First Participant Dosed in ASCEND-ATTR, a Phase 3b/4 Study Evaluating the Long-Term Effects of Acoramidis on Disease Regression in ATTR-CM – BridgeBio
BridgeBio Pharma, Inc. announced that the first participant has been dosed in ASCEND-ATTR, a Phase 3b/4 study designed to further characterize the long-term effects of acoramidis on the improvement of cardiac structure, function, and amyloid burden in individuals with transthyretin amyloid cardiomyopathy (ATTR-CM).
Acoramidis is the only selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer.
“Serial cardiac imaging from the ATTRibute-CM CMR substudy gave us the first real signal that TTR stabilization can do more than slow disease progression, it may allow the heart to recover function and remodel favorably over time,” said Ahmad Masri, M.D., M.S. of Oregon Health and Science University. “ASCEND-ATTR will allow us to study these structural and functional changes prospectively and in far greater depth, across a notably larger patient cohort and with two complementary imaging modalities, to better understand the extent to which favorable remodeling can be achieved with long-term acoramidis treatment.”
ASCEND-ATTR is a single-arm, prospective, longitudinal, open-label study that will enroll appro
ximately 150 participants with ATTR-CM. Cardiovascular magnetic resonance (CMR) and cardiac echocardiography will be performed annually over 36 months. The primary efficacy endpoint is responder status at Month 36 by CMR, based on improvement from baseline in LV systolic function. Secondary endpoints include CMR measures of cardiac function, structure, and amyloid burden at Month 36, along with echocardiographic measures, circulating biomarkers, and imaging assessments at Months 12 and 24. This study reflects BridgeBio’s relentless pursuit in advancing care and addressing the unmet needs of the ATTR-CM community.
The previously presented CMR substudy of ATTRibute-CM found treatment with acoramidis suggested disease improvement across multiple measurements of cardiac structure and function through month 30, including mean improvement from baseline in Left Ventricular Mass Index (LVMi), Left Ventricular Stroke Volume Index (LVSVi), and Left Ventricular Ejection Fraction (LVEF) with evidence of amyloid regression in a subset of patients. TTR stabilization with acoramidis may allow the rate of innate amyloid clearance mechanisms to exceed the rate of amyloid formation, thereby enabling cardiac remodeling and functional recovery. These findings suggest acoramidis may be capable of altering the trajectory of this otherwise progressive disease and actively restoring heart health. Additional data from the CMR substudy of ATTRibute-CM and its open-label extension compared to a natural history cohort will be shared at the European Society of Cardiology (ESC) Congress 2026.
