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Sarepta Therapeutics Inc (NASDAQ: 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.
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.
Crypto Whales Are Loading Up — Are You?
New research shows the biggest crypto buyers are back. And this time? They could hold for the possibility that Bitcoin will surpass $100,000 in 2024. You don’t want to miss the next massive crypto bull run like we saw in 2020 and 2021. To know exactly what’s going on and what to buy… Get Access To Benzinga’s Best Crypto Research and Investments For Only $1.
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