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AZD7503 Intervention

Phase 1

Non-alcoholic Fatty Liver Disease | Small molecule | Infectious Disease |AstraZeneca PLC|Last Updated: Jul 20, 2025

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment19
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05560607Knockdown of HSD17B13 mRNA, Pharmacokinetics, Safety, and Tolerability, of AZD7503 in Non-Alcoholic Fatty Liver DiseasePHASE1 COMPLETED 19Aug 12, 2022Feb 27, 2024Jul 20, 20251 United States
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Study Endpoints
Primary Endpoints
Number of participants with Adverse Events (AEs).
99 days

To assess adverse events as a variable of safety and tolerability of AZD7503.

Secondary Endpoints
Change in HSD17B13 mRNA Expression
31 days
Number of participants with positive anti-drug antibodies to AZD7503
99 days
Area under plasma concentration time-curve from zero to infinity (AUCinf) of AZD7503
99 days
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeOTHER
Treatment Arms
ArmTypeDescription
Intervention/ DrugEXPERIMENTALInvestigation of the knockdown of hepatic HSD17B13 mRNA expression, PK, safety, and tolerability following multiple dose administration of AZD7503 in male participants and female participants of non-childbearing potential with NAFLD or NASH
Interventions
NameTypeDescription
AZD7503 InterventionDRUGPart A: Participants will be screened for histologic evidence of NAFLD or NASH and all eligibility criteria in part A prior to enrollment in part B. Part B: Participants consented to part B will be administered the study drug over the course of 31 days. At the end of the study a liver biopsy will be collected to measure for endpoints.
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria # Part A 1. Participant must be ≥ 18 to ≤ 70 years of age at the time of signing the informed consent. 2. Participants with suspected or confirmed NAFLD or NASH including laboratory values with any of the following deviations at screening 1. ALT \> ULN, 2. Imaging demonstr...

Countries:United States
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