Ratziu V, Rinella ME, Neuschwander-Tetri BA, et al. - EDP-305 (an oral farnesoid X receptor agonist) resulted in reduction in alanine aminotransferase (ALT) levels and magnetic resonance imaging-proton density fat fraction when used for treating nonalcoholic steatohepatitis (NASH). Thus, further development of EDP-305 in patients with NASH with longer term studies is supported.

• A phase 2, randomized, double-blind, placebo-controlled study in which 134 non-cirrhotic patients with fibrotic NASH were randomized to EDP-305 1 mg, EDP-305 2.5 mg, or placebo, for 12 weeks, and 132 were evaluated.

• With 2.5 mg EDP-305 and 1 mg EDP-305, the least squares mean reduction at Week 12 from baseline for ALT was -27.9 U/L and -21.7 U/L, respectively, vs -15.4 U/L with placebo.

• With these doses, absolute liver fat reduction was -7.1% and -3.3%, respectively, vs -2.4% with placebo.

• Patients most commonly (≥5%) suffered pruritus, nausea, vomiting, diarrhea, headache, and dizziness as adverse events.