Neuren Pharmaceuticals Limited is an Australia-based biopharmaceutical company. The Company is developing new drug therapies to treat multiple serious neurological disorders that are caused by genetic abnormalities or brain injury and have no or limited approved treatment options. Its product DAYBUE (trofinetide) is used to treat Rett syndrome in adults and pediatric patients two years of age and older. Its other product, NNZ-2591, is an investigational medicine in Phase III clinical trial for Phelan-McDermid syndrome. Its NNZ-2591 other programs include Pitt Hopkins syndrome, Angelman syndrome, Prader-Willi Syndrome and SYNGAP1-related disorder. The United States Food and Drug Administration (FDA) and European Medicines Agency have both granted Orphan drug designation for trofinetide in Rett syndrome and Fragile X syndrome and for NNZ-2591. The Company has granted a worldwide license to Acadia Pharmaceuticals Inc. for the development and commercialization of trofinetide.