Acoramidis significantly reduces the risk of all-cause and cardiovascular mortality in patients with ATTR-CM through month 54 – BridgeBio

BridgeBio Pharma, Inc. presented long-term efficacy and safety data from the ATTRibute-CM open-label extension (OLE) trial, demonstrating sustained clinical benefit of acoramidis through Month 54 in patients with ATTR-CM. These data were presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo by Prem Soman, M.D., Ph.D. of University of Pittsburgh School of Medicine. These data are now simultaneously published in JAMA Cardiology. Acoramidis is the only selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer.

“Despite dramatic therapeutic advances in the field, many patients with ATTR-CM still continue to suffer progressive heart failure, high mortality risk, and many have limited long-term treatment options,” said Dr. Soman. “The ATTRibute-CM long-term data show that early and continuous treatment with acoramidis can meaningfully change the trajectory of this disease, with sustained reductions in all-cause and cardiovascular mortality, cardiovascular hospitalization, continued mitigation of NT-proBNP progression, and a favorable long-term safety profile. These findings reinforce the importance of early diagnosis followed by prompt, durable treatment to deliver sustained clinical benefit for patients.”

The findings from the OLE trial included:

i) At Month 54, continuous acoramidis treatment led to a statistically significant risk reduction of 44.7% in all-cause mortality (ACM) (p<0.0001) and 49.3% in cardiovascular mortality (CVM) (p<0.0001) versus placebo-to-acoramidis, which is the earliest timepoint in an open-label extension with this magnitude of risk reduction in these events

ii) Through Month 54, acoramidis mitigated the rise in NT-proBNP versus placebo-to-acoramidis to an extent not seen in the era of disease modifying treatments

iii) Early and continuous acoramidis treatment stabilized and maintained all measures of heart failure-related QOL scores (KCCQ-OS), which demonstrates that the improvement in duration and quality of life in patients with ATTR-CM by acoramidis is sustained

iv) Acoramidis continued to be well tolerated with no long-term safety concerns.

Citation: Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid CardiomyopathyOpen-Label Extension of the ATTRibute-CM Randomized Clinical Trial. Authors: Prem Soman, MD, PhD; Sarah A. M. Cuddy, MD; Julian D. Gillmore, MD, PhD et al. JAMA CardiolPublished Online: March 30, 2026. doi: 10.1001/jamacardio.2026.0819