Discovery of MDI-114215: A Potent and Selective LIMK Inhibitor To Treat Fragile X Syndrome

J Med Chem. 2025 Jan 9;68(1):719-752. doi: 10.1021/acs.jmedchem.4c02694. Epub 2024 Dec 22.

Abstract

LIMKs are serine/threonine and tyrosine kinases responsible for controlling cytoskeletal dynamics as key regulators of actin stability, ensuring synaptic health through normal synaptic bouton structure and function. However, LIMK1 overactivation results in abnormal dendritic synaptic development that characterizes the pathogenesis of Fragile X Syndrome (FXS). As a result, the development of LIMK inhibitors represents an emerging disease-modifying therapeutic approach for FXS. We report the discovery of MDI-114215 (85), a novel, potent allosteric dual-LIMK1/2 inhibitor that demonstrates exquisite kinome selectivity. 85 reduces phospho-cofilin in mouse brain slices and rescues impaired hippocampal long-term potentiation in brain slices from FXS mice. We also show that LIMK inhibitors are effective in reducing phospho-cofilin levels in iPSC neurons derived from FXS patients, demonstrating 85 to be a potential therapeutic candidate for FXS that could have broad application to neurological disorders or cancers caused by LIMK1/2 overactivation and actin instability.

MeSH terms

  • Actin Depolymerizing Factors / metabolism
  • Animals
  • Drug Discovery
  • Fragile X Syndrome* / drug therapy
  • Fragile X Syndrome* / metabolism
  • Hippocampus / drug effects
  • Hippocampus / metabolism
  • Humans
  • Induced Pluripotent Stem Cells / drug effects
  • Induced Pluripotent Stem Cells / metabolism
  • Lim Kinases* / antagonists & inhibitors
  • Lim Kinases* / metabolism
  • Long-Term Potentiation / drug effects
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Neurons / drug effects
  • Neurons / metabolism
  • Protein Kinase Inhibitors* / chemical synthesis
  • Protein Kinase Inhibitors* / chemistry
  • Protein Kinase Inhibitors* / pharmacology
  • Protein Kinase Inhibitors* / therapeutic use
  • Structure-Activity Relationship

Substances

  • Lim Kinases
  • Protein Kinase Inhibitors
  • LIMK1 protein, human
  • LIMK2 protein, human
  • Actin Depolymerizing Factors