Spark Therapeutics (ONCE) and Pfizer Inc. (PFE) announced updated results of the first cohort from the ongoing Phase 1/2 clinical trial of SPK-9001, the lead investigational candidate in the companies' SPK-FIX program, in development for the treatment of hemophilia B as a potential one-time therapy.
According to the companies, data presented today, current as of July 12, 2016, showed that the low dose cohort of four subjects enrolled in the study experienced consistent and sustained factor IX activity levels following a single administration of SPK-9001 at the initial dose level studied in the trial.
Across the four subjects, average steady-state factor IX activity levels are 31.8 percent ±6.9 percent, (range 20% - 44%) of normal, determined by averaging levels beginning at 8 weeks post vector administration through follow up over 12-31 weeks. No sustained elevation in liver enzyme levels was seen. The first four subjects all with follow up beyond 12 weeks without any need for immunosuppression.
SPK-9001 has received breakthrough therapy designation from the U.S. Food and Drug Administration.
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