Albireo Announces Presentations at NASPGHAN 2020 Annual Meeting
Albireo Pharma (NASDAQ: ALBO) is showcasing new data on its investigational drug, odevixibat, at the NASPGHAN 2020 Annual Meeting. Odevixibat, developed for treating rare pediatric liver diseases like PFIC, biliary atresia, and Alagille syndrome, is a non-systemic ileal bile acid transport inhibitor. The company plans to complete regulatory filings for odevixibat in PFIC by early 2021, aiming for approval and market launch in the second half of 2021. The largest Phase 3 trial for biliary atresia, the BOLD trial, is also ongoing, underscoring Albireo’s commitment to rare liver diseases.
- Odevixibat is a highly potent, once-daily treatment for rare pediatric liver diseases.
- Albireo plans to file for regulatory approval of odevixibat in PFIC by early 2021.
- The BOLD trial is the largest Phase 3 trial conducted for biliary atresia.
- Odevixibat's approval is contingent on successful outcomes from ongoing clinical trials.
- Potential delays in regulatory approval could impact market launch timelines.
-Research continues to show potential of odevixibat as first drug therapy for PFIC and in other rare liver diseases-
-Scientific leadership demonstrates company’s commitment to increase understanding of rare pediatric cholestatic liver diseases and reduce burden of illness-
BOSTON, Oct. 23, 2020 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced upcoming poster presentations at the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) 2020 Annual Meeting, being held virtually November 1-7. The company’s presentations will include studies on lead product candidate odevixibat, a highly potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) currently being developed for the treatment of progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome.
“Our leading approach to rare cholestatic liver disease research has been underscored this year, therefore, we are very pleased to present new data on odevixibat at NASPGHAN,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Poster presentations will also include a trial design overview of our global, gold standard approach with the BOLD clinical trial evaluating odevixibat, which is the largest Phase 3 trial in biliary atresia, as well as health economics and outcomes research focused on the burden of disease in PFIC.”
The Albireo poster presentations will be included in Poster Session III on Friday, November 6 from 11 a.m. to 1 p.m. EST:
- Abstract #649: The BOLD Study: A Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Infants with Biliary Atresia After Kasai Portoenterostomy
- Abstract #638: Drug-drug Interaction Study to Evaluate the Interaction of A4250 (Odevixibat) with Itraconazole, a P-gp Inhibitor in Healthy Adult Subjects
- Abstract #157: The PICTURE Burden of Illness Study: Quantifying the Socio-economic Burden of Progressive Familial Intrahepatic Cholestasis (PFIC) in the U.S., UK, France and Germany
The abstracts are available as a supplement to the November issue of the Journal of Pediatric Gastroenterology and Nutrition and can be viewed at https://journals.lww.com/jpgn/Documents/NASPGHAN%202020.pdf.
About Odevixibat
Odevixibat is an investigational product candidate being developed to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome. A highly potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), odevixibat acts locally in the small intestine. Odevixibat does not require refrigeration and can be taken as a capsule for older children, or opened and sprinkled onto food, which are factors of key importance for adherence in a pediatric patient population. Albireo conducted the largest ever global Phase 3 trial in PFIC1 and PFIC2. The PEDFIC 1 trial evaluated odevixibat 40 µg/kg/day or 120 µg/kg/day or placebo in 62 patients, ages 6 months to 15.9 years. Positive results from the trial were announced on September 8, 2020: News Release – Albireo Phase 3 Trial Meets Both Primary Endpoints for Odevixibat in PFIC. Odevixibat is currently being evaluated in the ongoing PEDFIC 2 open-label trial (NCT03659916) and the BOLD Phase 3 trial in patients with biliary atresia (NCT04336722). A pivotal Phase 3 trial of odevixibat for Alagille syndrome is also anticipated by the end of 2020.
Odevixibat has received fast track, rare pediatric disease and orphan drug designations in the United States. In addition, the FDA has granted orphan drug designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. The EMA has granted odevixibat orphan designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. Its Pediatric Committee has agreed to Albireo’s odevixibat Pediatric Investigation Plan for PFIC. EMA has also granted orphan designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. Odevixibat has the potential to become the first approved drug treatment for patients with PFIC. The Company intends to complete regulatory filings in the EU and U.S. for odevixibat in PFIC no later than early 2021, in anticipation of potential regulatory approval, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021.
About Albireo
Albireo Pharma is a clinical-stage biopharmaceutical company focused on the development of novel bile acid modulators to treat orphan pediatric 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 and is in Phase 3 development in progressive familial intrahepatic cholestasis (PFIC) and biliary atresia, with a third Phase 3 trial being planned in Alagille syndrome. The Company expects to complete IND-enabling studies for a new preclinical candidate this year. 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, 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 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 planned pivotal trial for odevixibat in Alagille syndrome; 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 planned 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, elobixibat, 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 planned 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.
Media Contacts:
Colleen Alabiso, 857-356-3905, colleen.alabiso@albireopharma.com
Heather Anderson, 6 Degrees, 919-827-5539, handerson@6degreespr.com
Investor Contact:
Hans Vitzthum, LifeSci Advisors, LLC., 857-272-6177
FAQ
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