Benzinga | Jul 14, 2020 07:32AM EDT

Albireo Enrolls First Patient In Phase 3 Clinical Trial Of Odevixibat In Biliary Atresia



-- BOLD is first-ever pivotal trial for biliary atresia --

-- Trial expands odevixibat's development program into second rare cholestatic liver disease --

BOSTON, July 14, 2020 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (NASDAQ:ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced the first patient has been enrolled in the BOLD (Biliary atresia and the use of Odevixibat in treating Liver Disease) global Phase 3 clinical trial of odevixibat, a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), for the treatment of biliary atresia. There are no approved pharmacological treatments for biliary atresia which is the most common pediatric cholestatic liver disease and the leading cause of pediatric liver transplant across all diseases. BOLD is the largest, prospective intervention trial ever conducted in biliary atresia, expanding the development of odevixibat to a second rare cholestatic liver disease indication. Two clinical sites in the United States are currently active for patient enrollment in the second odevixibat Phase 3 pivotal trial.

Biliary atresia is a rare pediatric liver disease with symptoms typically developing about two to eight weeks after birth and no approved pharmacological therapies. Damaged or absent bile ducts outside the liver result in bile and bile acids being trapped inside the liver, quickly resulting in cirrhosis, and even liver failure. The disease impacts an estimated 15,000-20,000 people in the United States and European Union and is the leading cause of liver transplants among children. Odevixibat has received orphan drug designation for biliary atresia in the United States and European Union.

"The initiation of the BOLD trial is encouraging progress for biliary atresia patients, their families and clinicians, as it is the first-ever pivotal trial of a pharmacological therapy for this devastating rare disease," said Saul Karpen, M.D., Ph.D., Pediatric Hepatologist at Children's Healthcare of Atlanta and lead investigator of the BOLD trial. "There is an incredible unmet need for these patients with biliary atresia, since there are no effective therapies to stem the expected progression of disease. This lack of effective therapeutics leads to the current state of the field where approximately 70 percent of biliary atresia patients will need a liver transplant during childhood. I look forward to seeing how a targeted therapeutic, odevixibat, one that specifically addresses the accumulation of bile acids in the liver, may delay or prevent liver transplant for babies with this serious disease."

BOLD (NCT04336722) is a double-blind, randomized, placebo-controlled trial to evaluate the efficacy and safety of odevixibat in children who have biliary atresia and have undergone a Kasai procedure before age three months. Children in the treatment arm will receive odevixibat and escalate to 120 ?g/kg orally once daily for 24 months. The primary efficacy endpoint is improvement in the proportion of patients who are alive and have not undergone a liver transplant after two years of treatment compared to placebo, and secondary outcome measures include time to onset of any sentinel events, total bilirubin levels and serum bile acid levels. The trial will enroll approximately 200 patients at up to 75 sites globally.

"We're pleased to expand the evaluation of odevixibat into a second pivotal trial with the initiation of the precedent-setting BOLD trial for patients with biliary atresia, the most common rare pediatric liver disease," said Ron Cooper, President and Chief Executive Officer of Albireo. "We look forward to continuing our work to realize the potential of odevixibat as a much-needed treatment option for patients across multiple cholestatic liver diseases with BOLD and our PEDFIC 1 Phase 3 trial of odevixibat in progressive familial intrahepatic cholestasis, which we continue to expect topline data from in mid-2020."

In addition to the BOLD and PEDFIC 1 trials, Albireo is finalizing a third pivotal trial of odevixibat in Alagille syndrome and anticipates initiating the trial by the end of 2020.