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

BEIJING & CAMBRIDGE, Mass.

CANbridge Pharmaceuticals Inc. (HKEX:1228), a leading China and US-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that the first patient has been dosed in the Phase 2 EMBARK study of CAN108 (maralixibat) in biliary atresia (BA) in China, at Children’s Hospital of Capital Institute of Pediatrics (CIP), in Beijing. The clinical trial in China is part of the global EMBARK study in BA. The multi-center randomized controlled Phase 2 study to evaluate the efficacy and safety of CAN108 in the treatment of patients with BA after Kasai surgery is expected to enroll up to 20 patients in China and 72 patients globally.

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 dosed the first patient in our CAN108 trial in biliary atresia at Beijing’s prestigious Children’s Hospital of Capital Institute of Pediatrics,” said James Xue, Ph.D., Founder, Chairman and CEO, CANbridge Pharmaceuticals. “With the approval of CAN108 for the treatment of Alagille syndrome under the Early and Pilot Implementation Policy in Boao Lecheng International Medical Tourism Pilot Zone, we are advancing the study of CAN108 in two indications in China, where we hope to bring new treatment options for rare liver disease patients.”

About Biliary Atresia
Biliary atresia (BA) is a progressive inflammatory condition of the biliary tree that presents in the first weeks of life and can present with 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 injury, fibrosis and cirrhosis that leads to portal hypertension and liver failure, necessitating liver transplantation, and may be fatal if untreated. Standard of care is the Kasai procedure as a first line intervention. However, approximately 40%–50% of patients undergoing Kasai will require a liver transplantation by 2 years of age, making BA the leading cause of pediatric liver transplantation worldwide, and underscoring the large unmet medical need in BA.

About the EMBARK Study
EMBARK is a Mirum Pharmaceuticals-sponsored global Phase 2 study 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, to 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 orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.

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 committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs 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, as well as glioblastoma multiforme. CANbridge is also building next-generation gene therapy development capability through a combination of collaboration with world-leading researchers and biotech companies and internal capacity. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School, the University of Washington School of Medicine, Scriptr Global and LogicBio.

For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.

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