Wave Life Sciences Announces Initiation of Dosing in Phase 1b/2a FOCUS-C9 Clinical Trial of WVE-004 in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

Excerpt from the Press Release:

CAMBRIDGE, Mass., July 20, 2021 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced that multiple participants have initiated dosing in the Phase 1b/2a FOCUS-C9 clinical trial evaluating WVE-004 as an investigational treatment for C9orf72-associated amyotrophic lateral sclerosis (C9-ALS) and frontotemporal dementia (C9-FTD). WVE-004 is a stereopure antisense oligonucleotide designed to selectively target transcriptional variants containing a hexanucleotide repeat expansion (G₄C₂) associated with the C9orf72 gene, thereby sparing C9orf72 protein. G₄C₂ expansions in C9orf72 are one of the most common genetic causes of the sporadic and inherited forms of ALS and FTD.

“ALS and FTD are devastating illnesses where therapeutic progress has been extremely limited. Advancing discovery and development of new treatments for ALS and FTD is an urgent need that requires creativity, expediency and innovative thinking,” said Michael Panzara, MD, MPH, Chief Medical Officer and Head of Therapeutics Discovery and Development at Wave Life Sciences. “The predicted pharmacology of WVE-004, afforded by PN chemistry and based upon in vivo models, allowed us to design FOCUS-C9 to be adaptive, enabling data-driven decisions regarding dose level and frequency as the trial proceeds and potentially accelerating time to proof-of-concept. Opening the FOCUS-C9 trial to those diagnosed with C9orf72-associated ALS or FTD may also facilitate the ability to pursue both indications in the future. We anticipate generating clinical data in 2022 that will enable decision-making on next steps for the program.”

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