Discovery of First-in-Class Protein Arginine Methyltransferase 5 (PRMT5) Degraders

J Med Chem. 2020 Sep 10;63(17):9977-9989. doi: 10.1021/acs.jmedchem.0c01111. Epub 2020 Aug 21.

Abstract

The aberrant expression of protein arginine methyltransferase 5 (PRMT5) has been associated with multiple cancers. Using the proteolysis targeting chimera technology, we discovered a first-in-class PRMT5 degrader 15 (MS4322). Here, we report the design, synthesis, and characterization of compound 15 and two structurally similar controls 17 (MS4370) and 21 (MS4369), with impaired binding to the von Hippel-Lindau E3 ligase and PRMT5, respectively. Compound 15, but not 17 and 21, effectively reduced the PRMT5 protein level in MCF-7 cells. Our mechanism studies indicate that compound 15 degraded PRMT5 in an E3 ligase- and proteasome-dependent manner. Compound 15 also effectively reduced the PRMT5 protein level and inhibited growth in multiple cancer cell lines. Moreover, compound 15 was highly selective for PRMT5 in a global proteomic study and exhibited good plasma exposure in mice. Collectively, compound 15 and its two controls 17 and 21 are valuable chemical tools for exploring the PRMT5 functions in health and disease.

Publication types

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

MeSH terms

  • Animals
  • Cell Line, Tumor
  • Dipeptides / chemical synthesis
  • Dipeptides / pharmacokinetics
  • Dipeptides / pharmacology*
  • Drug Design
  • Enzyme Inhibitors / chemical synthesis
  • Enzyme Inhibitors / pharmacokinetics
  • Enzyme Inhibitors / pharmacology*
  • Humans
  • Ligands
  • Male
  • Mice
  • Molecular Structure
  • Protein-Arginine N-Methyltransferases / antagonists & inhibitors*
  • Protein-Arginine N-Methyltransferases / metabolism
  • Proteolysis / drug effects
  • Structure-Activity Relationship
  • Von Hippel-Lindau Tumor Suppressor Protein / metabolism

Substances

  • Dipeptides
  • Enzyme Inhibitors
  • Ligands
  • PRMT5 protein, human
  • Protein-Arginine N-Methyltransferases
  • Von Hippel-Lindau Tumor Suppressor Protein