Albireo (ALBO)

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ALBO > 14M trading float > up 70%. FDA phase III news:

Albireo Announces Phase 3 Trial Of Odevixibat Met Both Primary Endpoints
6:37 am ET September 8, 2020 (Benzinga) Print
-Highly statistically significant reductions in both pruritus and serum bile acids-


-Well tolerated with very low incidence of diarrhea-

-Similar efficacy in children with PFIC1 or PFIC2-

-Pivotal trial results substantiate potential for odevixibat to be first drug for PFIC patients-

-Regulatory submissions for approval on track-

-Conference call to be held today at 8:30 a.m. EDT-

BOSTON, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (NASDAQ:ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced positive topline results from PEDFIC 1, a global Phase 3 clinical trial evaluating the efficacy and safety of odevixibat and the largest study ever conducted in PFIC1 and PFIC2. PEDFIC 1 met its two primary endpoints, demonstrating that odevixibat reduced serum bile acid responses (sBAs) (p=0.003) and improved pruritus assessments (p=0.004) with a single digit diarrhea rate. Odevixibat is a highly potent, non-systemic ileal bile acid transport inhibitor (IBATi), for the treatment of progressive familial intrahepatic cholestasis (PFIC) patients.

In the primary analysis, the study met the U.S. regulatory primary endpoint with the proportion of positive pruritus assessments being 53.5% in the odevixibat arms compared to 28.7% in the placebo arm (p=0.004). As a secondary endpoint, 42.9% of patients in the odevixibat arms had a clinically meaningful improvement in the pruritus score, defined as a drop from baseline of 1.0 point or more on the 0-4 point scale, at week 24 compared to 10.5% in the placebo arm (p=0.018). The study also met the EU regulatory primary endpoint with 33.3% of subjects in the odevixibat arms experiencing either a 70% reduction in sBAs or reaching a level of 70 µmol/L compared to no patients in the placebo arm (p=0.003). As a secondary endpoint, mean reduction of bile acids was 114.3 µmol/L in the odevixibat arms compared to an increase of 13.1 µmol/L in the placebo arm (p=0.002). Both doses of odevixibat were statistically significant for each of the endpoints. Odevixibat was well tolerated, with an overall adverse event incidence 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. Full results from the Phase 3 clinical trial will be presented at a future scientific meeting.

"The successful clinical application of IBAT inhibition is all about the ability to lower bile acids and reduce diarrhea rates. Odevixibat reduced bile acids in both PFIC1 and PFIC2 patients and demonstrated a clinically meaningful outcome in pruritus. This is exciting news for children suffering from PFIC who, if odevixibat is approved, may soon have an easy to take, once-daily drug for their life-threatening liver disease," said Ron Cooper, President and Chief Executive Officer of Albireo. "These strong results from PEDFIC 1 increase our confidence in the ongoing BOLD pivotal trial in biliary atresia and the Alagille syndrome study planned for later this year."

"The results of the PEDFIC 1 Phase 3 trial represent the potential for a paradigm shift in the treatment of PFIC," said Richard Thompson, MD, Professor of Molecular Hepatology at King's College London and principal investigator of the study. "These data demonstrate that odevixibat reduced serum bile acids and improved pruritus in patients with PFIC. Coupled with the favourable safety and tolerability profile odevixibat exhibited, these data underscore the potential to improve upon the current standard of care, which typically consists of off-label medications or invasive surgeries including transplant."

"PFIC is a devastating and progressive disease that impacts every facet of a person's life. These positive results represent an important milestone for the PFIC community, many of whose only option is a liver transplant or other invasive surgeries," saidEmily Ventura, President & Co-Founder of the PFIC Network."We appreciate Albireo's vital work in this area, and believe these positive data could bring us one step closer to a safe, effective drug therapy to make a real difference for patients with PFIC and their families."