Shanghai, China, October 25, 2024 — Belief BioMed Inc. (BBM), a globally innovative biotech company focusing on cutting-edge gene therapies, announced today that the European Medicines Agency (EMA) has granted its gene therapy drug BBM-H901 the Advanced Therapy Medicinal Product (ATMP) designation for the treatment of hemophilia B.

BBM-H901, independently developed and manufactured by Belief BioMed, is a recombinant adeno-associated virus (rAAV) gene therapy vector carrying an optimized human Factor IX (FIX) gene expression cassette. This EMA designation, from one of the leading global regulatory authorities, validates our breakthrough research, advances our global expansion plans, and strengthens our position in the European market.

In accordance with EU regulations, the EMA typically designates Advanced Therapy Medicinal Products (ATMPs) to therapies based on genes, tissues, or somatic-cells that are anticipated to provide transformative treatment options for human diseases or injuries. ATMP developers can receive a range of regulatory guidelines for pre-clinical research, clinical development and manufacturing, as well as incentives, such as fee reduction for scientific advice.

As one of Belief BioMed’s core products, BBM-H901 has received a lot of recognition for its clinical potential and academic significance: It has received a Breakthrough Therapy Designation (BTD) and was granted Priority Review under the New Drug Application (NDA) by the National Medical Products Administration (NMPA) of China; it has also obtained the Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA); Results from Investigator Initiated Trial (IIT) related to BBM-H901 have been published in prestigious international journals such as The Lancet Haematology, The New England Journal of Medicine, and presented at the International Society on Thrombosis and Haemostasis (ISTH) 2024 Congress.

Reference:

1.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.

2.Xue F et al. Total Knee Arthroplasty after Gene Therapy for Hemophilia B.N EnglJ Med.2022 Oct 27;387(17):1622-1624

3.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

About Hemophilia B

Hemophilia B is a genetic disorder caused by the deficiency of factor IX (FIX), and it is marked by repeated, spontaneous bleeding episodes within joints, muscles, and soft tissues, often enduring for prolonged periods. Left unmanaged, it can culminate in joint deformity, muscle atrophy, and, in severe cases, fatal outcomes. Presently, patients with hemophilia B in the world face multiple challenges, including limited treatment options and a lack of standardized diagnostic and therapeutic procedures, and there is a pressing need for more innovative treatment solutions.

About BBM-H901

BBM-H901 is independently developed and manufactured by Belief BioMed. It is administered intravenously to introduce the human factor Ⅸ gene into the body of a 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 adult patients. 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. In addition, the IIT of BBM-H901 for the treatment of hemophilia B in paediatric patients was officially initiated in 2023 and dosing of all subjects was completed in mid-2024.

About Belief BioMed

Belief BioMed Inc. (BBM) is a global biotech company that integrates the research and 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. 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 unmet clinical needs in different therapeutic areas such as hemophilia, DMD, Parkinson’s disease, osteoarthritis, etc. Several product pipelines have entered clinical studies or submitted IND filings. The NDA of a gene therapy for the treatment of adult patients with hemophilia B, has been accepted by the NMPA of China. For more information, visit www.beliefbiomed.com.

Statement

- 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.

- For information about any company products, diseases and/or treatments, please consult a healthcare professional.

- BBM-H901 described herein has not been approved for marketing.