Design, Synthesis, and Characterization of I-BET567, a Pan-Bromodomain and Extra Terminal (BET) Bromodomain Oral Candidate

J Med Chem. 2022 Feb 10;65(3):2262-2287. doi: 10.1021/acs.jmedchem.1c01747. Epub 2022 Jan 7.

Abstract

Through regulation of the epigenome, the bromodomain and extra terminal (BET) family of proteins represent important therapeutic targets for the treatment of human disease. Through mimicking the endogenous N-acetyl-lysine group and disrupting the protein-protein interaction between histone tails and the bromodomain, several small molecule pan-BET inhibitors have progressed to oncology clinical trials. This work describes the medicinal chemistry strategy and execution to deliver an orally bioavailable tetrahydroquinoline (THQ) pan-BET candidate. Critical to the success of this endeavor was a potency agnostic analysis of a data set of 1999 THQ BET inhibitors within the GSK collection which enabled identification of appropriate lipophilicity space to deliver compounds with a higher probability of desired oral candidate quality properties. SAR knowledge was leveraged via Free-Wilson analysis within this design space to identify a small group of targets which ultimately delivered I-BET567 (27), a pan-BET candidate inhibitor that demonstrated efficacy in mouse models of oncology and inflammation.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Aminoquinolines / chemistry*
  • Aminoquinolines / metabolism
  • Aminoquinolines / pharmacokinetics
  • Aminoquinolines / therapeutic use
  • Animals
  • Benzoates / chemistry
  • Benzoates / metabolism
  • Binding Sites
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Crystallography, X-Ray
  • Dogs
  • Drug Design*
  • Half-Life
  • Humans
  • Male
  • Mice
  • Molecular Conformation
  • Molecular Dynamics Simulation
  • Neoplasms / drug therapy
  • Proteins / antagonists & inhibitors
  • Proteins / metabolism*
  • Rats
  • Structure-Activity Relationship

Substances

  • 4-(1-acetyl-4-((4-chlorophenyl)amino)-2-methyl-1,2,3,4-tetrahydroquinolin-6-yl)benzoic acid
  • Aminoquinolines
  • Benzoates
  • Proteins
  • bromodomain and extra-terminal domain protein, human