Delocalized quinolinium-macrocyclic peptides, an atypical chemotype for CNS penetration

Sci Adv. 2024 Jul 12;10(28):eado3501. doi: 10.1126/sciadv.ado3501. Epub 2024 Jul 10.

Abstract

Macrocyclic drugs can address an increasing range of molecular targets but enabling central nervous system (CNS) access to these drugs has been viewed as an intractable problem. We designed and synthesized a series of quinolinium-modified cyclosporine derivatives targeted to the mitochondrial cyclophilin D protein. Modification of the cation to enable greater delocalization was confirmed by x-ray crystallography of the cations. Critically, greater delocalization improved brain concentrations. Assessment of the compounds in preclinical assays and for pharmacokinetics identified a molecule JP1-138 with at least 20 times the brain levels of a non-delocalized compound or those reported for cyclosporine. Levels were maintained over 24 hours together with low hERG potential. The paradigm outlined here could have widespread utility in the treatment of CNS diseases.

MeSH terms

  • Animals
  • Brain / drug effects
  • Brain / metabolism
  • Central Nervous System / drug effects
  • Central Nervous System / metabolism
  • Crystallography, X-Ray
  • Cyclosporine / chemistry
  • Cyclosporine / pharmacokinetics
  • Humans
  • Mice
  • Peptides / chemistry
  • Peptides / pharmacokinetics
  • Quinolinium Compounds* / chemistry
  • Quinolinium Compounds* / pharmacokinetics

Substances

  • Quinolinium Compounds
  • Cyclosporine
  • Peptides