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CANbridge Pharmaceuticals Granted Orphan Drug Designation for CAN 106 for the Treatment of Myasthenia Gravis


BEIJING & BURLINGTON, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals, Inc. (1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, announced that CAN106, a clinical-stage recombinant humanized monoclonal antibody targeting C5, has been granted Orphan Drug Designation by the United States Food and Drug administration (FDA) for the treatment of myasthenia gravis (MG), an autoimmune neuromuscular disease that causes weakness in skeletal muscles. CAN106 is in an ongoing Phase 1b/2 trial in paroxysmal nocturnal hemoglobinuria (PNH) in China.

“Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation,” said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. “We continue to advance our global development strategy for CAN106 and look forward to developing CAN106 for myasthenia gravis and other complement-mediated diseases.”

About CAN106

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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 (MAC) 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.

About Myasthenia Gravis

Myasthenia gravis (MG) 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.

About CANbridge Pharmaceuticals Inc.

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 (SMA) 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. For more on CANbridge Pharmaceuticals Inc., please go to:

Forward-Looking Statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the data on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.


U.S. Investor Relations:
Chris Brinzey

ICR Westwicke

China Investor Relations
CANbridge Pharmaceuticals Inc.

Deanne Eagle

Planet Communications

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