Business Summary

Neuren Pharmaceuticals Limited is an Australia-based biopharmaceutical company that is engaged in developing drugs for neurological disorders. The Company’s lead drug compound, Trofinetide, is in a Phase III clinical trial in the United States for Rett syndrome and has completed a Phase II clinical trial in Fragile X syndrome. Its both programs have each received Fast Track designation by the United States Food and Drug Administration (FDA) and Orphan Drug designation in both the United States and the European Union. The Company has granted a license to Acadia Pharmaceuticals Inc. for the development and commercialization of trofinetide in North America, while retaining all rights outside North America. The Company is preparing to initiate Phase II trials of its second drug candidate, NNZ-2591, for each of PhelanMcDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome.