First Subject Receives New Drug Therapy for Hemophilia B

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.