Discovery of a Potent and Selective ATAD2 Bromodomain Inhibitor with Antiproliferative Activity in Breast Cancer Models

J Med Chem. 2022 Feb 24;65(4):3306-3331. doi: 10.1021/acs.jmedchem.1c01871. Epub 2022 Feb 8.

Abstract

ATAD2 is an epigenetic bromodomain-containing target which is overexpressed in many cancers and has been suggested as a potential oncology target. While several small molecule inhibitors have been described in the literature, their cellular activity has proved to be underwhelming. In this work, we describe the identification of a novel series of ATAD2 inhibitors by high throughput screening, confirmation of the bromodomain region as the site of action, and the optimization campaign undertaken to improve the potency, selectivity, and permeability of the initial hit. The result is compound 5 (AZ13824374), a highly potent and selective ATAD2 inhibitor which shows cellular target engagement and antiproliferative activity in a range of breast cancer models.

MeSH terms

  • ATPases Associated with Diverse Cellular Activities / antagonists & inhibitors*
  • Antineoplastic Agents / chemical synthesis*
  • Antineoplastic Agents / pharmacology*
  • Breast Neoplasms / drug therapy*
  • Cell Line, Tumor
  • Crystallography, X-Ray
  • DNA-Binding Proteins / antagonists & inhibitors*
  • Drug Discovery
  • Drug Screening Assays, Antitumor
  • Female
  • Humans
  • Models, Molecular
  • Small Molecule Libraries
  • Structure-Activity Relationship
  • Substrate Specificity
  • Tumor Stem Cell Assay

Substances

  • Antineoplastic Agents
  • DNA-Binding Proteins
  • Small Molecule Libraries
  • ATAD2 protein, human
  • ATPases Associated with Diverse Cellular Activities