Spark Therapeutics Presents Updated Interim Hemophilia B Data Supporting Consistent and Sustained Response at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress
Data demonstrate a 99-percent reduction in annualized infusion rate (AIR) and a 96-percent reduction in annualized bleeding rate (ABR) in 10 participants as of
Five trial participants are now at least one-year post investigational SPK-9001 administration, including one participant out approximately 18 months; all have discontinued routine factor IX concentrate infusions and have sustained increases in factor IX activity levels
The interim data will be presented today by
“We continue to be encouraged by the SPK-9001 clinical trial results observed to date, with all participants having discontinued routine infusions of factor IX concentrates,” said Katherine A. High, M.D., president and chief scientific officer at Spark Therapeutics. “The growing body of data showing a sustained response is a promising sign for this investigational hemophilia B gene therapy program.”
The first participant enrolled in the trial, who, as of
As of the
As reported in
Improvements in Health-Related Quality of Life in Adults with Hemophilia B
A separate prospective interim data analysis suggests a one-time infusion of SPK-9001 results in meaningful health-related quality of life (HRQoL) improvements in several measures, according to a poster that will be presented by
All participants were considered HRQoL responders based on the hemophilia-specific Haem-A-QoL Total Score, with the most marked statistically significant changes after vector infusion in responses related to “being dependent on factor concentrate” (p < 0.0001); “feeling different from others because of their hemophilia” (p < 0.001); and “worrying that their condition is worsening” (p < 0.003). Other measures, including, but not limited to, fear of complications, dependency on physician and not feeling contented about their body, did not reach statistical significance.
Participants on average reported “already good” HRQoL pre-vector infusion in the generic EuroQ-VAS (M=80.5±8.3), a standardized instrument for measuring HRQoL that comprises a health state classification followed by a health evaluation using a visual analogue scale (VAS), which still improved post-vector infusion (M=86.1±9.1), but was not statistically significant.
All participants completed generic and hemophilia-specific HRQoL questionnaires prior to vector infusion and four, 12, 26 and 52 weeks after vector infusion.
About Hemophilia B
Hemophilia, a rare genetic bleeding disorder that causes the blood to take a long time to clot because of a deficiency in one of several blood clotting factors, is almost exclusively found in males. People with hemophilia 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 also is called congenital factor IX deficiency or Christmas disease. The current standard of care requires recurrent intravenous infusions of either plasma-derived or recombinant factor IX to control and prevent bleeding episodes. There exists a significant need for novel therapeutics to treat people living with hemophilia.
About the SPK-FIX Program and SPK-9001
Spark Therapeutics and Pfizer entered a collaboration in December 2014 for the SPK-FIX program, including SPK-9001, under which Spark Therapeutics is responsible for conducting all Phase 1/2 studies for any product candidates, while Pfizer will assume responsibility for pivotal studies, any regulatory activities and potential global commercialization of any products that may result from the collaboration.
Spark Cautionary Note on Forward-looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the company's SPK-FIX program. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that: (i) our lead SPK-FIX product candidate, SPK-9001, may not produce sufficient data in our Phase 1/2 clinical trial to warrant further development; and (ii) our overall collaboration with Pfizer may not be successful. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in our Annual Report on Form 10-K, our Quarterly Reports on Form 10-Q and other filings we make with the
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