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Wave Skips Towards Approval With Impressive DMD Data

 
• By Elizabeth Cairns

The exon 53 skipper WVE-N531 looks highly competitive with approved therapies, but a biomarker hit might not correlate with functional improvement.    

DNA helix colorful genes chromosomes DNA sequence, DNA structure with glow.
• Source: Alamy

With two exon 53 skippers already approved for Duchenne muscular dystrophy (DMD), any new candidate was going to have to post remarkable data in order to have a chance of competing. Fortunately for Wave Life Sciences Ltd., WVE-N531 has done just that, and the company’s NASDAQ-listed shares soared by 53% to close at $8.19 on 24 September.

Key Takeaways
  • Interim Phase II data on Wave Life Sciences’ WVE-N531 suggests it boosts dystrophin expression in Duchenne muscular dystrophy patients, potentially teeing up accelerated approval.
  • WVE-N531’s safety and...

Interim data from 11 boys enrolled in the open-label FORWARD-53 trial show impressive efficacy for WVE-N531 after six months’ treatment. The data could allow the company to approach regulators about...

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