Study of OCA in Combination With BZF Evaluating Efficacy, Safety and Tolerability in Participants With PBC

Serum samples were collected at scheduled visits during the double-blind treatment period. Changes in ALP were evaluated using a mixed-effects repeated-measures model (MMRM) to assess the effects of treatment groups over time. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from Baseline was calculated as post Baseline value minus Baseline value.

Responders were defined as participants achieving a ≥10%, ≥20%, ≥30%, or ≥40% reduction from baseline in serum ALP at week 12 during the DB treatment period with non-responder imputation applied. Baseline ALP was defined as the last evaluations prior to the first administration of investigational product. The percentage of responders were summarized by treatment group for each response threshold and compared using a Cochran Mantel Haenszel test stratified by the randomization stratification factor.

Percentage of participants achieving a response in serum ALP at week 12 during the DB treatment period was assessed using non-responder imputation. Analyses of ALP normalization rates were performed using a Cochran-Mantel-Haenszel test stratified by the randomization stratification factor.

Percentage of participants achieving a response in biochemical disease markers including ALP, alanine amino transferase (ALT), aspartate amino transferase (AST), gamma-glutamyl transferase (GGT), total and conjugated bilirubin and lipid panel (cholesterol, high density lipoprotein [HDL] and low density lipoprotein [LDL]) at Week 12 during the DB treatment period has been presented. Analyses of response rates and normalization rates were performed using a Cochran-Mantel-Haenszel test stratified by the randomization stratification factor.

Blood samples were collected at indicated timepoint and Change from Baseline in GGT, ALT and AST were analyzed using the MMRM model and results were summarized in standard International System of Units (SI) by treatment group using descriptive statistics at baseline and at each on-study evaluation. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from baseline was calculated as post Baseline value minus Baseline value.

Blood samples were collected at indicated timepoints and the changes in total and conjugated bilirubin were evaluated using MMRM to assess the effects of treatment groups over time. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from baseline was calculated as post Baseline value minus Baseline value.

Blood samples were collected at indicated timepoints and the changes in lipid panel including cholesterol, HDL and LDL were evaluated using MMRM to assess the effects of treatment groups over time. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from baseline was calculated as post Baseline value minus Baseline value.

Blood samples were collected at indicated timepoints for the assessment of C4. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from baseline was calculated as post Baseline value minus Baseline value.

Blood samples were collected for the assessment of bile acids. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product.