Alnylam Reports Mutant Protein Reduction in Highest-Dose Trial Group

Alnylam Pharmaceuticals announced Friday, Oct. 23, that its investigational Huntington’s disease drug brezosiran lowered mutant huntingtin in spinal fluid in the highest-dose group, an early sign of target engagement that does not show whether treatment slows the disease. That group had a mean reduction of 60% to 64% from baseline three months after one intrathecal dose. The largest individual reduction was 83%. These biomarker results do not establish a clinical benefit or slower disease progression. The interim results covered 48 participants across five single-dose cohorts. A sixth cohort of 12 people was still enrolling and had not been evaluated.

Source: TokenPost