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
First-ever demonstration of substantial improvements in muscle health with exon skipping – statistically significant reduction in fibrosis driven by decreases in inflammation and necrosis, coupled with transition from regenerative to mature muscle; decreases in creatine kinase and circulating inflammatory biomarkers
Dystrophin expression stabilized between 24 and 48 weeks and averaged 7.8%, with 88% of boys above 5% average dystrophin; WVE-N531 remains safe and well-tolerated with no Serious Adverse Events
Following recent feedback from FDA, Wave intends to file a New Drug Application in 2026 for accelerated approval, with data to support monthly dosing at launch
Wave expects to file CTAs in 2026 for multiple DMD candidates for other exons, with preclinical data supporting a best-in-class exon skipping franchise
Wave to host investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., March 26, 2025 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today 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 (DMD) 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 (Q2W).
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. WVE-N531 continues to be safe and well-tolerated.
Additionally, the data demonstrate substantial decreases in inflammation and necrosis, a statistically significant reversal of muscle fibrosis (28.6% reduction between week 24 to 48; p