Beijing, China; Burlington, Mass., January, 18, 2023 — 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 and oncology, announced that the first patient has been dosed in the Phase 2 part of the ongoing 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.

Gaucher disease (GD), one of the most common lysosomal storage disorders, is caused by a genetic enzyme deficiency leading to an accumulation of the sphingolipid, glucocerebroside, primarily in macrophages, which results in hepatosplenomegaly, anemia, thrombocytopenia, bone pain and fractures, 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.  Most patients with Gaucher disease in China do not have access to approved treatments due to cost barriers.

“We are pleased to be moving into the Phase 2 part of our registrational Chinese trial in Gaucher disease,” said James Xue, Ph.D., CANbridge founder, chairman and CEO.  “Gaucher disease was the first rare disease to have an enzyme replacement therapy approved in China. Despite this, most patients still do not have access to treatment.  We hope that, building on the foundation from Phase 1, this additional phase of study will further demonstrate  the safety and efficacy of CAN103 in a larger Gaucher patient group and ultimately pave way for a registration filing.  We are excited that Peking Union Medical College Hospital, which is the leader at the National Collaborative Network for the Diagnosis and Treatment of Rare Diseases, continues to be the leading site, as we advance this potentially new Gaucher disease treatment for medically underserved patients globally.”

“We look forward to moving into Phase 2 with CAN103, which has demonstrated a good safety profile to date,” said Bing Han MD, PhD, Chief Physician and Professor in the Department of Hematology at Peking Union Medical College Hospital. “CAN103 has the potential to provide a much-needed new treatment for Gaucher’s disease, where there is a high unmet medical need in China.”

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 (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 sphingolipid called glucocerebroside (glucosylceramide) within 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 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.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) 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 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. 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) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. Animal data from the SMA gene therapy was presented in 2022 at the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. 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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