Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

Summary

Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Timeline

Start

2021-10-27

Primary completion

2028-04-15

Completion

2028-05-15

Outcome

Met primary endpoint

paper Continuous acoramidis reduced time to first CVH (HR, 0.53; 95% CI, 0.42-0.69; P < .001) through month 54 PMID 41911489 ↗

Publications

• Testani JM, Judge DP, Borlaug BA, Cherney D, Cox ZL, Gillmore JD, Lewis JB, Adler SH, Cao X, Castano A, Fox JC, Katz L, Mathur V, Xiong K, Butler J, Masri A. Effects of Acoramidis on Kidney Function in Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. 2026 Jul 2:e014656. doi: 10.1161/CIRCHEARTFAILURE.126.014656. Online ahead of print.
• Soman P, Cuddy SAM, Gillmore JD, Sarswat N, Judge DP, Cappelli F, Stern LK, Wright R, Akinboboye O, Obici L, Bhatt K, Xiong K, Castano A, Chen C, Falvey H, Pecoraro ML, Tamby JF, Sinha U, Fox JC, Shah K, Grodin JL, Ambardekar AV, Alexander KM, Khouri MG, Cheng RK. Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy: Open-Label Extension of the ATTRibute-CM Randomized Clinical Trial. JAMA Cardiol. 2026 May 1;11(5):446-454. doi: 10.1001/jamacardio.2026.0819.
• Alexander KM, Davis MK, Akinboboye O, Berk J, Bhatt K, Cappelli F, Cuddy SAM, Fontana M, Garcia-Pavia P, Gillmore JD, Griffin JM, Grodin JL, Judge DP, Khouri MG, Lam K, Masri A, Maurer MS, Obici L, Ruberg FL, Sarswat N, Shah K, Soman P, Stern L, Wright R, Xiong K, Cao X, Lystig T, Tamby JF, Castano A, Katz L, Sinha U, Fox JC, Solomon SD, Grogan M. Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension. JAMA Cardiol. 2026 Jan 1;11(1):57-67. doi: 10.1001/jamacardio.2025.4477.
• Judge DP, Gillmore JD, Alexander KM, Ambardekar AV, Cappelli F, Fontana M, Garcia-Pavia P, Grodin JL, Grogan M, Hanna M, Masri A, Nativi-Nicolau J, Obici L, Hvitfeldt Poulsen S, Sarswat N, Shah K, Soman P, Lystig T, Cao X, Wang K, Pecoraro ML, Tamby JF, Katz L, Sinha U, Fox JC, Maurer MS. Long-Term Efficacy and Safety of Acoramidis in ATTR-CM: Initial Report From the Open-Label Extension of the ATTRibute-CM Trial. Circulation. 2025 Mar 4;151(9):601-611. doi: 10.1161/CIRCULATIONAHA.124.072771. Epub 2024 Nov 18.

Drugs

Indications

No indication recorded.