Albireo Presents New Bylvay® (odevixibat) Data at Annual ESPGHAN Congress

• Four presentations highlight newest data from landmark Phase 3 PEDFIC 1 and PEDFIC 2 trials, largest studies completed in PFIC
• New analysis show potential for greater response and higher proportion of pruritus responders with dose escalation and consistent tolerability across doses
• Long-term data provide evidence of consistent improvements in hepatic parameters, growth, sleep, and biomarkers in patients with all PFIC types with treatment up to 72 weeks

BOSTON, June 24, 2022 (GLOBE NEWSWIRE) — Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, today announced the presentation of new data at the annual meeting of the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) being held in Copenhagen, Denmark. The data provided new analyses of the Phase 3 PEDFIC 1 and PEDFIC 2 trials of Bylvay^® (odevixibat), demonstrating that dose escalation can provide greater benefits in relieving patients’ pruritus, with similar tolerability, as well as showing that reductions in pruritus are associated with important improvements in sleep and other quality of life measures in children with all types of progressive familial intrahepatic cholestasis (PFIC).

“These data analyses, from the largest studies ever completed in children with PFIC, reinforce that when you give patients’ relief from their pruritus, you can dramatically improve many aspects of their life and that of their families – perhaps most importantly resulting in better sleep,” said Jan Mattsson, Ph.D., Chief Scientific Officer and Head of R&D. “The data also provide physicians insight into dose escalation, providing them confidence that they can use a higher dose of Bylvay to increase patients’ pruritus relief and help more patients become pruritus responders, with a tolerability profile that is similar to that seen with a lower dose.”

Bylvay PEDFIC 1 and PEDFIC 2 Data
Bylvay is a potent, non-systemic once daily ileal bile acid transport inhibitor (IBATi) that is the only treatment approved in Europe for the treatment of all types of PFIC in patients aged 6 months or older and the only treatment approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of PFIC. The PEDFIC 1 trial was 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 (sBAs) in a randomized, double-blind, placebo-controlled trial, and PEDFIC 2 is a long-term, open-label Phase 3 extension study.

Evidence of Improved Pruritus Response with Bylvay Dose Escalation

• Oral Presentation: Efficacy and Safety Outcomes in Patients with Progressive Familial Intrahepatic Cholestasis Who Had an Odevixibat Dose Escalation: Pooled Results from the PEDFIC 1 and PEDFIC 2 Studies. In the pooled analysis population (N=84), 21 patients with PFIC treated with Bylvay who had a treatment response on 40 μg/kg/day maintained their response and/or had further improvements with dose escalation to 120 μg/kg/day. Among those with dose escalation, additional patients became pruritus responders at the higher dose and some non-responders became pruritus responders at the higher dose in the PEDFIC studies. Bylvay was generally well tolerated throughout dose escalation. In patients who received 40 μg/kg/day of Bylvay during PEDFIC 1 and escalated to 120 μg/kg/day in PEDFIC 2, the majority of TEAEs were mild or moderate in severity.

Long-Term Data Demonstrate Efficacy of Bylvay Across PFIC Subtypes

• Oral Presentation: Analysis of Quality of Life, Hepatic Biochemical Markers, and Sleep in Patients With Progressive Familial Intrahepatic Cholestasis Who Had a Pruritus Response With Odevixibat Treatment. Results of pooled analysis population of 82 patients treated with Bylvay including 44 pruritus responders of PEDFIC 1 and PEDFIC 2 show that patients across various types of PFIC who had a pruritus response with Bylvay experienced improvements in quality of life, sleep and hepatic biomarkers that were sustained for up to 72 weeks. From baseline to week 72, pruritus responders had significant quality of life improvements, as assessed by mean PedsQL total scores (p=0.048) and PedsQL FI total scores (p=0.007). Data also indicated that pruritus responders had significant improvements in several caregiver-reported sleep parameters (all P<0.001), including reductions in days with blood due to scratching, days needing help falling asleep, days needing soothing, and days sleeping with caregiver. Among all pruritus responders, 86% had any TEAE, none of which were drug related serious TEAEs.​Two patients experienced TEAEs that led to study discontinuation, which included splenomegaly, diarrhoea, jaundice, decreased weight, and/or hypophagia​.
• Oral Presentation: Changes in Hepatic Parameters, Growth, Sleep, and Biochemical Markers With Odevixibat Treatment Across Patients With Various Types of Progressive Familial Intrahepatic Cholestasis. Patients with all types of PFIC treated with Bylvay for up to 72 weeks, with some tracked up to 128 weeks, experienced reductions in autotaxin levels and increases in C4 levels, as well as variable changes in hepatic parameters, sleep characteristics and growth. 84 patients received Bylvay during the pooled analysis period. Patients of almost all PFIC types experienced mean improvements in caregiver-reported sleep parameters. Patients of most PFIC types generally had mean increases in height and/or weight Z scores with Bylvay treatment; patients with PFIC1 and PFIC3 experienced more variable changes in weight Z scores. Among all patients, 85% had any TEAE, which was similar across all PFIC types and mild or moderate in severity​. All serious TEAEs were assessed as unrelated to study drug.

Evidence of Bylvay Efficacy, With and Without Concomitant UDCA

• e-Poster Abstract #363: Total, Primary, and Secondary Serum Bile Acid Concentrations in Patients With Progressive Familial Intrahepatic Cholestasis With Serum Bile Acid Response or Not With Odevixibat Treatment: Assessing the Contribution of Ursodeoxycholic Acid Concentration. A large proportion of patients in the PEDFIC 1 trial were on UDCA at baseline, but still had elevated sBAs and pruritus. This analysis assessed whether excluding UDCA concentrations from total sBA measurements altered the proportion of patients who had a sBA response to Bylvay and showed no appreciable changes. Moreover, patients who had a sBA response experienced large reductions in sBAs, whether or not they were on concomitant UDCA.

Albireo will also be hosting a company-sponsored symposium that will feature case-based expert discussion on real-life experience of treating PFIC with an IBAT inhibitor. Details for attendance:

Breakfast Symposium: Real-life experience of treating PFIC with an IBAT inhibitor
Expert panel: Prof. Patrick McKiernan, Birmingham Children’s Hospital, NHS Foundation Trust, UK, Dr. Christoph Leiskau, Hannover Medical School, Germany, and Dr. Angelo Di Giorgio, Hospital Papa Giovanni XXIII, Bergamo, Italy
Date & Time: Saturday, 25 June, 7:15-8:15 CEST

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, 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; 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;; 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: 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; 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 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