Alnylam Announces Positive Initial Phase 1b Data for Brezosiran (ALN-HTT02) Demonstrating Deep and Durable Huntingtin Lowering in Huntington’s Disease
Alnylam reported positive initial Phase 1b data for brezosiran (ALN-HTT02), an investigational RNAi therapeutic for Huntington's disease, with mean CSF mutant huntingtin reductions of 60-64% at month 3 and up to 83% in individuals in the highest dose group evaluated. The data, presented at the EHDN Clinical Research Congress in Krakow, show an encouraging safety profile through up to 12 months of follow-up with no dose-dependent increases in CSF inflammatory markers, NfL, or brain ventricular volume, supporting potential twice-yearly dosing.
Key figures
- Drug Name
- Brezosiran (ALN-HTT02)
- Phase Of Trial
- Phase 1b
- Dosing potential
- twice-yearly
- Ole duration months
- 36
- Pivotal study start
- 2027
- Enrolled participants
- 48
- Dose cohorts evaluated
- 5
- Csf mhtt mean reduction
- 60-64%
- Csf mhtt max individual reduction
- 83%
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