Albireo Pharma presents positive Phase 2 data for pediatric cholestasis candidate


Albireo Pharma (NASDAQ:ALBO) presented positive Phase 2 data for its drug candidate, A4250, at The International Liver Congress 2019.

A4250 is being developed to treat rare pediatric cholestatic liver diseases, where bile acid flow is disrupted. An inhibitor of the ileal bile acid transporter, A4250 is designed to reduce bile acid reuptake in the small intestine.

The Phase 2 clinical trial assessed the safety and tolerability of orally-administered A4250 in 20 children with cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome and biliary atresia. At the ILC 2019, Albireo presented data where A4250 improved pruritis in the majority Alagille syndrome patients, with serum bile acid reduction of up to 92%. Reductions in serum bile acids and pruritis were also observed in biliary atresia patients.

“The Alagille patient data from this Phase 2 trial, including the meaningful reductions in serum bile acids and improved pruritus and sleep scores, indicate that A4250 could be a promising therapy for cholestatic liver diseases like Alagille, and suggest that further investigation of A4250 in children with Alagille is warranted,” the study’s lead investigator, Dr. Ulrich Baumann, said in a statement.

Albireo plans to initiate a A4250 pivotal program in biliary atresia in the second half of 2019. A4250 is also in Phase 3 development for PFIC.

Abby Hardy