Dosing complete in Phase 3 trial of Belief BioMed’s gene therapy candidate

BBM-H901 is an investigational gene therapy for hemophilia B patients

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Patient dosing has been completed in a registrational Phase 3 clinical trial in China testing BBM-H901, Belief BioMed‘s (BBM)’s investigational gene therapy for hemophilia B, the company announced.

BBM-H901 is designed to restore the production of factor IX (FIX) — the blood-clotting protein that is faulty or missing in people with hemophilia B — by delivering a functional copy of the F9 gene to liver cells, which are the main producers of clotting factors in the body. Gene therapy components are delivered to liver cells by a modified, harmless adeno-associated virus (AAV) that works as a vehicle.

The gene therapy is given as a single intravenous (into-the-vein) infusion.

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“Featuring ‘one-time administration and long-term effectiveness,’ this innovative therapy will bring better survival benefits to patients,” Xiao Xiao, PhD, co-founder, chairman, and chief science officer of Belief BioMed, said in a press release.

According to the company, BBM-H901 is the first investigational AAV gene therapy for adults with hemophilia B being tested in a registrational clinical trial in China.

“We are pleased to see patient dosing has been successfully completed in the [Phase 3] 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,” Xiao said.

The registrational Phase 3 trial (CTR20212816), which started dosing participants last year, intends to obtain sufficient data to support a future application requesting the therapy’s approval. It’s a multicenter open-label study designed to assess the safety and effectiveness of a single intravenous infusion of BBM-H901 in adult men with hemophilia B whose FIX activity levels are 2% or lower than normal.

Prior dose exploration and escalation studies have been completed in China with positive results.

As previously reported, all patients treated with BBM-H901 in an open-label Phase 1 trial (NCT04135300) no longer required FIX replacement therapy infusions. Also, the median annualized bleeding rate decreased from 12 to zero, and the median number of joints with active bleeds dropped from 1.5 to zero. This was accompanied by a sustained increase in FIX activity levels. There were no reports of serious or severe adverse side effects.

“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,” Xiao said.

“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!,” he added.

BBM-H901 is being developed and manufactured by BBM’s wholly-owned subsidiary, Shanghai Belief-Delivery Biomed.