CANbridge Pharmaceuticals has dosed the first subject in Phase I/II CAN103 clinical trial in treatment-naïve Gaucher disease (GD) Types I and III patients in China. 

A recombinant human glucocerebrosidase enzyme replacement therapy (ERT), CAN103 is offered intravenously.

It is developed to supplement glucocerebrosidase deficiency in the lysosomes of patients with GD. 

CANbridge is developing CAN103 under a rare disease collaboration with WuXi Biologics, as long-term therapy for Gaucher disease Types I and III in adults and children.

Peking Union Medical College Hospital in Beijing, China will be the initial trial site of the study.

The multi-site trial with two parts will enrol nearly 40 subjects. 

An open-label study, Part A (Phase I) will analyse the safety, tolerability and pharmacokinetics of different doses of CAN103 ERT in treatment-naïve participants with GD Type I. 

The randomised, parallel-group, double-blind, dose-comparison Part B (Phase II) study will evaluate the safety and efficacy of CAN103 in GD Type I or III patients.

CANbridge Pharmaceuticals founder, chairman and CEO James Xue said: “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. 

“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 organisation in the National Collaborative Network for the Diagnosis and Treatment of Rare Diseases, as we advance this potentially new Gaucher disease treatment.” 

Autosomal recessive mutations in the GBA gene on chromosome 1 causes GD, a rare inherited genetic metabolic ailment.