Discovery of novel FABP1 inhibitors for the treatment of metabolic dysfunction-associated steatohepatitis and hepatic fibrosis

发现新型FABP1抑制剂用于治疗代谢功能障碍相关性脂肪性肝炎和肝纤维化

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Abstract

The fatty acid-binding protein 1 (FABP1) has drawn increasing attention as a promising target for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). However, efforts to validate pharmacological effects of FABP1 are restricted by the lack of relevant inhibitors. Herein, we identified the lead compound 1 with potent inhibition on FABP1 through screening from our in-house library. Further comprehensive structure-activity relationship (SAR) study based on compound 1 resulted in the identification of the optimal compound 12 (IC(50) = 3.6 µM). Moreover, compound 12 exerted stronger efficacy on reducing hepatic lipid accumulation, inflammation and fibrosis than that of clinical candidate GFT505 in MASH models. In addition, compound 12 significantly inhibited fibrosis-related gene expression in TGF-β treated hepatic stellate cells and exerted stronger effects than Pirfenidone in CCl(4)-induced liver fibrosis mice model. These results indicated that compound 12 may serve as a novel FABP1 inhibitor for the treatment of MASH and liver fibrosis.

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