Alnylam's Huntington's Drug Cuts Huntingtin 60% — SkimNews
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- Alnylam said Friday that its experimental Huntington's drug showed promise in a small study, suggesting it can partially silence the mutant gene that drives the neurological disorder.
- Brezosiran at its highest dose lowered mutant huntingtin levels in patients' spinal fluid by 60% or 64% on average, depending on the assay used.
- Alnylam described the safety profile as "encouraging" and said it plans to start a placebo-controlled study next year to show whether brezosiran can actually slow the disease.
- Pushkal Garg, Alnylam's head of research and development, said the company is seeing "high levels of knockdown with good safety and tolerability."
- European Huntington's Disease Network: Alnylam presented the data Friday at the network's Clinical Research Congress.
Why it matters: For Alnylam, the 60%-64% cut in spinal-fluid mutant huntingtin at the top dose provides the biomarker case for a placebo-controlled trial starting next year; for Huntington's patients, that trial, not this small study, is what establishes whether the drug actually slows the disease.
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