SUZHOU, China, March 13, 2025 - 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 velaglucerase-beta for injection, with its wholly - owned subsidiary, CANbridge (Shanghai) Life Sciences Ltd. as the holder, successfully passed the Pre-approval inspection and pre - marketing GMP compliance inspection for the pilot biological product of Divided manufacturing. The inspection was coordinated by the CFDI (Center of Food and Drug Inspection)  of the National Medical Products Administration and carried out by a collaborative inspection team composed of the Shanghai Drug Evaluation and Inspection Center and the CDI (Center of Drug Inspection) of Jiangsu province.

Velaglucerase-beta for injection is the first innovative biological product in China to pass the inspection of Divided manufacturing of biological products.

Under the overall coordination of the CFDI of NMPA the Shanghai Drug Evaluation and Inspection Center and CDI of Jiangsu MPA (Medical Product Adminstration) covered the entire dynamic production process in Shanghai and Jiangsu through serial inspections. This enabled seamless traceability of production and inspection data at all stages, achieved information sharing and intercommunication in the whole - chain production quality management, ensured that "segmentation does not reduce responsibilities and collaboration does not break the chain", and accelerated the Approval process of innovative drugs for rare diseases.

“Velaglucerase-beta for injection was approved as one of the first domestic pilot projects for Divided manufacturing of biological products in Shanghai after the NMPA  issued the "Pilot Work Plan for Biological products divided manufacturing" on October 21, 2024. Based on the characteristics of rare - disease drug research, development, and production, CANbridge (Shanghai) Life Sciences Ltd. directly applied for NDA using the existing segmented production process, which greatly accelerated the speed of NDA. Under the overall coordination and careful organization of the CFDI of NMPA , the drug regulatory departments of the two regions collaborated across provinces, advanced in an integrated manner, broke geographical boundaries, improved the development progress and application quality of innovative drugs for rare diseases, and will help the first domestic ERT (enzyme replacement treatment) to be launched in Shanghai.” said James Xue, Ph.D., CANbridge Founder, Chairman, and CEO. “Here, we would like to express our gratitude to our partners who have empowered CANbridge to develop velaglucerase-beta for injection, including WuXi Biologics. We also thank the experts of the inspection team for their strict and professional inspection. We are grateful for the opportunity given by the times to witness history and create the future together. We believe that the successful implementation of the inspection for the Divided manufacturing  pilot of velaglucerase-beta for injection will provide useful experience for the NMPA to explore and establish a replicable and promotable regulatory system for the divided manufacturing of biological products.”

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 disease. CANbridge has a differentiated drug portfolio, with 2 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, 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.

Forward - Looking Statements

The forward - looking statements made in this article are only related to the events or information as of the date of making the statements in this article. Unless required by law, we have no obligation to publicly update or revise any forward - looking statements, whether due to new information, future events, or other reasons, after the date of the data on which the statements are based or to reflect the occurrence of unforeseen events. You should read this article in its entirety and be aware that our actual future results or performance may differ materially from our expectations. In this article, any statements or references to the intentions of us, any of our directors, or our company are made as of the date of this article. Any of these intentions may change with future developments.

Contact:

China Investor Relations

CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com