Business
Mirum Pharmaceuticals Announces Enrollment Completion in Phase 2b EMBARK Biliary Atresia Study
Topline data are expected in second half of 2023. EMBARK will be the first completed randomized study of an IBAT inhibitor for the treatment of biliary
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n\nTopline data are expected in second half of 2023.\n\n\n\nEMBARK will be the first completed randomized study of an IBAT inhibitor for the treatment of biliary atresia.\n\n\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced it has completed enrollment of its Phase 2b EMBARK study for pediatric patients with biliary atresia, the most common indication for pediatric liver transplant. The EMBARK study is a global, double-blind, randomized, placebo-controlled, 26-week trial evaluating the efficacy and safety of LIVMARLI® (maralixibat) oral solution in infants with biliary atresia who have previously undergone a hepatoportoenterostomy (HPE), more commonly known as a Kasai procedure. Topline results are expected to be announced in the second half of 2023.\n\n\n“We are pleased to have fully enrolled our EMBARK study with LIVMARLI as there is an urgent need for new therapies in biliary atresia. We are looking forward to reporting topline data later this year,” said Lara Longpre, chief development officer at Mirum. “Further, these topline data will represent the first randomized 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.”\n\n\nAbout the EMBARK Study\n\n\nThe Phase 2 EMBARK study is evaluating LIVMARLI® (maralixibat) oral solution in patients with biliary atresia following a hepatoportoenterostomy (HPE), or Kasai procedure. EMBARK is a double-blind, placebo-controlled study assessing changes in total bilirubin and serum bile acids, both of which are leading indicators of efficacy on key markers of disease progression. Secondary endpoints also include bilirubin normalization, biomarkers of liver injury and safety. Following the 26-week placebo-controlled portion of the study, a long-term open label period will commence during which all patients will receive LIVMARLI up to Week 104.\n\n\nAbout Biliary Atresia\n\n\nBiliary atresia (BA) is a rare liver disorder in which there is a blockage or absence of large bile ducts that lead to bile accumulation in the liver and ultimately results in progressive cholestasis and liver damage. BA occurs in infants and is estimated to affect one in every 10,000-15,000 live births in the United States. While the underlying...