Sarepta (SRPT) to Update on Additional Eteplirsen Phase IIb Data

Sarepta Therapeutics (NASDAQ: SRPT) announced 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:

• Individual patient data on the primary endpoint of change in dystrophin-positive fibers from baseline;
  
• Additional biochemical findings including RT-PCR and western blot images from selected patients;
  
• Additional information on the two patients in the 30 mg/kg cohort who showed a rapidly progressive decline on the 6-minute walk test and were excluded from the analysis; and
  
• A summary of treatment-emergent adverse events comparing eteplirsen-treated patients versus placebo, which demonstrated that eteplirsen was well-tolerated through 48 weeks of treatment. No treatment-related adverse events, serious adverse events, or treatment discontinuations related to eteplirsen were observed. In addition, no treatment related changes were detected on any safety laboratory parameters, including several biomarkers for renal function.

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

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