Discovery of Novel Dual Mechanism of Action Src Signaling and Tubulin Polymerization Inhibitors (KX2-391 and KX2-361)

J Med Chem. 2018 Jun 14;61(11):4704-4719. doi: 10.1021/acs.jmedchem.8b00164. Epub 2018 Apr 17.

Abstract

The discovery of potent, peptide site directed, tyrosine kinase inhibitors has remained an elusive goal. Herein we describe the discovery of two such clinical candidates that inhibit the tyrosine kinase Src. Compound 1 is a phase 3 clinical trial candidate that is likely to provide a first in class topical treatment for actinic keratosis (AK) with good efficacy and dramatically less toxicity compared to existing standard therapy. Compound 2 is a phase 1 clinical trial candidate that is likely to provide a first in class treatment of malignant glioblastoma and induces 30% long-term complete tumor remission in animal models. The discovery strategy for these compounds iteratively utilized molecular modeling, along with the synthesis and testing of increasingly elaborated proof of concept compounds, until the final clinical candidates were arrived at. This was followed with mechanism of action (MOA) studies that revealed tubulin polymerization inhibition as the second MOA.

MeSH terms

  • Acetamides / metabolism
  • Acetamides / pharmacology*
  • Amino Acid Sequence
  • Catalytic Domain
  • Cell Line, Tumor
  • Drug Discovery*
  • Humans
  • Molecular Docking Simulation
  • Morpholines / metabolism
  • Morpholines / pharmacology*
  • Protein Kinase Inhibitors / metabolism
  • Protein Kinase Inhibitors / pharmacology*
  • Pyridines / metabolism
  • Pyridines / pharmacology*
  • Signal Transduction / drug effects
  • Tubulin Modulators / metabolism
  • Tubulin Modulators / pharmacology*
  • src-Family Kinases / antagonists & inhibitors*
  • src-Family Kinases / chemistry
  • src-Family Kinases / metabolism

Substances

  • Acetamides
  • KX2-361
  • Morpholines
  • Protein Kinase Inhibitors
  • Pyridines
  • Tubulin Modulators
  • tirbanibulin
  • src-Family Kinases