Shanghai, China, July 24th, 2024 —— Belief BioMed (BBM), an innovative Chinese biotechnology company focused on developing cutting-edge gene therapies, announced today that the Medical Products Administration (NMPA) of China has accepted the company’s new drug application (NDA) for BBM-H901 for treatment of adults with hemophilia B.

BBM-H901 is the first core product independently developed and manufactured by BBM, it's also the first gene therapy product indicated for inborn genetic diseases for which an NDA has been submitted in China. By achieving this milestone, BBM-H901 is expected to become the first approved recombinant AAV-based gene therapy product for hemophilia B in China and to reshape the landscape of AAV-based gene therapy worldwide.

Professor Lei Zhang from the Hematology Hospital of the Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences) and the Leading PI of BBM-H901 registrational study said,

“There are pressing unmet medical needs for hemophilia B patients in China. Currently, most patients are treated with prothrombin complex concentrate (PCC) as a replacement therapy, this treatment has infection risk and at the same time, it’s inconvenient for the patients. Furthermore, due to insufficient or non-standardized treatment, patients with moderate to severe hemophilia B often experience joint bleeding and deformities. With the latest BBM-H901 clinical data showing both good safety and efficacy, we look forward to bringing BBM-H901 to market and the clinical benefits it will bring to patients.”

Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said,

“We are very delighted to share the good news that the new drug application for BBM-H901 has been accepted by China NMPA. Looking back on the journey, we’ve experienced a lot of challenges, behind this milestone is the professionalism and dedication of all BBM employees day after day, as well as the trust and support from investigators, trial participants and their families, governments at all levels, our partners and friends. On behalf of the company, I would like to express our sincerest gratitude to all of you. With even more opportunities to expect, BBM will continue to work at the forefront of gene therapy, striving to bring more first-in-class and best-in-class therapies to patients.”

This registrational study (CTR20212816) is a multi-center, single-arm, open label and single-dose clinical study designed 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, all subjects participating in the confirmatory phase III study have completed the 52-week follow-up visits after treatment, desirable safety and efficacy outcome was achieved.

About BBM-H901

BBM-H901 was independently developed and manufactured by Belief BioMed. It is administered intravenously to introduce the human factor Ⅸ 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, for the prevention of bleeding. In 2022, BBM-H901 was granted Breakthrough Therapy Designation (BTD) by China Center for Drug Evaluation (CDE), NMPA. In 2024, It became the first AAV-based gene therapy, for which the New Drug Application was submitted in China. The results of Investigator Initiated Trial (IIT) related to BBM-H901 were successively published in two prestigious international journals, i.e. The Lancet Haematology and New England Journal of Medicine, and were also announced in an oral presentation at the ISTH 2024 Congress.

About Belief BioMed

Belief BioMed Inc. (BBM) is a biotech company that integrates the research & development, manufacturing and clinical application of gene therapy products. The company is committed to providing innovative and more effective gene therapies for severe genetic and chronic diseases through safe and efficient viral 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 clinical-grade vector manufacturing process. It has also established an extensive R&D pipeline covering a wide range of unmet medical needs in therapeutic areas such as hemophilia. Several product pipelines have entered clinical studies or submitted IND filings. For more information, visit www.beliefbiomed.com.

Statement

1.This information is only for the purpose of introducing the company's event and information on that date, 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.