BridgeBio reports 54-month ATTRibute-CM data showing acoramidis reduces mortality, stabilizes cardiac biomarkers, and maintains quality of life in ATTR-CM patients.
Written By: Vennela Reddy, BPharm
Reviewed By: Pharmacally Editorial Team
BridgeBio Pharma has reported long-term efficacy and safety data from the ATTRibute-CM open-label extension (OLE) trial (NCT04988386), demonstrating sustained clinical benefit of acoramidis through 54 months in patients with Transthyretin Amyloid Cardiomyopathy. The findings were presented at the American College of Cardiology Annual Scientific Sessions and simultaneously published in JAMA Cardiology.
Sustained mortality reduction and cardiac benefit
The OLE data showed that continuous treatment with acoramidis resulted in a 44.7% reduction in all-cause mortality and a 49.3% reduction in cardiovascular mortality at Month 54 compared with patients who switched from placebo. Both outcomes reached strong statistical significance (p<0.0001).
Acoramidis also slowed disease progression, as reflected by mitigation of NT-proBNP increases, a biomarker of cardiac stress. This effect was described as exceeding what has been observed in the era of disease-modifying therapies for ATTR-CM.
Dr. Prem Soman, M.D., Ph.D. of University of Pittsburgh School of Medicine noted that despite recent advances, ATTR-CM remains associated with progressive heart failure and high mortality, and emphasized that early and continuous acoramidis treatment can meaningfully alter disease progression, with sustained improvements in survival, hospitalization risk, and cardiac biomarkers alongside a favorable long-term safety profile.
Quality of life and functional outcomes
Patients receiving early and continuous treatment maintained stable scores on the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS), indicating preserved heart failure–related quality of life over time. These findings suggest that both survival and functional status benefits are sustained with long-term therapy.
Safety profile remains favorable
Long-term treatment with acoramidis was well tolerated, with no new safety concerns identified over the 54-month period. Reported adverse events, including diarrhea and upper abdominal pain, were generally mild and did not lead to higher discontinuation rates compared with placebo.
Supporting analyses highlight early treatment impact
Additional analyses presented at the meeting reinforced the importance of early intervention. Data showed that early increases in serum transthyretin levels were associated with slower decline in patient-reported outcomes, while long-term stabilization of transthyretin correlated with sustained clinical benefit.
A real-world survey analysis also highlighted ongoing unmet needs, with more than one-third of patients not receiving treatment and physicians reporting dissatisfaction with existing options in over half of treated cases. Oral therapies were preferred by patients, underscoring the relevance of acoramidis as an oral agent.
Regulatory status and indication
Acoramidis is approved in the United States as Attruby and in multiple global markets under the name BEYONTTRA. It is indicated for adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization.
BridgeBio indicated that additional data on acoramidis will be presented at future medical meetings, as the company continues to evaluate its long-term impact in ATTR-CM.
Reference
Acoramidis Significantly Reduces the Risk of All-Cause and Cardiovascular Mortality in Patients with ATTR-CM through Month 54, 30 March 2026, BridgeBio Pharma Inc. – Acoramidis Significantly Reduces the Risk of All-Cause and Cardiovascular Mortality in Patients with ATTR-CM through Month 54
Soman P, Cuddy SAM, Gillmore JD, et al. Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy: Open-Label Extension of the ATTRibute-CM Randomized Clinical Trial. JAMA Cardiol. Published online March 30, 2026. https://jamanetwork.com/journals/jamacardiology/fullarticle/2847055
Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants (AG10), ClinicalTrials.gov ID NCT04988386, https://clinicaltrials.gov/study/NCT04988386
About the Writer
Vennela Reddy, BPharm is a pharmacy graduate with a keen interest in clinical research, pharmacovigilance, and medical writing, with a growing focus on publishable and scientific content development. In her words, she is passionate about translating complex medical data into clear, evidence-based communication.