Core Insights - Acoramidis has shown significant early and sustained clinical efficacy in reducing cumulative cardiovascular outcomes in patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM) [1][2][3] Group 1: Clinical Efficacy - Acoramidis reduced cumulative cardiovascular mortality (CVM) or recurrent cardiovascular-related hospitalizations (CVH) by 49% through Month 30 compared to placebo, with a p-value of less than 0.0001 [1][3] - At Month 30, 53 events were avoided per 100 treated participants, with a 95% confidence interval of 29 to 79 [1][3] - Continuous treatment with acoramidis at Month 42 resulted in a 45% reduction in CVM compared to a switch from placebo to acoramidis, with a p-value of 0.0011 [3] Group 2: Patient Population Insights - Acoramidis demonstrated a consistent mitigation of NT-proBNP levels in patients with variant ATTR-CM, starting from Month 3 and continuing through Month 30 [3] - The findings highlight the importance of early and continuous treatment with acoramidis, regardless of TTR genotype, in both wild-type and variant ATTR-CM patients [3] Group 3: Regulatory Approvals - Acoramidis is approved as Attruby® by the U.S. FDA and as BEYONTTRA® by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency, and the UK Medicines and Healthcare Products Regulatory Agency [4][5] Group 4: Future Developments - Additional data on the benefits of Attruby for ATTR-CM patients is planned for future medical meetings [4]

Core Insights - The UK has granted marketing authorization for acoramidis, branded as BEYONTTRA, for treating wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) [1][2] - Acoramidis is the first and only approved treatment for ATTR-CM in the U.S., EU, UK, and Japan, with a label specifying near-complete stabilization (≥90%) of transthyretin (TTR) [1][3] - The approval is based on positive results from the Phase 3 ATTRibute-CM study, which demonstrated significant cardiovascular benefits [2][3] Summary by Category Product Approval - The Medicines and Healthcare products Regulatory Agency in the UK has approved acoramidis for ATTR-CM treatment [1][2] - Acoramidis has previously received approvals in the U.S. as Attruby™ and in the EU and Japan as BEYONTTRA [3] Clinical Study Results - The ATTRibute-CM study involved 632 participants and showed a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalization events at Month 30 compared to placebo [1][2] - A 50% reduction in the cumulative frequency of cardiovascular-related hospitalization events was also observed at Month 30 [1] Market and Financial Aspects - BridgeBio will receive royalties in a tiered structure starting in the low-thirties percent on sales of BEYONTTRA in the UK [1][4][5] - Bayer is responsible for all commercial activities related to acoramidis in the UK [1][3]