Motoneurone Disease Stroke Peripheral Neuropathy Multiple Sclerosis Lennox-Gastaut Eye Diseases
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Initial Discovery

  1. The first family member of the Neural Regeneration Peptides (NRPs) was discovered by Dr Frank Sieg during an in vitro screening effort using rat central nervous system (CNS) tissue aimed at the identification of novel neuronal migration inducing factors.
    1. Successful purification to homogeneity of the newly identified compound, named NRP, confirmed that the factor is a small peptide secreted by cells located within the central nervous system.
    2. Apart from the induction of neuronal migration, neuronal cell-dividing stimulating properties as well as neuronal differentiation and survival-promoting activities could be contributed to this exciting new molecule.
  2. Bioinformatic-based searches for similar genes occurring within the human genome revealed various gene sequences with domain homology to the initially identified rat NRP.

NRP forming new neuronal tissue

The above diagram shows thalamic CNS neurons expressing the calcium binding protein parvalbumin (green) are migrating and forming new neuronal tissue (red) 24 - 48hrs after contact with NRPs. Arrows indicate double-localization of both thalamus-typical proteins, parvalbumin (green) and MAP-2 (red) are neuronal protein markers.

Pre-clinical Development Completed

  1. Synthesis of small length peptide mimetics with optimized activities regarding peptide stability and activity led to the selection of the current lead compound.
  2. Successful selection of the lead with superior physico-chemical stability with preserved potency and bioactivity.
  3. Successful formulation of lead compound regarding stability as well as suitability for peripheral administration routes.
  4. The selected lead compound has to date shown highly effective therapeutic pharmacodynamic activities within animal models mimicking acute and refractory epilepsy, Motorneurone Disease, sever retinal diseases and peripheral neuropathy.
  5. Moreover, NRP compounds have shown preferential activities in Multiple Sclerosis and stroke animal models.
  6. Successful Rat Toxicology completed
  7. Dog Toxicolgy completed
  8. CMC Pathway completed
  9. Potential Biomarker identified

Clinical Development

  • Clinical Phase I study in healthy volunteers initiated in March 2017 in Melbourne Australia and completion date by end of 2017
  • IND filing early 2018
  • Clinical Phase II in Lennox Gastaut Syndrome children planned for 2018

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Background to the Discovery Drug Platform Collaborations Target Diseases Publications