Recently, Belief Pharmaceutical Group announced that the Phase III trial of the registered clinical study (CTR20212816) of BBM-H901 injection, independently developed by its wholly-owned subsidiary Shanghai Xinzhi Pharmaceutical Technology Co., Ltd., has been successfully completed for all subjects.
BBM-H901 injection is suitable for preventing bleeding in adult patients with hemophilia B. It is the first adeno-associated virus (AAV) gene therapy drug approved for registration in clinical trials for hemophilia B.
It is reported that this study is a multicenter, single arm, open, single dose registered clinical study aimed at evaluating the safety and efficacy of a single intravenous infusion of BBM-H901 injection in hemophilia B patients aged ≥ 18 years and with endogenous coagulation factor IX (F IX) activity ≤ 2IU/dL (i.e. ≤ 2%). As of now, the dose exploration and dose increase phase studies have been completed in China, and encouraging data have been obtained in terms of effectiveness and safety: 100% of the subjects stopped using the F IX product treatment, with an annualized bleeding rate (ABR) of 0, and significantly increased levels of coagulation factors in the body and long-term stable expression; Good safety, no serious adverse events reported.
About hemophilia
Hemophilia, as a hereditary bleeding disease, is mainly caused by mutations in the coagulation factor VIII or IX genes. Spontaneous bleeding may be caused by a significant decrease in the activity of coagulation factor VIII (hemophilia A) or factor IX (hemophilia B) in patients, and repeated bleeding in joints and muscles can lead to lifelong disability. Nowadays, prevention and on-demand use of coagulation factor replacement therapy remain the standard clinical treatment for hemophilia. Patients need to repeatedly inject plasma derived products or recombinant coagulation factors throughout their lives to maintain coagulation function. Therefore, developing drugs that can cure hemophilia is a persistent goal pursued by scientists around the world, and gene therapy has become a cutting-edge new technology for treating hemophilia.
About BBM-H901 Injection
BBM-H901 injection is an adeno-associated virus (AAV) gene therapy drug with independent intellectual property rights owned by Faith Pharmaceutical. The human coagulation factor IX (F IX) gene is introduced into the body of hemophilia B patients through intravenous administration, thereby improving and maintaining the coagulation factor levels in the patient's body for a long time, in order to achieve a "one-time administration, long-term effectiveness" treatment and prevention of bleeding. The design of BBM-H901 injection drug adopts liver targeted serotypes and efficient gene expression boxes with fully independent intellectual property rights, and uses the company's self-developed serum free suspension culture and chromatography process to produce drugs that meet the requirements of Good Manufacturing Practice (GMP).
BBM-H901 injection is one of the earliest AAV gene therapy drugs to be clinically studied in China, and has been a clinical study initiated by researchers since 2019 (IIT, NCT04135300). In April 2021, Shanghai Xinzhi Pharmaceutical Technology Co., Ltd., a wholly-owned subsidiary of Faith Pharmaceutical, officially submitted a clinical trial application (IND) for BBM-H901 injection to the National Drug Administration (NMPA). On May 14, 2021, the Drug Evaluation Center (CDE) officially accepted the application and officially approved BBM-H901 injection to enter the clinical trial phase (CTR20212816) on August 6, 2021.
In 2022, the clinical research results related to BBM-H901 injection were published in the internationally authoritative journals "Lancet Hematology" and "New England Journal of Medicine". In the same year, the drug also received orphan drug recognition from the US Food and Drug Administration and was included in the breakthrough treatment variety list by the National Drug Administration's Drug Evaluation Center.
