Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication REDEMPLO is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). REDEMPLO is an apolipoprotein C-III ( apoC-III )-directed small interfering ribonucleic acid (siRNA) indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). ( 1 ) FDA label
Also known as Plozasiran, Plozasiran Injection, Redemplo, SAR449124, VSA001
ARO-APOC3 · 12 trials · 7 indications
Blood samples for lipid parameters were collected at the specified time points. Fasting serum TG at Month 10 was defined as geometric mean of 2 measurements taken during Month 10, or the other non-missing measurement if any one of the two measurements was missing during Month 10. Analysis was performed on the basis of the handling strategy for intercurrent events, missing fasting serum was imputed mainly based on the Pattern Mixture Models (PMM). Descriptive statistics were calculated based on non-imputed data.
Least square (LS) mean and standard error (SE) were calculated using mixed model repeated measures (MMRM) which included treatment arm, study visit, and baseline value as model terms and treatment by visit and treatment by baseline as interaction terms.
Incidence, frequency, and severity of adverse events (AEs) and serious adverse events (SAEs), and the relationship with VSA-01001. Clinically significant abnormalities in laboratory tests, vital signs, physical examination, 12-lead electrocardiograms (ECG).
| Arm | Type | Description |
|---|---|---|
| Plozasiran Injection | EXPERIMENTAL | Plozasiran by SC injection every 3 months (Q3M) through completion of the randomized period Plozasiran by SC injection Q3M through completion of the OLE period |
| Placebo | EXPERIMENTAL | calculated volume to match active treatment by SC injection (randomized period) |
| Plozasiran 25 mg | EXPERIMENTAL | Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months |
| Plozasiran 50 mg | EXPERIMENTAL | Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months |
| ARO-APOC3 (Plozasiran) 25 mg | EXPERIMENTAL | Randomized Period: plozasiran 25 mg Q3M for a total of 4 doses. Open-label Period (Parts A and B): plozasiran 25 mg Q3M for a total of 8 doses. |
| Placebo for ARO-APOC3 (Plozasiran) 25 mg | PLACEBO_COMPARATOR | Randomized Period: volume-matched placebo every 3 months (Q3M) for a total of 4 doses. Open-label Period (Parts A and B): plozasiran 25 mg Q3M for a total of 8 doses. |
| ARO-APOC3 (Plozasiran) 50 mg | EXPERIMENTAL | Randomized Period: plozasiran 25 mg Q3M for a total of 4 doses. Open-label Period: plozasiran 50 mg (Part A), then 25 mg (Part B) Q3M for a total of 8 doses. |
| Placebo for ARO-APOC3 (Plozasiran) 50 mg | PLACEBO_COMPARATOR | Randomized Period: volume-matched placebo every 3 months (Q3M) for a total of 4 doses. Open-label Period: plozasiran 50 mg (Part A), then 25 mg (Part B) Q3M for a total of 8 doses. |
| ARO-APOC3 | EXPERIMENTAL | 1 dose of ARO-APOC3 by subcutaneous (sc) injection every 3 months |
| ARO-APOC3 10 mg Q12W | EXPERIMENTAL | ARO-APOC3 10 milligrams (mg) subcutaneous (SC) injection on Day 1 and Week 12 (Q12W) for a total of 2 injections |
| ARO-APOC3 25 mg Q12W | EXPERIMENTAL | ARO-APOC3 25 mg SC injection on Day 1 and Week 12 (Q12W) for a total of 2 injections |
| ARO-APOC3 50 mg Q12W | EXPERIMENTAL | ARO-APOC3 50 mg SC injection on Day 1 and Week 12 (Q12W) for a total of 2 injections |
| ARO-APOC3 50 mg Q24W | EXPERIMENTAL | ARO-APOC3 50 mg SC injection on Day 1 and Week 24 (Q24W) for a total of 2 injections |
| Placebo, Day 1 and Week 12 | EXPERIMENTAL | Participants received a total of 2 placebo SC injections on Day 1 and Week 12 for a total of 2 injections. |
| ARO-APOC3 10 mg | EXPERIMENTAL | Participants received a total of 2 ARO-APOC3 10 mg SC injections on Day 1 and Week 12 for a total of 2 injections. |
| ARO-APOC3 25 mg | EXPERIMENTAL | Participants received a total of 2 ARO-APOC3 25 mg SC injections on Day 1 and Week 12 for a total of 2 injections. |
| ARO-APOC3 50 mg | EXPERIMENTAL | Participants received a total of 2 ARO-APOC3 50 mg SC injections on Day 1 and Week 12 for a total of 2 injections. |
| VSA001 injection | ACTIVE_COMPARATOR | A single dose of active drug (VSA001, low or high dose) will be administered by subcutaneous injection on Day 1. |
| Name | Type | Description |
|---|---|---|
| Plozasiran | DRUG | ARO-APOC3 injection |
| Placebo | DRUG | sterile normal saline (0.9% NaCl) |
| Plozasiran Injection | DRUG | ARO-APOC3 injection |
| Placebon | DRUG | Subcutaneous injection |
| ARO-APOC3 | DRUG | ARO-APOC3 Injection |
| VSA001 injection | DRUG | The active drug is VSA001 injection. The active pharmaceutical ingredient (API) contained in VSA001 is a synthetic, double-stranded, hepatocyte targeted NAG-conjugated RNAi. |
| sterile normal saline (0.9% NaCl) | DRUG | calculated volume to match active treatment |
Inclusion Criteria: * Males, or nonpregnant (who do not plan to become pregnant) nonlactating females * Established diagnosis of SHTG and prior documented evidence of fasting TG levels of ≥ 880 mg/dL (≥ 10 mmol/L) * Documented evidence of at least 1 prior AP event not attributed to other etiologies...
ARO-APOC3 is an investigational RNA therapy developed by Arrowhead Pharmaceuticals for dyslipidemias, including hypertriglyceridemia, mixed dyslipidemia, and severe hypertriglyceridemia. It is designed to reduce apolipoprotein C3 production. It has completed Phase 2 clinical testing and is not approved for any indication.
ARO-APOC3 is being studied for the treatment of dyslipidemias, including hypertriglyceridemia, mixed dyslipidemia, and severe hypertriglyceridemia. It is an investigational therapy and has not been approved for commercial use. All clinical trials of ARO-APOC3 to date have been completed.
ARO-APOC3 is developed by Arrowhead Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ARWR. Arrowhead is a biopharmaceutical company focused on RNA-based therapies. ARO-APOC3 is one of its clinical-stage cardiovascular candidates.
ARO-APOC3 has completed Phase 2 clinical development. It is an investigational RNA therapy and is not FDA approved. No active trials of ARO-APOC3 are currently listed, and the completed studies enrolled a total of 418 participants across the Phase 1 and Phase 2 programs.
ARO-APOC3 has been evaluated in completed trials including NCT05413135 in adults with dyslipidemia, NCT04998201 in mixed dyslipidemia, NCT04720534 in severe hypertriglyceridemia, and NCT03783377 in healthy volunteers, hypertriglyceridemic patients, and patients with familial chylomicronemia syndrome. These studies were conducted across the United States, Australia, Canada, Europe, and New Zealand.
Yes, plozasiran is an alternative name for ARO-APOC3. The Phase 2 trial NCT04998201 is titled as a study of plozasiran (ARO-APOC3) in adults with mixed dyslipidemia, confirming that both names refer to the same investigational RNA therapy developed by Arrowhead Pharmaceuticals.