Wave Life Sciences (NASDAQ:WVE) slips 11% premarket on light volume on the heels of final safety and tolerability data on Duchenne muscular dystrophy (DMD) candidate suvodirsen (WVE-210201) that appears worse than Sarepta’s EXONDYS 51 (eteplirsen). The results are being presented today at the Muscular Dystrophy Association Clinical and Scientific Conference in Orlando, FL.
36 DMD patients received one of five ascending doses of suvodirsen or placebo and were followed for 85 days. No serious adverse events, deaths or discontinuations were reported.
67% (n=16/24) of participants who received suvodirsen and 80% (n=8/10) of those receiving placebo experienced one or more adverse events (AEs). The most common AEs, all mild or moderate, in the suvodirsen group was pyrexia (high temperature), headache, vomiting and tachycardia (rapid heart rate), all consistent with infusion-related reactions. All resolved spontaneously or with symptomatic treatment.
Safety data on EXONDYS 51 showed a 38% rate of balance disorder and vomiting and 25% rate of contact dermatitis at at 1.7x the recommended dosage.
The company will initiate a Phase 2/3 trial, DYSTANCE 51, in DMD boys amenable to exon 51 skipping in July. Participants will receive 4.5 mg/kg or 3 mg/kg of suvodirsen or placebo administered intravenously once per week for 48 weeks. The primary endpoints will be the change in dystrophin protein level and the change in North Star Ambulatory Assessment score.
Interim muscle biopsy data from an ongoing open-label extension study should be available in H2.
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