Mirum has completed enrollment for its Phase IIb EMBARK study evaluating Livmarli (maralixibat) in infants with biliary atresia who have previously undergone a hepatoportoenterostomy (HPE).
The double-blind, placebo-controlled study (NCT04524390) is assessing mean changes in total bilirubin as a primary endpoint. Secondary endpoints include mean change in total serum bile acids, liver transplantation times, serum enzyme and platelet levels. A total of 72 participants have been enrolled, according to clinicaltrials.gov.
Livmarli is orally administered, once daily and is approved by the U.S. Food and Drug Administration for treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older. It is an ileal bile acid transporter (IBAT) inhibitor, which reduces absorption of bile acids and increases elimination in faeces.
Biliary atresia is a rare liver disorder where there is an accumulation of bile in the liver due to blockages or damage in bile ducts. It can result in progressive cholestasis and liver damage. It occurs in 1 of every 15,000 births and is the most common reason for liver transplants in children.
An open label period will commence with all patients receiving Livmarli following a 26-week placebo-controlled portion of the study. Topline results are expected to be announced by the California, US-based company in the second half of 2023.
“Further, these topline data will represent the first randomised data showing the effects of IBAT inhibition in patients with biliary atresia and an important step forward for a potential new treatment for patients in this difficult setting,” said Mirum chief development officer Lara Longpre.
Livmarli could also be cleared for progressive familial intrahepatic cholestasis (PFIC), with Mirum submitting the candidate for approval in the US and Europe.