Paul Bolno, MD, President and Chief Executive Officer of Wave Life Sciences, discusses positive results from the FORWARD-53 clinical trial evaluating WVE-N531 in patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.
DMD affects the muscles, leading to muscle wasting that gets worse over time. DMD occurs primarily in males, though in rare cases may affect females. The symptoms of DMD include progressive weakness and atrophy of both skeletal and heart muscles. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. DMD is caused by genetic changes in the Dystrophin gene.
The FORWARD-53 study is a phase 2 clinical trial evaluating WVE-N531 in patients with DMD who are amenable to exon 53 skipping. WVE-N631 is an exon skipping oligonucleotide. The analysis of the study was conducted after 48 weeks of treatment with a 10 mg/kg dose of WVE-N531 every two weeks.
Observations from this study revealed significant improvements in muscle health with exon skipping, with a 28.6% reduction in fibrosis and transition from regenerative to mature muscle. A statistically significant and clinically meaningful improvement of 3.8 seconds in Time-to-Rise vs natural history was also observed. Additional functional benefits were observed on the North Star Ambulatory Assessment (NSAA) versus natural history, and in hand grip strength versus baseline.
Dystrophin expression was stabilized between 24 and 48 weeks and averaged 7.8%, with 88% of participants above 5% average dystrophin. A 50% decline in creatine kinase, as well as decreases in IL-6 and MCP-1, were also observed. Additionally, the safety and tolerability profile of WVE-N531 was favorable with no serious adverse events
A New Drug Application for accelerated approval is expected to be filed in 2026.
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To learn more about DMD and other rare musculoskeletal conditions, visit https://checkrare.com/diseases/musculoskeletal-diseases/
