Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Pelacarsen (TQJ230) 80mg, Pelacarsen (TQJ230)
Pelacarsen · 7 trials · 7 indications
Change in log-transformed Lipoprotein A (Lp(a)) concentration from baseline to Day 180
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
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
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 | EXPERIMENTAL | Pelacarsen (TQJ230) 80 mg s.c. QM |
| Placebo | PLACEBO_COMPARATOR | Corresponding placebo s.c. QM |
| Pelacarsen (TQJ230) | EXPERIMENTAL | open-label pelacarsen 80mg |
| 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 | DRUG | Pelacarsen 80 mg subcutaneously (s.c.) once a month (QM) |
| Placebo | DRUG | Placebo subcutaneously (s.c.) once a month (QM) |
| Pelacarsen (TQJ230) | DRUG | pelacarsen 80mg s.c. monthly |
| 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. |
| Pelacarsen (TQJ230) 80mg | DRUG | Pelacarsen (TQJ230) 80mg |
| Matching placebo | DRUG | Matching placebo |
Inclusion Criteria: * Lp(a) ≥ 150 nmol/L * Within 10 days of hospitalization for ACS event and meets all of the following criteria: * Clinical syndrome consistent with spontaneous cardiac ischemia * Diagnosis of ACS: ST-elevation myocardial infarction (STEMI) or non-ST-elevation myocardial infarcti...
Pelacarsen is an investigational drug being studied for cardiovascular conditions including Acute Coronary Syndrome, Hepatic Impairment, Hyperlipoproteinemia (a), Atherosclerotic Cardiovascular Disease, Cardiovascular Disease with elevated Lipoprotein(a), and Aortic Stenosis. It is currently in Phase 3 clinical development and has not been approved by the FDA.
Pelacarsen is an antisense oligonucleotide (ASO), a class of drugs that targets genetic material. It is being studied to reduce levels of lipoprotein(a), a risk factor for cardiovascular disease. The drug is designed to interfere with the production of this protein in the liver.
Pelacarsen is being developed by Novartis AG, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. Novartis is conducting clinical trials to evaluate the drug's safety and efficacy for cardiovascular indications.
Pelacarsen is currently in Phase 3 clinical development. It is an investigational drug, meaning it has not yet received regulatory approval. The company is conducting late-stage trials to assess its long-term safety and tolerability in patients with cardiovascular conditions.
Pelacarsen is being studied in several clinical trials, including NCT05026996, a completed Phase 1 study in hepatic impairment; NCT05900141, an active Phase 3 open-label extension; NCT06875973, a recruiting Phase 3 roll-over extension; and NCT07517263, a recruiting Phase 3 open-label extension with an enrollment of 5,700 participants.
Yes, Pelacarsen is also known as TQJ230. In clinical trial records, the drug is referred to as Pelacarsen (TQJ230), and some studies list it as Pelacarsen (TQJ230) 80mg. These names refer to the same investigational compound being developed by Novartis.