SUZHOU – September 30, 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, announced today that CAN103 (velaglucerase-beta) for Gaucher disease has been granted priority review status by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA).

CAN103 is a product of CANbridge’s collaboration with WuXi Biologics (2269.HK) and is the first enzyme replacement therapy (ERT) to be developed in China for Gaucher disease (GD). CAN103 is intended for the long-term treatment of adult and pediatric patients with GD Types I and III. According to Frost & Sullivan, there were approximately 3,000 GD patients in China in 2020. 

 “We are pleased that the CDE has granted CAN103 priority review status, which recognizes the high unmet need of Chinese Gaucher patients and the strength of our clinical trial results. CAN103 has the potential to be a transformational treatment that will contribute significantly to the rare disease ecosystem.”  said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.  “More than 30 years after the approval of the first recombinant ERT for Gaucher disease, most patients in China and many globally still do not have access to existing therapies and represent a continuing underserved population. We are working hard toward accelerating the approval of CAN103 as the first domestically developed and manufactured enzyme replacement therapy in China.

About CAN103

CAN103 (velaglucerase-beta) is a recombinant human glucocerebrosidase ERT that is being developed to treat GD Types I and III, which are the chronic non-neuronopathic and neuronopathic forms of the disease, respectively, that constitute the vast majority of patients.  CAN103 is administered as an intravenous infusion and is intended to supplement the lack of glucocerebrosidase in the lysosomes of GD patients.  

About Gaucher disease (GD)

Gaucher disease, one of the most common lysosomal storage disorders, is a rare inherited genetic metabolic disease caused by autosomal recessive mutations in the GBA gene located on chromosome 1q22 and affects both males and females equally. Gaucher disease is a clinical spectrum that comprises perinatal-lethal, Type I (chronic non-neuronopathic), Type II (acute neuronopathic), and Type III (chronic neuronopathic) forms, with Types I and III surviving into adulthood. Gaucher disease is caused by a deficiency of glucocerebrosidase (acid b-glucosidase), an enzyme that helps break down a cellular membrane glycosphingolipid called glucocerebroside (glucosylceramide) inside lysosomes. As a result, glucocerebroside accumulates primarily in cells of the monocyte-macrophage lineage (Gaucher cells) within certain organs, leading to splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and fractures, and in the most severe forms (perinatal-lethal, Types II and III), early neurological symptoms.  For 30 years, recombinant human glucocerebrosidase enzyme replacement therapy (ERT) has been the standard of care for Gaucher disease, with clinical trials and real-world data demonstrating significant improvement in the major non-neurological signs and symptoms of disease and quality of life. There were 3,000 patients with Gaucher disease in China in 2020, according to Frost & Sullivan.

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. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 9 assets, targeting prevalent rare disease indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, paroxysmal nocturnal hemoglobinuria and other 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.

Contact:

U.S. Investor Relations:

Chris Brinzey

ICR Westwicke

Chris.brinzey@westwicke.com

China Investor Relations

CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com