On Tuesday, Wave Life Sciences Ltd. ( WVE ) revealed interim data from the ongoing Phase 2 FORWARD-53 study of WVE-N531 for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.

DMD is a genetic disease that causes progressive muscle weakness and degeneration caused by a mutation in the dystrophin gene. It’s the most common inherited neuromuscular disease and affects boys more often than girls.

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Dystrophin is a protein that helps strengthen and protect muscle fibers.

Eleven boys amenable to exon 53 skipping (age 5-11; 10 ambulatory and one non-ambulatory) are enrolled.

Dystrophin results from a pre-specified analysis of ambulatory (who can walk) boys showed:

The mean absolute muscle content-adjusted dystrophin expression was 9.0%, and the mean absolute unadjusted dystrophin expression was 5.5% of normal.

89% of ambulatory participants achieved muscle content-adjusted dystrophin levels of at least 5%.

The mean exon skipping was 57%.

WVE-N531 was detected in myocyte nuclei in all participants and myogenic stem cells in most participants. Myogenic stem cells are the progenitor cells for new myoblasts, which give rise to new myocytes and ultimately aid in skeletal muscle regeneration.

Participants showed multiple indicators of improvement in muscle health.

Significant reductions were observed in serum biomarkers associated with muscle damage.

WVE-N531 was safe and well tolerated. Treatment-related adverse events (four events total in three participants) were mild in intensity.

There were no oligonucleotide class-related safety events.

In the first quarter of 2025, Wave expects to complete the FORWARD-53 trial and receive feedback from regulators on a pathway to accelerated approval.

Price Action: WVE stock is up 47.90% at $7.88 at the last check on Tuesday.

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