Shanghai, China, 24^th April, 2023 —— Belief BioMed Group (BBM), an industry-leading biotech company focusing on innovative gene therapies, announced today the completion of patient dosing in its Phase III Registrational Clinical Trial (CTR20212816) of BBM-H901, a gene therapy for Hemophilia B developed and manufactured in-house by Shanghai Belief-Delivery Biomed Co., Ltd., its wholly owned subsidiary. 

BBM-H901 is indicated for prophylactic treatment of bleeding in adults with hemophilia B. It is the first adeno-associated virus (AAV) gene therapy for Hemophilia B with IND approval for registrational clinical study in China.

This registrational study is a multi-center, single-arm, open and single-dose clinical study. Its objective is to evaluate the safety and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B patients ≥18 years of age with endogenous blood coagulation factor Ⅸ (FⅨ) activity ≤2 IU/dL (≤2%). As of today, its dose exploration and dose escalation studies have been completed in China, and positive results have been obtained in terms of efficacy and safety: 100% patients stopped FⅨ treatment, and the annual bleeding rate (ABR) was reduced to zero; the level of coagulation factors was significantly increased and persisted in all patients; safety profile was good with no serious adverse event reported.   

Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, "We are pleased to see patient dosing has been successfully completed in the phase III registrational trial of BBM-H901, the first blockbuster product independently developed and manufactured by BBM with the potential to become the first AAV gene therapy approved for marketing in China for prophylactic treatment of bleeding in adults with hemophilia B. Featuring ‘one-time administration and long-term effectiveness’, this innovative therapy will bring better survival benefits to patients. I want to express my heartfelt thanks to all investigators, patients and their families as well as the project team for their contribution to the trials. I believe it is a milestone that will lay a solid foundation for the future clinical application of more of our gene therapies and help promote the vigorous development of the entire gene therapy industry. Being fully committed to addressing unmet medical needs, BBM will continue to tap into the potential of gene therapy, and strive to bring more first-in-class and best-in-class products to patients!”

About Hemophilia

Hemophilia, an inherited bleeding disorder, is mainly caused by mutations in the coagulation factor Ⅷ or Ⅸ genes. Spontaneous bleeding may occur due to a significant reduction in coagulation factor Ⅷ activity (hemophilia A) or factor Ⅸ activity (hemophilia B) in patients. Repeated bleeding in joints and muscles may cause lifelong disability. Nowadays, prophylaxis and on-demand therapy with coagulation factors remain the standard of care for hemophilia. Patients need repeated injections of plasma-derived or recombinant coagulation factors throughout their life to maintain their coagulation function. As a result, the development of drugs that can cure hemophilia is a goal that scientists relentlessly pursue worldwide, and gene therapy has become a cutting-edge technology to cure hemophilia.

About BBM-H901

BBM-H901 is an adeno-associated virus (AAV) gene therapy with independent intellectual property rights owned 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, in order to achieve "one-time administration, long-term effectiveness" and prevention of bleeding. BBM-H901 utilizes liver-tropic AAV serotypes and full gene expression cassette exclusively owned by Belief BioMed, and the company uses self-developed serum free suspension culture and chromatographic process to produce drugs that meet the requirements of Good Manufacturing Practice (GMP).

BBM-H901 is one of the earliest AAV gene therapies to be clinically studied in China, with Investigator Initiated Trial (IIT) NCT04135300 started in 2019. In April 2021, Shanghai Belief-Delivery Biomed Co., Ltd., a wholly-owned subsidiary of Belief BioMed, submitted the IND application for BBM-H901 to China National Drug Administration (NMPA). The IND application was officially accepted by the Center of Drug Evaluation (CDE) of NMPA on May 14, 2021. On August 6, 2021, BBM-H901 obtained approval to enter the clinical trial phase (CTR20212816).

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 Orphan Drug Designation (ODD) by U.S. Food and Drug Administration (FDA), and Breakthrough Therapy Designation (BTD) by China Center for Drug Evaluation (CDE), NMPA.

About BBM

Belief BioMed (BBM) is an industry-leading, 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 genetic disorders, neurodegenerative diseases, age-related degenerative diseases and some major malignant diseases through safe and efficient adeno-associated virus (AAV) vector technology. The company is headquartered in Shanghai, China, with offices or R&D and manufacturing centers in Beijing, Suzhou, Hong Kong and North Carolina, USA.

BBM has developed hundreds of key AAV vector technologies and owns dozens of world-leading patented technologies, including the world-leading HEK293 cell suspension serum-free culture process and full-scale chromatography purification process, and has established the first commercial production platform for clinical used AAV 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, advanced clinically-used vector manufacturing process, and innovative clinical development pathways. 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, lysosomal storage disorders, hereditary neuromuscular diseases, oncology, ophthalmology, and degenerative diseases of the elderly. Several product pipelines have entered clinical studies or IND filings, and clinical data have been collected showing that the drug candidates have good safety and efficacy profiles.

For more information about BBM, please visit: www.beliefbiomed.com.

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