Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as ISIS 703802
IONIS ANGPTL3-LRx · 2 trials · 5 indications
An ANCOVA model was performed on the log ratio of Primary Analysis Time Point to Baseline. The estimate of the log ratio was converted back to the original scale and percent change was calculated using formula: = (ratio of Primary Analysis Time Point to Baseline - 1) × 100.
The safety and tolerability of IONIS ANGPTL3-LRx will be assessed by determining the incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters by dose. Safety results in subjects dosed with IONIS ANGPTL3-LRx will be compared with those from subjects dosed with placebo.
The plasma pharmacokinetics (concentration-time results) of IONIS ANGPTL3-LRx (unconjugated and conjugated ASO) will be assessed following single and multiple-dose SC administration. The amount of IONIS ANGPTL3-LRx excreted in urine at selected 24-hour intervals will also be determined.
Changes in serum angiopoietin-like 3 (ANGPTL3) levels compared to baseline.
| Arm | Type | Description |
|---|---|---|
| Pooled Placebo | PLACEBO_COMPARATOR | Participants from each cohort received placebo at a dose-matched volume of study drug, subcutaneously (SC). |
| Cohort B: ISIS 703802, 40 mg Q4W | EXPERIMENTAL | Participants received ISIS 703802, 40 milligrams (mg) SC once every 4 weeks for 6 doses. |
| Cohort C: ISIS 703802, 80 mg Q4W | EXPERIMENTAL | Participants received ISIS 703802, 80 mg SC once every 4 weeks for 6 doses. |
| Cohort A: ISIS 703802, 20 mg QW | EXPERIMENTAL | Participants received ISIS 703802, 20 mg once every week for 26 doses. |
| Cohorts A, D: Placebo | PLACEBO_COMPARATOR | Participants received a single-dose of IONIS ANGPTL3-LRx-matching placebo subcutaneously on Day 1. |
| Cohorts A, D: IONIS ANGPTL3-LRx 20 mg | EXPERIMENTAL | Participants received a single-dose of IONIS ANGPTL3-LRx 20 milligrams (mg) subcutaneously on Day 1. |
| Cohorts A, D: IONIS ANGPTL3-LRx 120 mg | EXPERIMENTAL | Participants received a single-dose of IONIS ANGPTL3-LRx 120 mg subcutaneously on Day 1. |
| Cohorts B, C: Placebo | PLACEBO_COMPARATOR | Participants received a single-dose of IONIS ANGPTL3-LRx-matching placebo subcutaneously on Day 1. |
| Cohorts B, C: IONIS ANGPTL3-LRx 40 mg | EXPERIMENTAL | Participants received a single-dose of IONIS ANGPTL3-LRx 40 mg subcutaneously on Day 1. |
| Cohorts B, C: IONIS ANGPTL3-LRx 80 mg | EXPERIMENTAL | Participants received a single-dose of IONIS ANGPTL3-LRx 80 mg subcutaneously on Day 1. |
| Cohorts AA-DD: Placebo | PLACEBO_COMPARATOR | Participants received IONIS ANGPTL3-LRx-matching placebo subcutaneously once per week for 6 weeks. |
| Cohorts AA-DD: IONIS ANGPTL3-LRx 10 mg | EXPERIMENTAL | Participants received IONIS ANGPTL3-LRx 10 mg subcutaneously once per week for 6 weeks. |
| Cohorts AA-DD: IONIS ANGPTL3-LRx 20 mg | EXPERIMENTAL | Participants received IONIS ANGPTL3-LRx 20 mg subcutaneously once per week for 6 weeks. |
| Cohorts AA-DD: IONIS ANGPTL3-LRx 40 mg | EXPERIMENTAL | Participants received IONIS ANGPTL3-LRx 40 mg subcutaneously once per week for 6 weeks. |
| Cohorts AA-DD: IONIS ANGPTL3-LRx 60 mg | EXPERIMENTAL | Participants received IONIS ANGPTL3-LRx 60 mg subcutaneously once per week for 6 weeks. |
| Name | Type | Description |
|---|---|---|
| Placebo | DRUG | Placebo (Matched with ISIS 703802) |
| ISIS 703802 40 mg | DRUG | ISIS 703802 40 mg, administered via SC injection, once every 4 weeks for 6 doses. |
| ISIS 703802 80 mg | DRUG | ISIS 703802 80 mg, administered via SC injection, once every 4 weeks for 6 doses. |
| ISIS 703802 20 mg | DRUG | ISIS 703802 20 mg, administered via SC injection, once every week for 26 doses. |
| IONIS ANGPTL3-LRx | DRUG | - |
Key Inclusion Criteria: * Plasma triglycerides (TG) at Screening greater than (\>)150 milligrams per deciliter (mg/dL) and at qualification of \>150 mg/dL. * Documented history of hepatic steatosis with baseline magnetic resonance imaging (MRI) indicating hepatic fat fraction (HFF) greater than (\>...
IONIS ANGPTL3-LRx is an investigational drug being developed for hypertriglyceridemia, a condition characterized by elevated triglyceride levels in the blood. It is a small molecule being studied in a Phase 1 clinical trial that also includes participants with familial hypercholesterolemia.
IONIS ANGPTL3-LRx is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol IONS. The company is conducting clinical trials to evaluate the drug's safety and efficacy.
IONIS ANGPTL3-LRx is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The Phase 1 trial has been completed, and the drug remains under investigation for its potential use in treating hypertriglyceridemia.
IONIS ANGPTL3-LRx has been studied in one completed Phase 1 clinical trial with the identifier NCT02709850. This trial evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug in healthy volunteers with elevated triglycerides and participants with familial hypercholesterolemia.
IONIS ANGPTL3-LRx is not FDA approved. It is an investigational drug currently in Phase 1 clinical development. The completed Phase 1 trial assessed its safety and tolerability, but further clinical studies are needed before it can be considered for regulatory approval.