Seladelpar Development Status
RESPONSE (NCT04620733), a 52-week Phase 3 global registration study of seladelpar in patients with PBC who have demonstrated intolerance or inadequate response to UDCA, was initiated in the first quarter of 2021. The primary outcome of RESPONSE is composite responder rate (ALP<1.67xULN, ≥15% decrease in ALP, total bilirubin ≤ULN) and secondary outcomes are the proportion of patients with ALP ≤1.0 x ULN at 12 months and change from baseline in pruritus in subjects with baseline Numerical Rating Scale >4 using e-diary at 6 months.
IDEAL, A 52-week Phase 3 study of seladelpar in patients with PBC who have an incomplete response or intolerance to ursodeoxycholic acid (UDCA), in each case with ALP greater than the upper limit of normal (ULN) but less than 1.67xULN, and total bilirubin less than or equal to 2xULN was initiated in August 2023. The primary outcome measure is the normalization of ALP at 12 months and additional key outcomes evaluating efficacy include the percent change from baseline in ALP at 12 months and the level of self-reported pruritus (itch) at 6 months for patients with moderate to severe symptoms at baseline, assessed by a numerical rating scale (NRS) and recorded by electronic diary.
We also are conducting ASSURE (NCT03301506), an open-label, long-term study of seladelpar in patients with PBC intended to collect additional long-term safety data to support registration. The first subjects will consist of patients who have participated in our prior studies of seladelpar in PBC, including the patients who completed the open-label Phase 2 study and enrolled in the previous long-term study and ENHANCE. Patients who complete RESPONSE, and potentially other future PBC studies with seladelpar, will also have the opportunity to enroll in ASSURE.