BridgeBio Shares Data on Serum TTR Increase When Switching Participants from Placebo and Tafamidis to Acoramidis in ATTRibute-CM and its Open-Label Extension

Core Insights - The study presented by BridgeBio Pharma indicates that acoramidis, a TTR stabilizer, shows significant improvements in serum TTR levels and clinical outcomes for patients with ATTR-CM [1][2][3] Group 1: Clinical Study Results - In the ATTRibute-CM study, participants switching to acoramidis from tafamidis and placebo experienced a mean increase of 3.0 mg/dL in serum TTR at Month 1 and 3.4 mg/dL at Month 6 of the open-label extension [1][2] - Increased serum TTR levels at Day 28 were correlated with reduced risks of all-cause mortality, cardiovascular mortality, and cardiovascular-related hospitalization [1][3] - Acoramidis demonstrated a 42% reduction in composite cardiovascular hospitalization and all-cause mortality events relative to placebo at Month 30 [1] - A 50% reduction in cumulative frequency of cardiovascular hospitalization events was observed at Month 30 compared to placebo [1] Group 2: Regulatory and Commercial Developments - BridgeBio submitted a New Drug Application to the U.S. FDA, accepted with a PDUFA action date of November 29, 2024, and a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025 [4] - The company granted Bayer exclusive rights to commercialize acoramidis for ATTR-CM in Europe, leveraging Bayer's expertise in cardiovascular disease [6] Group 3: Future Directions and Collaborations - The ongoing partnership with the Cardiovascular Data Science Lab aims to utilize AI tools for screening ATTR-CM across diverse health systems, focusing on undiagnosed cases [3] - The data presented may support the rationale for monitoring serum TTR levels in patients and inform treatment decisions in the context of multiple disease-modifying therapies for ATTR-CM [3]