Patient dosing in a phase 3 registrational clinical trial of BBM-H901, a gene therapy for hemophilia B, has been completed, Belief BioMed Group announced.
“We are pleased to see patient dosing has been successfully completed in phase 3 registrational trial of BBM-H901, the first blockbuster product independently developed and manufactured by Belief BioMed with the potential to become the first adeno-associated virus gene therapy approved for marketing in China for prophylactic treatment of bleeding in adults with hemophilia B,” said Dr. Xiao Xiao, co-founder, chairman, and chief science officer of Belief BioMed.
“Featuring ‘one-time administration and long-term effectiveness,’ this innovative therapy will bring better survival benefits to patients,” he added.
This multicenter, single-arm, open, and single-dose clinical study enrolled hemophilia B patients older than 18 years with endogenous blood coagulation factor IX activity 2 IU/dL (≤2%) or less and explored the safety and efficacy of a single intravenous infusion of BBM-H901. The medication has been granted the Investigational New Drug Application approval for registrational clinical study in China.
Read more about hemophilia experimental therapies
The researchers have now completed dose exploration and dose escalation studies with promising results indicating high efficacy and a favorable safety profile of the medication. In fact, 100% of patients stopped their regular treatment for factor IX, and their annual bleeding rate was reduced to 0%. The decrease of coagulation factors was maintained in all patients, and there were no reports of serious adverse events associated with the use of BBM-H901.
The results of previous clinical studies investigating the novel medication were published in The Lancet Haematology and The New England Journal of Medicine in 2022. Based on these data, the experimental drug received Orphan Drug Designation by the US Food and Drug Administration, as well as the Breakthrough Therapy Designation by the China Center for Drug Evaluation.
BBM-H901 is an adeno-associated virus gene therapy administered intravenously. It delivers the gene coding the human factor IX into the recipient’s cells, which is meant to increase coagulation factor levels.
Belief BioMed announces a key milestone of dosing completion for all subjects in its registrational clinical trial of BBM-H901. News release. Belief BioMed Group; April 23, 2023.