- 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 Life Sciences Announced Results From Its Phase 1B/2A SELECT-HD Trial Of WVE-003 For Huntington's Disease, Demonstrating Mutant Huntingtin Lowering Of 46%, With Preservation Of Wild-type Huntingtin
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