Position 5.46 of the serotonin 5-HT2A receptor contributes to a species-dependent variation for the 5-HT2C agonist (R)-9-ethyl-1,3,4,10b-tetrahydro-7-trifluoromethylpyrazino[2,1-a]isoindol-6(2H)-one: impact on selectivity and toxicological evaluation

Mol Pharmacol. 2009 Dec;76(6):1211-9. doi: 10.1124/mol.109.059204. Epub 2009 Sep 18.

Abstract

Successful development of 5-HT(2C) agonists requires selectivity versus the highly homologous 5-HT(2A) receptor, because agonism at this receptor can result in significant adverse events. (R)-9-Ethyl-1,3,4,10b-tetrahydro-7-trifluoromethylpyrazino[2,1-a]isoindol-6(2H)-one (compound 1) is a potent 5-HT(2C) agonist exhibiting selectivity over the human 5-HT(2A) receptor. Evaluation of the compound at the rat 5-HT(2A) receptor, however, revealed potent binding and agonist functional activity. The physiological consequence of this higher potency was the observation of a significant increase in blood pressure in conscious telemeterized rats that could be prevented by ketanserin. Docking of compound 1 in a homology model of the 5-HT(2A) receptor indicated a possible binding mode in which the ethyl group at the 9-position of the molecule was oriented toward position 5.46 of the 5-HT(2A) receptor. Within the human 5-HT(2A) receptor, position 5.46 is Ser242; however, in the rat 5-HT(2A) receptor, it is Ala242, suggesting that the potent functional activity in this species resulted from the absence of the steric bulk provided by the -OH moiety of the Ser in the human isoform. We confirmed this hypothesis using site-directed mutagenesis through the mutation of both the human receptor Ser242 to Ala and the rat receptor Ala242 to Ser, followed by radioligand binding and second messenger studies. In addition, we attempted to define the space allowed by the alanine by evaluating compounds with larger substitutions at the 9-position. The data indicate that position 5.46 contributed to the species difference in 5-HT(2A) receptor potency observed for a pyrazinoisoindolone compound, resulting in the observation of a significant cardiovascular safety signal.

MeSH terms

  • Animals
  • Binding, Competitive / drug effects
  • Blood Pressure / drug effects
  • Calcium / metabolism
  • Cell Line
  • Dogs
  • Genetic Variation
  • Humans
  • Isoindoles / metabolism
  • Isoindoles / pharmacology*
  • Ketanserin / pharmacology
  • Macaca fascicularis
  • Male
  • Motor Activity / drug effects
  • Mutagenesis, Site-Directed
  • Protein Binding / drug effects
  • Pyrazines / metabolism
  • Pyrazines / pharmacology*
  • Rats
  • Rats, Sprague-Dawley
  • Receptor, Serotonin, 5-HT2A / drug effects
  • Receptor, Serotonin, 5-HT2A / metabolism
  • Receptor, Serotonin, 5-HT2C / genetics
  • Receptor, Serotonin, 5-HT2C / physiology
  • Sequence Homology, Amino Acid
  • Serotonin 5-HT2 Receptor Agonists*
  • Serotonin Antagonists / pharmacology
  • Species Specificity
  • Structural Homology, Protein

Substances

  • 9-ethyl-1,3,4,10b-tetrahydro-7-trifluoromethylpyrazino(2,1-a)isoindol-6(2H)-one
  • Isoindoles
  • Pyrazines
  • Receptor, Serotonin, 5-HT2A
  • Receptor, Serotonin, 5-HT2C
  • Serotonin 5-HT2 Receptor Agonists
  • Serotonin Antagonists
  • Ketanserin
  • Calcium