Press release
Alnylam Completes Enrollment in HELIOS-B Phase 3 Study of Investigational Vutrisiran in Patients with Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
− Full Study Enrollment Completed Well Ahead of Schedule − CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n− Full Study Enrollment Completed Well Ahead of Schedule −\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it has achieved full patient enrollment in its HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of cardiomyopathy in patients with transthyretin-mediated (ATTR) amyloidosis. Enrollment was completed, significantly ahead of schedule, with more than 600 ATTR amyloidosis patients across 123 activated sites in 32 countries.\n\nThe HELIOS-B study was designed to evaluate the safety and efficacy of investigational vutrisiran, an RNAi therapeutic subcutaneously administered once every three months for the potential treatment of cardiomyopathy in ATTR amyloidosis patients. The primary endpoint will evaluate the efficacy of vutrisiran versus placebo on the composite endpoint of all-cause mortality and recurrent cardiovascular (CV) events (CV hospitalizations and urgent heart failure visits) at 30 months in ATTR amyloidosis patients with cardiomyopathy.\n\n“Today’s milestone represents another exciting step forward as we advance the investigation of RNAi therapeutics like vutrisiran in ATTR amyloidosis patients with cardiomyopathy. We reached full study enrollment for HELIOS-B, our largest clinical trial to date, approximately 20 months from study initiation, reflecting the high interest from patients and physicians in a potential treatment option with subcutaneous administration, quarterly dosing, and potent and reversible serum TTR reduction,” said Rena Denoncourt, Vice President, TTR Franchise Lead. “If positive, the HELIOS-B study would support our efforts to advance the development of an industry leading franchise of RNAi therapeutics for the treatment of ATTR amyloidosis.”\n\nTopline full results from HELIOS-B are expected in early 2024. The HELIOS-B protocol includes an optional interim analysis which, if pursued, would be conducted following the data readout from Alnylam’s APOLLO-B Phase 3 clinical study of patisiran in patients with ATTR amyloidosis with cardiomyopathy. The APOLLO-B study completed enrollment in June 2021 and is expected to have topline data available in mid-2022. Based on those data and subsequent regulatory interactions, the potential path forward fo...