Wave Life Sciences (WVE) shares were higher premarket Wednesday after the company reported "positive" 48-week data from a phase 2 trial of its WVE-N531 drug candidate in boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping.
The biotechnology company said the boys received 10 milligrams of the exon skipping oligonucleotide every two weeks for each kilogram of their weight, with WVE-N531 meeting all trial goals, including sustained and industry-leading exon skipping, muscle concentrations and dystrophin restoration,
WVE-N531 also demonstrated a 61-day tissue half-life support in monthly dosing. It also continues to be safe and well-tolerated, the company said.
Wave also said it recently met with the US Food and Drug Administration to discuss its interim 24-week data and initial plans for a confirmatory phase 3 trial, with the agency confirming WVE-N531 remains on an accelerated approval pathway using dystrophin expression as a surrogate endpoint.
The company also expects to file a new drug application in 2026 for accelerated approval of WVE-N531 based on FDA feedback and the new 48-week data.
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