing our product candidates to market, due to a variety of
reasons, some 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 the
COVID-19 pandemic; and even if Sarepta’s programs result in new
commercialized products, Sarepta may not achieve the expected
revenues from the sale of such products; 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 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.
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, 617-274-4052,
iestepan@sarepta.comMedia:Tracy Sorrentino, 617-301-8566,
tsorrentino@sarepta.com
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