Sarepta Announces Results From SRP-9001 Study

Sarepta today announced topline results from Part 2 of Study 102, an ongoing, randomized, double-blind, placebo-controlled clinical trial evaluating its investigational gene transfer therapy SRP-9001 in Duchenne patients.  

In its placebo crossover cohort, the mean NSAA at 48 weeks scored a statistically significant 2.0 points higher than an external control comparison group of Duchenne patients, and the safety profile of SRP-9001 was consistent with that seen in Part 1. There were no treatment-related serious adverse events, no deaths, and no study discontinuations due to an adverse event. 

This study is ongoing, and all participants continue to be monitored for safety and longer-term assessments of functional outcomes.

For more information, read the press release here.