- Albireo Pharma (NASDAQ:ALBO) announces positive data from a Phase 2 clinical trial evaluating lead candidate odevixibat (A4250) in patients with three rare liver/bile duct disorders: biliary atresia, Alagille syndrome and progressive familial intrahepatic cholestasis. The results were presented at the European Society for Paediatric Gastroenterology, Hepatology and Nutrition Annual Meeting in Glasgow, Scotland.
- Pediatric cholestasis patients treated with odevixibat, an ileal bile acid transporter inhibitor, experienced reductions in serum bile acids as high as 92%. Two biliary atresia patients experienced reductions in serum bile acids of 58% and 51%.
- The two biliary atresia patients and most Alagille patients showed improvement in pruritis (itchy skin).
- On the safety front, odevixibat was generally well-tolerated. Two Alagille patients who had high baseline transaminase levels showed further further increases (biomarker for liver damage) so dosing was stopped at 200 ug.
- Topline data from a Phase 3 clinical trial, PEDFIC 1, should be available by year-end or early 2020.