First Patient Dosed in CAN106 Phase 1b/2 Trial for Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

BEIJING & CAMBRIDGE, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals, Inc. (“CANbridge,” stock code 1228.HK), a China-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that the first patient has been dosed in the CAN106 Phase 1b/2 trial for treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China.

The multi-center, open-label, Phase 1b/2 study to evaluate the tolerability, efficacy, safety and PK/PD of CAN106 administered intravenously to complement inhibitor treatment-naïve PNH patients, is under the direction of principal investigator, Dr. Bing Han, Director of Hematology, at the Peking Union Medical College Hospital.

CAN106 was previously shown to be safe and well-tolerated, with dose-dependent and linear pharmacokinetic exposure, in a study of healthy volunteers in Singapore. The data also showed that free C5 and CH50 could be effectively inhibited. Based on these results, China’s National Medical Products Administration approved the CAN106 Phase 1b/2 trial for the treatment of patients with PNH.

“We are pleased to have dosed the first patient in the CAN106 PNH trial in China so soon after reporting the first human data validating CAN106 as a potential treatment for PNH and other complement-mediated diseases,” said James Xue, Ph.D., Founder, Chairman and CEO, CANbridge Pharmaceuticals Inc. “We look forward to working with the Peking Union Medical College Hospital, one of China’s leading research institutions, with whom we recently signed a rare disease research collaboration, and to bringing this potential new treatment to patients in China, where access to anti-C5 therapies is limited, leaving them with few treatment options.”

About CAN106

CAN106 is a novel, long-acting recombinant human 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), which in the case of paroxysmal nocturnal hemoglobinuria (PNH), is red blood cells. 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 Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) belongs to a group of fatal and rare disorders that occur when the complement system is dysregulated. In patients with PNH, the proteins that normally protect their red blood cells are not present, leaving these denuded cells susceptible to complement attack, which results in their destruction (hemolysis). This leads to severe anemia, thromboembolism, gastrointestinal pain and dysfunction, fatigue, cardiac failure, pulmonary hypertension, renal impairment, and eventually, death. Treatment options include steroids, allogeneic bone marrow transplantation, the anti-C5 monoclonal antibodies eculizumab and ravulizumab, and the C3 inhibitor, pegcetacoplan. PHN is an acquired genetic condition that can occur at any age across genders and race, but 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-to-2 per million people per year. In Asia, the rate is approximately 10 per million people per year, according to the 2019 China Rare Diseases Diagnosis and Treatment Guide. There is only one approved C5-targeting monoclonal therapy approved in China, where cost is also a prohibiting factor.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (“CANbridge,” stock code 1228.HK) is a China-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology.

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.

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Investor Relations:

Chris Brinzey
ICR Westwicke

CANbridge Pharmaceuticals Inc.


Deanne Eagle
Planet Communications

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