Discovery of a Potent Nonpeptidomimetic, Small-Molecule Antagonist of Cellular Inhibitor of Apoptosis Protein 1 (cIAP1) and X-Linked Inhibitor of Apoptosis Protein (XIAP)

J Med Chem. 2017 Jun 8;60(11):4611-4625. doi: 10.1021/acs.jmedchem.6b01877. Epub 2017 May 24.

Abstract

XIAP and cIAP1 are members of the inhibitor of apoptosis protein (IAP) family and are key regulators of anti-apoptotic and pro-survival signaling pathways. Overexpression of IAPs occurs in various cancers and has been associated with tumor progression and resistance to treatment. Structure-based drug design (SBDD) guided by structural information from X-ray crystallography, computational studies, and NMR solution conformational analysis was successfully applied to a fragment-derived lead resulting in AT-IAP, a potent, orally bioavailable, dual antagonist of XIAP and cIAP1 and a structurally novel chemical probe for IAP biology.

MeSH terms

  • Animals
  • Cell Line, Tumor
  • Crystallography, X-Ray
  • Drug Discovery
  • HEK293 Cells
  • Heterocyclic Compounds, 2-Ring / chemistry*
  • Heterocyclic Compounds, 2-Ring / pharmacology*
  • Humans
  • Inhibitor of Apoptosis Proteins / antagonists & inhibitors*
  • Mice
  • Mice, Inbred BALB C
  • Mice, SCID
  • Peptidomimetics
  • Piperazines / chemistry*
  • Piperazines / pharmacology*
  • Small Molecule Libraries
  • Structure-Activity Relationship
  • X-Linked Inhibitor of Apoptosis Protein / antagonists & inhibitors*

Substances

  • AT-IAP compound
  • Heterocyclic Compounds, 2-Ring
  • Inhibitor of Apoptosis Proteins
  • Peptidomimetics
  • Piperazines
  • Small Molecule Libraries
  • X-Linked Inhibitor of Apoptosis Protein
  • XIAP protein, human