Final Results of Albireo’s Phase 2 Study of A4250 in Children with Cholestatic Liver Disease Presented at The Liver Meeting® 2017
— A4250 reduced serum bile acids and improved pruritus in most patients,
exhibited a favorable overall tolerability profile —
— Albireo to conduct Phase 3 study in children with progressive familial intrahepatic cholestasis —
BOSTON — October 20, 2017 — Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced that final results from its Phase 2 study of lead product candidate A4250 in children with cholestatic liver disease will be presented at the American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting® 2017 in Washington, D.C. A poster presentation of the study will be made by Ekkehard Sturm, M.D., Ph.D., an investigator for the study, on October 21, followed by an ePoster presentation chosen by the leaders of the AASLD Special Interest Groups on October 22.
In the study, A4250 reduced serum bile acids (sBA) and improved pruritus in most patients, particularly patients with progressive familial intrahepatic cholestasis (PFIC). A4250 exhibited a favorable overall tolerability profile in the study, with all patients completing the four-week treatment period and no reports of diarrhea associated with multiple dose therapy.
“The findings from this Phase 2 study of A4250 on various efficacy measures, and the safety and tolerability profile shown, are promising,” said Dr. Sturm, head of pediatric gastroenterology-hepatology, liver and intestinal transplantation at Children’s Hospital, University of Tuebingen in Germany. “The compelling data in PFIC patients in particular illustrate that A4250 has potential to become a welcomed treatment alternative to the current standards of care, which are bile diversion surgery or transplantation. Further study of A4250 in PFIC is warranted.”
A4250 is a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), which has minimal systemic exposure and acts locally in the gut. The open label, multicenter, dose-finding Phase 2 clinical trial assessed the safety and tolerability of A4250 and explored changes in sBA levels, pruritus and sleep disturbance. Twenty 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 study, with four of the patients reenrolling into a second dose group. A4250 was administered orally once daily for four weeks. Five different doses of A4250 were evaluated, ranging from 10 µg/kg to 200 µg/kg.
A4250 demonstrated a mean reduction in sBA levels in all five dose groups in the study, with substantial sBA reductions (ranging from 43 to 98 percent) in 80 percent of the PFIC patients. In addition, the majority of patients showed improvement in pruritus on three different assessment scales, with a significant correlation between reduction in sBA and improvement in pruritus, as well as improvement in sleep disturbance. The open label study was not powered for formal statistical analyses.
There were no serious adverse events reported in the study that were considered to be drug related. Most adverse events, including some increased transaminases, were mild, transient and assessed as either unrelated to study drug or the relationship was unclear.
“We are encouraged by the findings from the Phase 2 study in pediatric cholestatic liver disease and look forward to getting underway our planned Phase 3 clinical trial of A4250 in patients with PFIC, a devastating rare liver disease for which there is currently no approved drug treatment,” said Paresh Soni, M.D., Ph.D., Albireo’s Chief Medical Officer.
In addition to the final results from Albireo’s Phase 2 study in children with cholestatic liver disease, data from an investigator-initiated Phase 2 study of A4250 in adults with primary biliary cholangitis (PBC) is scheduled to be presented by the investigator at The Liver Meeting®. Albireo has no plans to develop A4250 to treat PBC.
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 has been granted orphan drug designation for PFIC in the United States and the European Union. The European Medicines Agency (EMA) has also granted A4250 access to the PRIority MEdicines (PRIME) program for the treatment of PFIC, and its Paediatric Committee has agreed to Albireo’s A4250 Pediatric Investigation Plan.
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.
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; the timing for initiation or completion of or reporting of results from any clinical trial; 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 that results in a delay in its 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.
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Source: Albireo Pharma, Inc.