Neuren is developing new drug therapies to treat multiple serious neurological disorders characterised by impaired connections and signalling between brain cells, that emerge in early childhood and have no or limited approved treatment options. Recognising the urgent unmet need, all programs have been granted “orphan drug” designation in the United States.

DAYBUE™ (trofinetide) is approved by the US Food and Drug Administration (FDA) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Neuren has granted an exclusive worldwide licence to Acadia Pharmaceuticals Inc. for the development and commercialisation of trofinetide.

Neuren’s second drug candidate, NNZ-2591, is in Phase 2 development for each of Phelan-McDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome. Neuren has recently announced positive results in a Phase 2 trial of NNZ-2591 in Phelan-McDermid syndrome and is planning a Phase 3 trial pending consultation with the FDA.