Alnylam says drug for Huntington's shows promise

Elaine’s stories explore the boom in new obesity treatments, new drug payment models, the ways companies are affected by FDA changes, and the emerging psychedelics drug sector. She co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70.

An experimental Huntington’s drug from Alnylam Pharmaceuticals showed promise in a small study, the company said Friday, suggesting it can partially silence the mutant gene that drives the devastating neurological disorder.

Patients who received the highest dose of the drug, called brezosiran, saw the levels of mutant huntingtin in their spinal fluid fall by 60% or 64% on average, depending on the type of assay used. The company described the safety profile as “encouraging” and said it planned to start a placebo-controlled study next year to prove the drug can actually slow the disease.

“We’re getting high levels of knockdown with good safety and tolerability, and so this is all very, very encouraging,” said Pushkal Garg, head of research and development at Alnylam, which presented the data Friday at the European Huntington’s Disease Network Clinical Research Congress.

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