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Also known as Olezarsen, AKCEA-APOCIII-LRx
ISIS 678354 · 10 trials · 6 indications
An analysis of covariance (ANCOVA) model was performed on the log ratio of TG value at the Primary Analysis Time Point to TG value at Baseline. The estimate of the log ratio was converted back to the original scale and percent change was calculated using formula: (ratio of TG value at the Primary Analysis Time Point to TG value at Baseline - 1) × 100.
An adverse event (AE) was defined as any unfavorable and unintended sign (including a clinically-significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered to be related to the investigational drug product. A TEAE was defined as any AE starting on or after the first dose of the study drug.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Participants aged 12 to \<18 years will receive multiple doses of olezarsen, at a dose level that depends on body weight, once every month by subcutaneous injection for up to 1 year. All participants may continue to the optional 1-year long-term extension period with the last dose received being after 2 years of treatment. |
| Cohort 2 | EXPERIMENTAL | Participants aged 2 to \<12 years will receive multiple doses of olezarsen, at a dose level based on information obtained from Cohort 1, once every month by subcutaneous injection for up to 1 year. All participants may continue to the optional 1-year long-term extension period with the last dose received being after 2 years of treatment. |
| Olezarsen | EXPERIMENTAL | Participants who completed either ISIS 678354-CS5 (NCT05079919) or ISIS 678354-CS6 (NCT05552326) study would be enrolled to receive olezarsen, subcutaneous (SC) injection, once every 4 weeks from Week 1 through Week 153. |
| Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive olezarsen-matching placebo, once every 4 weeks by SC injection up to Week 49. |
| Olezarsen 50 mg | EXPERIMENTAL | Participants received olezarsen, 50 milligrams (mg), once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period. |
| Olezarsen 80 mg | EXPERIMENTAL | Participants received olezarsen 80 mg, once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period. |
| Pooled Placebo | PLACEBO_COMPARATOR | Participants in each cohort (A, B, C and D) were randomized to receive placebo at a dose-matched volume of study drug (ISIS 678354). |
| Cohort A: ISIS 678354: 10 mg Q4W | EXPERIMENTAL | Cohort A participants received 10 milligrams (mg) ISIS 678354, subcutaneous (SC) injection, once every 4 weeks (Q4W), for up to 49 weeks and a maximum of 13 doses. |
| Cohort C: ISIS 678354: 15 mg Q2W | EXPERIMENTAL | Cohort C participants received 15 mg ISIS 678354, SC injection, once every 2 weeks (Q2W) for up to 51 weeks and a maximum of 26 doses. |
| Cohort D: ISIS 678354: 10 mg QW | EXPERIMENTAL | Cohort D participants received 10 mg ISIS 678354, SC injection, once weekly (QW) for up to 52 weeks and a maximum of 52 doses. |
| Cohort B: ISIS 678354: 50 mg Q4W | PLACEBO_COMPARATOR | Cohort B participants received 50 mg ISIS 678354, SC injection, once Q4W for up to 49 weeks and a maximum of 13 doses. |
| Name | Type | Description |
|---|---|---|
| Olezarsen | DRUG | Olezarsen will be administered by subcutaneous injection. |
| Placebo | DRUG | Olezarsen-matching placebo will be administered by SC injection. |
| ISIS 678354 | DRUG | ISIS 678354 solution for SC injection. |
Key Inclusion Criteria: 1. Parental or legally authorized representative consent must be obtained, and the participants must provide age-appropriate or cognition-appropriate assent, as determined by the Investigator. The parent or legal guardian must be able to understand and comply with the study ...
ISIS 678354 is an investigational therapy being developed for hypertriglyceridemia, a condition marked by elevated triglyceride levels in the blood. It has also been studied in patients with established cardiovascular disease. The drug has completed a Phase 2 clinical trial and is not yet approved for any indication.
ISIS 678354 is being developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker symbol IONS. The company is the sponsor of the clinical program evaluating the drug in hypertriglyceridemia and cardiovascular disease.
ISIS 678354 has completed a Phase 2 clinical trial. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. No active trials are currently listed for this asset.
ISIS 678354 was evaluated in a single Phase 2 trial, NCT03385239, titled Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Participants With Hypertriglyceridemia and Established Cardiovascular Disease (CVD). The trial enrolled 114 participants across the United States and Canada and has been completed.
Yes, ISIS 678354 is also known as AKCEA-APOCIII-LRx. The two names refer to the same investigational drug candidate. The alternative name appears in the title of its Phase 2 clinical trial, NCT03385239, which studied the drug in patients with hypertriglyceridemia and established cardiovascular disease.