(Shanghai, China, October 17, 2023) – Belief BioMed Inc. announced today that it has signed an exclusive cooperation agreement with Takeda China. According to the agreement, Belief BioMed will grant Takeda China the exclusive license for the commercialization of its investigational product, BBM-H901, in mainland China, Hong Kong China, and Macau China. The collaboration underscores the shared commitment of both sides to leverage their respective strengths and resources in their respective fields, to accelerate the commercialization process of the product, help hemophilia B patients access innovative treatment option, and jointly promote the development of the gene therapy industry.

Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, “Belief BioMed focuses on the cutting-edge gene therapy field, with our product pipeline covering a wide range of rare and common diseases, striving to fill a large number of unmet clinical needs. BBM-H901 is one of the blockbuster products independently developed and produced by Belief BioMed, and it is the first adeno-associated virus (AAV)-based therapy under registrational clinical trials for the treatment of hemophilia B in China. We are very pleased to have announced this collaboration with Takeda China during the 2023 INTERNATIONAL BIOPHARMA INDUSTRY WEEK, and we believe that combining Belief BioMed’s solid clinical development capabilities and Takeda China’s strong commercialization experiences, together we will accelerate the accessibility of this innovative drug and work together to bring more innovative treatment solutions to the patients in China.”

Dr. Xiao Xiao

Co-founder, Chairman and Chief Science Officer, Belief BioMed

Hemophilia B is a genetic disorder caused by the deficiency of factor IX (FIX), and it is marked by repeated, spontaneous bleeding episodes within joints, muscles, and soft tissues, often enduring for prolonged periods. Left unmanaged, it can culminate in joint deformity, muscle atrophy, and, in severe cases, fatal outcomes. In China, there are an estimated 3,800 registered cases of patients with hemophilia B¹. Presently, patients with hemophilia B in China face multiple challenges, including limited treatment options and a lack of standardized diagnostic and therapeutic procedures. Most patients rely on prothrombin complex concentrate (PCC) as a replacement therapy, despite the risk of exposure to infection, together with inconvenient administration. Furthermore, due to low adoption rate and insufficient dosage of treatment, joint bleeds and joint deformity are commonly observed in patients with moderate-to-severe hemophilia B. Consequently, there is a pressing unmet need for medical care among these patients.

BBM-H901 is a gene therapy, independently developed and produced by Belief BioMed. It is administered intravenously to introduce the human factor Ⅸ (FⅨ) gene into the body of hemophilia B patient, thereby improving and maintaining the levels of coagulation factors in the patient's body for a long time, and in order to achieve the prevention of bleeding. It is also the first AVV-based therapy under registrational clinical trials for the treatment of hemophilia B in China. At present, the dosing to subjects in Phase III registration clinical trial has been successfully completed². In 2022, the results of clinical studies related to BBM-H901 were successively published in two prestigious international journals, i.e. The Lancet Haematology and New England Journal of Medicine. In the same year, BBM-H901 was granted Breakthrough Therapy Designation (BTD) by China Center for Drug Evaluation (CDE), NMPA.

The clinical study published in The Lancet Hematology (NCT04135300) is an investigator-initiated, single-center, single-arm clinical trial designed to evaluate the safety and efficacy of a single intravenous infusion of BBM-H901. According to their findings, patients with hemophilia B over 18 years of age with FIX residual levels ≤2IU/ dl who received BBM-H901 injection at 58 weeks of follow-up showed: The median annual bleeding rate was reduced from 12 to 0 (p=0.0092), the median number of target joints was reduced from 1.5 to 0 (p=0.0031), and the median annual infusions of factor IX were reduced from 53.5 to 0 (p<0.0001). There were no serious adverse events during follow-up, and no grade 3-4 adverse events. Grade 1-2 adverse events associated with BBM-H901 included fever (1 case [10%]) and elevated aminotransferase (1 case [10%])³.

The collaboration with Belief BioMed is an important milestone that will further enrich our existing product portfolios. “By combining the R&D technology of Belief BioMed with Takeda China's extensive experience and expertise in commercialization, we can accelerate the pace of bringing breakthrough gene therapies to more patients”, Sean Shan, Senior Vice President of Takeda Pharmaceutical Company and President of Takeda, said. “The local advanced gene therapy technology as that developed by Belief BioMed is emerging rapidly day by day, and we are convinced that China's pharmaceutical innovation will fast integrate into the world in the future and become the innovation source of global biomedical technology.”

Mr. Sean Shan

Senior Vice President of Takeda Pharmaceutical Company and President of Takeda

Belief BioMed is a high-tech company that is engaged in the research and development, production and clinical application of gene therapy products for the treatment of a wide range of diseases with unmet clinical needs such as hemophilia, Parkinson's disease, arthritis and neuromuscular disease. The collaboration, which is built on the mutual trust and dedication of the two sides, will greatly accelerate the commercialization of the gene therapies for hemophilia developed by Belief BioMed in China, brighten the life of more patients, and contribute to the vigorous development of China's gene therapy industry.

Reference

[1] Demographics, clinical profile and treatment landscape of patients with hemophilia B in China [J]. Haemophilia, 2022 Mar, 28(2): e56-e60

[2] https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c

[3] Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial [J]. The Lancet, 2022 May, 9(7): E504-E513

Statement

1.This information is only for the purpose of introducing the company's business activities, and is not intended to promote any company's products and/or services, nor should it be construed as providing any advice or recommendation on the selection of any drugs, medical devices and treatment options.

2.For information about any company products, diseases and/or treatments, please consult a healthcare professional.

3.BBM-H901 described herein has not been approved for marketing in China.

About Belief BioMed

Belief BioMed Inc. (BBM) is a high-tech company that integrates the development, manufacturing and clinical application of gene therapy products. The company is committed to providing innovative and more effective gene therapies for monogenic diseases, neurodegenerative diseases, age-related degenerative diseases and some other major malignant diseases through safe and efficient virus vector technology. BBM has developed hundreds of key vector technologies, including HEK293 cell suspension serum-free culture process and full-scale chromatography purification process, and has established a commercial production platform for gene therapy drugs in China. The company has been building up its capabilities in a variety of fields including novel AAV capsids targeting different tissues, efficient transgene expression cassette design, and advanced clinically applicable vector manufacturing process. It has also established an extensive R&D pipeline covering a wide range of major, unmet clinical needs in therapeutic areas such as hemophilia, Parkinson's disease, arthritis, neuromuscular disease, etc. Several product pipelines have entered clinical studies or IND filings. For more information, visit www.beliefbiomed.com.

About Takeda

Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare disease, plasma-derived therapies, neuroscience, oncology and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com.

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