New deal aims to bring BBM’s hemophilia A gene therapy to China
Grand Life Sciences to market BBM-H803 on mainland, beyond if approved
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Grand Life Sciences will have exclusive rights to market Belief Biomed's hemophilia A gene therapy in greater China if the experimental treatment is approved.
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BBM-H803 is a one-time gene therapy delivering a working F8 gene to liver cells to increase factor VIII production.
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The therapy is undergoing registrational Phase 1/2/3 trials in China, where no hemophilia A gene therapies are yet approved.
Grand Life Sciences Group has entered into an exclusive collaboration agreement with Belief Biomed (BBM) to commercialize BBM-H803, BBM’s one-time investigational gene therapy for hemophilia A, in Greater China if it is eventually approved in the region.
The agreement covers Mainland China, as well as Hong Kong, Macau, and Taiwan.
“By combining Belief BioMed’s solid clinical development expertise with Grand Life Sciences’ robust commercialization capabilities, we are creating a powerful synergy to jointly advance the commercialization of gene therapies for hemophilia A,” Xiao Xiao, BBM’s cofounder, chairman, and chief science officer, said in a company press release announcing the deal. “We look forward to bringing this new treatment option to hemophilia A patients in China.”
Zenghui Feng, chairman of Grand Life Sciences, said the healthcare enterprise group is looking forward to partnering with BBM.
“Together, we aim to efficiently accelerate … patient access to this innovative therapy in China,” Feng said. “Our shared goal is to enable more hemophilia A patients to benefit earlier, relieving them from the physical burden of frequent injections and the fear of bleeding episodes.”
Hemophilia A is A blood disorder usually caused by mutations in the F8 gene, which contains the instructions for making clotting factor VIII (FVIII), a protein needed for blood clotting. When FVIII is missing or doesn’t work properly, people with hemophilia A experience excessive bleeding that may occur unexpectedly or after injuries.
No hemophilia A gene therapy has been approved to date in China
BBM-H803, also known as BBM-002, is a gene therapy designed to supply cells in the liver with a working version of F8, delivered via a harmless viral carrier called an adeno-associated virus (AAV). After a one-time delivery, liver cells are expected to begin producing functional FVIII, leading to long-term increases in FVIII activity and a reduced risk of bleeding.
To date, there are no approved hemophilia A gene therapies in China. The company has said the planned clinical dose for BBM-H803 is lower than doses used for other AAV-based gene therapy approaches, which is expected to help reduce the risk of side effects.
A registrational Phase 1/2/3 trial (NCT06111638), which began dosing in 2024, is evaluating BBM-H803 as a single intravenous, or into-the-vein, infusion for adults with severe hemophilia A. That trial is still recruiting participants at sites in China.
As a registrational trial, it aims to obtain sufficient data to support a future application requesting the therapy’s approval.
An initial dose-escalation phase is intended to establish a recommended dose for future testing. That first stage is also used to closely monitor side effects, including potential liver-related changes, which can occur with AAV-based gene therapies.
In a subsequent dose-expansion phase, researchers will evaluate whether the gene therapy reduces the annualized bleeding rate, or the number of expected bleeding episodes in a one-year period. Researchers will also track FVIII activity as a key marker of how well the therapy restores clotting function.
BBM-H803 is being tested in a small clinical trial in China
In addition to the BBM-sponsored registrational study, BBM-H803 is being evaluated in an ongoing investigator-initiated Phase 1 clinical trial (NCT05454774) at the Institute of Hematology & Blood Diseases Hospital in Tianjin. In that study, eight men are expected to receive the gene therapy at a dose of 10 trillion vector genomes per kilogram.
“We look forward to integrating Belief BioMed’s cutting-edge [research and development] capabilities with Grand Life Sciences’ mature commercialization platform and market channels,” Feng said.
In the U.S., BBM-H803 holds orphan drug and rare pediatric disease designations, both of which provide financial and regulatory incentives to speed a therapy’s development.
Belief is also working to expand the reach of its hemophilia B gene therapy BBM-H901 (dalnacogene ponparvovec), which was approved for people with moderate to severe forms of the bleeding disorder in China last year and, more recently, in Macao.
“Belief BioMed focuses on the cutting-edge gene therapy field … striving to fill a large number of unmet clinical needs,” Xiao said.
Xiao noted that, last year, the company launched China’s first hemophilia B gene therapy. That drug is now “providing a novel treatment option for patients in this field,” Xiao said.
“Now, turning to the treatment of hemophilia A, we are delighted to forge this strategic partnership with Grand Life Sciences,” he said.
