ALBIREO PHARMA, INC. Two key oral presentations on the PEDFIC trials provided evidence of the disease modifying effects of Bylvay in patients with PFIC. The first underscored that a decrease in serum bile acids was strongly associated with native liver survival for up to three years in PFIC patients treated with Bylvay. A second late-breaking oral presentation showed that Bylvay restored bile acid secretion in PFIC patients with bile salt export pump deficiency. The late-breaking abstract was selected by AASLD for inclusion as a key presentation in 'The Best of The Liver Meeting' in the Pediatric Hepatology category.
'We are pleased to share long term data in PFIC, providing evidence that Bylvay could modify the course of disease, preserving patients' native liver and improving families' quality of life by alleviating disease burden,' said
'Patients with PFIC have long needed a disease modifying treatment,' said Dr.
Along with results observed with Bylvay treatment in PFIC patients, Albireo shared the first detailed results of the Phase 3 ASSERT trial in Alagille syndrome (ALGS) in a late-breaking oral presentation. The data showed that Bylvay provided early, rapid, clinically meaningful, and sustained improvements in pruritus, as well as significant reductions in bile acids and improvements in sleep quality in patients with ALGS. The ASSERT abstract was selected by AASLD for inclusion as a key presentation in 'The Best of The Liver Meeting' in the Pediatric Hepatology category.
Analyses of the ASSERT and PEDFIC trials and real-world data on Bylvay treatment, as well as data on the investigational therapy A3907, were highlighted in the following presentations at AASLD The Liver Meeting:
Early, Rapid, Sustained Effects in Alagille Syndrome
Oral, Late-Breaking Parallel Session Presentation (Abstract 5005; Publication 38786): Efficacy and Safety of Odevixibat in Patients with Alagille Syndrome: Top-Line Results from ASSERT, a Phase 3, Double-Blind, Randomized, Placebo-Controlled Study
Presenter: Dr. Nadia Ovchinsky,
Session: Late-Breaking Oral Abstract Session 1;
Top-line, unblinded data from the ASSERT study demonstrated that Bylvay treatment led to significant and clinically meaningful improvements in pruritus, as well as reductions in bile acid levels and improvements in sleep parameters in patients with ALGS. Over 90% of patients were pruritus responders and the treatment effects were early, rapid, and sustained. Bylvay was generally well tolerated; the overall incidence of treatment emergent adverse events (TEAEs) was similar to placebo. No patients discontinued the study and 96% of patients rolled over into the open-label extension study.
Disease Modification in PFIC
Oral Presentation (Abstract 865): Native Liver Survival in Odevixibat Serum Bile Acid Responders: Data from the PEDFIC Studies in Patients with Progressive Familial Intrahepatic Cholestasis
Presenter: Dr.
Session: Genes to Cures: What's New in Pediatric Liver Disease;
Pooled data analysis showed that PFIC patients who responded to Bylvay remained liver transplant free for up to three years. A decrease in serum bile acids (sBAs) at six months of treatment was strongly associated with native liver survival (p=0.0050) for sBA responders vs non-responders.
Oral, Late-Breaking Parallel Session Presentation (Abstract 5004; Publication 38801): Odevixibat Treatment in Responsive Patients with Bile Salt Export Pump Deficiency Restores Biliary Bile Acid Secretion, as Indicated by Serum Bile Acid Composition
Presenter: Dr. Mark Nomden,
Session: Late-Breaking Oral Abstract Session 1;
The analysis found that responders not only had a decrease in sBA concentration, but also alternations in sBA composition, more like that of a healthy individual. Data indicated that Bylvay restored biliary bile acid secretion in treatment-responsive patients with BSEP deficiency.
Poster (Abstract 37939): Long-term Efficacy and Safety of Odevixibat in Patients with Progressive Familial Intrahepatic Cholestasis: Results with 96 Weeks or More of Treatment
Presenter: Dr.
Session: Poster Session IV;
Pooled data analysis showed Bylvay treatment for 96 weeks or more was associated with durable clinical benefits in patients with PFIC, with improvements over time in mean sBA and aminotransferase levels and growth. Mean levels of alanine aminotransferase (ALT) and aspartate aminotransferase (AST) decreased over time, while mean total bilirubin levels were relatively unchanged. Mean height and weight Z scores also increased over time and Bylvay was generally well tolerated.
Poster (Abstract 37254): Serum Bile Acid Levels, Pruritus Scores, and Growth Over Time in Odevixibat Responders: Pooled Data from the PEDFIC Studies in Patients with Progressive Familial Intrahepatic Cholestasis
Presenter: Dr.
Session: Poster Session IV;
This analysis of pooled data examined treatment effects in 49 Bylvay responders who had a mean duration of exposure of 110 weeks and showed mean reductions in sBAs and pruritus were sustained over time, across PFIC types in general. Patients also had mean improvements in growth.
Poster (Abstract 37273): Effect of Odevixibat in Patients with Progressive Familial Intrahepatic Cholestasis Type 2 with at Least 1 Severe Mutation (BSEP3 Compound Heterozygotes): Pooled Data from the PEDFIC 1 and PEDFIC 2 Studies
Presenter: Dr. Henkjan J. Verkade,
Session: Poster Session IV;
Partial external biliary diversion (PEBD) is not effective in PFIC2 patients with BSEP 3 mutations, but this pooled data analysis showed that treatment with Bylvay provided substantial reductions in sBAs and/or pruritus severity in most patients with BSEP2/BSEP3 mutations, demonstrating that Bylvay may provide an alternative to surgical intervention for these patients.
Poster (Abstract 37608): Odevixibat Therapy in Patients with FIC1-Deficient Progressive Familial Intrahepatic Cholestasis and Diarrhea Following Liver Transplantation That Impacted Daily Activities: A Retrospective Case Series
Presenter:
Session: Poster Session IV;
Chologenic diarrhea can be a frequent and severe symptom after liver transplant in patients with FIC1 deficiency. Real-world data in three PFIC1 patients indicated that treatment with Bylvay can improve diarrhea and quality of life in PFIC1 patients with severe diarrhea after transplant.
Poster (Abstract 850): Odevixibat Treatment in Patients with Recurrent Episodic Cholestasis and Biallelic Mutations in ATP8B1: A Retrospective Case Series
Presenter: Dr.
Session: Poster Session IV;
Recurrent episodic cholestasis is a rare disease characterized by episodes of cholestasis followed by periods of remission. In a case series of six patients treated with Bylvay during a cholestasis episode, most patients experienced clinical improvement, including improvements in sBA levels, hepatic laboratory parameters, pruritus, sleep disturbances and impacts on daily life. Before starting Bylvay treatment, all 6 patients had high sBAs and severe pruritus leading to sleep or mood disturbances and/or the inability to attend school, play sports or work.
About the Phase 3 PEDFIC & ASSERT Studies
The PEDFIC trials represent the largest studies ever completed in children with PFIC, or progressive familial intrahepatic cholestasis, a rare genetic disorder that causes progressive, life-threatening liver disease. PEDFIC 1 was a randomized, double-blind, placebo-controlled Phase 3 trial that evaluated the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids (sBAs) in children with PFIC, and PEDFIC 2 is a long-term, open-label Phase 3 extension study. Patients with PFIC have impaired bile flow, or cholestasis, and the resulting bile build-up in liver cells causes liver disease and symptoms, such as intense itching, poor sleep, delayed growth, and diminished quality of life. The harmful impacts of the disease extend to parents and caregivers, as the 2022 multinational PICTURE study revealed that PFIC negatively affects caregivers' quality of life, relationships, and career prospects.
ASSERT is a gold standard, prospective intervention trial in Alagille syndrome, or ALGS, a rare, multisystem genetic disorder that can affect the liver, heart, skeleton, eyes, central nervous system, kidneys, and facial features. With 32 sites across
About Bylvay (odevixibat)
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