Spark Therapeutics will present novel results regarding the ongoing Phase 1/2 clinical trial (NCT02484092) investigating SPK-9001 in hemophilia B patients at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, to be held July 8-13 in Berlin.
Two presentations focus on data from the trial: “SPK-9001: Adeno-associated Virus Mediated Gene Transfer for Haemophilia B Achieved Durable Endogenous Prophylaxis at Levels of Activity Sufficient to Achieve Significant Mean Reduction in Annual Bleeding and Infusions Rates in Preliminary Data from an Ongoing Phase 1/2a Trial,” by Dr. Lindsey George, and “Preliminary Results of SPK-9001 Gene Transfer Demonstrate Statistical Improvements on the Health-related Quality-of-Life in Adults with Haemophilia B Speaker,” by researcher Sylvia von Mackensen.
Dr. Katherine A. High, president and chief scientific officer at Spark, will give an overview of gene therapy research in hemophilia. She’ll also discuss results from the trial in a presentation, “Gene Therapy Replacement.”
SPK-9001 is an investigational gene therapy that helps the body produce factor IX, the protein missing in hemophilia B patients. The ongoing trial enrolled 10 patients to assess the benefits of this treatment; all had been receiving regular factor IX infusions, but stopped before enrolling the study.
Previously presented results from the trial showed that SPK-9001 reduced their bleeding rate by 96 percent; patients had a mean 9.2 annual bleeding episodes before the study, falling to 0.39 after the treatment.
Treatment with SPK-9001 also decreased the mean number of annual infusions by 99 percent, from 68.5 before the study to 0.98 after treatment.
Importantly, so far SPK-9001 has been safe and well-tolerated, with no reports of safety concerns.
Hemophilia B is caused by missing or defective protein called factor IX, which is essential in clotting the blood. As such, hemophilia B patients bleed longer than others when injured. Doctors generally treat the disease through intravenous factor IX, but this procedure is expensive and painful.