Design and Preclinical Characterization Program toward Asundexian (BAY 2433334), an Oral Factor XIa Inhibitor for the Prevention and Treatment of Thromboembolic Disorders

J Med Chem. 2023 Sep 14;66(17):12203-12224. doi: 10.1021/acs.jmedchem.3c00795. Epub 2023 Sep 5.

Abstract

Activated coagulation factor XI (FXIa) is a highly attractive antithrombotic target as it contributes to the development and progression of thrombosis but is thought to play only a minor role in hemostasis so that its inhibition may allow for decoupling of antithrombotic efficacy and bleeding time prolongation. Herein, we report our major efforts to identify an orally bioavailable, reversible FXIa inhibitor. Using a protein structure-based de novo design approach, we identified a novel micromolar hit with attractive physicochemical properties. During lead modification, a critical problem was balancing potency and absorption by focusing on the most important interactions of the lead series with FXIa while simultaneously seeking to improve metabolic stability and the cytochrome P450 interaction profile. In clinical trials, the resulting compound from our extensive research program, asundexian (BAY 2433334), proved to possess the desired DMPK properties for once-daily oral dosing, and even more importantly, the initial pharmacological hypothesis was confirmed.

MeSH terms

  • Anticoagulants
  • Benzamides* / chemistry
  • Benzamides* / pharmacology
  • Benzamides* / therapeutic use
  • Factor XIa*
  • Fibrinolytic Agents*
  • Hydrocarbons, Fluorinated* / chemistry
  • Hydrocarbons, Fluorinated* / pharmacology
  • Hydrocarbons, Fluorinated* / therapeutic use
  • Triazoles* / chemistry
  • Triazoles* / pharmacology
  • Triazoles* / therapeutic use

Substances

  • Anticoagulants
  • Factor XIa
  • Fibrinolytic Agents
  • asundexian
  • Benzamides
  • Hydrocarbons, Fluorinated
  • Triazoles