Structure- and Property-Based Optimization of Efficient Pan-Bromodomain and Extra Terminal Inhibitors to Identify Oral and Intravenous Candidate I-BET787

J Med Chem. 2024 Jun 27;67(12):10464-10489. doi: 10.1021/acs.jmedchem.4c00959. Epub 2024 Jun 12.

Abstract

The bromodomain and extra terminal (BET) family of bromodomain-containing proteins are important epigenetic regulators that elicit their effect through binding histone tail N-acetyl lysine (KAc) post-translational modifications. Recognition of such markers has been implicated in a range of oncology and immune diseases and, as such, small-molecule inhibition of the BET family bromodomain-KAc protein-protein interaction has received significant interest as a therapeutic strategy, with several potential medicines under clinical evaluation. This work describes the structure- and property-based optimization of a ligand and lipophilic efficient pan-BET bromodomain inhibitor series to deliver candidate I-BET787 (70) that demonstrates efficacy in a mouse model of inflammation and suitable properties for both oral and intravenous (IV) administration. This focused two-phase explore-exploit medicinal chemistry effort delivered the candidate molecule in 3 months with less than 100 final compounds synthesized.

MeSH terms

  • Administration, Intravenous*
  • Administration, Oral
  • Animals
  • Humans
  • Mice
  • Molecular Structure
  • Structure-Activity Relationship
  • Transcription Factors / antagonists & inhibitors
  • Transcription Factors / metabolism

Substances

  • Transcription Factors