Sarepta Plunges on Duchenne Muscular Dystrophy Trial Data
Shares of Sarepta Therapeutics Inc. (SRPT) plunged 50% in after-hours Thursday, following top-line results from Part 1 of a phase II trial of investigational gene therapy SRP-9001 in patients with Duchenne muscular dystrophy, dubbed Study 102.
According to the company, the study met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, in SRP-9001-treated participants versus placebo. However, the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post-treatment.
The NSAA or North Star Ambulatory Assessment is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne. It is used to monitor the progression of the disease and treatment effects which makes it suitable as an endpoint in clinical trials for Duchenne.
In the Study 102, all 41 participants have completed their Part 1, 48-week assessment and have entered the Part 2 crossover phase.
SRPT closed Thursday's trading at $168.95, up 1.50%. In after-hours, the stock fell 50.01% to $84.45.
https://www.finanznachrichten.de/...plunges-on-dmd-trial-data-020.htm
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