Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication ATTRUBY is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. FDA label
Also known as Acoramidis (AG10), AG10 oral tablet, AG10, ALXN2060
Acoramidis · 7 trials · 14 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.
Change in Diastolic Blood Pressure from Baseline to Day 28 (Postdose)
Change in Heart Rate from Baseline to Day 28 (Postdose)
Change in Respiratory Rate from Baseline to Day 28 (Postdose)
Change in Temperature from Baseline to Day 28
Change in Systolic Blood Pressure from Baseline to Day 28
Maximum Concentration (Cmax)
Area under the plasma concentration-time curve (AUC)
Time to maximum concentration (Tmax)
To evaluate the safety and tolerability of single and multiple doses of AG10 administered to healthy adult subjects
| 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. |
| AG10 Low Dose | ACTIVE_COMPARATOR | AG10 400mg tablets twice daily for 28 days |
| AG10 High Dose | ACTIVE_COMPARATOR | AG10 800mg tablets twice daily for 28 days |
| AG10 single oral dose | EXPERIMENTAL | AG10 oral tablet, administered by mouth, once |
| Placebo single oral dose | PLACEBO_COMPARATOR | Placebo Oral Tablet, administered by mouth, once |
| 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) |
| AG10 | DRUG | TTR stabilizer |
| AG10 oral tablet | DRUG | Active single ascending dose |
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...
Acoramidis is an oral small molecule drug in Phase 3 development for transthyretin (TTR) amyloidosis, including transthyretin amyloid cardiomyopathy (ATTR-CM) and related amyloidosis conditions. It is being developed by BridgeBio Pharma, Inc. (ticker BBIO) as a disease-directed therapy for patients with TTR amyloidosis.
Acoramidis targets transthyretin (TTR) and acts as a TTR stabilizer. By binding and stabilizing the TTR protein, the drug is designed to address the amyloidogenic process central to transthyretin amyloidosis. Its molecular target is TTR, and it belongs to the stabilizer class of small molecules.
Acoramidis is being developed by BridgeBio Pharma, Inc., which trades under the ticker BBIO. BridgeBio is the sponsor of the acoramidis clinical program, including the Phase 3 ACT-EARLY and ACT-EARLY OLE studies and the open-label safety study in symptomatic ATTR participants.
Acoramidis is in Phase 3 clinical development. It is an investigational drug and is not approved; it is still being studied in clinical trials for transthyretin amyloidosis, including ATTR-CM. Across its program, three Phase 3 trials and one Phase 1 pharmacokinetic study have been registered.
Acoramidis trials include NCT06563895 (ACT-EARLY), a recruiting Phase 3 prevention study in asymptomatic carriers of a pathogenic TTR variant; NCT07116473 (ACT-EARLY OLE), a not-yet-recruiting Phase 3 long-term safety study in newly diagnosed ATTR-CM; and NCT04988386, an active, not recruiting open-label Phase 3 safety study in symptomatic ATTR participants.
Yes, acoramidis is the same drug as AG10. AG10 is the earlier code name for acoramidis, and the oral tablet is sometimes referred to as AG10 oral tablet. Searches for AG10, AG10 oral tablet, or acoramidis (AG10) all refer to the same TTR stabilizer developed by BridgeBio Pharma.