Sarepta gains on 6MWD data for Duchenne patients
Sarepta Therapeutics Inc. (NASDAQ:SRPT) jumped $8.02 (40%) to $28 on Thursday after presenting data at the JPMorgan Healthcare Conference from 10 patients in the open-label extension of the Phase IIb Study 4658-US-201 trial evaluating eteplirsen ( AVI-4658) to treat Duchenne muscular dystrophy (DMD). Patients who received either dose of eteplirsen for 120 weeks (30 or 50 mg/kg) had a 9.8 meter reduction in mean six-minute walk test (6MWT) from baseline to week 120 compared to a 67.8 meter mean reduction for patients who received placebo for 24 weeks followed by eteplirsen for an additional 96 weeks (p=0.016). Sarepta plans to start a confirmatory Phase III trial of eteplirsen -- a phosphorodiamidate morpholino oligomer (PMO) targeting exon 51 -- next quarter. ...