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Eplontersen

Phase 3

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Small molecule | Neurology |Ionis Pharmaceuticals, Inc.|Last Updated: Dec 4, 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 Enrollment151
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05071300A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid PolyneuropathyPHASE3 ACTIVE NOT_RECRUITING 151 — — Jan 4, 2022Aug 1, 2029Dec 4, 202533 United States, Argentina +11
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Study Endpoints
Primary Endpoints
Change From Baseline in Platelet Count
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Renal Function
Baseline to Week 181
Number of Participants with Clinically Significant Changes from Baseline in Transaminases
Baseline to Week 181
Change From Baseline in Adverse Events
Baseline to Week 181
Change From Baseline in Number of Concomitant Medications Used
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Baseline to Week 181
Change From Baseline in Body Weight
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Physical Examination Findings
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Tests
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Thyroid Panel Tests
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Coagulation Tests
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Inflammatory Panel Tests
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Complement and Immunogenicity Tests
Baseline to Week 181
Secondary Endpoints
Change From Baseline in Neuropathy Impairment Score (NIS)
Baseline to Week 181
Change From Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Questionnaire
Baseline to Week 181
Change From Baseline in Neuropathy Symptom and Change Score (NSC)
Baseline to Week 181
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
EplontersenEXPERIMENTALEplontersen will be administered by subcutaneous (SC) injection once every 4 weeks for up to 3 years (157 weeks).
Interventions
NameTypeDescription
EplontersenDRUGEplontersen will be administered by SC injection.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites33

Inclusion Criteria: 1. Satisfactory completion of ION-682884-CS3 (NCT04136184) (Index Study) as judged by the Investigator and Sponsor, or diagnosis of hATTR-PN and satisfactory completion of either study ISIS 420915-CS101 or study 2018-P001436 (NCT03702829) (both are Investigator-Sponsored studies...

Countries:United StatesArgentinaAustraliaBrazilCanadaCyprusFranceItalyPortugalSpainSwedenTaiwanTurkey (Türkiye)
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05071300primaryCompletionDate: changed
LOWMay 24, 2026NCT05071300studyFirstPostDate: changed