SUZHOU – August [19], 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 positive topline results from the CAN103 pivotal trial, in treatment-naïve subjects aged 12 or above with Gaucher disease (GD) Types I and III.

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 (Center for Drug Evaluation of NMPA).

All key secondary endpoints were also met with statistical significance at the 60 U/kg dose, including reductions in liver volume, increases in hemoglobin levels, and increases in platelet counts from baseline. At the 30 U/kg dose, statistically significant improvements were observed in liver volume and hemoglobin levels, along with an increase in platelet counts from baseline. Greater improvements were observed with the higher dose than with the lower dose.

Furthermore, both the 60 U/kg and 30 U/kg treatments resulted in statistically significant reductions in the Gaucher Disease biomarker plasma Lyso-GL-1 levels compared to baseline, with a more rapid reduction seen at the 60 U/kg dose, further supporting the clinical response to treatment. CAN103 was well tolerated at both doses, with no drug-related serious adverse events reported. Most adverse events were mild, unrelated to the drug, and transient in nature. Less than 10% of subjects had hypersensitivity reactions and all continued treatment, and less than 10% of subjects tested positive for anti-drug antibodies at more than one timepoint.

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

“We are extremely encouraged by the strong data showing that CAN103 has promising efficacy results with a  favorable safety profile,” said Bing Han MD, PhD, Chief Physician and Professor in the Department of Hematology at Peking Union Medical College Hospital in Beijing, China, and principal investigator for this multi-site trial, “Today, most patients in China still do not have access to existing enzyme replacement therapies for Gaucher disease. The results of this pivotal trial suggest that CAN103 has the potential to become an attractive treatment alternative for Gaucher disease patients.”

“We would like to express our deepest gratitude to the investigators and patients who participated in this study. Their dedication and commitment have brought us one step closer to new treatment option for GD patients - the first GD ERT treatment that is locally developed and manufactured in China. said James Xue, Ph.D., founder, chairman and CEO of CANbridge Pharmaceuticals Inc.  “This breakthrough reinforces our commitment to improving patient outcomes and addressing the significant unmet needs in this therapeutic area. We plan to submit the New Drug Application (NDA)  to NMPA in the fourth quarter of 2024.“

About CAN103

CAN103 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 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-glucosidase), an enzyme that helps break down a cellular membrane sphingolipid 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 more than 25 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, 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 relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the data on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.