Discovery of Potent and Brain-Penetrant Tau Tubulin Kinase 1 (TTBK1) Inhibitors that Lower Tau Phosphorylation In Vivo

J Med Chem. 2021 May 13;64(9):6358-6380. doi: 10.1021/acs.jmedchem.1c00382. Epub 2021 May 4.

Abstract

Structural analysis of the known NIK inhibitor 3 bound to the kinase domain of TTBK1 led to the design and synthesis of a novel class of azaindazole TTBK1 inhibitors exemplified by 8 (cell IC50: 571 nM). Systematic optimization of this series of analogs led to the discovery of 31, a potent (cell IC50: 315 nM) and selective TTBK inhibitor with suitable CNS penetration (rat Kp,uu: 0.32) for in vivo proof of pharmacology studies. The ability of 31 to inhibit tau phosphorylation at the disease-relevant Ser 422 epitope was demonstrated in both a mouse hypothermia and a rat developmental model and provided evidence that modulation of this target may be relevant in the treatment of Alzheimer's disease and other tauopathies.

MeSH terms

  • Animals
  • Brain / metabolism*
  • Drug Design*
  • Humans
  • Indazoles / chemistry
  • Indazoles / metabolism
  • Indazoles / pharmacology
  • Mice
  • Molecular Targeted Therapy
  • Phosphorylation / drug effects
  • Protein Kinase Inhibitors / chemistry
  • Protein Kinase Inhibitors / metabolism*
  • Protein Kinase Inhibitors / pharmacology*
  • Protein Serine-Threonine Kinases / antagonists & inhibitors*
  • Rats
  • tau Proteins / metabolism*

Substances

  • Indazoles
  • Protein Kinase Inhibitors
  • tau Proteins
  • tau-tubulin kinase
  • Protein Serine-Threonine Kinases