Preliminary data from an ongoing Phase 1/2 clinical trial evaluating Spark Therapeutics' (ONCE +1.4%) factor IX product SPK-9001 show a significant treatment effect. The results will be presented tomorrow at the Hemostasis and Thrombosis Research Society 2017 Scientific Symposium in Scottsdale, AZ.
As of March 24, 10 patients who received a single administration SPK-9001 have discontinued routine factor IX infusions. Nine of 10 have not taken factor IX concentrates to prevent or control bleeding events. Two of 10 experienced a temporary elevation in liver enzymes or decline in factor IX activity, potentially indicating an immune response to the Spark100 vector capsid. Treatment with corticosteroids resolved both.
Based on patient histories prior to the study, annualized bleed rates (ABRs) were reduced 96% to a mean of 0.39 annual bleeds. Annual infusion rates (AIRs) were reduced 99% to an average of 0.98 annual infusions.
SPK-9001 is a bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimized high-activity human factor IX variant designed to deliver and express a therapeutic gene in the liver. It was developed using Spark's proprietary technology platform called SPK-FIX which is being advanced under a 2014 collaboration with Pfizer.
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