Wave's DMD Drug Shows Promise in Latest Phase 2 Data, FDA Submission on Horizon

Wave Life Sciences has unveiled encouraging 48-week data from its phase 2 trial of WVE-N531, an exon skipping oligonucleotide therapy for Duchenne muscular dystrophy (DMD). The results demonstrate significant improvements in dystrophin production and muscle health, positioning the company for a potential FDA submission next year.

Dystrophin Production Exceeds Target Threshold

The Forward-53 trial, involving 11 boys with exon 53 DMD, showed a mean absolute unadjusted dystrophin level of 6.4% in eight participants. Notably, seven of these eight boys achieved greater than 5% average dystrophin between 24 and 48 weeks, surpassing Wave's previously established target.

"WVE-N531's demonstrated ability to reach both myofibers and myogenic stem cells—the regenerative muscle cells—and sustain dystrophin restoration over time is impacting muscle health in ways never before seen in DMD," stated Wave CEO Paul Bolno, M.D.

Improvements in Muscle Health and Functionality

Beyond dystrophin production, the trial revealed significant enhancements in muscle health indicators:

• A 28.6% reduction in median muscle necrosis between weeks 24 and 48
• A decrease in median muscle necrosis and inflammation scores from 2 to 1
• A statistically significant 3.8-second improvement in the time taken to rise from the floor in 10 of the participants

Analysts from Leerink Partners noted that the 3.8-second improvement surpasses the 2.7-second benefit observed one year after administration of Sarepta's DMD gene therapy Elevidys.

Regulatory Path and Market Response

With these promising results, Wave Life Sciences is preparing to file its first New Drug Application (NDA) with the FDA. "We are now moving toward our first NDA filing, which puts us on the path toward becoming a commercial company," Bolno added.

The market responded positively to the announcement, with Wave's shares rising 19% in premarket trading on March 26, reaching $11.23 from the previous day's closing price of $9.44.

As Wave looks to expand its DMD treatment portfolio, the company is planning to seek permission for testing treatments targeting other exons beyond exon 53. Leerink analysts suggest that while exon 53 appears easier to skip, dystrophin expression seems more challenging, making the current data particularly impressive and potentially de-risking efforts for other exons where even greater expression might be achieved.