Zitat aus dem 10K Filing:
A4250 — our lead product candidate for PFIC and potentially other orphan pediatric liver diseases . A4250 is a novel, minimally absorbed, orally administered IBAT inhibitor. Our initial target indication for A4250 is PFIC. A4250 is currently being evaluated in an open label, dose finding Phase 2 clinical trial in children with chronic cholestasis that is intended to support a planned Phase 3 clinical trial in patients with PFIC. The Phase 2 trial includes children with chronic cholestasis caused by any of a number of different liver conditions, including PFIC, biliary atresia, Alagille syndrome, or ALGS, and sclerosing cholangitis. Because it is an open label study, preliminary data are available as of March 27, 2017 from all cohorts. These data show a reduction in serum bile acids in a substantial majority of patients and improvement in pruritus that was significantly correlated with the reduction in serum bile acids. In addition, A4250 was generally well tolerated in the trial. We expect final data from the trial to become available in the first half of 2017.
Vorläufige Daten aus allen Kohorten am 27.3.2017 Verfügbar Endgültige Daten im ersten Halbjahr 2017 verfügbar.
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