Gilead Sciences has announced promising results from an open-label extension study of its investigational PPAR delta agonist, seladelpar, in patients with primary biliary cholangitis (PBC).
Affecting approximately 130,000 people in the US, PBC is a rare cholestatic liver disease mainly affecting women.
The most common early symptoms of PBC are pruritus, or itching, and fatigue, and disease progression is associated with an increased risk of liver-related mortality.
The ongoing ASSURE study has been evaluating the long-term safety and efficacy of seladelpar in adults with PBC who had previously participated in a trial of the candidate and who had not responded adequately to ursodeoxycholic acid (UDCA), with the majority of patients being female and also receiving UDCA treatment.
Results showed that 70% of 148 patients receiving an oral 10mg dose of seladelpar once daily achieved the clinically meaningful composite response endpoint and 37% achieved alkaline phosphatase normalisation after 12 months.
Of the 20 patients who completed 24 months of treatment, 70% achieved the composite response endpoint and 25% experienced ALP normalisation.
Seladelpar also reduced other important biomarkers of liver injury, demonstrated a rapid and durable reduction in patient-reported pruritus in those with moderate-to-severe symptoms, and was generally well tolerated.
Merdad Parsey, chief medical officer, Gilead Sciences, said: “The initial data from ASSURE further supports the efficacy and safety profile of seladelpar observed across the robust development programme and continues to indicate that seladelpar has the potential to be a best-in-class therapy that could help transform treatment for people living with PBC.”
Gilead gained access to seladelpar in March through its $4.3bn acquisition of CymaBay Therapeutics .
The candidate has already been accepted for priority review by the US Food and Drug Administration, with a Prescription Drug User Fee Act target action date of 14 August set.
Daniel O’Day, chairman and chief executive officer at Gilead, said at the time of the acquisition announcement in February: “Building on the strong research and development work by the CymaBay team to date, we have the potential to address a significant unmet need for people living with PBC and expand on our existing broad range of transformational therapies.”