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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 antisense inhibitor being developed for Familial Chylomicronemia Syndrome, Severe Hypertriglyceridemia, and Hypertriglyceridemia. It is also being studied in healthy participants. The drug is in Phase 3 clinical development and has not been approved by the FDA.
Olezarsen targets APOC3, a protein involved in triglyceride metabolism. It is an antisense inhibitor, meaning it works by reducing the production of APOC3. This mechanism is being studied for its potential to lower triglyceride levels in patients with hypertriglyceridemia and related conditions.
Olezarsen is developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker IONS. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with severe hypertriglyceridemia and other lipid disorders.
Olezarsen is in Phase 3 clinical development. It has received FDA designations including Priority Review, Orphan Drug, Fast Track, and Breakthrough Therapy. However, it is still investigational and has not been approved by the FDA for any indication.
Olezarsen has been studied in four clinical trials, including NCT05355402 (Phase 2, completed), NCT05579860 (Phase 1, completed), NCT05610280 (Phase 3, completed), and NCT05681351 (Phase 3, active). These trials evaluate the drug in patients with hypertriglyceridemia, severe hypertriglyceridemia, and healthy participants.
Yes, Olezarsen was formerly known as AKCEA-APOCIII-LRx. It is also referred to as ISIS 678354 in some clinical trials. These names refer to the same investigational drug being developed by Ionis Pharmaceuticals for the treatment of hypertriglyceridemia and related conditions.