. 2019 Aug 9;1(3):203–213. doi: 10.1016/j.jhepr.2019.05.005

Table 2.

Main published studies (or presented with a minimum 52 week-follow-up) of drugs in development for PBC.

Main Target	Agent	Study design	Inclusion criteria	Duration	No. patients	Main results
Immunity	Ustekinumab⁶⁷	Open-label	Inadequate response to UDCA	28 wk	20	Modest decrease in ALP (-12%)
Immunity	Rituximab (1,000 mg x2)⁶⁵	Open-label	Inadequate response to UDCA	6-10 mo	14	Modest decrease in ALP (-16%) at 6 months
Immunity	Rituximab (1,000 mg x2)⁶⁶	Randomised Placebo-controlled phase II	Moderate or severe fatigue	12 mo	57	No improvement in fatigue score
Cholestasis	Seladelpar (50 or 200 mg/d)⁶⁸	Randomised Placebo-controlled phase II	Inadequate response to UDCA	12 wk	70	-53% to -63% decrease in ALP 3 grade-3 ALT increases
Cholestasis	Seladelpar (5-10 or 10 mg/d)⁶⁹	Randomised dose ranging phase II	Inadequate response to UDCA	52 wk	119 (results in 34)	-47% and -46% decrease in ALP No ALT flare
Cholestasis	NGM282⁷²	Randomised Placebo-controlled phase II	Inadequate response to UDCA	4 wk	45	-16% to 19% decrease in ALP Diarrhoea in 1/4
Cholestasis	GSK2330672⁷⁶	Placebo-controlled, cross-over, phase II	Pruritus	2 wk	22	Significant decrease in itch scores Diarrhoea in 1/3

ALP, alkaline phosphatase; ALT, alanine aminotransferase; PBC, primary biliary cholangitis; UDCA, ursodeoxycholic acid; ULN, upper limit of normal.