BridgeBio(US:BBIO) Newsfilter·2024-01-10 22:15Core Insights - BridgeBio Pharma announced positive results from its Phase 3 ATTRibute-CM study of acoramidis for patients with ATTR-CM, demonstrating significant treatment effects on all-cause mortality and cardiovascular-related hospitalization with a Win Ratio of 1.8 (p<0.0001) [1][2] - Acoramidis showed an observed 30-month survival rate of 80.7% in the treatment arm, compared to 74.3% in the placebo arm, and 70.5% in the combined tafamidis treatment arms of ATTR-ACT [2] - The company has submitted a New Drug Application (NDA) to the U.S. FDA and plans to submit additional marketing authorization applications in 2024 [1][3] Efficacy and Safety - Acoramidis was well-tolerated with no safety signals of potential clinical concern identified [1][2] - The study demonstrated statistically significant treatment benefits in various parameters, including 6-minute walk distance and serum TTR levels [2] - The annualized cardiovascular-related hospitalization rate in the treatment arm was 0.29, comparable to the overall hospitalization rate of 0.26 in the U.S. Medicare population [1][2] Regulatory and Market Potential - The NDA submission to the FDA marks a significant step towards making acoramidis available to patients, with additional regulatory submissions planned for 2024 [3] - The results from the ATTRibute-CM study support the hypothesis that greater stabilization of transthyretin (TTR) is associated with improved clinical outcomes [2][3] - BridgeBio's commitment to advancing genetic medicine is reflected in its pipeline, which includes various development programs from early science to advanced clinical trials [4]