Pfizer: Beqvez Gene Therapy Decreases Bleeding Rates in Hemophilia B Patients – 2 Minute Medicine

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A recent Phase III open-label, single-arm study conducted by Pfizer in collaboration with Spark Therapeutics investigated the safety and efficacy of a gene therapy named Beqvez in adult male patients with moderately severe to severe hemophilia B. Spark Therapeutics was responsible for conducting all Phase 1 and 2 studies, whilePfizer assumed responsibility for pivotal studies, regulatory activities, and potential global commercialization. The clinical trial named the BENEGENE-2 study found that during follow-up, there were decreased annualized bleeding rates in patients when they received the gene therapy compared to when theywere treatedwith routine factor IX prophylaxis. The results showed that patientsgenerallytolerated Beqvez well, with no reports of death, serious adverse events, or thrombotic events.

Physicians Perspective

Hemophilia B is a rare genetic disorder that prevents normal blood clotting for people who cannot generate the factor IX protein, causing them to bleed more frequently and profusely. According to the World Federation of Hemophilia, 38,000 people are affected by this disorder. Patients with hemophilia B struggle with the commitment and lifestyle disruptions of regular (factor IX protein) infusions, spontaneous bleeding episodes, painful joint damage, and mobility issues. Traditionally, patientsneed toreceive routine infusions of Factor IX treatment to prevent and control bleeding. However, thiscurrentstandard treatment method does not alter the underlying disease process. With a gene therapy infusion, patients can receive a one-time treatment that helps them produce their own Factor IX. This alternative active source of endogenous Factor IX helps improve bleeding outcomes and reduces the need for Factor IX infusions. The single gene therapy injectionis expectedto be effective for at least10years.

Molecular Targets

Hemophilia Bis causedby mutations in the F9 genewhichcarries instructions for making Factor IX, a protein involved inthe formation ofblood clots. Beqvez delivers a highly functional version of F9 to liver cells, where blood clotting factorsare produced. The functional F9 gene is called Padua and encodes for a version of Factor IXthat has a5-10greaterclotting activity than the endogenously produced protein. The F9 geneis packagedinside a specialized adeno-associated virus, surrounded by an outer shell capsid thatis taken upby liver cells. Once the F9 gene componentis taken upby the liver cells, the endogenous machinery can begin to produce the enhanced version of the Factor IX protein.

Company History

Pfizer is an American pharmaceutical and biotechnology corporation headquartered in Manhattan, New York City. In addition to Beqvez, Pfizeralsohas gene therapies under investigation in phase 3 trials for hemophilia A and Duchenne muscular dystrophy. The companyis also testinganti-tissue factor pathway inhibitors to treat people with hemophilia A and B.

Further Reading: https://www.pfizer.com/news/press-release/press-release-detail/us-fda-approves-pfizers-beqveztm-fidanacogene-elaparvovec

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Pfizer: Beqvez Gene Therapy Decreases Bleeding Rates in Hemophilia B Patients - 2 Minute Medicine