Benzinga | Nov 13, 2020 08:10AM EST

Albireo Pharma Highlights Presentation Of Data Showing 'Durable Response to Odevixibat in a Rare Pediatric Liver Disease' At AASLD

- PEDFIC 1 meets both U.S. and EU primary and secondary endpoints with highly statistically significant reductions in serum bile acids and pruritus -



- Interim results from long-term extension study show sustained improvements in serum bile acids, pruritus -

- Improvements in growth measures and liver parameters observed with long-term administration suggests disease modifying potential of odevixibat -

- Treatment effect consistent across wide range of PFIC patient types 1, 2 and 3 -

- Next-generation, novel bile acid modulators for adult liver diseases unveiled, including compound A3907 -

BOSTON, Nov. 13, 2020 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (NASDAQ:ALBO), a clinical-stage rare liver disease company developing novel bile acid modulators, today announced new data in progressive familial intrahepatic cholestasis (PFIC) confirming statistically significant reductions in serum bile acids (sBAs) and improvements in pruritus for odevixibat, a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi). Interim results from the extension study also showed continued treatment effect for sBAs, pruritus, growth and liver parameters across PFIC1, PFIC2 and PFIC3 patients. Data to be presented at the American Association for the Study of Liver Diseases (AASLD) Liver Meeting November 13-16.

Full results from PEDFIC 1, the first and largest, global, phase 3 study ever conducted in PFIC, confirm both U.S. and EU primary endpoints were met in the randomized, double-blind, placebo-controlled trial. Additionally, long-term data from PEDFIC 2, an open-label Phase 3 extension study, demonstrate continued and durable reductions in sBAs, improvements in pruritus assessments and encouraging markers of liver and growth function in patients treated up to 48 weeks. Across both studies, odevixibat was generally well tolerated, and treatment-emergent adverse events (TEAEs) were mostly mild or moderate. Collectively, these studies reaffirm odevixibat's potential to be the first drug treatment approved for patients living with PFIC, a devastating disease which is currently treated with surgical options including liver transplantation. The data support near-term regulatory filings in the U.S. and EU.

"The full results from the PEDFIC 1 Phase 3 trial confirm the magnitude of treatment effects seen in the topline data and reinforce the potential for odevixibat to alter the biology of PFIC disease. There were highly statistically significant reductions in both pruritus and serum bile acids in the PEDFIC 1 study," said Richard Thompson, M.D., Ph.D., Professor of Molecular Hepatology at King's College London and principal investigator of PEDFIC 1 and PEDFIC 2. "We saw durability and sustained effect in the interim results in PEDFIC 2, which are encouraging signs that IBAT inhibition with odevixibat may offer a transformative treatment and genuine alternative to surgery for patients with PFIC."

Final Results from PEDFIC 1, First and Largest Study Ever in PFIC1 and PFIC2

Full results from PEDFIC 1, the global Phase 3 clinical trial evaluating the efficacy and safety of odevixibat in children with PFIC, confirms both U.S. and EU primary endpoints were met in the placebo-controlled trial. Key findings include:

* Significant reductions in pruritus and SBAs: Overall, treatment with odevixibat at both doses of 40 and 120 ?g/kg/day led to statistically significant reductions in pruritus symptoms and serum bile acids over 24 weeks, compared with placebo. Statistically significant improvement was seen in the proportion of positive pruritus assessments (p=0.004), which is the U.S. regulatory primary endpoint. The EU regulatory primary endpoint was also achieved, which was a 70% reduction in serum bile acids (sBAs) or reaching a level of 70 ?mol/L (p=0.003).

* Rapid, sustained effect: Rapid onset of treatment effects, sustained through week 24.

* Well tolerated: Odevixibat was well tolerated, with an overall adverse event incidence not dose dependent and similar to placebo. There were no drug-related serious adverse events (SAEs) reported during the study. Diarrhea/frequent bowel movements were the most common treatment-related gastrointestinal adverse events, which occurred in 9.5% of odevixibat treated patients vs. 5.0% of placebo patients. Only one patient in the 120 ?g/kg/day group discontinued treatment due to an AE of diarrhea.