Drug intelligence / Profile preview

ym-53601

Development stage
Preclinical
Lead developer
Astellas Pharma
Modality
Small Molecules
Administration
Oral
01

Overview

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].

Other names
(E)-2-[2-fluoro-2-(quinuclidin-3-ylidene)ethoxy]-9H-carbazole monohydrochlorideYM-53601 hydrochlorideYM53601 hydrochlorideYM 53601 hydrochloride2-[(2E)-2-(1-azabicyclo[2.2.2]oct-3-ylidene)-2-fluoroethoxy]-9H-carbazole, monohydrochloride2-[(2E)-2-(1-azabicyclo[2.2.2]octan-3-ylidene)-2-fluoroethoxy]-9H-carbazole:hydrochloride9H-Carbazole, 2-[(2E)-2-(1-azabicyclo[2.2.2]oct-3-ylidene)-2-fluoroethoxy]-, hydrochloride (1:1)YM-53601 ExclusiveYM53601 ExclusiveYM 53601 ExclusiveYM-53601 free baseYM53601 free baseYM 53601 free base
02

Targets

FDFT1 (Squalene synthase)

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