Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
acoramidis · 5 trials · 12 indications
Proportion of participants with: treatment-emergent AEs and SAEs, AEs leading to treatment discontinuation, abnormal physical examination findings of clinical relevance, abnormal vital signs of clinical relevance, abnormal ECG parameters of clinical relevance, changes in clinical safety laboratory parameters of potential concern
* ATTR-CM defined by biopsy or imaging-based diagnosis * ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis
Incidence of each treatment-emergent adverse events measured over 60 Months or study completion
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 |
| AG10 | EXPERIMENTAL | Open-label study all participants will receive AG10 during this study. |
| 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 (AG10) | DRUG | TTR stabilizer administered orally twice daily (BID) |
| Acoramidis | DRUG | TTR stabilizer administered orally twice daily (BID) |
| Placebo oral tablet | DRUG | Non-active control administered orally twice daily (BID) |
Key Inclusion Criteria: Participants must have completed the AG10-501 study (ACT-EARLY study) within the past 60 calendar days with a diagnosis of ATTR-CM (based on the AG10-501 protocol definition of ATTR-CM). Key Exclusion Criteria: 1. Participants who completed the AG10-501 study with a diagno...