Disease Stabilization Translates into Slower Functional Decline for Patients with Transthyretin Amyloid Cardiomyopathy

Early and continuous treatment with transthyretin (TTR) stabilizer acoramidis led to significant improvements in health status and slower functional decline in individuals with transthyretin amyloid cardiomyopathy (ATTR-CM), according to data presented at the 2026 Annual Meeting of the American Association of Heart Failure Nurses (AAHFN 2026) in San Diego, California. 

Acoramidis, a highly selective oral TTR stabilizer that achieves near-complete (≥90%) disease stabilization, is approved in the United States, Europe, Japan, Switzerland, and the United Kingdom for the treatment of variant or wild-type ATTR-CM in adults. In the phase 3 ATTRibute-CM study, which enrolled more than 600 patients who were randomized to receive acoramidis hydrochloride at a dose of 800 mg or matching placebo twice daily for 30 months, treatment with acoramidis achieved ATTR-CM disease stabilization, reflected in sustained increases in serum TTR concentrations and stabilized N-terminal pro–B-type natriuretic peptide levels. An interim analysis showed that acoramidis significantly increased serum TTR concentrations early, by day 28 after treatment initiation, and that the increase in concentrations was sustained through month 30 [Maurer MS, et al. J Am Coll Cardiol 2025; 85:1911-23]. The acoramidis-mediated early increase in serum TTR concentrations was independently associated with improved survival. The researchers also assessed Kansas City Cardiomyopathy Questionnaire (KCCQ) scores, which measure a patient's health status, symptom burden, physical limitations, and quality of life caused by heart failure or cardiomyopathy, and found that acoramidis treatment also reduced the decline in heart failure-related health status (as assessed by KCCQ scores) compared with placebo at month 30. 

An efficacy analysis conducted in the ATTRibute-CM modified intention-to-treat population, presented at AAHFN 2026, showed that early increases in serum TTR concentrations at day 28 in patients treated with acoramidis were associated with a clinically meaningful slower decline in KCCQ scores at month 30 compared with placebo. Serum TTR concentrations below the normal range (< 20 mg/dL) were associated with lower KCCQ-OS scores at month 30 compared with higher serum TTR concentrations, regardless of treatment group. The modified intention-to-treat population consisted of all randomized participants in ATTRibute-CM who had received at least one dose of acoramidis or placebo, had at least one efficacy evaluation after baseline, and had baseline estimated glomerular filtration rates of ≥ 30 mL/min/1.73 m2

A separate analysis showed that switching from placebo to acoramidis rapidly increased and maintained serum TTR concentrations and stabilized heart failure-related health status through month 54 in the open-label extension phase of the trial. 

“The KCCQ measures a patient’s perspective of their own health status,” said presenting author Kellie Walsh, MSN, a nurse practitioner specializing in cardiac amyloidosis. “Worsening of KCCQ-OS score over time in patients with ATTR-CM is associated with hospitalization and death.” Patients’ quality of life represents a major focus of clinical interventions, Walsh noted, as does improving functional status or slowing functional decline in vulnerable patients with heart failure. 

“These findings demonstrate durable benefits of acoramidis with both early and delayed initiation [of treatment] in a contemporary ATTR-CM population, but with greater treatment effects when therapy is started early, underscoring the importance of early diagnosis and treatment,” the authors concluded.