Albireo Receives Positive CHMP Opinion for Bylvay™ (odevixibat) for Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)

Albireo Receives Positive CHMP Opinion for Bylvay™ (odevixibat) for Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)

– Bylvay has the potential to become first approved drug to treat all forms of PFIC –

– CHMP opinion is based on the PEDFIC Phase 3 studies –

– PEDFIC 1 met the bile acid (P=0.003) and pruritus (P=0.004) primary endpoints, with a diarrhea rate of 10% vs 5% placebo –

– Integrated EU commercial, access and medical organization in place ready to launch following EU marketing authorization –

BOSTON, May 21, 2021 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending approval of Bylvay (odevixibat) for the treatment of PFIC. Bylvay is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) and could be the first non-surgical treatment for this rare disease. With this positive CHMP opinion, Bylvay continues on the accelerated approval path for European Commission (EC) decision on the marketing authorization application (MAA) and an anticipated launch in the second half of this year.

“Today’s positive CHMP opinion for Bylvay brings us one step closer to having the first-ever, non-surgical treatment that could reduce the burden of PFIC on families and children,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Our hope is to provide children both short-term relief from symptoms such as pruritus and long-term improvements in important parameters such as growth with Bylvay.”

PFIC is a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life. The most prominent and problematic ongoing manifestation of PFIC is pruritus, or intense itching, which often results in a severely diminished quality of life.

“The novel IBAT inhibition of odevixibat holds the promise to transform the treatment paradigm for children with PFIC types 1, 2 and 3,” remarked Richard Thompson, Professor of Molecular Hepatology at King’s College London and principal investigator of PEDFIC 1 and PEDFIC 2. “The current standard of liver transplants come with real challenges, such as life-long immunosuppression, and challenges finding organs for young children.”

Of the estimated 100,000 patients with cholestatic liver disease worldwide without an approved drug treatment, there are approximately 15,000 PFIC patients, excluding China and India. Albireo plans to directly commercialize Bylvay in the EU and is prepared for launch with commercial, access and medical affairs personnel on the ground. To support future access negotiations with countries in Europe, Albireo is building a compelling value package with the PEDFIC, gold standard, Phase 3 data, natural history information and a study to reflect the burden of PFIC on caregivers and families.

In addition to the pending EC decision for the MAA for Bylvay in PFIC, the Company also anticipates an upcoming regulatory decision by the U.S. FDA for the treatment of pruritus in patients with PFIC. The FDA has granted a Priority Review for the NDA and has set an action date of July 20, 2021 under the Prescription Drug User Fee Act (PDUFA). Albireo is also studying the use of Bylvay in other rare pediatric cholestatic diseases with the BOLD Phase 3 clinical trial in patients with biliary atresia and the ASSERT Phase 3 clinical trial in Alagille syndrome. Topline data from the ASSERT trial is expected in 2022, and data from the BOLD trial is expected in 2024.

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 17-20 May 2021:
https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-17-20-may-2021

EMA News:
https://www.ema.europa.eu/en/news/first-treatment-rare-liver-disease

About PFIC

Progressive familial intrahepatic cholestasis (PFIC) is a rare disorder that causes progressive, life-threatening liver disease. Patients have impaired bile flow, or cholestasis, caused by genetic mutations. The resulting bile build-up in liver cells causes liver disease and symptoms. The most prominent and problematic ongoing manifestation of the disease is pruritus, or intense itching, which often results in a severely diminished quality of life. Other symptoms include jaundice, poor weight gain and slowed growth. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all people with PFIC require treatment before age 30. There are no drugs currently approved for PFIC, only surgical options that include partial external biliary diversion (PEBD) and liver transplantation. For information on patient advocacy and disease education, check out resources from the Children’s Liver Disease Foundation (CLDF) at childliverdisease.org and PFIC Advocacy and Resource Network at PFIC.org.

About the PEDFIC Global Phase 3 Program

The positive opinion for Bylvay was supported by data from PEDFIC 1 and PEDFIC 2, the largest, global, Phase 3 trials ever conducted in PFIC. In PEDFIC 1, a randomized, double-blind study, placebo-controlled study, Bylvay met both its pruritus (p=0.004) and serum bile acid (p=0.003) primary endpoints and was well tolerated with very low incidence of diarrhea/frequent bowel movements (9.5% of treated patients vs. 5.0% of placebo patients). PEDFIC 2, a long-term, open-label Phase 3 extension study, reaffirmed Bylvay delivered sustained reductions in sBAs as well as improvements in pruritus assessments, growth and other markers of liver function in patients treated up to 48 weeks. Across both studies, Bylvay was well tolerated with diarrhea/frequent stools being the most common treatment-related gastrointestinal adverse events. There were no serious treatment related adverse events.

About Bylvay (odevixibat)
Bylvay is an investigational product candidate being developed to treat rare pediatric cholestatic liver diseases, including PFIC, biliary atresia and Alagille syndrome. 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. In addition to the PEDFIC studies, the Company also provides an Expanded Access Program (EAP) for eligible patients with PFIC in the U.S., Europe, Canada and Australia.

The FDA has granted Priority Review and set a PDUFA goal date of July 20, 2021. In Europe, Bylvay is the only IBATi granted accelerated assessment by the EMA. Bylvay has 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. 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 Alagille syndrome.

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. 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. 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; 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 and the Phase 1 clinical trial for A3907; the potential approval and commercialization of Bylvay and the timing for such potential approval and commercialization; 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 or that the MAA will not be approved despite CHMP’s opinion recommending approval of Bylvay for the treatment of PFIC; whether the FDA or EMA will require additional information, whether we will be able to provide in a timely manner any additional information that the FDA or EMA requests, and whether such additional information will be satisfactory to the FDA and EMA; 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, colleen.alabiso@albireopharma.com