Design and Activity of Specific Hypoxia-Inducible Factor-2α (HIF-2α) Inhibitors for the Treatment of Clear Cell Renal Cell Carcinoma: Discovery of Clinical Candidate ( S)-3-((2,2-Difluoro-1-hydroxy-7-(methylsulfonyl)-2,3-dihydro-1 H-inden-4-yl)oxy)-5-fluorobenzonitrile (PT2385)

J Med Chem. 2018 Nov 8;61(21):9691-9721. doi: 10.1021/acs.jmedchem.8b01196. Epub 2018 Oct 18.

Abstract

HIF-2α, a member of the HIF family of transcription factors, is a key oncogenic driver in cancers such as clear cell renal cell carcinoma (ccRCC). A signature feature of these cancers is the overaccumulation of HIF-2α protein, often by inactivation of the E3 ligase VHL (von Hippel-Lindau). Herein we disclose our structure based drug design (SBDD) approach that culminated in the identification of PT2385, the first HIF-2α antagonist to enter clinical trials. Highlights include the use of a putative n → π*Ar interaction to guide early analog design, the conformational restriction of an essential hydroxyl moiety, and the remarkable impact of fluorination near the hydroxyl group. Evaluation of select compounds from two structural classes in a sequence of PK/PD, efficacy, PK, and metabolite profiling identified 10i (PT2385, luciferase EC50 = 27 nM) as the clinical candidate. Finally, a retrospective crystallographic analysis describes the structural perturbations necessary for efficient antagonism.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / pharmacology
  • Basic Helix-Loop-Helix Transcription Factors / antagonists & inhibitors*
  • Basic Helix-Loop-Helix Transcription Factors / chemistry
  • Carcinoma, Renal Cell / pathology*
  • Cell Line, Tumor
  • Dogs
  • Drug Design*
  • Indans / chemistry*
  • Indans / pharmacokinetics
  • Indans / pharmacology*
  • Kidney Neoplasms / pathology*
  • Mice
  • Models, Molecular
  • Protein Conformation
  • Rats
  • Structure-Activity Relationship
  • Sulfones / chemistry*
  • Sulfones / pharmacokinetics
  • Sulfones / pharmacology*
  • Tissue Distribution

Substances

  • Antineoplastic Agents
  • Basic Helix-Loop-Helix Transcription Factors
  • Indans
  • PT2385
  • Sulfones
  • endothelial PAS domain-containing protein 1