Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in
precision genetic medicine for rare diseases, will present at the
29th Annual Congress of the World Muscle Society 2024 Congress (WMS
2024), taking place Oct. 8-12, 2024, in Prague, Czechia.
Among the data to be presented are new safety and efficacy
results from several studies in the delandistrogene moxeparvovec
clinical development program, including data from Studies 9001-101,
9001-103 (ENDEAVOR) and 9001-301 (EMBARK).
“New data to be presented at WMS from clinical studies of
delandistrogene moxeparvovec include the first look at skeletal
muscle and cardiac MRI outcomes from the EMBARK study, a
late-breaking analysis of safety data from across all
delandistrogene moxeparvovec clinical studies, and five-year
functional results from Study SRP-9001-101, the longest-term data
to date for a gene therapy in Duchenne,” said Louise Rodino-Klapac,
Ph.D., executive vice president, chief scientific officer and head
of research and development, Sarepta Therapeutics. “The results add
to the substantial and growing body of data for delandistrogene
moxeparvovec.”
Podium Presentation:
19O: Muscle MRI outcomes in patients with
Duchenne Muscular Dystrophy treated with delandistrogene
moxeparvovec: Findings from EMBARK Part 1
Oct. 12, 2024
1:45-2:00 a.m. EDT
(07:45-08:00 CEST)
Late Breaking Poster:
726LBP: Long-term safety and tolerability
of delandistrogene moxeparvovec in Duchenne muscular dystrophy:
phase 1 to phase 3 clinical trials
Oct. 11, 2024
9:45-10:45 a.m. EDT
(15:45-16:45 CEST)
Poster Presentations (*Denotes encore presentation)
391P: Agreement and accuracy of ambulatory
definitions in Duchenne muscular dystrophy (DMD): a cross-sectional
analysis*
Oct. 9, 2024
8:30-9:30 a.m. EDT
(14:30-15:30 CEST)
94P: JOURNEY: a natural history study of
Limb Girdle muscular dystrophies R3-R5: baseline characteristics of
study cohort
Oct. 9, 2024
11:15 a.m.-12:15 p.m. EDT
(17:15-18:15 CEST)
423P: Safety and efficacy of
delandistrogene moxeparvovec versus placebo in Duchenne muscular
dystrophy: Phase 3 EMBARK primary results*
Oct. 9, 2024
11:15 a.m.-12:15 p.m. EDT
(17:15-18:15 CEST)
424P: Micro-dystrophin expression and
safety with delandistrogene moxeparvovec gene therapy for DMD in a
broad population: Phase 1B trial (ENDEAVOR)
Oct. 9, 2024
11:15 a.m.-12:15 p.m. EDT
(17:15-18:15 CEST)
425P: Five-year outcomes with
delandistrogene moxeparvovec in patients with Duchenne Muscular
dystrophy (DMD): a phase 1/2a study
Oct. 9, 2024
11:15 a.m.-12:15 p.m. EDT
(17:15-18:15 CEST)
428P: Cardiac MRI outcomes in patients
with Duchenne Muscular Dystrophy treated with delandistrogene
moxeparvovec: Findings from EMBARK Part 1
Oct. 9, 2024
11:15 a.m.-12:15 p.m. EDT
(17:15-18:15 CEST)
The full WMS 2024 program is available at
https://www.wms2024.com/page/programme.
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 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 related to our
research and development programs, technologies, scientific
approaches, and product and product candidates, including potential
benefits related to safety and efficacy, and our mission.
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, clinical development
plans, and bringing its products to markets for various reasons
including 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; 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; 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;
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, 2023, 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, except as required by law.
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version on businesswire.com: https://www.businesswire.com/news/home/20240926302872/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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