EMA's Paediatric Committee Agrees to Albireo's A4250 Pediatric Investigation Plan

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EMA's Paediatric Committee Agrees to Albireo's A4250 Pediatric Investigation Plan

Agreement an essential step for a potential EU marketing authorization application 
Completion of PIP would provide an additional two years of market exclusivity 

Planned Phase 3 clinical trial a key component of agreed PIP

BOSTON, Sept. 26, 2017 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq:ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced that the Paediatric Committee (PDCO) of the European Medicines Agency (EMA) has agreed to Albireo's pediatric investigation plan (PIP) for lead product candidate A4250 in patients with progressive familial intrahepatic cholestasis (PFIC). PFIC is a rare and life-threatening genetic liver disease for which there are currently no approved drug therapies.  

"Because we are developing A4250 to treat a pediatric patient population, we are especially pleased that the PDCO, which is responsible for activities on potential medicines for pediatric populations and supporting their development in the European Union, has agreed to our pediatric investigation plan for A4250 in PFIC," said Ron Cooper, President and Chief Executive Officer of Albireo. "We believe A4250 has the potential with Phase 3 success to make a significant difference in the lives of children suffering from this debilitating rare liver disease and their families. We plan to initiate our previously announced Phase 3 trial of A4250 by year end."

As part of the regulatory process for the registration of new medicines with the EMA, companies are required to agree with PDCO on a PIP that outlines a development program for the investigational product in the pediatric population. The agreed PIP for A4250 includes Albireo's previously announced double-blind, placebo-controlled, Phase 3 clinical trial in patients with PFIC (type 1 or 2), ages six months to 18 years, as well as Albireo's completed Phase 2 study of A4250 in children with cholestatic liver disease. In addition, the PIP includes a small clinical trial in neonates with PFIC, which will be deferred until after completion of the planned Phase 3 trial, and the development of a liquid formulation.

A4250 has received orphan designation in the European Union (EU) (as well as in the United States) for the treatment of PFIC, which, if maintained at approval, would be expected to provide 10 years of post-approval EU market exclusivity. Completion of the agreed PIP would provide an additional two years of market exclusivity in the EU, making a total of 12 years.

In a recently completed Phase 2 clinical trial in children with cholestatic liver disease, A4250 exhibited a favorable overall tolerability profile, and most patients showed both a reduction in serum bile acid levels and an improvement in pruritus across multiple scales after four weeks of treatment with A4250.

About A4250

A4250 is a first-in-class product candidate in development for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC) and potentially other orphan pediatric cholestatic liver diseases. A4250 is a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), has minimal systemic exposure and acts locally in the gut.

A4250 has been granted orphan drug designation for PFIC in the United States and the European Union and has been granted support through the PRIority MEdicines (PRIME) program of the European Medicines Agency (EMA) for the treatment of PFIC.

About Albireo 

Albireo is a clinical-stage biopharmaceutical company focused through its operating subsidiary on the development of novel bile acid modulators to treat orphan pediatric liver diseases and other liver and gastrointestinal diseases and disorders. Albireo's clinical pipeline includes a Phase 3 product candidate, a Phase 2 product candidate and a product candidate for which an application for regulatory approval has been submitted in Japan. Albireo was spun out from AstraZeneca in 2008.

Albireo Pharma is located in Boston, Massachusetts, and its key operating subsidiary, Albireo AB, is located in Gothenburg, Sweden. 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: the plans for, or progress or scope of, development of A4250, including regarding the planned Phase 3 clinical program for A4250 in patients with PFIC or the other activities comprising the pediatric investigation plan for A4250; the timing for initiation or completion of or reporting of results from any clinical trial, including the timing for initiation of the planned Phase 3 PFIC clinical program or pediatric investigation plan for A4250; the competitive position of A4250 or the commercial opportunity in PFIC; 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 and uncertainties, including, but not limited to, risks and uncertainties relating to: whether favorable findings from clinical trials of A4250 to date, including findings in indications other than PFIC, will be predictive of results from future clinical trials of A4250, including the trials comprising the planned Phase 3 PFIC program; whether either or both of the FDA and EMA will determine that the primary endpoint and duration of the planned double blind Phase 3 trial in patients with PFIC is sufficient, even if such primary endpoint is met with statistical significance, to support approval of A4250 in the United States or the European Union, to treat PFIC, a symptom of PFIC or otherwise; the outcome and interpretation by regulatory authorities of the ongoing third-party study pooling and analyzing long-term PFIC patient data; whether Albireo's cash resources will be sufficient to advance A4250 through completion of the planned Phase 3 PFIC program; the timing for initiation or completion of, or for availability of data from, ongoing or future trials of A4250, including the trials comprising the planned Phase 3 PFIC program, and the outcomes of such trials; delays or other challenges in the initiation of, or recruitment of patients for, the planned double blind Phase 3 trial; and whether Albireo receives additional feedback from regulatory authorities on the planned Phase 3 PFIC program for A4250 prior to initiation. 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, in Albireo's Current Report on Form 8-K filed May 23, 2017 and in other 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.

Source: Albireo Pharma, Inc.

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Source: Albireo, Inc.

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