BioCentury
ARTICLE | Clinical News

Sarepta gains on additional DMD data

October 4, 2012 12:59 AM UTC

Sarepta Therapeutics Inc. (NASDAQ:SRPT) gained $29.94 (200%) to $44.93 on Wednesday after reporting additional data for eteplirsen to treat Duchenne muscular dystrophy (DMD). In an open-label extension of a Phase IIb trial, high-dose eteplirsen for 48 weeks led to significantly better clinical outcomes at week 48 compared to placebo for 24 weeks followed by low- or high-dose eteplirsen for an additional 24 weeks. The four patients in the high-dose arm had a 21 m increase in six-minute walk test (6MWT) from baseline to week 48 compared to a 68.4 m reduction for the four patients in the placebo/delayed eteplirsen group (p=0.016). The low-dose did not lead to a significant difference in 6MWT.

Both 48-week arms of eteplirsen and the placebo/delayed eteplirsen arm also led to significant mean increases in dystrophin-positive muscle fibers from baseline to week 48. Sarepta plans to request an end-of-Phase IIb meeting with FDA by year end or early 2013 to discuss the design of a confirmatory trial. Eteplirsen is a phosphorodiamidate morpholino oligomer (PMO) targeting exon 51. ...