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 it has dosed the first patient in the CAN008 Phase 2 clinical trial to treat glioblastoma multiforme (GBM) in Mainland China.

The multi-center, randomized, double-blind, placebo-controlled Phase 2 trial will compare standard-of-care (tumor removal, followed by radiation therapy plus temozolomide (TMZ)) with placebo, to standard-of-care with CAN008. The trial will investigate efficacy, as well as explore how select biomarkers correlate to outcome, to provide potential benefits to GBM patients.

The compound has been recognized by regulatory bodies in key international markets as potentially promising in the treatment of glioblastoma. It was granted orphan drug designation by the United States Food and Drug Administration (FDA) and received an orphan medicinal product designation by the European Medicines Agency (EMA) for the treatment of glioblastoma multiforme. In addition, it also was accepted into the EMA�s PRIME (Priority Medicines) program, which provides early and enhanced support to medicines that have the potential to address patients' unmet needs. CANbridge completed the Phase 1 clinical trial of CAN008 plus temozolomide (TMZ), during and after radiation therapy, in patients with newly diagnosed glioblastoma multiforme (GBM). The results demonstrate that CAN008 has excellent safety and tolerability in patients with GBM and could potentially improve the quality life and the survival time of the patients

�Glioblastoma multiforme is the most common primary intracranial malignant tumor,� said Professional Wenbin Li, Director of Comprehensive Treatment Ward of Neuro-Oncology, Beijing Tiantan Hospital, Capital Medical University. �Under the current standard treatment, the media survival time of patients is only 14.6 months, and the two-year survival rate is only 27%. The incidence in China has been increasing in recent years. The preliminary clinical trial results of CAN008 show that it has a good treatment effect trend, as well as very good safety and tolerability. We are pleased to participate in the domestic clinical study of CAN008 to provide a potential new treatment option for patients with glioblastoma.�

�CAN008 is our first clinical product approved to commence clinical trials that has received a Class 1 new drug designation by China Food�s National Medical Products Administration,� said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals, Inc. �The dosing of the first patient in CAN008 Phase 2 trial for the treatment of GBM in China represents a major milestone for CANbridge. We look forward to advancing this truly novel, fusion protein treatment for glioblastoma along the clinical pathway in China, where it might provide new options for patients.�

Class 1 new drug refers to new chemical entities, demonstrating clinical value, which have never been marketed anywhere in the world, and confers five years of regulatory exclusivity in China.

About Glioblastoma (GBM)

Glioblastoma (GBM) is the most common malignant tumor of the central nervous system, accounting for 35.26% � 60.9% of intracranial tumors. It is classified by the World Health Organization (WHO) as a stage-IV cancer, according to the latest classification standard of intracranial tumors. GBM is highly infiltrative and aggressive and is not surgically curable, which leads to a high recurrence rate. Although the treatment of GBM, through resection radiotherapy and chemotherapy, has continuously improved, prognosis has not been substantially improved. The median survival time is still only 14.6 months.

According to the American Association of Neurological Surgeons AANS Statistics, the number of patients with GBM in western countries is 2-3/100,000 every year, accounting for 52% of intracranial tumors. The two-year and five-year survival rates are about 30% and 4-5%, respectively.

According to Frost & Sullivan, GBM patients in China accounted for 46.6 % of intracranial tumors, totaling 54,700 patients in 2020. With the aging population and aggravated levels of air pollution and ionizing radiation exposure, the number of patients with newly diagnosed glioblastoma in China is expected to reach 59,800 in 2025, and 64,400 in 2030.

About CAN008

CAN008 is a CD95 Fc fusion protein that blocks the interaction between CD95 receptor, and its cognate ligand CD95L, through binding to CD95L. CAN008 has a unique dual mechanism of action, which can not only inhibit the invasive growth and migration of tumor cells, but could also reduce the apoptosis of T cells induced by Caspase, and enhance immune recognition. Previous clinical trial data show that CAN008 has good safety, can effectively improve the quality-of-life of GBM patients, and can greatly prolong the survival time of patients. Based on this, European Medicines Agency (EMA) included CAN008 in the Priority Medicines (PRIME) program.

Note: The European Medicines Agency (EMA) officially launched the �PRIority MEdicines (PRIME)� plan on March 7, 2016, to accelerate the review process of drugs that could address medically underserved conditions.

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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Media:

Deanne Eagle

Planet Communications

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