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Investing.com -- Shares of Wave Life Sciences Ltd . (NASDAQ:WVE) rose 14% today, following the company’s announcement of positive results from its FORWARD-53 clinical trial in Duchenne muscular dystrophy (DMD), which showed significant functional benefits and reversal of muscle damage after 48 weeks of dosing with its investigational drug WVE-N531.
The Cambridge-based biotech firm revealed that the Phase 2 trial met all its goals, demonstrating sustained and leading exon skipping, muscle concentrations, and dystrophin restoration. The trial’s results also indicated a 61-day tissue half-life for the drug, which supports the potential for monthly dosing. The drug was reported to be safe and well-tolerated, with no Serious Adverse Events.
Notably, the trial showed a statistically significant and clinically meaningful improvement of 3.8 seconds in Time-to-Rise (TTR) compared to natural history, and additional functional benefits were observed in other outcome measures, including the North Star Ambulatory Assessment (NSAA). Moreover, the trial demonstrated a significant reduction in muscle fibrosis and decreases in creatine kinase and circulating inflammatory biomarkers.
Wave Life Sciences also intends to file a New Drug Application (NDA) for accelerated approval of WVE-N531 in 2026, following recent feedback from the FDA. The company plans to file clinical trial applications (CTAs) for multiple DMD candidates for other exons in 2026, with preclinical data supporting a best-in-class exon skipping franchise.
Jones Research analyst Catherine Novack commented on the results, stating, "Muscle-content adjusted dystrophin at 48 weeks was 6.4% vs 9.0% at 24 weeks (avg: 7.8%), with 88% of patients averaging above 5%. This falls below the 10% threshold that is expected to confer clinical benefit, but still significantly greater than other exon 53 skippers at 48 weeks. Time to rise from the floor improved by 3.8 seconds vs natural history. This is impressive, though we would like to see placebo-controlled functional data given the heterogeneity of the disease."
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