Discovery of Novel Pyrazolo-pyridone DCN1 Inhibitors Controlling Cullin Neddylation

J Med Chem. 2019 Sep 26;62(18):8429-8442. doi: 10.1021/acs.jmedchem.9b00410. Epub 2019 Sep 13.

Abstract

Chemical control of cullin neddylation is attracting increased attention based largely on the successes of the NEDD8-activating enzyme (E1) inhibitor pevonedistat. Recently reported chemical probes enable selective and time-dependent inhibition of downstream members of the neddylation trienzymatic cascade including the co-E3, DCN1. In this work, we report the optimization of a novel class of small molecule inhibitors of the DCN1-UBE2M interaction. Rational X-ray co-structure enabled optimization afforded a 25-fold improvement in potency relative to the initial screening hit. The potency gains are largely attributed to additional hydrophobic interactions mimicking the N-terminal acetyl group that drives binding of UBE2M to DCN1. The compounds inhibit the protein-protein interaction, block NEDD8 transfer in biochemical assays, engage DCN1 in cells, and selectively reduce the steady-state neddylation of Cul1 and Cul3 in two squamous carcinoma cell lines harboring DCN1 amplification.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Amides / chemistry
  • Carcinoma, Squamous Cell / drug therapy
  • Cell Line, Tumor
  • Cell Proliferation
  • Cullin Proteins / chemistry*
  • Cyclopentanes / pharmacology
  • Drug Design
  • Fibroblasts / metabolism
  • Glycine / chemistry
  • Humans
  • Intracellular Signaling Peptides and Proteins / antagonists & inhibitors*
  • Intracellular Signaling Peptides and Proteins / chemistry
  • NEDD8 Protein / chemistry*
  • Protein Domains
  • Protein Interaction Mapping
  • Pyrazoles / chemistry*
  • Pyridones / chemistry*
  • Pyrimidines / pharmacology
  • Reactive Oxygen Species / chemistry
  • Structure-Activity Relationship
  • Ubiquitin-Conjugating Enzymes / chemistry

Substances

  • Amides
  • CUL3 protein, human
  • Cullin 1
  • Cullin Proteins
  • Cyclopentanes
  • DCUN1D1 protein, human
  • Intracellular Signaling Peptides and Proteins
  • NEDD8 Protein
  • NEDD8 protein, human
  • Pyrazoles
  • Pyridones
  • Pyrimidines
  • Reactive Oxygen Species
  • Ubiquitin-Conjugating Enzymes
  • UBE2M protein, human
  • pevonedistat
  • Glycine