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Note: The following is an edited version of a press release from Spark Therapeutics.聽Read the press release from Spark Therapeutics聽in it鈥檚 entirety here.


Spark Therapeutics recently announced that 10 participants in its ongoing Phase 1/2 clinical trial of聽SPK-9001 for hemophilia B, as of the June 5, 2017 data cut off, had their AIR reduced approximately 99 percent to a mean of 1.0 annual infusion as of the data cut-off date, compared with 67.5 annual infusions before聽SPK-9001聽administration. Nine of the 10 participants have not experienced a bleed since vector infusion; overall ABR was reduced by approximately 96 percent to a mean of 0.4 annual bleeds, compared with 11.1 bleeds before聽a single administration of 5 x 1011 vector genomes (vg)/kg body weight of SPK-9001. These data represent approximately 9.63 cumulative patient years of SPK-9001exposure from the start of the trial, with one participant out approximately 18 months post-infusion and four additional participants out at least one year post-infusion. All 10 trial participants have shown consistent and sustained increases in factor IX activity level and a discontinuation of routine infusions of factor IX concentrates. Their mean steady-state factor IX activity level, or the average of each participant鈥檚 average activity level after 12 weeks, was 33 percent.

The interim data was聽presented聽at ISTH 2017 Congress in Berlin.


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