Wave Life Sciences: No Clinical Benefit Observed in Phase 1b/2a Trial for ALS and Frontotemporal Dementia Treatment

Clinical stage RNA medicines company, Wave Life Sciences (Nasdaq: WVE) has failed to meet primary endpoints in a Phase 1b/2a clinical trial investigating a potential treatment for Amyotrophic Lateral Sclerosis (ALS) and frontotemporal dementia (FTD).

The company said it was “deeply disappointed that we were not able to see any evidence of potential benefits that would be expected to drive meaningful outcomes for these patients,” and thanked participants, their families, the clinical sites, and its study advisory committees for their participation and support.

The Alzheimer’s Drug Discovery Foundation provided support for the company’s FOCUS-C9 clinical trial.

Highlights 

The FOCUS-C9 trial is a global, multicenter, randomized, double-blind, placebo-controlled Phase 1b/2a clinical trial evaluating the safety and tolerability of WVE-004 in patients with C9-ALS and/or C9-FTD.

WVE-004 was found to be safe and well-tolerated across the trial’s single and multi-dose cohorts.

There was no clinical benefit observed in WVE-004-treated participants compared with placebo in either ALS or FTD.

Wave has decided to discontinue development of WVE-004, including stopping the FOCUS-C9 study and the open label extension study. A webcast of the company’s call with investors regarding the trial results can be accessed at: https://ir.wavelifesciences.com/events-and-presentations.

About Wave Life Sciences

Wave Life Sciences (Nasdaq: WVE) is a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future.

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Wave Life Sciences: No Clinical Benefit Observed in Phase 1b/2a Trial for ALS and Frontotemporal Dementia Treatment

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