Discovery of Nanomolar Melanocortin-3 Receptor (MC3R)-Selective Small Molecule Pyrrolidine Bis-Cyclic Guanidine Agonist Compounds Via a High-Throughput "Unbiased" Screening Campaign

J Med Chem. 2021 May 13;64(9):5577-5592. doi: 10.1021/acs.jmedchem.0c02041. Epub 2021 Apr 22.

Abstract

The central melanocortin-3 and melanocortin-4 receptors (MC3R, MC4R) are key regulators of body weight and energy homeostasis. Herein, the discovery and characterization of first-in-class small molecule melanocortin agonists with selectivity for the melanocortin-3 receptor over the melanocortin-4 receptor are reported. Identified via "unbiased" mixture-based high-throughput screening approaches, pharmacological evaluation of these pyrrolidine bis-cyclic guanidines resulted in nanomolar agonist activity at the melanocortin-3 receptor. The pharmacological profiles at the remaining melanocortin receptor subtypes tested indicated similar agonist potencies at both the melanocortin-1 and melanocortin-5 receptors and antagonist or micromolar agonist activities at the melanocortin-4 receptor. This group of small molecules represents a new area of chemical space for the melanocortin receptors with mixed receptor pharmacology profiles that may serve as novel lead compounds to modulate states of dysregulated energy balance.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Algorithms
  • Animals
  • Drug Evaluation, Preclinical
  • Energy Metabolism / drug effects
  • Guanidine / analogs & derivatives
  • Guanidine / metabolism*
  • Guanidine / pharmacology
  • Guanidine / therapeutic use
  • High-Throughput Screening Assays
  • Humans
  • Mice
  • Mice, Knockout
  • Protein Isoforms / agonists
  • Protein Isoforms / genetics
  • Protein Isoforms / metabolism
  • Pyrrolidines / chemistry*
  • Pyrrolidines / metabolism
  • Pyrrolidines / pharmacology
  • Pyrrolidines / therapeutic use
  • Receptor, Melanocortin, Type 3 / agonists*
  • Receptor, Melanocortin, Type 3 / genetics
  • Receptor, Melanocortin, Type 3 / metabolism
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / metabolism
  • Small Molecule Libraries / pharmacology
  • Small Molecule Libraries / therapeutic use
  • Structure-Activity Relationship

Substances

  • Protein Isoforms
  • Pyrrolidines
  • Receptor, Melanocortin, Type 3
  • Small Molecule Libraries
  • Guanidine