Health · Pharma & Biotech · 1 hr ago
Alnylam plans larger study of Huntington’s disease drug
Alnylam’s experimental drug brezosiran showed promise in a small study involving people with Huntington’s disease.
The disorder is driven by a mutant gene that produces huntingtin, a protein the drug is designed to reduce.
At the highest dose, patients’ spinal fluid showed an average 60% or 64% drop in mutant huntingtin, depending on the test used.
Alnylam described the drug’s safety profile as encouraging, but the early results do not show that it slows the disease.
The company presented the findings at the European Huntington’s Disease Network Clinical Research Congress.
Alnylam plans a placebo-controlled study next year to test whether the drug can slow the disease.
Alnylam said its experimental Huntington’s disease drug brezosiran showed promise in a small study.
At the highest dose, patients had an average 60% or 64% fall in mutant huntingtin levels in spinal fluid, depending on the assay.
The company said it plans to start a placebo-controlled study next year to test whether the drug can slow the disease.
Alnylam presented the data at the European Huntington’s Disease Network Clinical Research Congress.
- Who
- Alnylam Pharmaceuticals and patients in a small study of its experimental drug brezosiran.
- What
- The drug showed promise, reducing mutant huntingtin levels in spinal fluid among patients who received the highest dose.
- When
- The company reported the results on Friday, October 23, 2026.
- Where
- The data were presented at the European Huntington’s Disease Network Clinical Research Congress.
- Why
- Alnylam plans a placebo-controlled study to test whether the drug can slow Huntington’s disease.
This story does not have two clearly opposing sides.
Alnylam presented early-study data at the European Huntington’s Disease Network Clinical Research Congress.
Alnylam plans to start a placebo-controlled study to test whether brezosiran can slow Huntington’s disease.
- Drug
- Brezosiran
- Highest dose result
- Mutant huntingtin levels in spinal fluid fell by 60% or 64% on average, depending on the assay.
- Next study
- Placebo-controlled study planned for next year.
- Presentation
- European Huntington’s Disease Network Clinical Research Congress
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What people are saying
We’re getting high levels of knockdown with good safety and tolerability, and so this is all very, very encouraging