Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, will be presenting five posters on Bylvay (odevixibat) at the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) meeting being held December 12–18. Data will be presented that show evidence of correlations in serum bile acid reductions and long-term improvements in pruritus and sleep in patients with progressive familial intrahepatic cholestasis (PFIC); reductions in serum bile acids and improvements in pruritus with and without concomitant ursodeoxycholic acid (UDCA) and/or rifampicin use; and the efficacy and safety of Bylvay in patients with PFIC and prior partial external biliary diversion (PEBD). Data on disease burden and natural history of PFIC will be presented to describe the clinical characteristics of the disease. There will also be a poster presentation on the Phase 3 double-blind, randomized, placebo-controlled ASSERT study of Bylvay in Alagille syndrome (ALGS). Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi) that 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.

“We are pleased to present additional data supporting Bylvay’s potential to reduce disease burden and improve the standard of care in the treatment of pruritus in PFIC,” said Jan Mattsson, Chief Scientific Officer at Albireo. “As we continue to see sustained correlations between the reduction of serum bile acids and long-term improvements in pruritus and quality of life measures, as well as certain results from the use of Bylvay with post-PEBD patients, we are encouraged that data could demonstrate Bylvay’s impact on disease modification over time.”

Bylvay PEDFIC 1 & 2 Treatment Data PEDFIC 1 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.

Poster of Distinction Poster #69: 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 CholestasisPoster Session I: Monday, December 13, 1:30-2:15pm ET

  • Significant correlations were observed between reductions in sBAs and reductions in pruritus and most sleep disturbance scores.
  • Percentage change in sBAs from baseline to weeks 49−72 was significantly correlated with change in pruritus scores during that interval (r=0.58; P<0.001).
  • Moderate correlations were also observed between percentage change in sBAs from baseline to weeks 49−72 and changes during that interval in caregiver-reported percentage of days where patients had bleeding associated with scratching, needed soothing or help falling asleep, and were sleeping with caregivers.
  • No serious drug-related treatment emergent adverse events (TEAEs) or deaths occurred. The overall incidence of any event of diarrhea was 21% and all instances were mild to moderate in severity and resolved, most without intervention.

Poster of Distinction Poster #78: Efficacy and Safety of Odevixibat Therapy With Concomitant UDCA or Rifampicin in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials Poster Session I: Monday, December 13, 1:30-2:15pm ET

  • Patients with PFIC receiving Bylvay treatment experienced reductions in sBAs and improvements in pruritus with and without concomitant ursodeoxycholic acid (UDCA) and/or rifampicin use.
  • After 48 weeks of Bylvay treatment, percentages of patients meeting criteria for sBA response were similar among patients using vs not using UDCA and/or rifampicin (using vs not using, each 67%)
    • 74% and 40% in patients using vs not using UDCA, respectively.
    • 54% and 82% in patients using vs not using rifampicin.
  • Mean proportions of positive pruritus assessments (PPAs) were similar in patients using vs not using UDCA (65% vs 72%, respectively) and in patients using vs not using UDCA and/or rifampicin (66% vs 69%); mean proportions of PPAs in patients using vs not using rifampicin (56% and 81%).
  • Safety and tolerability were comparable in patients using vs not using UDCA and/or rifampicin with Bylvay. Incidence of TEAEs was similar in patients using vs not using UDCA and/or rifampicin (78% vs 90%, respectively), as well as in patients using vs not using UDCA (77% vs 87%) and rifampicin (75% vs 86%).

Poster #303: Efficacy And Safety Of Odevixibat In Children With Progressive Familial Intrahepatic Cholestasis With Prior Partial External Biliary Diversion Poster Session II: Wednesday, December 15, 1:30-2:15pm ET

  • Improvements were observed in sBA levels, pruritus, quality of life (QoL), and sleep parameters in some patients who had prior partial external biliary diversion, or PEBD surgery. Improvements were observed despite elevated sBAs and pruritus in these patients at baseline, suggesting that some patients with poor response to PEBD could potentially respond to Bylvay treatment.
  • All of the 10 patients with prior PEBD had elevated sBAs and pruritus scores prior to the first dose of Bylvay, indicating that prior PEBD surgery was unsuccessful or only partially successful.
    • 7 patients had reductions in pruritus score and 4 met criteria for pruritus response (i.e., ≥1-point drop in pruritus score).
    • 5 patients had reductions in sBA levels; 1 patient, who also met pruritus response criteria, met criteria for sBA response (i.e., sBAs reduced by ≥70% or levels ≤70 μmol/L) at last assessment.
    • Of the 9 patients with post-baseline QoL assessments, 6 had improved observer-reported PedsQL total scores, including all 4 pruritus and/or sBAs responders.
    • Pruritus and/or sBA responders also had reductions from baseline to last assessment in percentage of days with bleeding associated with scratching, needing soothing or help falling asleep, and sleeping with caregivers.
  • TEAEs, including mild abdominal pain, were observed in the subgroup of patients with prior PEBD; all TEAEs were mild to moderate in severity, and no patients discontinued due to a TEAE.

Poster #292: Disease Burden and Natural History of Progressive Familial Intrahepatic Cholestasis: Baseline Clinical Characteristics Among Odevixibat-Treated Patients in the Phase 3 PEDFIC Studies Poster Session II: Wednesday, December 15, 1:30-2:15pm ET

  • Baseline characteristics of the patient population studied reflect the broader population of patients with PFIC; specifically this is a pediatric population with cholestasis, impaired hepatic function, growth deficits, and impacted sleep parameters; and PFIC had a considerable impact on the QoL of patients and their caregivers.
  • The complexity of PFIC was illustrated by the heterogeneity and severity of the clinical signs and symptoms in these patients with different types of PFIC.

Bylvay in ASSERT StudyPoster #281: The ASSERT Study: A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat In Patients With Alagille SyndromePoster Session II: Wednesday, December 15, 1:30-2:15pm ET

  • ASSERT is an on-going, global Phase 3 pivotal trial designed to evaluate the safety and efficacy of Bylvay for 24 weeks in relieving pruritus in patients with Alagille syndrome.
  • ASSERT is expected to enroll approximately 63 patients, including approximately 45 patients aged <18 years and an additional exploratory cohort of up to 18 patients aged ≥18 years. 

Full scientific abstracts are available and can be viewed here: Journal of Pediatric Gastroenterology and Nutrition.

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). 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 for sale in Germany 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 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 Alagille syndrome, 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 Alagille syndrome.

About AlbireoAlbireo 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 and biliary atresia, as well as an Open-label Extension (OLE) study for PFIC. In Europe, Bylvay has been approved for the treatment of PFIC and has been submitted for pricing and reimbursement approval. The Company has also initiated a Phase 1 clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies moving ahead 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. The Boston Business Journal named Albireo one of the 2019 and 2020 Best Places to Work in Massachusetts. 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 and expectations for commercializing Bylvay in the U.S. and Europe; 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 pivotal trial for Bylvay in biliary atresia (BOLD); the pivotal trial for Bylvay in Alagille syndrome (ASSERT); the Phase 1 trial for A3907; the IND-enabling studies for A2342; the target indication(s) for development or approval; the size, design, population, location, conduct, cost, objective, enrollment, duration or endpoints of any clinical trial, or 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, Phase 1 trial for A3907 and the IND-enabling studies for A2342; potential regulatory approval by and discussions with the FDA or EMA regarding our programs; the potential benefits or competitive position of Bylvay or any other Albireo product candidate or program or the commercial opportunity in any target indication; the potential effects of Bylvay of the treatment of PFIC patients and its potential to improve the current standard of care; 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; 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 1 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

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

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