|
Synonyms: ARO-APOC3
Compound class:
Nucleic acid
Comment: Plozasiran (ARO-APOC3) is a small interfering RNA (siRNA) class ligand. The molecule contains modified nucleotides to improve stability, and it is congugated with N-acetylgalactosamine (GalNAc; at the R1 position), which binds to asialoglycoprotein receptors expressed on hepatocyte membranes to promote delivery of the drug to the liver [4]. Once bound, the siRNA is internalised in lysosomes and is subsequently released into the cytosol. Plozasiran blocks synthesis of apolipoprotein C-III (APOC3), as a mechanism to reduce dyslipidemia that is linked to cardiovascular disease risk [1-2,5].
We have been unable to locate a full chemical SMILES for the ligand, or its HELM notation. |
| No information available. |
Summary of Clinical Use  |
| Plozasiran (ARO-APOC3) is an advanced stage clinical candidate for hypertriglyceridemia and associated atherosclerotic cardiovascular disease, nonalcoholic steatohepatitis, and acute pancreatitis. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT05089084 | Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS) | Phase 3 Interventional | Arrowhead Pharmaceuticals | ||
| NCT05902598 | A Phase 3 Study of VSA001 in Chinese Adults With Familial Chylomicronemia Syndrome | Phase 3 Interventional | Visirna Therapeutics HK Limited | ||
| NCT06347133 | Phase 3 Study of Plozasiran in Adults With Hypertriglyceridemia | Phase 3 Interventional | Arrowhead Pharmaceuticals | ||
| NCT06347003 | Study of Plozasiran (ARO-APOC3) in Adults With Severe Hypertriglyceridemia | Phase 3 Interventional | Arrowhead Pharmaceuticals | ||
| NCT06347016 | Study of Plozasiran in Adults With Severe Hypertriglyceridemia | Phase 3 Interventional | Arrowhead Pharmaceuticals | ||
| NCT04720534 | Study to Evaluate ARO-APOC3 in Adults With Severe Hypertriglyceridemia | Phase 2 Interventional | Arrowhead Pharmaceuticals | 3 | |
