ALBO Albireo Pharma Inc

Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company developing novel bile acid modulators to treat pediatric and adult liver diseases, today announced the presentation of data at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting® 2022, being held November 4 – 8, 2022 in Washington, D.C. Data to be presented include one oral presentation and five posters demonstrating long-term data on the efficacy of Bylvay in patients with progressive familial intrahepatic cholestasis (PFIC). Albireo will also present one abstract highlighting data on A3907, the Company’s ASBT inhibitor in clinical development for the treatment of adult liver disease. A full list of presentations can be found on The Liver Meeting Digital Experience™ 2022 website.

Pediatric Liver Presentations

Oral Presentation (Abstract #865): Native Liver Survival in Odevixibat sBA Responders: Data from the PEDFIC Studies in Patients with Progressive Familial Intrahepatic CholestasisPresenter: Dr. Richard J. Thompson, Institute of Liver Studies, King’s College LondonSession Title: Genes to Cures: What’s New in Pediatric Liver DiseaseDate & Time: Sunday, November 6, 2:10 PM EST

Poster (Abstract #37273): Effect of Odevixibat in Patients with Progressive Familial Intrahepatic Cholestasis Type 2 with at Least 1 Severe Mutation (BSEP3 Compound Heterozygotes): Pooled Data from the PEDFIC 1 and PEDFIC 2 StudiesPresenter: Dr. Henkjan J. Verkade, Department of Paediatrics, University of Groningen, Beatrix Children’s Hospital/University Medical Centre Groningen Session Title: Poster Session IVDate & Time: Monday, November 7, 1:00 PM – 2:00 PM EST

Poster (Abstract #37939): Long-term Efficacy and Safety of Odevixibat in Patients with Progressive Familial Intrahepatic Cholestasis: Results with 96 Weeks or More of TreatmentPresenter: Dr. Richard J. Thompson, Institute of Liver Studies, King’s College London Session Title: Poster Session IVDate & Time: Monday, November 7, 1:00 PM – 2:00 PM EST

Poster (Abstract #850): Odevixibat Treatment in Patients with Recurrent Episodic Cholestasis and Biallelic Mutations in ATP8B1: A Retrospective Case SeriesPresenter: Dr. Angelo Di Giorgio, Hospital Papa Giovanni XXIII, Bergamo, Italy   Session Title: Poster Session IVDate & Time: Monday, November 7, 1:00 PM – 2:00 PM EST

Poster (Abstract #37608): Odevixibat Therapy in Patients with FIC1-Deficient Progressive Familial Intrahepatic Cholestasis and Diarrhea Following Liver Transplantation That Impacted Daily Activities: A Retrospective Case SeriesPresenter: Georg-Friedrich Vogel, Assistant Professor, Medical University of Innsbruck Innsbruck, AustriaSession Title: Poster Session IVDate & Time: Monday, November 7, 1:00 PM – 2:00 PM EST

Poster (Abstract #37254): Serum Bile Acid Levels, Pruritus Scores, and Growth Over Time in Odevixibat Responders: Pooled Data from the PEDFIC Studies in Patients with Progressive Familial Intrahepatic Cholestasis Presenter: Dr. Lorenzo D'Antiga, Department of Paediatric Hepatology, Gastroenterology, and Transplantation, Azienda Ospedaliera Papa Giovanni XXIIISession Title: Poster Session IVDate & Time: Monday, November 7, 1:00 PM – 2:00 PM EST

Adult Liver Presentation

Poster (Abstract #37733): Systemic ASBT Inhibition with A3907 Stimulates Urinary Excretion of Bile Acids and Halts Liver Disease Progression in Bile-Duct–Obstructed Mice Presenter: Francisco J. Caballero-Camino, Instituto de Investigación Sanitaria Biodonostia, San Sebastián, Basque Country, Spain Session Title: Poster Session IIIDate & Time: Sunday, November 6, 1:00 PM – 2:00 PM

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 Italy, Germany, Belgium, 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 A3907

A3907 is the first oral systemic apical sodium-dependent bile acid transporter (ASBT) inhibitor with high oral bioavailability that has potential to inhibit intestinal and renal bile acid reuptake as well as ASBT expressed by cholangiocytes. Due to high oral bioavailability, A3907 can inhibit ASBT in the intestine and kidney, with the potential to increase elimination of bile acids by both fecal and urinary excretion. By using dual pathway diversion of bile acids, next generation modulators like A3907 seek to increase efficacy without the dose limiting diarrhea seen with bile acid transport inhibitors today. A3907 is being developed for adult cholestatic liver diseases such as primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC).

About Albireo

Albireo Pharma is a rare disease company focused on the development of novel bile acid modulators to treat 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 is reimbursed for the treatment of PFIC in Germany, England, Wales & Northern Ireland, Scotland, Belgium, and Italy. 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, A3907, A2342 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); the Phase 2 study for A3907 the IND-enabling or clinical studies for 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, the Phase 2 study for A3907, and the IND-enabling and clinical studies for A2342; the impact of the Expanded Access Program; expectations that biliary atresia is the most common pediatric cholestatic liver disease with no approved drug treatment; 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; 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; 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 Phase 2 clinical trial of A3907, 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 Contacts: 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