- Results from an open-label, dose-finding Phase 2 clinical trial assessing Albireo Pharma's (ALBO) lead candidate A4250 in children with cholestatic liver disease showed a treatment effect and favorable tolerability profile. The data will be presented at the Liver Meeting in Washington, D.C.
- Treatment with A4250, a selective inhibitor of the ileal bile acid transporter (IBAT), resulted in reduced serum bile acids and less itching in most patients with an inherited liver disease called progressive familial intrahepatic cholestasis (PFIC).
- IBAT transports bile acids from the distal part of the small intestine back to the liver so they can be used again to promote digestion.
- On the safety front, all patients completed four weeks of treatment with no reports of drug-related diarrhea. Most adverse events were mild and transitory.
- The company plans to advance A4250 into Phase 3 development.
- A4250 has Orphan Drug status in the U.S. and EU for PFIC and has PRIME status in the EU.
- Previously: Albireo adds to A4250-stoked up move; shares ahead 41% (Nov. 21, 2016)
- Now read: Albireo Pharma (ALBO) Presents At Ladenburg Thalmann 2017 Healthcare Conference - Slideshow
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