Identification and Optimization of Benzimidazole Sulfonamides as Orally Bioavailable Sphingosine 1-Phosphate Receptor 1 Antagonists with in Vivo Activity

J Med Chem. 2015 Sep 10;58(17):7057-75. doi: 10.1021/acs.jmedchem.5b01078. Epub 2015 Aug 26.

Abstract

We report here a novel series of benzimidazole sulfonamides that act as antagonists of the S1P1 receptor, identified by exploiting an understanding of the pharmacophore of a high throughput screening (HTS)-derived series of compounds described previously. Lead compound 2 potently inhibits S1P-induced receptor internalization in a cell-based assay (EC50 = 0.05 μM), but has poor physical properties and metabolic stability. Evolution of this compound through structure-activity relationship development and property optimization led to in vivo probes such as 4. However, this compound was unexpectedly found to be a potent CYP3A inducer in human hepatocytes, and thus further chemistry efforts were directed at addressing this liability. By employing a pregnane X receptor (PXR) reporter gene assay to prioritize compounds for further testing in human hepatocytes, we identified lipophilicity as a key molecular property influencing the likelihood of P450 induction. Ultimately, we have identified compounds such as 46 and 47, which demonstrate the desired S1P1 antagonist activity while having greatly reduced risk of CYP3A induction in humans. These compounds have excellent oral bioavailability in preclinical species and exhibit pharmacodynamic effects of S1P1 antagonism in several in vivo models following oral dosing. Relatively modest antitumor activity was observed in multiple xenograft models, however, suggesting that selective S1P1 antagonists would have limited utility as anticancer therapeutics as single agents.

MeSH terms

  • Administration, Oral
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology
  • Benzimidazoles / chemical synthesis
  • Benzimidazoles / chemistry*
  • Benzimidazoles / pharmacology
  • Biological Availability
  • Cells, Cultured
  • Cytochrome P-450 CYP3A / biosynthesis
  • Cytochrome P-450 CYP3A Inducers / chemical synthesis
  • Cytochrome P-450 CYP3A Inducers / chemistry
  • Cytochrome P-450 CYP3A Inducers / pharmacology
  • Genes, Reporter
  • Hepatocytes / drug effects
  • Hepatocytes / metabolism
  • Humans
  • Models, Molecular
  • Molecular Structure
  • Pregnane X Receptor
  • Pyridines / chemical synthesis
  • Pyridines / chemistry*
  • Pyridines / pharmacology
  • Receptors, Lysosphingolipid / antagonists & inhibitors*
  • Receptors, Steroid / genetics
  • Stereoisomerism
  • Structure-Activity Relationship
  • Sulfonamides / chemical synthesis
  • Sulfonamides / chemistry*
  • Sulfonamides / pharmacology
  • Xenograft Model Antitumor Assays

Substances

  • 6-cyano-N-(1-(1-ethyl-6-(trifluoromethyl)-1H-imidazo(4,5-c)pyridin-2-yl)ethyl)pyridine-3-sulfonamide
  • 6-cyano-N-(1-(6-cyclopropyl-1-ethyl-1H-imidazo(4,5-c)pyridin-2-yl)ethyl)pyridine-3-sulfonamide
  • Antineoplastic Agents
  • Benzimidazoles
  • Cytochrome P-450 CYP3A Inducers
  • Pregnane X Receptor
  • Pyridines
  • Receptors, Lysosphingolipid
  • Receptors, Steroid
  • Sulfonamides
  • Cytochrome P-450 CYP3A

Associated data

  • PDB/5A86