CymaBay Therapeutics announces oral late-breaking presentation of positive results from the ENHANCE global phase III study of seladelpar for primary biliary cholangitis at The Liver Meeting 2020.

-CymaBay Therapeutics, Inc. announced an oral late-breaking presentation of the positive results from the ENHANCE phase III study evaluating seladelpar for primary biliary cholangitis (PBC) . These data were presented online during the late-breaking session at The Liver Meeting of the American Association for the Study of Liver Diseases (AASLD). In an oral presentation titled, “ENHANCE: Safety and efficacy of Seladelpar in Patients with Primary Biliary Cholangitis (PBC) – A Phase III, International, Randomized, Placebo-Controlled Study,” Professor Gideon Hirschfield, FRCP PhD, Lily and Terry Horner Chair in Autoimmune Liver Disease at the University of Toronto, presented results from ENHANCE, a phase III study of seladelpar in patients with PBC. Eligible PBC patients with either an inadequate response, defined as alkaline phosphatase (ALP) greater than or equal to 1.67 times the upper limit of normal (ULN), or intolerance to ursodeoxycholic acid (UDCA) were randomized to daily seladelpar 5 or 10 mg or placebo (pbo). The primary endpoint was a composite response of ALP <1.67x uln alp decrease greater than 15 and total bilirubin tb less than uln at month 12. secondary endpoints were alp normalization at month 12 and change in pruritus numerical rating scale nrs at month 6. due to the early termination of the study and the small number of patients who had reached the 52 week timepoint the primary outcome measure was amended prior to database lock to a 3 month timepoint. after only 3 months 78.2 of patients on seladelpar 10 mg versus 12.5 on placebo achieved the primary composite outcome p><0.0001). in addition 27.3 of patients on seladelpar 10 mg versus zero on placebo experienced normalization of alp by 3 months p><0.0001). treatment with seladelpar 10 mg also resulted in a statistically significant improvement in pruritus p><0.05) for patients with moderate-to-severe itch at baseline versus placebo. additional data highlighted the sustained anti-cholestatic anti-inflammatory and anti-pruritic effects of seladelpar through six months. overall seladelpar appeared to be safe and well-tolerated in this study. the only ae in greater than 10 of patients was pruritus in 12.6 3.4 and 11.2 in the pbo 5 and 10 mg groups respectively. there were no treatment-related serious adverse events and 2 treatment-emergent aes led to study discontinuation. these findings suggest that seladelpar treatment is well positioned to be a preferred second line treatment for patients with pbc said professor hirschfield. data from this study show that seladelpar appears efficacious safe and well-tolerated. given the high unmet need that exists in the pbc population i believe investigators and patients will continue to support cymabays efforts to make this breakthrough therapy available to patients. a second clinical presentation titled a 52-week multi-center double-blind randomized phase ii study of seladelpar a potent and selective peroxisome proliferator-activated receptor delta ppar-delta agonist in patients with nonalcoholic steatohepatitis nash was presented by dr. stephen a. harrison md medical director of pinnacle clinical research. this electronic poster presentation was selected by aasld for special recognition as a poster of distinction and highlighted the effects of seladelpar on liver fat liver enzymes and key histologic endpoints recognized by regulators for registration including nash resolution and reduction in fibrosis..>