CANbridge Pharmaceuticals CAN106 Investigational New Drug Application Approved in China for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

BEIJING & CAMBRIDGE, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that the Chinese National Medical Products Administration (NMPA) has approved an Investigational New Drug (IND) application for CAN106, a long-acting humanized monoclonal antibody targeting complement C5 of the complement system, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). PNH is a fatal disease in which the complement system eventually destroys red blood cells. CAN106 was granted an IND by Singapore�s Health Sciences Authority (HSA) in December 2020, where it is currently in a Phase I safety trial.

CANbridge is developing CAN106 for the treatment of multiple diseases associated with complement dysregulation, including PNH. CAN106 comes out of the strategic partnership between CANbridge and WuXi Biologics (2269.HK), in collaboration with Privus Biologics, for the development of rare disease therapeutics.

“This is the first NMPA IND approval for CAN106, which is also under evaluation in a Phase I trial in Singapore,” said James Xue, Ph.D. Founder, Chairman and CEO of CANbridge Pharmaceuticals Inc.“ It demonstrates and reinforces our commitment to, and our strategy of, building global integrated preclinical and clinical capabilities for the efficient development of global treatments that could address multiple indications with significant market potential.”

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) 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 the destruction of the red blood cells, which leads to severe anemia and, eventually, death. Treatment options are limited to allogenic bone marrow transplant and the monoclonal antibody treatments, eculizumab and ravulizumab-cwvz. PNH may occur at any age across gender and race, but it is more prevalent among females and people in their 30s and 40s. It continues throughout the life of the patient. The PNH incidence rate in Western countries is 1-2 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 China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies.

CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with significant market potential, targeting some of the most prevalent rare diseases and rare oncology.

These include Hunter syndrome (MPS II) and other lysosomal storage disorders (LSDs), complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases as well as glioblastoma multiforme (GBM).

CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments. CANbridge global partners include, but are not limited to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Medical School (UMass) and LogicBio.

For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.

Contacts

Investor Relations:

ir@canbridgepharma.com

Media:

Deanne Eagle

Planet Communications

deanne@planetcommunications.nyc
917.837.5866