Neuren Pharmaceuticals Ltd

Neuren Pharmaceuticals Limited is an Australia-based biopharmaceutical company developing therapies for neurodevelopmental disorders. The Company is developing new drug therapies to treat multiple serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. The Company’s product DAYBUE (trofinetide) is used to treat Rett syndrome in adults and pediatric patients two years of age and older. The United States (US) Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted orphan drug designation to the Company for trofinetide in Fragile X syndrome. Its second product, NNZ-2591, is in phase II clinical trials in Phelan-McDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome. The Company has granted an exclusive worldwide license to Acadia Pharmaceuticals Inc. for the development and commercialization of NNZ-259 for Rett syndrome and Fragile X syndrome.