Australian biopharmaceuticals developer Neuren Pharmaceuticals (ASX: NEU) has been granted a patent from the European Patent Office covering NNZ-2591, a world-first drug being developed to treat three neurodevelopmental disorders which affect children, and are currently incurable.
The granting of the new patent is the third jurisdiction to do so for Neuren with the same application previously granted in the United States and Japan. Titled “Neuroprotective bicyclic compounds and methods for their use in treating autism spectrum disorders and neurodevelopmental disorders,” the patent will enable advancing the development of NNZ-2591 which has already delivered encouraging results when applied to mice.
The three neurodevelopmental diseases being targeted by Neuren with NNZ-2591 are Phelen-McDermaid syndrome, Angelman syndrome and Pitt Hopkins syndrome which share underlying impairment of signalling between brain cells.
All three developmental diseases impact the development of a child’s brain which lead to varying levels of intellectual disability and are caused by gene mutations and deletion.
Because the three diseases are so rare, Neuren has been granted Orphan Drug designation from the United States Food & Drug Administration (FDA) which provides Government assistance to drug research for diseases so rare that there would otherwise be no commercial value to conduct the research.
Neuren is currently undertaking the non-clinical studies for NNZ-2591 but anticipate submitting an FDA application to commence clinical trials in 2020.
Beyond the development of NNZ-2591, Neuron is currently undertaking Phase 3 clinical trials for its trofinetide product aimed at treating Rett syndrome, another rare neurological disorder which the Company has attained FDA Orphan Drug and Fast Track designation for.