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An early-stage Alzheimer’s disease treatment from Alnylam Pharmaceuticals showed promising signs of benefit in a small clinical trial, the company said Wednesday, suggesting the medicine might be able to prevent the toxic brain plaques thought to drive the disease.

Unlike recently approved medicines that clear out those plaques, called beta-amyloid, Alnylam’s treatment is designed to silence the gene responsible for their production. In a Phase 1 study enrolling 20 patients with early-onset Alzheimer’s, a single dose of the therapy was well tolerated, the company said, and led to lasting reductions of APP, the protein that folds into amyloid plaques. Side effects of the treatment were mild or moderate, Alnylam said. The company said it would withhold detailed data on the drug until a later scientific meeting.


Treatment with the therapy, which is injected into the spine, reduced APP by a maximum of about 90%, the company said. Patients who received the highest dose saw their APP decline by a median of 70% for at least three months, according to Alnylam.

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