– PEDFIC 1 and PEDFIC 2 Phase 3 data presentations on long-term safety, tolerability, and improved outcomes for patients with PFIC –
– Data showing long-term treatment benefits of Bylvay™ (odevixibat), including improvements in total bilirubin, growth, and sleep –
BOSTON, May 19, 2021 (GLOBE NEWSWIRE) — Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare pediatric liver disease company developing novel bile acid modulators, today announced the presentation of new data from its Phase 3 PEDFIC 1 study and PEDFIC 2 long-term extension study of its lead product candidate, Bylvay, at the 6th World Congress of Pediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) Meeting, being held virtually June 2 – 5. Bylvay is 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.
“We have an abundance of data from our PEDFIC studies with Bylvay and are pleased to be able to present key findings for PFIC at WCPGHAN.,” said Patrick Horn, M.D., Ph.D., Chief Medical Officer at Albireo. “We look forward to presenting the full results in three oral presentations, which support the potential of Bylvay to provide benefits to patients with PFIC and improve the standard of care if approved as the first non-surgical treatment option.”
The full list of Albireo presentations at WCPGHAN includes:
Oral Presentation:
Long-Term Safety and Tolerability of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis: Results From the Phase 3 PEDFIC 1 and PEDFIC 2 Studies
Presenter: Tassos Grammatikopoulos, M.D., FRCPCH Consultant in Paediatric Hepatology & Honorary Clinical Senior Lecturer
Session: Highest Scoring Abstracts
Date & Time: Thursday, June 3, 3:15 p.m. CEST
Oral Presentation:
Long-term Treatment Benefits of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis: Improvements in Total Bilirubin, Growth, and Sleep as Secondary and Exploratory Outcome Measures from the Phase 3 PEDFIC 1 and PEDFIC 2 Studies
Presenter: Richard J. Thompson, M.D., Ph.D., Professor of Molecular Hepatology at King’s College London and principal investigator of the study
Session: Hepatology Session 3
Date & Time: Friday, June 4, 11:30 a.m. CEST
Oral Presentation:
Validation of the PRUCISION
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Caregiver-Reported Pruritus Measure Using Data From the Phase 3, Randomised PEDFIC 1 Trial in Paediatric Patients With Progressive Familial Intrahepatic Cholestasis
Presenter: Chad Gwaltney, Ph.D., President and Principal Consultant at Gwaltney Consulting
Session: Hepatology Session 3
Date & Time: Friday, June 4, 11:30 a.m. CEST
ePoster:
Drug-Drug Interaction Study to Evaluate the Interaction of A4250 (Odevixibat) With Midazolam, a Sensitive CYP3A4 Substrate, and Itraconazole, a P-gp Inhibitor, in Healthy Adult Subjects
Presenter: Philip Stein, M.D. Medical Director, Albireo
Session: ePoster Session
Date & Time: Saturday, June 5, 10:40 a.m. CEST
The abstracts will also be published as an abstract book in the Journal of Pediatric Gastroenterology and Nutrition (JPGN).
About Bylvay (odevixibat)
Bylvay is an investigational product candidate being developed to treat rare pediatric cholestatic liver diseases, including PFIC, biliary atresia and ALGS. A potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), Bylvay acts locally in the small intestine. Bylvay 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. The FDA has granted Priority Review and set a PDUFA goal date of July 20, 2021. In Europe, the EMA validated MAA. Bylvay is the only IBATi granted accelerated assessment by the EMA.
Bylvay also been granted Orphan Designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. The EMA’s Pediatric Committee has agreed to Albireo’s Bylvay Pediatric Investigation Plans for PFIC and biliary atresia. In addition to PFIC, Bylvay has Orphan Drug Designations for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. With FDA and EMA regulatory submissions complete, Bylvay has the potential to become the first approved drug treatment for patients with PFIC in the U.S and Europe. The Company anticipates potential regulatory approvals, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021.
The MAA and NDA filings are supported by results from PEDFIC 1 and PEDFIC 2 Phase 3 studies. 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 in a randomized, double-blind, placebo-controlled trial. In the PEDFIC 1 study, Bylvay met both primary endpoints and was well tolerated with very low incidence of diarrhea/frequent bowel movements (9.5% of Bylvay treated patients vs. 5.0% of placebo patients). ir.albireopharma.com/news-releases/news-release-details/albireo-phase-3-trial-meets-both-primary-endpoints-odevixibat. PEDFIC 2 is a long-term, open-label Phase 3 extension study. The Company also provides an Expanded Access Program (EAP) for eligible patients with PFIC in the U.S., Europe, Canada and Australia. Bylvay is also currently being evaluated in the BOLD Phase 3 trial in patients with biliary atresia, and the global Phase 3 ASSERT trial for ALGS.
About Albireo
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. Albireo’s lead product candidate, Bylvay, is being developed to treat rare pediatric cholestatic liver diseases with Phase 3 trials in PFIC, Alagille syndrome and biliary atresia. For PFIC, the FDA recently granted Priority Review and set a PDUFA goal date of July 20, 2021. In Europe, the EMA validated MAA. Bylvay is the only IBATi granted accelerated assessment by the EMA. Bylvay has been provisionally accepted by both the FDA and EMA as the brand name for odevixibat. 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 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 Bylvay or any other Albireo product candidate or program; including expectations regarding the impact of the COVID-19 pandemic on our business and our ability to adapt our plans and activities as appropriate; the pivotal trial for Bylvay in biliary atresia (BOLD), and the pivotal trial for Bylvay 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 Bylvay in PFIC, BOLD, ASSERT ; the Phase 1 clinical trial for A3907, the potential approval and commercialization of Bylvay; the potential for Bylvay to become the first approved drug for PFIC patients; 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; 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,” 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 NDA for Bylvay for the treatment of pruritus in patients with PFIC will be approved by the FDA and whether the MAA for Bylvay in PFIC will be approved by the EMA; whether the FDA or EMA will complete their respective reviews within the target timelines, including the FDA’s PDUFA goal date, as a potential result of the impact of the COVID-19 pandemic or otherwise; the risk that the NDA will not be approved despite the FDA’s acceptance of the NDA for review; whether the FDA will require additional information, whether we will be able to provide in a timely manner any additional information that the FDA requests, and whether such additional information will be satisfactory to the FDA; other 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 indications other than PFIC, will be predictive of results from other clinical trials of Bylvay; 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 Bylvay 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 Bylvay or A3907, 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 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 Contact:
Colleen Alabiso, 857-356-3905, [email protected]
Lisa Rivero, 617-947-0899, [email protected]
Investor Contact:
Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578
