Sarepta reports Phase I/II data for exon 53 DMD candidate
Sarepta Therapeutics Inc. (NASDAQ:SRPT) reported data from 25 boys with Duchenne muscular dystrophy (DMD) with confirmed deletions of the DMD gene amenable to skipping exon 53 from the Phase I/II 4053-101 trial showing that once-weekly 30 mg/kg IV golodirsen (SRP-4053) increased mean dystrophin protein levels, a co-primary endpoint, to 1.019% of normal at week 48 compared to 0.095% of normal at baseline as measured by Western blot (p<0.001). Golodirsen also increased exon 53 skipping, a secondary endpoint, from baseline in all patients (p<0.001).
The 2-part, double-blind, placebo-controlled, European trial analyzed biopsies of the bicep muscle at baseline and at week 48 and is slated to continue for 144 weeks. Co-primary endpoints also include safety and the 6-minute walk test (6MWT). Secondary endpoints include pharmacokinetics, pulmonary function tests and percentage of dystrophin-positive fibers. Golodirsen is a phosphorodiamidate morpholino oligomer (PMO) targeting exon 53...
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