Evaluation of the hepatotoxicity of the novel GPR40 (FFAR1) agonist CPL207280 in the rat and monkey

PLoS One. 2021 Sep 23;16(9):e0257477. doi: 10.1371/journal.pone.0257477. eCollection 2021.

Abstract

GPR40 (FFAR1) is a promising target for the managing type 2 diabetes (T2D). The most advanced GPR40 agonist TAK-875 exhibited satisfactory glucose-lowering effects in phase II and III studies. However, the phase III studies of TAK-875 revealed drug-induced liver injury (DILI). It is unknown whether DILI is a consequence of a specific GPR40 agonist or is an inherent feature of all GPR40 agonists. CPL207280 is a novel GPR40 agonist that improves diabetes in Zucker Diabetic Fatty (ZDF) rats, Goto Kakizaki (GK) rats and db/db mice. In this report, the DILI-related toxicity of CPL207280 was compared directly with that of TAK-875. In vitro studies evaluating hepatic biliary transporter inhibition, mitochondrial function, and metabolic profiling were performed in hepatocytes from different species. The long term toxicity of CPL207280 was studied in vivo in rats and monkeys. Activity of CPL207280 was one order of magnitude lesser than that of TAK-875 for the inhibition of bile acid transporters. CPL207280 had a negligible effect on the hepatic mitochondria. In contrast to TAK-875, which was metabolized through toxic glucuronidation, CPL207280 was metabolized mainly through oxidation. No deleterious hepatic effects were observed in chronically treated healthy and diabetic animals. The study presents promising data on the feasibility of creating a liver-safe GPR40 agonist. Additionally, it can be concluded that DILI is not a hallmark of GPR40 agonists; it is linked to the intrinsic properties of an individual agonist.

MeSH terms

  • Animals
  • Benzofurans
  • Chemical and Drug Induced Liver Injury / metabolism
  • Haplorhini
  • Hepatocytes / drug effects
  • Hepatocytes / metabolism
  • Humans
  • Liver / drug effects
  • Liver / metabolism
  • Liver / pathology
  • Macaca fascicularis
  • Male
  • Mice
  • Rats
  • Rats, Zucker
  • Receptors, G-Protein-Coupled* / agonists
  • Receptors, G-Protein-Coupled* / metabolism
  • Sulfones / pharmacology

Substances

  • Receptors, G-Protein-Coupled
  • TAK-875
  • Sulfones
  • G-protein-coupled receptor 40, rat
  • FFAR1 protein, human
  • Benzofurans

Grants and funding

The study was co-financed by Celon Pharma SA and the National Center of Research and Development grant number “POIR.01.01.01.-00-0334/17” (awarded: MW) on behalf of European Regional Development Fund. The funder provided support in the form of salaries for authors KB-G, PB, MM, JP, MJ, ED, IK, JD, HZ, MW but did not have any additional role in the study design, data collection and analysis, decision to publish, or preparation of the manuscript. The specific roles of these authors are articulated in the ‘author contributions’ section. Study was created under the contract with the Ministry of Science and Higher Education No. 50//DW/2017/01/1 as a part of the "Implementation doctorate" program (awarded: KB-G). Gad Consulting Services had no role in funding of the study, SCG affiliated to it merely provided the external scientific expertise.