Synthesis and Pharmacological Characterization of 2-(2,6-Dichlorophenyl)-1-((1 S,3 R)-5-(3-hydroxy-3-methylbutyl)-3-(hydroxymethyl)-1-methyl-3,4-dihydroisoquinolin-2(1 H)-yl)ethan-1-one (LY3154207), a Potent, Subtype Selective, and Orally Available Positive Allosteric Modulator of the Human Dopamine D1 Receptor

J Med Chem. 2019 Oct 10;62(19):8711-8732. doi: 10.1021/acs.jmedchem.9b01234. Epub 2019 Sep 30.

Abstract

Clinical development of catechol-based orthosteric agonists of the dopamine D1 receptor has thus far been unsuccessful due to multiple challenges. To address these issues, we identified LY3154207 (3) as a novel, potent, and subtype selective human D1 positive allosteric modulator (PAM) with minimal allosteric agonist activity. Conformational studies showed LY3154207 adopts an unusual boat conformation, and a binding pose with the human D1 receptor was proposed based on this observation. In contrast to orthosteric agonists, LY3154207 showed a distinct pharmacological profile without a bell-shaped dose-response relationship or tachyphylaxis in preclinical models. Identification of a crystalline form of free LY3154207 from the discovery lots was not successful. Instead, a novel cocrystal form with superior solubility was discovered and determined to be suitable for development. This cocrystal form was advanced to clinical development as a potential first-in-class D1 PAM and is now in phase 2 studies for Lewy body dementia.

MeSH terms

  • Acetylcholine / metabolism
  • Administration, Oral
  • Allosteric Regulation / drug effects
  • Animals
  • Binding Sites
  • Crystallography, X-Ray
  • Cyclic AMP / metabolism
  • HEK293 Cells
  • Half-Life
  • Humans
  • Isoquinolines / chemistry
  • Isoquinolines / pharmacokinetics
  • Isoquinolines / pharmacology*
  • Kidney / drug effects
  • Kidney / metabolism
  • Locomotion / drug effects
  • Mice
  • Molecular Conformation
  • Protein Isoforms / agonists
  • Protein Isoforms / metabolism
  • Rats
  • Receptors, Dopamine D1 / agonists*
  • Receptors, Dopamine D1 / metabolism
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / metabolism
  • Small Molecule Libraries / pharmacology
  • Structure-Activity Relationship

Substances

  • Isoquinolines
  • LY3154207
  • Protein Isoforms
  • Receptors, Dopamine D1
  • Small Molecule Libraries
  • Cyclic AMP
  • Acetylcholine