YM-53601 is a potent squalene synthase (SQS) inhibitor that inhibits adipogenic biosynthesis and lipid secretion in rodents.YM-53601 is a cholesterol-lowering agent that inhibits farnesyl diphosphate farnesyltransferase 1 (FDFT1).YM-53601 exhibits potential antiviral activity and enhances adriamycin-mediated HCC arrest and cell death in vivo. MJN228 is a lipid-based enzyme.

HepG2 cells (guinea pig liver microsomes):46 nM, HepG2 cells (human liver microsomes):79 nM, Squalene synthase (hamster liver):170 nM, HepG2 cells (rhesus monkey liver microsomes):45 nM, HepG2 cells (rat liver microsomes):90 nM, HepG2 cells (hamster liver microsomes):170 nM

YM-53601 inhibited squalene synthase activity in a variety of animal and human cell lines, with IC50s of 90, 170, 46, 45, and 79 nM in liver microsomes of rat, hamster, guinea pig, rhesus monkey, and human HepG2 cells, respectively. [1]
YM-53601 inhibited the conversion of [3H] farnesyl diphosphate to [3H] squalene by squalene synthase in hamster liver with an IC50 of 170 nM. [2]
In H35 cells, 1 μM M-53601 enhanced sensitivity to toxic carotenoids, lonidamine and adriamycin, and reduced mitochondrial cholesterol levels in H35 and HepG2 cells. [4]

YM-53601 inhibited cholesterol biosynthesis in rats at an ED50 of 32 mg/kg . [1]
In addition, in a hamster model, YM-53601 reduced plasma non-HDL cholesterol levels by approximately 70% when administered orally at 50 mg/kg/day for 5 days. [2]
YM-53601 also enhanced doxorubicin-mediated inhibition of hepatocellular carcinoma (HCC) cell proliferation and cell death in vivo. [4]