China OKs Hemophilia B Trial Testing BBM-H901 Gene Therapy

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by Patricia Inácio, PhD |

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Belief Biomed is launching a Phase 1/2 clinical trial in China to test BBM-H901, its investigational one-time gene therapy for hemophilia B.

The announcement comes after China’s National Medical Products Administration (NMPA) approved the company’s investigational new drug application, which had been submitted in April, Belief Biomed said in a press release.

According to the company, this is the first investigational new drug approval for an intravenous (into-the-vein) infusion gene therapy for hemophilia B in China, as well as the first intravenous infusion gene therapy for rare diseases in the country.

“Gene therapy is the third wave of biopharma industry revolution, following small molecules and antibodies,” said Jane Zheng, Belief Biomed‘s CEO.

“The gene therapy market has proliferated in recent years,” Zheng said.

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Patients’ Priorities for Gene Therapy: Durability and Bleeding Control

BBM-H901 uses a modified and harmless version of an adeno-associated virus (AAV) to deliver a working copy of the F9 gene — which provides instructions for making factor IX, the blood-clotting protein missing or defective in people with hemophilia B — to liver cells, the main producers of clotting factors in the body.

The gene therapy is expected to increase the levels of factor IX in hemophilia B patients. Its goal is to prevent and help control bleeds and eliminate the need for regular treatments.

Developed using Belief Biomed’s own new manufacturing processes, BBM-H901 has demonstrated high efficacy and safety in a pilot Phase 1 study in China, the company said. The open-label trial (NCT04135300) is underway at the Institute of Hematology & Blood Diseases Hospital, in Tianjin.

It is evaluating the safety, tolerability, and kinetics (motion) of a single injection of BBM-H901 in hemophilia B patients with low factor IX levels.

After infusion — at a dose of 5 trillion vector genomes per kilogram — patients are followed for about one year. They are then encouraged to enroll in an extension study that will evaluate the safety of BBM-H901 for a total of five years.

According to Belief Biomed, the results so far have shown that the annualized bleeding rate declined significantly after the gene therapy’s infusion, with patients reaching stable factor IX activity during follow-up and with no indication of serious adverse events.

BBM-H901 showed signs of “one-time treatment, long-term benefit,” the company states in the release.