Drug

Pelacarsen

In clinical development

ClassLPA mRNA antisense inhibitorEmerging researchLate-stage development

Also known as IONIS-APO(a)-LRx, Isis-681257 (galnac3--conjugated aso targeting human lipoprotein (a)) free acid.

RxNorm2471442 ChEMBLCHEMBL4298202UNIILSO9H7UZ90

Research papers

Active clinical trials

Protein targets

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of Pelacarsen (TQJ230) With Background Inclisiran in Participants With Atherosclerotic Cardiovascular Disease (ASCVD), and Elevated LDL-C and Lp(a)

Clinical trial2026-07-14Results expected Q1 2028 · ClinicalTrials.gov

A Single Arm, Multicenter, Open-label Extension (OLE) Trial to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230) in Participants Who Completed the Parent Lp(a)HORIZON Trial

Clinical trial2026-07-08Results expected Q1 2030 · ClinicalTrials.gov

A Randomized Double-blind, Placebo-controlled, Multicenter Trial Assessing the Impact of Lipoprotein(a) Lowering With Pelacarsen (TQJ230) on the Progression of Calcific Aortic Valve Stenosis [Lp(a)FRONTIERS CAVS]

Clinical trial2026-06-23Results expected Q1 2030 · ClinicalTrials.gov

A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open-Label Pelacarsen in Participants With Elevated Lp(a) and Established ASCVD

Clinical trial2026-06-11Results expected Q4 2030 · ClinicalTrials.gov

A Randomized, Double-blind, Placebo-controlled, Multicenter Study Evaluating the Efficacy, Safety, Tolerability, and Coronary Plaque Effects of Early Pelacarsen (TQJ230) Initiation in Patients in the US With Elevated Lp(a) and Recent Acute Coronary Syndrome

Clinical trial2026-06-04Results expected Q4 2028 · ClinicalTrials.gov

A Single Arm, Open Label Extension (OLE), Multicenter Study to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230) in Patients With Cardiovascular Disease Who Have Successfully Completed the Apheresis Parent Study.

Clinical trial2025-08-06Results expected Q3 2028 · ClinicalTrials.gov

Design and Rationale of Lp(a)HORIZON Trial: Assessing the Effect of Lipoprotein(a) Lowering With Pelacarsen on Major Cardiovascular Events in Patients With CVD and Elevated Lp(a).

Research2025-04-02American heart journal

A Single-dose, Open-label, Parallel-group Study to Assess the Pharmacokinetics of Pelacarsen (TQJ230) in Participants With Mild Hepatic Impairment Compared to Matched Healthy Participants

Clinical trial2022-10-19Completed · ClinicalTrials.gov

Influence of Pelacarsen on Arterial Wall Properties and Risk Factors in Patients After Myocardial Infarction With High Lp(a) Values

Clinical trial2022-10-01Withdrawn · ClinicalTrials.gov

A Randomized Double-blind, Placebo-controlled, Non-confirmatory Study to Assess Safety, Tolerability, PK, and PD of Single Ascending and Multiple Doses of ISIS 681257 in Healthy Japanese Participants

Clinical trial2019-12-18Completed · ClinicalTrials.gov

A Blinded, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ISIS 681257 Administered Subcutaneously to Healthy Volunteers With Elevated Lipoprotein(a)

Clinical trial2016-02-01Completed · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Pelacarsen

Aliases & brands

IONIS-APO(a)-LRxIsis-681257 (galnac3--conjugated aso targeting human lipoprotein (a)) free acid

RxNorm CUI

2471442

ChEMBL ID

CHEMBL4298202

UNII

LSO9H7UZ90

Primary mechanism

LPA mRNA antisense inhibitor

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

ENSG00000198670

ANTISENSE INHIBITORLPA mRNA antisense inhibitor

Clinical trials

10 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme

CLINICALTRIALS.GOV · LIVE REGISTRY

registered trials across all phases

LATEST COMPLETION 2022

Active studies

Recruiting

Late-stage (III+)

Completed

Discontinued

PHASE DISTRIBUTIONn = 10

Phase 13Phase 21Phase 35Phase N / A1

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open-Label Pelacarsen in Participants With Elevated Lp(a) and Established ASCVD

RecruitingPH IIINovartis Pharmaceuticalsn=5992030

A Single Arm, Multicenter, Open-label Extension (OLE) Trial to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230) in Participants Who Completed the Parent Lp(a)HORIZON Trial

RecruitingPH IIIKey trialNovartis Pharmaceuticalsn=5,7002030

Not yet recruitingPH IIINovartis Pharmaceuticalsn=2402028

Active, not recruitingPH IIINovartis Pharmaceuticalsn=412028

RecruitingPH IIINovartis Pharmaceuticalsn=3402028

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Single-dose, Open-label, Parallel-group Study to Assess the Pharmacokinetics of Pelacarsen (TQJ230) in Participants With Mild Hepatic Impairment Compared to Matched Healthy Participants

CompletedPH INovartis Pharmaceuticalsn=172022

A Randomized Double-blind, Placebo-controlled, Non-confirmatory Study to Assess Safety, Tolerability, PK, and PD of Single Ascending and Multiple Doses of ISIS 681257 in Healthy Japanese Participants

CompletedPH IIonis Pharmaceuticals, Inc.n=292019

CompletedPH IIonis Pharmaceuticals, Inc.n=602016

Research activity

1 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Most influential

Design and Rationale of Lp(a)HORIZON Trial: Assessing the Effect of Lipoprotein(a) Lowering With Pelacarsen on Major Cardiovascular Events in Patients With CVD and Elevated Lp(a).

American heart journal · 2025 · 49 cites