Alnylam’s Huntington’s drug brezosiran shows promising results
The intrathecal therapy lowered mutant huntingtin protein by up to 64 % in a small phase‑I study.
23 Oct 2026 0 reads Compiled with AI
#Alnylam #brezosiran #Huntington’s disease
In a phase‑Ib trial, 48 adults with early Huntington’s disease received single doses of the intrathecal RNAi drug brezosiran (ALN‑HTT02). The highest dose lowered the mutant huntingtin protein in cerebrospinal fluid by 60–64 % on average, with a single patient achieving an 83 % reduction, according to data presented at the European Huntington’s Disease Network conference.
The company said patients tolerated the drug well and the study did not show a dose‑dependent rise in inflammatory markers, neurofilament light protein or brain ventricular volume. The results support the idea that brezosiran could be dosed twice a year, and Alnylam plans a placebo‑controlled pivotal study to start in 2027.
The therapy targets exon 1 of the HTT mRNA, using interference to silence the mutant gene that drives the disease. Early data suggest durable knockdown of the protein, a key goal for treating this fatal neurodegenerative disorder.