Pelacarsen

Summary

Pelacarsen (CHEMBL4298202) is a phase-3 clinical-stage oligonucleotide; indicated across 6 conditions including hyperlipoproteinemia and cardiovascular disorder.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• Indications: 6 conditions
• Clinical trials: 14

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4298202
Name	Pelacarsen
Type	Oligonucleotide
Max phase	3

Also known as: AKCEA-APO(a)-LRx, Ionis-apo(a)-lrx, ISIS 681257, ISIS-681257 FREE ACID, Pelacarsen, PELACARSEN

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

6 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
hyperlipoproteinemia	3	MONDO:0037748	MONDO:0037748
cardiovascular disorder	2	MONDO:0004995	EFO:0000319
aortic valve stenosis	2	MONDO:0042981	HP:0001650
kidney disorder	1	MONDO:0005240	EFO:0003086
liver disorder	1	MONDO:0005154	EFO:0001421
coronary artery disorder	1	MONDO:0005010	EFO:0001645

Clinical trials

Total trials: 14.

Phase distribution

Phase	Trials
PHASE1	6
PHASE3	4
PHASE2	2
Not specified	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05900141	PHASE3	ACTIVE_NOT_RECRUITING	An Open Label Extension (OLE) Study to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230)
NCT06813911	PHASE3	RECRUITING	Lp(a) Lowering Study of Pelacarsen (TQJ230) With Background Inclisiran in Participants With Elevated Lp(a) and Established ASCVD
NCT06875973	PHASE3	RECRUITING	Pelacarsen Roll-over Extension Program
NCT07517263	PHASE3	RECRUITING	An Open Label Extension (OLE) Study (Following Completion of CTQJ230A12301) to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230)
NCT05646381	PHASE2	RECRUITING	A Multicenter Trial Assessing the Impact of Lipoprotein(a) Lowering With Pelacarsen (TQJ230) on the Progression of Calcific Aortic Valve Stenosis
NCT03070782	PHASE2	COMPLETED	Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease
NCT02414594	PHASE1	COMPLETED	Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)
NCT03392051	PHASE1	COMPLETED	Drug-drug Interaction Study to Evaluate the Effect of ISIS 681257 on Clopidogrel
NCT03426033	PHASE1	COMPLETED	Drug-drug Interaction Study to Evaluate the Effect of ISIS 681257 on Warfarin
NCT03506854	PHASE1	COMPLETED	Study of ISIS 681257 in Patients With Renal Impairment Compared to Healthy Patients
NCT05026996	PHASE1	COMPLETED	Study to Assess the Pharmacokinetics of Pelacarsen (TQJ230) in Participants With Mild Hepatic Impairment Compared to Matched Healthy Participants
NCT05337878	PHASE1	COMPLETED	A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants
NCT07000695	Not specified	AVAILABLE	Managed Access Programs for TQJ230, Pelacarsen
NCT04993664	Not specified	WITHDRAWN	Influence of Pelacarsen on Patients After Myocardial Infarction With High Lp(a) Values (PEMILA)

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).