CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 8 assets, targeting prevalent rare disease indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, paroxysmal nocturnal hemoglobinuria and other complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases. 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,, Duchenne muscular dystrophy (DMD) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, and Scriptr Global.
We are led by a management team with significant industry experience in rare diseases, spanning R&D, clinical development, regulatory affairs, business development and commercialization.As of now, 42% of our employees have a Ph.D. and/or M.D. degree. And more than 70% of our employees have prior experience working at multinational biopharmaceutical companies. Our management team has a track record of successfully achieving approval and commercializing of rare disease therapies across the key markets, including China, the United States. We leverage this expertise to play an active role in advancing the rare disease industry and shaping the rare disease ecosystem in China.
For example, our founder, Dr. James Qun Xue (“Dr. Xue”), Ph. D., is currently serving as the Deputy Director General of China’s Alliance for Rare Disease (CHARD).
Key pipelines and their milestones:
Livmarli® (maralixibat oral solution, formerly known as CAN108), an oral, minimally absorbed, reversible inhibitor of the ileal bile acid transporter (IBAT) that is under development to treat rare cholestatic liver diseases including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC)
In 2023, CANbridge received multiple marketing approvals for Livmarli in mainland China, Hong Kong, and Taiwan. These approvals make Livmarli the first and only approved product marketed for the treatment of cholestatic pruritus in patients with ALGS in these regions.
Hunterase® (idursulfase beta, formerly known as CAN101), an enzyme replacement therapy (ERT) for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome
In September 2020, we received marketing approval for Hunterase (CAN101) for MPS II in mainland China.
CAN106 (omoprubart), a novel, long-acting monoclonal antibody for the treatment of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis (MG) and other diseases that may benefit from treatment with an anti-C5 antibody
In June 2023, CANbridge Reported positive preliminary top-line data from the ongoing Phase 1b study of CAN106 being conducted in PNH patients in China.
In February 2022, a Phase I single ascending dose study of CAN106 in Singapore recorded positive topline results with complete complement blockade.
CAN103, an ERT for the treatment of Gaucher Disease (GD)
In January 2023, the first patient was dosed in phase 2 trial for the treatment of GD in China.In October Full Enrollment Reached in the Core Part of The CAN103 Phase 2 Trial for GD in China.In July 2024,Last Patient Last Visit in CAN103 Phase 2/Registration Trial for GD.
In August 2024,Positive Topline Data from CAN103 Pivotal Trial for GD in China, Plans NDA Filing in Q4 2024.
Gene therapy
In addition to biologics and small molecules, we are investing in next-generation technology for gene therapies. Gene therapies provide a potentially one-time durable treatment for rare genetic diseases that have limited treatment options. As of December 31, 2022, we are developing gene therapies for the treatments of Fabry disease and Pompe disease, which we licensed from LogicBio Therapeutics. In addition, we are internally developing an adeno-associated virus (AAV) delivery platform targeting different tissues, such as the central nervous system (CNS) and muscle.
CANbridge established.
Acquires license for Apogenix's CAN008 for the treatment of glioblastoma in China
Receives approval for CAN008 GBM Phase I/II trial in Taiwan
Doses first patient in CAN008 GBM Phase I/II trial in Taiwan
Submits IND application for CAN008 GBM Phase II/III trial in China
Completes patient enrollment for CAN008 GBM Phase I trial in Taiwan
Receives approval to commence CAN008 GBM Phase II/III trial in China
Enters strategic partnership with WuXi Biologics for rare disease therapeutics development
Submits NDA for CAN101 (Hunterase®) for Hunter syndrome in China
Granted priority review for CAN101 (Hunterase®) in China
Enters research agreement with UMass Medical School for rare disease gene therapy
Enters into second research agreement with Umass Medical school for rare disease gene therapy
First CANbridge rare disease treatment marketing approval in China:
Hunterase®, the first enzyme replacement therapy for Hunter syndrome in the region
CANbridge Pharma announces strategic collaboration with LogicBio Therapeutics, securing licenses to gene delivery and editing platforms
Mirum Pharma and CANbridge Pharma enter into exclusive licensing agreement to develop and commercialize Maralixibat in Greater China for rare liver disease
Obtains CDE clearance from NMPA for an updated Phase II clinical first line trial application for CAN008 in China
Completes $98 million series D and $58 million series E financing
Begins commercialization of Hunterase® (CAN101) in mainland China
Reported Positive Top-Line CAN106 Phase 1 Data
First Patient Dosed in CAN106 Phase 1b/2 Trial for Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
LIVMARLI® New Drug Application/Orphan Drug Registration (NDA/ORD) for Alagille Syndrome Accepted by the Taiwan Food and Drug Administration
CANbridge-UMass Chan Medical School Gene Therapy Research Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
LIVMARLI® New Drug Application (NDA), for Alagille Syndrome, Accepted by China’s National Medical Products Administration
Forms Scientific Advisory Board to Guide Global Development of CAN106 in Complement-mediated Diseases
First Patient Dosed in LIVMARLI® (Maralixibat) EMBARK Phase 2 Study in Biliary Atresia in China
First Patient Dosed in CANbridge Pharmaceuticals CAN103 Phase 1/2 Trial for the Treatment of Gaucher Disease in China
CANbridge-UMass Chan Medical School Gene Therapy Research in Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual Congress
Orphan Drug Designation Granted to CAN 106 for the Treatment of Myasthenia Gravis
First Patient Dosed in CAN103 Phase 2 Trial for the Treatment of Gaucher Disease in China
CANbridge and UMass Chan Medical School reported at the 26th ASGCT Annual Meeting that Spinal Muscular Atrophy gene therapy (CAN203) extends life and improves motor function in mice via intracerebroventricular injection
Livmarli® was approved for marketing in mainland China
Positive preliminary data were obtained from the Phase 1b clinical trial of omoprubart (CAN106) injection for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China
Livmarli® was approved for marketing in Hong Kong
Livmarli® was approved for marketing in Taiwan
CAN103 for the treatment of Gaucher disease completed patient enrollment in the core part of the Phase 2 clinical trial in China
Abstract of gene therapy for Fabry disease presented at the 30th Annual Meeting of the European Society for Gene and Cell Therapy