Shares of Albireo Pharma Inc. (ALBO) are up nearly 18% over the last 4 trading days.
Albireo Pharma is a clinical-stage company focused on developing therapies for pediatric liver diseases and disorders. Spun out from AstraZeneca in 2008, Albireo went public on the NASDAQ Capital Market on November 4, 2016 under the symbol “ALBO”, following a reverse merger with Biodel Inc.
The Company’s lead product candidate is A4250, in development for progressive familial intrahepatic cholestasis.
Progressive familial intrahepatic cholestasis, or PFIC, is a class of chronic cholestasis disorders causing progressive liver disease, which typically leads to liver failure. In people with PFIC, liver cells are less able to secrete a digestive fluid called bile. The buildup of bile in liver cells causes liver disease in affected individuals. (Source: Genetics Home Reference).
Albireo is planning to advance A4250 to phase III program in patients with progressive familial intrahepatic cholestasis in the second half of 2017.
The planned phase III PFIC program includes a single randomized, double blind, placebo controlled, multicenter, six-month treatment clinical trial designed to enroll 60 patients and an open label long-term extension study. The primary endpoint for FDA evaluation, and a key secondary endpoint for EMA evaluation, will be an assessment of change in pruritus (severe itching). The primary endpoint for EMA evaluation, and a key secondary endpoint for FDA evaluation, will be serum bile acid (sBA) responder rate, according to the Company.
Elevated serum bile acids are linked to PFIC, and pruritus (severe itching) is a common and debilitating symptom of this disease.
A4250 is also being evaluated in a phase II trial in children with chronic cholestasis.
The most-advanced product candidate in the pipeline is Elobixibat for chronic constipation, which is under regulatory review in Japan.
Elobixibat is being developed in Japan by Albireo’s licensee EA Pharma Co. Ltd. This drug…