Sarepta jumps on clinical outcome data for eteplirsen

Sarepta Therapeutics Inc. (NASDAQ:SRPT) jumped $5.06 (146%) to $8.52 on Tuesday after reporting that once-weekly 50 mg/kg IV eteplirsen led to significantly better clinical outcomes at week 36 compared with placebo as evaluated by a six-minute walk test in an extension of a Phase IIb trial to treat Duchenne muscular dystrophy (DMD). Sarepta (formerly AVI BioPharma Inc.) said four patients who received eteplirsen for all 36 weeks had only a 8.7 m reduction from a baseline mean of about 396 m in the six-minute walk test. In comparison, four patients who received placebo for 24 weeks followed by eteplirsen for 12 weeks had a 78 m reduction from a baseline mean of 394.5 m. The difference between the groups was significant (p<=0.019). The open-label study was an extension of the double-blind, U.S. Phase IIb Study 4658-US-201 trial, which enrolled 12 patients aged 7-13. Sarepta said it plans to report 48-week data in October and request an end-of-Phase IIb meeting with FDA by year end to discuss the design of a confirmatory trial. ...