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Wave Life Sciences Receives FDA Rare Pediatric Disease Designation for WVE-N531 for the Treatment of Duchenne Muscular Dystrophy
Designation highlights significant unmet needs in DMD; dystrophin data from potentially registrational FORWARD-53 trial of WVE-N531 are on track for 3Q 2024
About this update from Wave Life Sciences Ltd.
[{"type":"text","content":"Designation highlights significant unmet needs in DMD; dystrophin data from potentially registrational FORWARD-53 trial of WVE-N531 are on track for 3Q 2024 In previous Part A trial, WVE-N531 achieved industry-leading exon skipping of 53% and muscle tissue concentrations that were approximately 20-30 times higher than those reported by exon skipping technologies leveraging muscle delivery conjugates CAMBRIDGE, Mass., Aug. 12, 2024 (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 that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to WVE-N531 for the treatment of boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. WVE-N531 is currently being evaluated in the potentially registrational FORWARD-53 clinical trial and Wave expects to deliver data, including dystrophin protein expression from muscle biopsies after 24 weeks of treatment, in the third quarter of 2024. “This designation from FDA underscores that significant unmet needs remain in DMD, and it also supports Wave’s innovative and purposeful approach to drug development in the rare disease space,” said Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, Chief Development Officer at Wave Life Sciences. “With our WVE-N531 program, we are aiming to restore clinically meaningful levels of near full length, functional dystrophin protein. Positive data from FORWARD-53 would also unlock additional programs for other exons in our pipeline, with the goal of developing best-in-class medicines that address the underlying cause of the disease for up to 40% of boys with DMD.” WVE-N531 is an exon skipping oligonucleotide designed to induce production of endogenous, functional dystrophin protein. In the previously completed Part A study (three 10 mg/kg doses every other week), WVE-N531 achieved industry-leading mean exon skipping levels of 53% and mean muscle tissue concentrations of ~42,000 ng/g (~6,100 nM), which is approximately 20-30 times higher than levels reported by exon-skipping technologies leveraging muscle delivery conjugates in DMD patients. The Part A data also demonstrated distribution to myogenic stem cells (also known as satellite cells) in all study partici...