6-month liver disease data reinforces CymaBay's phase 3 move

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CymaBay is developing seladelpar, a peroxisome proliferator-activated receptor delta (PPARδ) agonist, for nonalcoholic steatohepatitis (NASH), as well as for primary biliary cholangitis. (Image: Enrique Meseguer)

CymaBay will unveil new data from a phase 2 study of its liver med seladelpar at The International Liver Congress Thursday. The 26-week data show that the drug had "potent and sustained" efficacy in patients with primary biliary cholangitis (PBC), an autoimmune disease that causes progressive destruction of bile ducts.

CymaBay was up 18% Wednesday morning.

In the study, 71 patients were given at least one dose of seladelpar, of whom 53 underwent 12 weeks of treatment and 42 continued the treatment for another 14 weeks, the company said in a statement. The patients were divided into three dosing groups: 2 mg, 5 mg and 10 mg. 

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The drug demonstrated anti-inflammatory and anti-cholestatic—cholestasis describes a decrease in bile duct flow—effects. At 12 weeks, it reduced levels of the liver disease marker alkaline phosphatase (AP) by 21%, 33% and 45% in the 2 mg, 5 mg and 10 mg groups respectively. After 12 weeks, the investigators tweaked the dose for patients whose AP levels stayed higher than normal and where lowering AP could reduce the risk of disease progression. The dosing groups were 5 mg, 5 to 10 mg titration and 10 mg; all three exhibited similar drops in AP levels: 45%, 43% and 43% respectively. 

The majority of patients across all three groups "had an AP less than 1.67 times the upper limit of normal, with at least a 15% decrease in AP from baseline" and 29% of patients had a normal AP at 26 weeks. Patients saw a drop in transaminase, another marker of liver disease, and did not develop drug-induced pruritis, an itching sensation commonly seen in liver disease.

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“Seladelpar continues to demonstrate an impressive level of activity that is now sustained over 26 weeks of treatment. The decreased level of pruritus that was noted in the 10 mg group, a group that demonstrated a clinically relevant level of pruritus at baseline, is particularly intriguing and needs to be confirmed in additional studies," said Gideon Hirschfield of the University of Birmingham, who will present the findings. 

CymaBay is developing seladelpar, a peroxisome proliferator-activated receptor delta (PPARδ) agonist, for nonalcoholic steatohepatitis (NASH), as well as for PBC. The data reaffirms its plans to take seladelpar into phase 3 later this year. 

“We have now firmly established doses of seladelpar with compelling efficacy and tolerability which we expect to further confirm in a Phase 3 study planned to start in the second half of the year,” said Pol Boudes, M.D., CymaBay's chief medical officer. “These data continue to support the potential for seladelpar to significantly improve treatment options for patients with PBC. We are thankful for the commitment and dedication of the patients, PBC support groups, investigators and study coordinators who are all essential to our efforts to advance seladelpar for patients with PBC.”