Discovery of epigenetic regulator I-BET762: lead optimization to afford a clinical candidate inhibitor of the BET bromodomains

J Med Chem. 2013 Oct 10;56(19):7501-15. doi: 10.1021/jm401088k. Epub 2013 Sep 25.

Abstract

The bromo and extra C-terminal domain (BET) family of bromodomains are involved in binding epigenetic marks on histone proteins, more specifically acetylated lysine residues. This paper describes the discovery and structure-activity relationships (SAR) of potent benzodiazepine inhibitors that disrupt the function of the BET family of bromodomains (BRD2, BRD3, and BRD4). This work has yielded a potent, selective compound I-BET762 that is now under evaluation in a phase I/II clinical trial for nuclear protein in testis (NUT) midline carcinoma and other cancers.

MeSH terms

  • Animals
  • Anti-Inflammatory Agents / chemical synthesis
  • Anti-Inflammatory Agents / pharmacokinetics
  • Anti-Inflammatory Agents / pharmacology
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / pharmacology*
  • Apolipoprotein A-I / biosynthesis
  • Benzodiazepines / chemical synthesis
  • Benzodiazepines / pharmacokinetics
  • Benzodiazepines / pharmacology*
  • Cell Cycle Proteins
  • Dogs
  • Epigenesis, Genetic
  • Humans
  • Macaca fascicularis
  • Mice
  • Models, Molecular
  • Nuclear Proteins / antagonists & inhibitors*
  • Permeability
  • Protein Structure, Tertiary
  • Rats
  • Stereoisomerism
  • Structure-Activity Relationship
  • Transcription Factors / antagonists & inhibitors*

Substances

  • APOA1 protein, human
  • Anti-Inflammatory Agents
  • Antineoplastic Agents
  • Apolipoprotein A-I
  • BRD4 protein, human
  • Cell Cycle Proteins
  • Nuclear Proteins
  • Transcription Factors
  • Benzodiazepines
  • molibresib

Associated data

  • PDB/4C66
  • PDB/4C67