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.
Wave Skips Towards Approval With Impressive DMD Data
The exon 53 skipper WVE-N531 looks highly competitive with approved therapies, but a biomarker hit might not correlate with functional improvement.
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