Alnylam announces publication of results from Phase 3 study of patisiran

Alnylam announced results from the APOLLO-B Phase 3 study of investigational patisiran in patients with the cardiomyopathy of transthyretin-mediated – ATTR – amyloidosis published online in the New England Journal of Medicine. 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 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. The study also met its first secondary endpoint, with patisiran demonstrating a significant difference in the change from baseline in health status and quality of life compared with placebo. For the composite endpoint of all-cause mortality, frequency of cardiovascular events, and change from baseline in 6-MWT over 12 months compared with placebo, the win ratio favored patisiran but did not reach statistical significance. Patisiran demonstrated an encouraging safety and tolerability profile in the APOLLO-B Phase 3 study, including no cardiac safety concerns relative to placebo, through 12 months. The majority of adverse events were mild or moderate in severity.