Conference Coverage

ASCEND-ATTR Phase 3b/4 Study to Assess Acoramidis and Cardiac Imaging Outcomes in ATTR-CM

Edited by:
Jennifer J. Spector, DPM, FACFAS

Key Highlights

  • ASCEND-ATTR plans to enroll approximately 150 adults with wild-type or variant ATTR-CM.
  • Its planned primary endpoint is responder status at month 36, based on improvement from baseline in left ventricular ejection fraction or stroke volume index on cardiac magnetic resonance imaging.
  • The study will also assess cardiac structure, amyloid burden, biomarkers, and acoramidis safety and tolerability. It is currently enrolling; no outcomes have been reported.

The ASCEND-ATTR study, as reported in a poster at the Heart Failure Society of America 2026 Annual Scientific Meeting, plans to use serial cardiac imaging to examine long-term changes in heart structure, function, and amyloid burden during acoramidis treatment in patients with ATTR-CM. ASCEND-ATTR is a prospective, longitudinal, open-label, single-arm phase 3b/4 study. Researchers plan to follow approximately 150 acoramidis-treated participants for 36 months using cardiac magnetic resonance imaging (CMR) and echocardiography. Eligible adults must be aged 18 to 80 years and have confirmed wild-type or variant ATTR-CM, a history or clinical evidence of heart failure, left ventricular ejection fraction greater than 35%, and New York Heart Association class I or II symptoms. The protocol also specifies left ventricular wall thickness of at least 12 mm and an NT-proBNP level of at least 300 but less than 6000 pg/mL at screening.

Study Design and Status

ASCEND-ATTR is currently enrolling. The primary efficacy endpoint is CMR-based responder status at month 36, defined by improvement from baseline in left ventricular ejection fraction or left ventricular stroke volume index. Secondary endpoints include individual CMR measures of systolic function, global longitudinal strain, left ventricular mass index, and extracellular volume, a measure used in the study to assess amyloid burden.

Exploratory assessments will include echocardiographic measures, serum transthyretin, NT-proBNP, high-sensitivity troponin I, and imaging measures at months 12 and 24. CMR and echocardiographic data will be analyzed centrally after each participant completes the study; readers will be masked to the chronological order of the imaging assessments. Researchers will also evaluate acoramidis safety and tolerability.

Clinical Implications

According to the investigators, ASCEND-ATTR is intended to determine whether long-term transthyretin stabilization is associated with sustained improvement in myocardial structure, function, and amyloid burden. They also aim to identify imaging measures that may characterize response during treatment.

Expert Commentary

“By integrating serial CMR and echocardiography, the study aims to provide mechanistic insights into cardiac remodeling during treatment and help define imaging biomarkers of therapeutic response,” the researchers shared.

Reference

Masri A, Carmona Rubio A, Garcia-Pavia P, et al. Evaluating the long-term effects of acoramidis on cardiac function, structure, and amyloid burden in transthyretin amyloid cardiomyopathy: ASCEND-ATTR. HFSA ePoster Library. October 10, 2026.