Sarepta's Stock Trading Higher As Duchenne Muscular Dystrophy Gene Therapy Shows Promise In Open-Label Study

Sarepta Therapeutics Inc 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 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.

Overview Rating:

Speculative

0100