Later this week, the American Association for the Study of Liver Diseases (AASLD) will hold its virtual The Liver Meeting Digital Experience (TLMdX). Altogether, TLMdX will last from November 13-16. Recently, clinical-stage biopharmaceutical company Albireo Pharma (“Albireo”) announced that data from its Phase 3 PEDFIC 1 trial and its PEDFIX 2 extension trial were accepted as late breakers for the meeting. Albireo will share data from these sessions and explain the impact of odevixibat on pediatric patients with progressive familial intrahepatic cholestasis (PFIC). For a full list of late-breaking abstracts, look here.
TLMdX Data and Presentations
Odevixibat was developed by Albireo, keeping with the company’s general mission of treating rare liver diseases. In addition to PFIC, odevixibat is being evaluated as a potential treatment for biliary atresia and Alagille syndrome. Odevixibat is an investigational non-systemic ideal bile acid transport inhibitor (IBATi). It is orally administered and taken 1x per day. In the Phase 3 PEDFIC 1 clinical trial, researchers analyzed the safety, efficacy, and tolerability of odevixibat. 62 patients enrolled in the trial. Patients ranged in age from 6 months to 15.9 years old. Ultimately, odevixibat reduced bile acids, itching, and diarrhea. Simultaneously, it improved growth and patient quality of life (QOL).
Currently, the treatment was granted Orphan Drug, Rare Pediatric Disease, and Fast Track designations. It has also been granted Orphan Medicinal Products designation in the EU. If the drug becomes FDA-approved, it would mark the first approved treatment option for PFIC.
TLMdX: What Will Be Shared
In the first presentation at TLMdX (#L04), Dr. Richard J. Thompson, MD, PhD, will discuss the results from the Phase 3 PEDFIC 1 trial. During the presentation, Dr. Thompson will explain the safety and efficacy of odevixibat for patients with PFIC type 1 and PFIC type 2. This presentation will be held on November 15 at 5:30pm ET.
Next comes #LP19 in the late-breaking abstract posters session. This poster also explores the safety and efficacy of odevixibat. However, data for this poster was sourced from the extension trial. Then, Poster #348 will discuss IBATis – how to discover them, how to characterize them, and how they might be used for treating various liver diseases.
Finally, Albireo has a Poster of Distinction (#509). Unlike the other presentations, #509 does not discuss previous clinical trials. Rather, this poster describes A3907, Albireo’s preclinical drug candidate for patients with nonalcoholic steatohepatitis.
Progressive Familial Intrahepatic Cholestasis (PFIC)
Generally, patients experience symptoms of progressive familial intrahepatic cholestasis (PFIC) before age 2. However, in rarer cases, PFIC diagnoses don’t come until adolescence. PFIC is a group of rare, inherited liver conditions caused by biliary epithelial transporter defects. Typically, ATP8B1, ABCB11, and ABCB4 gene mutations cause PFIC. These mutations prevent biliary epithelial transporters to remove bile from the liver. As a result, cholestasis occurs – meaning bile is obstructed. Symptoms include:
- Failure to thrive
- Pruritis (severe itching)
- Inability to gain weight
- Liver and spleen enlargement
- Poor vitamin solubility
- High blood pressure
- Vision and balance problems
- Suicidal ideation
- Liver cancer or cirrhosis
Learn more about PFIC.