Discovery of Potent, Highly Selective, and Orally Bioavailable MTA Cooperative PRMT5 Inhibitors with Robust In Vivo Antitumor Activity

J Med Chem. 2025 Jan 23;68(2):1940-1955. doi: 10.1021/acs.jmedchem.4c02732. Epub 2025 Jan 9.

Abstract

Protein arginine methyltransferase 5 (PRMT5), which catalyzes the symmetric dimethylation of arginine residues on target proteins, plays a critical role in gene expression regulation, RNA processing, and signal transduction. Aberrant PRMT5 activity has been implicated in cancers and other diseases, making it a potential therapeutic target. Here, we report the discovery of a methylthioadenosine (MTA) cooperative PRMT5 inhibitor. Compound 20 exhibited strong antiproliferation activity in multiple MTAP-deleted cancer cell lines, excellent selectivity over MTAP wild-type cell lines, as well as satisfactory oral pharmacokinetic properties over various preclinical species. Notably, compound 20 demonstrated a dose-dependent reduction of symmetric dimethylarginine (SDMA) expression in the LU99 cell line and robust in vivo antitumor activity in the LU99 subcutaneous model.

MeSH terms

  • Administration, Oral
  • Animals
  • Antineoplastic Agents* / chemistry
  • Antineoplastic Agents* / pharmacokinetics
  • Antineoplastic Agents* / pharmacology
  • Biological Availability
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Drug Discovery
  • Enzyme Inhibitors / chemical synthesis
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacokinetics
  • Enzyme Inhibitors / pharmacology
  • Female
  • Humans
  • Male
  • Mice
  • Protein-Arginine N-Methyltransferases* / antagonists & inhibitors
  • Protein-Arginine N-Methyltransferases* / metabolism
  • Rats
  • Structure-Activity Relationship
  • Thionucleosides / pharmacology

Substances

  • Protein-Arginine N-Methyltransferases
  • Antineoplastic Agents
  • PRMT5 protein, human
  • Enzyme Inhibitors
  • Thionucleosides