• CAN106 Phase 1 results showed rapid and potent C5 inhibition, complete and sustained blockade of complement activity
• Data presented at European Hematology Association 2022 Congress and 14^thInternational Conference on Complement Therapeutics
• CAN106 in Phase 1b/2 study of paroxysmal nocturnal hemoglobinuria in China
• Shows potential for treating other complement-mediated diseases

BEIJING and CAMBRIDGE, Mass., July 6, 2022—CANbridge Pharmaceuticals Inc. (HKEX:1228), a China and US-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that it has formed a Complement Disease Scientific Advisory Board focused on the global development of CAN106, a novel, long-acting monoclonal antibody directed against C5 complement.  CANbridge is developing CAN106 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and other complement-mediated diseases. CAN106 is currently in a Phase 1b/2 PNH trial in China. The Board will offer guidance on the CAN106 clinical development program, as well as explore the potential for CAN106 in other indications.

The Scientific Advisory Board members are:

Anthony Amato, MD, Brigham and Women’s Hospital Distinguished Chair in Neurology, Chief of the Neuromuscular Division and Director of the Clinical Neurophysiology Laboratory at Brigham and Women’s Hospital, and Professor of Neurology at Harvard Medical School. Dr. Amato’s clinical research interests include neuromuscular junction disorders, myopathies, amyotrophic lateral sclerosis, and peripheral neuropathies. 

Robert Colvin, MD, Pathologist-in-Chief, Emeritus at Massachusetts General Hospital, and the Benjamin Castleman Distinguished Professor of Pathology at Harvard Medical School.  Dr. Colvin’s research interests include acute and chronic antibody-mediated organ rejection, chronic arteriopathy following long-term organ grafts, and the immunopathogenesis of renal diseases.

Jean Francis, MD, Medical Director of the Kidney Transplant Program at Boston Medical Center and Boston University School of Medicine, Medical Director of the Combined Pancreas Transplant Program at Boston Medical Center and Brigham and Women’s Hospital, and Associate Professor of Medicine at Boston University School of Medicine.  In addition to organ transplantation, Dr. Francis’ clinical research interests include thrombotic microangiopathy.

Richard Polisson, MD, MHSc, Clinical Development Consultant and, most recently, former Chief Medical Officer at Artax Biopharma.  Previously, Dr. Polisson was Senior Vice President and Head of Translational Medicine at Sanofi-Genzyme Research and Development Center, Clinical Director of the Arthritis Unit and Chair of the Mallinckrodt Clinical Research Unit Scientific Advisory Committee at Massachusetts General Hospital, and Associate Professor of Medicine at Harvard Medical School.  Dr. Polisson’s clinical research interests include rheumatologic, autoimmune, and immunologic diseases as well as drug development for rare diseases.

Sushrut Waikar, MD, MPH, Chief of Nephrology at Boston Medical Center and the Norman G. Levinsky Professor of Medicine at Boston University School of Medicine,and formerly the Constantine L. Hampers, MD Distinguished Chair in Renal Medicine at Brigham and Women’s Hospital.  Dr. Waikar’s research focuses on biomarkers and therapeutic targets for acute and chronic kidney disease, noninvasive biomarkers of kidney pathology and fibrosis and randomized controlled trials in nephrology.

Brian Weinshenker, MD, Professor of Neurology at the University of Virginia, and formerly Professor of Neurology at Mayo Clinic.  Dr. Weinshenker’s clinical research interests include the diagnosis and treatment of inflammatory demyelinating diseases of the central nervous system, including fulminant demyelination, neuromyelitis optica and multiple sclerosis.

Phase 1 results from a single ascending dose study in healthy subjects were presented at the European Hematology Association (EHA) 2022 Congress held in Vienna, Austria from June 9-12, and at the 14th International Conference on Complement Therapeutics held in Rhodes, Greece from June 17-22.  CAN106 achieved rapid, dose-dependent reductions in both free C5 complement, the target of CAN106, and in CH50, a measure of serum hemolytic activity, within 24 hours of dosing.  All subjects in the two highest dose groups of CAN106 (8 and 12 mg/kg) showed >99% reduction in free C5 and ³90% inhibition of CH50.  The latter indicates complete blockade of terminal complement activity, which was sustained for up to 4 weeks. CAN106 was well tolerated and had a favorable safety profile.  The half-life of CAN106 in healthy subjects was 31 days, suggesting the potential for an extended dosing interval in patients. CAN106 is currently being investigated in a Phase 1b/2 clinical trial in subjects with PNH in China. Based on its mechanism of action, CANbridge believes CAN106 holds promise for the treatment of several complement-mediated diseases.

“Our CAN106 program for complement-mediated diseases is gaining momentum this year with the first public presentation of the Phase 1 clinical trial results at two major scientific conferences and the announcement of a CAN106 Scientific Advisory Board,” stated Gerry Cox, MD, PhD, Interim Chief Medical Officer and Chief Development Strategist at CANbridge and Chair of the CANbridge Complement Disease Scientific Advisory Board.  “Based on encouraging Phase 1 results, we initiated a Phase 1/2 study of CAN106 in patients with PNH in China in March 2022.  We have assembled a distinguished CAN106 Scientific Advisory Board whose members have a wealth of clinical experience across diverse disease areas in which C5 activation is either known or believed to play an important role and where an anti-C5 therapeutic may be beneficial.  We have only skimmed the surface in terms of opportunities for CAN106, and our board members will help us to prioritize which diseases to study next.”

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) is a China and US-based global biopharmaceutical company 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. CANbridge is also building next-generation gene therapy development capability through a combination of collaboration with world-leading researchers and biotech companies and internal capacity. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School, the University of Washington School of Medicine, Scriptr Global and LogicBio.

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

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.

Contact:

Investor Relations:

US

Chris Brinzey

ICR Westwicke

Chris.brinzey@westwicke.com

China

CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com

Media:

Deanne Eagle

Planet Communications

deanne@planetcommunications.nyc

917.837.5866