OP-1 LONG-TERM EFFICACY AND SAFETY OF OPEN-LABEL SELADELPAR TREATMENT IN PATIENTS WITH PRIMARY BILIARY CHOLANGITIS: INTERIM 2-YEAR RESULTS FROM THE ASSURE STUDY

Yes, Full disclosures sent separately. Seladelpar reduces biochemical markers of cholestasis and pruritus in patients with primary biliary cholangitis. ASSURE (NCT03301506) is an ongoing, open-label, long-term Phase 3 trial of seladelpar in patients rolling over from Phase 3 RESPONSE (NCT04620733) or legacy studies (NCT03602560, NCT02955602, NCT03301506, and NCT04950764). We report interim 2-year efficacy and safety results. Patients with insufficient response/intolerance to ursodeoxycholic acid could enroll in ASSURE. Key endpoints were composite biochemical response (alkaline phosphatase [ALP] <1.67 × upper limit of normal [ULN], ALP decrease ≥15%, and total bilirubin ≤ULN) and ALP normalization. Pruritus was measured using numerical rating scale (NRS; 0–10). For patients enrolling from RESPONSE, baseline was entry to RESPONSE and analyzed as continuous seladelpar or crossover from placebo; legacy patients were analyzed separately with baseline defined as entry to ASSURE. As of 01/2024, 158 RESPONSE and 179 legacy patients received seladelpar 10 mg daily for up to 155 weeks. In RESPONSE, 61.7% of patients met the endpoint at 12 months (M) vs 20% for placebo. In ASSURE, 61.8% (6M) and 72.4% (12M) met the composite endpoint; 75% (6M) and 93.8% (12M) of placebo crossover patients met the endpoint. In RESPONSE, ALP normalized in 25% of seladelpar and 0 placebo patients at 12M. With continued treatment, 33.3% (6M) and 17.2% (12M) had ALP normalization; 26.9% (6M) and 50% (12M) of crossover patients had ALP normalization. In ASSURE, 6-month change from baseline in pruritus NRS was similar to RESPONSE: −3.8 and −3.7 in continuous and crossover patients, respectively. At 12M and 24M, 73.2% and 69.7% of legacy patients met the endpoint in ASSURE; 42.1% and 42.4% achieved ALP normalization, and reduction in pruritus NRS was −3.8 and −3.1, respectively. There were no treatment-related serious adverse events. Seladelpar treatment led to improvements in biochemical markers and pruritus, and was well tolerated with long-term use.

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Journal
Annals of Hepatology
Published
2024-12-01
DOI
https://doi.org/10.1016/j.aohep.2024.101599
Citations
1
Primary Topic
Gallbladder and Bile Duct Disorders
Type
article
Field-Weighted Citation Impact
0.42
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article

OP-1 LONG-TERM EFFICACY AND SAFETY OF OPEN-LABEL SELADELPAR TREATMENT IN PATIENTS WITH PRIMARY BILIARY CHOLANGITIS: INTERIM 2-YEAR RESULTS FROM THE ASSURE STUDY

Marlyn J. Mayo, Sarah Proehl, Ziad Younes, Daria B. Crittenden et al.
1 citations
Annals of Hepatology
Gallbladder and Bile Duct Disorders
0.42
article

OP-1 LONG-TERM EFFICACY AND SAFETY OF OPEN-LABEL SELADELPAR TREATMENT IN PATIENTS WITH PRIMARY BILIARY CHOLANGITIS: INTERIM 2-YEAR RESULTS FROM THE ASSURE STUDY

Marlyn J. Mayo, Sarah Proehl, Ziad Younes, Daria B. Crittenden, Aparna Goel, Christopher L. Bowlus, Adam Peyton, Oren Shibolet, Charles A. McWherter, Jeong Heo, Stuart C. Gordon, Daniel S. Pratt, Cynthia Levy, Carrie Heusner, Palak Trivedi, Alejandra Villamil, Pietro Andreoné, Eric Lawitz, Kidist Yimam, Andreas Emanuel Kremer, Christophe Corpechot, Kris V. Kowdley, Ulrike Morgera, Maria Carlota Londoño Hurtado, Shuqiong Zhou, Hany Elbeshbeshy, Gideon M. Hirschfield, Aliya F. Gulamhusien, Alma Ladron de Guevara Cetina
article en
1 citations

Abstract

Yes, Full disclosures sent separately. Seladelpar reduces biochemical markers of cholestasis and pruritus in patients with primary biliary cholangitis. ASSURE (NCT03301506) is an ongoing, open-label, long-term Phase 3 trial of seladelpar in patients rolling over from Phase 3 RESPONSE (NCT04620733) or legacy studies (NCT03602560, NCT02955602, NCT03301506, and NCT04950764). We report interim 2-year efficacy and safety results. Patients with insufficient response/intolerance to ursodeoxycholic acid could enroll in ASSURE. Key endpoints were composite biochemical response (alkaline phosphatase [ALP] <1.67 × upper limit of normal [ULN], ALP decrease ≥15%, and total bilirubin ≤ULN) and ALP normalization. Pruritus was measured using numerical rating scale (NRS; 0–10). For patients enrolling from RESPONSE, baseline was entry to RESPONSE and analyzed as continuous seladelpar or crossover from placebo; legacy patients were analyzed separately with baseline defined as entry to ASSURE. As of 01/2024, 158 RESPONSE and 179 legacy patients received seladelpar 10 mg daily for up to 155 weeks. In RESPONSE, 61.7% of patients met the endpoint at 12 months (M) vs 20% for placebo. In ASSURE, 61.8% (6M) and 72.4% (12M) met the composite endpoint; 75% (6M) and 93.8% (12M) of placebo crossover patients met the endpoint. In RESPONSE, ALP normalized in 25% of seladelpar and 0 placebo patients at 12M. With continued treatment, 33.3% (6M) and 17.2% (12M) had ALP normalization; 26.9% (6M) and 50% (12M) of crossover patients had ALP normalization. In ASSURE, 6-month change from baseline in pruritus NRS was similar to RESPONSE: −3.8 and −3.7 in continuous and crossover patients, respectively. At 12M and 24M, 73.2% and 69.7% of legacy patients met the endpoint in ASSURE; 42.1% and 42.4% achieved ALP normalization, and reduction in pruritus NRS was −3.8 and −3.1, respectively. There were no treatment-related serious adverse events. Seladelpar treatment led to improvements in biochemical markers and pruritus, and was well tolerated with long-term use.

Annals of HepatologyVol. 29
University of Modena and Reggio Emilia (IT), United Nations Industrial Development Organization (AT), University of Miami (US), Henry Ford Health System (US), The University of Texas at San Antonio Health Science Center (US), American College of Veterinary Surgeons (US), Tel Aviv Sourasky Medical Center (IL), California Pacific Medical Center (US), Hospital Italiano de Buenos Aires (AR), Massachusetts General Hospital (US), University Hospital of Zurich (CH), Pusan National University Hospital (KR), Liver Institute Northwest (US), Pacific Medical (China) (CN), Centro Médico ABC (MX), Texas Liver Institute (US), Toronto Liver Centre (CA), Fundació Clínic per a la Recerca Biomèdica (ES), Pusan National University (KR), Charité - Universitätsmedizin Berlin (DE), Miami University (US), University of California, Davis (US), The University of Texas Southwestern Medical Center (US)
Good health and well-being
Openalex Percentile: Top 30%
Gallbladder and Bile Duct Disorders
0.42
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