Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ALN-PCSSC · 2 trials · 4 indications
Due to the small number of subjects, and the fact that the data are not normally distributed, the data are presented as descriptive statistics with no inferential and limited summary statistics presented.
Percent Change in LDL-C (beta-quantification) from Baseline to Day 180 in MITT Population
| Arm | Type | Description |
|---|---|---|
| ALN-PCSSC | EXPERIMENTAL | 300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by \>70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy. |
| ALN-PCSSC 200 mg (bi-annual dosing) | EXPERIMENTAL | ALN-PCSSC 200 milligram (mg) SC administration once at Day 1 |
| ALN-PCSSC 300 mg (bi-annual dosing) | EXPERIMENTAL | ALN-PCSSC 300 mg SC administration once at Day 1 |
| ALN-PCSSC 500 mg (bi-annual dosing) | EXPERIMENTAL | ALN-PCSSC 500 mg SC administration once at Day 1 |
| Normal Saline (bi-annual dosing) | PLACEBO_COMPARATOR | Saline SC administration once at Day 1 |
| ALN-PCSSC 100 mg (quarterly dosing) | EXPERIMENTAL | ALN-PCSSC 100 mg SC administration twice at Day 1 and Day 90 |
| ALN-PCSSC 200 mg (quarterly dosing) | EXPERIMENTAL | ALN-PCSSC 200 mg SC administration twice at Day 1 and Day 90 |
| ALN-PCSSC 300 mg (quarterly dosing) | EXPERIMENTAL | ALN-PCSSC 300 mg SC administration twice at Day 1 and Day 90 |
| Normal Saline (quarterly dosing) | PLACEBO_COMPARATOR | Saline SC administration twice at Day 1 and Day 90 |
| Name | Type | Description |
|---|---|---|
| ALN-PCSSC | DRUG | ALN-PCSSC is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections. |
| Standard of Care | DRUG | Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies. |
| Normal Saline | DRUG | Saline (sterile, normal, 0.9%) solution given as SC injections |
Inclusion Criteria: * Males and females, ≥12 years of age with a diagnosis of homozygous familial hypercholesterolemia by genetic confirmation or a clinical diagnosis based on a history of an untreated low-density lipoprotein cholesterol (LDL-C) concentration \>500 mg/deciliter (dL) \[13 millimoles...
ALN-PCSSC is an investigational small molecule being developed for atherosclerotic cardiovascular disease and homozygous familial hypercholesterolemia. It is a Phase 2 drug candidate that has been studied in clinical trials for lowering low density lipoprotein cholesterol (LDL-C) in these patient populations.
ALN-PCSSC is being developed by Novartis AG (NVS). The company is conducting clinical trials to evaluate the drug's effect on LDL-C levels in patients with atherosclerotic cardiovascular disease and homozygous familial hypercholesterolemia.
ALN-PCSSC is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Two Phase 2 trials have been completed, one with 501 participants and another with 9 participants.
ALN-PCSSC has been studied in two completed Phase 2 trials. NCT02597127 evaluated its effect on LDL-C in 501 participants with atherosclerotic cardiovascular disease, familial hypercholesterolemia, or diabetes. NCT02963311 studied the drug in 9 participants with homozygous familial hypercholesterolemia.
ALN-PCSSC is a distinct investigational drug candidate developed by Novartis. No alternative names have been reported for this compound in the clinical trial data available.