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
"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
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
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.
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
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.
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
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