Wave Life Sciences: Phase 2 FORWARD-53 Achieves All Trial Goals - Quick Facts

(RTTNews) - Wave Life Sciences (WVE) reported 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 trial achieved all goals, demonstrating sustained exon skipping, muscle concentrations and dystrophin restoration through 48 weeks and a 61-day tissue half-life that supports monthly dosing.

Wave also announced that the company met with the FDA on WVE-N531 to discuss its interim 24-week data and initial plans for the confirmatory trial, where the Agency confirmed that the accelerated approval pathway using dystrophin expression as a surrogate endpoint remains open. Based on the FDA feedback and the 48-week data, Wave plans 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.

Shares of Wave Life Sciences are up 4% in pre-market trade on Wednesday.

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