First Patient Treated in EMBARK CAN108 (Maralixibat) Phase 2 Biliary Atresia Study in China

BEIJING AND CAMBRIDGE, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals Inc. (HKEX:1228), a leading global biopharmaceutical company based in China and the United States, engaged in the research, development and commercialization of transformative therapies for rare diseases and oncology rare, announced that the first patient received a dose in the Phase 2 EMBARK Study of CAN108 (maralixibat) in Biliary Atresia (BA) in China, at the Capital Institute of Pediatrics (CIP) Children’s Hospital, Beijing. The clinical trial in China is part of the global EMBARK study in BA. Multicenter Randomized Controlled Phase 2 Study to Evaluate the Efficacy and Safety of CAN108 in Treating Patients with BA After Surgery in Kasai is Expected to Recruit Up to 20 Patients in China and 72 Patients Worldwide .

CANbridge and Mirum Pharmaceuticals, Inc. have an exclusive license agreement for the development, commercialization and manufacture, under certain conditions, of maralixibat (CAN108) in Greater China. Under the terms of the agreement, CANbridge has the right to develop and commercialize CAN108 for three indications: Alagille syndrome, progressive familial intrahepatic cholestasis and biliary atresia in Greater China.

“We are delighted to have assayed the first patient in our CAN108 trial in biliary atresia at the prestigious Children’s Hospital of the Capital Institute of Pediatrics,” said James Xue, Ph.D., Founder, President and CEO of CANbridge Pharmaceuticals. “With the approval of CAN108 for the treatment of Alagille syndrome under the early and pilot implementation policy in the Boao Lecheng international medical tourism pilot area, we are advancing the study of CAN108 in two indications in China, where we hope to bring new treatment options for patients with rare liver disease.

About biliary atresia

Biliary atresia (BA) is a progressive inflammatory condition of the biliary tree that presents during the first weeks of life and may manifest as prolonged jaundice, acholic stools and dark urine. The etiology and pathogenesis of BA remain elusive and are likely to be multifactorial. The estimated prevalence of BA in Western countries is between 0.5 and 0.8 per 10,000 live births, which is lower than that reported in Asians (Japan 1.04/10,000, Taiwan region 1.78 /10,000). BA is recognized as an orphan disease in Western countries. It is associated with cholestatic liver damage, fibrosis, and cirrhosis that lead to portal hypertension and liver failure, requiring liver transplantation, and can be fatal if untreated. The standard of care is the Kasai procedure as the first line intervention. However, approximately 40% to 50% of patients undergoing Kasai will require liver transplantation by age 2, making BA the leading cause of pediatric liver transplantation worldwide and underscoring the significant unmet medical need. in BA.

About the EMBARK Study

EMBARK is a global Phase 2 study sponsored by Mirum Pharmaceuticals to evaluate the efficacy and safety of maralixibat in the treatment of patients with BA after Kasai surgery (NCT04524390). The 26-week randomized controlled trial, which will be followed by a 78-week open-label extension study, is being conducted at multiple sites in North America, Europe and Asia, including China. There are currently no pharmacological agents approved for the treatment of patients with biliary atresia.

About LIVMARLI® (maralixibat) oral solution LIVMARLI® (maralixibat) oral solution is an oral, once-daily ileal bile acid transporter (IBAT) inhibitor approved by the United States Food and Drug Administration for the treatment of cholestatic pruritus in patients with the syndrome d’Alagille (ALGS) ages one year and older and is the only drug approved by the FDA to treat cholestatic pruritus associated with Alagille syndrome. For more information, visit

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC) and biliary atresia. LIVMARLI has received Breakthrough Therapy Designation for ALGS and PFIC Type 2 and Orphan Designation for ALGS, PFIC and Biliary Atresia.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX: 1228) is a China-based global biopharmaceutical company dedicated to the research, development and commercialization of transformative therapies for rare diseases and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 10 assets, targeting prevalent rare diseases and rare oncology indications that have unmet need and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, and glioblastoma multiforme. CANbridge is also developing next-generation gene therapy development capability through collaboration with world-renowned researchers and biotech companies and in-house capability. CANbridge’s global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, University of Washington School of Medicine, Scriptr Global and LogicBio.

To learn more about CANbridge Pharmaceuticals Inc., visit:

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