Early-stage trial shows positive profile for Sarepta's DMD candidate SRP-9001

Shares of Sarepta Therapeutics (Nasdaq: SRPT) rose as much as 12% yesterday, after the US biotech announced positive 12-week expression and safety results from the first 11 participants enrolled in Study SRP-9001-103, an open-label study known as ENDEAVOR, which is being conducted in partnership with Roche (ROG: SIX).

In results from the first clinical study using commercially representative material, SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) demonstrated robust expression of micro-dystrophin and no new safety signals from prior studies, supporting its potentially differentiated profile for the treatment of Duchenne muscular dystrophy (DMD). 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.

Swiss pharma giant Roche entered the partnership in December 2019, when it paid Sarepta an upfront $750 million in cash and $400 million in equity.