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Adjusted LOA
ML Risk
Also known as ISIS 703802, IONIS-ANGPTL3-LRx, Vupanorsen
AKCEA-ANGPTL3-LRx · 3 trials · 8 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.
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.
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.
| 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. |
| 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. |
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 ...
AKCEA-ANGPTL3-LRx is an investigational drug being studied for the treatment of Familial Chylomicronemia Syndrome and Familial Partial Lipodystrophy, both rare diseases. It is in Phase 2 clinical development and has not been approved by regulatory authorities.
AKCEA-ANGPTL3-LRx targets ANGPTL3, a protein involved in lipid metabolism. By inhibiting this target, the drug aims to reduce triglyceride and lipid levels in patients with rare lipid disorders. This mechanism is being evaluated in clinical trials for Familial Chylomicronemia Syndrome and Familial Partial Lipodystrophy.
AKCEA-ANGPTL3-LRx is developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company. Ionis is listed on the NASDAQ under the ticker symbol IONS. The drug is currently in Phase 2 clinical development for rare lipid disorders.
AKCEA-ANGPTL3-LRx is in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. Two Phase 2 studies have been completed, one in Familial Chylomicronemia Syndrome and one in Familial Partial Lipodystrophy.
AKCEA-ANGPTL3-LRx has been studied in two completed Phase 2 trials. NCT03360747 enrolled 3 participants with Familial Chylomicronemia Syndrome in Canada. NCT03514420 enrolled 4 participants with Familial Partial Lipodystrophy in the United States. Both trials were uncontrolled and open-label.
Yes, AKCEA-ANGPTL3-LRx is also known as ISIS 703802. Both names refer to the same investigational drug developed by Ionis Pharmaceuticals. It is being studied for Familial Chylomicronemia Syndrome and Familial Partial Lipodystrophy.