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plozasiran   Click here for help

GtoPdb Ligand ID: 13642

Synonyms: ARO-APOC3 | Redemplo®
Approved drug
plozasiran is an approved drug
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 [1-2,6].
We have been unable to locate a full chemical SMILES for the ligand, or its HELM notation.
No information available.
Summary of Clinical Use Click here for help
Plozasiran (ARO-APOC3) was progressed as a clinical candidate for hypertriglyceridemia and associated atherosclerotic cardiovascular disease, nonalcoholic steatohepatitis, and acute pancreatitis. First approval was granted by the US FDA in November 2025, to reduce triglycerides in adults with familial chylomicronemia syndrome [5].
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