Phase III Clinical Trial Results of BBM-H901 Gene Therapy for Adult Hemophilia B Presented at ASH 2024
Takeda
Biopharmaceutical Manufacturer
Shanghai, China, December 9, 2024 —— Belief BioMed (hereinafter referred to as Belief BioMed) and Takeda China jointly announced today:The Phase III clinical trial results of BBM-H901 Injection, a gene therapy drug for treating adult Hemophilia B, have been successfully selected for the 66th American Society of Hematology (ASH) Annual Meeting and will be presented in the form of an academic poster.The annual meeting is being grandly held in San Diego, USA, in a combination of online and offline formats.
The study results showed that, compared with prophylactic FIX replacement therapy, BBM-H901 injection significantly reduced the annualized bleeding rate (ABR) in patients, rapidly increased the FIX activity level in patients and maintained stable expression. Meanwhile, the research data also showed that this drug has good safety and tolerability.
As of April 21, 2023, 26 subjects have been successfully enrolled and completed a single intravenous infusion treatment of BBM-H901 injection (5×10¹² vg/kg). The currently completed 52-week follow-up data is as follows:
The mean annualized bleeding rate (ABR) for subjects was 0.6 (95% CI: 0.18-1.99), with the upper limit of the 95% confidence interval (95% CI) for the mean ABR being 1.99, which is lower than the superiority margin of 5.0 (assumed ABR for prophylactic treatment in China, set at 5.0).
At Week 52 post-infusion, the mean FIX activity reached 55.08 IU/dL (SD 35.93) (one-stage SynthASil method); by Day 3 post-infusion, the mean FIX activity had already increased to 49.70 IU/dL (one-stage SynthASil method).
The average number of infusions for the 52-week FIX medication decreased from 58.2 (SD 30.67) before treatment to 2.9 (SD 10.71) after treatment. Twenty-one of the 26 subjects (80.8%) experienced no bleeding events after treatment. The average number of target joints decreased from 1.1 (SD 1.2) before treatment to 0 after treatment.
No serious adverse events, no FIX inhibitor-positive adverse events, no thromboembolic events, no grade 3-4 adverse events
The 1-2 grade adverse events related to BBM-H901 injection included: increased alanine aminotransferase (7 cases [26.9%]), increased aspartate aminotransferase (2 cases [7.7%]), and decreased fibrinogen (3 cases [11.5%]).
The investigator-initiated clinical study (IIT) of BBM-H901 injection was launched as early as 2019, and its research results have been successively published in the international authoritative journal The Lancet Haematology.2and The New England Journal of Medicine3, and was presented in an oral report at the 2024 International Society on Thrombosis and Haemostasis (ISTH) Congress.4The New Drug Application (NDA) for this drug was officially accepted by the National Medical Products Administration (NMPA) of China in July 2024 and has been included in the priority review and approval process. Belief BioMed has reached an exclusive commercialization cooperation agreement with Takeda China in the field of Hemophilia B, and Takeda China will be responsible for the commercial promotion of this product in mainland China, Hong Kong, and Macao.
Notes
1 Feng X，et al. 2024 ASH. Poster 3582
2 Xue F et al. 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. Lancet Haematol. 2022;9(7): e504-e513.
3 Xue F et al. Total Knee Arthroplasty after Gene Therapy for Hemophilia B.N EnglJ Med.2022 Oct 27;387(17):1622-1624
4 Xue F et al. More than 3 years follow-up after gene transfer with BBM-H901 in adults with Hemophilia B. 2024 ISTH.OC 30.4
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Statement
- This article is only related to the events and materials stated on the day of publication. It is not intended to promote any company products and/or services, nor should it be construed as providing any opinions or recommendations regarding the selection of any drugs or treatment options.
- For any information related to company products, diseases, and/or diagnosis and treatment, please be sure to consult a healthcare professional.
- The BBM-H901 injection described in this article is an investigational drug and has not yet been approved for marketing.
About Hemophilia B
Hemophilia B is a hereditary coagulation disorder caused by a deficiency of coagulation factor IX (FIX) in the patient's blood. Its clinical manifestations include recurrent, spontaneous bleeding in joints, muscles, and soft tissues. Prolonged and repeated bleeding can also lead to joint deformities, muscle atrophy, and even death. Overall, hemophilia patients worldwide still face challenges such as limited treatment options and non-standardized diagnosis and treatment. There is an urgent clinical need for more innovative treatment solutions.
About BBM-H901 Injection
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. It aims to elevate and maintain the coagulation factor levels in patients over the long term, preventing bleeding in adult patients with hemophilia B. This product is China's first gene therapy drug for genetic disorders to submit a New Drug Application (NDA). It has been designated as a Breakthrough Therapy Drug (BTD) and granted priority review status for new drug applications by the National Medical Products Administration (NMPA) of China. Additionally, it has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA), as well as Advanced Therapy Medicinal Product (ATMP) certification from the European Medicines Agency (EMA).
About Belief BioMed
Belief BioMed Inc. is a global high-tech enterprise integrating the research and development, production, and clinical application of gene therapy products. The company is committed to providing more effective and innovative gene therapies for severe genetic disorders and chronic diseases through safe and efficient viral vector technologies. It has developed hundreds of key vector technologies, including HEK293 cell suspension serum-free culture processes and fully chromatographic large-scale purification processes, and established a commercial production platform for gene therapy drugs. The company has made comprehensive advancements in areas such as novel AAV capsids targeting different tissues, efficient transgene expression cassette designs, and advanced clinical-grade vector manufacturing processes. Additionally, it has built a robust R&D pipeline covering therapeutic areas with unmet clinical needs, such as hemophilia, Duchenne muscular dystrophy, Parkinson's disease, and osteoarthritis. Multiple product pipelines have entered clinical research or IND submission stages, and the new drug application for its gene therapy targeting hemophilia B has been accepted by the National Medical Products Administration (NMPA) of China. For more information, please visit: www.beliefbiomed.com.
About Takeda
Takeda Pharmaceutical Company Limited (TSE: 4502) (NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical company headquartered in Japan. Takeda is dedicated to translating science into highly innovative medicines to ensure a healthier future for patients. Takeda focuses on developing medicines in four therapeutic areas: oncology, gastroenterology, neuroscience, and rare diseases, with additional specialized investments in plasma-derived therapies and vaccines. Takeda consistently prioritizes the development of highly innovative medicines, exploring new treatment modalities and strengthening its collaborative R&D engine to build a robust and diverse pipeline aimed at improving people’s lives. For more information, please visit https://www.takeda.com.
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