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AG10

Phase 2

Amyloid Cardiomyopathy | Small molecule | Cardiovascular |BridgeBio Pharma, Inc.|Last Updated: Mar 13, 2025

Target and mechanism

Molecular targetTTR
Target classProtein
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment55

FDA Designations

No designations recorded

Clinical trial landscape

AG10 · 3 trials · 4 indications

Phase 2 2Phase 1 1
NCT03536767Open-Label Study of AG10 in Patients with CardiomyopathyAmyloid Cardiomyopathy
ACTIVE NOT_RECRUITING55 Analytics
NCT03458130Study of AG10 in Amyloid CardiomyopathyFamilial ATTR-CM (ATTRm-CM, or FAC)
COMPLETED49 Analytics
PHASE2ACTIVE NOT_RECRUITING
Open-Label Study of AG10 in Patients with Cardiomyopathy
Amyloid CardiomyopathyUnlock trial analytics
PHASE2COMPLETED
Study of AG10 in Amyloid Cardiomyopathy
Familial ATTR-CM (ATTRm-CM, or FAC)Unlock trial analytics

Study Endpoints

Primary Endpoints

Assessment of long-term safety and tolerability: Incidence of each treatment-emergent adverse events
up to 60 Months or study completion by recommendation from safety monitoring committee

Incidence of each treatment-emergent adverse events

Change in Diastolic Blood Pressure
Baseline to Day 28

Change in Diastolic Blood Pressure from Baseline to Day 28 (Postdose)

Change in Heart Rate
Baseline to Day 28

Change in Heart Rate from Baseline to Day 28 (Postdose)

Change in Respiratory Rate
Baseline to Day 28

Change in Respiratory Rate from Baseline to Day 28 (Postdose)

Change in Temperature
Baseline to Day 28

Change in Temperature from Baseline to Day 28

Change in Systolic Blood Pressure
Baseline to Day 28

Change in Systolic Blood Pressure from Baseline to Day 28

Safety & tolerability: individual and summary blood pressures, heart rate, ECG and lab data presented in tabular form with descriptive statistics. Adverse events will be tabulated and summarized by Part A (SAD) vs. B (MAD), and treatment.
30 days

To evaluate the safety and tolerability of single and multiple doses of AG10 administered to healthy adult subjects

Secondary Endpoints

AG10 Pharmacokinetics AUC
up to 60 Months or study completion by recommendation from safety monitoring committee
AG10 Pharmacodynamic Assessments of TTR stabilization by Fluorescent Polarization Exclusion Assay
up to 60 Months or study completion by recommendation from safety monitoring committee
AG10 Pharmacodynamic Assessments of TTR stabilization by Western Blot
up to 60 Months or study completion by recommendation from safety monitoring committee
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Open-LabelEXPERIMENTAL -
AG10 Low DoseACTIVE_COMPARATORAG10 400mg tablets twice daily for 28 days
AG10 High DoseACTIVE_COMPARATORAG10 800mg tablets twice daily for 28 days
PlaceboPLACEBO_COMPARATORPlacebo tablets twice daily for 28 days
AG10 single oral doseEXPERIMENTALAG10 oral tablet, administered by mouth, once
Placebo single oral dosePLACEBO_COMPARATORPlacebo Oral Tablet, administered by mouth, once

Interventions

NameTypeDescription
AG10DRUGAG10 800mg twice daily, oral administration
Placebo Oral TabletDRUGNonactive control
AG10 oral tabletDRUGActive single ascending dose
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Eligibility Criteria

Age Range18 Years to 90 Years
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: 1. Completed participation in study AG10-201. 2. Willing and able to comply with the study medication regimen and all study requirements. 3. The ability to understand and provide informed consent, which must be obtained prior to initiation of study participation. 4. Male patient...

Countries:United States
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Frequently asked questions about AG10

What is AG10 used for?

AG10 is an investigational small molecule being developed for the treatment of amyloid cardiomyopathy, including transthyretin-related amyloid cardiomyopathy and familial ATTR-CM (ATTRm-CM, or FAC). It is designed to address the underlying causes of these conditions, which affect the heart's function.

What does AG10 target?

AG10 targets the transthyretin (TTR) protein. By binding to TTR, AG10 aims to stabilize the protein and prevent its misfolding and aggregation, which are key steps in the development of transthyretin-related amyloid cardiomyopathy.

Who makes AG10?

AG10 is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company. BridgeBio is focused on discovering and developing innovative therapies for genetic diseases and cancers. The company's ticker symbol is BBIO.

What phase is AG10 in?

AG10 is currently in Phase 2 clinical development. It has completed Phase 1 and Phase 2 trials, and an additional Phase 2 study is active but not recruiting participants. AG10 is not yet approved by regulatory authorities and remains investigational.

What clinical trials is AG10 in?

AG10 has been studied in several clinical trials. NCT03294707 was a Phase 1 study of safety and tolerability. NCT03458130 was a Phase 2 study in amyloid cardiomyopathy. NCT03536767 is an active Phase 2 open-label study in patients with cardiomyopathy.

Is AG10 the same as acoramidis?

AG10 is also known as acoramidis. This alternative name is used in some clinical and scientific contexts. Both names refer to the same investigational drug being developed by BridgeBio Pharma for the treatment of transthyretin-related amyloid cardiomyopathy.