BridgeBio Pharma Shares Positive Results of Single-Arm Phase 3 Study of Acoramidis in Japanese Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Including No Mortality Reported in the Trial at 30 Months

Core Insights - BridgeBio Pharma, Inc. announced positive results from a Phase 3 trial of acoramidis for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in Japan, showing consistency with global trial results [1][2] - The treatment demonstrated no mortality over a 30-month period and was well-tolerated with no safety concerns identified [1][2] - The findings support local regulatory submission in Japan, with plans for data presentation at a medical meeting [2] Group 1 - The Phase 3 trial was an open-label, single-arm study conducted by Alexion, AstraZeneca Rare Disease, showing a Win Ratio of 1.8 and a highly statistically significant p-value of p<0.0001 in the global ATTRibute-CM trial [1] - At 30 months, an 81% survival rate and a 0.29 annualized cardiovascular hospitalization rate were observed in patients treated with acoramidis [1] - The trial also measured improvements in functional capacity and quality of life, assessed through the six-minute walk test and the Kansas City Cardiomyopathy Questionnaire Overall Summary Score [1] Group 2 - Acoramidis is an investigational oral small molecule designed to stabilize transthyretin (TTR) and is exclusively licensed to Alexion, AstraZeneca Rare Disease for development in Japan [2] - The company aims to leverage advances in genetic medicine to deliver transformative treatments for genetic diseases and cancers [3] - BridgeBio was founded in 2015 and focuses on a pipeline that spans from early science to advanced clinical trials [3]