Discovery of GSK251: A Highly Potent, Highly Selective, Orally Bioavailable Inhibitor of PI3Kδ with a Novel Binding Mode

J Med Chem. 2021 Sep 23;64(18):13780-13792. doi: 10.1021/acs.jmedchem.1c01102. Epub 2021 Sep 11.

Abstract

Optimization of a previously reported lead series of PI3Kδ inhibitors with a novel binding mode led to the identification of a clinical candidate compound 31 (GSK251). Removal of an embedded Ames-positive heteroaromatic amine by reversing a sulfonamide followed by locating an interaction with Trp760 led to a highly selective compound 9. Further optimization to avoid glutathione trapping, to enhance potency and selectivity, and to optimize an oral pharmacokinetic profile led to the discovery of compound 31 (GSK215) that had a low predicted daily dose (45 mg, b.i.d) and a rat toxicity profile suitable for further development.

MeSH terms

  • Animals
  • Class I Phosphatidylinositol 3-Kinases / metabolism*
  • Crystallography, X-Ray
  • Female
  • Male
  • Mice
  • Mice, Inbred BALB C
  • Molecular Structure
  • Phosphoinositide-3 Kinase Inhibitors / chemical synthesis
  • Phosphoinositide-3 Kinase Inhibitors / metabolism
  • Phosphoinositide-3 Kinase Inhibitors / pharmacology*
  • Protein Binding
  • Rats
  • Rats, Wistar
  • Structure-Activity Relationship
  • Sulfonamides / chemical synthesis
  • Sulfonamides / metabolism
  • Sulfonamides / pharmacology*

Substances

  • Phosphoinositide-3 Kinase Inhibitors
  • Sulfonamides
  • Class I Phosphatidylinositol 3-Kinases