SUZHOU – Aug 7, 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 disease, is pleased to share the news that the Chinese Journal of Pediatrics published the Expert Consensus on Diagnosis and Treatment of Alagille Syndrome (ALGS) Associated Liver Disease (2024), recommending maralixibat (Livmarli) as drug treatment for ALGS.

This consensus was jointly initiated by the Genetic Liver Disease Branch of the China Rare Disease Alliance, the Infectious Diseases Group of the Pediatrics Branch of the Chinese Medical Association, and the Editorial Committee of the Chinese Pediatric Journal in October 2023. It was developed by 5 experts and an additional invited 15 experts with rich clinical and basic research experience in the field of pediatric liver disease by incorporating clinical experience and considerable discussion based upon systemic literature retrieval and evaluation on ALGS related evidence, for the purpose of promoting the precision diagnosis and treatment of ALGS.  Prior to this, there were no disease consensus or guidelines about ALGS in China.

The consensus mentions that ileal bile acid transporter ( IBAT) inhibitors can alleviate the toxic effects of high-load bile acids on the liver and improve cholestasis by inhibiting the enterohepatic circulation of bile acids to treat cholestasis and pruritus in ALGS patients. Real-world data from Global Alagille Alliance (GALA) found that treatment with maralixibat can reduce the risk of clinical events (biliary diversion surgery, decompensated cirrhosis, liver transplantation or death) in ALGS patients by 70% over 6 years. Long-term treatment with maralixibat is expected to reduce the risk of liver-related adverse events.

“We believe the publication of this consensus will tremendously assist the diagnosis and treatment of rare disease ALGS in China. We hope to reduce the risk of liver-related complications in children with ALGS, improve their quality of life and survival rate, and bring more welfare to the group of patients with rare diseases,” said Prof. Wang Jianshe from Center for Children's Liver Disease in Children's Hospital of Fudan University 

“We are pleased that the first consensus on ALGS related liver disease was published in China.  Livmarli is recommended in the consensus, with the hope of improving the symptom and long term outcome of more ALGS patients” said James Xue, Ph.D., CANbridge founder, chairman, and CEO. “CANbridge has always been committed to helping rare disease patients through the development of breakthrough therapies. Livmarli is the first and only drug approved in China for the treatment of cholestatic pruritus in ALGS patients aged 3 months and above. We look forward to collaborating with medical society to disseminate the consensus and promote the early diagnosis and treatment of ALGS.”

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

LIVMARLI^® (Maralixibat Chloride Oral Solution /迈芮倍^®) 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 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), Duchenne muscular dystrophy (DMD) 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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