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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
First clinical dosing of a compound using PN backbone chemistry modifications Enrolling participants with C9-ALS, C9-FTD or mixed phenotype FOCUS-C9 is
About this update from Wave Life Sciences Ltd.
[{"type":"text","content":"First clinical dosing of a compound using PN backbone chemistry modifications Enrolling participants with C9-ALS, C9-FTD or mixed phenotype FOCUS-C9 is adaptive to enable rapid optimization of WVE-004 dosing Clinical data to enable decision-making on next steps for WVE-004 program expected in 2022 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.” C9-ALS and C9-FTD are believed to be caused by multiple factors related to the G4C2 expansion. The expansion may lead to accumulation of repeat-containing RNA transcripts and aberrantly translated dipeptide repeat proteins (DPRs) leading to neurotoxicity, as well as insufficient levels of C9orf72 protein, affecting ...