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YM-53601 is a small molecule, cell-penetrant, and potent inhibitor of squalene synthase, an enzyme in the mevalonate pathway responsible for the biosynthesis of cholesterol and other sterols[1][5]. By selectively inhibiting squalene synthase, YM-53601 reduces cholesterol and triglyceride levels in plasma in vivo, with animal studies showing greater efficacy than HMG-CoA reductase inhibitors (e.g., pravastatin) and fibrates (e.g., fenofibrate)[4][5]. YM-53601 has also been shown to inhibit hepatitis C virus (HCV) propagation at concentrations that do not affect cell viability, likely due to its disruption of cholesterol biosynthesis, which is essential for HCV replication[1][3]. The drug is currently for research use only and has not been approved for therapeutic use in humans[1].
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