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First data for Spark's hemophilia B gene therapy

May 20, 2016 12:21 AM UTC

Spark Therapeutics Inc. (NASDAQ:ONCE) gained $5.94 (14%) to $47.17 on Thursday after it and partner Pfizer Inc. (NYSE:PFE) said the first three hemophilia B patients enrolled in a Phase I/II study of SPK-9001 ( SPK-FIX) had "AAV-mediated Factor IX activity levels" after one administration of the low dose of the gene therapy. An European Hematology Association meeting.

In the two patients without history of liver disease, one had Factor IX activity levels stabilize at 28% of normal 18 weeks post-administration, while the other stabilized at 30% of normal after seven weeks. The partners said Factor IX activity levels "rose consistently" and were at 16% of normal after three weeks in the third subject, who the abstract said had a history of HCV infection that had spontaneously cleared. ...