Faith Medicine AAV gene therapy completed the phase III clinical enrollment of hemophilia B.

Recently, Faith Pharmaceutical Group announced that the registered clinical study (CTR20212816) Phase III trial of BBM-H901 injection independently developed by its wholly-owned subsidiary Shanghai Xinzhi Pharmaceutical Technology Co., LTD has successfully completed the administration of all subjects.

BBM-H901 injection is suitable for preventing bleeding in adult patients with hemophilia B, and is the first adeno-associated virus (AAV) gene therapy drug for hemophilia B approved to enter registered clinical trials in China.

It is reported that this study is a multicenter, single-arm, open, single-dose registration clinical study to evaluate the safety and efficacy of a single intravenous infusion of BBM-H901 injection in hemophiliac B patients ≥18 years of age with endogenous coagulation factor Ⅸ (FⅨ) activity ≤2IU/dL (i.e., ≤2%). So far, the dose-exploration and dose-escalation phase studies have been completed in China, and encouraging data have been obtained in terms of efficacy and safety: 100% of the subjects stopped treatment with the FⅨ product, the annual bleeding rate (ABR) showed 0, the level of coagulation factor in vivo significantly increased and long-term stable expression; Safety was good with no serious adverse events reported.

About hemophilia

Hemophilia, as a hereditary bleeding disease, is mainly caused by gene mutation of coagulation factor Ⅷ or Ⅸ. Spontaneous bleeding may be caused by A significant decrease in the activity of Factor VIII (Hemophilia A) or Factor IX (Hemophilia B) in the patient, and repeated bleeding of joints and muscles can lead to permanent disability. Today, prophylactic and on-demand use of clotting factor replacement therapy remains the standard clinical treatment for hemophilia. Patients need repeated injections of plasma-derived products or recombinant clotting factors throughout their lives to maintain clotting function. Therefore, the development of drugs that can cure hemophilia is the goal of scientists all over the world, and gene therapy has become a cutting-edge new technology for the treatment of hemophilia.

About BBM-H901 injection

BBM-H901 injection is Adeno-associated virus (AAV) gene therapy drug with independent intellectual property rights of Faith Medicine. It introduced human FactorⅨ (FⅨ) gene into hemophilia B patients by intravenous administration. Thus, the level of coagulation factors in patients can be improved and maintained for a long time, in order to achieve the "one-time administration, long-term effective" treatment and bleeding prevention effect. The design of BBM-H901 injection drug adopts the liver targeting serotype and high efficiency gene expression box with fully independent intellectual property rights, and adopts the serum-free suspension culture and chromatography process independently developed by the company to produce the drug in line with the requirements of GMP.

BBM-H901 injection is one of the earliest AAV gene therapy drugs to be clinically studied in China, and investigator-initiated clinical studies have been conducted since 2019 (IIT,NCT04135300). In April 2021, Shanghai Xinzhi Medical Technology Co., LTD., a wholly-owned subsidiary of Faith Pharma, formally submitted an application (IND) for clinical trials of BBM-H901 injection to the National Medical Products Administration (NMPA). On May 14, 2021, the Center for Drug Evaluation (CDE) officially accepted and officially approved BBM-H901 injection into the clinical trial stage (CTR20212816) on August 6, 2021.

In 2022, the results of clinical studies related to BBM-H901 injection were published successively in the international authoritative journals The Lancet Hematology and the New England Journal of Medicine. In the same year, the drug also received orphan drug designation from the US Food and Drug Administration and was included in the list of breakthrough treatment varieties by the Drug Review Center of the State Drug Administration.