Discovery of (2 S)- N-(6-Cyano-5-(trifluoromethyl)pyridin-3-yl)-3-(6-(4-cyanophenyl)-3,6-diazabicyclo[3.1.1]heptan-3-yl)-2-hydroxy-2-methylpropanamide as a Highly Potent and Selective Topical Androgen Receptor Antagonist for Androgenetic Alopecia Treatment

J Med Chem. 2024 Jan 11;67(1):322-348. doi: 10.1021/acs.jmedchem.3c01417. Epub 2023 Dec 21.

Abstract

Androgenetic alopecia (AGA) is the most prevalent form of progressive hair loss disorder in both men and women, significantly impacting their appearance and overall quality of life. Overactivation of the AR signaling pathway in dermal papilla cells (DPCs) plays a crucial role in the development and progression of AGA. Considering the severe systemic side effects associated with oral AR antagonists, the idea of developing of topical AR antagonists with rapid metabolic deactivation properties emerged as a promising approach. Herein, through systematic structural optimization, we successfully identified compound 30a as a potent and selective AR antagonist with favorable pharmacokinetic properties, resulting in high skin exposure and low plasma exposure following topical administration. Importantly, in both hair-growth and AGA mouse models, compound 30a showed potent hair-growth-promoting effects without any noticeable toxicity. These findings suggest that compound 30a holds significant potential as a topical AR antagonist for treating AGA patients.

MeSH terms

  • Alopecia / drug therapy
  • Androgen Receptor Antagonists* / pharmacology
  • Androgen Receptor Antagonists* / therapeutic use
  • Animals
  • Female
  • Humans
  • Male
  • Mice
  • Quality of Life*
  • Receptors, Androgen / metabolism
  • Skin / metabolism

Substances

  • Androgen Receptor Antagonists
  • Receptors, Androgen