Zinger Key Points
- Wave Life's WVE-N531 met all Phase 2 goals, showing exon skipping, dystrophin restoration.
- The therapy showed a 28.6% muscle fibrosis reduction, a 50% CK decline, and decreased IL-6/MCP-1, supporting its potential for DMD treatment
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Wave Life Sciences Ltd. WVE released 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.
DMD is the most common hereditary neuromuscular disease. Mutations in the dystrophin gene lead to progressive muscle fiber degeneration and weakness.
The company on Wednesday said FORWARD-53 achieved all trial goals, demonstrating sustained 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 company said the data demonstrate substantial decreases in inflammation and necrosis, a statistically significant reversal of muscle fibrosis (28.6% reduction between weeks 24 and 48), and a transition from muscle regeneration to maturation.
A 50% decline in creatine kinase (CK) and decreases in IL-6 and MCP-1 were also observed.
Time-to-Rise (TTR) data demonstrate a statistically significant and clinically meaningful 3.8-second improvement versus natural history.
Additional functional benefits were observed on the North Star Ambulatory Assessment (NSAA) compared to natural history and in-hand grip strength compared to baseline.
On Wednesday, Wave also announced that it met with the U.S. Food and Drug Administration (FDA) on WVE-N531 to discuss its interim 24-week data and initial plans for the confirmatory trial.
Wave intends to file a New Drug Application in 2026 for accelerated approval of WVE-N531.
WVE-N531, as well as Wave’s programs for exons 52, 51, 45, and 44, collectively could address around 40% of the DMD population and represent over $2.4 billion in total market opportunity in the United States alone.
Wave expects to submit multiple clinical trial applications for other exon-skipping programs in 2026.
Price Action: WVE stock is up 14.9% at $10.85 at the last check on Wednesday.
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