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AZD4063

Phase 1

Dilated Cardiomyopathy | Small molecule | Cardiovascular |AstraZeneca PLC|Last Updated: Mar 31, 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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment31
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07241104A Study of AZD4063 in PLN R14del Dilated CardiomyopathyPHASE1 RECRUITING 31 — — Dec 1, 2025Nov 22, 2027Mar 31, 20264 Netherlands
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Study Endpoints
Primary Endpoints
Number of participants with adverse events (AEs)
Cohort 1 (SAD): From Day 1 to Day 109, Cohorts 2 and 3 (SAD) From Day 1 to Day 99; For Cohorts 1,2 and 3 (MAD): Day 1 to Day 155

The safety and tolerability of AZD4063 following the SC administration of single and repeated doses in participants with PLN R14del dilated cardiomyopathy

Secondary Endpoints
Area under plasma concentration-time curve from 0 to infinity (AUCinf)
Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 141
Area under the plasma concentration-curve from 0 to the last quantifiable concentration (AUClast)
Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 141
Maximum plasma drug concentration (Cmax)
Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 141
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1 (SAD): Dose 1 of AZD4063EXPERIMENTALParticipants will receive Dose 1 of AZD4063 via SC injection in Cohort 1 of SAD.
Cohort 2 (SAD): Dose 2 of AZD4063EXPERIMENTALParticipants will receive Dose 2 of AZD4063 via SC injection in Cohort 2 of SAD.
Cohort 3 (SAD): Dose 3 of AZD4063EXPERIMENTALParticipants will receive Dose 3 of AZD4063 via SC injection in Cohort 3 of SAD.
Cohort 1 (MAD): Dose 4 of AZD4063EXPERIMENTALParticipants will receive Dose 4 of AZD4063 via SC injection in Cohort 1 of MAD.
Cohort 2 (MAD): Dose 5 of AZD4063EXPERIMENTALParticipants will receive Dose 5 of AZD4063 via SC injection in Cohort 2 of MAD.
Cohort 3 (MAD): Dose 6 of AZD4063EXPERIMENTALParticipants will receive Dose 6 of AZD4063 via SC injection in Cohort 3 of MAD.
Optional Cohort 1 (SAD): Dose 7 of AZD4063EXPERIMENTALParticipants will receive Dose 7 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Optional Cohort 2 (SAD): Dose 8 of AZD4063EXPERIMENTALParticipants will receive Dose 8 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Optional Cohort 1 (MAD): Dose 9 of AZD4063EXPERIMENTALParticipants will receive Dose 9 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Optional Cohort 2 (MAD): Dose 10 of AZD4063EXPERIMENTALParticipants will receive Dose 10 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Interventions
NameTypeDescription
AZD4063DRUGAZD4063 will be administered in the SAD and MAD part of the study as solution for injection via subcutaneous route of administration.
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Participants must be 18 to 80 years of age inclusive, at the time of Screening * Participants with pre-existing positive screening for R14 del PLN mutation * Participants with screening Left ventricular eject fraction ≤ 45% as assessed by echocardiography * Participants with N...

Countries:Netherlands
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07241104primaryCompletionDate: changed
LOWMay 24, 2026NCT07241104studyFirstPostDate: changed