Our pipeline consists of two clinical-stage product candidates with mechanisms of action that have potential utility across a wide range of rare liver diseases. We are advancing our lead candidate, maralixibat for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) — conditions with a profound and devastating impact on quality of life for young patients and their families.
If left untreated, these conditions can lead to liver failure and inflict life-altering symptoms including stunted growth and severe, non-stop itching. Under current standard of care, patients often require a liver transplant and are at higher risk of many serious health issues. Working closely with patients and caregiver communities, we intimately understand the challenges of living with cholestatic liver diseases.
Based on a wealth of data from clinical trials spanning multiple years and involving more than one hundred patients, we believe our approach can address the underlying cause of symptoms and liver damage. Our goal is to give families their lives back.