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
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
Wave life sciences宣佈，其WVE-003用於亨廷頓病的1B/2A SELECT-HD試驗結果顯示，突變亨廷頓蛋白降低了46％，同時對野生型亨廷頓蛋白進行了保存。
- 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
- 統計學上有意義、強效且持久的等位選擇性沉默：與安慰劑相比，30毫克多劑量隊列平均降低了46%腦脊液突變獵頭蛋白（mHTT），維持野生型獵頭蛋白（wtHTT），並且普遍安全和耐受的譜系。
- 突變獵頭蛋白降低與尾狀核萎縮減緩之間存在顯著相關性 - 這是一個預測臨床結果的成像生物標誌物。
- 揮手邀請監管機構參與WVE-003的臨床開發計劃，以支持潛在的加速批准，並將其選擇性收入方案提交給方案夥伴武田公司。
- 數據進一步驗證了Wave的RNA藥物平台，包括PN化學和流水線; Wave仍然在計劃在2024年第3季度爲DMD中的WVE-N531提供6個月的dystrophin數據，並在2024年爲AATD中的WVE-006提供RNA編輯機制驗證數據，並在2025年第1季度開始進行其INHBE GalNAc siRNA（WVE-007）用於肥胖的臨床試驗。
譯文內容由第三人軟體翻譯。
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