Sarepta's Stock Trading Higher As Duchenne Muscular Dystrophy Gene Therapy Shows Promise In Open-Label Study
- Sarepta Therapeutics Inc (NASDAQ: SRPT) has announced positive 12-week results from the first 11 participants enrolled in open-label study 9001-103 ENDEAVOR being conducted in partnership with Roche Holdings AG's (OTC: RHHBY).
- Results demonstrated robust expression of micro-dystrophin and no new safety signals from prior studies.
- SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.
- All patients demonstrated robust transduction, with a mean micro-dystrophin expression of 55.4% of normal, as measured by western blot.
- Muscle dystrophin levels demonstrated a mean of 70.5% (baseline 12.8%) muscle fibers expressing micro-dystrophin at 12 weeks with a mean intensity at the sarcolemma of 116.9% (baseline 41.0%) compared to normal biopsies.
- Mean vector genome copies per nucleus reached 3.87.
- The safety profile was consistent with the safety seen in earlier studies.
- Two patients experienced serious adverse events (transaminase elevation in one patient and nausea and vomiting in a second patient) that fully resolved.
- Price Action: SRPT shares are up 11.5% at $83.75 during the market session on the last check Tuesday.
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