Discovery of a Potent, Selective, and Orally Available MTHFD2 Inhibitor (DS18561882) with in Vivo Antitumor Activity

J Med Chem. 2019 Nov 27;62(22):10204-10220. doi: 10.1021/acs.jmedchem.9b01113. Epub 2019 Nov 18.

Abstract

We report the discovery of a potent and isozyme-selective MTHFD2 inhibitor, DS18561882 (2). Through investigation of the substituents on our tricyclic coumarin scaffold (1,2,3,4-tetrahydrochromeno[3,4-c]pyridin-5-one), MTHFD2 inhibitory activity was shown to be elevated by incorporating an amine moiety at the 8-position and a methyl group at the 7-position of the initial lead 1. X-ray structure analysis revealed that a key interaction for enhanced potency was salt bridge formation between the amine moiety and the diphosphate linker of an NAD+ cofactor. Furthermore, ortho-substituted sulfonamide in place of benzoic acid of 1 significantly improved cell permeability and cell-based growth inhibition against a human breast cancer cell line. The thus-optimized DS18561882 showed the strongest cell-based activity (GI50 = 140 nM) in the class, a good oral pharmacokinetic profile, and thereby tumor growth inhibition in a mouse xenograft model upon oral administration.

MeSH terms

  • Administration, Oral
  • Aminohydrolases / antagonists & inhibitors*
  • Animals
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / pharmacology*
  • Breast Neoplasms / drug therapy
  • Breast Neoplasms / pathology
  • Cell Line, Tumor
  • Cell Membrane Permeability / drug effects
  • Crystallography, X-Ray
  • Female
  • Humans
  • Male
  • Methylenetetrahydrofolate Dehydrogenase (NADP) / antagonists & inhibitors*
  • Mice, Inbred BALB C
  • Multifunctional Enzymes / antagonists & inhibitors*
  • Structure-Activity Relationship
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • MTHFD2 protein, human
  • Multifunctional Enzymes
  • Methylenetetrahydrofolate Dehydrogenase (NADP)
  • Aminohydrolases