Albireo Presents Data on Greater Efficacy in PFIC with Earlier Bylvay® Treatment at NASPGHAN

– New data presented at the North American Society for Pediatric Gastroenterology, Hepatology & Nutrition meeting (NASPGHAN)

– Earlier treatment with Bylvay (odevixibat) provided greater efficacy in children with PFIC

– Direct link between level of pruritus relief, liver function, and sleep-related outcomes

– Bylvay improved bile acids, sleep, growth, and quality of life even in patients with low pruritus

BOSTON, Oct. 13, 2022 (GLOBE NEWSWIRE) — 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 new data at the NASPGHAN Annual Meeting being held in Orlando, Florida from October 12-15, 2022. A new analysis of pooled data from the landmark Phase 3 PEDFIC 1 and PEDFIC 2 trials demonstrates that Bylvay (odevixibat) reduces serum bile acids and pruritus in children with progressive familial intrahepatic cholestasis (PFIC) who have varying levels of baseline hepatic impairment. Importantly, the analysis shows that significantly more patients with mild hepatic impairment at baseline had a serum bile acid response, suggesting children may benefit from earlier treatment with Bylvay.

Additional data analyses presented at the meeting show that Bylvay reduced pruritus in patients regardless of their baseline pruritus scores. Further, pruritus reductions were associated with improvements in serum bile acids, hepatic parameters, growth, and disease aspects related to sleep, with greater reductions in pruritus associated, in general, with greater improvements across these additional measures.

“These new data provide important evidence that treating children with Bylvay earlier in their disease course could result in greater efficacy,” said Jan Mattsson, Ph.D., Chief Scientific Officer and Head of R&D at Albireo. “Furthermore, the data we are presenting reinforce the medicine’s efficacy in patients with mild pruritus, suggesting patients’ benefit from beginning treatment at the first sign of itching.”

“If a physician is trying to determine when to start treatment with Bylvay, this new data suggest the earlier the better,” said Dr. Lorenzo D’Antiga, Department of Paediatric Hepatology, Gastroenterology, and Transplantation, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo. “We should consider treating patients at an early stage of the disease to preserve their native liver and provide relief from the incessant itching associated with the condition. When we see babies stop scratching and sleeping better, we see quality of life improvements for them and their parents.”

PFIC is a rare genetic disorder that causes progressive, life-threatening liver disease. Patients with PFIC have impaired bile flow, or cholestasis, and the resulting bile build-up in liver cells causes liver disease and symptoms, such as intense itching, poor sleep, delayed growth, and diminished quality of life. The harmful impacts of the disease extend to parents and caregivers, as the 2022 multinational PICTURE study revealed that PFIC negatively affects caregivers’ quality of life, relationships, and career prospects.

New Analyses of the PEDFIC 1 and PEDFIC 2 Trials
The global PEDFIC trials represent the largest studies ever completed in children with PFIC. New pooled data analyses from PEDFIC 1, a randomized, double-blind, placebo-controlled Phase 3 trial that evaluated the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids (sBAs) in children with PFIC, and PEDFIC 2, a long-term, open-label Phase 3 extension study, are being shared at NASPGHAN in an oral presentation and two poster presentations. An additional poster presents data showing that Bylvay may be mixed in liquids to give to the youngest patients:

Benefit to Treating Earlier with Bylvay

Oral Presentation: Hepatic Impairment Classifications at Baseline in Responders to Odevixibat Therapy in Children with Progressive Familial Intrahepatic Cholestasis
Lead Author: Dr. Lorenzo D’Antiga, Department of Paediatric Hepatology, Gastroenterology, and Transplantation, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy
Session Title: Concurrent Session V – Potent P’s in Hepatology: A daily double
Date & Time: Saturday, October 15, 2:00pm – 3:30pm ET

Pooled data analysis showed that treatment with Bylvay is associated with improvements in serum bile acids and pruritus and suggests greater efficacy when treatment with Bylvay occurs earlier in the disease. Significantly more patients with mild hepatic impairment at baseline, according to Child-Pugh scores, had a serum bile acid response than patients with more severe hepatic impairment at baseline. Among patients with mild and moderate hepatic impairment at baseline, 45% and 24% were Bylvay responders, respectively (p value=0.039). No patients had Child-Pugh scores of severe hepatic impairment at baseline. Across baseline hepatic impairment levels, pruritus response rates were comparable and Bylvay was generally well tolerated.

Direct Link Between Sustained Level of Pruritus Reduction and Magnitude of Improvement in Other Disease Parameters

Poster Abstract #301: Outcomes with Odevixibat in Patients with Progressive Familial Intrahepatic Cholestasis by Level of Pruritus Reduction: Pooled Analysis from the PEDFIC Trials
Lead Author: Dr. Ekkehard Sturm, Pediatric Gastroenterology and Hepatology, University Children’s Hospital Tübingen, Tübingen, Germany
Session Title: Poster Session II
Date & Time: Friday, October 14, 12:00pm – 2:30pm ET

An analysis of pooled data from 77 patients treated with Bylvay showed that pruritus reductions were associated with improvements in serum bile acids, hepatic parameters, growth, and disease aspects related to sleep and, in general, patients who had larger-magnitude pruritus reductions had larger-magnitude improvements in other outcomes. The median percentage change from baseline to weeks 70–72 in serum bile acid level was –12% in patients who had pruritus reductions of <1, –54% in patients who had reductions of ≥1 to <2, –94% in patients who had reductions of ≥2 to <3,–96% in patients who had reductions of ≥3 to <4, and –94% in patients who achieved a pruritus score of 0 or 1. Bylvay was generally well tolerated regardless of the level of pruritus reduction.

Evidence of Bylvay Efficacy in Patients with Lower Baseline Pruritus Severity

Poster Abstract #547: Outcomes in Patients with Progressive Familial Intrahepatic Cholestasis Treated with Odevixibat who had Medium or Lower Pruritus Severity at Baseline: Pooled Analysis from the PEDFIC 1 and PEDFIC 2 Studies
Lead Author: Dr. Kathleen M. Loomes, Children’s Hospital of Philadelphia, Philadelphia, PA, USA
Session Title: Poster Session III
Date & Time: Saturday, October 15, 12:00pm – 2:30pm ET

A pooled data analysis of 16 patients who had medium or lower pruritus severity at baseline showed that treatment with Bylvay was associated with improvement in serum bile acids, pruritus, hepatic parameters, growth, sleep, and quality of life (QoL), suggesting even patients with lower baseline pruritus severity may benefit from Bylvay treatment. Treatment with Bylvay led to a serum bile acid response in 31% of patients; improvements in mean percentage of days needing help falling asleep, falling from baseline of 41% to 18%; needing soothing falling from 37% to 19%; and sleeping with a caregiver falling from 36% to 16%; but not in percentage of days with scratching associated with bleeding, which was 9% at baseline and 30% at weeks 61-72. Bylvay was generally well tolerated.

Stability of Bylvay Mixed in Liquid for Pediatric Dosing

Poster Abstract #70: Stability of Odevixibat Oral Capsule Contents in Liquids
Lead Author: Dr. Prince Korah, Albireo, Boston, MA, USA
Session Title: Poster Session I
Date & Time: Thursday, October 13, 5:00pm – 7:00pm ET

An in vitro study showed that when Bylvay is mixed in a range of liquids often used to administer medicines to young infants, it is stable and provides children with an adequate dose. As the symptoms of PFIC often begin in infancy, when a child is not yet eating solid food, it is important that parents have an option to mix Bylvay in a liquid to give to their young children.

Albireo will also host a product theater featuring an expert perspective on treating PFIC patients with Bylvay:

Product Theater: Relief Made Possible with Bylvay^® (odevixibat)
Expert: Dr. Saeed Mohammad, Monroe Carell Jr. Children’s Hospital at Vanderbilt, Nashville, TN, USA
Date & Time: Friday, October 14, 1:45pm -2:15pm ET
Location: Promenade in front of Exhibit Hall

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. A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay has minimal systemic exposure and 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 open-label trial 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 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 a completed Phase 3 trial in Alagille syndrome (ALGS), an ongoing Phase 3 study in 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, Italy, and Belgium. 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 expected cash runway; 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 target indication(s) for development or approval; the timing for anticipated regulatory filings; discussions with the FDA or EMA regarding our programs; potential regulatory approval and plans for potential commercialization of Bylvay in biliary atresia or ALGS or Albireo’s other product candidates; the impact of the Expanded Access Program; 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: whether the regulatory filings to be made for Bylvay in patients with ALGS will be made on the timelines we expect and be approved by the FDA and EMA; whether the FDA and EMA will complete their respective reviews within target timelines, once determined; whether the FDA and EMA will require additional information, whether we will be able to provide in a timely manner any additional information that the FDA and EMA request, and whether such additional information will be satisfactory to the FDA and EMA; 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 or other approved indications 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 PFIC, ALGS and other indications, 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 (such as biliary atresia or ALGS) beyond the jurisdictions in which or indications for which Bylvay is currently approved; there is no guarantee that our other product 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 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; any repurchase by the Company of Sagard’s interest in the royalty interest payments under our royalty monetization agreement with Sagard could materially impact our financial condition; 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