Promising Pediatric Data for Albireo’s A4250 to

Data supports advancement of A4250 into a planned Phase 3 trial in patients with progressive familial intrahepatic cholestasis

Pruritus improvement and reduction in serum bile acid levels in most patients in Phase 2 clinical trial in children with cholestatic liver disease

BOSTON, April 22, 2017 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO ), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, announced that the embargo on its late-breaker abstract discussing preliminary data from a Phase 2 clinical trial of its lead product candidate A4250 in children with cholestatic liver disease has been lifted and the data will be presented today at The International Liver Congress™ 2017 in Amsterdam. The data demonstrated improvement in pruritus and reduction in serum bile acids (sBA) in most patients, particularly patients with progressive familial intrahepatic cholestasis (PFIC), after four weeks of treatment with A4250.

“This study represents the first time we have studied A4250 in children and we are very pleased with the data, especially the responses seen in patients with the rare, life-threatening liver disease PFIC,” said Ron Cooper, President and Chief Executive Officer of Albireo. “We are currently engaging with regulatory authorities in the United States and Europe with the objective to design a coordinated Phase 3 program in patients with PFIC, which we expect to initiate in the second half of 2017.”

A4250 is a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT) that has minimal systemic exposure. The open label, multicenter, dose finding Phase 2 trial assessed the safety and tolerability of A4250 and explored changes in sBA levels and pruritus. Nineteen patients aged one to 17 years old with a pediatric cholestatic liver disease, including PFIC (subtype 1, 2 or 3), Alagille syndrome, biliary atresia or intrahepatic cholestasis, were enrolled in the trial’s first five cohorts. Five different doses of A4250 were evaluated, ranging from 10 µg/kg to 200 µg/kg.

A4250 reduced mean levels of sBA in all five dose groups, with substantial sBA reductions observed in seven of nine PFIC patients (ranging from 43 percent to 98 percent). In addition, 14 of 19 patients showed improvement in pruritus using a visual analogue scale (VAS-Itch 0-10). The dose with the greatest improvement showed a mean decrease of 2.86 points from baseline. The trial was not powered for formal statistical analyses.

“The findings in this study illustrate the potential for A4250 to improve the treatment of children with PFIC or other rare cholestatic liver diseases,” said Ulrich Baumann, MD, an investigator in the A4250 Phase 2 clinical trial and professor at Hannover Medical School in Germany. “There is no drug proven to be effective in treating these devastating diseases, and surgery, with its associated limitations and drawbacks, is often the only viable alternative. A safe and effective new treatment option for these patients is greatly needed.”

The data showed a significant correlation between reduction in sBA and improvement of pruritus. In addition, no treatment-related serious adverse events were observed, and A4250 was well tolerated.

Subsequent to the submission date for ILC, the trial’s remaining patients completed the study.

About A4250  A4250 is a first-in-class product candidate in development for 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. Albireo anticipates initiating a Phase 3 clinical trial of A4250 in patients with PFIC in the second half of 2017.

A4250 has been granted orphan drug designation for PFIC in the United States and the European Union and has been granted access to 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 two Phase 2 product candidates and one Phase 3 product candidate. 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 progress or scope of development of A4250, including regarding a planned Phase 3 clinical trial of A4250 in patients with PFIC; the timing for initiation of such planned Phase 3 clinical trial of A4250; the competitive position of A4250 or the commercial opportunity in any target indication; 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 preliminary data from the Phase 2 trial of A4250 in children with chronic cholestasis will be confirmed following database lock; whether the FDA, EMA or other regulatory authorities will determine clinical results for A4250 to be sufficient to support advancement into a planned Phase 3 clinical trial in patients with PFIC in the United States and Europe; whether favorable findings from clinical trials of A4250 to date will be predictive of results from future clinical trials, including the planned Phase 3 PFIC trial; the designs, endpoints, sizes and durations for trials that will be required to support approval of A4250 to treat patients with PFIC or any other orphan pediatric liver disease; whether Albireo’s cash resources will be sufficient to advance A4250 through completion of a planned Phase 3 PFIC trial; the timing for initiation or completion of, or for availability of data from, ongoing or future trials of A4250, including the planned Phase 3 PFIC trial, and the outcomes of such trials; and delays or other challenges in the recruitment of patients for the planned Phase 3 PFIC trial. 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 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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