Alnylam Pharmaceuticals, Inc.ALNY

Alnylam Pharmaceuticals, Inc. (ALNY) focuses on neurology. Their drug Amvuttra (vutrisiran) is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. In the HELIOS-A trial, 164 patients were randomized. The incidence of Infusion Related Reactions (IRRs) for patisiran in HELIOS-A was 23.8%. In Study 004, the IRR in the patisiran-LNP group was 18.9%, while in the placebo group it was 9.1%. A total of 223 IRR symptoms (events) were observed in the patisiran-LNP group, compared to 105 events in the placebo group. In the ALN-TTR02-004 Safety Population, 18.9% of patients in the patisiran-LNP group experienced at least 1 IRR, while 9.1% of patients in the placebo group experienced at least 1 IRR.

Safety findings for vutrisiran in HELIOS-A included 4 patients with Grade 3 abnormalities in chemistry parameters and 2 subjects with Grade 3 haematology abnormalities. Additionally, 3 subjects in the vutrisiran group had Grade 3 abnormalities in coagulation parameters. For patisiran, 3 subjects in HELIOS-A had Grade 3 chronic kidney disease, and 2 subjects in HELIOS-A had Grade 3 chronic kidney disease with vutrisiran. In Study 004, 0 patients had platelet counts <50 x10^9/L (CTCAE Grade 3).