Primary endpoint met in phase 3 trial with vutrisiran in patients with ATTR-CM
The primary endpoint of all-cause mortality and recurrent CV events was significantly reduced with the RNAi therapeutic vutrisiran in patients with ATTR amyloidosis with cardiomyopathy in the HELIOS-B phase 3 study.
The phase 3 HELIOS-B study of vutrisiran, an investigational RNAi therapeutic against TTR for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), met its primary composite endpoint of all-cause mortality and recurrent CV events. A reduction in the primary endpoint with vutrisiran was demonstrated in the overall population (HR 0.718, P=0.0118, n=654) and in the monotherapy population (patients not receiving tafamidis at baseline; HR 0.672, P=0.0162, n=395).
Significant improvements were observed across all secondary endpoints, including 6-MWT, KCCQ and NYHA class at month 30 (P<0.025 for all). Moreover, all-cause mortality was reduced in the group receiving vutrisiran in the overall population (HR 0.645, P<0.025) and in the monotherapy population (HR 0.655, P<0.05) up to month 42.
The safety and tolerability profile of vutrisiran in the HELIOS-B study was encouraging, consistent with previous findings. Rate of adverse events, serious adverse events, and adverse events leading to study drug discontinuation were similar between the vutrisiran and placebo groups.
The HELIOS-B study is a phase 3, randomized, double-bling, placebo-controlled multicenter global trial to evaluate the efficacy and safety of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy in the overall and monotherapy populations. In this study, 655 adult patient with ATTR amyloidosis with cardiomyopathy were randomized in a 1:1 ratio to receive vutrisiran 25 mg or placebo subcutaneously once every 3 months during a period of 36 months.
Presentation of the results from the HELIOS-B study is planned at the ESC congress this year and plans to proceed with global regulatory submissions will start later this year.