Additional findings demonstrate that seladelpar can help reduce pruritus (itch) in people living with primary biliary cholangitis (PBC). There are currently no treatments indicated to treat PBC-related pruritis. This data will be shared in an oral presentation during the Presidential Plenary of the Digestive Disease Week 2024 Conference in
ASSURE is an open-label study evaluating the long-term safety and efficacy of seladelpar, a once daily potent and selective peroxisome proliferator-activated receptor (PPAR) delta agonist, or delpar. ASSURE enrolled adult patients with PBC who previously participated in a study of seladelpar where a key eligibility criterion included having an inadequate response or intolerance to ursodeoxycholic acid (UDCA). This interim data analysis did not include patients from the Phase 3 RESPONSE study, which will be reported separately. Of the 174 patients included, the majority had a gap of one year or more between completion of the respective primary study (seladelpar or placebo) and enrollment into ASSURE. Enrolled patients received an open-label oral dose of 10 mg seladelpar once daily, with the majority (97%) also receiving UDCA treatment.
Most patients enrolled in ASSURE were female (94%), with a mean age of 59 years. Baseline characteristics included mean alkaline phosphatase (ALP) 270.5 U/L and total bilirubin (TB) 0.75 mg/dL; 19% of enrolled patients met the criteria for cirrhosis. The study evaluated several prespecified biochemical endpoints, including the composite response of an alkaline phosphatase (ALP) below 1.67 x upper limit of normal (ULN), a decrease in ALP of at least 15%, and a total bilirubin (TB) below the ULN. Endpoints were evaluated over an interim observation period extending from enrollment through a data cutoff date of
'We are encouraged by these positive interim results from ASSURE, which are highly consistent with those observed in the Phase 3, double-blind placebo-controlled RESPONSE study of seladelpar in people with an inadequate response or intolerance to UDCA,' said
Patient-reported pruritus was monitored throughout the study using the numerical rating scale (NRS; 0-10). In the 60 patients with moderate-to-severe pruritus at baseline with an NRS score of 4, a rapid improvement in pruritus was observed at Month 1. By Month 6 the patients reported a mean reduction of 3.5 points, and this impact was sustained through Month 12.
'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,' said
Additional results from ASSURE, including the long-term study of patients rolled over from RESPONSE to the ASSURE study, will be available at a future medical meeting.
A New Drug Application (NDA) for seladelpar for the treatment of PBC, including pruritus, in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to UDCA, has been accepted for priority review by the
About ASSURE (NCT03301506)
ASSURE is an open label study to evaluate the long-term safety and tolerability of seladelpar in people with primary biliary cholangitis (PBC) who have already participated in other PBC clinical trials of seladelpar. The study is currently enrolling up to 500 people living with PBC from across 160 sites around the world. ASSURE will also address the long-term efficacy of seladelpar and its impact on important patient reported outcomes such as pruritus, or severe itch, which can have a significant impact on the quality of life of people living with PBC.
Interim results of ASSURE (Session #2060), titled 'Efficacy and Safety of Seladelpar in Patients with Primary Biliary Cholangitis in the ASSURE Study: Interim Results,' will be presented Dr.
About Seladelpar
Seladelpar, an investigational treatment for people with PBC, is a first-in-class oral, selective PPAR-delta agonist, or delpar. PPAR-delta has been shown to regulate critical metabolic and liver disease pathways. Preclinical and clinical data support its ability to regulate genes involved in bile acid synthesis, inflammation, fibrosis and lipid metabolism, storage, and transport. Seladelpar is not approved by the FDA or any other regulatory authority globally and has not been determined to be safe or efficacious for any use.
About PBC
PBC is a rare, chronic inflammatory liver disease primarily affecting women (1 in 1,000 women over the age of 40 or about 130,000 total people in the
About CymaBay
About
For decades, Gilead has pioneered the way forward to improve the lives of people living with liver disease around the world. We have helped to transform hepatitis C from a chronic condition into one that can be cured for millions of people. For people living with hepatitis B or D, our focus on advancing our medicines drives hope that today's research will turn into tomorrow's cures. Beyond viral hepatitis, we're working to deliver advanced treatments for people living with primary biliary cirrhosis (PBC). But our commitment doesn't stop there. Through our ground-breaking science and collaborative partnerships, we strive to create healthier futures for everyone living with liver disease. We are committed to a future without liver disease.
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Forward-Looking Statements
This press release includes forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the ability of Gilead and CymaBay to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing or additional clinical studies, including those involving seladelpar; the possibility that Gilead and CymaBay may make a strategic decision to discontinue development of programs for indications that are currently under evaluation and, as a result, these programs may never be successfully commercialized for such indications; uncertainties relating to regulatory applications and related filing and approval timelines, including the risk that the FDA, MHRA or EMA may not approve seladelpar the treatment of primary biliary cholangitis, and the risk that any such approvals, if granted, may be subject to significant limitations on use and any assumptions underlying any of the foregoing. These and other risks, uncertainties and factors are described in detail in Gilead's Quarterly Report on Form 10-Q for the quarter ended
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