BridgeBio Pharma, Inc. (Nasdaq: BBIO), a leading biopharmaceutical company specializing in genetic diseases and cancers, unveiled findings from the exploratory CMR imaging substudy of its Phase 3 trial, ATTRibute-CM, evaluating acoramidis in patients with ATTR-CM. Presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo by Dr. Yousef Razvi of University College London, these results shed light on the efficacy and safety of acoramidis, a next-generation, orally-administered TTR stabilizer. The positive outcomes from ATTRibute-CM prompted BridgeBio to submit an NDA to the U.S. FDA and an MAA to the European Medicines Agency, anticipating potential regulatory approvals.
Dr. Marianna Fontana, Principal Investigator of the Substudy and Professor of Cardiology at the National Amyloidosis Centre, emphasized the significance of CMR imaging in assessing cardiac structure, function, and amyloid burden in ATTR-CM patients. The substudy’s findings indicate a potential enhancement in cardiac structure and function with acoramidis treatment, aligning with clinical observations in the Phase 3 trial. Notably, acoramidis demonstrated the potential to reduce cardiac amyloid load, a promising prospect not previously observed in controlled clinical settings.
The CMR imaging substudy involved baseline assessments before the initial dose and subsequent evaluations at months 12, 24, and 30, revealing:
- Stable or improved cardiac structure parameters, such as mean indexed left ventricular mass, with acoramidis compared to deterioration on placebo over 30 months.
- Preservation or improvement trends in cardiac function measures, including left ventricular ejection fraction and stroke volume, with acoramidis treatment relative to baseline and placebo.
BridgeBio’s previous announcement in July 2023 highlighted the highly significant results from ATTRibute-CM, demonstrating the efficacy of acoramidis across various endpoints. The drug exhibited favorable tolerability profiles, with no notable safety concerns. Additional analyses from ATTRibute-CM have been presented at major scientific conferences, reinforcing the robustness of the findings. Furthermore, positive results from a Phase 3 study of acoramidis in Japanese patients with ATTR-CM underscore its potential across diverse populations.
BridgeBio’s ongoing efforts underscore its commitment to advancing treatments for ATTR-CM and addressing unmet medical needs in this complex disease landscape.
