CANbridge Pharmaceuticals CAN103 Investigational New Drug Application Accepted by China’s National Medical Products Administration

BEIJING & CAMBRIDGE, Mass.

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 Investigational New Drug (IND) application for CAN103 has been accepted by the Chinese National Medical Products Administration (NMPA). CAN103 is an enzyme replacement therapy (ERT) being developed by CANbridge as part of its rare disease partnership with WuXi Biologics (2269.HK) for the long-term treatment of adults and children with Gaucher disease, Types I and III.

“Twenty years after the pioneering introduction of the first Gaucher disease treatment in China, over 90% of Chinese patients still do not have access to it, underscoring the need for a total solution,” said James Xue, Ph.D. Founder, Chairman and CEO of CANbridge Pharmaceuticals Inc., who also serves as the Deputy Director General of the China Alliance for Rare Disease (CHARD). “To that end, we collaborated with CHARD on precedent-setting pre-IND meetings with all stakeholders, including regulatory authorities and patient representatives, which paved the way to this CAN103 submission. CANbridge is committed to developing a sustainable solution to addressing Gaucher in China, where patients are significantly underserved, and potentially worldwide.”

“The local development and production of drugs for rare diseases is good news for patients with rare diseases and a major move toward perfecting a rare disease ecosystem in our country,” said Li Linkang, Executive Director General of the China Alliance for Rare Diseases (CHARD). “Society is expressing care and concern for patients with rare diseases today, which coupled with the progress made on various fronts in recent years, demonstrates that the government takes patients with rare diseases very seriously. For rare disease patients, the availability of targeted treatment is just the beginning. Providing a rare disease solution will eventually require a complete ecosystem, including research and development, evaluation and approval of new treatments, implementation of guidelines for diagnosis and treatment, as well as creating systems for drug accessibility and payment. I believe that bringing this ecosystem together as early as possible will inspire and incentivize even more local innovations in rare diseases therapies.”

About Gaucher disease (GD)

Gaucher disease is one of the most common lysosomal storage disorders (LSDs). It is a rare hereditary genetic metabolic disease caused by a recessive mutation in the GBA gene, located on chromosome 1, and affects both males and females. It causes a deficiency of glucocerebrosidase, an enzyme that helps break down the muscle cell membrane component, glucocerebroside. As a result, glucocerebroside accumulates in cells and certain organs, a process which is characterized by splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and neurological symptoms. For nearly 30 years, glucocosylase ERT has been the standard treatment for Gaucher disease, with clinical trial and real-world data demonstrating significant improvement in the major non-neurological symptoms and quality of life. There were 3,000 patients with Gaucher disease in China in 2020, according to Frost & Sullivan.

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.

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