18 November 2022 - - China-based biopharmaceutical company CANbridge Pharmaceuticals, Inc's (1228.HK) CAN106, a clinical-stage recombinant humanized monoclonal antibody targeting C5, has been granted Orphan Drug Designation by the United States Food and Drug administration for the treatment of myasthenia gravis, an autoimmune neuromuscular disease that causes weakness in skeletal muscles, the company said.

CAN106 is in an ongoing Phase 1b/2 trial in paroxysmal nocturnal hemoglobinuria in China.

CAN106 is a clinical-stage investigational novel, long-acting recombinant humanized monoclonal antibody that binds to and neutralizes C5, a key component of the complement system.

By preventing the cleavage of C5 into C5a and C5b, CAN106 is intended to prevent the C5b-dependent formation and activation of the membrane attack complex on susceptible cell surfaces, with resulting cell lysis (destruction).

CAN106 acts downstream of C3 in the complement pathway, preserving the generation of C3a and C3b, which are important for innate immunity.

CAN106 has demonstrated a favorable PK/PD profile, safety, and tolerability, indicating that CAN106 has the potential to effectively inhibit C5 in patients with certain complement-mediated diseases.

Myasthenia gravis is a rare, acquired, neuromuscular disease that causes weakness in the muscles under voluntary control, including those involved in breathing, swallowing, eye movement and moving the arms and legs.

MG is caused when the immune system blocks or destroys muscle fiber sites that normally receive acetylcholine, a brain chemical that activates muscle contraction. MG can result in hospitalization and the need for ventilation.

MG can occur at any age, although it most commonly affects women under 40 and men over 60.

According to the Myasthenia Gravis Foundation of America, it is estimated that 20 in 100,000 Americans have MG, or approximately 36,000 to 60,000 cases, but given that the disease is underdiagnosed, these numbers could be much higher. There is no cure.

CANbridge Pharmaceuticals Inc. (HKEX: 1228) global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology.

CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology 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, as well as glioblastoma multiforme.

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 and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies.

Animal data from the SMA gene therapy was presented at the American Society for Gene and Cell Therapy (ASCGT), the European Society for Gene and Cell Therapy (ESCGT) and the World Muscle Congress.

CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global.