Albireo Enrolls First Patient in Phase 3 PFIC Trial of A4250
Albireo Pharma announced the first patient has been enrolled in PEDFIC-1, a Phase 3 clinical trial of lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor being studied for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC).
“A PFIC diagnosis for our children was shocking and devastating, and we quickly found out that current treatment options are inadequate,” said Kristen and Michael Busby of New York, parents of two children with PFIC. “We applaud all research in this area, and it is great news to see a clinical trial as an option for children with PFIC.”
PFIC is estimated to affect between one in every 50,000 to 100,000 children born worldwide and causes progressive, life-threatening liver disease. Moderate to severe pruritus is a common and problematic clinical presentation of PFIC that can severely diminish quality of life. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all patients with PFIC require treatment before age 30. There are currently no approved pharmacological treatment options for PFIC.
“We are very excited to have the first patient enrolled into the PEDFIC-1 pivotal study to determine the potential of A4250, with the goal of providing new options to patients with this debilitating disease,” said Professor Richard Thompson, King’s College London and principal investigator of the study. “The results of the Phase 2 trial, where A4250 reduced serum bile acids and decreased pruritus in most patients, while showing a favourable overall tolerability profile, gives us optimism and confidence that A4250 could be an excellent medical treatment option.”
The Phase 3 program includes a single randomized, double-blind, placebo-controlled clinical trial designed to evaluate A4250 in 60 patients, ages 6 months to 18 years, with PFIC (subtype 1 or 2), elevated serum bile acid (sBA) levels and pruritus, and an open-label extension study to assess long-term safety and durability of response. Patients in the double-blind trial will receive a 40 or 120 μg/kg oral dose of A4250 or placebo once daily for 24 weeks. The primary endpoint for the US Food and Drug Administration (FDA) evaluation will be an assessment of change in pruritus, and the primary endpoint for the European Medicines Agency (EMA) evaluation will be sBA responder rate.
“The team at Albireo is proud to start a Phase 3 trial with A4250 in PFIC to generate data to support potential approval and provide the first approved treatment for patients with PFIC,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Our team is committed to working expeditiously to activate clinical trial sites around the world, complete the study and seek regulatory approval as soon as possible.”
A4250 has received orphan drug designation for PFIC in the United States and European Union, and has been granted access to the EMA’s PRIority MEdicines (PRIME) program for the treatment of PFIC.