Approval Probability
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Also known as IONIS ANGPTL3-LRx, AKCEA-ANGPTL3-LRx, Vupanorsen
ISIS 703802 · 4 trials · 9 indications
The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.
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.
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
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 |
|---|---|---|
| AKCEA-ANGPTL3-LRx 20 mg | EXPERIMENTAL | Participants received AKCEA-ANGPTL3-LRx 20 milligrams (mg) administered every week for 26 weeks by subcutaneous (SC) injection. |
| 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 |
|---|---|---|
| AKCEA-ANGPTL3-LRx | DRUG | AKCEA-ANGPTL3-LRx solution for SC injection. |
| 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: * Must give written informed consent to participate in the study. * Clinical diagnosis of familial partial lipodystrophy plus diagnosis of type 2 diabetes mellitus and hypertriglyceridemia. * Diagnosis of diabetes mellitus, made at least 6 months prior to the Screening with ...
ISIS 703802 is an investigational small molecule developed by Ionis Pharmaceuticals, Inc. for metabolic disease. It has been studied in a Phase 2 clinical trial in participants with nonalcoholic fatty liver disease (NAFLD), type 2 diabetes mellitus, and hypertriglyceridemia. It is also referred to as ISIS 703802 40 mg.
ISIS 703802 has been studied for nonalcoholic fatty liver disease (NAFLD), a metabolic condition. The Phase 2 trial also enrolled participants with type 2 diabetes mellitus and hypertriglyceridemia, so the research population combined these metabolic and liver conditions rather than NAFLD alone.
ISIS 703802 is developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS. The company sponsored the Phase 2 study of the drug in participants with NAFLD, type 2 diabetes mellitus, and hypertriglyceridemia.
ISIS 703802 is in Phase 2 development. Its single registered trial, NCT03371355, was a Phase 2 study that has been completed. The drug is investigational and has not been approved for any indication.
ISIS 703802 has been evaluated in one clinical trial, NCT03371355, titled Study of ISIS 703802 in Participants With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease. The Phase 2 trial enrolled 105 participants across the United States and Canada and is completed.
The Phase 2 trial NCT03371355 was randomized, double blind, and placebo controlled, enrolling 105 participants aged 18 years and older of all sexes. It was conducted in the United States and Canada in participants with NAFLD, type 2 diabetes mellitus, and hypertriglyceridemia, and it has been completed.