Wave Life Sciences' Stock Falls: Investigational Therapy Fails To Show Clinical Benefit In Rare Neurological Diseases

Wave Life Sciences Ltd WVE announced topline results from the Phase 1b/2a FOCUS-C9 study evaluating WVE-004 as an investigational treatment for C9orf72-associated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) (C9-ALS/FTD). 

WVE-004 did not show clinical benefit compared to placebo; poly(GP) reductions did not correlate with clinical outcomes. Wave will discontinue the development of WVE-004.

The company says robust, sustained reductions in poly(GP) from baseline were observed, with a maximal mean reduction of 48% (p<0.0001) in the Q12W dose and 50% (p=0.0001) in the Q4W dose of WVE-004. 

Poly(GP) is a pharmacodynamic biomarker indicating WVE-004 lowers C9orf72 hexanucleotide repeat expansion (G4C2) transcripts, which are hypothesized to contribute to pathogenesis in C9-ALS/FTD. 

WVE-004 was generally safe and well-tolerated across doses, with most adverse events presenting as mild in intensity. 

There were no clinically meaningful changes in cerebrospinal fluid (CSF) protein or white blood cell count and no new safety signals since the previous data update in April 2022.

Wave remains on track to share data from its Phase 1b/2a SELECT-HD study in Huntington's disease of WVE-003 in 2H of 2023. 

The company is also rapidly advancing WVE-N531 for Duchenne muscular dystrophy, amenable to exon 53 skipping into the potentially registrational Part B (Phase 2) clinical study.

Price Action: WVE shares are down 15.10% at $3.15 during the premarket session on the last check Tuesday.