STAT News· Jason Mast and Elaine Chen·· 2 hours agoPickScore52
STAT+: Alnylam says Huntington’s drug shows promise in early study
In brief
Alnylam reported results from a small early study of brezosiran, in which the highest dose reduced mutant huntingtin levels in cerebrospinal fluid by an average of 60% or 64%. The company described the drug as having good safety and tolerability and plans to start a placebo-controlled study next year to test whether it can slow disease progression. The data were presented at the European Huntington's Disease Network Clinical Research Congress.
Why it matters
Highest-dose CSF mutant huntingtin fell 60% to 64%, with safety and tolerability reported, setting an early baseline for a placebo-controlled trial.
Source: STAT News · statnews.com
