Inotersen

Summary

Inotersen (CHEMBL4297770) is an approved oligonucleotide (ATC N07XX15); indicated across 2 conditions including amyloidosis and familial amyloid neuropathy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: N07XX15
• Indications: 2 conditions
• Clinical trials: 7

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297770
Name	Inotersen
Type	Oligonucleotide
Max phase	4
ATC	N07XX15

Also known as: GSK-2998728, Inotersen, ISIS 420915, ISIS-420915, INOTERSEN

Parent form; salt/anhydrous children: CHEMBL4297537

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 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
amyloidosis	3	MONDO:0019065	EFO:1001875
familial amyloid neuropathy	3	MONDO:0007100	EFO:0004129

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE3	2
PHASE2	2
Not specified	2
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01737398	PHASE2/PHASE3	COMPLETED	Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
NCT02175004	PHASE3	COMPLETED	Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
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
NCT02627820	PHASE2	WITHDRAWN	The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart
NCT03702829	PHASE2	TERMINATED	24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients
NCT04270058	Not specified	NOT_YET_RECRUITING	TEGSEDI Pregnancy Surveillance Program
NCT03400098	Not specified	APPROVED_FOR_MARKETING	ATTR Expanded Access Program (EAP) by Ionis

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).