Sustained Efficacy and Safety of Seladelpar for up to 36 Months in Patients with Primary Biliary Cholangitis from the Placebo-Controlled Response Study to the Open-Label Assure Study
Recommended Citation
Schwenken M, Levy C, Hirschfield GM, Lawitz EJ, Trivedi PJ, Kowdley KV, Bowlus CL, Villamil AM, Kwon KM, Proehl S, Qi X, Crittenden DB, Gordon SC. Sustained Efficacy and Safety of Seladelpar for up to 36 Months in Patients with Primary Biliary Cholangitis from the Placebo-Controlled Response Study to the Open-Label Assure Study. Innere Med 2026; 67(SUPPL2):135-136.
Document Type
Conference Proceeding
Publication Date
4-13-2026
Publication Title
Innere Med
Keywords
General & Internal Medicine
Abstract
Introduction: Seladelpar (SEL) is a first-in-class delpar (selective peroxisome proliferator-activated receptor delta [PPAR6] agonist) indicated for the treatment of primary biliary cholangitis (PBC) combined with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in patients (pts) intolerant to UDCA. In the Phase 3, randomized, placebo (PBO)-controlled RESPONSE study (NCT04620733), SEL significantly reduced biochemical markers of cholestasis and pruritus. After completing RESPONSE, pts were eligible to enroll in ASSURE (NCT03301506), an ongoing, open-label, Phase 3 trial. Here, we report interim, long-term efficacy and safety data for pts enrolled in RESPONSE and upon rollover to ASSURE with up to 3 yrs of data. Methods: Data were analyzed from RESPONSE and ASSURE up to 1/31/2025. Pts with PBC with an inadequate response or intolerance to UDCA and ALP ~ 1.67 x the upper limit of normal (ULN) received blinded, daily oral SEL 10 mg or PBO in RESPONSE; pts who enrolled in ASSURE received open-label, daily oral SEL 10 mg. Pts were analyzed based on receipt of SEL (continuous SEL) or PBO (crossover SEL) in RESPONSE. Efficacy endpoints included a composite biochemical response (CBR; ALP < 1.67 x ULN, ALP decrease ~ 15%, and total bilirubin s ULN), ALP normalization, ALT normalization (among pts with ALT> ULN at baseline [BL]), and ALP percent change from BL. Exposure-adjusted adverse events (AEs) were analyzed. Results: Of 193 pts enrolled in RESPONSE (SEL, n = 128; PBO, n = 65), 158 pts (SEL, n = 104; PBO, n = 54) enrolled in ASSURE. As of the cutoff date, 94% of continuous SEL pts (98/104) received SEL for ~ 2 yrs and 27% (28/104) received SEL for~ 3 yrs; 24% of crossover SEL pts (13/54) received SEL for~ 2 yrs. Among continuous SEL pts, the CBR, ALP normalization, and ALT normalization rates were 69% (20/29), 24% (7/29), and 56% (10/18) through 36 months (M). Among crossover SEL pts, the CBR rate at 6M was 74% (40/54) and remained similar through 24M (85% [11/13]). Among crossover SEL pts, ALP normalized in 26% of pts (14/54) at 6M and 54% of pts (7/13) at 24M; ALP percent change from BL was also assessed (Figure). In total, 71% (20/28) achieved ALT normalization at 6M and 75% (3/4) at 24M. Exposure-adjusted pt incidence of AEs was 90.9 per 100 pt-yrs for SEL vs 91-4 for PBO in RESPONSE; rates were generally similar or lower in yrs 2 and 3 of SEL exposure in ASSURE. Conclusion: Biochemical efficacy and safety with SEL in the pivotal RESPONSE study was sustained up to 36M in the open-label ASSURE study.
Volume
67
Issue
SUPPL2
First Page
135
Last Page
136
