Wave down on Phase I safety data for DMD therapy

Following a setback last week for its Huntington disease program, Wave lost $9.55 (28%) to $24.47 on Tuesday after the company announced plans to test two lower doses of suvodirsen in a Phase II/III trial after final Phase I data revealed toxicity concerns with higher doses of the Duchenne muscular dystrophy candidate.

Wave Life Sciences Ltd. (NASDAQ:WVE) said doses of up to 5 mg/kg IV suvodirsen (WVE-210201) were generally well tolerated in the Phase I trial in 36 boys with DMD amenable to exon 51 skipping. Patients received placebo or a single dose of 0.5, 1, 2, 5, 7 or 10 mg/kg suvodirsen. At the 5 mg/kg dose, patients reported adverse events within 24 hours of infusion that were associated with transient increases in high-sensitivity C-reactive protein (hsCRP) and complement factor Bb levels; both resolved within a week. At the 7 and 10 mg/kg doses, patients reported similar adverse events but with increased severity...