SHANGHAI, April 24, 2023 /PRNewswire/ — Belief BioMed Group (BBM), an industry-leading biotech company focusing on innovative gene therapies, announced today the completion of patient dosing in its Phase III Registrational Clinical Trial (CTR20212816) of BBM-H901, a gene therapy for Hemophilia B developed and manufactured in-house by Shanghai Belief-Delivery Biomed Co., Ltd., its wholly owned subsidiary.
BBM-H901 is indicated for prophylactic treatment of bleeding in adults with hemophilia B. It is the first adeno-associated virus (AAV) gene therapy for Hemophilia B with IND approval for registrational clinical study in China.
This registrational study is a multi-center, single-arm, open and single-dose clinical study. Its objective is to evaluate the safety and efficacy 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 of today, its dose exploration and dose escalation studies have been completed in China, and positive results have been obtained in terms of efficacy and safety: 100% patients stopped FⅨ treatment, and the annual bleeding rate (ABR) was reduced to zero; the level of coagulation factors was significantly increased and persisted in all patients; safety profile was good with no serious adverse event reported.
Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, “We are pleased to see patient dosing has been successfully completed in the phase III registrational trial of BBM-H901, the first blockbuster product independently developed and manufactured by BBM with the potential to become the first AAV gene therapy approved for marketing in China for prophylactic treatment of bleeding in adults with hemophilia B. Featuring ‘one-time administration and long-term effectiveness’, this innovative therapy will bring better survival benefits to patients. I want to express my heartfelt thanks to all investigators, patients and their families as well as the project team for their contribution to the trials. I believe it is a milestone that will lay a solid foundation for the future clinical application of more of our gene therapies and help promote the vigorous development of the entire gene therapy industry. Being fully committed to addressing unmet medical needs, BBM will continue to tap into the potential of gene therapy, and strive to bring more first-in-class and best-in-class products to patients!”
The forward-looking statements made in this article only relate to events or information as of the date when the statements are made in this article. Except as required by law, we undertake no obligation to update or publicly revise any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. All statements in this article are made on the date of publication of this article and may change due to future developments.