Discovery of IACS-9439, a Potent, Exquisitely Selective, and Orally Bioavailable Inhibitor of CSF1R

J Med Chem. 2020 Sep 10;63(17):9888-9911. doi: 10.1021/acs.jmedchem.0c00936. Epub 2020 Aug 18.

Abstract

Tumor-associated macrophages (TAMs) have a significant presence in the tumor stroma across multiple human malignancies and are believed to be beneficial to tumor growth. Targeting CSF1R has been proposed as a potential therapy to reduce TAMs, especially the protumor, immune-suppressive M2 TAMs. Additionally, the high expression of CSF1R on tumor cells has been associated with poor survival in certain cancers, suggesting tumor dependency and therefore a potential therapeutic target. The CSF1-CSF1R signaling pathway modulates the production, differentiation, and function of TAMs; however, the discovery of selective CSF1R inhibitors devoid of type III kinase activity has proven to be challenging. We discovered a potent, highly selective, and orally bioavailable CSF1R inhibitor, IACS-9439 (1). Treatment with 1 led to a dose-dependent reduction in macrophages, promoted macrophage polarization toward the M1 phenotype, and led to tumor growth inhibition in MC38 and PANC02 syngeneic tumor models.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / therapeutic use*
  • Benzothiazoles / chemical synthesis
  • Benzothiazoles / pharmacokinetics
  • Benzothiazoles / therapeutic use*
  • Drug Stability
  • Humans
  • Microsomes, Liver / metabolism
  • Molecular Structure
  • Neoplasms / drug therapy*
  • Pyrimidines / chemical synthesis
  • Pyrimidines / pharmacokinetics
  • Pyrimidines / therapeutic use*
  • Receptors, Granulocyte-Macrophage Colony-Stimulating Factor / antagonists & inhibitors*
  • Structure-Activity Relationship
  • THP-1 Cells
  • Tumor-Associated Macrophages / drug effects

Substances

  • Antineoplastic Agents
  • Benzothiazoles
  • CSF1R protein, human
  • Pyrimidines
  • Receptors, Granulocyte-Macrophage Colony-Stimulating Factor