Pfizer's Hemophilia B Gene Therapy Beqvez Nears FDA Approval with Breakthrough Clinical Data

The day before yesterday was the 35th World Hemophilia Day. On this special day, we can't help but turn our attention to a major breakthrough in the medical field — the hemophilia B gene therapy developed by Pfizer.Expected in the second quarterApproved for marketing by the U.S. FDA. This news is undoubtedly a hopeful福音 for hundreds of thousands of hemophilia patients worldwide.

As early asIn January this year,HuiRui's Hemophilia B Gene TherapyBeqvez is inApproved in Canada，For the treatment of adults aged 18 years or older with moderately severe to severe hemophilia B. Notably,Beqvez's US marketing application is based onThe data from the Phase 3 clinical trial BENEGENE-2 is the same as the clinical study submitted for the approval of this therapy in Canada.

BENEGENE-2 data shows,Compared with the use of FIX prophylactic treatment regimens in conventional therapy,Annualized Bleeding Rate (ABR) of Total Bleeding Events in Patients After Beqvez InfusionDecreased by 71%, with an annualized coagulation factor infusion rate reduced by 92%.

▲ BENEGENE-2 Trial Data,Beqvez Reduces Patients' Annualized Bleeding Rate

Data from the World Federation of Hemophilia shows that more than 38,000 people worldwide were affected in 2021.Hemophilia B. ForHemophilia BPatients: The current standard of care requires frequent intravenous infusions of plasma-derived or recombinant FIX to control and prevent bleeding episodes. Relevant data shows that 65% of pediatric hemophilia patients and 16.5% of adult hemophilia patients actively receive prophylactic treatment, and the dosage of clotting factors is also increasing year by year.

In terms of genetic diseases, gene therapy has been successfully applied to treat conditions such as congenital blindness, hemophilia, and Duchenne muscular dystrophy. By introducing normal genes into the patient's body to replace or repair defective genes, gene therapy...TherapyCan restore the normal physiological functions of patients and improve their quality of life. Hemophilia, caused by a single gene mutation, is one of the most suitable diseases for gene therapy.

Pfizer's Hemophilia B Gene TherapyBeqvezContaining bioengineered adeno-associated virus (AAV) capsids (protein shells) and a high-activity variant of the FIX gene. For patients with hemophilia B, the goal of this gene therapy is to enable them toOne-time treatmentProducing endogenous FIX protein, rather than requiring regular intravenous infusion of FIX as in the current standard treatment.

▲ BENEGENE-2 trial data shows,After Beqvez infusion, FIX activity in patients remained stable.

In addition, in August 2022, SichuanZhiShan WeiXinBiotech Co., Ltd.'s AAV Gene Therapy ZS801 for Hemophilia B Receives CDE Approval for Clinical Application, Targeting Bleeding Control and Prevention in Male Patients Aged 18 and Above with Severe or Moderately Severe Hemophilia B. The product has previously undergone IIT clinical research at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences. The first subject showed a significant and sustained increase in plasma FIX activity levels after receiving treatment in March 2021, with good safety reported.

April 2022, ShanghaiTianze YuntaiVGB-R04 Injection, an AAV gene therapy candidate independently developed by Bio for the treatment of Hemophilia B, has been granted clinical trial approval.VGB-R04 is administered intravenously, delivering the target gene (therapeutic gene) to the liver cell nucleus mediated by the AAV capsid. It expresses a variant of coagulation factor IX (hFIX Padua) protein in liver cells. FIX-Padua is a naturally occurring, highly active FIX variant (R338L) with activity approximately eight times that of normal wild-type FIX, meaning it can achieve normal blood clotting function at lower expression levels.

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