BridgeBio Pharma Publishes Positive Phase 3 Results for Acoramidis in NEJM

BridgeBio Pharma, Inc., a biopharmaceutical company specializing in genetic diseases and cancers, announced promising results from its Phase 3 ATTRibute-CM study of acoramidis for patients with ATTR-CM, published in the New England Journal of Medicine (NEJM). The study aimed to evaluate the efficacy and safety of acoramidis, an orally-administered small molecule stabilizer of transthyretin (TTR).

Professor Julian Gillmore, head of University College London’s Centre for Amyloidosis, noted the striking benefits acoramidis provided in treating ATTR-CM, expressing hope that it will soon be available globally. The trial results demonstrated a significant positive impact of acoramidis on all-cause mortality (ACM), cardiovascular-related hospitalization (CVH), N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and six-minute walk distance (6MWD).

Published findings support acoramidis as both a safe and effective treatment option, reinforcing the idea that greater stabilization of TTR can lead to improved clinical outcomes. Notably, the majority of comparisons in the study's primary hierarchical analysis, specifically 58% in the associated Win Ratio, were determined by ACM and CVH. Statistical significance was achieved for these two cardiovascular outcomes alone. Additionally, significant treatment benefits were observed for changes in 6MWD, the Kansas City Cardiomyopathy Questionnaire (KCCQ), and serum TTR levels.

The survival rate data from the study is also noteworthy. The placebo arm exhibited a 30-month survival rate of 74.3%, higher than the 70.5% survival rate seen in the combined tafamidis treatment arms of the ATTR-ACT study, the only other cardiovascular outcomes study reported for ATTR-CM. For context, the recent U.S. Social Security Administration data indicated a 30-month survival rate of 85% in an age-matched cohort from the general population. Additionally, the annualized CVH rate of 0.29 in the treatment arm can be compared to the overall hospitalization rate of 0.26 in the U.S. Medicare population.

Patients treated with acoramidis showed consistent elevation in serum TTR due to near-complete stabilization of the protein. Acoramidis was well-tolerated, with no significant safety concerns identified.

Jonathan Fox, President and Chief Medical Officer of BridgeBio Cardiorenal, emphasized that the data suggests a stronger stabilization of TTR correlates with greater clinical benefits. BridgeBio has submitted a New Drug Application (NDA) to the FDA and plans further regulatory submissions to bring acoramidis to more patients in need.

In addition to the publication in NEJM, BridgeBio presented detailed results of the ATTRibute-CM study at several significant medical conferences, including the European Society of Cardiology Congress 2023 and the American Heart Association Scientific Sessions 2023. The company is also preparing to submit additional marketing authorization applications to various regulatory bodies in 2024.

BridgeBio, founded in 2015, focuses on discovering and developing transformative medicines for genetic diseases and cancers. With a robust pipeline ranging from early-stage science to advanced clinical trials, BridgeBio is dedicated to swiftly applying advances in genetic medicine to help patients.