Design, Synthesis, and Evaluation of a Novel Series of Indole Sulfonamide Peroxisome Proliferator Activated Receptor (PPAR) α/γ/δ Triple Activators: Discovery of Lanifibranor, a New Antifibrotic Clinical Candidate

J Med Chem. 2018 Mar 22;61(6):2246-2265. doi: 10.1021/acs.jmedchem.7b01285. Epub 2018 Feb 27.

Abstract

Here, we describe the identification and synthesis of novel indole sulfonamide derivatives that activate the three peroxisome proliferator activated receptor (PPAR) isoforms. Starting with a PPARα activator, compound 4, identified during a high throughput screening (HTS) of our proprietary screening library, a systematic optimization led to the discovery of lanifibranor (IVA337) 5, a moderately potent and well balanced pan PPAR agonist with an excellent safety profile. In vitro and in vivo, compound 5 demonstrated strong activity in models that are relevant to nonalcoholic steatohepatitis (NASH) pathophysiology suggesting therapeutic potential for NASH patients.

MeSH terms

  • Animals
  • Benzothiazoles / chemical synthesis*
  • Benzothiazoles / pharmacokinetics
  • Benzothiazoles / pharmacology*
  • Carbon Tetrachloride Poisoning / drug therapy
  • Cell Line
  • Drug Discovery
  • Fibrosis / prevention & control*
  • Hepatocytes / drug effects
  • High-Throughput Screening Assays
  • Humans
  • Hypoglycemic Agents / chemical synthesis
  • Hypoglycemic Agents / pharmacology
  • Indoles / chemical synthesis*
  • Indoles / pharmacokinetics
  • Indoles / pharmacology*
  • Mice
  • Mice, Inbred C57BL
  • Models, Molecular
  • Molecular Structure
  • Non-alcoholic Fatty Liver Disease / drug therapy
  • Peroxisome Proliferator-Activated Receptors / agonists*
  • Rats
  • Rats, Sprague-Dawley
  • Structure-Activity Relationship
  • Sulfonamides / chemical synthesis*
  • Sulfonamides / pharmacokinetics
  • Sulfonamides / pharmacology*

Substances

  • Benzothiazoles
  • Hypoglycemic Agents
  • Indoles
  • Peroxisome Proliferator-Activated Receptors
  • Sulfonamides
  • lanifibranor