The discovery of macrocyclic XIAP antagonists from a DNA-programmed chemistry library, and their optimization to give lead compounds with in vivo antitumor activity

J Med Chem. 2015 Mar 26;58(6):2855-61. doi: 10.1021/jm501892g. Epub 2015 Mar 12.

Abstract

Affinity selection screening of macrocycle libraries derived from DNA-programmed chemistry identified XIAP BIR2 and BIR3 domain inhibitors that displace bound pro-apoptotic caspases. X-ray cocrystal structures of key compounds with XIAP BIR2 suggested potency-enhancing structural modifications. Optimization of dimeric macrocycles with similar affinity for both domains were potent pro-apoptotic agents in cancer cell lines and efficacious in shrinking tumors in a mouse xenograft model.

MeSH terms

  • Animals
  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / therapeutic use*
  • Breast / drug effects
  • Breast / metabolism
  • Breast / pathology
  • Breast Neoplasms / drug therapy*
  • Breast Neoplasms / metabolism
  • Breast Neoplasms / pathology
  • Caspase 3 / metabolism
  • Cell Line, Tumor
  • Crystallography, X-Ray
  • Drug Discovery
  • Female
  • Gene Library
  • Humans
  • Macrocyclic Compounds / chemistry*
  • Macrocyclic Compounds / pharmacokinetics
  • Macrocyclic Compounds / therapeutic use*
  • Mice
  • Models, Molecular
  • X-Linked Inhibitor of Apoptosis Protein / antagonists & inhibitors*
  • X-Linked Inhibitor of Apoptosis Protein / metabolism

Substances

  • Antineoplastic Agents
  • Macrocyclic Compounds
  • X-Linked Inhibitor of Apoptosis Protein
  • Caspase 3