Design and Synthesis of Clinical Candidate PF-06852231 (CVL-231): A Brain Penetrant, Selective, Positive Allosteric Modulator of the M4 Muscarinic Acetylcholine Receptor

J Med Chem. 2024 Jul 11;67(13):10831-10847. doi: 10.1021/acs.jmedchem.4c00293. Epub 2024 Jun 18.

Abstract

Selective activation of the M4 muscarinic acetylcholine receptor subtype offers a novel strategy for the treatment of psychosis in multiple neurological disorders. Although the development of traditional muscarinic activators has been stymied due to pan-receptor activation, muscarinic receptor subtype selectivity can be achieved through the utilization of a subtype of a unique allosteric site. A major challenge in capitalizing on this allosteric site to date has been achieving a balance of suitable potency and brain penetration. Herein, we describe the design of a brain penetrant series of M4 selective positive allosteric modulators (PAMs), ultimately culminating in the identification of 21 (PF-06852231, now CVL-231/emraclidine), which is under active clinical development as a novel mechanism and approach for the treatment of schizophrenia.

MeSH terms

  • Allosteric Regulation / drug effects
  • Animals
  • Brain* / drug effects
  • Brain* / metabolism
  • CHO Cells
  • Cricetulus
  • Drug Design*
  • Humans
  • Muscarinic Agonists / chemical synthesis
  • Muscarinic Agonists / chemistry
  • Muscarinic Agonists / pharmacology
  • Rats
  • Receptor, Muscarinic M4* / agonists
  • Receptor, Muscarinic M4* / metabolism
  • Schizophrenia / drug therapy
  • Schizophrenia / metabolism
  • Structure-Activity Relationship

Substances

  • Receptor, Muscarinic M4
  • Muscarinic Agonists