Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, today announced the publication of the PICTURE study in Orphanet Journal of Rare Disease. A multinational, retrospective, cross-sectional study, PICTURE evaluated and quantified the impact that progressive familial intrahepatic cholestasis (PFIC) has on caregivers and found a significant caregiver-reported burden on health-related quality of life (HRQoL), impairment of daily activities, reduced sleep, impact on work productivity, career building challenges and relationship strain. The study illustrates for the first time that PFIC levies a substantial burden that extends beyond the individuals with the disease to those caring for them.

“PFIC is a devastating diagnosis. As a mother of a child with PFIC, I could not ask for a greater gift than being my daughter’s advocate. With that said, the PICTURE study reinforces what many of us intuitively know – the challenges of caregiving are ever present and often overwhelming and exhausting,” said Emily Ventura, Chief Executive Director of PFIC Network, co-author of the PICTURE study and caregiver. “From loss of sleep to stress on finances and relationships, PICTURE reinforces that we must recognize the burden this disease puts on families and the importance of providing resources for PFIC caregivers, while continuing to fund research to improve treatment and care.”

PFIC is a spectrum of rare, pediatric, genetic diseases of cholestasis, characterized by inadequate bile secretion, often requiring liver transplantation, and leading to liver failure and early death. The rare nature of PFIC has presented challenges to understanding and quantifying its impact on the daily lives of patients and their caregivers. The PICTURE study is the first and largest global study to evaluate and quantify the health-related quality of life responses from PFIC caregivers. The study showed that while caregivers report feeling fulfillment from their caregiving responsibilities, they also reported measurable negative impacts on many important health-related quality of life measures and work productivity. Specifically, the study found that:

  • PFIC substantially impacted caregivers’ quality of life
    • 82% of caregivers reported a strain on their relationships.
    • 86% of caregivers reported difficulty sleeping.
    • Notably, caregivers in the PICTURE study reported a median quality of life score of 67.7%. This score is lower in comparison with quality of life reported in a separate study of caregivers for individuals with cystic fibrosis, a similarly rare, genetic, progressive and lifespan-shortening condition (median 84.7 and 89.2 for mothers and fathers, respectively).
  • On average, PFIC caregivers reported high impairment in daily activities; more than half also experienced loss of work productivity
    • 50% of caregivers reported an impact of their child’s PFIC on their career-building efforts, with 73% of these citing the prevention of either their career progression or working more hours.
    • A third (36%) of caregivers reported missing an average of 13 workdays in the last three months, equating to 52 workdays lost in 12 months.
    • Of those who were working in paid employment, 36% stopped work due to caregiving needs, missing an average of 2.8 years of employment during their career.

“The PICTURE study provides, for the first time, a look at what it’s really like for caregivers to live with the impact of this devastating, rare disease and the effect it has every day on families and adjacent communities,” said Ron Cooper, President and CEO of Albireo. “At Albireo, we are committed to being the gold standard partner in the fight against PFIC and the findings from the PICTURE study reinforce the importance of providing access to Bylvay, the first approved drug treatment for patients with PFIC.”

PFIC is a rare and devastating disorder affecting young children 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 and diminished quality of life. Albireo is committed to advancing research in PFIC and other rare cholestatic diseases. The Company recently received approval by the U.S. FDA for Bylvay, the first drug for the treatment of pruritus in all types of PFIC, and in Europe, Bylvay is approved for the treatment of PFIC. The Company is working with the community and patient advocacy groups like the PFIC Network and Children’s Liver Disease Foundation to raise awareness of PFIC and support families managing the burden of this devastating disease.

About the PICTURE Study The PICTURE study was a cross-sectional burden of illness study of physician and caregiver-reported information for 22 patients with PFIC type 1 or 2 in France, Germany, the United Kingdom and the United States from September 2020 through to March 2021. Physicians provided clinical and resource use data of PFIC patients at the time of consultation, via an electronic Case Report Form (eCRF). Caregivers of PFIC patients, recruited by the physician as they attended a clinical appointment with the patient, completed online specific self-completion questionnaires about the impact of the disease on their lives. The study was conducted under the guidance of an Expert Reference Group (ERG), consisting of a representative of academia as principal investigator, partnering charity and advocacy representatives as well as experts in the field of liver diseases. Funding for this study was provided by Albireo Pharma.

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 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 https://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 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 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 with pricing listing in Germany and draft guidance from the National Institute for Health and Care Excellence (NICE), which recommends Bylvay for use in the National Health Service in the England, Wales and Northern Ireland UK. 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. 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: 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 IND-enabling 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 and the IND-enabling studies for A2342; or the potential benefits or competitive position of Bylvay or any other Albireo product candidate or program or the commercial opportunity in any target indication; 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 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 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.comLance Buckley, 917-439-2241, lbuckley@lippetaylor.com

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

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