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Eplontersen

Phase 3

Transthyretin Amyloid Cardiomyopathy | Small molecule | Cardiovascular |AstraZeneca PLC|Last Updated: Apr 27, 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 Enrollment64
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06194825EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid CardiomyopathyPHASE3 ACTIVE NOT_RECRUITING 64Dec 1, 2023Jan 5, 2027Apr 27, 202626 China
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Study Endpoints
Primary Endpoints
Percent change of serum TTR concentration from baseline
up to 24 weeks

To evaluate the effect of eplontersen compared to placebo on serum transthyretin (TTR) concentration up to 24 weeks

Secondary Endpoints
Changes from baseline in NT-proBNP serum level
up to 24 weeks
Changes from baseline in hs-cTnT serum level
up to 24 weeks
Plasma concentrations of eplontersen at specified time-points (Week0,2,4,8,12,16,20,24,28,32,36,48,60,72,84,96,104)
up to 124 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
EplontersenEXPERIMENTALEplontersen by subcutaneous injection once every 4 weeks
placeboPLACEBO_COMPARATOREplontersen-matching placebo by subcutaneous injection once every 4 weeks
Interventions
NameTypeDescription
EplontersenDRUGEplontersen by subcutaneous injection
PlaceboDRUGEplontersen-matching placebo by subcutaneous injection
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Eligibility Criteria
Age Range20 Years — 90 Years
SexALL
Healthy VolunteersNo
Study Sites26

Inclusion Criteria: 1. Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements. 2. 20 to 90 years of age (inclusive). 3. Females: must be non-pregnant and non-lactating and either: 1. surgically s...

Countries:China
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06194825primaryCompletionDate: changed
LOWMay 24, 2026NCT06194825studyFirstPostDate: changed