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Investigational Agent for Primary Biliary Cholangitis Shows Promise

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Upon ursodiol failure, patients with primary biliary cholangitis (PBC) have few treatment options. Alkaline phosphatase (ALP) and bilirubin levels in these patients are associated with the risk of liver transplant or death. A phase 2, multicenter, randomized, double‐blind, placebo‐controlled trial assessing the investigational agent NGM282, an engineered analogue of fibroblast growth factor 19, showed significant improvements in ALP and transaminase levels compared with placebo in patients with PBC. The agent also showed good safety and tolerability.

The 28-day study randomly assigned 45 patients with PBC and an inadequate response to ursodiol to subcutaneous daily doses of NGM282 0.3 mg (n = 14), NGM282 3 mg (n = 16), or placebo (n = 15). At day 28, ALP was significantly reduced with both NGM282 doses versus placebo. Seven of 14 patients (50%) receiving NGM282 0.3 mg and 6 of 13 (46%) receiving NGM282 3 mg achieved a ≥15% reduction in ALP levels from baseline, compared with 1 of 15 patients (7%) receiving placebo. NGM282 also significantly reduced serum concentrations of transaminases and immunoglobulins. Adverse events were mostly grade 1 or 2, with gastrointestinal disorders observed more often with NGM282 treatment. No patients receiving NGM282 had worsening of pruritus.

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Mayo MJ, Wigg AJ, Leggett BA, et al. NGM282 for treatment of patients with primary biliary cholangitis: a multicenter, randomized, double-blind, placebo-controlled trial. Hepatol Commun. 2018;2(9):1037-1050. doi: 10.1002/hep4.1209.

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