Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Pelacarsen (TQJ230), TQJ230
Pelacarsen · 8 trials · 7 indications
Number of participants with Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs), TEAEs of special interest, including abnormal laboratory evaluations and vital signs. Number of participants discontinuing due to treatment emergent adverse events (TEAEs) or treatment emergent serious adverse events (TESAEs) will also be presented.
Evaluate long-term safety and tolerability of pelacarsen (TQJ230) in participants with elevated Lp(a) and established atherosclerotic cardiovascular disease who have completed the parent study
To evaluate the efficacy of Pelacarsen compared to placebo, in reducing Lp(a) levels at Month 6 in ASCVD participants who have elevated Lp(a), and who are on background inclisiran for elevated LDL-C
The primary aim of the study is to demonstrate the superiority of pelacarsen to placebo in lowering the Lp(a) level at 12 months of treatment in US Black/African American and US Hispanic participants with established ASCVD and a Lp(a) level of ≥ 125 nmo/L.
Evaluate long-term safety and tolerability of pelacarsen (TQJ230) in participants with elevated Lp(a) and established cardiovascular disease who have competed the parent study
Duration of drug exposure will be collected
Demonstrate the superiority of pelacarsen (TQJ230) compared to placebo in reducing the risk of expanded MACE (cardiovascular death, non-fatal MI, non-fatal stroke and urgent coronary re-vascularization requiring hospitalization) in the overall study population with established CVD and (Lp(a) ≥ 70 mg/dL)
Demonstrate the superiority of pelacarsen (TQJ230) compared to placebo in reducing the risk of expanded MACE (cardiovascular death, non-fatal MI, non-fatal stroke and urgent coronary re-vascularization requiring hospitalization) in the overall study population with established CVD and (Lp(a) ≥ 90 mg/dL)
To demonstrate the superiority of pelacarsen (TQJ230) vs. placebo in slowing the progression of calcific aortic valve stenosis by evaluating the change from baseline to month 36 in peak aortic jet velocity by echocardiography
To demonstrate the superiority of pelacarsen (TQJ230) vs. placebo in slowing the progression of calcific aortic valve stenosis by evaluating the change from baseline to month 36 in aortic valve calcium score by computed tomography
The maximum (peak) observed drug concentration in after single-dose administration (mass x volume-1). To assess the PK properties of plasma pelacarsen after a single s.c. injection in participants with mild HI (Child-Pugh classification) as compared to matched healthy participants with normal hepatic function.
The area under the concentration-time curve (AUC) from time zero to the last measurable concentration sampling time (mass x time x volume-1). To assess the PK properties of plasma pelacarsen after a single s.c. injection in participants with mild HI (Child-Pugh classification) as compared to matched healthy participants with normal hepatic function.
The AUC from time zero to infinity (mass x time x volume-1). To assess the PK properties of plasma pelacarsen after a single s.c. injection in participants with mild HI (Child-Pugh classification) as compared to matched healthy participants with normal hepatic function.
| Arm | Type | Description |
|---|---|---|
| Pelacarsen (TQJ230) | EXPERIMENTAL | open-label pelacarsen 80mg |
| Pelacarsen | ACTIVE_COMPARATOR | Participants randomized in Pelacarsen arm, will be administered pelacarsen (TQJ230) subcutaneous injection once monthly for 12 months. |
| Placebo | PLACEBO_COMPARATOR | Participants will be administered placebo subcutaneous injection once monthly for 6 months. After Month 6, all participants will receive pelacarsen 80 mg injection for the remaining 6 months during the open-label treatment period. |
| TQJ230 | EXPERIMENTAL | TQJ230 80mg QM s.c. |
| Pelacarsen (TQJ230) 80mg | EXPERIMENTAL | Pelacarsen (TQJ230) 80 mg prefilled syringe injected monthly, administered subcutaneously |
| Matching placebo | PLACEBO_COMPARATOR | Placebo to match pelacarsen (TQJ230) prefilled syringe injected monthly, administered subcutaneously |
| Mild hepatic impairment patients | EXPERIMENTAL | Participants with mild hepatic impairment |
| Healthy participants | EXPERIMENTAL | Matched healthy participants with normal hepatic function |
| Name | Type | Description |
|---|---|---|
| Pelacarsen (TQJ230) | DRUG | pelacarsen 80mg s.c. monthly |
| Pelacarsen | DRUG | Pelacarsen will be provided as solution for injection in prefilled syringe 80 mg for subcutaneous injection. |
| Placebo | DRUG | Placebo will be provided as solution for injection in prefilled syringe for subcutaneous injection. |
| Inclisiran | DRUG | All participants will be administered two loading doses of inclisiran as background treatment at Run-in 1 and Run-in 2, separated by 3 months, according to the approved label. After that inclisiran will be administered every 6 months, i.e., Month 5 and Month 11. |
| TQJ230 | DRUG | TQJ230 80mg QM s.c. |
| Pelacarsen (TQJ230) 80mg | DRUG | Pelacarsen (TQJ230) 80mg |
| Matching placebo | DRUG | Matching placebo |
Inclusion Criteria: * Participants who have provided informed consent prior to initiation of any study-specific activities/procedures. * Participants who have completed the parent study EOS visit while still on assigned investigational product. Exclusion Criteria: * Participants who for any reaso...
Pelacarsen is an investigational RNA therapy developed by Novartis AG for cardiovascular conditions linked to elevated lipoprotein(a). It is being studied in adults with atherosclerotic cardiovascular disease, cardiovascular disease with elevated lipoprotein(a), hyperlipoproteinemia (a), and aortic stenosis. Pelacarsen is also known as TQJ230.
Pelacarsen targets LPA, the gene that encodes apolipoprotein(a), a component of lipoprotein(a). It is an antisense inhibitor, a class of RNA therapy designed to bind LPA RNA and reduce production of the lipoprotein(a) protein. Lowering lipoprotein(a) is the intended pharmacologic effect under study in its cardiovascular trials.
Novartis AG, which trades under the ticker NVS, develops pelacarsen. Novartis is running the clinical program for the drug, including the open label extension studies and the Lp(a) lowering study combining pelacarsen with background inclisiran in participants with elevated Lp(a) and established atherosclerotic cardiovascular disease.
Pelacarsen is in Phase 3 clinical development. Its registered trials are listed as Phase 3 studies, and the program includes open label extension studies evaluating long term safety and tolerability following earlier trial completion. Pelacarsen is investigational and is not described as an approved therapy.
Pelacarsen trials include NCT07517263, an open label extension study in cardiovascular disease and lipoprotein(a) with 5,700 participants across many countries, and NCT06875973, a roll-over extension program in atherosclerotic cardiovascular disease. Other studies are NCT06813911, testing pelacarsen with background inclisiran in ASCVD, and NCT05900141, an open label extension in hyperlipoproteinemia (a).
Yes, pelacarsen and TQJ230 refer to the same investigational drug from Novartis. The names appear together in trial titles, including studies of pelacarsen (TQJ230) in cardiovascular disease and lipoprotein(a). Some listings also refer to it as pelacarsen (TQJ230) 80mg, reflecting the dose form used in studies.