Alnylam to take Huntington's RNAi brezosiran into a placebo-controlled study
- Alnylam will advance its Huntington's disease RNAi drug brezosiran into a placebo-controlled study in 2027.
- Timing depends on first reaching regulatory alignment.
- BioSpace's headline describes the data as ‘best-in-class knockdown’; detailed figures were not in the feed text.
💡 Points
Knockdown of huntingtin is only the first hurdle; the pivotal trial will have to show clinical benefit, and that is where HTT-lowering programs have stumbled before. The regulatory-alignment caveat means the endpoint question is open. For the RNAi field, a CNS indication with strong target engagement would extend Alnylam's case beyond liver-centric products.
Alnylam plans pivotal trial as Huntington’s drug notches ‘best-in-class knockdown’ ↗