Wave Life Sciences Achieves Breakthrough in Huntington’s Disease Treatment with WVE-003, Eyes Accelerated Approval

1. Positive Results: Wave Life Sciences announced positive results from its Phase Ib/IIa SELECT-HD trial, demonstrating a 46% mean reduction in mutant huntingtin (mHTT) protein levels in patients with Huntington's disease.
2. Allele-Selective Silencing: WVE-003, an investigational antisense oligonucleotide, achieved statistically significant and durable allele-selective silencing of mHTT, preserving wild-type huntingtin (wtHTT) protein levels.
3. Correlation with Caudate Atrophy: The reduction in mHTT levels was significantly correlated with the slowing of caudate atrophy, a known imaging biomarker of clinical outcomes.
4. Accelerated Approval: Wave is preparing to engage regulators for a potential accelerated approval of WVE-003.
5. Pipeline Progress: The company remains on track to deliver 6-month dystrophin data for WVE-N531 in Duchenne muscular dystrophy (DMD) in 3Q 2024 and RNA editing proof-of-mechanism data for WVE-006 in alpha-1 antitrypsin deficiency (AATD) in 2024.
6. Cash Runway: Wave's current cash and cash equivalents are expected to be sufficient to fund operations into the fourth quarter of 2025.