Discovery of a Novel Silybin Derivative for the Treatment of Metabolic Dysfunction-Associated Steatohepatitis

J Med Chem. 2025 Jun 26;68(12):12786-12799. doi: 10.1021/acs.jmedchem.5c00672. Epub 2025 Jun 10.

Abstract

MASH has become the leading cause of liver disease worldwide, and the prevalence of MASH is steadily increasing. The development of new drugs for the treatment of MASH is urgent. Silybin has been used for decades in liver protection, but its insufficient antioxidant capacity and poor oral bioavailability have limited its clinical use. In this paper, we discovered a novel silybin derivative A2 as a potent agent for MASH treatment. In vitro, A2 showed excellent activity in inhibiting lipid accumulation, antioxidation, anti-inflammation, and antifibrosis. In the acute-liver-damage rat model experiment, A2 showed notable hepatoprotective efficacy. In the MASH mouse model experiment, A2, better than silybin, significantly ameliorated the pathological features of the MASH liver including steatosis, inflammation, and fibrosis. In addition, A2 displayed a good oral bioavailability and a good safety profile. Collectively, these findings demonstrated that A2 serves as a promising candidate for MASH treatment.

MeSH terms

  • Animals
  • Antioxidants / chemistry
  • Antioxidants / pharmacology
  • Antioxidants / therapeutic use
  • Biological Availability
  • Drug Discovery*
  • Fatty Liver* / drug therapy
  • Fatty Liver* / metabolism
  • Fatty Liver* / pathology
  • Humans
  • Liver / drug effects
  • Liver / pathology
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Rats
  • Rats, Sprague-Dawley
  • Silybin* / analogs & derivatives
  • Silybin* / chemistry
  • Silybin* / pharmacology
  • Silybin* / therapeutic use
  • Silymarin* / pharmacology
  • Silymarin* / therapeutic use

Substances

  • Silybin
  • Antioxidants
  • Silymarin