Press release
Alnylam Announces Publication of Results from APOLLO-B Phase 3 Study of Patisiran in Patients with the Cardiomyopathy of ATTR Amyloidosis in the New England Journal of Medicine
– Treatment with an RNAi Therapeutic Preserved Functional Capacity and Health Status and Quality of Life Compared with Placebo at 12 Months – – Patisiran
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Treatment with an RNAi Therapeutic Preserved Functional Capacity and Health Status and Quality of Life Compared with Placebo at 12 Months –\n\n\n– Patisiran Demonstrated an Encouraging Safety and Tolerability Profile in Patients with the Cardiomyopathy of ATTR Amyloidosis –\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that results from the APOLLO-B Phase 3 study of investigational patisiran in patients with the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis were published online in the New England Journal of Medicine (NEJM). The data reported in the APOLLO-B Phase 3 study publication demonstrate that patisiran, an RNAi therapeutic targeting transthyretin (TTR), preserved functional capacity and health status and quality of life compared with placebo at 12 months. In addition, treatment with patisiran demonstrated favorable effects on biomarkers of cardiac stress and injury and on measures of cardiac structure and function. The full manuscript, titled “Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis,” will appear in the October 26, 2023 issue of NEJM.\n\n\n“ATTR amyloidosis is a rapidly progressive disease, with cardiac manifestations that can have a devastating impact on patients’ lives, and include arrhythmias, conduction disease, and heart failure. Current treatment options are limited, with many patients still experiencing declines in their functional capacity and quality of life, underscoring the need for additional therapeutic approaches,” said Mathew Maurer, M.D., Arnold and Arlene Goldstein Professor of Cardiology at Columbia University Irving Medical Center, principal investigator of the APOLLO-B Phase 3 study, and lead author of the manuscript. “The results of the APOLLO-B Phase 3 study published in the New England Journal of Medicine support the hypothesis that a reduction in circulating TTR protein can provide benefit in patients with the cardiomyopathy of ATTR amyloidosis, warranting further evaluation of investigational RNAi therapeutics to treat this devastating disease.”\n\n\nThe APOLLO-B Phase 3 study achieved its primary endpoint at 12 months, with patisiran demonstrating a significant difference in the change from baseline in functional capacity compared with placebo, as mea...