Dravet syndrome (DS) is a catastrophic epileptic encephalopathy. It typically presents around 5–8 months of age in previously well children. Approximately 85% of DS cases are due to heterozygous loss-of-function mutations in the sodium channel alpha-1 (SCN1A) gene encoding voltage-gated sodium channel type 1 (NaV1.1). It is hypothesised that a pharmacological agent that enhances NaV1.1 activity should provide effective therapy in DS. Researchers at The University of Queensland (UQ) in collaboration with the Florey Institute of Neuroscience & Mental Health have developed novel peptide agonists as a potential targeted therapy for DS. Electrophysiology assays show that the two lead peptides Hm1a and Hm1b are selective for NaV1.1 over other sodium and potassium channels.

Key features

  • Peptide-based agonist of voltage-gated sodium channel type 1 (NaV1.1)
  • Potent and selective for NaV1.1 over other NaV isoforms
  • Proof of concept data in mouse Dravet syndrome model
  • Potential for use in other indications
  • First-in-class disease-modifying opportunity.

Applications

Therapeutic applications of the NaV1.1 agonist include:

  • Dravet syndrome (primary indication)
  • Other SC1A-associated epilepsies
  • Schizophrenia, and
  • Alzheimer’s disease.

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