Design, Synthesis, and Evaluation of Dihydropyrimidine Derivatives as Selective PDE1 Inhibitors for the Treatment of Liver Fibrosis

J Med Chem. 2024 May 23;67(10):8309-8322. doi: 10.1021/acs.jmedchem.4c00461. Epub 2024 Apr 26.

Abstract

Liver fibrosis is a common pathological feature of most chronic liver diseases with no effective drugs available. Phosphodiesterase 1 (PDE1), a subfamily of the PDE super enzyme, might work as a potent target for liver fibrosis by regulating the concentration of cAMP and cGMP. However, there are few PDE1 selective inhibitors, and none has been investigated for liver fibrosis treatment yet. Herein, compound AG-205/1186117 with the dihydropyrimidine scaffold was selected as the hit by virtual screening. A hit-to-lead structural modification led to a series of dihydropyrimidine derivatives. Lead 13h exhibited the IC50 of 10 nM against PDE1, high selectivity over other PDEs, as well as good safety properties. Administration of 13h exerted significant anti-liver fibrotic effects in bile duct ligation-induced fibrosis rats, which also prevented TGF-β-induced myofibroblast differentiation in vitro, confirming that PDE1 could work as a potential target for liver fibrosis.

MeSH terms

  • Animals
  • Cyclic Nucleotide Phosphodiesterases, Type 1* / antagonists & inhibitors
  • Cyclic Nucleotide Phosphodiesterases, Type 1* / metabolism
  • Drug Design*
  • Humans
  • Liver Cirrhosis* / drug therapy
  • Liver Cirrhosis* / pathology
  • Male
  • Molecular Docking Simulation
  • Molecular Structure
  • Phosphodiesterase Inhibitors* / chemical synthesis
  • Phosphodiesterase Inhibitors* / chemistry
  • Phosphodiesterase Inhibitors* / pharmacology
  • Phosphodiesterase Inhibitors* / therapeutic use
  • Pyrimidines* / chemical synthesis
  • Pyrimidines* / chemistry
  • Pyrimidines* / pharmacology
  • Pyrimidines* / therapeutic use
  • Rats
  • Rats, Sprague-Dawley
  • Structure-Activity Relationship

Substances

  • Cyclic Nucleotide Phosphodiesterases, Type 1
  • Pyrimidines
  • Phosphodiesterase Inhibitors