CN128: A New Orally Active Hydroxypyridinone Iron Chelator

J Med Chem. 2020 Apr 23;63(8):4215-4226. doi: 10.1021/acs.jmedchem.0c00137. Epub 2020 Apr 2.

Abstract

Deferoxamine, deferiprone, and deferasirox are used for the treatment of systemic iron overload, although they possess limitations due to lack of oral activity, lower efficacy, and side effects. These limitations led to a search for an orally active iron chelator with an improved therapeutic index. The lower efficacy of deferiprone is due to rapid glucuronidation, leading to the formation of a nonchelating metabolite. Here, we demonstrate that the influence of metabolism can be reduced by introducing a sacrificial site for glucuronidation. A log P-guided investigation of 20 hydroxpyridinones led to the identification of CN128. The Fe(III) affinity and metal selectivity of CN128 are similar to those of deferiprone, the log P value is more lipophilic, and its iron scavenging ability is superior. Overall, CN128 was demonstrated to be safe in a range of toxicity assessments and is now in clinical trials for the treatment of β-thalassemia after regular blood transfusion.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Animals
  • Dose-Response Relationship, Drug
  • Guinea Pigs
  • Humans
  • Iron Chelating Agents / administration & dosage*
  • Iron Chelating Agents / chemistry*
  • Iron Overload / blood
  • Iron Overload / drug therapy*
  • Mice
  • Pyridones / administration & dosage*
  • Pyridones / chemistry*
  • Rats
  • Rats, Sprague-Dawley
  • Tissue Distribution / drug effects
  • Tissue Distribution / physiology

Substances

  • Iron Chelating Agents
  • Pyridones