Outcomes of HELIOS-B Monotherapy Analysis

This document is a post hoc analysis report focusing on the outcomes of the HELIOS-B study, specifically evaluating the efficacy and safety of vutrisiran in a monotherapy population of patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The analysis compares results with and without censoring observations after the initiation of tafamidis, as 21.5% of patients in the monotherapy group began tafamidis treatment post-randomization. The report outlines the study's objectives, which include assessing the impact of vutrisiran on all-cause mortality (ACM) and recurrent cardiovascular (CV) events. It details the study populations and provides statistical results demonstrating that vutrisiran significantly reduced ACM and recurrent CV events compared to placebo, irrespective of censoring. Additionally, the safety profile of vutrisiran is discussed, indicating that adverse events were consistent across the monotherapy population. The findings contribute to understanding the treatment's effectiveness in managing ATTR-CM.