Discovery and Preclinical Characterization of Usmarapride (SUVN-D4010): A Potent, Selective 5-HT4 Receptor Partial Agonist for the Treatment of Cognitive Deficits Associated with Alzheimer's Disease

J Med Chem. 2021 Aug 12;64(15):10641-10665. doi: 10.1021/acs.jmedchem.1c00703. Epub 2021 Jul 12.

Abstract

A series of oxadiazole derivatives were synthesized and evaluated as 5-hydroxytryptamine-4 receptor (5-HT4R) partial agonists for the treatment of cognitive deficits associated with Alzheimer's disease. Starting from a reported 5-HT4R antagonist, a systematic structure-activity relationship was conducted, which led to the discovery of potent and selective 5-HT4R partial agonist 1-isopropyl-3-{5-[1-(3-methoxypropyl) piperidin-4-yl]-[1,3,4]oxadiazol-2-yl}-1H-indazole oxalate (Usmarapride, 12l). It showed balanced physicochemical-pharmacokinetic properties with robust nonclinical efficacy in cognition models. It also showed disease-modifying potential, as it increased neuroprotective soluble amyloid precursor protein alpha levels, and dose-dependent target engagement and correlation of efficacy with oral exposures. Phase 1 clinical studies have been completed and projected efficacious concentration was achieved without any major safety concerns. Phase 2 enabling long-term safety studies have been completed with no concerns for further development.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alzheimer Disease / drug therapy*
  • Alzheimer Disease / metabolism
  • Cognition Disorders / drug therapy*
  • Cognition Disorders / metabolism
  • Dose-Response Relationship, Drug
  • Drug Discovery*
  • Humans
  • Molecular Structure
  • Neuroprotective Agents / chemical synthesis
  • Neuroprotective Agents / chemistry
  • Neuroprotective Agents / pharmacology*
  • Receptors, Serotonin, 5-HT4 / metabolism*
  • Serotonin 5-HT4 Receptor Agonists / chemical synthesis
  • Serotonin 5-HT4 Receptor Agonists / chemistry
  • Serotonin 5-HT4 Receptor Agonists / pharmacology*
  • Structure-Activity Relationship

Substances

  • Neuroprotective Agents
  • Serotonin 5-HT4 Receptor Agonists
  • Receptors, Serotonin, 5-HT4