BridgeBio reports positive results from Phase 3 ATTRibute-CM study of acoramidis

BridgeBio Pharma announced that positive results from its Phase 3 ATTRibute-CM study of acoramidis for patients with ATTR-CM were published in the New England Journal of Medicine. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, small molecule stabilizer of transthyretin. “The consistent benefits of acoramidis treatment demonstrated by the ATTRibute-CM results, especially in the context of contemporary ATTR-CM care, are striking and encourage its potential use,” said Professor Julian Gillmore, M.D., Ph.D., head of University College London’s Centre for Amyloidosis and research lead at the UK National Amyloidosis Centre. “Given the efficacy and safety of acoramidis demonstrated in this trial, I am hopeful that it will soon be available to the benefit of the growing global population of patients diagnosed with ATTR-CM.” The ATTRibute-CM study demonstrated a significant treatment effect of acoramidis in the primary analysis that compared, in a hierarchical manner, all-cause mortality, cardiovascular-related hospitalization, N-terminal prohormone of brain natriuretic peptide, and 6-minute walk distance. Findings presented in the NEJM support acoramidis as an effective and safe treatment option for patients with ATTR-CM and reinforce the hypothesis that greater stabilization of TTR may be associated with improved clinical outcomes.