Albireo Presented Data in Cholestatic Liver Diseases at Digestive Disease Week (DDW) 2022

– Six abstracts presented highlighting data on Bylvay^TM (odevixibat) and effects on pruritus, serum bile acids, growth and sleep parameters

– Pre-clinical study of systemic ASBT/NTCP inhibitors, shows potential of dual-acting ileal/kidney and liver bile acid modulators for cholestatic liver diseases

BOSTON, May 23, 2022 - Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, today announced that it presented analyses of its pivotal PEDFIC 1 and PEDFIC 2 studies which evaluated Bylvay for the treatment of progressive familial intrahepatic cholestasis (PFIC), at the Digestive Disease Week Annual Meeting held May 21-24, 2022. Also presented at the meeting, were new results from a preclinical study showing the potential of the company’s investigational dual-acting ileal/kidney and liver bile acid inhibitors to significantly increase bile acid excretion. All abstracts are available on the DDW website.

“The PEDFIC 1 and PEDFIC 2 Phase 3 clinical trials represent the largest body of data ever collected in PFIC, a rare disease that causes tremendous suffering for patients and their families,” said Ron Cooper, President and Chief Executive Officer of Albireo. “We’re pleased to share data demonstrating Bylvay’s efficacy on a wide range of all types of PFIC as well as positive impact on all levels of pruritus in PFIC.”

Bylvay is a potent, non-systemic once daily ileal bile acid transport inhibitor (IBATi) which is 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, based on the PEDFIC 1 study and PEDFIC 2 open-label extension study.

Bylvay PEDFIC 1 & 2 Treatment Data
The following data presentations at DDW provided analyses from PEDFIC 1, the first and largest, global, pivotal Phase 3 study conducted in PFIC, which evaluated the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids in a randomized, double-blind, placebo-controlled trial, and PEDFIC 2, a long-term, open-label Phase 3 extension study.

E-poster #1229: Relationships Between Decreases in Serum Bile Acids, Pruritus, and Sleep Disturbance Scores with up to 72 Weeks of Odevixibat Treatment in Patients with Progressive Familial Intrahepatic Cholestasis
Lead Author: Dr. Buket Dalgic, Department of Pediatric Gastroenterology, Gazi University Faculty of Medicine

E-poster #1230: Efficacy and Safety of Odevixibat in Children with Progressive Familial Intrahepatic Cholestasis with Prior Partial External Biliary Diversion
Lead Author: Dr. Binita M. Kamath, Hospital for Sick Children and the University of Toronto

E-poster #1231: Odevixibat Therapy Improves Clinically Meaningful Endpoints in Children with Progressive Familial Intrahepatic Cholestasis: Data from the PEDFIC 1 and PEDFIC 2 Trials
Lead Author: Dr. Richard J. Thompson, Institute of Liver Studies, King’s College London

E-poster #1232: Odevixibat Effects on Cholestasis-Related Parameters: Analysis of Pooled Data from the PEDFIC 1 and PEDFIC 2 Studies in Children with Progressive Familial Intrahepatic Cholestasis
Lead Author: Dr. Richard J. Thompson, Institute of Liver Studies, King’s College London

E-poster #1233: Long-Term Treatment with Odevixibat Improves Multiple Sleep Parameters in Patients with Progressive Familial Intrahepatic Cholestasis: A Pooled Responder Analysis from the Phase 3 PEDFIC Studies
Lead Author: Dr. Richard J. Thompson, Institute of Liver Studies, King’s College London

E-poster #1228: Disease Burden and Natural History of Progressive Familial Intrahepatic Cholestasis: Baseline Clinical Characteristics Among Odevixibat-Treated Patients in the Phase 3 PEDFIC Studies
Lead Author: Dr. Girish Gupte, Liver Unit and Small Bowel Transplantation, Birmingham Women’s and Children’s NHS Foundation Trust

Presentation on Dual-Acting Bile Acid Transport Inhibitors
E-poster #1327: Dual Acting Ileal/Renal-Liver Bile acid Transporter Inhibitors Significantly Increase Urinary Excretion of Non-Sulfated Bile Acids in a Diethoxy-Carbonyl-Dihydro-Collidine-Induced Mouse Model of Cholestasis
Lead Author: Dr Anuradha Rao, Department of Pediatrics, Emory University School of Medicine, Atlanta.

“The ASBT and NTCP bile acid transporters play important roles in maintaining bile acid homeostasis,” said Dr. Jan Mattsson, Ph.D., Chief Scientific Officer and Co-Founder of Albireo. “This study shows that dual-acting ASBT/NTCP with different selectivity may represent an attractive strategy to reduce bile acid burden in hepatobiliary diseases, reinforcing the potential of our A3907 and A2342 programs as well as our earlier staged novel bile acid modulators.”