This segment focuses on the research, development, and commercialization of therapies for neurodevelopmental disorders with high unmet medical needs. The primary focus is on Trofinetide (DAYBUE) for Rett syndrome, a debilitating genetic neurological disorder. Research and development activities include clinical trials to evaluate the safety and efficacy of Trofinetide in various patient populations, as well as exploring its potential in other related disorders. The segment also encompasses the development of NNZ-2591 for the treatment of Phelan-McDermid, Angelman, Pitt Hopkins, and Prader-Willi syndromes. The goal is to improve the quality of life for patients and their families by providing effective and targeted treatments. Future opportunities include expanding the indications for existing therapies and developing new innovative treatments for a range of neurodevelopmental conditions.

This segment is dedicated to the discovery and advancement of novel therapeutic candidates for neurological disorders. It involves preclinical studies, clinical trials, and regulatory affairs to bring innovative treatments to market. The R&D efforts are focused on understanding the underlying mechanisms of neurodevelopmental disorders and identifying potential drug targets. Technologies and methodologies used include molecular biology, biochemistry, and advanced imaging techniques. The segment also collaborates with academic institutions and research organizations to accelerate the development of new therapies. The ultimate goal is to create a pipeline of innovative treatments that address unmet medical needs and improve patient outcomes. This segment is crucial for the long-term growth and sustainability of Neuren Pharmaceuticals.