CANbridge Pharmaceuticals said that it has dosed the first patient in a phase 1/2 trial of CAN103 in treatment-naïve patients in China having Gaucher disease (GD) Types I and III.

The clinical trial of the recombinant human glucocerebrosidase enzyme replacement therapy is likely to enroll nearly 40 Gaucher disease patients.

Gaucher disease is a lysosomal storage disease resulting from a genetic enzyme deficiency. It leads to an accumulation of the sphingolipid, glucocerebroside, which causes multiple organ complications, bone pain, and death as well.

CAN103 is being developed by CANbridge Pharmaceuticals as part of its partnership in rare diseases with WuXi Biologics.

The multicenter CAN103 phase 1/2 clinical trial will comprise two parts.

Part A, which is phase 1, is an open-label study to assess the safety, tolerability, and pharmacokinetics of various dose levels of the enzyme replacement therapy in very few treatment-naïve patients with Gaucher disease Type I.

Part B or phase 2 will be a randomized, double-blind, parallel group, dose comparison study for evaluating the safety and efficacy of CAN103 in a larger number of patients with Gaucher disease Type I or III.

James Xue — CANbridge Pharmaceuticals Founder, Chairman, and CEO 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 organization in the National Collaborative Network for the Diagnosis and Treatment of Rare Diseases, as we advance this potentially new Gaucher disease treatment.”

Recently, CANbridge Pharmaceuticals dosed the first patient in the phase 2 EMBARK study of CAN108 (maralixibat) in biliary atresia.

  CAN103, CANbridge Pharmaceuticals, China, Gaucher disease, James Xue, Rare diseases, WuXi Biologics