Small Molecule Neuropilin-1 Antagonists Combine Antiangiogenic and Antitumor Activity with Immune Modulation through Reduction of Transforming Growth Factor Beta (TGFβ) Production in Regulatory T-Cells

J Med Chem. 2018 May 10;61(9):4135-4154. doi: 10.1021/acs.jmedchem.8b00210. Epub 2018 Apr 24.

Abstract

We report the design, synthesis, and biological evaluation of some potent small-molecule neuropilin-1 (NRP1) antagonists. NRP1 is implicated in the immune response to tumors, particularly in Treg cell fragility, required for PD1 checkpoint blockade. The design of these compounds was based on a previously identified compound EG00229. The design of these molecules was informed and supported by X-ray crystal structures. Compound 1 (EG01377) was identified as having properties suitable for further investigation. Compound 1 was then tested in several in vitro assays and was shown to have antiangiogenic, antimigratory, and antitumor effects. Remarkably, 1 was shown to be selective for NRP1 over the closely related protein NRP2. In purified Nrp1+, FoxP3+, and CD25+ populations of Tregs from mice, 1 was able to block a glioma-conditioned medium-induced increase in TGFβ production. This comprehensive characterization of a small-molecule NRP1 antagonist provides the basis for future in vivo studies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Angiogenesis Inhibitors / chemistry
  • Angiogenesis Inhibitors / pharmacology
  • Animals
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology
  • Cell Line, Tumor
  • Drug Design
  • Humans
  • Immunomodulation / drug effects*
  • Mice
  • Models, Molecular
  • Molecular Conformation
  • Neuropilin-1 / antagonists & inhibitors*
  • Pentanoic Acids / chemistry
  • Pentanoic Acids / pharmacology
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / pharmacology*
  • T-Lymphocytes, Regulatory / drug effects*
  • T-Lymphocytes, Regulatory / immunology
  • T-Lymphocytes, Regulatory / metabolism*
  • Transforming Growth Factor beta / biosynthesis*
  • Vascular Endothelial Growth Factor A / pharmacology

Substances

  • Angiogenesis Inhibitors
  • Antineoplastic Agents
  • Pentanoic Acids
  • Small Molecule Libraries
  • Transforming Growth Factor beta
  • Vascular Endothelial Growth Factor A
  • Neuropilin-1