Discovery of AMG 232, a potent, selective, and orally bioavailable MDM2-p53 inhibitor in clinical development

J Med Chem. 2014 Feb 27;57(4):1454-72. doi: 10.1021/jm401753e. Epub 2014 Feb 5.

Abstract

We recently reported the discovery of AM-8553 (1), a potent and selective piperidinone inhibitor of the MDM2-p53 interaction. Continued research investigation of the N-alkyl substituent of this series, focused in particular on a previously underutilized interaction in a shallow cleft on the MDM2 surface, led to the discovery of a one-carbon tethered sulfone which gave rise to substantial improvements in biochemical and cellular potency. Further investigation produced AMG 232 (2), which is currently being evaluated in human clinical trials for the treatment of cancer. Compound 2 is an extremely potent MDM2 inhibitor (SPR KD = 0.045 nM, SJSA-1 EdU IC50 = 9.1 nM), with remarkable pharmacokinetic properties and in vivo antitumor activity in the SJSA-1 osteosarcoma xenograft model (ED50 = 9.1 mg/kg).

MeSH terms

  • Acetates / chemistry
  • Acetates / pharmacology*
  • Administration, Oral
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology*
  • Biological Availability
  • Crystallography, X-Ray
  • Drug Discovery
  • Humans
  • Piperidones / chemistry
  • Piperidones / pharmacology*
  • Protein Conformation
  • Proto-Oncogene Proteins c-mdm2 / antagonists & inhibitors*
  • Tumor Suppressor Protein p53 / antagonists & inhibitors*

Substances

  • 2-(5-(3-chlorophenyl)-6-(4-chlorophenyl)-1-(1-(isopropylsulfonyl)-3-methylbutan-2-yl)-3-methyl-2-oxopiperidin-3-yl)acetic acid
  • Acetates
  • Antineoplastic Agents
  • Piperidones
  • TP53 protein, human
  • Tumor Suppressor Protein p53
  • MDM2 protein, human
  • Proto-Oncogene Proteins c-mdm2