Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicines to treat rare neuromuscular
diseases, will present a total of six posters at the 22nd
International Annual Congress of the World Muscle Society (WMS),
including a late-breaking poster presentation titled, SRP-4053
Induces Exon Skipping Leading to Sarcolemmal Dystrophin Expression
in Duchenne Muscular Dystrophy Patients Amenable to Exon 53
Skipping, on Saturday, October 7, 2017 from 12:30-13:30 CEST by
Francesco Muntoni, M.D., principal investigator for the 4053-101
study. The 4053-101 study was supported by the European Community
FP7-HEALTH-2012-Grant 305370, SKIP-NMD, and by Sarepta
Therapeutics. The International Congress of the World Muscle
Society will take place October 3-7, 2017 at the Palais du Grand
Large in Saint Malo, France.
Dr. Muntoni is a Pediatric Neurologist, Great
Ormond Street Hospital for Children NHS Foundation Trust and the
UCL Great Ormond Street Institute of Child Health. He also serves
as Director of the Dubowitz Neuromuscular Center, a leading
clinical and research institution for children affected by
neuromuscular disorders, and Deputy Director for the MRC
Neuromuscular Translational Research Centre at University College
London.
Details of Sarepta’s other four poster
presentations and its joint poster with Catabasis Pharmaceuticals,
Inc. are as follows:
Title: Optimization and
Implementation of Best Practices for Collection and Preparation of
Muscle Biopsies for Analysis During Clinical Trials of
Neuromuscular Disease TherapeuticsDate and Time:
Thursday, October 5, 2017; 17:00-18:30 CEST
Title: Edasalonexent
(CAT-1004), an NF-kB Inhibitor, Enhances Myotube Formation In
Vitro, and Increases Exon-Skipped Sarcolemmal Dystrophin in Muscle
of Mdx MiceDate and Time: Thursday, October 5,
2017; 17:00-18:30 CEST
Title: Development of Novel
Observer-Reported Outcome Assessments in Clinical Trials of
Patients with Duchenne Muscular DystrophyDate and
Time: Thursday, October 5, 2017; 17:00-18:30 CEST
Title: Effects of Long-Term
Treatment with Eteplirsen on Cardiac FunctionDate and
Time: Wednesday, October 4, 2017; 14:30-16:00 CEST
Title: Effects of Long-Term
Eteplirsen Treatment on Upper Limb Function in Patients With
Duchenne Muscular Dystrophy: Findings of Two Phase 2 Clinical
TrialsDate and Time: Thursday, October 5, 2017;
17:00-18:30 CEST
About EteplirsenEteplirsen uses
Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO)
chemistry and exon-skipping technology to skip exon 51 of the
dystrophin gene. Eteplirsen is designed to bind to exon 51 of
dystrophin pre-mRNA, resulting in exclusion of this exon during
mRNA processing in patients with genetic mutations that are
amenable to exon 51 skipping. Exon skipping is intended to allow
for production of an internally truncated dystrophin protein. Data
from clinical studies of eteplirsen in a small number of DMD
patients have demonstrated a consistent safety and tolerability
profile. The pivotal trials were not designed to evaluate long-term
safety and a clinical benefit of eteplirsen has not been
established.
Important Safety Information
About EteplirsenAdverse reactions in DMD patients
(N=8) treated with eteplirsen 30 or 50 mg/kg/week by intravenous
(IV) infusion with an incidence of at least 25% more than placebo
(N=4) (Study 1, 24 weeks) were (eteplirsen, placebo): balance
disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%,
0%). The most common adverse reactions were balance disorder and
vomiting. Because of the small numbers of patients, these represent
crude frequencies that may not reflect the frequencies observed in
practice. The 50 mg/kg once weekly dosing regimen of eteplirsen is
not recommended.
In the 88 patients who received ≥30 mg/kg/week
of eteplirsen for up to 208 weeks in clinical studies, the
following events were reported in ≥10% of patients and occurred
more frequently than on the same dose in Study 1: vomiting,
contusion, excoriation, arthralgia, rash, catheter site pain, and
upper respiratory tract infection.
There have been reports of transient erythema,
facial flushing, and elevated temperature occurring on the day of
eteplirsen infusion.
For further information, please see the full
Prescribing Information.
About Sarepta
TherapeuticsSarepta Therapeutics is a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicines 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 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 statements regarding Sarepta and
Professor Muntoni presenting at the 22nd International Annual
Congress of the World Muscle Society and SRP-4053 inducing exon
skipping that leads to Sarcolemmal Dystrophin Expression in DMD
patients amenable to Exon 53 skipping.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta's control.
Known risk factors include, among others: Sarepta and/or Professor
Muntoni may not be able to present at the 22nd International Annual
Congress of the World Muscle Society due to reasons outside of
their control; the results of Sarepta’s ongoing research and
development efforts and clinical trials for its product candidates,
including SRP-4053, may not be positive or consistent with prior
results or demonstrate a safe treatment benefit; there may be
delays in Sarepta's projected development or regulatory timelines
for its product candidates for various reasons, some of which may
be outside of Sarepta’s control, including regulatory, court or
agency decisions, and any or all of Sarepta's product candidates
may fail in development or may not receive required regulatory
approvals for commercialization; 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, 2016 and Sarepta’s
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 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
Sarepta's 2016 Annual Report on Form 10-K and most recent Quarterly
Report on Form 10-Q for the quarter ended June 30, 2017 filed with
the Securities and Exchange Commission (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
InformationWe 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 Group Brian Reid,
212-257-6725 breid@w2ogroup.com
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