Sarepta Dosing First Patients In Phase 3 Trial Of Duchenne Muscular Dystrophy Drug

Sarepta Therapeutics Inc SRPT revealed that it dosed first patient in final stage clinical study of SRP-4045 and SRP-4053 for treatment of Duchenne Muscular Dystrophy Amenable to exon 45 or 53 skipping.

The company indicated the third stage trial, ESSENCE, is a double-blind, placebo-controlled, multi-center trial to assess the safety and efficacy of both the drug candidates. It would randomize patients to get once weekly intravenous infusions of 30 mg/kg SRP-4045 or SRP-4053 for 96 weeks.

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Following this, an open label extension period would commence with all patients getting open-label active treatment for a maximum of 96 weeks.

Sarepta CEO Edward Kaye viewed, "It is our goal to treat as many patients amenable to exon skipping as possible, and therefore is important to advance our clinical pipeline candidates. We have made great effort to develop a meaningful clinical trial, using our learnings from our previous clinical programs and observations of the natural history of Duchenne."

The stock closed regular trading at $62.24 on Wednesday, gaining 5.31 percent.