Identification and development of biphenyl substituted iminosugars as improved dual glucosylceramide synthase/neutral glucosylceramidase inhibitors

J Med Chem. 2014 Nov 13;57(21):9096-104. doi: 10.1021/jm501181z. Epub 2014 Oct 17.

Abstract

This work details the evaluation of a number of N-alkylated deoxynojirimycin derivatives on their merits as dual glucosylceramide synthase/neutral glucosylceramidase inhibitors. Building on our previous work, we synthesized a series of D-gluco and L-ido-configured iminosugars N-modified with a variety of hydrophobic functional groups. We found that iminosugars featuring N-pentyloxymethylaryl substituents are considerably more potent inhibitors of glucosylceramide synthase than their aliphatic counterparts. In a next optimization round, we explored a series of biphenyl-substituted iminosugars of both configurations (D-gluco and L-ido) with the aim to introduce structural features known to confer metabolic stability to drug-like molecules. From these series, two sets of molecules emerge as lead series for further profiling. Biphenyl-substituted L-ido-configured deoxynojirimycin derivatives are selective for glucosylceramidase and the nonlysosomal glucosylceramidase, and we consider these as leads for the treatment of neuropathological lysosomal storage disorders. Their D-gluco-counterparts are also potent inhibitors of intestinal glycosidases, and because of this characteristic, we regard these as the prime candidates for type 2 diabetes therapeutics.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • 1-Deoxynojirimycin / analogs & derivatives
  • Biphenyl Compounds / chemical synthesis*
  • Biphenyl Compounds / pharmacology
  • Enzyme Inhibitors / chemical synthesis*
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology
  • Glucosylceramidase / antagonists & inhibitors*
  • Glucosyltransferases / antagonists & inhibitors*
  • Humans
  • Imino Sugars / chemical synthesis*
  • Imino Sugars / pharmacology
  • beta-Glucosidase / antagonists & inhibitors

Substances

  • Biphenyl Compounds
  • Enzyme Inhibitors
  • Imino Sugars
  • 1-Deoxynojirimycin
  • Glucosyltransferases
  • ceramide glucosyltransferase
  • beta-Glucosidase
  • GBA2 protein, human
  • Glucosylceramidase