Eplontersen

Summary

Eplontersen (CHEMBL4594334) is a phase-3 clinical-stage oligonucleotide (ATC N07XX21); indicated across 2 conditions including familial amyloid neuropathy and amyloidosis.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• ATC class: N07XX21
• Indications: 2 conditions
• Clinical trials: 9

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4594334
Name	Eplontersen
Type	Oligonucleotide
Max phase	3
ATC	N07XX21

Also known as: Akcea ttr lrx, Akcea-ttr-lrx, AKCEA-TTR-LRx, Eplontersen, ION-682884, ION-682884 FREE ACID, Ion-ttr-lrx, IONIS-TTR-LRx, ISIS-682884 FREE ACID, EPLONTERSEN

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

2 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
familial amyloid neuropathy	3	MONDO:0007100	EFO:0004129
amyloidosis	1	MONDO:0019065	EFO:1001875

Clinical trials

Total trials: 9.

Phase distribution

Phase	Trials
PHASE3	5
PHASE1	2
PHASE1/PHASE2	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04136171	PHASE3	ACTIVE_NOT_RECRUITING	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)
NCT05071300	PHASE3	ACTIVE_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
NCT05667493	PHASE3	ENROLLING_BY_INVITATION	An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
NCT06194825	PHASE3	ACTIVE_NOT_RECRUITING	EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
NCT04136184	PHASE3	COMPLETED	NEURO-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 Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
NCT07608354	PHASE2	NOT_YET_RECRUITING	A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM.
NCT03728634	PHASE1/PHASE2	COMPLETED	Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).
NCT04302064	PHASE1	COMPLETED	A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Healthy Japanese Participants
NCT06527755	PHASE1	COMPLETED	A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).