CymaBay announces results from RESPONSE Phase 3 study of seladelpar

CymaBay Therapeutics announced that the results from its RESPONSE Phase 3 registration study evaluating seladelpar, a potent, selective, orally-active delpar, or PPAR agonist, in adult patients with primary biliary cholangitis will be featured as an oral presentation in today’s Late Breaker Session of the American Association for the Study of Liver Diseases’, AASLD, The Liver Meeting 2023 in Boston, Massachusetts. RESPONSE was a double-blind, placebo-controlled, global study of one-year duration that randomized 193 PBC patients in a 2:1 ratio to seladelpar 10 mg or placebo, once daily. The primary endpoint was a composite of ALP and total bilirubin previously accepted by the U.S. Food and Drug Administration and European Medicines Agency for registration studies in PBC. The composite endpoint was achieved in 61.7% of patients on seladelpar vs. 20.0% on placebo. The key secondary endpoint of ALP normalization occurred in 25% of patients receiving seladelpar vs. 0% for patients on placebo. The average decrease in ALP for patients on seladelpar was -133.9 U/L vs. -16.9 U/L for patients on placebo. Compared to placebo, seladelpar reduced alanine aminotransferase at Month 12 by 23.5% vs. 6.5% and gamma-glutamyl transferase by 39.1% vs. 11.4%. The study also measured change in patient-reported pruritus, as a key secondary endpoint using the daily numerical rating scale. The pruritus endpoint was met at Month 6 among patients with baseline NRS greater than 4 reporting decreases of 3.2 points with seladelpar vs. 1.7 for patients on placebo. Notably, these improvements were sustained through Month 12. Approximately 37.3% of patients enrolled had moderate-to-severe pruritus based on an NRS score greater than or equal to 4 at baseline. A statistically significant reduction in pruritus was also observed at Month 6 and at Month 12 for patients in the intent-to-treat population, which includes all patients irrespective of their NRS score at baseline.