Discovery of the Soluble Guanylate Cyclase Activator Runcaciguat (BAY 1101042)

J Med Chem. 2021 May 13;64(9):5323-5344. doi: 10.1021/acs.jmedchem.0c02154. Epub 2021 Apr 19.

Abstract

Herein we describe the discovery, mode of action, and preclinical characterization of the soluble guanylate cyclase (sGC) activator runcaciguat. The sGC enzyme, via the formation of cyclic guanosine monophoshphate, is a key regulator of body and tissue homeostasis. sGC activators with their unique mode of action are activating the oxidized and heme-free and therefore NO-unresponsive form of sGC, which is formed under oxidative stress. The first generation of sGC activators like cinaciguat or ataciguat exhibited limitations and were discontinued. We overcame limitations of first-generation sGC activators and identified a new chemical class via high-throughput screening. The investigation of the structure-activity relationship allowed to improve potency and multiple solubility, permeability, metabolism, and drug-drug interactions parameters. This program resulted in the discovery of the oral sGC activator runcaciguat (compound 45, BAY 1101042). Runcaciguat is currently investigated in clinical phase 2 studies for the treatment of patients with chronic kidney disease and nonproliferative diabetic retinopathy.

MeSH terms

  • Animals
  • Binding Sites
  • Crystallography, X-Ray
  • Cytochrome P-450 CYP3A / chemistry
  • Cytochrome P-450 CYP3A / metabolism
  • Dogs
  • Drug Design*
  • Enzyme Activators / chemistry*
  • Enzyme Activators / metabolism
  • Enzyme Activators / pharmacology
  • Enzyme Activators / therapeutic use
  • Half-Life
  • Heart Rate / drug effects
  • Hemodynamics / drug effects
  • Hypertension / drug therapy
  • Hypertension / pathology
  • Molecular Dynamics Simulation
  • Rats
  • Rats, Inbred SHR
  • Solubility
  • Soluble Guanylyl Cyclase / chemistry*
  • Soluble Guanylyl Cyclase / metabolism
  • Structure-Activity Relationship

Substances

  • Enzyme Activators
  • Cytochrome P-450 CYP3A
  • Soluble Guanylyl Cyclase