WASHINGTON (dpa-AFX) - Alnylam Pharmaceuticals (ALNY) reported positive initial Phase 1b clinical trial results for ALN-HTT02, an investigational RNAi therapeutic in development for the treatment of Huntington's disease. ALN-HTT02 will be developed under the International Nonproprietary Name brezosiran. In the ongoing Phase 1b study, participants on single ascending intrathecal doses of brezosiran experienced robust, dose-dependent reductions of mutant huntingtin protein in cerebrospinal fluid.
Alnylam plans to initiate a placebo-controlled study in 2027, pending regulatory alignment, to evaluate the ability of brezosiran to slow disease progression in adults with Huntington's disease.
At last close on NasdaqGS, Alnylam shares were trading at $227.28.
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