Noncovalent Mutant Selective Epidermal Growth Factor Receptor Inhibitors: A Lead Optimization Case Study

J Med Chem. 2015 Nov 25;58(22):8877-95. doi: 10.1021/acs.jmedchem.5b01412. Epub 2015 Nov 12.

Abstract

Because of their increased activity against activating mutants, first-generation epidermal growth factor receptor (EGFR) kinase inhibitors have had remarkable success in treating non-small-cell lung cancer (NSCLC) patients, but acquired resistance, through a secondary mutation of the gatekeeper residue, means that clinical responses only last for 8-14 months. Addressing this unmet medical need requires agents that can target both of the most common double mutants: T790M/L858R (TMLR) and T790M/del(746-750) (TMdel). Herein we describe how a noncovalent double mutant selective lead compound was optimized using a strategy focused on the structure-guided increase in potency without added lipophilicity or reduction of three-dimensional character. Following successive rounds of design and synthesis it was discovered that cis-fluoro substitution on 4-hydroxy- and 4-methoxypiperidinyl groups provided synergistic, substantial, and specific potency gain through direct interaction with the enzyme and/or effects on the proximal ligand oxygen atom. Further development of the fluorohydroxypiperidine series resulted in the identification of a pair of diastereomers that showed 50-fold enzyme and cell based selectivity for T790M mutants over wild-type EGFR (wtEGFR) in vitro and pathway knock-down in an in vivo xenograft model.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Antineoplastic Agents / chemical synthesis*
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / pharmacology*
  • Carcinoma, Non-Small-Cell Lung / drug therapy
  • Cell Line, Tumor
  • Dogs
  • Drug Design
  • ErbB Receptors / antagonists & inhibitors*
  • Gene Knockdown Techniques
  • Genes, erbB-1 / drug effects*
  • Humans
  • In Vitro Techniques
  • Lipids / chemistry
  • Lung Neoplasms / drug therapy
  • Macaca fascicularis
  • Microsomes, Liver / metabolism
  • Models, Molecular
  • Mutation
  • Rats
  • Stereoisomerism
  • Structure-Activity Relationship
  • Substrate Specificity
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Lipids
  • ErbB Receptors