Alnylam reports Phase 1b data showing 60‑64% huntingtin reduction and plans 2027 pivotal trial
Alnylam Pharmaceuticals presented initial Phase 1b results for its investigational Huntington’s disease RNAi therapy brezosiran (ALN‑HTT02). Single ascending intrathecal doses produced a mean 60‑64% reduction of mutant huntingtin protein in cerebrospinal fluid, with a maximum individual drop of 83%. The company said a placebo‑controlled pivotal study is slated for 2027, subject to regulatory alignment. The data were discussed at an Alnylam RNAi Roundtable on 26 October 2026.
Why it matters
The early proof‑of‑concept suggests the program could become a disease‑modifying asset for Huntington’s disease, potentially adding a high‑value product to Alnylam’s pipeline. The company’s statement that a pivotal trial will follow in 2027 signals continued R&D spending and future regulatory milestones.
Key facts
- 1Mean reduction of cerebrospinal fluid mutant huntingtin protein was 60‑64% at month 3 in the highest dose group. finanznachrichten.de
- 2Maximum individual reduction of mutant huntingtin protein reached 83%. stocktitan.net
- 3The Phase 1b study enrolled 48 participants across five single‑dose cohorts. marketscreener.com
- 4A placebo‑controlled pivotal study is planned for 2027, pending regulatory alignment. stocktitan.net
- 5Alnylam will discuss the Huntington’s disease program at its RNAi Roundtable on 26 October 2026. stocktitan.net
Summary written by AlphAI AI Desk from 5 of 5 sources. Not investment advice. Figures are as stated by the linked sources.