Cilofexor is a small molecule nonsteroidal agonist of farnesoid X receptor (FXR). K. Patel et al. (University of Toronto, Canada) evaluated the safety and efficacy of cilofexor in patients with NASH in a double-blind, placebo-controlled, phase 2 trial. 140 non-cirrhotic patients with NASH were randomized to receive cilofexor 100 mg, 30 mg, or placebo orally once daily for 24 weeks.
At week 24, patients receiving cilofexor 100 mg had a median relative decrease in MRI-PDFF of -22.7%, compared with an increase of 1.9% in those receiving placebo.
Serum gamma-glutamyltransferase, C4, and primary bile acids decreased significantly at week 24 in both cilofexor treatment groups, whereas significant changes in ELF and liver stiffness were not observed. Cilofexor was generally well tolerated. Moderate to severe pruritus was more common in patients receiving cilofexor 100 mg than in those receiving cilofexor 30 mg and placebo.