Belief BioMed Group (BBM) has concluded dosing patients in its Phase III registrational trial evaluating BBM-H901 for the treatment of haemophilia B.

The multi-centre, single-arm, open and single-dose trial aims to assess the safety and efficacy of a single intravenous infusion of BBM-H901 in patients aged 18 years or above.

Patients with endogenous blood coagulation factor Ⅸ (FⅨ) activity ≤2 IU/dL are included in the trial.

The trial has so far completed its dose exploration and dose escalation portions, with positive efficacy and safety data.

The data also showed that 100% of patients stopped FⅨ treatment, while the annual bleeding rate (ABR) came down to zero.

The level of coagulation factors grew significantly and was found in all patients, with a good safety profile and no serious adverse event observed.  

BBM-H901, an adeno-associated virus (AAV) gene therapy to treat haemophilia B, has been developed and produced by BBM’s wholly owned subsidiary Shanghai Belief-Delivery Biomed.

Haemophilia is a hereditary bleeding disorder and is primarily triggered by mutations in the coagulation factor Ⅷ or Ⅸ genes.

Belief BioMed co-founder, chairman and chief science officer Dr Xiao Xiao 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 haemophilia B.

“Featuring ‘one-time administration and long-term effectiveness’, this innovative therapy will bring better survival benefits to patients.”

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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