Trial of Hemophilia B Gene Therapy BBM-H901 Begins Dosing in China

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by Steve Bryson, PhD |

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The first man with hemophilia B has been dosed in a clinical trial in China to evaluate Belief BioMed’s investigational one-time gene therapy BBM-H901.

According to the company, BBM-H901 is the first investigational gene therapy given intravenously, or directly into a vein, for hemophilia B or any genetic disease to enter clinical testing.

“We are thankful to our clinical collaborators, our patient community and our entire team who made this happen,” Xiao Xiao, co-founder, chairman, and chief science officer at Belief, said in a press release.

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Hemophilia B is caused by mutations in the F9 gene, leading to the lack or resulting in the production of a defective blood clotting called protein factor IX (FIX). As a result, hemophilia B patients are unable to prevent or control bleeds. Because the F9 gene is found on the X chromosome, males are more susceptible to the disease than females.

BBM-H901 is designed to deliver a working copy of F9 using a modified, harmless version of an adeno-associated virus (AAV) to liver cells, where blood clotting factors are produced. The gene therapy aims to increase FIX levels to prevent or control bleeding episodes and eliminate the need for regular treatments.

This registrational trial (CTR20212816) — aimed at obtaining sufficient data to support a future application requesting the therapy’s approval — is an open-label study designed to evaluate the efficacy, safety, tolerability, and pharmacokinetic properties of a single intravenous infusion of BBM-H901 in adult men with hemophilia B. (Pharmacokinetics is the study of how the medicine moves into, through, and out of the body.)

Much of the therapy’s production will occur in Belief BioMed’s in-house manufacturing facility, which follows current good manufacture practice (cGMP) regulatory requirements.

“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,” Xiao said.

BBM-H901 is being tested in an ongoing Phase 1 pilot study (NCT04135300) in China, which has enrolled 10 hemophilia B patients, ages 18 and older. According to the company, the gene therapy significantly increased and sustained FIX levels in the bloodstream and safely reduced the annualized bleeding rate (ABR).

After one year of follow-up, participants can enroll in an extension study that aims to continue evaluating the safety of BBM-H901 for up to five years.

“BBM-H901 has previously demonstrated good safety and long-term efficacy,” Xiao added. “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 [intravenous] injection of BBM-901. No serious adverse event (SAE) was reported.”