Albireo Pharma, Inc. announced the completion of patient enrollment in the ASSERT study, a Phase 3 pivotal trial of Bylvay (odevixibat) in patients with Alagille syndrome (ALGS). Topline results are expected to be available by the end of the year, consistent with guidance, and with the enrollment of 52 patients versus an original target of 45. ASSERT is a gold standard, global, double-blind, randomized, placebo-controlled trial designed to evaluate the safety and efficacy of Bylvay in patients with ALGS over 24 weeks.

Both the U.S. FDA and EMA have agreed on the Phase 3 study design and have indicated that this single study would be sufficient for regulatory filings. Bylvay is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) already approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of PFIC, and in Europe for the treatment of all types of PFIC in patients aged 6 months or older. ALGS is a rare, multisystem genetic disorder that can affect the liver, heart, skeleton, eyes, central nervous system, kidneys and facial features.

Liver damage is caused by a paucity of bile ducts preventing bile flow from the liver to the small intestine. Approximately 95% of patients with the condition present with chronic cholestasis, usually within the first three months of life, and as many as 88% also present with severe, intractable pruritus. Aligned to the Company's mission of providing hope for families, Albireo continues to prioritize access and continued scientific research for patients living with rare cholestatic liver diseases.

Albireo opened an Expanded Access Program (EAP) with the first patient already enrolled. The program is available in the U.S. and Europe, which aims to provide access to Bylvay for patients suffering from ALGS, prior to the product's planned approval and reimbursement. Timing of availability in Europe will vary due to country-specific regulations and stock availability.

This program is available for patients with a clinical diagnosis of ALGS who have no other therapeutic options. The Company continues to enroll and dose patients in the Phase 3 BOLD study, which is the first and only pivotal trial of an IBATi in biliary atresia, that passed the 50% enrollment milestone and remains on track for topline data in 2024. Biliary atresia is the most common pediatric cholestatic liver disease with no approved drug treatment.