Neuren Pharmaceuticals (ASX: NEU) is a leading company focused on developing innovative therapies for severe neurodevelopmental disorders that affect young children. These disorders are characterized by impaired connections and signaling between brain cells, leading to profound developmental challenges. With an extensive background in neurodevelopmental disorder therapy, Neuren has made significant strides in its clinical development.

Its flagship product, DAYBUE™ (trofinetide), has received approval from the US FDA for the treatment of Rett syndrome, making it the first and only drug approved for this condition. In addition, Neuren's pipeline includes NNZ-2591, a promising drug currently in Phase 2 trials for Phelan-McDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome, and Prader-Willi syndrome. These disorders currently have no approved treatments, and Neuren's innovative approach holds great promise.

As a testament to the importance of Neuren's work, both the FDA and the EMA have granted orphan drug designation for trofinetide and NNZ-2591, recognizing the critical need for therapeutically important drugs in these conditions. With a deep commitment to improving the lives of patients and their families, Neuren Pharmaceuticals is at the forefront of neurodevelopmental disorder therapy, offering hope and groundbreaking solutions where none currently exist