Belief BioMed Completed Dosing of the First Subject in the Registrational Gene Therapy Clinical Trial for Hemophilia B
On December 30th, 2021, Belief BioMed Group（BBM）announced that it has successfully dosed the first subject in the registrational gene therapy clinical trial by intravenous (IV) infusion of BBM-H901, an adeno-associated virus (AAV) vector expressing factor IX gene for treatment of adult male hemophilia B patients. BBM-H901 is the first investigational new drug (IND) approved in China for gene therapy by the IV delivery route for genetic diseases, and in specific, for hemophilia B. At present, no AAV gene therapy product has been approved for marketing for hemophilia in China and globally.
This registrational clinical study (CTR20212816) is a multi-center, single-arm, open-label and single-treatment study. Its objective is to evaluate the safety, tolerability, pharmacokinetics, as well as long-term efficacy and safety 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 the company's first major product made in our own state-of-art cGMP facility, we are pleased that Belief BioMed has reached an important milestone in the clinical research after dosing the first subject in the pivotal clinical study. BBM-H901 has previously demonstrated good safety and long-term efficacy in an investigator initiated clinical trial (IIT). In that study the annualized bleeding rate (ABR) was greatly reduced, and the level of coagulation factors FIX was significantly increased and persisted in all patients after IV injection of BBM-901. No serious adverse event (SAE) was reported.” said Dr. Xiao Xiao, co-founder, Chairman and Chief Science Officer (CSO) of Belief BioMed.
"We are thankful to our clinical collaborators, our patient community and our entire team who made this happen”, Dr. Xiao added. Belief BioMed, as a leading gene therapy company in China, will continue to make stride in both preclinical and clinical studies to bring more innovative drugs to the needy patients. In addition, this milestone has laid a solid foundation for the clinical application of our gene therapy drugs and paved the way for the clinical development of these drugs for a variety of major and unmet clinical needs in the future. Our goal is to make innovative gene therapy drugs available and affordable to the patients, and at the same time, to facilitate the growth of gene therapy industry.
Hemophilia is a bleeding disorder that slows the blood clotting process. People with this condition experience prolonged bleeding or oozing following an injury, surgery, or having a tooth pulled. In severe cases of hemophilia, continuous bleeding occurs after minor trauma or even in the absence of injury (spontaneous bleeding). Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. This disease has seriously jeopardized the life quality of patients and brought huge impact and heavy burden to patients, their families and the whole society.
BBM-H901 is a cutting-edge bio-engineered adeno-associated viral (AAV) vector utilizing a novel recombinant AAV capsid, containing a codon-optimized human factor IX gene under the control of a liver-specific promoter. Belief BioMed owns the proprietary patents of the capsid and the cassette. Leveraging the serum-free production and chromatographic purification process developed in house by Belief BioMed to produce drugs in line with cGMP requirements, BBM-H901 has demonstrated high efficacy and safety with the FIX (Factor IX) well-expressed in patient’s plasma, achieving “one-single treatment, long-term benefit” effect.
The Investigator Initiated Trial (IIT) of BBM-H901 is one of the earliest clinical trials for AAV gene therapy carried out in China. The IIT(NCT04135300) of BBM-H901 started in 2019. Belief BioMed submitted its original IND application to NMPA in April 2021 and received clearance on August 6th, 2021.
About Belief BioMed Group
Founded in September 2016, Belief BioMed Group (BBM) is an industry-leading innovative pharmaceutical company that fully integrates gene therapy drug R&D, manufacturing platforms and clinical transformation. The company was co-founded by Dr. Xiao Xiao, who currently serves as the Chairman and Chief Science Officer (CSO) of the company. Dr. Xiao is a renowned expert and leader in the gene therapy industry, with international reputation and over 35 years of experience in research of adeno-associated virus (AAV) and its utilities.
BBM is committed to providing more effective and innovative therapies for monogenic disorder diseases, neurodegenerative diseases and malignant diseases using AAV vector technology. BBM has its own patents for a variety of AAV vectors, as well as dozens of new capsids of specific tissue tropisms. Its recombinant AAV packaging technology has also been widely used in the field of AAV gene therapy. The company has extensive R&D pipelines covering a wide range of major, unmet clinical needs, including hemophilia, neurodegenerative diseases, lysosomal storage diseases, hereditary neuromuscular diseases and ophthalmology diseases and so on.
As an industry leader, Belief BioMed’s strong R&D capability has been recognized by the top investment institutions, and the company has scientists deeply engaged in the research field and executives with expertise in commercialization. The completion of the first subject administration of hemophilia gene therapy registered clinical trial marks another significant milestone for the company. Belief BioMed will continue to uphold its vision to help the health of all people and benefit patients in China and around the globe.
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