Rare Disease Report

CEO of Albireo Pharma Talks PFIC Orphan Drug

JUNE 19, 2017
James Radke

A4250 – a potent and selective inhibitor of the ileal bile acid transporter (IBAT) – is being developed by Albireo Pharma for the treatment of the rare liver disease progressive familial intrahepatic cholestasis (PFIC).

As the company begins to prepare for a Phase 3 clinical trial, RDR spoke with the the company’s president and CEO, Ron Cooper.

Cooper boasts 25 years of pharmaceutical experience, and in just a few years, has built Albireo up from a small group of scientists into a company that is about to begin several Phase 2 and Phase 3 clinical trials for a variety of liver conditions.

Cooper noted that the company’s mission statement is simply to provide ‘hope for children with rare liver diseases’. Albireo started in 2008 as an offshoot of AstraZeneca and scientists from that company brought in assets to provide a unique means to treat rare liver conditions like PFIC.

PFIC is a rare genetic disorder affecting an estimated 3,200 children in the United States. The most prominent symptom of the PFIC is very deliberating pruritus, but most children also have a failure to thrive. At present, there is no approved treatment for the disorder, and the only options available for these children include cream to control the itching or to undergo 1 of 2 surgical options – either a liver transplant or partial external biliary diversion (PEBD) surgery. In the latter case, the excess bile acids are mechanically shunted from the liver to an external bag.

Cooper hopes the company’s lead candidate A4250 can act as a pharmaceutical means of the PEBD surgery, namely to have the bile shunted out of the liver via inhibition of IBAT.

And since the drug works on IBAT, its side effect profile is very limited. As Cooper noted, the drug works non-systemically and focuses on removal bile acids from the gut.

A4250 is administered orally, once a day in children as young as 1 year of age. To date, the data in an open label Phase 2 study show the drug to be well tolerated with reductions observed in serum bile acids and pruritus.

Moving forward, the company plans to begin a Phase 3 multicenter with sites in United States, Europe, and the Middle East later this year.

A4250 is a simple treatment and easily ingested, so the company is finalizing the trial protocol with the hope that it would allow patients to conduct the majority of the study while at home to limit travel time for these sick children and their families.

For more information about A4250 and PFIC, readers can click here. Additionally, be on the lookout for our video interview with Mr Cooper in the coming weeks. 

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2019 Rare Disease Communications. All Rights Reserved.