Gilead's new liver disease med continues to deliver the goods ahead of FDA verdict

Three months after spending $4.3 billion on CymaBay Therapeutics for its primary biliary cholangitis (PBC) med, Gilead Sciences has presented a fresh slice of data as a reminder of seladelpar’s potential while an FDA decision is pending.

The open-label ASSURE study comprised 174 patients who had already previously participated in another study of seladelpar and who had not responded adequately to ursodeoxycholic acid (UDCA), the typical treatment for PBC. They received an oral dose of 10 mg seladelpar each day, with nearly all patients being female and also receiving UDCA treatment.

A total of 148 patients completed 12 months of treatment. Of these, 70% reached the clinically meaningful composite response endpoint, Gilead explained in a presentation of the interim data in a May 18 release. Across all patients who received seladelpar, 37% experienced an average change from baseline alkaline phosphatase (ALP) level of 44%, considered a return to normal levels.

Twenty patients received the selective PPAR-delta agonist for a full two years. Of these, 70% reached the response endpoint, but only 25% saw ALP normalization.

Other biomarkers of liver injury were also reduced. Specifically, total bilirubin fell by 9%, gamma-glutamyl transferase by 36% and alanine aminotransferase by 25% from baseline levels.

There were no treatment-related adverse events, while 4.6% of patients discontinued due to adverse events, Gilead reported.

“The initial data from ASSURE further support the efficacy and safety profile of seladelpar observed across the robust development program and continue to indicate that seladelpar has the potential to be a best-in-class therapy that could help transform treatment for people living with primary biliary cholangitis,” Gilead Chief Medical Officer Merdad Parsey, M.D., Ph.D., said in the release.

“The PBC community has been waiting for new treatments that help slow the progression of their liver disease as well as address difficult symptoms like pruritus that can have a significant impact on their quality of life,” Parsey added. “We are excited to offer a new option for this community as we work to bring seladelpar to people living with PBC, if approved.”

Seladelpar was submitted to the FDA to treat PBC with pruritus, or itching, without cirrhosis or with compensated cirrhosis in December 2023. The rare liver disease primarily affects women over 40 and is characterized by impaired bile flow and the accumulation of bile acids in the liver, causing an elevation of liver enzymes that can be dangerous.

The FDA is set to make a call on Aug. 14 on approving seladelpar for the treatment of PBC, including pruritus, in adults without cirrhosis or with compensated cirrhosis who are inadequate responders or intolerant to UDCA.