Alnylam Pharmaceuticals (ALNY) said Sunday its phase 1 study of nucresiran as a potential treatment for transthyretin amyloidosis showed the drug's efficacy.
Transthyretin amyloidosis is a condition characterized by the buildup of abnormal protein deposits in the body's organs and tissues.
The results showed that a single dose of the drug reduced transthyretin protein levels by more than 90% at day 15 and more than 70% after 360 days, with nucresiran being well tolerated at all tested doses, the company said.
Alnylam plans to share phase 3 testing plans in Q1.
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