This segment focuses on the research, development, and commercialization of small molecule drugs targeting central nervous system (CNS) disorders, with a primary focus on Parkinson's disease psychosis and Rett syndrome. Research and development activities include preclinical studies, clinical trials, and the ongoing evaluation of new drug candidates. Technologies and methodologies employed encompass advanced medicinal chemistry, pharmacology, and clinical trial design. The segment's therapeutic areas include Parkinson's disease, Rett syndrome, and other neurological conditions. The patient impact is significant, as these therapies aim to alleviate debilitating symptoms and improve the quality of life for patients suffering from these conditions. Market positioning is strong due to the company's first-to-market advantage with NUPLAZID and DAYBUE, and its focus on unmet medical needs. Future opportunities include expanding the indications for existing drugs and developing new therapies for other CNS disorders. Regulatory and clinical aspects involve navigating FDA approval processes and conducting post-market surveillance. Partnerships and collaborations are essential for clinical trial execution and commercialization efforts.

This segment is dedicated to the development and commercialization of therapies for rare diseases, with a current focus on Rett syndrome. Research and development efforts are centered on identifying and developing novel treatments that address the underlying causes and symptoms of these rare conditions. Technologies and methodologies include advanced drug discovery techniques, preclinical models, and clinical trial design tailored to rare disease populations. The therapeutic area is primarily Rett syndrome, with potential expansion into other rare neurological disorders. The patient impact is substantial, as these therapies aim to provide much-needed treatment options for patients with limited or no existing treatments. Market positioning is enhanced by the company's focus on underserved patient populations and its commitment to developing innovative therapies. Future opportunities include expanding the pipeline with additional rare disease programs and exploring new therapeutic modalities. Regulatory and clinical aspects involve navigating the complexities of rare disease drug development and working closely with regulatory agencies. Partnerships and collaborations are crucial for conducting clinical trials and ensuring access to patients.