Alpha-1 antitrypsin deficiency (AATD), Huntington’s disease (HD), obesity, and Duchenne muscular dystrophy (DMD) are among the major unmet medical needs that the company seeks to solve. The news was released prior to the company’s January 13, 2025, presentation at the 43rd Annual J.P. Morgan Healthcare Conference.
Plans for Exon 53 Skipping Study
- WVE-N531 is an exon skipping oligonucleotide designed to induce production of endogenous, functional dystrophin protein for the treatment of boys with DMD amenable to exon 53 skipping.
- Key achievements in 2024
- Interim results of the Phase 2 FORWARD-53 study of WVE-N531 showed highly consistent, mean muscle content-adjusted dystrophin expression of 9.0% (range: 4.6-13.9%), best-in-class muscle delivery, multiple indicators of improved muscle health, and a safe and well-tolerated profile.
- Expected upcoming milestones:
- The FORWARD-53 trial is ongoing and all patients have elected to continue treatment in the planned extension portion of the study with monthly doses of WVE-N531.
- Wave expects to deliver the 48-week FORWARD-53 data and receive feedback from regulators on a pathway to accelerated approval in the first quarter of 2025.