Lead Optimization of the 5-Phenylpyrazolopyrimidinone NPD-2975 toward Compounds with Improved Antitrypanosomal Efficacy

J Med Chem. 2024 Feb 22;67(4):2849-2863. doi: 10.1021/acs.jmedchem.3c01976. Epub 2024 Feb 8.

Abstract

Human African trypanosomiasis (HAT) still faces few therapeutic options and emerging drug resistance, stressing an urgency for novel antitrypanosomal drug discovery. Here, we describe lead optimization efforts aiming at improving antitrypanosomal efficacy and better physicochemical properties based on our previously reported optimized hit NPD-2975 (pIC50 7.2). Systematic modification of the 5-phenylpyrazolopyrimidinone NPD-2975 led to the discovery of a R4-substituted analogue 31c (NPD-3519), showing higher in vitro potency (pIC50 7.8) against Trypanosoma brucei and significantly better metabolic stability. Further, in vivo pharmacokinetic evaluation of 31c and experiments in an acute T. brucei mouse model confirmed improved oral bioavailability and antitrypanosomal efficacy at 50 mg/kg with no apparent toxicity. With good physicochemical properties, low toxicity, improved pharmacokinetic features, and in vivo efficacy, 31c may serve as a promising candidate for future drug development for HAT.

MeSH terms

  • Animals
  • Antiprotozoal Agents* / therapeutic use
  • Drug Development
  • Humans
  • Mice
  • Trypanocidal Agents* / pharmacology
  • Trypanocidal Agents* / therapeutic use
  • Trypanosoma brucei brucei*
  • Trypanosomiasis, African* / drug therapy

Substances

  • Trypanocidal Agents
  • Antiprotozoal Agents