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Spark Therapeutics and Pfizer Announce Updated Data from First Cohort in Hemophilia B Phase 1/2 Trial Demonstrating Consistent, Sustained Therapeutic Levels of Factor IX Activity

First four subjects all with follow up beyond 12 weeks without any need for immunosuppression

PHILADELPHIA and NEW YORK, July 25, 2016 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE) and Pfizer Inc. (NYSE:PFE) announced today 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. SPK-9001, a novel bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimized, high-activity human factor IX variant, was developed using Spark's proprietary technology platform for selecting, designing, manufacturing and formulating highly optimized gene therapies. SPK-9001 has received breakthrough therapy designation from the U.S. Food and Drug Administration.

Data presented today, current as of July 12, 2016, show 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 (5 x 1011 vg/kg) studied in the trial. Across the four subjects, average steady-state factor IX activity levels are 31.8% ±6.9% (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 were seen.

To date, SPK-9001 has been well-tolerated and no subjects have needed, or received, immunosuppression. None of the first four subjects, through a combined 76 weeks of observation, has received infusions of factor IX concentrates to prevent bleeding events. Only one precautionary infusion has taken place due to a suspected ankle bleed in one subject two days after administration of vector.

"The clinical data emerging from our hemophilia B program, which is partnered with Pfizer, continue to show early promise in achieving our goal of eliminating the need for regular factor IX infusions to control and prevent bleeding episodes in patients with these diseases through a potentially one-time, intravenous administration of highly optimized gene therapies," said Dr. Katherine High, president and chief scientific officer of Spark Therapeutics.

These data from the Phase 1/2 clinical trial of SPK-9001 will be presented at a moderated poster presentation today, Monday July 25, at 4:00 pm (ET) at the 2016 International Congress of the World Federation of Hemophilia (WFH) by Dr. Lindsey George, a hematologist at the Children's Hospital of Philadelphia and the lead clinical investigator in the Phase 1/2 clinical trial of SPK-9001 for the treatment of hemophilia B. Dr. George also will present at a Late Breaking Gene Therapy session on Wednesday July 27, 2016 at 2:15 pm (ET).

About Hemophilia B

Hemophilia is a rare genetic bleeding disorder that causes the blood to take a long time to clot as a result of a deficiency in one of several blood clotting factors, and occurs almost exclusively in males. People with hemophilia face specific risks as they are not able to form blood clots efficiently and are at risk for excessive and recurrent bleeding from modest injuries, which have the potential to be life threatening. People with severe hemophilia often bleed spontaneously into their muscles or joints. The incidence of hemophilia B is one in 25,000 male births. People with hemophilia B have a deficiency in clotting factor IX, a specific protein in the blood. Hemophilia B is also called congenital factor IX deficiency or...