Seladelpar, which Gilead acquired when it bought CymaBay in February for $4.3 billion, showed positive results for reducing pruritus in primary biliary cholangitis patients and reducing inflammation.
Gilead Sciences announced Saturday that Seladelpar, part of the $4.3 billion purchase of CymaBay in February 2024, posted positive interim late-stage results in patients with the autoimmune disease primary biliary cholangitis.
The Phase III ASSURE study is investigating the once-daily selective peroxisome proliferator-activated receptor (PPAR) delta agonist, with data showing a reduction in pruritus, or itch, for patients with primary biliary cholangitis (PBC).
Those who participated in the trial were adults with PBC who had already participated in a seladelpar study and had an “inadequate response or intolerance” to ursodeoxycholic acid (UDCA), according to Gilead. UDCA is the only agent currently approved by the FDA for first-line treatment of PBC.
The interim results for ASSURE showed that 70% of the 148 patients who finished 12 months of treatment had a clinically meaningful composite response endpoint. The results also showed that 37% of seladelpar patients had alkaline phosphatase (ALP) normalization with a mean ALP change from the baseline of -44%.
For the 20 patients who finished 24 months of treatment, 70% reached the composite response endpoint, and 25% had ALP normalization. Gilead also reported that the drug had reduced other biomarkers of liver injury, such as total bilirubin (TB) by 9%, alanine aminotransferase (ALT) by 25%, and gamma-glutamyl transferase (GGT) levels by 36%.
“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 CMO Merdad Parsey said in a statement. “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 significantly impact their quality of life.”
The pharma said the study had no serious adverse events, and the drug was well tolerated. Discontinuation due to adverse events occurred in 4.6% of patients. The results did not include patients from its Phase III Response study, which will be reported independently.
An investor note from the Bank of Montreal said that seladelpar continues to show “an interesting opportunity” for Gilead to grow and differentiate its liver portfolio.
The drug candidate has a PDUFA date of Aug. 14, 2024, and has also been accepted for review by the EMA and the U.K.’s Medicines and Healthcare Products Regulatory Agency.
Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.
