Study results to be presented at NASPGHAN demonstrate reductions in bile acids and bilirubin are highly associated with decreased aggregate need for liver transplant in PFIC patients
Findings to be presented by NAPPED Consortium at AASLD The Liver Meeting demonstrate native liver survival rates and impact of serum bile acid reduction across PFIC types
BOSTON, Oct. 15, 2018 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, announced today that important findings from multiple studies on progressive familial intrahepatic cholestasis (PFIC) and other cholestatic liver diseases will be presented at major upcoming North American liver meetings.
Findings from two studies on cholestatic liver diseases will be presented during poster sessions at the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) 2018 Annual Meeting, being held Oct. 24-27, 2018, in Hollywood, Fla. In addition, data from the NAPPED (NAtural course and Prognosis of PFIC and Effect of biliary Diversion) Consortium study evaluating the natural history of PFIC will be presented by an international research team at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting, being held Nov. 9-13, 2018, in San Francisco.
“These studies deepen our understanding of cholestatic liver diseases and have important applications for the development of our lead product candidate A4250 for pediatric cholestatic liver diseases,” said Ron Cooper, President and Chief Executive Officer of Albireo. “We are assessing pruritus in our PFIC program using a patient- and caregiver-reported instrument developed based on the qualitative study being presented at NASPGHAN. The partial external biliary diversion (PEBD) surgery data being presented at NASPGHAN show that decreases in serum bile acids and bilirubin were highly associated with decreased pruritus and decreased need for liver transplant. The results of these studies, alongside the NAPPED results, illustrate the unmet need in PFIC and support the approach we are pursuing with A4250.”
At NASPGHAN, the following posters will be presented on October 26: “Symptoms and daily impacts associated with progressive familial intrahepatic cholestasis and other pediatric cholestatic liver diseases: a qualitative study with patients and caregivers,” abstract number 352, and “Systematic literature review of the effect of partial external biliary diversion surgery on clinical and biochemical outcomes in progressive familial intrahepatic cholestasis patients,” abstract number 353.
At AASLD, the following presentations on November 11 are particularly relevant for the development of A4250: “The Natural Course of F1C1 Deficiency: Results from the NAPPED Consortium,” abstract number 1847, and “The Natural Course of BSEP Deficiency: Results from the Global NAPPED Consortium,” abstract number 0185. NAPPED is supported by an unrestricted grant from Albireo. Additional data on the relevance of bile acids in cholestatic liver disease will be presented elsewhere at AASLD.
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 lead product candidate, A4250, is being developed to treat rare pediatric cholestatic liver diseases and is in Phase 3 development in its initial target indication, progressive familial intrahepatic cholestasis. Albireo’s clinical pipeline also includes two Phase 2 product candidates. Albireo’s elobixibat, approved in Japan for the treatment of chronic constipation, is the first ileal bile acid transporter (IBAT) inhibitor approved anywhere in the world. 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
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 progress, scope, duration or results or timing for availability of results of, development of A4250 including regarding the Phase 3 clinical program for A4250 in patients with PFIC; the target indication(s) for development, design, population, conduct, objectiveor endpoints of any clinical trial, or the timing for completion of or reporting of results from the double-blind Phase 3 PFIC trial for A4250; 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, uncertainties and other factors, including, but not limited to: whether favorable findings from clinical trials of A4250 to date, including findings in indications other than PFIC, will be predictive of results from the trials comprising the Phase 3 PFIC program or any other clinical trials of A4250; 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, even if the 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, 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 completion of, or for availability of data from, clinical trials of A4250, including the trials comprising the Phase 3 PFIC program, and the outcomes of such trials; delays or other challenges in the recruitment of patients for, or the conduct of, the double-blind Phase 3 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 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.
Investor Contact:
Hans Vitzthum
LifeSci Advisors, LLC
212-915-2568
Media Contact:
Heather Anderson
6 Degrees
980-938-0260
handerson@6degreespr.com
Source: Albireo Pharma, Inc.