RESPONSE Study Overview

RESPONSE: A phase 3 trial1

The efficacy of LIVDELZI was evaluated in the RESPONSE trial, a 12-month, randomized, double-blind, placebo-controlled trial. The study included 193 adult patients with primary biliary cholangitis (PBC) with an inadequate response or intolerance to ursodeoxycholic acid (UDCA). Patients were included in the trial if their alkaline phosphatase (ALP) was greater than or equal to 1.67-times upper limit of normal (ULN) and total bilirubin (TB) was less than or equal to 2-times ULN. Patients were excluded from the trial if they had other chronic liver diseases, clinically important hepatic decompensation including portal hypertension with complications, or cirrhosis with complications (eg, Model for End-Stage Liver Disease [MELD] score of 12 or greater, known esophageal varices or history of variceal bleeds, history of hepatorenal syndrome).2

Patients were randomized to receive LIVDELZI 10 mg (n=128) or placebo (n=65) once daily for 12 months. LIVDELZI or placebo was administered in combination with UDCA in 181 (94%) patients during the trial, or as a monotherapy in 12 (6%) patients who were unable to tolerate UDCA.2

Table showing select demographics and baseline characteristics of patients in the RESPONSE trial in the LIVDELZI and placebo arms. The median age of patients in each arm was approximately 57 years, and exactly 93.8% of patients in each arm were treated with ursodeoxycholic acid, or UDCA, at baseline.Table showing select demographics and baseline characteristics of patients in the RESPONSE trial in the LIVDELZI and placebo arms. The median age of patients in each arm was approximately 57 years, and exactly 93.8% of patients in each arm were treated with ursodeoxycholic acid, or UDCA, at baseline.

ALT=alanine transaminase; AST=aspartate aminotransferase; F=female; GGT=gamma-glutamyltransferase; M=male; NRS=Numerical Rating Scale; SD=standard deviation; TB=total bilirubin.

  • Compensated cirrhosis allowed2

RESPONSE study design1,2

Diagram depicting the study design of the RESPONSE trial, a placebo-­controlled, randomized, phase 3 study evaluating the efficacy and safety of LIVDELZI 10 mg oral once daily ± ursodeoxycholic acid, or UDCA, versus placebo once daily ± UDCA. The primary endpoint was a biochemical response assessed at month 12, and defined as: • Alkaline phosphatase (ALP) levels <1.67 times the upper limit of normal (ULN) • A decrease in ALP levels of at least 15% from baseline • Normalization of total bilirubin serum levelsDiagram depicting the study design of the RESPONSE trial, a placebo-­controlled, randomized, phase 3 study evaluating the efficacy and safety of LIVDELZI 10 mg oral once daily ± ursodeoxycholic acid, or UDCA, versus placebo once daily ± UDCA. The primary endpoint was a biochemical response assessed at month 12, and defined as: • Alkaline phosphatase (ALP) levels <1.67 times the upper limit of normal (ULN) • A decrease in ALP levels of at least 15% from baseline • Normalization of total bilirubin serum levels

NRS=Numerical Rating Scale.

§The NRS is a scale ranging from 0 (no itch) to 10 (worst itch imaginable).2

References: 1. Hirschfield GM, Bowlus CL, Mayo MJ, et al. A phase 3 trial of seladelpar in primary biliary cholangitis. N Engl J Med. 2024;390(9):783-794. doi:10.1056/NEJMoa2312100 2. LIVDELZI [prescribing information]. Foster City, CA: Gilead Sciences, Inc.; 2024.