Sarepta’s Next-Gen DMD Drug SRP-5051 Shows Advantages, But Safety Concerns Too
The next-generation DMD drug showed dramatic improvement on exon dystrophin and exon skipping, but small numbers and adverse events give pause.
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The FDA placed a hold on the Phase II study of SRP-5051, which uses the company’s next-generation peptide conjugation technology, but Sarepta thinks it can address safety questions in short order.
The company plans to start its Phase III trial of SRP-9001 in the middle of this year, with a potential readout by the end of 2022.
Arising from an Oxford and Cambridge university collaboration, the spin-out has selected Duchenne muscular dystrophy as the first target for its peptide platform.