Discovery, Optimization, and Biological Evaluation of Arylpyridones as Cbl-b Inhibitors

J Med Chem. 2024 Jan 25;67(2):1500-1512. doi: 10.1021/acs.jmedchem.3c02083. Epub 2024 Jan 16.

Abstract

Casitas B-lymphoma proto-oncogene-b (Cbl-b), a member of the Cbl family of RING finger E3 ubiquitin ligases, has been demonstrated to play a central role in regulating effector T-cell function. Multiple studies using gene-targeting approaches have provided direct evidence that Cbl-b negatively regulates T, B, and NK cell activation via a ubiquitin-mediated protein modulation. Thus, inhibition of Cbl-b ligase activity can lead to immune activation and has therapeutic potential in immuno-oncology. Herein, we describe the discovery and optimization of an arylpyridone series as Cbl-b inhibitors by structure-based drug discovery to afford compound 31. This compound binds to Cbl-b with an IC50 value of 30 nM and induces IL-2 production in T-cells with an EC50 value of 230 nM. Compound 31 also shows robust intracellular target engagement demonstrated through inhibition of Cbl-b autoubiquitination, inhibition of ubiquitin transfer to ZAP70, and the cellular modulation of phosphorylation of a downstream signal within the TCR axis.

MeSH terms

  • Phosphorylation
  • Proto-Oncogene Proteins c-cbl* / metabolism
  • T-Lymphocytes / metabolism
  • Ubiquitin / metabolism
  • Ubiquitin-Protein Ligases* / metabolism

Substances

  • Proto-Oncogene Proteins c-cbl
  • Ubiquitin-Protein Ligases
  • Ubiquitin