Sarepta Announces Clinical Hold Lifted for its Duchenne Muscular Dystrophy Micro-dystrophin Gene Therapy Program
September 24 2018 - 7:00AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicine to treat rare neuromuscular diseases,
announced today that the Food and Drug Administration (FDA) has
lifted the clinical hold for the Company’s Duchenne muscular
dystrophy (DMD) micro-dystrophin gene therapy program. Sarepta
previously announced on July 25, 2018, that the FDA placed the
program on clinical hold due to the presence of trace amounts of
DNA fragment in research-grade third-party supplied plasmid in a
manufacturing lot. In response, and in collaboration with
Nationwide Children’s Hospital, an action plan was developed and
submitted to the FDA, including an audit of the plasmid supplier
and a commitment to use GMP-s plasmid for all future production
lots.
“Thanks to the diligent and rapid work of my
Sarepta colleagues and Nationwide Children’s Hospital in compiling
and submitting a complete response and the expeditious evaluation
by the FDA in reviewing the response and removing this clinical
hold, we have been able to address the clinical hold in record time
and without delay to this profoundly important clinical program,”
stated Doug Ingram, Sarepta’s president and chief executive
officer. “Our focus now is on meeting with the Division to take
guidance and gain alignment around what we hope to be our
registration trial for our micro-dystrophin program and achieving
our goal of commencing that trial by year-end
2018.”
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicine to treat rare neuromuscular diseases.
The Company is primarily focused on rapidly advancing the
development of its potentially disease-modifying Duchenne muscular
dystrophy (DMD) drug candidates. For more information, please visit
www.sarepta.com.
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 focus on meeting with the
Division to take guidance and gain alignment around what Sarepta
hopes to be its registration trial for its micro-dystrophin
program; Sarepta’s expectation that the DMD micro-dystrophin gene
therapy program will not be delayed due to the clinical hold; and
Sarepta’s goal to commence the DMD micro-dystrophin gene therapy by
year-end 2018.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Known risk factors include, among others: Sarepta may not be able
to execute on its business plans, including meeting its expected or
planned regulatory milestones and timelines, research and clinical
development plans and timelines, and bringing its product
candidates to market, for various reasons including that study data
may not consistently or sufficiently demonstrate the safety or
efficacy of any of Sarepta’s 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 CHMP on eteplirsen or the United States Patent and
Trademark Office with respect to patents that cover Sarepta’s
product candidates; and even if Sarepta’s programs result in new
commercialized products, Sarepta may not achieve any significant
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, 2017 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 2017 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.
Media and Investors: Sarepta Therapeutics, Inc. Ian Estepan,
617-274-4052 iestepan@sarepta.com or W2O GroupRachel Hutman,
301-801-5540rhutman@w2ogroup.com
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