Potent and Selective Human 5-HT2B Serotonin Receptor Antagonists: 4'-Cyano-(N)-methanocarba-adenosines by Synthetic Serendipity

J Med Chem. 2024 Dec 12;67(23):21264-21291. doi: 10.1021/acs.jmedchem.4c02174. Epub 2024 Nov 26.

Abstract

Rigidified nucleoside derivatives with (N)-methanocarba replacement of ribose have been repurposed as peripheral subtype-selective 5-HT2B serotonin receptor antagonists for heart and lung fibrosis and intestinal/vascular conditions. 4'-Cyano derivative 40 (MRS8209; Ki, 4.27 nM) was 47-fold (human binding, but not rat and mouse) and 724-fold (functionally) selective at 5-HT2BR, compared to antitarget 5-HT2CR, and predicted to form a stable receptor complex using docking and molecular dynamics. 4'-Cyano substituents enhanced 5-HT2BR affinity (typically 4-5-fold compared to 4'-CH2OH), depending on an N6 group larger than methyl. Asymmetric N6 groups (4'-cyano-2-halo derivatives 33-35 and 37) provided potent 5-HT2BR Ki values (7-22 nM). A 4'-CH2CN substituent was less effective than 4'-CN at increasing 5-HT2BR affinity, while a 4'-CHF2 group produced high 5-HT2B affinity/selectivity. A 2-benzylthio-adenine group with unsubstituted 6-NH2 shifted the typical selectivity pattern toward potent 5-HT2C binding. Thus, the SAR suggests that N6-cyclopentyl-4'-cyano modifications are promising, with an interdependence among the substituent positions.

MeSH terms

  • Animals
  • Humans
  • Mice
  • Molecular Docking Simulation
  • Nitriles / chemistry
  • Nitriles / pharmacology
  • Rats
  • Receptor, Serotonin, 5-HT2B* / metabolism
  • Serotonin 5-HT2 Receptor Antagonists* / chemical synthesis
  • Serotonin 5-HT2 Receptor Antagonists* / chemistry
  • Serotonin 5-HT2 Receptor Antagonists* / pharmacology
  • Structure-Activity Relationship

Substances

  • Receptor, Serotonin, 5-HT2B
  • Serotonin 5-HT2 Receptor Antagonists
  • Nitriles