Sarepta Therapeutics Shares Halted as Trial Shows Positive Topline Results

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by Chris Wack

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Sarepta Therapeutics Inc. shares were halted at $84.58 because the company said it had seen positive topline results from part 2 of its study to evaluate SRP-9001 delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy.

Of the 41 patients, 21 were in the placebo crossover cohort. SRP-9001 is an investigational gene transfer therapy that aims to deliver its micro-dystrophin-encoding gene to muscle tissue for targeted production of the micro-dystrophin protein.

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Sarepta said that SRP-9001-treated participants in the placebo crossover group scored a statistically significant 2 points higher on the mean North Star ambulatory assessment at 48 weeks compared with propensity-score-weighted external controls.

The mean NSAA scores of these Part 2 participants improved by 1.3 points from baseline for the SRP-9001 treated group, and NSAA scores decreased by 0.7 points from baseline in the external control group.

The safety profile of patients treated in Part 2 of the trial is consistent with that observed in Part 1. There were no treatment-related serious adverse events, no deaths, and no study closures due to an adverse event. The most common treatment-related adverse event in patients treated in Part 2 was vomiting, similar to Part 1. For patients treated in Part 1, no new safety indications emerged after two years of follow-up.

The study is ongoing and all participants are monitored for safety in addition to a long-term assessment of functional outcomes.

Write to Chris Wack at [email protected]

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