Sarepta Therapeutics to Present Additional 48-Week Data From the Phase IIb Study of Eteplirsen for the Treatment of …

CAMBRIDGE, MA--(Marketwire - Oct 12, 2012) - Sarepta Therapeutics ( NASDAQ : SRPT ), a developer of innovative RNA-based therapeutics, announced today that data from a Phase IIb study evaluating eteplirsen, an investigational treatment for boys with Duchenne muscular dystrophy (DMD), will be presented Saturday, October 13th at the World Muscle Society in Perth, Australia.Principal investigator, Jerry R. Mendell, M.D. of Nationwide Children's Hospital, will present the data in an oral presentation of the abstract titled, "Results at 48 Weeks of a Phase IIb Extension Study of the Exon-Skipping Drug Eteplirsen in Patients with Duchenne muscular dystrophy (DMD)."Dr. Mendell will present tomorrow from 2:30 to 4:00 p.m. WST UTC +8 hours/2:30 to 4:00 a.m. EDT.

The presentation will describe new and previously reported efficacy and safety data from the Phase IIb study examining 48 weeks of treatment with eteplirsen in boys with DMD.Results from the Phase IIb extension study confirmed that treatment with Sarepta's lead exon-skipping compound, eteplirsen, met the primary efficacy endpoint, increase in novel dystrophin, and achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT) over the placebo/delayed treatment cohort.

Additional data to be presented includes:

Dr. Mendell's presentation will be posted on the Sarepta website in the "Events & Presentations" section after the session is completed.

About Study 201 and Study 202 (Phase IIb Eteplirsen Study)

Study 4658-US-201 was conducted at Nationwide Children's Hospital in Columbus, Ohio.Twelve boys meeting the inclusion criteria being between 7 and 13 years of age with appropriate deletions of the dystrophin gene that confirm eligibility for treatment with an exon-51 skipping drug, received double-blind IV infusions of placebo (n=4), 30 mg/kg of eteplirsen (n=4), or 50 mg/kg of eteplirsen once weekly for 24 weeks (n=4).Muscle biopsies for evaluation of dystrophin were obtained at baseline for all subjects, and after 12 weeks for patients in the 50 mg/kg cohort and after 24 weeks for patients in the 30 mg/kg cohort.Two placebo patients were randomized to the 30 mg/kg cohort and two placebo patients were randomized to the 50 mg/kg cohort.This study design allowed Sarepta to investigate the relationship of dose and duration of eteplirsen treatment on the production of dystrophin over the course of the 24-week study.

Study 4658-US-202 is the extension study to 201 and continues to assess the long-term safety and efficacy of open-label eteplirsen.The four placebo patients were rolled over to open-label eteplirsen at week 24, with six patients on 30 mgs/kg, and six patients on 50 mgs/kg.Third biopsies occurred at 48 weeks in the original study 201 treated patients, and at 24 weeks, the same time point, in the original placebo patients.6MWT was performed at 32 weeks, 36 weeks, 48 weeks and will continue to be performed every 12 weeks going forward.

About Dystrophin

Dystrophin, a large structural protein, is critical to the stability of myofiber membranes in skeletal, diaphragmatic and cardiac muscle, protecting muscle fibers from contraction-induced damage.Loss of functional dystrophin destabilizes the dystroglycan protein complex, impairing its localization to the muscle membrane, and compromising the integrity of the membrane structure. The absence of functional dystrophin results in muscle membrane breakdown with muscle fibers being replaced by adipose and fibrotic tissue.

About the 6-Minute Walk Test

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Sarepta Therapeutics to Present Additional 48-Week Data From the Phase IIb Study of Eteplirsen for the Treatment of ...