Wave Life Sciences presents final results from Phase 1 suvodirsen study at MDA
Wave Life Sciences announced the final results from its Phase 1 clinical trial of investigational suvodirsen in boys with Duchenne muscular dystrophy, or DMD, who are amenable to exon 51 skipping. The company also announced the design of its planned Phase 2/3 clinical trial of suvodirsen in DMD, DYSTANCE 51. The Phase 1 data and DYSTANCE 51 details will be presented at the Muscular Dystrophy Association, or MDA. Wave's Phase 1 clinical trial was a global, multicenter, double-blind, placebo-controlled study designed to evaluate the safety, tolerability and plasma concentrations of single ascending doses of suvodirsen administered intravenously. Thirty-six patients received a dose of suvodirsen or placebo in five ascending dose cohorts and were followed for 85 days. No serious adverse events, deaths or discontinuations due to adverse events were reported in any study patients treated with suvodirsen. Suvodirsen was generally safe and well tolerated with 67% of patients who received suvodirsen and 80% of patients who received placebo experienced one or more adverse events. The most common adverse events occurring in two or more patients who received suvodirsen were pyrexia, headache, vomiting and tachycardia, consistent with infusion-associated reactions. Adverse events in patients receiving suvodirsen were mild to moderate in intensity and resolved spontaneously or with symptomatic treatment. No clinically relevant changes were observed in renal or hepatic parameters or platelet levels. In patients receiving 5 mg/kg of suvodirsen, the adverse events that occurred within 24 hours of infusion were associated with transient increases in high-sensitivity C-reactive protein and complement factor Bb levels, both of which were resolved within a week and no changes were observed in complement C3 levels. Based on results of the first four ascending dose cohorts, the independent safety monitoring committee of the Phase 1 clinical trial endorsed continued dose exploration by proceeding to the last planned cohort. Doses of 7 mg/kg or 10 mg/kg of suvodirsen were administered to two patients in the fifth cohort and were associated with similar adverse events as those observed at lower doses but were more severe in intensity. The full data from the Phase 1 clinical trial will be presented in an oral presentation and poster at the MDA conference.