Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
Summary
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
Timeline
- Start
- 2019-03-19
- Primary completion
- 2023-05-11
- Completion
- 2023-05-11
Outcome
Sources disagree · no verdict is set; each source's reading is shown below
paper The primary analysis favored acoramidis over placebo (P<0.001); reads as met primary endpoint PMID 38197816 ↗
registry NUMBER Acoramidis HCl 800 mg: 63.7 Percent of Wins from Win Ratio reads as met primary endpoint NCT03860935 ↗
registry analysis (superiority test); Acoramidis HCl 800 mg vs Placebo; p <0.0001; Finkelstein-Schoenfeld Method reads as met primary endpoint NCT03860935 ↗
release “ATTRibute-CM did not meet its primary endpoint at Month 12.” reads as missed primary endpoint bridgebio.com ↗
release “As previously announced, a highly statistically significant result was observed on the primary endpoint of ATTRibute-CM with a Win Ratio of 1.8 (p<0.0001).” reads as met primary endpoint bridgebio.com ↗
release “global ATTRibute-CM Phase 3 trial, wherein the primary endpoint was met (Win Ratio of 1.8)” reads as met primary endpoint bridgebio.com ↗
release “In the Phase III ATTRibute-CM study, acoramidis met the primary endpoint.” reads as met primary endpoint bayer.com ↗
release “The study met its primary clinical endpoints at month 30 by significantly reducing cardiovascular-related hospitalization, improving survival, and preserving functional capacity and quality of life for patients.” reads as met primary endpoint bridgebio.com ↗
release “The study met its primary clinical endpoints at Month 30 by significantly reducing cardiovascular-related hospitalization, improving survival, and preserving functional capacity” reads as met primary endpoint bridgebio.com ↗
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.
- Judge DP, Ji AX, Graef IA, Grogan M, Soman P, Cappelli F, Fontana M, Masri A, Gillmore JD, Garcia-Pavia P, Cao X, Fox JC, Sinha U. Differential Transthyretin Stabilization in Patients With Wild Type and Variant Transthyretin Amyloidosis. JACC CardioOncol. 2026 Jun;8(3):299-313. doi: 10.1016/j.jaccao.2026.04.006.
- Fontana M, Cappelli F, Garcia-Pavia P, Judge DP, Obici L, Masri A, Agostinho JR, Tschope C, Vogtlander K, Ciaccia A, Zazula A, Zeitler S, Tamby JF, Fox JC, Gillmore JD, Damy T. Outpatient worsening heart failure in transthyretin amyloid cardiomyopathy: Findings from ATTRibute-CM. Eur J Heart Fail. 2026 May 29:xuag177. doi: 10.1093/ejhf/xuag177. Online ahead of print.
- Tahara N, Raizner A, Chen Y, Mercuri MF, Minamisawa M; Phase 3 Japan Study Investigators. Efficacy of Acoramidis in Adults With Transthyretin Amyloid Cardiomyopathy: Propensity Score Comparison Between a Single-Arm Open-Label Phase 3 Trial in Japan and a Global Double-Blind Phase 3 Trial. Circ Rep. 2026 Apr 23;8(5):797-806. doi: 10.1253/circrep.CR-25-0304. eCollection 2026 May 8.
- Gillmore JD, Judge DP, Cappelli F, Fontana M, Garcia-Pavia P, Gibbs S, Grogan M, Hanna M, Hoffman J, Masri A, Maurer MS, Nativi-Nicolau J, Obici L, Poulsen SH, Rockhold F, Shah KB, Soman P, Garg J, Chiswell K, Xu H, Cao X, Lystig T, Sinha U, Fox JC; ATTRibute-CM Investigators. A plain language review of the ATTRibute-CM study: efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy. Future Cardiol. 2025 Dec;21(12):1017-1025. doi: 10.1080/14796678.2025.2591426. Epub 2025 Dec 12.
- 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.
- Dasgupta N, Poulsen SH, Emdin M, Ambardekar AV, Shah KB, Hennum L, Marwah R, Allison M, Shivanna P, Siddhanti S, Tamby JF, Falvey H, Grodin JL. Contemporary Oral Medication Use and Frequency in Patients with Transthyretin Amyloid Cardiomyopathy. Am J Cardiovasc Drugs. 2025 Nov;25(6):829-839. doi: 10.1007/s40256-025-00752-x. Epub 2025 Aug 1.
- Maurer MS, Judge DP, Gillmore JD, Garcia-Pavia P, Masri A, Cappelli F, Alexander KM, Sarswat N, Grogan M, Ambardekar AV, Ducharme A, Poulsen SH, Lam K, Obici L, Soman P, Rao S, Tamby JF, Castano A, Fox JC, Adam B, Chepyala SR, Poland B, Sinha U, Fontana M. Early Increase in Serum Transthyretin by Acoramidis Independently Predicts Improved Survival in TTR Amyloid Cardiomyopathy. J Am Coll Cardiol. 2025 May 27;85(20):1911-1923. doi: 10.1016/j.jacc.2025.03.542.
- Judge DP, Alexander KM, Cappelli F, Fontana M, Garcia-Pavia P, Gibbs SDJ, Grogan M, Hanna M, Masri A, Maurer MS, Obici L, Soman P, Cao X, Lystig T, Tamby JF, Siddhanti S, Castano A, Katz L, Fox JC, Mahaffey KW, Gillmore JD. Efficacy of Acoramidis on All-Cause Mortality and Cardiovascular Hospitalization in Transthyretin Amyloid Cardiomyopathy. J Am Coll Cardiol. 2025 Mar 18;85(10):1003-1014. doi: 10.1016/j.jacc.2024.11.042.
- Soman P, Khouri MG, Lenihan D, Reyentovich A, Sperry BW, Sowalsky K, Bai Y, Du J, Katz L, Siddhanti S, Fox JC. Comparison of in-clinic assessment of 6MWT by conventional method and using wearable sensors for patients with ATTR-CM. Future Cardiol. 2025 Feb;21(2):75-81. doi: 10.1080/14796678.2025.2457881. Epub 2025 Jan 29.
- Gillmore JD, Judge DP, Cappelli F, Fontana M, Garcia-Pavia P, Gibbs S, Grogan M, Hanna M, Hoffman J, Masri A, Maurer MS, Nativi-Nicolau J, Obici L, Poulsen SH, Rockhold F, Shah KB, Soman P, Garg J, Chiswell K, Xu H, Cao X, Lystig T, Sinha U, Fox JC; ATTRibute-CM Investigators. Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2024 Jan 11;390(2):132-142. doi: 10.1056/NEJMoa2305434.
- Stern LK, Kittleson MM. Updates in Cardiac Amyloidosis Diagnosis and Treatment. Curr Oncol Rep. 2021 Mar 16;23(4):47. doi: 10.1007/s11912-021-01028-8.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Acoramidis | Unknown | 800 mg | — |