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인용수 1
·2025
Cholesterol-Lowering Effects of BMS-303141 Analogues via Inhibition of Adenosine Triphosphate-Citrate Lyase
In‐Gyu Je, Joon-Tae Park, Hyeong Jun Lee, A‐Rang Im, Jaecheol Lee, Ki‐Young Kim
IF 2.8Current Pharmaceutical Design
초록

BACKGROUND: Cholesterol is considered a major factor contributing to cardiovascular diseases. Statins, the most commonly prescribed cholesterol-lowering drugs, are known to have various limitations. Inhibition of Adenosine Triphosphate-Citrate Lyase (ACLY) has been proposed as an alternative therapeutic strategy for managing hypercholesterolemia by lowering cholesterol levels. This has led to the discovery of a cell-permeable small molecule ACLY inhibitor. METHODS: ACLY enzyme activity was assessed using an ACLY Assay Kit with the ADP-Glo Kinase Assay Kit. HepG2 cells were treated with test compounds to demonstrate cholesterol and fatty acid synthesis. Pharmacokinetic studies were performed on CD-1 mice following a single oral dose of the compounds. Hypercholesterolemia was induced in mice through a High-Fat and High Cholesterol Diet (HFHCD), and drugs were administered orally for six weeks. Serum and hepatic lipid profiles were subsequently analyzed. RESULTS: : 45 nM, 10-fold lower than BMS- 303141) and achieved near-complete suppression in cholesterol and fatty acid synthesis at the highest concentration. Pharmacokinetic studies revealed improved half-lives and systemic exposures for all analogues. In hypercholesterolemic mouse models, test compounds significantly reduced serum total cholesterol (32.0-57.3%) and low-density lipoprotein cholesterol (67.5-80.2%) levels compared to the vehicle group. Notably, ID0085 also increased high-density lipoprotein cholesterol levels. DISCUSSION: Among the synthesized analogues, ID0085 exhibited the most potent ACLY inhibition, superior pharmacokinetic properties, and significant improvements in both serum and hepatic cholesterol profiles compared to BMS-303141. CONCLUSION: Based on the results, ID0085 appears to be the most promising therapeutic candidate for the treatment of hypercholesterolemia.

키워드
ATP citrate lyaseCholesterolIC50PharmacologyChemistryFatty acid synthesisAdenosine triphosphateBiochemistryPharmacokineticsEnzyme
타입
article
IF / 인용수
2.8 / 1
게재 연도
2025

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