Wave Life Sciences Announces Upcoming Presentations at MDA Conference that Highlight Best-in-Class Potential for WVE-N531 in Duchenne Muscular Dystrophy

Presentations include data for WVE-N531 that provide first clinical evidence of a potential therapeutic for DMD accessing satellite cells, which are important for potential muscle regeneration

Presentations also include non-human primate data demonstrating significant concentrations of WVE-N531 in heart, diaphragm and skeletal muscle, as well as preclinical data for potential future DMD programs targeting other exons

CAMBRIDGE, Mass., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced its upcoming presentations at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 3-6 in Orlando, FL.

Wave’s poster presentations will highlight the best-in-class potential of WVE-N531 in Duchenne muscular dystrophy (DMD), which is currently being evaluated in the Phase 2, potentially registrational FORWARD-53 study. The presentations will also illustrate the impact of Wave’s novel PN chemistry on pharmacology of its exon skipping oligonucleotides. Highlights from the presentations include:

• Data from the Phase 1b/2 proof-of-concept (Part A) study of WVE-N531 in boys with DMD amenable to exon 53 skipping, which demonstrate uptake of WVE-N531 in satellite cells of all participants in the study. Satellite cells, or muscle stem cells, are important for muscle regeneration, and this is the first clinical evidence of satellite cell uptake for any investigational or approved DMD therapeutic.
• Preclinical data for WVE-N531 in non-human primates, which demonstrate that Wave’s PN chemistry significantly enhanced drug concentrations in skeletal muscle, with even higher exposure in the heart and diaphragm. These data suggest that WVE-N531 muscle concentrations in the clinic may be higher in heart and diaphragm than in skeletal muscle. In the previous Phase 1b/2 Part A study, WVE-N531 demonstrated high skeletal muscle concentrations of 42 μg/g (42,000 ng/g) after three every-other-week doses, which translated to best-in-class exon skipping (mean of 53%).
• Preclinical data for Wave’s exon skipping programs beyond exon 53, which reinforce the impact of PN chemistry for enabling high tissue concentrations, exon skipping and dystrophin restoration in preclinical models. Success with WVE-N531 would unlock a multiexon strategy where Wave can potentially address up to 40% of the DMD population with its current DMD pipeline, which includes discovery programs for skipping exons 51, 52, 44 and 45, in addition to exon 53 with WVE-N531.