Albireo reports Phase II data for A4250 in cholestatic liver disease

Albireo Pharma Inc. (NASDAQ:ALBO) said A4250 was well tolerated and led to no treatment-related serious adverse events in a Phase II trial in 20 patients ages 1-17 with cholestatic liver disease, including progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome, biliary atresia and intrahepatic cholestasis. The open-label, international trial evaluated once-daily doses of 10-200 µg/kg oral A4250 for 4 weeks across 5 dose cohorts. The primary endpoint was safety.

On secondary endpoints, Albireo said A4250 led to a mean reduction in serum bile acid levels from baseline in all 5 dose cohorts, with "substantial" reductions, ranging from 43% to 98%, reported in 80% of PFIC patients. In patients receiving doses of ≥100 µg/kg, A4250 led to a mean absolute reduction in pruritus on a visual analog scale (VAS) itching score of 2.9 points and a mean absolute reduction in sleep disturbance of 2.9 points. The company said the reductions in VAS itching score were significantly correlated with reductions in serum bile acids (p<0.0008). Data were presented at the American Association for the Study of Liver Diseases meeting in Washington D.C...