Betagene and Takeda China Announce Exclusive Commercialization Partnership for Hemophilia B Gene Therapy in Greater China

Will be in ChinaPatients Bring New Choices for Gene Therapy

ShanghaiOctober 17, 2023PR Newswire -- Belief BioMed (hereinafter referred to as "Belief BioMed") and Takeda China today announced an exclusive cooperation agreement, under which Belief BioMed has authorized Takeda China to be responsible for the commercial operation of its investigational product BBM-H901 injection in mainland China, Hong Kong, and Macao. The two parties will combine their respective strengths and resources to accelerate the commercialization process of this product, help patients with hemophilia B gain access to innovative treatment options, and jointly promote the industrial development of gene therapy.

Belief BioMed and Takeda China Exclusive Commercialization Cooperation Signing Ceremony

Dr. Xiao Xiao, Co-founder, Chairman, and Chief Scientist of Belief BioMed, stated: "Belief BioMed is dedicated to advancing cutting-edge gene therapy, with a broad product pipeline covering both rare and common diseases, aiming to address significant unmet clinical needs. BBM-H901 injection is one of the key products independently developed and produced by Belief BioMed and represents the first adeno-associated virus (AAV) gene therapy drug for Hemophilia B to enter a registrational clinical trial in China. We are thrilled to announce this strategic cooperation with Takeda China during the 2023 Shanghai International Biopharmaceutical Industry Week. With Belief BioMed's robust clinical development capabilities and Takeda China's strong commercialization abilities, we believe this collaboration will further solidify both parties' pipeline strategies and market presence. Together, through mutual trust and cooperation, we will bring more innovative treatment solutions to patients across China."

Hemophilia B is a hereditary coagulation disorder caused by a deficiency of clotting factor IX (FIX) in the patient's blood. It is characterized by recurrent, spontaneous bleeding in joints, muscles, and soft tissues, which, if prolonged and repeated, can lead to joint deformities, muscle atrophy, and even death. In China, approximately 3,800 patients with Hemophilia B are registered.^[1]Currently, patients with Hemophilia B in China still face challenges such as limited treatment options and non-standardized diagnosis and treatment. Patients mostly use Prothrombin Complex Concentrate (PCC) for replacement therapy, which carries risks of infection and inconvenient administration. Meanwhile, due to insufficient or non-standardized treatment, joint bleeding and deformities are common among moderate to severe patients, indicating that their medical needs remain unmet.

BBM-H901 Injection, independently developed and produced by Belief BioMed, delivers the human coagulation factor IX gene into patients with hemophilia B through intravenous administration to achieve sustained expression, enhancing and maintaining the patients' coagulation factor levels over the long term. It is used for preventing bleeding in adult patients with hemophilia B. This product is China's first AAV gene therapy drug for hemophilia B to be approved for registration clinical trials and has successfully completed dosing of participants in the Phase III registration clinical trial.^[2]In 2022, the clinical research results related to BBM-H901 injection were successively published in the prestigious international journals *The Lancet Haematology* and *The New England Journal of Medicine*. In the same year, it was included in the "Breakthrough Therapy Designation" list by the Center for Drug Evaluation of the National Medical Products Administration.

The clinical study (NCT04135300) published in The Lancet Haematology was 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 the study results, adult patients (aged 18 years or older) with Hemophilia B and FIX residual levels ≤2 IU/dl who received BBM-H901 injection showed the following outcomes during a 58-week follow-up: the median annualized bleeding rate decreased from 12 to 0 (p=0.0092), the median number of target joints reduced from 1.5 to 0 (p=0.0031), and the median annual infusion frequency of coagulation factor IX drugs decreased from 53.5 to 0 (p<0.0001). No serious adverse events occurred during follow-up, and no grade 3-4 adverse events were reported. BBM-H901-related grade 1-2 adverse events included fever (1 case [10%]) and elevated transaminase levels (1 case [10%]).^[3]。

"Collaborating with Belief BioMed marks a significant milestone, further enriching our existing portfolio of products and therapies. We look forward to combining Belief BioMed's R&D technology with Takeda China’s extensive experience and expertise in commercialization, which will accelerate the availability of breakthrough gene therapies for more patients," said Shan Guohong, Global Senior Vice President of Takeda and President of Takeda China. "The rise of local gene therapy technologies represented by Belief BioMed is becoming increasingly prominent. We firmly believe that China's pharmaceutical innovation will also accelerate its integration into the global landscape, becoming an innovative source of global biopharmaceutical technology."

Belief BioMed is a high-tech enterprise that integrates the research and development, production, and clinical application of gene therapy products, with a treatment scope widely covering various major diseases with unmet clinical needs such as hemophilia, Parkinson's disease, arthritis, and neuromuscular disorders. This collaboration, built on the foundation of mutual trust and cooperation between the two parties, will greatly accelerate Belief BioMed’s commercialization of gene therapies in the field of hemophilia treatment in China, bringing the light of life to more patients and contributing strength to the vigorous development of China’s gene therapy industry.

Annotation Description:

[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 material is only for introducing the company's business and operational activities, and is not intended to promote any company products and/or services. It should not be construed as providing any opinions or suggestions regarding the selection of any drugs, medical devices, or treatment options.

2. For any information related to company products, diseases, and/or diagnosis and treatment, please be sure to consult a healthcare professional.

3. The BBM-H901 injection described in this article has not yet been approved for marketing in China.

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