BEIJING & CAMBRIDGE, Mass. – CANbridge Pharmaceuticals, Inc., a biopharmaceutical company developing and commercializing innovative drugs to treat rare diseases and targeted cancers in China and globally, announced that the first subject has been dosed in the Phase I trial of CAN106 in Singapore, which is being developed for the treatment of complement dysregulation diseases. The first indication is paroxysmal nocturnal hemoglobinuria (PNH), a fatal disease in which the complement system destroys red blood cells and leads to severe medical problems such as anemia, thromboembolism, gastrointestinal pain and dysfunction, fatigue, pulmonary hypertension, and renal impairment.
This first-in-human trial is a randomized, double-blind, placebo-controlled, single ascending dose study in healthy subjects to assess the safety, pharmacokinetics, and biological activity of CAN106. CAN106 is a recombinant human monoclonal antibody that specifically binds to and neutralizes C5, a component of the complement system, and is intended to prevent the formation of membrane attack complexes on red blood cell surfaces, thereby reducing cell lysis and other symptoms associated with PNH. This proof-of-concept study will evaluate key biomarkers indicating C5 inhibition. Singapore’s Health Sciences Authority (HSA) approved the investigational new drug (IND) application for CAN106 in December 2020. CANbridge is developing CAN106 in conjunction with WuXi Biologics (2269.HK), a global company with leading open-access biologics technology platforms, as part of a strategic partnership for the development of rare disease therapeutics.
“This first clinical trial to arise out of our strategic partnership with WuXi Biologics is a significant milestone for CANbridge as we advance our rare disease programs,” said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals, Inc. “We expect the trial to yield important dosing information and surrogate efficacy signals that will help us effectively advance CAN106 in PNH in Singapore and the rest of the world, and in a full range of complement regulation disorders.”
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PHN) belongs to a group of fatal and rare disorders that occur when the complement system, a part of the immune system, is dysregulated. In PNH, this results in severe anemia, thromboembolism, gastrointestinal pain and dysfunction, fatigue, pulmonary hypertension, renal impairment, and eventually, death. Treatment options are limited to allogenic bone marrow transplant and the anti-C5 monoclonal antibodies, eculizumab and ravulizumab-cwvz. PHN is an acquired condition that can occur at any age across genders and race, but it most commonly presents in adults in their 30s to 40s and continues for the life of the patient. The incidence of PNH in Western countries is estimated to be 1-10 per million per year. In Asia, the rate is approximately 10 per million per year, according to the 2019 China Rare Diseases Diagnosis and Treatment Guide.
About CANbridge Pharmaceuticals Inc.
CANbridge Pharmaceuticals Inc. is a biopharmaceutical company accelerating development and commercialization of specialty healthcare products for orphan diseases and targeted cancers to address unmet medical needs.
CANbridge has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases. In greater China, where it is a recognized leader in orphan diseases, CANbridge has an exclusive licensing agreement to commercialize Hunterase®, an enzyme replacement therapy for the treatment of Hunter syndrome (also known as Mucopolysaccharidosis type II), developed by GC Pharma and marketed in more than 11 countries worldwide. CANbridge also has a collaborative agreement with the Horae Gene Therapy Center at UMass Medical School for the research and development of gene therapies to treat rare genetic diseases.
CANbridge Pharmaceuticals Inc.