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Wave Life Sciences Announces Positive Update to Ongoing Phase 1b/2a FOCUS-C9 Study Driven by Potent, Durable Reductions of Poly(GP) with Low, Single Doses of WVE-004
Reductions in poly(GP), a key disease biomarker indicating target engagement, observed across all treatment groups after single doses Extending dose
About this update from Wave Life Sciences Ltd.
[{"type":"text","content":"Reductions in poly(GP), a key disease biomarker indicating target engagement, observed across all treatment groups after single doses Extending dose observation period from three months (day 85) to six months to identify the maximum reduction of poly(GP) and duration of effect of low, single doses Dosing in multidose 10 mg cohort well underway; longer-term follow-up from single dose cohorts and multidose data expected throughout 2022 First human data supporting preclinical to clinical translation of next-generation PN chemistry-containing molecules; clinical data from HD and DMD programs also expected in 2022 Wave to host investor conference call and webcast at 8:30 a.m. ET today CAMBRIDGE, Mass., April 04, 2022 (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 a positive update to the ongoing Phase 1b/2a FOCUS-C9 trial of WVE-004, the company’s clinical candidate for C9orf72-associated amyotrophic lateral sclerosis (C9-ALS) and frontotemporal dementia (C9-FTD). FOCUS-C9 (NCT04931862) is an adaptive trial that was designed to rapidly optimize dose level and frequency based on early indicators of target engagement. The trial update announced today is being driven by the observation of potent, durable reductions of poly(GP) dipeptide repeat proteins in cerebrospinal fluid (CSF) with low, single doses of WVE-004. Poly(GP) is a key C9-ALS/C9-FTD disease biomarker that, when reduced in CSF, indicates WVE-004’s engagement of target in the brain and spinal cord. “ALS and FTD are serious, life threatening disorders where advances in disease-modifying therapeutics have been extremely limited. While early, these data are encouraging and open an opportunity to target the disease at the RNA level,” said Merit Cudkowicz, MD, Chief of the Neurology Department, Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and chair of the FOCUS-C9 Clinical Advisory Committee. “Additionally, it is encouraging to see the benefits of the study’s adaptive design, where this early analysis has already helped narrow the doses being explored and enabled more precise, real-time exploration of dose response and optimization.” Reductions in poly(GP) were observed across all...