Discovery of (10R)-7-amino-12-fluoro-2,10,16-trimethyl-15-oxo-10,15,16,17-tetrahydro-2H-8,4-(metheno)pyrazolo[4,3-h][2,5,11]-benzoxadiazacyclotetradecine-3-carbonitrile (PF-06463922), a macrocyclic inhibitor of anaplastic lymphoma kinase (ALK) and c-ros oncogene 1 (ROS1) with preclinical brain exposure and broad-spectrum potency against ALK-resistant mutations

J Med Chem. 2014 Jun 12;57(11):4720-44. doi: 10.1021/jm500261q. Epub 2014 Jun 3.

Abstract

Although crizotinib demonstrates robust efficacy in anaplastic lymphoma kinase (ALK)-positive non-small-cell lung carcinoma patients, progression during treatment eventually develops. Resistant patient samples revealed a variety of point mutations in the kinase domain of ALK, including the L1196M gatekeeper mutation. In addition, some patients progress due to cancer metastasis in the brain. Using structure-based drug design, lipophilic efficiency, and physical-property-based optimization, highly potent macrocyclic ALK inhibitors were prepared with good absorption, distribution, metabolism, and excretion (ADME), low propensity for p-glycoprotein 1-mediated efflux, and good passive permeability. These structurally unusual macrocyclic inhibitors were potent against wild-type ALK and clinically reported ALK kinase domain mutations. Significant synthetic challenges were overcome, utilizing novel transformations to enable the use of these macrocycles in drug discovery paradigms. This work led to the discovery of 8k (PF-06463922), combining broad-spectrum potency, central nervous system ADME, and a high degree of kinase selectivity.

MeSH terms

  • Aminopyridines
  • Anaplastic Lymphoma Kinase
  • Animals
  • Antineoplastic Agents / chemical synthesis*
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / pharmacology
  • Brain / metabolism*
  • Crystallography, X-Ray
  • Drug Resistance, Neoplasm
  • Humans
  • Lactams
  • Lactams, Macrocyclic / chemical synthesis*
  • Lactams, Macrocyclic / pharmacokinetics
  • Lactams, Macrocyclic / pharmacology
  • Mice
  • Microsomes, Liver / metabolism
  • Models, Molecular
  • Mutation
  • NIH 3T3 Cells
  • Protein-Tyrosine Kinases / antagonists & inhibitors*
  • Proto-Oncogene Proteins / antagonists & inhibitors*
  • Pyrazoles
  • Rats
  • Receptor Protein-Tyrosine Kinases / antagonists & inhibitors*
  • Receptor Protein-Tyrosine Kinases / genetics
  • Stereoisomerism
  • Structure-Activity Relationship

Substances

  • Aminopyridines
  • Antineoplastic Agents
  • Lactams
  • Lactams, Macrocyclic
  • Proto-Oncogene Proteins
  • Pyrazoles
  • ALK protein, human
  • Alk protein, mouse
  • Alk protein, rat
  • Anaplastic Lymphoma Kinase
  • Protein-Tyrosine Kinases
  • ROS1 protein, human
  • Receptor Protein-Tyrosine Kinases
  • lorlatinib

Associated data

  • PDB/4CLI
  • PDB/4CLJ
  • PDB/4CMO
  • PDB/4CMT
  • PDB/4CMU
  • PDB/4CNH
  • PDB/4CTB
  • PDB/4CTC