Discovery of 6-Phenylhexanamide Derivatives as Potent Stereoselective Mitofusin Activators for the Treatment of Mitochondrial Diseases

J Med Chem. 2020 Jul 9;63(13):7033-7051. doi: 10.1021/acs.jmedchem.0c00366. Epub 2020 Jun 18.

Abstract

Mutations in the mitochondrial fusion protein mitofusin (MFN) 2 cause the chronic neurodegenerative condition Charcot-Marie-Tooth disease type 2A (CMT2A), for which there is currently no treatment. Small-molecule activators of MFN1 and MFN2 enhance mitochondrial fusion and offer promise as therapy for this condition, but prototype compounds have poor pharmacokinetic properties. Herein, we describe a rational design of a series of 6-phenylhexanamide derivatives whose pharmacokinetic optimization yielded a 4-hydroxycyclohexyl analogue, 13, with the potency, selectivity, and oral bioavailability of a preclinical candidate. Studies of 13 cis- and trans-4-hydroxycyclohexyl isostereomers unexpectedly revealed functionality and protein engagement exclusively for the trans form, 13B. Preclinical absorption, distribution, metabolism, and excretion (ADME) and in vivo target engagement studies of 13B support further development of 6-phenylhexanamide derivatives as therapeutic agents for human CMT2A.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amides / chemistry*
  • Amides / pharmacokinetics
  • Amides / pharmacology*
  • Amides / therapeutic use
  • Animals
  • Drug Design*
  • GTP Phosphohydrolases / metabolism*
  • Mice
  • Mitochondrial Diseases / drug therapy*
  • Mitochondrial Membrane Transport Proteins / metabolism*
  • Mitochondrial Proteins / metabolism*
  • Stereoisomerism
  • Substrate Specificity
  • Tissue Distribution

Substances

  • Amides
  • Mitochondrial Membrane Transport Proteins
  • Mitochondrial Proteins
  • GTP Phosphohydrolases
  • MFN2 protein, human
  • Mfn1 protein, human
  • caproamide