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Wave Life Sciences Announces Topline Results from Phase 1b/2a FOCUS-C9 Study of WVE-004 for C9orf72-associated Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Potent and durable target engagement observed across cohorts, including with 10 mg doses administered every 12 weeks which were also generally safe and
About this update from Wave Life Sciences Ltd.
[{"type":"text","content":"Potent and durable target engagement observed across cohorts, including with 10 mg doses administered every 12 weeks which were also generally safe and well-tolerated WVE-004 did not show clinical benefit compared with placebo; additionally, poly(GP) reductions did not correlate with clinical outcomes -- Wave to discontinue development of WVE-004 Wave to host investor conference call at 8:30 a.m. ET today CAMBRIDGE, Mass., May 23, 2023 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases, today 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). The results include data from a planned analysis of the study, where participants received multiple 10 mg doses of WVE-004 or placebo every 12 weeks (Q12W) or every 4 weeks (Q4W), as well as an additional 20 mg single dose cohort. 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. Robust, sustained reductions in poly(GP) from baseline were observed, with a maximal mean reduction of 48% (p","length":1763,"tagName":"div"}]