A phase 3 trial evaluated the efficacy and safety of acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The study randomly assigned 632 patients to receive acoramidis (800 mg twice daily) or matching placebo for 30 months. The primary outcome was a four-step hierarchical analysis including death from any cause, cardiovascular-related hospitalization, change in NT-proBNP levels, and 6-minute walk distance. Acoramidis showed significant benefits over placebo, with a win ratio of 1.8 (95% CI, 1.4 to 2.2), indicating better outcomes in most pairwise comparisons. The primary analysis favored acoramidis over placebo (P<0.001). Key secondary outcomes included improved 6-minute walk distance, better quality of life as measured by the Kansas City Cardiomyopathy Questionnaire–Overall Summary (KCCQ-OS), and higher serum TTR levels in the acoramidis group. Adverse events were similar between groups, with 98.1% and 97.6% incidence in the acoramidis and placebo groups, respectively. Serious adverse events were reported in 54.6% and 64.9% of patients. Acoramidis demonstrated a significant improvement in mortality, morbidity, and function compared to placebo. The study was funded by BridgeBio Pharma and conducted in accordance with Good Clinical Practice guidelines. The results support the use of acoramidis as an effective and safe treatment for ATTR-CM.A phase 3 trial evaluated the efficacy and safety of acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The study randomly assigned 632 patients to receive acoramidis (800 mg twice daily) or matching placebo for 30 months. The primary outcome was a four-step hierarchical analysis including death from any cause, cardiovascular-related hospitalization, change in NT-proBNP levels, and 6-minute walk distance. Acoramidis showed significant benefits over placebo, with a win ratio of 1.8 (95% CI, 1.4 to 2.2), indicating better outcomes in most pairwise comparisons. The primary analysis favored acoramidis over placebo (P<0.001). Key secondary outcomes included improved 6-minute walk distance, better quality of life as measured by the Kansas City Cardiomyopathy Questionnaire–Overall Summary (KCCQ-OS), and higher serum TTR levels in the acoramidis group. Adverse events were similar between groups, with 98.1% and 97.6% incidence in the acoramidis and placebo groups, respectively. Serious adverse events were reported in 54.6% and 64.9% of patients. Acoramidis demonstrated a significant improvement in mortality, morbidity, and function compared to placebo. The study was funded by BridgeBio Pharma and conducted in accordance with Good Clinical Practice guidelines. The results support the use of acoramidis as an effective and safe treatment for ATTR-CM.