Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06563895 | Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant | PHASE3 | RECRUITING | 587 | — | — | May 12, 2025 | Dec 1, 2032 | May 12, 2026 | 101 | United States, Argentina +22 |
| NCT03860935 | Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy | PHASE3 | COMPLETED | 632 | — | — | Mar 19, 2019 | May 11, 2023 | Jun 27, 2024 | 104 | United States, Australia +16 |
| NCT04769479 | A Single Dose Study to Evaluate the Pharmacokinetics of Acoramidis Modified Release Formulations in Healthy Subjects | PHASE1 | COMPLETED | 14 | — | — | Mar 28, 2021 | Sep 3, 2021 | Sep 19, 2024 | 1 | United Kingdom |
* ATTR-CM defined by biopsy or imaging-based diagnosis * ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis
The endpoint was analyzed using Finkelstein-Schoenfeld method. The method combines all-cause mortality, cumulative frequency of CV-related hospitalizations, change from baseline in NT-proBNP and change from baseline in 6MWT in a hierarchical fashion. The method compares every participant with every other participant within strata, assigning a +1 to the "better" participant and a -1 to the "worse" participant and 0 if they are "tied". Participants who had heart transplantation or implantation of a cardiac mechanical assist device were handled in the same manner as death. 'Win' represents a participant doing better based on hierarchical comparison. The reported unit is the total percent of "wins" for each treatment group from performing such a hierarchical comparison across stratification factors in the study.
Maximum Concentration (Cmax)
Area under the plasma concentration-time curve (AUC)
Time to maximum concentration (Tmax)
| Arm | Type | Description |
|---|---|---|
| acoramidis | EXPERIMENTAL | Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID) |
| Placebo | PLACEBO_COMPARATOR | Subjects will receive placebo to match twice daily |
| acoramidis HCl 800 mg | EXPERIMENTAL | Subjects will receive acoramidis HCl 800 mg twice daily. 6MWT primary outcome will be assessed at the end of 12 months. The hierarchical combination of All-Cause mortality, cumulative frequency of cardiovascular-related hospitalizations, change from baseline in NT-proBNP levels, and change from baseline in distance walked on the 6MWT will be assessed after 30 months of treatment. |
| Name | Type | Description |
|---|---|---|
| Acoramidis | DRUG | TTR stabilizer administered orally twice daily (BID) |
| Placebo oral tablet | DRUG | Non-active control administered orally twice daily (BID) |
Key Inclusion Criteria: * Male or female ≥ 18 to ≤ 75 years of age inclusive. * Participants must have an established genotype (hetero- or homozygosity) through a medically-indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic (eg, V30M/p.V50M, V122I/p.V14...