Sarepta Therapeutics Inc. (NASDAQ:SRPT) shared topline data from the ESSENCE Phase 3 trial of Amondys 45 (casimersen) and Vyondys 53 (golodirsen) compared with placebo in 225 patients aged 6-13 years with Duchenne muscular dystrophy amenable to exon 45 or 53 skipping.
The treatment favored placebo; however, the observed difference of 0.05 steps/second in least-squares means (LSM) did not reach statistical significance (P=0.309) for the primary endpoint, the 4-step ascend velocity at 96 weeks.
