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Wave Life Sciences Announces Positive Results from Phase 1b/2a SELECT-HD Trial with First Clinical Demonstration of Allele-Selective Mutant Huntingtin Lowering in Huntington’s Disease
Statistically significant, potent, and durable allele-selective silencing: 46% mean reduction in CSF mutant huntingtin (mHTT) protein compared to placebo,
About this update from Wave Life Sciences Ltd.
[{"type":"text","content":"Statistically significant, potent, and durable allele-selective silencing: 46% mean reduction in CSF mutant huntingtin (mHTT) protein compared to placebo, preservation of wild-type huntingtin (wtHTT) protein, and generally safe and well-tolerated profile achieved in 30 mg multidose cohort Statistically significant correlation between mHTT lowering and slowing of caudate atrophy - an imaging biomarker predictive of clinical outcomes Wave to engage regulators on a clinical development path for WVE-003 that would support a potential accelerated approval, and will submit its opt-in package to program partner Takeda Data provide further validation for Wave’s RNA medicines platform, including PN chemistry, and pipeline; Wave remains on track to deliver 6-month dystrophin data for WVE-N531 in DMD in 3Q 2024 and RNA editing proof-of-mechanism data for WVE-006 in AATD in 2024, as well as to initiate a clinical trial for its INHBE GalNAc siRNA (WVE-007) for obesity in 1Q 2025 Wave to host investor conference call and webcast at 8:30 a.m. ET today CAMBRIDGE, Mass., June 25, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced positive results from its Phase 1b/2a SELECT-HD clinical trial of WVE-003, which is being developed as a potential disease modifying therapeutic for Huntington’s disease (HD). WVE-003 is a first-in-class, allele-selective antisense oligonucleotide (ASO) designed to lower mutant huntingtin (mHTT) protein and preserve healthy, wild-type huntingtin (wtHTT) protein. In the multidose portion of the SELECT-HD study (n=23), participants received either every-eight-week (Q8W) intrathecal doses of 30 mg WVE-003 (n=16) or placebo (n=7), with 12 weeks of follow up (total study period of 28 weeks). Key results are as follows: WVE-003 was generally safe and well-tolerated, with no Serious Adverse Events (SAEs) reported; ventricular volume was in line with natural history.Significant mHTT protein lowering was observed throughout the 28-week assessment period. At 24 weeks (8 weeks after last dose), mean mHTT lowering in cerebrospinal fluid (CSF) was 46% versus placebo (p=0.0007).At 28 weeks (12 weeks after last dose), mean mHTT lowering in CSF was 44% versus placebo (p=0.0002), which ...