BOSTON, Jan. 22, 2021 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver disease company developing novel bile acid modulators, today joins the Alagille community in commemorating the second annual International Alagille Syndrome Awareness Day on January 24, 2021 in honor of Dr. Daniel Alagille's birthday. Alagille syndrome (ALGS) is a rare genetic disorder that can affect the liver, heart and other parts of the body. Albireo leaders reaffirm the Company’s commitment to advancing life-changing research and join the community in bringing greater awareness to patients and families affected by this rare and devastating disorder.
“Connecting with families who manage Alagille syndrome and hearing firsthand the burdens associated with the disease reaffirms our mission at Albireo, which is to provide hope and improve treatment options and outcomes for children and families with rare cholestatic liver diseases,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Their feedback has been invaluable in helping design our Phase 3 study in Alagille syndrome, called ASSERT, which is currently enrolling eligible patients.”
Alagille syndrome Alliance (ALGSA) is an international advocacy group representing patients and families. The nonprofit facilitates in-person and digital forums to increase community education and awareness among families, healthcare providers and scientists. ALGSA also provides assistance programs to families in need and sponsors grants to advance scientific research. To recognize International Alagille Syndrome Awareness Day, the organization will be hosting a 24-hour “Aware-A-Thon” with the objective of connecting families in raising awareness of ALGS through sharing stories via social media. For more information, visit www.alagille.org, or find the ALGSA on social media under @ALGSAlliance.
“The Alagille syndrome community is one of tremendous courage, determination and camaraderie. Our support groups with families spanning the globe are filled with people dealing with all phases and aspects of the syndrome. They encourage, lift up, lean on, educate and help one another in so many ways,” said Roberta Smith, President of Alagille Syndrome Alliance. “In honor of our families, International Alagille Syndrome Awareness Day is a day to elevate the patient voice, with focus on disease awareness and highlighting Alagille education resources for all stakeholders as well as new families stepping into this complex rare disease diagnosis.”
Alagille syndrome (ALGS) is a rare, multisystem genetic disorder that can affect the liver, heart, skeleton, eyes, central nervous system, kidneys and facial features. Liver damage is caused by a paucity of bile ducts preventing bile flow from the liver to the small intestine. Approximately 95% of patients with ALGS present with chronic cholestasis, usually within the first three months of life, and up to 88% also present with severe, intractable pruritus. Currently, there are no approved drug treatments.
Albireo Pharma is a clinical-stage biopharmaceutical company focused on the development of novel bile acid modulators to treat rare pediatric and adult liver diseases, and other adult liver diseases and disorders. Albireo’s lead product candidate, odevixibat, is being developed to treat rare pediatric cholestatic liver diseases. The company recently completed U.S. and EU regulatory submissions for odevixibat in patients with progressive familial intrahepatic cholestasis (PFIC), and anticipates potential regulatory approvals, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021. Odevixibat is also currently being evaluated in the ASSERT Phase 3 trial in ALGS, BOLD Phase 3 trial in patients with biliary atresia and the ongoing PEDFIC 2 Phase 3 open-label trial in patients with PFIC. The Company also completed IND-enabling studies for new preclinical candidate A3907 and plans to advance development in adult 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 2020 Best Places to Work in Massachusetts for the second consecutive year. For more information on Albireo: www.albireopharma.com.
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 odevixibat or any other Albireo product candidate or program, including regarding expectations regarding the impact of COVID-19 on our business and our ability to adapt our approach as appropriate; the Phase 3 clinical program for odevixibat in patients with PFIC, the pivotal trial for odevixibat in biliary atresia (BOLD), and the pivotal trial for odevixibat in Alagille syndrome (ASSERT); 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 odevixibat in PFIC, the pivotal trial for odevixibat in biliary atresia, the pivotal trial for odevixibat in Alagille syndrome; the potential approval and commercialization of odevixibat; discussions with the FDA or EMA regarding our programs; the potential benefits or competitive position of odevixibat or any other Albireo product candidate or program or the commercial opportunity in any target indication; the potential effects of odevixibat of the treatment of PFIC patients and its potential to improve the current standard of care; the potential benefits of an orphan drug designation; the potential issuance of a rare pediatric disease priority review voucher; 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,” and 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: 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 odevixibat to date, including findings in indications other than PFIC, will be predictive of results from other clinical trials of odevixibat; whether either or both of the FDA and EMA will determine that the primary endpoint for their respective evaluations and treatment duration of the double-blind Phase 3 trial in patients with PFIC are sufficient to support approval of odevixibat in the United States or the European Union, to treat PFIC, a symptom of PFIC, a specific PFIC subtype(s) or otherwise; 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 odevixibat, including the pivotal program in biliary atresia or the pivotal program in Alagille syndrome, and the outcomes of such trials; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the United States or European Union; delays or other challenges in the recruitment of patients for, or the conduct of, company’s clinical trials; and Albireo’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.
Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578