Alnylam Presents Additional Results from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy at Heart Failure Society of America Annual Meeting

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced results from exploratory endpoints and additional analyses of prespecified patient subgroups for the primary and secondary endpoints in the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. The results were presented during a moderated poster session at the Heart Failure Society of America’s Annual Scientific Meeting on September 30, 2022. The Company previously announced that the 12-month study achieved its primary endpoint and met its first secondary endpoint at the 18^th International Symposium on Amyloidosis in September 2022.

The 12-month findings across a comprehensive set of exploratory endpoints suggest that treatment with patisiran was associated with favorable impacts on key measures of cardiac stress and injury, NT-proBNP and Troponin I, respectively. In addition, favorable impact of patisiran relative to placebo was seen in several echocardiographic parameters and technetium scintigraphy uptake. Results from an analysis of prespecified patient subgroups were also presented, demonstrating generally consistent benefit across subgroups for the primary and secondary endpoints of 6-MWT and KCCQ-OS, respectively. As previously shared, patisiran also demonstrated an encouraging safety and tolerability profile in patients with ATTR amyloidosis with cardiomyopathy.

“The cardiac manifestations associated with ATTR amyloidosis can have a devastating impact on patients’ lives and current treatment options are limited,” said Parag Kale, M.D., Staff Cardiologist, Heart Transplant Department at Baylor University Medical Center. “It’s impressive to see that the effects of patisiran on functional capacity and quality of life in the APOLLO-B study were maintained across key patient subgroups, and corroborated by favorable changes in exploratory measures of NT-proBNP and Troponin I, cardiac biomarkers that are often used in clinical practice to assess patients with cardiomyopathy due to ATTR amyloidosis.”

APOLLO-B Study Results

APOLLO-B is a Phase 3, randomized, double-blind, placebo-controlled multicenter global study designed and powered to evaluate the effects of patisiran on functional capacity, health status and quality of life in patients with ATTR amyloidosis with cardiomyopathy. The study enrolled 360 adult patients with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy at 69 sites in 21 countries. Patients were randomized 1:1 to receive 0.3 mg/kg of patisiran or placebo intravenously administered every three weeks over a 12-month treatment period. After 12 months, all patients will receive patisiran in an open-label extension.