SUZHOU – November 13, 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, announced today that the Chinese National Medical Products Administration (NMPA) has accepted the New Drug Application (NDA) for CAN103 (velaglucerase-beta for jnjection) for the treatment of Gaucher disease (GD).

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 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. 

“The acceptance of the NDA for CAN103 by the NMPA is a significant milestone for CANbridge and the Gaucher community. Once approved, CAN103 will be the first domestically developed ERT to be clinically prescribed  in China. It is a  Class-I innovative drug that can fully replace similar imported products.  In the meantime, CAN103 has accumulated a full set of R&D data and cost information spanning its entire development process, providing a first case study on the development and commercialization of a rare diseases therapy for  meeting the long-standing treatment needs of patients.” said James Xue, Ph.D., CANbridge Founder, Chairman, and CEO. “We expect to launch CAN103 in 2025, marking the first self-developed product to enter the market.  CAN103 will be the third rare disease treatment commercialized by CANbridge after Hunterase and Livmarli.

In August this year, CANbridge Pharmaceuticals announced positive top-line data results from the pivotal clinical trial of CAN103 for adult and pediatric patients with GD. In September, the Center for Drug Evaluation (CDE) of the NMPA granted CAN103 priority review status. In October, CANbridge Pharmaceuticals successfully passed the production and quality management system audit and on-site inspection for the Marketing Authorization Holder (MAH), obtaining the Drug Manufacturing License Type B Certificate. On October 22, 2024, the NMPA officially issued the ‘Pilot Work Plan for Segmented Production of Biological Products.’ Through close cooperation between CANbridge Pharmaceuticals and WuXi Biologics, both parties successfully obtained the relevant biological product manufacturing licenses, becoming the first practitioners of segmented manufacturing of biological products under the MAH system in China.

About CAN103

CAN103 (velaglucerase-beta for injection) is the first domestically developed recombinant human glucocerebrosidase ERT in China, soon to enter clinical application for the treatment of Type I and Type III Gaucher disease patients. Most Gaucher disease patients are Type I and Type III, which are chronic non-neuronopathic and chronic neuronopathic forms, respectively. CAN103 is administered as an intravenous infusion and is intended to supplement the lack of glucocerebrosidase in the lysosomes of GD patients.  

The pivotal clinical trial of CAN103 achieved positive top-line data in August 2024. The CAN103 pivotal trial is a randomized, double-blind, dose-comparison study designed to evaluate the efficacy, safety, and pharmacokinetics of intravenous CAN103 administered every other week in newly treated Gaucher Disease (GD) patients, with an extension period. The results demonstrate that the study successfully met its primary efficacy endpoint, showing a statistically significant mean percentage reduction from baseline in spleen volume at nine months for both the 60 U/kg dose (P<0.0001) and the lower 30 U/kg dose (P<0.001).  The primary endpoint of this trial protocol has been agreed upon by the CDE.

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 Type 0 (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 diseases. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 8 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,  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, the University of Washington School of Medicine and Scriptr Global.

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

China Investor Relations

CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com