• Marketing Authorization Application for trofinetide in Rett syndrome submitted to European Medicines Agency
• Potential for approval in Q1 2026
• Acadia anticipates initiating Managed Access Programs in Europe in Q2 2025

Neuren Pharmaceuticals (ASX: NEU) today announced that its partner Acadia Pharmaceuticals (NASDAQ: ACAD) has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for trofinetide for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. Acadia anticipates potential approval in Q1 2026. If granted marketing authorization, trofinetide will be the first and only approved therapy for Rett syndrome in the European Union. Acadia also anticipates initiating Managed Access Programs in Europe in the second quarter of 2025. Trofinetide (DAYBUE™) is already approved in the United States and Canada for Rett syndrome. Under the worldwide licence agreement with Acadia, Neuren is eligible to receive milestone payments and royalties related to development and commercialization of trofinetide outside North America.

Neuren is eligible to receive milestone payments of US$35 million upon first commercial sale of trofinetide for Rett syndrome in Europe and US$15 million upon first commercial sale in Japan. Additional milestone payments of up to US$363 million are also possible upon achievement of escalating annual net sales thresholds in Europe, Japan, and the Rest of World.

If granted marketing authorization, trofinetide will be the first and only approved therapy for Rett syndrome in the European Union. Acadia also anticipates initiating Managed Access Programs in Europe in the second quarter of 2025.