Sarepta gains on Phase I/II data for exon 53 DMD candidate

Sarepta Therapeutics Inc. (NASDAQ:SRPT) jumped $5.65 (14%) to $46.73 on Wednesday and ended the day with a market cap of just over $3 billion after reporting data from the Phase I/II 4053-101 trial of golodirsen (SRP-4053) to treat Duchenne muscular dystrophy amenable to exon 53 skipping. Golodirsen is a phosphorodiamidate morpholino oligomer (PMO) targeting exon 53.

On the trial's primary endpoint, golodirsen increased mean dystrophin protein 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 from baseline in all patients (p<0.001)...