Wave Life Sciences announces data from Phase 2 FORWARD-53 trial of WVE-N531

Wave Life Sciences (WVE) announced positive data from the Phase 2 FORWARD-53 trial of WVE-N531, which is an exon skipping oligonucleotide being investigated in boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. The analysis was conducted after 48 weeks of treatment with 10 mg/kg WVE-N531 dosed every two weeks. FORWARD-53 achieved all trial goals, demonstrating sustained and industry-leading exon skipping, muscle concentrations and dystrophin restoration through 48 weeks and a 61-day tissue half-life that supports monthly dosing. Statistically significant and clinically meaningful improvement of 3.8 seconds in Time-to-Rise vs. natural history with largest effect observed relative to any approved dystrophin restoration therapy at 48 weeks; additional functional benefits observed in other outcome measures including NSAA. Additionally, the data demonstrate substantial decreases in inflammation and necrosis, a statistically significant reversal of muscle fibrosis, and a transition from regeneration to maturation of muscle. A 50% decline in creatine kinase, as well as decreases in IL-6 and MCP-1, were also observed. Time-to-Rise data demonstrate a statistically significant and clinically meaningful 3.8-second improvement versus natural history, which is the largest effect observed relative to any approved dystrophin restoration therapy at 48 weeks. Based on the FDA feedback and the 48-week data, Wave intends to file a New Drug Application in 2026 for accelerated approval of WVE-N531. The NDA filing will be based on all FORWARD-53 data, which will include additional data to support monthly dosing.