Sarepta To Present Addl. Phase IIb Data Of Eteplirsen To Treat DMD – Quick Facts

(RTTNews.com) - Sarepta Therapeutics Inc. (SRPT) said Friday that data from a Phase IIb study of its investigational drug eteplirsen for the treatment of boys with Duchenne muscular dystrophy will be presented on October 13 at the World Muscle Society in Perth, Australia.

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 Duchenne muscular dystrophy, or DMD.

Results from the Phase IIb extension study confirmed that 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 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.

Data to be presented will also include 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.

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.

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Sarepta To Present Addl. Phase IIb Data Of Eteplirsen To Treat DMD - Quick Facts