Health

STAT+: Alnylam says Huntington’s drug shows promise in early study

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Briefings

Alnylam Pharmaceuticals reported that brezosiran, an experimental Huntington's drug, reduced mutant huntingtin levels in spinal fluid by 60% to 64% in a small study. The company described the safety profile as encouraging and plans a placebo-controlled trial next year to confirm disease-slowing effects. The findings were presented at the European Huntington's Disease Network Clinical Research Congress on October 23, 2026.

Key points

• Brezosiran reduced mutant huntingtin levels in spinal fluid by 60% to 64% in a small study.
• Alnylam plans a placebo-controlled trial next year to prove the drug slows Huntington's disease progression.

Context

Alnylam Pharmaceuticals presented data at the European Huntington's Disease Network Clinical Research Congress on October 23, 2026, confirming that brezosiran shows promise in silencing the mutant gene driving Huntington's disease.

Limitations

The study was small and conducted only in spinal fluid, limiting generalizability. The company's safety profile is described as encouraging but not yet proven to slow disease progression in larger trials.

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