RESPONSE topline data: Seladelpar normalized alkaline phosphatase, reduced pruritus in PBC
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Seladelpar reduced serum alkaline phosphatase and bilirubin at 12 months in more than 60% of patients with primary biliary cholangitis compared with 20% of placebo-treated patients, according to manufacturer, CymaBay Therapeutics.
These preliminary phase 3 trial results support filing of seladelpar, a selective peroxisome proliferator-activated receptor delta agonist, for regulatory approval with the FDA, the company stated in a press release.
“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, normalizes these measures,” Gideon Hirschfield, MD, chair of autoimmune liver disease research at Toronto Center for Liver Disease, said in the release.
In the double-blind, placebo-controlled trial, 193 patients with primary biliary cholangitis (PBC) received either seladelpar 10 mg (n = 128) or placebo (n = 65) once daily for 12 months. After 52 weeks, 61.7% of patients in the treatment group vs. 20% of patients in the placebo group reached the primary composite endpoint of reduced alkaline phosphatase (ALP) (< 1.67 x the upper limit of normal with 15% decrease in ALP) and total bilirubin (< 1 upper limit of normal).
Further, 25% of patients treated with seladelpar achieved ALP normalization at 12 months compared with 0% on placebo, with a least-squares mean percentage reduction in ALP of 42.4% vs. 4.3%, respectively. Patients experiencing moderate to severe pruritis at baseline also reported significant reduction after 6 months on seladelpar.
“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,” Hirschfield said.
According to the release, safety was comparable between groups and consistent with previous findings. There were no serious treatment-related adverse events.
“The results from RESPONSE support our conviction that seladelpar has the potential to advance patient care by improving measures of disease activity and reducing symptom burden,” Sujal Shah, president and CEO of CymaBay, said in the release. “They are consistent with previous findings in what we believe has been an exceptionally robust development program in PBC. We believe that the delpar mechanism is unique with its ability to normalize markers of cholestasis coupled with reductions in pruritus.”
Additional analyses of data from the RESPONSE trial are ongoing, the company stated.
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