Long-term efficacy and safety of open-label seladelpar treatment in patients with primary biliary cholangitis: pooled interim results for up to 3 years from the ASSURE study
Bibliographic record
Abstract
Introduction: ASSURE (NCT03301506) is an ongoing, open-label, long-term, Phase 3 trial of seladelpar — a selective PPARδ agonist — in patients (pts) with primary biliary cholangitis rolling over from the Phase 3, placebo-controlled RESPONSE trial (NCT04620733) or with prior participation in legacy trials (Phase 3 ENHANCE [NCT03602560], CB8025-21629 [NCT02955602], CB8025-31731 [NCT03301506], CB8025-21838 [NCT04950764]). The parent studies required an inadequate response or intolerance to first-line ursodeoxycholic acid. Objectives: Here, we report pooled interim efficacy and safety for all pts in ASSURE. Methodology: Data cutoff: January 31, 2024; pt exposure to seladelpar in ASSURE (including exposure in pts randomized to the active treatment arm in RESPONSE) was analyzed. Key efficacy endpoints included composite biochemical response (CBR; alkaline phosphatase [ALP]<1.67×upper limit of normal [ULN], ALP decrease≥15%, and total bilirubin [TB]≤ULN) and ALP normalization. Pruritus was recorded using a numeric rating scale (NRS; 0–10) collected daily through month (M) 6; change from baseline (BL) was assessed through M6 in pts with moderate-to-severe pruritus (NRS≥4) at BL. Exposure-adjusted adverse events (AEs) were calculated for each year on study as incidence per 100 pt-years. BL was based on first exposure to seladelpar in ASSURE or RESPONSE. Results: 337 pts received 10-mg seladelpar daily. 34 pts reached 30M on study; 90 pts had≥24M of seladelpar exposure. At BL, the mean (SD) age was 58.1 (9.7) years, 318/337 (94%) pts were female, mean (SD) ALP was 287.5 (128.4) U/L, mean (SD) TB was 0.75 (0.34) mg/dL, and 55/337 (16%) had cirrhosis. At M12, M24, and M30, 204/280 evaluable pts (73%), 90/124 (73%), and 30/37 (81%) met the CBR endpoint, respectively, and ALP normalized in 106/280 (38%), 47/124 (38%), and 15/37 (41%) pts, respectively. In the pruritus NRS, mean (SE) change from BL at 6M was−3.3 (0.24) among 99 evaluable pts. Outcomes are in [ Fig. 1 ]. Exposure-adjusted AEs were observed in 86, 70, and 63 pts per 100 pt-years at M12, M24, and M36, respectively. There were no treatment-related serious AEs. Fig. 1 Conclusion: By M30 of the long-term ASSURE study, seladelpar resulted in a durable and sustained biochemical response in 81% of pts, with an ALP normalization rate of 41%, and robust improvement in pruritus. Seladelpar continues to appear safe and well tolerated, with no new safety signals or change in frequency of AEs with up to 3 years of exposure. Abstracts Präsentiert in der Sitzung: Autoimmune und cholestatische Lebererkrankungen Donnerstag, 18. September 2025, 16:30 – 18:00, Saal 3 Publication History Article published online: 04 September 2025 © 2025. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.009 | 0.006 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.005 | 0.007 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.002 | 0.002 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.002 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".