Alnylam reports up to 83% huntingtin reduction
Pontos principais
- Alnylam Pharmaceuticals reported initial Phase 1b results for brezosiran (ALN-HTT02), showing mean reductions of 60%–64% in cerebrospinal fluid mutant huntingtin protein after three months at the highest dose evaluated.
- Individual reductions reached 83%, while early safety findings showed no dose-dependent increases in monitored inflammatory markers, neurofilament light chain or brain ventricular volume.
- Alnylam plans to initiate a placebo-controlled pivotal Huntington's disease trial in 2027, subject to regulatory alignment, and will discuss the program at its October 26 RNAi Roundtable.
Alnylam Pharmaceuticals (NASDAQ:ALNY) reported initial Phase 1b results showing that brezosiran reduced cerebrospinal fluid mutant huntingtin protein by an average of 60%–64% after three months in the highest dose group evaluated.
Individual protein reductions reached 83%, with dose-dependent effects observed across five treatment cohorts and confirmed using two independent assays.
“The deep and durable mutant huntingtin lowering observed after a single dose, together with the encouraging safety profile seen to date, give us confidence to advance brezosiran into a pivotal study,” said Alnylam Chief Research and Development Officer Pushkal Garg.
Participants followed for up to 12 months showed no dose-dependent increases in cerebrospinal fluid inflammatory markers, neurofilament light chain or brain ventricular volume.
Alnylam plans a placebo-controlled pivotal trial in 2027, pending regulatory alignment, to assess whether brezosiran can slow Huntington's disease progression.
The investigational RNA interference therapy targets both full-length mutant huntingtin and the shorter mutant HTT1a protein, with the company exploring twice-yearly dosing.
Eligible participants can enter a 36-month open-label extension receiving treatment every six months, while Alnylam has scheduled an RNAi Roundtable for October 26, 2026.
