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Olezarsen · 10 trials · 7 indications
| 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. |
| Olezarsen Dose Level 1 | EXPERIMENTAL | Participants will receive two doses of Dose Level 1 each, using one of the following two sequences: (i) AI on Day 1 of Treatment Period 1, followed by vial on Day 1 of Treatment Period 2; or (ii) vial on Day 1 of Treatment Period 1, followed by AI on Day 1 of Treatment Period 2. A washout period of 28-42 days will be maintained between the 2 treatment periods. |
| Olezarsen Dose Level 2 | EXPERIMENTAL | Participants will receive two doses of Dose Level 2 each, using one of the following two sequences: (i) AI on Day 1 of Treatment Period 1, followed by vial on Day 1 of Treatment Period 2; or (ii) vial on Day 1 of Treatment Period 1, followed by AI on Day 1 of Treatment Period 2 A washout period of at least 28 days will be maintained between the 2 treatment periods. |
| Name | Type | Description |
|---|---|---|
| Olezarsen | DRUG | Olezarsen will be administered by subcutaneous injection. |
| Placebo | DRUG | Olezarsen-matching placebo will be administered by 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 ...
Olezarsen is an investigational RNA therapy developed by Ionis Pharmaceuticals for lowering triglyceride levels in metabolic and cardiovascular conditions. It is being studied in familial chylomicronemia syndrome, hypertriglyceridemia, and severe hypertriglyceridemia. It has received FDA designations including Priority Review, Orphan Drug, Fast Track, and Breakthrough Therapy.
Olezarsen is being developed to treat familial chylomicronemia syndrome and hypertriglyceridemia, including severe hypertriglyceridemia, and is also being studied in patients with hypertriglyceridemia and atherosclerotic cardiovascular disease. It is an investigational therapy and has not been established as safe or effective for any indication.
Olezarsen targets apolipoprotein C-III, or APOC3. It is an antisense inhibitor designed to reduce production of APOC3, a protein involved in regulating plasma triglyceride levels. By lowering APOC3, the therapy aims to reduce triglycerides in patients with elevated levels.
Olezarsen is developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS. Ionis is the sponsor of the clinical development program for the drug across familial chylomicronemia syndrome and hypertriglyceridemia indications.
Olezarsen is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. The program includes Phase 3 trials in severe hypertriglyceridemia and hypertriglyceridemia with atherosclerotic cardiovascular disease, plus a Phase 3 pediatric study in familial chylomicronemia syndrome.
Olezarsen trials include NCT05681351, a Phase 3 study in severe hypertriglyceridemia; NCT05610280, a completed Phase 3 study in hypertriglyceridemia and atherosclerotic cardiovascular disease or severe hypertriglyceridemia; NCT07727538, a recruiting Phase 3 pediatric study in familial chylomicronemia syndrome; and NCT05579860, a completed Phase 1 study in healthy participants.
Yes. Olezarsen is also known as ISIS 678354, and both names refer to the same investigational antisense inhibitor targeting APOC3. Trial records for the Phase 3 severe hypertriglyceridemia and hypertriglyceridemia studies list the drug under the ISIS 678354 designation.