Discovery of a Potent and Selective PI3Kδ Inhibitor (S)-2,4-Diamino-6-((1-(7-fluoro-1-(4-fluorophenyl)-4-oxo-3-phenyl-4 H-quinolizin-2-yl)ethyl)amino)pyrimidine-5-carbonitrile with Improved Pharmacokinetic Profile and Superior Efficacy in Hematological Cancer Models

J Med Chem. 2020 Dec 10;63(23):14700-14723. doi: 10.1021/acs.jmedchem.0c01264. Epub 2020 Dec 1.

Abstract

PI3Kδ inhibitors have been approved for B-cell malignancies like CLL, small lymphocytic lymphoma, and so forth. However, currently available PI3Kδ inhibitors are nonoptimal, showing weakness against at least one of the several important properties: potency, isoform selectivity, and/or pharmacokinetic profile. To come up with a PI3Kδ inhibitor that overcomes all these deficiencies, a pharmacophoric expansion strategy was employed. Herein, we describe a systematic transformation of a "three-blade propeller" shaped lead, 2,3-disubstituted quinolizinone 11, through a 1,2-disubstituted quinolizinone 20 to a novel "four-blade propeller" shaped 1,2,3-trisubstituted quinolizinone 34. Compound 34 has excellent potency, isoform selectivity, metabolic stability across species, and exhibited a favorable pharmacokinetic profile. Compound 34 also demonstrated a differentiated efficacy profile in human germinal center B and activated B cell-DLBCL cell lines and xenograft models. Compound 34 qualifies for further evaluation as a candidate for monotherapy or in combination with other targeted agents in DLBCLs and other forms of iNHL.

MeSH terms

  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / metabolism
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / therapeutic use*
  • Cell Line, Tumor
  • Class I Phosphatidylinositol 3-Kinases / chemical synthesis
  • Class I Phosphatidylinositol 3-Kinases / metabolism
  • Class I Phosphatidylinositol 3-Kinases / pharmacokinetics
  • Class I Phosphatidylinositol 3-Kinases / therapeutic use*
  • Dogs
  • Drug Discovery
  • Female
  • Hematologic Neoplasms / drug therapy*
  • Humans
  • Male
  • Mice
  • Mice, Inbred BALB C
  • Mice, Inbred NOD
  • Mice, SCID
  • Molecular Docking Simulation
  • Molecular Structure
  • Phosphoinositide-3 Kinase Inhibitors / chemical synthesis
  • Phosphoinositide-3 Kinase Inhibitors / metabolism
  • Phosphoinositide-3 Kinase Inhibitors / pharmacokinetics
  • Phosphoinositide-3 Kinase Inhibitors / therapeutic use*
  • Quinolizines / chemical synthesis
  • Quinolizines / metabolism
  • Quinolizines / pharmacokinetics
  • Quinolizines / therapeutic use*
  • RAW 264.7 Cells
  • Rats, Sprague-Dawley
  • Structure-Activity Relationship
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Phosphoinositide-3 Kinase Inhibitors
  • Quinolizines
  • Class I Phosphatidylinositol 3-Kinases
  • Pik3cd protein, mouse