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AT-001

Phase 3

Diabetic Cardiomyopathies | Small molecule | Other |Applied Therapeutics, Inc.|Last Updated: Dec 8, 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_CONTROLLED
Total Trials1
Total Enrollment675
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04083339Safety and Efficacy of AT-001 in Patients With Diabetic CardiomyopathyPHASE3 ACTIVE NOT_RECRUITING 675Sep 20, 2019Dec 1, 2025Dec 8, 202279 United States, Australia +8
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Study Endpoints
Primary Endpoints
Peak VO2 during cardio-pulmonary exercise test (CPET);
15 months after randomization]

Changes in Peak VO2 during cardio-pulmonary exercise test (CPET) from baseline to approximately Month 15 (15-18 months). A CPET may be repeated at approximately Month 27 (27-30 months).

Secondary Endpoints
Progression to overt heart failure (Stage C Heart Failure)
27 months after randomization
Changes in NT-proBNP
27 months after randomization
Changes in the modified Kansas City Cardiomyopathy Questionnaire (KCCQ) score
27 months after randomization
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AT-001 High doseEXPERIMENTALThe total daily doses will be of 3g. The dose selection and the frequency of dosing was based on the results of the phase 1/2 study demonstrating that 3g/day of AT-001 is capable of producing the maximum inhibition of the production of sorbitol (a pharmacodynamic biomarker of biological activity).
AT-001 Low DoseEXPERIMENTALThe total daily doses will be of 2g. The dose selection and the frequency of dosing was based on the results of the phase 1/2 study demonstrating that 2g/day of AT-001 is capable of producing a sufficient inhibition of the production of sorbitol (a pharmacodynamic biomarker of biological activity).
Placebo ComparatorPLACEBO_COMPARATORPlacebo capsules will be used as comparator
Interventions
NameTypeDescription
AT-001DRUGAT-001 will be administered as 3 capsules twice daily, before breakfast and before dinner. At present AT001 is the sole name for the active substance. No INN/genetic name is available to date
PlaceboDRUGMatching placebo will be administered as 3 capsules twice daily, before breakfast and before dinner
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Eligibility Criteria
Age Range40 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites79

Inclusion Criteria: * Type 2 Diabetes Mellitus * Diabetic cardiomyopathy * Peak VO2 \< 75% of predicted normal value based on age and gender Exclusion Criteria: * Prior diagnosis or signs/symptoms of overt/symptomatic heart failure / stage C heart failure * Prior echocardiogrphic measurement of e...

Countries:United StatesAustraliaCanadaCzechiaFranceGermanyHong KongPolandSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04083339primaryCompletionDate: changed
LOWMay 24, 2026NCT04083339studyFirstPostDate: changed