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 Injection, ARO-APOC3, SAR449124, VSA001
Plozasiran · 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...
Plozasiran is an investigational RNA therapy being developed for rare lipid disorders. It is designed to treat familial chylomicronemia, familial chylomicronemia syndrome, hypertriglyceridemia, and severe hypertriglyceridemia. It is a Phase 3 candidate and is not yet approved. Plozasiran is also known as Plozasiran Injection.
Plozasiran is being studied for familial chylomicronemia, familial chylomicronemia syndrome, hypertriglyceridemia, and severe hypertriglyceridemia. These are rare lipid conditions marked by elevated triglyceride levels. The drug remains investigational and is being evaluated in Phase 3 clinical trials rather than being available as an approved treatment.
Plozasiran targets APOC3, or apolipoprotein C-III. It belongs to the -siran small interfering RNA class, meaning it is designed to reduce APOC3 production through RNA interference. Lowering APOC3 is intended to reduce triglyceride levels in patients with hypertriglyceridemia and related rare lipid disorders.
Plozasiran is developed by Arrowhead Pharmaceuticals, Inc., which trades under the ticker ARWR. The company is running the Phase 3 clinical program evaluating the drug in hypertriglyceridemia and severe hypertriglyceridemia, including a long-term safety and efficacy study.
Plozasiran is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. The Phase 3 program includes completed, active, and recruiting studies in hypertriglyceridemia and severe hypertriglyceridemia, with a combined enrollment of more than 2,000 participants across the trials.
Plozasiran has been studied in Phase 3 trials including NCT06347133 and NCT06347016, both completed, and NCT06822790, which is active but not recruiting. A fourth Phase 3 study, NCT06880770, is currently recruiting adults with severe hypertriglyceridemia at risk of acute pancreatitis. The trials are randomized, double-blind, and placebo-controlled.