Discovery and Optimization of the First Highly Effective and Orally Available Galectin-3 Inhibitors for Treatment of Fibrotic Disease

J Med Chem. 2022 Oct 13;65(19):12626-12638. doi: 10.1021/acs.jmedchem.2c00660. Epub 2022 Sep 26.

Abstract

Galectin-3 is a carbohydrate-binding protein central to regulating mechanisms of diseases such as fibrosis, cancer, metabolic, inflammatory, and heart disease. We recently found a high affinity (nM) thiodigalactoside GB0139 which currently is in clinical development (PhIIb) as an inhaled treatment of idiopathic pulmonary fibrosis. To enable treatment of systemically galectin-3 driven disease, we here present the first series of selective galectin-3 inhibitors combining high affinity (nM) with oral bioavailability. This was achieved by optimizing galectin-3 specificity and physical chemical parameters for a series of disubstituted monogalactosides. Further characterization showed that this class of compounds reduced profibrotic gene expression in liver myofibroblasts and displayed antifibrotic activity in CCl4-induced liver fibrosis and bleomycin-induced lung fibrosis mouse models. On the basis of the overall pharmacokinetic, pharmacodynamic, and safety profile, GB1211 was selected as the clinical candidate and is currently in phase IIa clinical trials as a potential therapy for liver cirrhosis and cancer.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Bleomycin / pharmacology
  • Carbon Tetrachloride
  • Fibrosis
  • Galectin 3* / metabolism
  • Idiopathic Pulmonary Fibrosis* / chemically induced
  • Idiopathic Pulmonary Fibrosis* / drug therapy
  • Idiopathic Pulmonary Fibrosis* / pathology
  • Liver Cirrhosis / drug therapy
  • Liver Cirrhosis / pathology
  • Lung
  • Mice
  • Thiogalactosides
  • Triazoles

Substances

  • Galectin 3
  • Thiogalactosides
  • Triazoles
  • Bleomycin
  • GB-0139
  • Carbon Tetrachloride