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ALN-APOC3

Phase 1

Dyslipidemia | Small molecule | Metabolic |Regeneron Pharmaceuticals, Inc.|Last Updated: Mar 16, 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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06784349ALN-APOC3 in Adult Participants With DyslipidemiaPHASE1 COMPLETED 32Jan 27, 2025Feb 25, 2026Mar 16, 20261 Moldova
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Study Endpoints
Primary Endpoints
Incidence of Treatment Emergent Adverse Events (TEAEs)
Through Week 44
Severity of TEAEs
Through Week 44
Percent changes in fasting concentrations of High-Density Lipoprotein Cholesterol (HDL-C)
Baseline to week 24

Part B Only

Secondary Endpoints
Concentration of combined ALN-APOC3 and metabolites in plasma
Through Week 44
Urinary excretion of combined ALN-APOC3 and metabolites
Through 24 Hours, After Treatment
Percent change in fasting concentrations of APOC3
Baseline to Week 12
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-APOC3 Ascending CohortsEXPERIMENTALPart A: Ascending Cohorts Randomized as described in the protocol May include optional cohorts
PlaceboPLACEBO_COMPARATORPart A and Part B: Randomized as described in the protocol
ALN-APOC3 low-doseEXPERIMENTALPart B: Parallel Arm Randomized as described in the protocol
ALN-APOC3 medium-doseEXPERIMENTALPart B: Parallel Arm Randomized as described in the protocol
ALN-APOC3 high-doseEXPERIMENTALPart B: Parallel Arm Randomized as described in the protocol
Interventions
NameTypeDescription
ALN-APOC3DRUGAdministered as per the protocol
Matching PlaceboDRUGAdministered as per the protocol
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersYes
Study Sites1

Key Inclusion Criteria: 1. Judged by the investigator to be in good health based on medical history, physical examination, vital signs, Electrocardiograms (ECG's) and laboratory safety testing, as defined in the protocol 2. Participants can enter the study under one of the following options, as def...

Countries:Moldova
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Recent Changes (Last 90 Days)
MEDIUMMay 21, 2026NCT06784349TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT06784349TRIAL_REMOVED: changed