This segment focuses on the research, development, and commercialization of novel therapeutics for rare diseases with high unmet medical needs. The primary focus is on developing treatments for conditions like Friedreich's ataxia (FA) and Alport syndrome. Research and Development activities include the investigation of small molecule activators of the Nrf2 pathway, such as omaveloxolone (for FA) and bardoxolone methyl (for Alport syndrome). Technologies and methodologies include advanced medicinal chemistry, clinical trial design, and biomarker development. Patient impact is significant, as these therapies aim to slow disease progression and improve the quality of life for patients with debilitating conditions. Market positioning is driven by the unmet need in rare disease treatment and the potential for first-in-class or best-in-class therapies. Future opportunities include expanding the application of Nrf2 activators to other rare diseases and exploring combination therapies. Regulatory and clinical aspects involve navigating the FDA approval process and conducting rigorous clinical trials to demonstrate safety and efficacy. Partnerships and collaborations with Biogen and Kyowa Kirin Co., Ltd. support the development and commercialization efforts.

This segment is dedicated to developing innovative therapies for various forms of chronic kidney disease (CKD). Research and development efforts are focused on identifying and validating novel therapeutic targets and developing small molecule drugs to address the underlying causes of CKD. The company is actively involved in Phase 3 clinical trials for bardoxolone methyl in patients with CKD caused by Alport syndrome, as well as for a form of pulmonary arterial hypertension associated with connective tissue disease. Technologies and methodologies include advanced drug discovery, clinical trial design, and biomarker development. The therapeutic areas covered include IgA nephropathy, type 1 and type 2 diabetic CKD, hypertensive CKD, and focal segmental glomerulosclerosis. The patient impact is significant, as these therapies aim to slow the progression of CKD and reduce the need for dialysis or kidney transplantation. Market positioning is based on the large and growing prevalence of CKD and the potential for disease-modifying therapies. Future opportunities include expanding the CKD pipeline to include additional indications and exploring combination therapies. Regulatory and clinical aspects involve conducting large-scale clinical trials to demonstrate safety and efficacy and obtaining regulatory approvals. Partnerships and collaborations with Kyowa Kirin Co., Ltd. support the development and commercialization efforts in specific geographic regions.