First Patient Enrolled in Phase 3 PFIC Trial of A4250

The first patient has been enrolled in PEDFIC-1, a phase 3 clinical trial of A4250, an ileal bile acid transporter (IBAT) inhibitor being studied for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC).

Albireo Pharma, Inc. recently announced that the first patient has been enrolled in PEDFIC-1, a Phase 3 clinical trial of A4250, an ileal bile acid transporter (IBAT) inhibitor being studied for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC).

"We are relentlessly focused on developing A4250 for cholestatic liver diseases, beginning with PFIC, and the initiation of our phase 3 clinical trial in PFIC marks a crucial milestone," stated Ron Cooper, President and Chief Executive Officer of Albireo in a recent comment. "PFIC is a rare genetic liver disease that frequently presents with debilitating pruritus and often leads to life-threatening liver disease. We’ve worked closely with regulators in the United States and Europe to develop our phase 3 program, and now are working to activate all of our clinical sites around the world with the goal of evaluating the safety and efficacy of A4250 in patients with PFIC.”

A4250 is a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT); it acts locally in the gut and has minimal systemic exposure.

PFIC causes progressive, life-threatening liver disease and is estimated to affect between 1 in every 50,000 to 100,000 children born worldwide. Currently, no approved pharmacological treatment exists for PFIC, only palliative care, surgical options, and liver transplant are available, which all have substantial risks.

The phase 3 program is comprised of a single randomized, double-blind, placebo-controlled clinical trial engineered to evaluate A4250. Sixty participants aged 6 months to 18 years with PFIC (subtype 1 or 2), elevated serum bile acid (sBA) levels, and pruritus will be eligible to participate. Subjects will also partake in an open-label extension study to assess long-term safety and durability of response. For 24 weeks, those enrolled in the double-blind trial will be administered a 40 or 120 μg/kg oral dose of A4250 or placebo once daily.

An assessment of change in pruritus will be the US Food and Drug Administration (FDA) evaluation’s primary endpoint. The sBA responder rate will be the European Medicines Agency (EMA) evaluation’s primary endpoint.

“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,” commented 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.”

Previously, A4250 has been granted orphan drug designation for PFIC in the United States and European Union. In addition, the European Medicines Agency (EMA) has granted A4250 access to the PRIority MEdicines (PRIME) program for the treatment of PFIC, and its Paediatric Committee has agreed to Albireo's A4250 Pediatric Investigation Plan.

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