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Release time:2023/4/11 9:03:00

The BBM-H901 injection independently developed by Shanghai Xinzhi Pharmaceutical Technology Co., Ltd. (an AAV gene therapy drug suitable for preventing bleeding in adult male patients with hemophilia B) has been approved for clinical trials by the National Medical Products Administration (NMPA) and will officially start registered clinical trials. This is the first domestically approved AAV gene therapy drug for hemophilia to enter registered clinical trials, and also the first domestically administered rare disease gene therapy.

Zheng Jing, CEO of Belief Pharmaceuticals, said, "If small molecule drugs and antibody drugs are referred to as the first two revolutions of biopharmaceuticals, gene therapy is seen as leading the third industrial revolution of biopharmaceuticals. After years of technological accumulation, the market size of the gene therapy industry has grown rapidly, and large pharmaceutical companies such as Pfizer, Novartis, and Johnson&Johnson have successively laid out their positions, Relying on a strong research and development team and core key technologies in the field of biotechnology, Belief Pharmaceuticals is a global field of gene therapy new drugs, and one of the few new startups that rivals the technology of multinational corporations in Europe and America

BBM-H901 injection is an AAV gene therapy drug with independent intellectual property rights owned by Faith Pharmaceuticals. The human factor IX (FIX) gene is introduced into the body of hemophilia B patients through intravenous administration, thereby improving and maintaining the level of coagulation factors 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 GMP requirements. BBM-H901 injection has the characteristics of good expression effect, high yield, and strong safety.

The gene therapy of BBM-H901 injection is one of the earliest AAV gene therapies to conduct clinical trials in China. Since 2019, researchers have initiated clinical studies (InvestigatorInitiated Trial, IIT, NCT04135300). Clinical research data shows that BBM-H901 injection has good safety and efficacy. After AAV gene therapy drug infusion, the levels of coagulation factors in patients' bodies significantly increase and remain stable for a long time. No significant adverse reactions were found during the clinical period, and the annualized bleeding rate (ABR) of patients was significantly reduced. In April 2021, Belief Pharmaceuticals officially submitted its Clinical Trial Application (IND) for BBM-H901 Injection to the National Drug Administration (NMPA). On May 14, 2021, the Center for Drug Evaluation (CDE) officially accepted and approved BBM-H901 injection for clinical trial on August 6, 2021.

In addition, according to CDE's recent announcement (August 9, 2021), Xinzhi Pharmaceutical Technology's BBM-H901 injection has been approved for clinical use in type B hemophilia.

Type B hemophilia is a type of hemophilia caused by a lack of coagulation factor IX, accounting for approximately 15% of the total number of hemophiliacs. The most common treatment method is intravenous injection of concentrated factor IX, and severe hemophilia B patients will also receive preventive treatment to maintain coagulation factor IX. BBM-H901 is an AAV based gene therapy that uses carriers to deliver missing human coagulation factor (FIX) coding genes to enhance endogenous FIX levels and treat hemophilia.

Although gene therapy, as a promising "once and for all" therapy, is expected. However, the difficulties in successful development are enormous, and currently the most advanced gene therapies for hemophilia are not progressing smoothly. UniQure hemophilia B gene therapy AMT-061 has been reported as a potential safety event. At the end of last year (December 21, 2020), a phase III clinical trial of AMT-061 reported a case of hepatocellular carcinoma in a patient. Due to suspicion that the adverse event was related to AMT-061, the FDA suspended the clinical trial. On March 29, 2021, uniQure released a comprehensive investigation into the incident, which found that hepatocellular carcinoma was highly unlikely to be caused by eAMT-061. At present, there has been no news of restarting clinical trials.

In addition to safety events, the persistence of gene therapy for hemophilia is also a concern. In August of last year, the FDA rejected the approval of the first Roctavian gene therapy for hemophilia A. The FDA pointed out in the CRL that BioMarin needed more data to support BLA, and recommended that BioMarin complete a phase III clinical study of BMN 270-301 and provide 2-year follow-up data as substantive evidence that the therapy can sustainably maintain annual bleeding rate efficacy. On June 28th of this year, BioMarin once again submitted a Marketing Authorization Application (MAA) to EMA, despite Roctavian's recent disclosure of the gradual decline in efficacy persistence.

Belief Pharmaceuticals (a subsidiary of Xinzhi Pharmaceuticals) was founded in September 2016. It is a high-tech enterprise that integrates the research and development, production, and clinical application of gene therapy drugs. The founder of the company is Professor Xiao Xiao, a renowned leader in the gene therapy industry. As a senior expert in the global gene therapy industry, Professor Xiao Xiao has over 35 years of experience in research and development transformation in the AAV gene therapy industry and profound international influence. He has led more than 20 domestic and foreign gene therapy research projects, and led the team to successfully develop dozens of AAV gene therapy drugs and related key technologies. His research results have been selected as one of the 100 major discoveries in the global scientific field by Discover magazine in the United States.

Professor Xiao Xiao is also a well-known serial entrepreneur in the global gene therapy industry. In August 2016, its founding company, Bamboo Therapeutics, was acquired by Pfizer for $645 million, and the drug is currently in clinical phase III; In October 2020, its founder, Asklepios BioPharmaceutical, was acquired by Bayer for $4 billion, and multiple drugs have entered the clinical stage.

Belief Pharmaceuticals has several globally leading patents and technologies, and has comprehensively laid out in areas such as targeting different tissues with novel AAV caps, efficient transgenic expression box design, advanced clinical level vector manufacturing processes, and innovative clinical development paradigms. It has successfully developed a globally leading HEK293 500L suspension culture process and a large-scale downstream purification process for complete chromatography. Multiple projects have entered the IND application and clinical validation stages. The company's R&D pipeline includes hemophilia A, hemophilia B, Parkinson's disease, age-related macular degeneration, glycosaminoglycan storage disease, spinal muscular atrophy and other indications.

The approval of clinical trials for the gene therapy for hemophilia by Faith Medicine is not only a milestone event for the company, but also a new chapter in domestic AAV gene therapy. Relying on leading technology, Belief Pharma will continue to provide more effective innovative solutions for single gene genetic diseases, age-related diseases, and some malignant diseases through AAV (adeno-associated virus) carrier technology. With technology as our belief and patient oriented approach, we will contribute to the health of the entire population.

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