Wave Life Sciences (NASDAQ:WVE) has announced positive interim data from its Phase 2 FORWARD-53 clinical trial investigating WVE-N531 for Duchenne muscular dystrophy amenable to exon 53 skipping.
The interim analysis conducted after 24 weeks of 10 mg/kg dosing every two weeks demonstrated substantial dystrophin expression and indicated that the treatment was safe and well-tolerated.
"The high and consistent dystrophin levels at this interim timepoint are compelling and speak to the potential of WVE-N531 for boys amenable to exon 53 skipping, where better therapeutic options are urgently needed," said Anne-Marie Li-Kwai-Cheung, Chief Development Officer at Wave Life Sciences.
Wave plans to complete the trial and obtain feedback from regulators on potential accelerated approval in the first quarter of 2025.