SHANGHAI, China I January 3, 2024 I Belief BioMed Group (BBM), an industry-leading biotech company focusing on innovative gene therapies, announced the completion of first subject dosing in its registrational clinical trial (CTR20233400) of BBM-H803, a gene therapy for hemophilia A, independently developed and produced by Belief BioMed.

BBM-H803 is Belief BioMed’s first gene therapy for the treatment of hemophilia A and is the company’s second product which has obtained IND approval from the National Medical Products Administration (NMPA). In December 2022, BBM-H803 was granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). As of today, there have not been adeno-associated virus (AAV)-based gene therapies available in China for the treatment of adults with hemophilia A.

This registrational study is a multi-center, single-arm, open-label and single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of a single-time intravenous administration of BBM-H803 in patients ≥18 years of age with severe hemophilia A. With an in-house developed engineered capsid, the product features relatively lower immunogenicity and higher liver delivery efficiency. The proposed clinical dose is also lower than that of similar AAV-based gene therapy products launched abroad, and the safety is expected to be better. Patient enrollment for the previous exploratory study was completed in August 2023, and from which data generated has been used to support subsequent registrational trials.

Dr. Xiao Xiao, Co-founder, Chairman, and Chief Science Officer of Belief BioMed, said, “As we welcome in the New Year with its sense of hope and new beginnings, it gives us great pleasure to share that today is a special day for both Belief BioMed and hemophilia A patients, as the first patient dosing has been successfully completed in the registrational trial of BBM-H803. The achievement of this milestone means that Belief BioMed has made another major breakthrough in the field of gene therapy in hematological therapeutical area, and it once again confirms the company’s strong and robust research and development capabilities. We will continue to pursue our original goals, working together – both internally and externally – to comprehensively and efficiently conduct this clinical study, all while striving to bring this innovative gene therapy to the vast number of Chinese patients that need it as soon as possible.”

About Hemophilia A

Hemophilia A is a genetic disorder caused by the deficiency of factor Ⅷ  (FⅧ), and it is characterized by repeated, spontaneous bleeding episodes within joints, muscles, and soft tissues. Repeated joints bleeding can culminate in joint deformity, and disabled outcomes in severe cases. In China, there are an estimated 14,000 registered cases of patients with hemophilia A. Currently, most patients rely on factor Ⅷ as a replacement therapy, despite the risk of exposure to infection, together with inconvenient administration. Furthermore, patients with hemophilia A face multiple challenges, including limited treatment options and a lack of standardized diagnostic and therapeutic procedures. Consequently, there is a pressing unmet need for medical care among these patients.

About BBM-H803

BBM-H803 is an AAV-based gene therapy with independent intellectual property rights owned by Belief BioMed. It is administered intravenously to deliver the coagulation factor Ⅷ gene into the body of patients with hemophilia A, thereby improving and maintaining the coagulation factor level in the patient’s body for a long time, for the prevention of bleeding. BBM-H803 uses liver-specific capsid and highly efficient gene expression cassette, and the drug production uses the company’s independently developed serum-free suspension culture process, which meets the requirements of the Good Manufacturing Practice for Pharmaceuticals.

Since 2022, the Investigator Initiated Trial (IIT, NCT05454774) of BBM-H803 has been on-going, and in December of the same year, BBM-H803 was granted Orphan Drug Designation (ODD) by the U.S. FDA. In May 2023, Belief BioMed submitted IND application to China NMPA, and received IND approval in July 2023.

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 age-related diseases, cancers, monogenic 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 the 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.

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SOURCE: Belief BioMed