Press Release Details

– 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 (GLOBE NEWSWIRE) -- 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.”

About Bylvay (odevixibat)
Bylvay is the first drug approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of progressive familial intrahepatic cholestasis (PFIC). Limitation of Use: Bylvay may not be effective in PFIC type 2 patients with ABCB11 variants resulting in non-functional or complete absence of bile salt export pump protein (BSEP-3). The European Commission (EC) and UK Medicines and Healthcare Products Regulatory Agency (MHRA) have also granted marketing authorization of Bylvay for the treatment of PFIC in patients aged 6 months or older. Bylvay is available in Germany and the UK and will be available for sale in other European countries following pricing and reimbursement approval. A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay acts locally in the small intestine. Bylvay can be taken as a capsule for patients that are able to swallow capsules, or opened and sprinkled onto food, which is a factor of key importance for adherence in a pediatric patient population. The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. The medicine can only be obtained with a prescription. For more information about using Bylvay, see the package leaflet or contact your doctor or pharmacist. For full prescribing information, visit www.bylvay.com.

In the U.S. and Europe, Bylvay has orphan exclusivity for its approved PFIC indications, and orphan designations for the treatment of ALGS, biliary atresia and primary biliary cholangitis. Bylvay is being evaluated in the ongoing PEDFIC 2 open-label trial in patients with PFIC, in the BOLD Phase 3 study for patients with biliary atresia and the ASSERT Phase 3 study for ALGS.

Important Safety Information

• The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.
• Liver Test Abnormalities: Patients should obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be required if abnormalities occur. For persistent or recurrent liver test abnormalities, consider treatment discontinuation.
• Diarrhea: Treat dehydration. Treatment interruption or discontinuation may be required for persistent diarrhea.
• Fat-Soluble Vitamin (FSV) Deficiency: Patient should obtain baseline vitamin levels and monitor during treatment. Supplement if deficiency is observed. If FSV deficiency persists or worsens despite FSV supplementation, discontinue treatment.

About Albireo
Albireo Pharma is a rare disease company focused on the development of novel bile acid modulators to treat rare pediatric and adult liver diseases. Albireo’s lead product, Bylvay, was approved by the U.S. FDA as the first drug for the treatment of pruritus in all types of progressive familial intrahepatic cholestasis (PFIC), and it is also being developed to treat other rare pediatric cholestatic liver diseases with Phase 3 trials in Alagille syndrome (ALGS) and biliary atresia, as well as Open-label Extension (OLE) studies for PFIC and ALGS. In Europe, Bylvay has been approved for the treatment of PFIC with pricing listing in Germany and guidance from the National Institute for Health and Care Excellence (NICE) recommending Bylvay for use in the National Health Service in England, Wales and Northern Ireland. The Company has also completed a Phase 1 clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies progressing with A2342 for viral and cholestatic liver disease. Albireo was spun out from AstraZeneca in 2008 and is headquartered in Boston, Massachusetts, with its key operating subsidiary in Gothenburg, Sweden. For more information on Albireo, please visit www.albireopharma.com.

Forward-Looking Statements
This press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements, other than statements of historical fact, regarding, among other things: Albireo’s commercialization plans; the plans for, or progress, scope, cost, initiation, duration, enrollment, results or timing for availability of results of, development of Bylvay, or any other Albireo product candidate or program; the PEDFIC 2 open-label trial in patients with PFIC; the pivotal trial for Bylvay in biliary atresia (BOLD); the pivotal trial for Bylvay in Alagille syndrome (ASSERT); clinical trials for A3907 and A2342, the target indication(s) for development or approval; the timing for initiation or completion of or availability or reporting of results from any clinical trial, including the long-term open-label extension study for Bylvay in PFIC, the BOLD and ASSERT trials, or A3907 or A2342 trials, potential regulatory approval and plans for potential commercialization of Bylvay in additional countries; the potential benefits or competitive position of Bylvay or any other Albireo product candidate or program or the commercial opportunity in any target indication; Bylvay’s funding for use in the National Health Service in England, Wales and Northern Ireland; future price listings and reimbursement approvals of Bylvay; the length of time for which Albireo’s cash resources are expected to be sufficient; or Albireo’s plans, expectations or future operations, financial position, revenues, costs or expenses. Albireo often uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “planned,” “continue,” “guidance,” or the negative of these terms or other similar expressions to identify forward-looking statements. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to: there are no guarantees that Bylvay will be commercially successful; we may encounter issues, delays or other challenges in commercializing Bylvay; whether Bylvay receives adequate reimbursement from third-party payors; the degree to which Bylvay receives acceptance from patients and physicians for its approved indication; challenges associated with execution of our sales activities, which in each case could limit the potential of our product; challenges associated with supply and distribution activities, which in each case could limit our sales and the availability of our product; results achieved in Bylvay in the treatment of patients with PFIC may be different than observed in clinical trials, and may vary among patients; other potential negative impacts of the COVID-19 pandemic, including on manufacturing, supply, conduct or initiation of clinical trials, or other aspects of our business; whether favorable findings from clinical trials of Bylvay to date, including findings in indications other than PFIC, will be predictive of results from other clinical trials of Bylvay; there is no guarantee that Bylvay will be approved in jurisdictions or for indications beyond the jurisdictions in which or indications for which Bylvay is currently approved; there is no guarantee that our other products candidates will be approved; estimates of the addressable patient population for target indications may prove to be incorrect; the outcome and interpretation by regulatory authorities of the ongoing third-party study pooling and analyzing of long-term PFIC patient data; the timing for initiation or completion of, or for availability of data from, clinical trials of Bylvay, including BOLD and ASSERT and the clinical trials of A3907 and A2342, and the outcomes of such trials; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the United States or Europe; delays or other challenges in the recruitment of patients for, or the conduct of, the Company’s clinical trials; and the Company’s critical accounting policies. These and other risks and uncertainties that Albireo faces are described in greater detail under the heading “Risk Factors” in Albireo’s most recent Annual Report on Form 10-K or in subsequent filings that it makes with the Securities and Exchange Commission. As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur. Albireo cautions you not to place undue reliance on any forward-looking statement. In addition, any forward-looking statement in this press release represents Albireo’s views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Albireo disclaims any obligation to update any forward-looking statement except as required by applicable law.

Media Contact:
Colleen Alabiso, 857-356-3905, colleen.alabiso@albireopharma.com
Lance Buckley, 917-439-2241, lbuckley@lippetaylor.com

Investor Contact:
Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578

Source: Albireo Pharma, Inc.