Albireo’s Lead Product Candidate for Orphan Pediatric Liver Disease Accepted Into European Medicines Agency’s PRIME Program

BOSTON, Nov. 15, 2016 -- Albireo Pharma, Inc. (NASDAQ:ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, announced today that its lead product candidate, A4250, has been granted access to the PRIority MEdicines (PRIME) program of the European Medicines Agency (EMA) for the treatment of progressive familial intrahepatic cholestasis (PFIC). 

“PFIC is a highly debilitating genetic liver disorder that is life altering for affected children and their families,” said Ron Cooper, President and Chief Executive Officer of Albireo.  “There is an urgent need for an effective pharmacological treatment for PFIC, as there are currently no approved drug options in the United States or Europe.  A4250’s acceptance into the PRIME program serves as validation of its promise to meet that need.  We look forward to collaborating with the EMA on the development plan for A4250, as well as the potential accelerated assessment of A4250 in Europe.”

The PRIME program was launched by the EMA to provide enhanced support to developers of investigational medicines that target an unmet medical need, with a focus on those that may offer a major therapeutic advantage over existing treatments or address a disease with no current treatment option.  The program is designed to provide early engagement with the EMA to optimize development plans and speed up evaluation, with the goal of helping patients benefit as early as possible from therapies that may significantly improve their quality of life.  As of August 2016, only 13 out of 57 requests for PRIME eligibility had been accepted into the program.  Having been accepted into the program, A4250 may be eligible for accelerated assessment of a potential future marketing authorization application (MAA) in Europe.

A4250 is currently being evaluated in children with chronic cholestasis in a Phase 2 clinical trial that is intended to support a potentially pivotal clinical trial in PFIC planned to be conducted in the United States and Europe.  Albireo anticipates meeting with the U.S. Food and Drug Administration (FDA) regarding the planned PFIC trial in the first quarter of 2017.  

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.  A4250 is currently being evaluated in children with chronic cholestasis in a Phase 2 clinical trial that is intended to support a potentially pivotal clinical trial in PFIC.

About Progressive Familial Intrahepatic Cholestasis
Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive, life-threatening liver disease, which may start early after birth or at a young age and rapidly progress to end-stage liver disease.  Three alternative gene defects have been identified that correlate to three separate PFIC subtypes, known as types 1, 2 and 3.  The precise prevalence of PFIC is unknown, but PFIC has been estimated to affect between one in every 50,000 to 100,000 children born worldwide.

PFIC is commonly associated with elevated serum bile acids.  Prominent symptoms of PFIC include pruritus, which is associated with severe sleep disturbance and diminished overall quality of life, and poor growth. First-line treatment in PFIC is typically off-label ursodeoxycholic acid (UDCA).  Notwithstanding treatment with UDCA, many PFIC patients will require partial external biliary diversion (PEBD) surgery and ultimately liver transplantation.  Although success rates vary, published third-party studies have shown that PEBD surgery can slow, and in some cases stop, the progression of liver disease and lead to reduced pruritus and improved sleep.

About Albireo 
Albireo Pharma 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 is located in Gothenburg, Sweden. For more information on Albireo, please visit www.albireopharma.com.

Forward-Looking Statements — Albireo
This press release includes “forward-looking statements.”  Forward-looking statements include statements, other than statements of historical fact, regarding Albireo’s intentions, plans, beliefs, expectations or forecasts for the future, including regarding a meeting with the FDA or the timing for such meeting, the potential for accelerated assessment by the EMA of a potential future MAA for A4250 or a potentially pivotal clinical trial of A4250 in PFIC.  Albireo 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: those described in the documents Albireo (f/k/a Biodel Inc.) has filed with the Securities and Exchange Commission, and whether the ongoing clinical trial of A4250 in children with chronic cholestasis will be sufficient to support advancement into a pivotal clinical trial in PFIC.  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.

Investor Contact:
Hans Vitzthum				
Managing Director			
LifeSci Advisors, LLC.		
212-915-2568				

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6 Degrees
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