NLY01 is a pegylated analogue of Glucagon Like Peptide Receptor (GLP-1R) agonist exendin-4. NLY01 penetrates the blood–brain barrier (BBB) in mice and its receptor (GLP-1R) is highly expressed on glial, but not neuronal cell.

The neuroprotective potential of NLY01 was studied in two mouse models of Parkinson’s disease (PD):

in α –syn PFF (α –synuclein pre-formed fibrils) model of sporadic Parkinson’s disease, and in a progressive, lethal constitutive α -synucleinopathy hA53T transgenic mouse model.

In both models NLY01 demonstrates unprecedented efficacy  manifested by:

  • Inhibition of secretion of neuroinflammatory cytokines by microglial cells
  • Prevention of formation of neurotoxic A1 astrocytes
  • limiting neuronal cell death both in vitro and in vivo
  • Limiting loss of motor function
  • Dramatic increase in survival of hA53T mice

NLY01 belongs to GLP-1R agonists, a class of drugs with well know safety profile approved for treatment of Type 2 diabetes. Combined with the overwhelming pre-clinical efficacy data in various models of neurodegenerative diseases and proven mechanism of action, NLY01 is expected to be potentially the first / best in class disease modifying agent with low development risks and fast path to the clinic.

NLY01 is currently in final stages of pre-clinical testing.