This segment focuses on the development and commercialization of therapies for rare liver diseases affecting children, with a primary emphasis on cholestatic conditions. The lead product, Bylvay (odevixibat), is a potent and selective inhibitor of the ileal bile acid transporter (IBAT) and is approved for the treatment of progressive familial intrahepatic cholestasis (PFIC). Research and development efforts are directed towards expanding the use of Bylvay to other cholestatic liver diseases, such as biliary atresia and Alagille syndrome. Clinical trials are conducted to evaluate the safety and efficacy of Bylvay in these patient populations. The segment aims to improve the quality of life for children with rare liver diseases by providing effective and targeted therapies.

This segment is dedicated to the discovery and development of novel therapies for adult liver and gastrointestinal diseases. The pipeline includes A3907, a compound in Phase I clinical trial for the treatment of adult liver diseases, and A2342, a preclinical candidate targeting adult viral and liver diseases. Research and development activities focus on identifying and validating new drug targets, developing innovative drug candidates, and conducting preclinical and clinical studies to evaluate their safety and efficacy. The segment leverages advanced technologies and methodologies to develop targeted therapies that address unmet medical needs in adult liver and gastrointestinal diseases. The goal is to provide effective treatment options for patients with these conditions and improve their overall health outcomes.