Discovery of novel PDE4 inhibitors targeting the M-pocket from natural mangostanin with improved safety for the treatment of Inflammatory Bowel Diseases

Eur J Med Chem. 2022 Nov 15:242:114631. doi: 10.1016/j.ejmech.2022.114631. Epub 2022 Aug 9.

Abstract

Inflammatory Bowel Diseases (IBDs) are chronic disorders with iterative intestinal mucosal inflammation which remain unmet medical needs. PDE4 inhibitors were reported to be novel anti-IBD agents, but their clinical use was hampered by side effects such as emesis and nausea. Herein, structure-based discovery of natural mangostanin (1) targeting the M-pocket resulted in the novel and potent PDE4 inhibitor 22d (IC50 = 3.5 nM) and favorable physico-chemical properties. X-Ray study revealed that 22d interacted tightly with the M-pocket and maintained the key interactions between PDE4 and roflumilast. Worthy to note that compounds 22d and our previously reported 4e and 18a, originating from mangostanin, all caused no emesis on beagle dogs at the oral dose of 10 mg/kg, confirming the safety superiority of scaffold in mangostanin derivatives over that in positive roflumilast. Finally, administration of 22d (5.0 mg/kg, twice-daily) exhibited comparable anti-IBD effects to the positive control dipyridamole (25.0 mg/kg, twice-daily) in the dextran sulfate sodium (DSS)-induced IBD mice model, indicating its potential as a novel anti-IBD agent.

Keywords: Inflammatory Bowel Diseases; Mangostanin; PDE4 inhibitor; Safety.

MeSH terms

  • Aminopyridines
  • Animals
  • Benzamides
  • Cyclic Nucleotide Phosphodiesterases, Type 4 / chemistry
  • Cyclopropanes
  • Dextran Sulfate
  • Dipyridamole / therapeutic use
  • Dogs
  • Inflammatory Bowel Diseases* / chemically induced
  • Inflammatory Bowel Diseases* / drug therapy
  • Mice
  • Phosphodiesterase 4 Inhibitors* / pharmacology
  • Phosphodiesterase 4 Inhibitors* / therapeutic use

Substances

  • Aminopyridines
  • Benzamides
  • Cyclopropanes
  • Phosphodiesterase 4 Inhibitors
  • Roflumilast
  • Dipyridamole
  • Dextran Sulfate
  • Cyclic Nucleotide Phosphodiesterases, Type 4