Albireo Achieves Significant Milestones for Odevixibat Across Multiple Diseases

Albireo Pharma, Inc. recently announced a number of advances and new initiatives in the clinical program for odevixibat, an oral once daily capsule in development for the treatment of progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome. The US FDA has cleared the company’s investigational new drug (IND) application to initiate a global pivotal trial in biliary atresia. Following scheduled FDA interactions in the first quarter of 2020, the company plans to commence an additional pivotal program in Alagille syndrome by the end of 2020. Management also continues to expect topline data from its Phase 3 trial in PFIC in mid-2020.

“We continue to believe in the potential for odevixibat to address unmet needs across multiple rare cholestatic liver diseases, and we remain steadfastly committed to patients and their families living with these devastating conditions,” said Ron Cooper, Albireo’s President and Chief Executive Officer. “We expect our Phase 3 trial in PFIC will be fully randomized in the coming days, and we expect to report topline data in mid-2020. Today, I am particularly excited to announce that the FDA has cleared our IND, and we expect to initiate a precedent-setting pivotal trial in biliary atresia in the first half of 2020. Following FDA input later this quarter, we plan to initiate a third odevixibat pivotal trial, in Alagille syndrome, later this year. We are a growing, energized and well-prepared organization, and look forward to 2020 being a transformational year for Albireo.”

The PEDFIC 1 trial is studying both high and low dose odevixibat in both PFIC type 1 and type 2 patients who are 6 months to 18 years of age. Patients randomized to odevixibat are being treated with high (120 µg/kg) or low (40 µg/kg) dose once-daily oral capsules or sprinkles. The trial uses Albireo’s planned commercial formulation of odevixibat, which does not require refrigeration. PEDFIC 1 has a target of 60 patients, 59 have been randomized and the randomization visit for the final patient has been scheduled in the coming days. Albireo expects top-line data from PEDFIC 1 in mid-2020 with potential first regulatory approval and odevixibat launch in the second half of 2021.

Biliary atresia is a rare disease that impacts an estimated 15,000-20,000 people in the United States and European Union and is the leading cause of liver transplants among children. Albireo’s planned double-blind, placebo controlled pivotal trial in biliary atresia is designed to enroll approximately 200 patients at 70 sites globally. Patients will receive either placebo or high-dose (120 µg/kg) odevixibat once daily. The primary endpoint is survival with native liver after 2 years of treatment. The FDA and European Commission have granted orphan designations for odevixibat in the treatment of biliary atresia.

In addition, Albireo has been evaluating a proposed pivotal study design for odevixibat in Alagille syndrome in consultation with physicians and regulators, and plans to initiate the trial later this year. The FDA and European Commission have granted orphan designations for odevixibat in the treatment of Alagille syndrome.

The company had cash and cash equivalents at September 30, 2019 of $142.7 million, and continues to anticipate that its current cash balance will be sufficient to meet its operating needs into 2021.