> Trialight public data. Index of pages: https://trialight.com/llms.txt
> Web page: https://trialight.com/data/asset/pelacarsen-bdcd4b0c-fd35-4862-abc9-48f7708d80e8
> Record changed: 2026-11-04

# Pelacarsen

Highest phase reached

Phase 3

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298202)

Drug type

Oligonucleotide

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298202)

## Development status

**Listing at source**

Listed

**Highest phase reached**

Phase 3

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298202)

## Names and chemistry

**Drug type**

Oligonucleotide

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298202)

**Drug name**

Pelacarsen

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298202)

### Other names (22)

- akcea apo a lrx
- AKCEA-APO(a)-LRx
- all p ambo 5 o 6 5 tris 3 6 2 acetamido 2 deoxy beta d galactopyranosyloxy hexylamino 3 oxopropoxymethyl methyl amino 5 oxopentanamido hexoxy phospho 2 o 2 methoxyethyl 5 methyl p thiouridylyl 3 o 5 o 2 o 2 methoxyethyl guanylyl 3 o 5 o 2 o 2 methoxyethyl 5 methylcytidylyl 3 o 5 o 2 o 2 methoxyethyl 5 methyluridylyl 3 o 5 o 2 o 2 methoxyethyl 5 methylcytidylyl 3 o 5 o 2 deoxy 5 methyl p thiocytidylyl 3 o 5 o 2 deoxy p thioguanylyl 3 o 5 o p thiothymidylyl 3 o 5 o p thiothymidylyl 3 o 5 o 2 deoxy p thioguanylyl 3 o 5 o 2 deoxy p thioguanylyl 3 o 5 o p thiothymidylyl 3 o 5 o 2 deoxy p thioguanylyl 3 o 5 o 2 deoxy 5 methyl p thiocytidylyl 3 o 5 o p thiothymidylyl 3 o 5 o 2 o 2 methoxyethyl 5 methyluridylyl 3 o 5 o 2 o 2 methoxyethyl guanylyl 3 o 5 o 2 o 2 methoxyethyl 5 methyl p thiouridylyl 3 o 5 o 2 o 2 methoxyethyl 5 methyl p thiouridylyl 3 o 5 o 2 o 2 methoxyethyl 5 methylcytidine
- ALL-P-AMBO-5-O-(((6-(5-((TRIS(3-(6-(2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYLOXY)HEXYLAMINO)-3-OXOPROPOXYMETHYL))METHYL)AMINO-5-OXOPENTANAMIDO)HEXOXY))PHOSPHO)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)GUANYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTIDYLYL-(3-O->5-O)-2-DEOXY-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-2-DEOXY-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)GUANYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTIDINE
- ionis apo a lrx
- Ionis-apo(a)-lrx
- isis 681257
- ISIS 681257
- isis 681257 free acid
- isis 681257 galnac3 conjugated aso targeting human lipoprotein a free acid
- ISIS-681257 (GALNAC3--CONJUGATED ASO TARGETING HUMAN LIPOPROTEIN (A)) FREE ACID
- ISIS-681257 FREE ACID
- pelacarsen
- Pelacarsen
- Pelacarsen (TQJ230) 80 mg s.c.
- pelacarsen \[INN\]
- PELACARSEN \[USAN\]
- Pelacarsen \[WHO-DD\]
- pelacarsen inn
- pelacarsen tqj230 80 mg s c
- pelacarsen usan
- pelacarsen who dd

## Identifiers

**CAS registry numbers**

- 1637637-70-7

**ChEMBL ID**

CHEMBL4298202

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298202)

**FDA ingredient codes (UNII)**

- LSO9H7UZ90

## Regulatory information

### Other regulatory facts

**First in class**

No

**Orphan designation**

No

## Pharmacology

### Drug classification

**Pharmacology name**

pelacarsen

**Type**

Nucleic acid

**Drug name stem**

\-rsen

### Mechanisms of action (2)

- **Mechanism**  
LPA mRNA antisense inhibitor

**Action type**  
ANTISENSE\_INHIBITOR

**Target**  
LPA mRNA

**Protein identifier**  
ENSG00000198670
- **Mechanism**  
LPA mRNA antisense inhibitor

**Action type**  
Inhibitor

**Target**  
LPA mRNA

**Protein identifier**  
ENSG00000198670

### Open Targets mechanisms

- **Mechanism**  
LPA mRNA antisense inhibitor

**Action type**  
ANTISENSE INHIBITOR

**Target**  
LPA mRNA

### Investigated indications (6)

- **Condition**  
Cardiovascular Diseases

**Highest recorded phase**  
2

**Condition identifier**  
EFO:0000319

**Subject identifier**  
D002318
- **Condition**  
Liver Diseases

**Highest recorded phase**  
1

**Condition identifier**  
EFO:0001421

**Subject identifier**  
D008107
- **Condition**  
Coronary Artery Disease

**Highest recorded phase**  
1

**Condition identifier**  
EFO:0001645

**Subject identifier**  
D003324
- **Condition**  
Kidney Diseases

**Highest recorded phase**  
1

**Condition identifier**  
EFO:0003086

**Subject identifier**  
D007674
- **Condition**  
Aortic Valve Stenosis

**Highest recorded phase**  
2

**Condition identifier**  
HP:0001650

**Subject identifier**  
D001024
- **Condition**  
Hyperlipoproteinemias

**Highest recorded phase**  
3

**Condition identifier**  
MONDO:0037748

**Subject identifier**  
D006951

### Open Targets indications (8)

- **Condition identifier**  
EFO\_0000319

**Highest recorded phase**  
3
- **Condition identifier**  
EFO\_0001421

**Highest recorded phase**  
1
- **Condition identifier**  
EFO\_0001645
- **Condition identifier**  
EFO\_0003086

**Highest recorded phase**  
1
- **Condition identifier**  
MONDO\_0004995

**Highest recorded phase**  
3
- **Condition identifier**  
MONDO\_0005010
- **Condition identifier**  
MONDO\_0005068
- **Condition identifier**  
MONDO\_0037748

**Highest recorded phase**  
3

### Trial references (8)

- NCT03070782  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03070782)
- NCT03392051  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03392051)
- NCT03426033  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03426033)
- NCT03506854  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03506854)
- NCT05026996  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05026996)
- NCT05305664  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05305664)
- NCT05900141  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05900141)
- NCT05646381  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05646381)

## Chemical properties

**Prodrug**

No

**Chirality**

Single enantiomer

**Inorganic**

No

**Natural product**

No

### Sequences

- **Component**  
Sequence

**Type**  
Nucleic Acid

**Sequence**  
(3'-5') R1-U=G-C-U-C-d(C=G=T=T=G=G=T=G=C=T=)U-G-U=U=C

## Related records

### Trials (16)

- [A Multicenter Trial Assessing the Impact of Lipoprotein(a) Lowering With Pelacarsen (TQJ230) on the Progression of Calcific Aortic Valve Stenosis](https://trialight.com/data/trial/a-multicenter-trial-assessing-the-impact-of-lipoprotein-a-51773799-ce02-463c-ae31-aa417295a17d.md)
- A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants
- [An Open Label Extension (OLE) Study (Following Completion of CTQJ230A12301) to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230)](https://trialight.com/data/trial/an-open-label-extension-ole-study-following-completion-of-377637e1-2c3d-44cc-a182-45f5a9dd3202.md)
- An Open Label Extension (OLE) Study to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230)
- Drug-drug Interaction Study to Evaluate the Effect of ISIS 681257 on Clopidogrel
- Drug-drug Interaction Study to Evaluate the Effect of ISIS 681257 on Warfarin
- Influence of Pelacarsen on Patients After Myocardial Infarction With High Lp(a) Values (PEMILA)
- Lp(a) Lowering Study of Pelacarsen (TQJ230) in Patients in the US With Elevated Lp(a) and Recent ACS (STEMI/NSTEMI) - Lp(a)FRONTIERS PEARL
- Lp(a) Lowering Study of Pelacarsen (TQJ230) With Background Inclisiran in Participants With Elevated Lp(a) and Established ASCVD
- Managed Access Programs for TQJ230, Pelacarsen
- Pelacarsen Roll-over Extension Program
- Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease
- Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)
- Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a)
- Study of ISIS 681257 in Patients With Renal Impairment Compared to Healthy Patients
- Study to Assess the Pharmacokinetics of Pelacarsen (TQJ230) in Participants With Mild Hepatic Impairment Compared to Matched Healthy Participants

### In development for (6)

- [Aortic valve stenosis](https://trialight.com/data/condition/aortic-valve-stenosis-d3f1a482-d908-5993-8a6c-7daad057bb89.md) Phase 2
- [Cardiovascular disorder](https://trialight.com/data/condition/cardiovascular-disorder-362e433e-88a4-575f-90af-50fe7239dec0.md) Phase 2
- [Coronary artery disorder](https://trialight.com/data/condition/coronary-artery-disorder-6f736849-d477-587a-b3fa-b0621d573407.md) Phase 1
- Hyperlipoproteinemia Phase 3
- [Kidney disorder](https://trialight.com/data/condition/kidney-disorder-e50371fe-f1a7-53fe-9cfb-9d6da9130e3c.md) Phase 1
- [Liver disorder](https://trialight.com/data/condition/liver-disorder-8388d30a-2581-5015-9ddf-adc5f6a331d7.md) Phase 1

### Other linked conditions (1)

- [Myocardial infarction](https://trialight.com/data/condition/myocardial-infarction-b4e97483-7435-5701-81dc-26d955e420ec.md)

### Targets (1)

- [LPA](https://trialight.com/data/target/lpa-7605327a-3607-533b-955f-c9eff32d6732.md) LPA mRNA antisense inhibitor

---

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