PALO ALTO - BridgeBio Pharma, Inc. (Nasdaq: BBIO) ('BridgeBio' or the 'Company'), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today presented results from the exploratory CMR imaging substudy of ATTRibute-CM, its Phase 3 trial of acoramidis in ATTR-CM.

These data were presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo in a moderated poster session by Yousef Razvi, M.D. of University College London. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of TTR. Based on the positive results from ATTRibute-CM, BridgeBio submitted an NDA to the U.S. FDA, which has been accepted with a PDUFA action date of November 29, 2024, and an MAA to the European Medicines Agency, with a decision expected in 2025.

'CMR is the reference non-invasive imaging method to evaluate cardiac structure, function, and amyloid burden in patients with ATTR-CM. The results shown in the imaging substudy are indicative of potential improvement of cardiac structure and function in patients with ATTR-CM, consistent with the clinical outcomes with acoramidis treatment observed in the ATTRibute-CM Phase 3 clinical trial. The impact on cardiac amyloid load highlights the potential that acoramidis could lead to cardiac amyloid regression in a proportion of patients with ATTR-CM, which we have not observed in such a controlled clinical trial to date,' said Marianna Fontana, M.D., Ph.D., Professor of Cardiology and Honorary Consultant Cardiologist at the National Amyloidosis Centre, Division of Medicine, University College London, and Principal Investigator of the Substudy.

CMR imaging was performed at baseline before the first dose in 35 participants or within three months after the first dose in 17 participants (range, 14-105 days); subsequent CMR imaging was performed at months 12, 24, and 30 in those individuals who were available to undergo imaging, which was influenced by survival. Images were read centrally at the National Amyloidosis Centre in a fashion blinded to other clinical data. Findings included: Key CMR imaging parameters measuring cardiac structure, including mean indexed left ventricular mass, were found to be stable or have a trend towards improvement on acoramidis vs. deteriorated on placebo over 30 months

Treatment with acoramidis preserved or was associated with a trend towards improvement in measures of cardiac function including left ventricular ejection fraction and stroke volume compared to initial measures, and relative to placebo

In July 2023, BridgeBio announced positive results from ATTRibute-CM, reporting a highly statistically significant result, demonstrated by a Win Ratio of 1.8 (p

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