SUZHOU —May 21, 2024 — CANbridge Pharmaceuticals, Inc. (1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, announced today that LIVMARLI^® (Maralixibat Chloride Oral Solution /邁芮倍^®) has been granted an expanded label by the National Medical Products Administration (NMPA). This approval extends the use of LIVMARLI for the treatment of cholestatic pruritus in patients with Alagille Syndrome (ALGS) to include those aged three months and older.

In the second half of 2023, LIVMARLI, a treatment of cholestatic pruritus in patients with ALGS aged one year and older, received multiple marketing approvals across mainland China, Hong Kong (3 months and older), and Taiwan. It is also the first and only approved product marketed for the treatment of this condition in these regions throughout Asia. LIVMARLI is approved in the United States for the treatment of cholestatic pruritus in patient with ALGS in patients three months of age and older and treatment of cholestatic pruritus in progressive familial intrahepatic cholestasis (PFIC) patients aged 5 years and older. It is also approved in Europe for patients two months and older. 

CANbridge has an exclusive license from Mirum Pharmaceuticals, Inc. for the development, commercialization and manufacturing, under certain conditions, of LIVMARLI in Greater China for rare liver disease indications including ALGS and PFIC, as well as other select indications.

About LIVMARLI^® (Maralixibat Chloride Oral Solution /邁芮倍^®)

LIVMARLI^® (Maralixibat Chloride Oral Solution /邁芮倍^®) 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 only medication approved in China, the US and EU (2 months and older) to treat cholestatic pruritus associated with Alagille syndrome (ALGS) aged 3 months and older.

LIVMARLI has been granted Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC by the FDA.

About Alagille Syndrome (ALGS)

Alagille syndrome (ALGS) is an autosomal dominant multisystem disorder which can lead to end-stage liver disease and death. Its incidence is between 1/30,000 and 1/50,000^[1]. It has been registered in National Rare Diseases Registry System of China (NRDRS). This disease is characterized by dysplasia of bile ducts and involvement of extrahepatic organs, such as the kidneys and eyes, as well as bones and the cardiovascular system. 100% of patients experience liver involvement ^{[2, 3]}, which often manifests as chronic cholestasis (slowed or stalled bile flow), usually in the neonatal period or within the first 3 months after birth. In addition to jaundice, skin xanthoma and hepatomegaly, patients will also experience severe pruritus ^[4], which can lead to skin disfigurement, emotional disorder, sleep deprivation and interruption of school learning, due to scratching in affected children ^[5]. It seriously affects the growth, development and quality of life of patients ^[6] and can lead to liver transplantation ^[7].

1.Kamath et al, JPGN 2018; 67: 148-156

2.Turnpenny PD, Ellard S. Eur J Hum Genet, 2012;20:251–57.

3.Saleh M, et al. Appl Clin Genet, 2016;9:75–82.

4.Elisofon SA, et al. J Pediatr Gastroenterol Nutr 2010; 51:759–765.

5.Elisofon et al. JPGN. 2010;51: 759-765.

6.Abetz-Webb et al. Hepatology. 2014, 60(4), 526-527.

7.Kamath BM, et al. Hepatol Comms 2020; 4:387–398

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 3 approved drugs and a pipeline of 9 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. 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. 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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China Investor Relations

CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com