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AZD0780

Phase 3

Heterozygous Familial Hypercholesterolaemia | Small molecule | Metabolic |AstraZeneca PLC|Last Updated: May 6, 2026

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 Enrollment473
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07000136A Phase III Study to Assess the Effect of AZD0780 on LDL-C in Patients With HeFHPHASE3 ACTIVE NOT_RECRUITING 473 — — Jun 10, 2025Jan 4, 2027May 6, 2026134 United States, Argentina +22
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Study Endpoints
Primary Endpoints
Relative change in LDL-C from baseline to 12 weeks
Baseline - 12 weeks

To compare the effect of treatment with AZD0780 versus placebo on LDL-C at 12 weeks

Secondary Endpoints
Relative change in LDL-C from baseline to 12 weeks
Baseline - 12 weeks
Indicator for LDL-C < 70 mg/dL (< 1.8 mmol/L) at 12 weeks
Baseline - 12 weeks
Indicator for LDL-C < 55 mg/dL (< 1.4 mmol/L) at 12 weeks
Baseline - 12 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AZD0780EXPERIMENTALParticipants will receive daily oral dose of AZD0780
PlaceboPLACEBO_COMPARATORParticipants will receive daily oral dose of placebo
Interventions
NameTypeDescription
AZD0780DRUGParticipants will receive daily oral dose of AZD0780
PlaceboDRUGParticipants will receive daily oral dose of placebo
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites134

Inclusion criteria: •≥ 18 years of age at the time of signing the ICF. * Diagnosis of HeFH by genetic confirmation or a definite clinical diagnosis, ie, a score \> x using the Dutch Lipid Network \[Nordestgaard et al 2013\] or equivalent as per internationally accepted diagnostic algorithms (AHA \...

Countries:United StatesArgentinaAustraliaBrazilBulgariaCanadaChileCzechiaDenmarkFinlandFranceGermanyHungaryJapanNetherlandsNew ZealandNorwaySlovakiaSouth KoreaSpainSwedenTaiwanTurkey (Türkiye)Vietnam
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07000136Enrollment: 455 → 473
LOWMay 24, 2026NCT07000136studyFirstPostDate: changed