Sarepta Therapeutics Announces Presentations at the 2017 MDA Scientific Conference
March 17 2017 - 8:30AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage
biopharmaceutical company focused on the discovery and development
of unique RNA-targeted therapeutics for the treatment of rare
neuromuscular diseases, today provided an update to investors that
it will present data from eteplirsen studies at the MDA Scientific
Conference in Arlington, Virginia on March 19-22. Sarepta is
scheduled to give an oral, as well as poster presentation, on the
cardiac function of eteplirsen-treated patients; a poster
presentation on non-ambulatory patients; and a poster presentation
on the pulmonary function of eteplirsen-treated patients.
Details of Sarepta’s presentations at the MDA Scientific
Conference are as follows:
Oral Presentation and Poster:
Title: Effects of Long-Term Treatment with
Eteplirsen on Cardiac Function: Left Ventricular Ejection Fraction
in Eteplirsen-Treated Patients vs Disease Natural History
Date and Time: Wednesday, March 22, 10:45-11:00am
ET
Posters: Title: Effects of
Long-Term Treatment with Eteplirsen on Cardiac Function: Left
Ventricular Ejection Fraction in Eteplirsen-Treated Patients vs
Disease Natural History
Title: Long-Term Treatment with Eteplirsen in
Non-ambulatory Patients: A Case Study in Identical Twins
Title: Effects of Long-Term Treatment With
Eteplirsen on Pulmonary Function in Patients With Duchenne Muscular
Dystrophy: Findings of Two Phase 2 Clinical Trials
About EXONDYS 51™
EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate
morpholino oligomer (PMO) chemistry and exon-skipping technology to
skip exon 51 of the dystrophin gene. EXONDYS 51 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 EXONDYS 51 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 EXONDYS 51 has not been
established.
Important Safety Information
- Adverse reactions in DMD patients (N=8) treated with 30 or 50
mg/kg/week of EXONDYS 51 with incidence of at least 25% more than
placebo (N=4) (Study 1) were: balance disorder (38%), vomiting
(38%) and contact dermatitis (25%). 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 EXONDYS 51 is not recommended.
- In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51
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 EXONDYS 51
infusion.
Please see the EXONDYS 51 (eteplirsen) U.S. Full Prescribing
Information at www.EXONDYS51.com.
About Sarepta Therapeutics Sarepta
Therapeutics is a commercial-stage biopharmaceutical company
focused on the discovery and development of unique RNA-targeted
therapeutics for the treatment of rare neuromuscular diseases. The
Company is primarily focused on rapidly advancing the development
of its potentially disease-modifying DMD drug candidates. For more
information, please visit us at www.sarepta.com.
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.
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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