Identification and Profiling of a Novel Diazaspiro[3.4]octane Chemical Series Active against Multiple Stages of the Human Malaria Parasite Plasmodium falciparum and Optimization Efforts

J Med Chem. 2021 Feb 25;64(4):2291-2309. doi: 10.1021/acs.jmedchem.1c00034. Epub 2021 Feb 12.

Abstract

A novel diazaspiro[3.4]octane series was identified from a Plasmodium falciparum whole-cell high-throughput screening campaign. Hits displayed activity against multiple stages of the parasite lifecycle, which together with a novel sp3-rich scaffold provided an attractive starting point for a hit-to-lead medicinal chemistry optimization and biological profiling program. Structure-activity-relationship studies led to the identification of compounds that showed low nanomolar asexual blood-stage activity (<50 nM) together with strong gametocyte sterilizing properties that translated to transmission-blocking activity in the standard membrane feeding assay. Mechanistic studies through resistance selection with one of the analogues followed by whole-genome sequencing implicated the P. falciparum cyclic amine resistance locus in the mode of resistance.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Anopheles / drug effects
  • Antimalarials / chemical synthesis
  • Antimalarials / metabolism
  • Antimalarials / pharmacology*
  • Female
  • Germ Cells / drug effects
  • High-Throughput Screening Assays
  • Humans
  • Male
  • Mice
  • Microsomes, Liver / metabolism
  • Molecular Structure
  • Parasitic Sensitivity Tests
  • Plasmodium falciparum / drug effects*
  • Rats
  • Spiro Compounds / chemical synthesis
  • Spiro Compounds / metabolism
  • Spiro Compounds / pharmacology*
  • Structure-Activity Relationship

Substances

  • Antimalarials
  • Spiro Compounds