Clinical Trials

The trial reached all of its primary and key secondary endpoints, with 61.7% of patients on a 10mg dose meeting the primary composite endpoint related to serum alkaline phosphatase and bilirubin at 12 months compared to only 20% of the placebo group. Treatment with seladelpar also demonstrated a statistically significant reduction in pruritus, or itch, after six months of treatment.

Results from the trial will support the company in seeking regulatory approval from the US Food and Drug Administration (FDA), the Medicines and Healthcare products Regulatory Agency (MHRA) and the European Medicines Agency (EMA).

Gideon Hirschfield, MD, Lily and Terry Horner chair in Autoimmune Liver Disease Research, Toronto Centre for Liver Disease, Canada, commented: “The topline results seen in the RESPONSE trial are exciting for highlighting the potential for an efficacious and safe new therapy that not only achieves the composite improvements in liver tests, but for a significant proportion of patients, normalises these measures. Further, the results support that seladelpar reduced itch, a particularly challenging symptom that continues to negatively impact quality of life for many PBC patients. While existing first- and second-line therapies have helped patients living with PBC, this is the first potential therapy to show promise in both significantly improving markers associated with risk of disease progression while also significantly reducing itch."