Safety and Sustained Efficacy of the Farnesoid X Receptor (FXR) Agonist Cilofexor Over a 96-Week Open-label Extension in Patients With PSC

• Published in: Clinical gastroenterology and hepatology Vol. 21; no. 6; pp. 1552 - 1560.e2
• Main Authors: Trauner, Michael, Bowlus, Christopher L., Gulamhusein, Aliya, Hameed, Bilal, Caldwell, Stephen H., Shiffman, Mitchell L., Landis, Charles, Muir, Andrew J., Billin, Andrew, Xu, Jun, Liu, Xiangyu, Lu, Xiaomin, Chung, Chuhan, Myers, Robert P., Kowdley, Kris V.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.06.2023
• Subjects: Adult; Alkaline Phosphatase; Bile Acids and Salts; Biomarkers; Cholangitis, Sclerosing - drug therapy; Cholestasis; Farnesoid X Receptor; Female; gamma-Glutamyltransferase; Gastroenterology and Hepatology; Humans; Large Duct PSC; Liver; Male; Primary Sclerosing Cholangitis; UDCA; Ursodeoxycholic Acid - therapeutic use; FXR; PSC; AST; TIMP-1; AE; Farnesoid X Receptor; ALP; Large Duct PSC; ALT; OLE; UDCA; IQR; CYP7A1; CK18; C4; GGT; FGF19; SAE; ULN; Primary Sclerosing Cholangitis; PIII-NP; Cholestasis; ELF; BA; aspartate aminotransaminase; Enhanced Liver Fibrosis; interquartile range; 7α-hydroxy-4-cholesten-3-one; gamma-glutamyl transferase; ursodeoxycholic acid; serious adverse event; alkaline phosphatase; fibroblast growth factor 19; cholesterol 7ɑ-hydroxylase; bile acid; adverse event; upper limit of normal; cytokeratin 18; open-label extension; alanine aminotransaminase; tissue inhibitor of metalloproteinase-1; procollagen III N-terminal peptide

Primary sclerosing cholangitis (PSC) is a major unmet medical need in clinical hepatology. Cilofexor is a nonsteroidal farnesoid X receptor agonist being evaluated for the treatment of PSC. Here, we describe the safety and preliminary efficacy of cilofexor in a 96-week, open-label extension (OLE) of...