Sarepta shares slump on DMD trial update

12 January 2022

Shares in US biotech Sarepta Therapeutics (Nasdaq: SRPT) lost 19% of their value on Monday and Tuesday following the presentation of new trial results at the 40th Annual JP Morgan Healthcare Conference.

Sarepta announced top-line results from Part 2 of the study SRP-9001-102, an ongoing, randomized, double-blind, placebo-controlled trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 (delandistrogene moxeparvovec) in 41 patients with Duchenne muscular dystrophy (DMD), 21 of whom were in the placebo crossover cohort.

"SRP-9001 is the greatest near-term hope we all have to address the need for a therapy that changes the trajectory of this disease"This investigational gene transfer therapy is intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. SRP-9001-treated participants from the placebo crossover group scored a statistically-significant 2.0 points higher on the mean North Star Ambulatory Assessment (NSAA) at 48 weeks compared to propensity-score weighted external controls.

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