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Eplontersen

Phase 3

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Small molecule | Neurology |Ionis Pharmaceuticals, Inc.|Last Updated: Sep 3, 2026

Target and mechanism

Molecular targetTTR
Target classAntisense Inhibitor
ModalitySmall molecule
ChEMBLCHEMBL4594334

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment151

FDA Designations

FAST_TRACK

Clinical trial landscape

Eplontersen · 4 trials · 3 indications

Phase 3 3Phase 1 1
NCT05667493An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
ENROLLING BY_INVITATION1,400 Analytics
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 PolyneuropathyHereditary Transthyretin-Mediated Amyloid Polyneuropathy
ACTIVE NOT_RECRUITING151 Analytics
NCT04136171CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
COMPLETED1,438 Analytics
PHASE3ENROLLING BY_INVITATION
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
NCT056674931,400
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PHASE3ACTIVE NOT_RECRUITING
A 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 Polyneuropathy
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
NCT05071300151
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PHASE3COMPLETED
CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
NCT041361711,438
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Study Endpoints

Primary Endpoints

Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
Baseline up to 36 months
Change From Baseline in Platelet Count
Baseline up to 36 months
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
Baseline up to 36 months
Change From Baseline in Urine Protein Creatinine Ratio (UPCR)
Baseline up to 36 months
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
Composite Outcome of Cardiovascular (CV) Mortality and Recurrent CV Clinical Events up to Week 140
Baseline up to Week 140
Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity
Up to 92 days
Percentage of Participants with TEAEs Potentially Related to Study Drug
Up to 92 days
Percentage of Participants with Changes in Clinically Significant (CS) Laboratory Value Abnormalities
Up to 92 days

Secondary Endpoints

Change From Baseline in Transthyretin (TTR) Serum Levels
Baseline up to 36 months
Change From Baseline in 6-minute Walk Test (6MWT)
Baseline up to 36 months
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)
Baseline up to 36 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
EplontersenEXPERIMENTALEplontersen will be administered once every month by sub-cutaneous (SC) injection for up to 36 months or 6 months after eplontersen is approved and available in the site's country, whichever occurs first.
PlaceboPLACEBO_COMPARATOREplontersen-matching placebo by subcutaneous injection once every 4 weeks

Interventions

NameTypeDescription
EplontersenDRUGEplontersen will be administered by SC injection.
PlaceboDRUGEplontersen-matching placebo by subcutaneous injection
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites134

Inclusion Criteria: 1. Satisfactory completion of Treatment Period and the End of Treatment Visit of the Index Study (ION-682884-CS2) OR diagnosis of ATTR-CM and satisfactory participation on ISIS 420915- CS101 study as judged by the Investigator and Sponsor. 2. Investigator is willing to treat the...

Countries:United StatesArgentinaAustraliaAustriaBelgiumBrazilCanadaCzechiaDenmarkFranceGermanyGreeceIsraelItalyJapanPolandPortugalSpainSwedenUnited KingdomCyprusTaiwanTurkey (Türkiye)Puerto Rico
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Recent Changes (Last 90 Days)

LOWSep 3, 2026NCT04136171lastUpdatePostDate: changed
LOWSep 3, 2026NCT04136171lastUpdatePostDate: changed
HIGHAug 25, 2026NCT04136171Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHAug 25, 2026NCT04136171Status: ACTIVE_NOT_RECRUITING → COMPLETED

Frequently asked questions about Eplontersen

What is Eplontersen used for?

Eplontersen is an investigational antisense oligonucleotide being developed for transthyretin-mediated amyloid cardiomyopathy (ATTR CM) and hereditary transthyretin-mediated amyloid polyneuropathy. It is also studied in healthy participants for safety and pharmacokinetic assessments. It has received Fast Track designation from the FDA.

What does Eplontersen target?

Eplontersen is an antisense oligonucleotide (ASO) that targets transthyretin messenger RNA to reduce production of the transthyretin protein, which forms amyloid deposits in tissues. This mechanism is being evaluated in clinical trials for ATTR CM and hereditary transthyretin-mediated amyloid polyneuropathy.

Who makes Eplontersen?

Eplontersen is developed by Ionis Pharmaceuticals, Inc., traded on NASDAQ under the ticker IONS. The company is conducting clinical trials to evaluate the drug's safety and efficacy in transthyretin-mediated amyloid diseases.

What phase is Eplontersen in?

Eplontersen is in Phase 3 clinical development for transthyretin-mediated amyloid cardiomyopathy and hereditary transthyretin-mediated amyloid polyneuropathy. It is also being studied in a Phase 1 trial in healthy Japanese participants. It is investigational and not yet approved by the FDA.

What clinical trials is Eplontersen in?

Eplontersen is being studied in several clinical trials, including NCT04136171 (CARDIO-TTRansform, Phase 3, completed), NCT04302064 (Phase 1, completed), NCT05071300 (Phase 3, active), and NCT05667493 (Phase 3 extension study). These trials assess safety, efficacy, and long-term outcomes in ATTR CM and polyneuropathy.

Is Eplontersen the same as ION-682884?

Yes, Eplontersen was formerly known as ION-682884, IONIS-TTR-LRx, and AKCEA-TTR-LRx. These names refer to the same investigational drug in clinical trials for transthyretin-mediated amyloid diseases.

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