CANbridge Pharmaceuticals Inc. (HKEX:1228), a leading China-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that the China region of the global Phase 2 EMBARK study of LIVMARLI® (maralixibat) oral solution (CAN108) in biliary atresia (BA) is fully enrolled, with nearly twice the expected number of patients. This global, multi-center, randomized controlled Phase 2 study, evaluating the efficacy and safety of LIVMARLI in patients with BA after Kasai surgery, is sponsored by Mirum Pharmaceuticals, Inc. and has also completed enrollment across the other global locations where the trial is open.

CANbridge has an exclusive license agreement for the development, commercialization and manufacturing, under certain conditions, of LIVMARLI in Greater China. In this region, CANbridge has the right to develop and commercialize LIVMARLI for three indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA). The National Medical Products Administration (NMPA) approved LIVMARLI for the treatment for ALGS under the Early and Pilot Implementation Policy in the Boao Lecheng International Medical Tourism Pilot Zone in China, allowing its use in the zone prior to national marketing authorization. LIVMARLI has been granted priority review by the NMPA for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age or older, with a potential for approval in the first half of 2023. CANbridge also expects to file an NDA for LIVMARLI for the treatment of PFIC with the NMPA soon.

“Enrollment in the LIVMARLI BA trial in China is nearly double what we had anticipated, which points to the dire need of effective treatments for this disease,” said Gerry Cox, MD, PhD, chief development strategist and interim chief medical officer, CANbridge Pharmaceuticals. “We are pleased to have met, and exceeded, full enrollment in this trial and look forward to the continued development of this promising treatment for patients in Greater China.”

About LIVMARLI

LIVMARLI® (maralixibat) oral solution (CAN108) is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor that blocks the enterohepatic circulation of bile acids, reduces bile acid levels in the liver and serum, reduces the resultant liver injury and relieves pruritus (extreme itching). LIVMARLI is the first, and currently, only medication approved in the US (3 months and older) and EU (2 months and older) to treat cholestatic pruritus associated with ALGS and has been granted FDA Breakthrough Therapy designation. Mirum has also filed for approval of LIVMARLI in U.S. and Europe for PFIC (in US for cholestatic pruritus in patients 3 months and older and in Europe for PFIC in patients two months of age and older).

LIVMARLI is under clinical development by CANbridge for the treatment of cholestatic liver diseases, including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA).

CANbridge acquired the exclusive right to develop and commercialize LIVMARLI in Greater China from Mirum Pharmaceuticals, Inc. for ALGS, PFIC and BA.

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 Asian countries (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.

IMPORTANT SAFETY INFORMATION, U.S. INDICATION

LIVMARLI can cause side effects, including:

Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen) or loss of appetite.

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.

A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.

Other common side effects reported during treatment were gastrointestinal bleeding and bone fractures.

US Prescribing Information

EU SmPC

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, 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. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. Animal data from the SMA gene therapy was presented in 2022 at the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global.

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

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