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CANbridge Pharmaceuticals CAN103 Phase 1/2 Trial for the Treatment of Gaucher Disease in China: 1st Patient Dosed

Peking Union Medical College Hospital Primary Clinical Trial Site

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CANbridge Pharmaceuticals Inc. (HKEX:1228), a China and US-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that the first patient has been dosed in the CAN103 Phase 1/2 trial in treatment-naïve patients with Gaucher disease (GD) Types I and III in China.

Bing Han MD, PhD, Chief Physician and Professor in the Department of Hematology at Peking Union Medical College Hospital in Beijing, China, is the principal investigator for this multi-site trial, which is expected to enroll approximately 40 subjects. Gaucher disease (GD), a lysosomal storage disease, is caused by a genetic enzyme deficiency leading to an accumulation of the sphingolipid, glucocerebroside, which results in multiple organ complications, bone pain and death. CAN103 is an enzyme replacement therapy (ERT) under development by CANbridge, as part of its rare disease partnership with WuXi Biologics (2269.HK), for the long-term treatment of adults and children with Gaucher disease Types I and III. Many GD patients in China do not have access to approved treatments due to cost barriers.

This multicenter Phase 1/2 clinical trial will consist of two parts. Part A (Phase 1) is an open-label study to evaluate the safety, tolerability and pharmacokinetics of different dose levels of CAN103 ERT in a small number of treatment-naïve subjects with Gaucher disease Type I. Part B (Phase 2) is a randomized, double-blind, parallel group, dose comparison study to assess the safety and efficacy of CAN103 in a larger number of subjects with Gaucher disease Type I or III.

“Dosing the first patient in the CAN103 Gaucher disease trial demonstrates the CANbridge commitment to developing rare disease solutions. More than 25 years after 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,” said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. “We are proud to have worked with the China Alliance for Rare Disease (CHARD) and other rare disease stakeholders to bring this trial to fruition and look forward to our collaboration with Peking Union Medical College Hospital, the lead organization in the National Collaborative Network for the Diagnosis and Treatment of Rare Diseases, as we advance this potentially new Gaucher disease treatment.”

About CAN103: CAN103 is a recombinant human glucocerebrosidase enzyme replacement therapy that is being developed to treat Gaucher disease (GD) Types I and III, which are the chronic non-neuronopathic and neuronopathic forms of the disease that constitute the majority of patients. CAN103 is delivered intravenously 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 1 and affects both males and females equally. Gaucher disease is a clinical spectrum that comprises perinatal-lethal, Type I (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 β-glucosidase), an enzyme that helps break down a cellular membrane sphingolipid called glucocerebroside (glucosylceramide) within lysosomes. As a result, glucocerebroside accumulates primarily in cells of the monocyte-macrophages 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 more than 25 years, recombinant human glucocerebrosidase enzyme replacement therapy (ERT) has been the standard of care for Gaucher disease, with clinical trial 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.

CANbridge Pharmaceuticals Inc. (HKEX:1228) is a China and US-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 10 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, as well as glioblastoma multiforme. CANbridge is also building next-generation gene therapy development capability through a combination of collaboration with world-leading researchers and biotech companies and internal capacity. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School, the University of Washington School of Medicine, Scriptr Global and LogicBio.

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