China’s CANbridge Reports Positive Phase 1 Data of Contender to AstraZeneca’s Complement Inhibitor

Chinese regulators have cleared CANbridge Pharmaceuticals to begin a Phase 1b/2 efficacy study of CAN106, its candidate for treating complement-mediated diseases, after the firm reported positive early phase data of the drug in paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare disease characterized by the destruction of red blood cells. 

Complement Diseases and Treatments

Certain proteins protect red blood cells from destruction. Due to a genetic mutation in the stem cells, red blood cells of people with PNH lack the protective proteins, resulting in their destruction by complements, part of the body’s innate immune system. 

Patients with PNH often require red blood cell transfusions to make up for the lost amount, and other treatments for their anemia, including folic acid and iron supplementation. 

In select cases, bone marrow transplantation is done to replace a patient’s defective stem cells with a donor’s healthy cells.

US-approved treatments for PNH include Soliris (eculizumab) and Ultomiris (ravulizumab), monoclonal antibodies that bind to C5 complements in the blood. 

CANbridge is positioning CAN106, a monoclonal antibody that inhibits C5, as the superior option to rival the mainstay anti-C5 therapies, which are developed by AstraZeneca’s Alexion. The company pointed out that these therapies have limited coverage around the world outside the US, and are gated by high costs. 

CANbridge’s push is especially relevant in China, where Soliris is the only approved drug for PNH. As Ultomiris has not been approved, the country still lacks a long-acting therapy for the disease. 

To address the medical need, CANbridge has worked with WuXi Biologics to co-develop CAN106 as a long-acting treatment for PNH and other complement diseases.  

Related Article: Sanofi’ Enjaymo Wins FDA Approval for Rare Autoimmune Anemia

Topline Data of the Phase 1 Trial

The Phase 1 trial involved 31 healthy volunteers and was conducted in Singapore. CANbridge reported that CAN106 was safe and well-tolerated with only mild to moderate side effects. 

The participants were followed for at least 112 days, and the data in the highest two dose cohorts (8 and 12 mg/kg) showed that CAN106 reduced levels of free C5 by over 99%. The groups also sustained over 90% inhibition of CH50 for 2 to 4 weeks. CH50>90% inhibition is a measure that indicates complete blockage of the classical and terminal complement pathways. 

James Xue, CEO of CANbridge, has applauded the positive results. 

“We look forward to advancing CAN106 in PNH as a first-in-class treatment in China, and as a best-in-class treatment in many markets, where PNH treatment options are few, and to expand CAN106 clinical development globally for other complement-mediated disease indications as either a first-in-class or best-in-class therapy,” he said. 

Related Article: How CDMOs are Restructuring the Biopharma Landscape – An Interview with Bobby Sheng

Author

Joy Lin

Without knowledge, there is no imagination. Without imagination, knowledge is meaningless