Mipomersen (sodium), a second-generation, 20-base phosphorothioate antisense oligonucleotide (ASO), targets human apoB-100.

Mipomersen predominantly distributes to the liver and decreases the production of apoB-100, the primary structural protein of the atherogenic lipoproteins including low density lipoprotein (LDL), thereby reducing plasma LDL-cholesterol and apoB-100 concentrations[2]. Apolipoprotein (apo) B, a large amphipathic protein, plays a fundamental role in human lipoprotein metabolism. ApoB-100 is also a ligand for LDL receptor-mediated endocytosis of LDL by cells[2].