Design of a Biased Potent Small Molecule Inhibitor of the Bromodomain and PHD Finger-Containing (BRPF) Proteins Suitable for Cellular and in Vivo Studies

J Med Chem. 2017 Jan 26;60(2):668-680. doi: 10.1021/acs.jmedchem.6b01583. Epub 2017 Jan 9.

Abstract

The BRPF (bromodomain and PHD finger-containing) family are scaffolding proteins important for the recruitment of histone acetyltransferases of the MYST family to chromatin. Evaluation of the BRPF family as a potential drug target is at an early stage although there is an emerging understanding of a role in acute myeloid leukemia (AML). We report the optimization of fragment hit 5b to 13-d as a biased, potent inhibitor of the BRD of the BRPFs with excellent selectivity over nonclass IV BRD proteins. Evaluation of 13-d in a panel of cancer cell lines showed a selective inhibition of proliferation of a subset of AML lines. Pharmacokinetic studies established that 13-d had properties compatible with oral dosing in mouse models of disease (Fpo 49%). We propose that NI-42 (13-d) is a new chemical probe for the BRPFs suitable for cellular and in vivo studies to explore the fundamental biology of these proteins.

MeSH terms

  • Adaptor Proteins, Signal Transducing / antagonists & inhibitors*
  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / pharmacology*
  • Cell Line, Tumor
  • DNA-Binding Proteins
  • Drug Screening Assays, Antitumor
  • Humans
  • Leukemia, Myeloid, Acute / drug therapy
  • Mice
  • Microsomes, Liver / metabolism
  • Nuclear Proteins / antagonists & inhibitors*
  • Protein Domains
  • Quinolones / chemical synthesis
  • Quinolones / chemistry
  • Quinolones / pharmacokinetics
  • Quinolones / pharmacology*
  • Structure-Activity Relationship
  • Sulfonamides / chemical synthesis
  • Sulfonamides / chemistry
  • Sulfonamides / pharmacokinetics
  • Sulfonamides / pharmacology*

Substances

  • 4-cyano-N-(1,3-dimethyl-2-oxo-1,2-dihydroquinolin-6-yl)benzenesulfonamide
  • Adaptor Proteins, Signal Transducing
  • Antineoplastic Agents
  • BRPF1 protein, human
  • DNA-Binding Proteins
  • Nuclear Proteins
  • Quinolones
  • Sulfonamides