New results have been reported from the phase 3 APOLLO-B trial of patisiran, an RNA interference therapy for patients with transthyretin amyloidosis (ATTR), including hereditary ATTR (hATTR) and cardiomyopathy, at the Heart Failure Society of America’s Annual Scientific Meeting in a moderated poster session.

Patisiran treatment led to improved measures of cardiac stress and injury, as well as N-terminal pro-brain natriuretic peptide and troponin I, after 12 months of treatment compared with placebo.

“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 [N-terminal pro-brain natriuretic peptide] and troponin I, cardiac biomarkers that are often used in clinical practice to assess patients with cardiomyopathy due to ATTR amyloidosis,” noted Parag Kale, MD, staff cardiologist, heart transplant department at Baylor University Medical Center, Texas.

APOLLO-B is a phase 3, randomized, double-blind, placebo-controlled study being carried out at 69 sites in 21 countries with the aim of assessing patisiran’s effects on functional capacity, health status, and quality of life in participants.

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Participants were randomized to receive 0.3 mg/kg of patisiran or placebo every 3 weeks, via intravenous administration, over 12 months. All patients will receive patisiran in the open-label extension after 12 months.

The positive results included benefits such as improvements in 6-minute walk test times and health status and quality of life (as assessed by the Kansas City Cardiomyopathy Questionnaire) compared with placebo. The results were consistent across patient subgroups, including age, type of ATTR (hereditary or wild type), and New York Heart Association functional classification. Patisiran also demonstrated an encouraging safety and tolerability profile across patient subgroups.


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. News release. Alnylam Pharmaceuticals, Inc.; September 30, 2022.