FDA grants Orphan Drug Designation for Neuren’s NNZ-2591 to treat Angelman, Phelan-McDermid and Pitt Hopkins syndromes

MELBOURNE, Australia–(BUSINESS WIRE)–Neuren Pharmaceuticals (ASX: NEU) today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Neuren’s NNZ-2591 for the treatment of Angelman syndrome, Phelan-McDermid syndrome and Pitt Hopkins syndrome.

Neuren Executive Chairman Richard Treagus commented: “Orphan Drug Designation is an important commercial milestone which adds significant value and momentum in respect of our plans to move NNZ-2591 into clinical trials in 2020. This confirms that NNZ-2591 is a valuable asset for Neuren and validates the encouraging preclinical data as well as our plans to develop NNZ-2591 in parallel for these three debilitating childhood disorders with urgent unmet need. We look forward to working with the FDA as we execute on our development plans.”

Neuren previously announced positive results for NNZ-2591 in separate mouse models for Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome, three debilitating neurodevelopmental disorders for which currently there are no approved drug therapies. Each disorder is caused by a mutation or deletion in a different gene; however, they share an underlying impairment in the connections and signaling between brain cells, which is the target for treatment with NNZ-2591.