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AG10

Phase 2

Familial ATTR-CM (ATTRm-CM, or FAC) | Small molecule | Other |BridgeBio Pharma, Inc.|Last Updated: Nov 16, 2022

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment49
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03458130Study of AG10 in Amyloid CardiomyopathyPHASE2 COMPLETED 49 — — Apr 27, 2018Oct 5, 2018Nov 16, 202213 United States
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Study Endpoints
Primary Endpoints
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

Secondary Endpoints
Number of Participants With Threshold Levels of Overall % Stabilization >= 95% and >= 99% by Fluorescent Probe Exclusion (FPE)
Day 1 to Day 28
Pharmacokinetic (PK): Steady State Trough Concentration of AG10
Day 14 and Day 28
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
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
Interventions
NameTypeDescription
AG10DRUGTTR stabilizer
Placebo Oral TabletDRUGNonactive control
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Eligibility Criteria
Age Range18 Years — 90 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: 1. Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures. 2. Be a male or female ≥18 to ≤90 years of age. 3. Have an established diagnosis of ATTR-CM with either wild-type transthyretin or a varian...

Countries:United States
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