Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious, and other diseases.
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|Sarepta Therapeutics Announces First Patient Dosed in Phase III Clinical Trial of SRP-4045 and SRP-4053 for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping|
“We are excited to announce the first patient dosed in our ESSENCE trial
of SRP-4045 and SRP-4053, for DMD patients amenable to Exon 45 and 53
skipping “ said
The Phase III study, ESSENCE, is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible patients with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 mg/kg SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active group, 66 patients) or placebo (33 patients) for up to 96 weeks (the placebo-controlled period of the trial). This will be followed by an open label extension period in which all patients will receive open-label active treatment for up to 96 weeks.
The study will enroll approximately 99 patients aged 7 to 13 years, inclusive, with a minimum target of 45 patients amenable to exon 45 skipping and 45 patients amenable to exon 53 skipping.
Twice as many patients will receive active treatment as will receive placebo. Approximately 66 patients will be randomized to receive active treatment with either SRP-4045 or SRP-4053 (depending on deletion mutation), and 33 patients will be randomized to receive placebo.
About Duchenne Muscular Dystrophy (DMD)
About SRP-4045 and SRP-4053
Forward Looking Statements
Each forward-looking statement contained in this press release is
subject to risks and uncertainties that could cause actual results to
differ materially from those expressed or implied by such statement.
Applicable risks and uncertainties include, among others: there may be
delays in the ESSENCE study timelines, Sarepta may not be able to
successfully complete the ESSENCE study for various reasons, including
the possibility that the data does not support safety or efficacy of
SRP-4045 SRP-4053; SRP-4045 and SRP-4053 could fail in development or
may never receive regulatory approvals required for commercialization as
therapeutics for DMD patients with mutations amenable to skipping of
exon 45 and 53, respectively, and those risks identified under the
heading “Risk Factors” in Sarepta’s Quarterly Report on Form 10-Q for
the quarter ended
For a detailed description of risks and uncertainties Sarepta faces, you
are encouraged to review the Company’s filings with the
Internet Posting of Information
“Safe Harbor” Statement under the Private Securities Litigation Reform Act of 1995: The statements that are not historical facts contained in this release are forward–looking statements that involve risks and uncertainties, including, but not limited to, the results of research and development efforts, the results of preclinical and clinical testing, the effect of regulation by the FDA and other agencies, the impact of competitive products, product development, commercialization and technological difficulties, and other risks detailed in the company’s Securities and Exchange Commission filings.