Spark Therapeutics Presents Preliminary Data on Investigational SPK-8011 Phase 1/2 Dose-escalation Clinical Trial in Hemophilia A at 59th American Society of Hematology (ASH) Annual Meeting and Exposition
Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, containing a codon-optimized human factor VIII gene under the control of a liver-specific promoter, is being studied as a potential one-time gene therapy for hemophilia A. It is the second investigational hemophilia gene therapy to emerge from Spark Therapeutics’ leading gene therapy platform.
“While still early in the dose-escalation clinical trial of SPK-8011, we are encouraged these data show clinically significant reductions in ABR and AIR with no serious adverse events reported to date,” said Katherine A. High, M.D., president and head of research and development at Spark Therapeutics. “We look forward to continuing to enroll additional participants to further increase our understanding of how to optimize the potential efficacy of this investigational gene therapy.”
As of the
Two additional participants, infused at a dose of 1 x 1012 vector genomes (vg)/kg body weight, have also achieved therapeutic levels of factor VIII. They have now been followed for 19 weeks and 14 weeks and have mean sustained factor VIII activity levels of 9 percent (7 to 12 percent) and 13 percent (7 to 24 percent) of normal, respectively, as of the data cutoff. Both participants have completed a precautionary tapering course of corticosteroids.
Three more participants have been infused, one at the 1 x 1012 vector genomes (vg)/kg body weight dose and two at a dose of 2 x1012 vector genomes (vg)/kg body weight. Results for these three participants are too early to report as of the data cutoff.
To date, none of the seven infused participants has reported a serious adverse event, including no factor VIII inhibitors, no thrombotic events and no factor VIII activity levels that may increase risk of thrombosis.
Please see Dr. George’s presentation, with data graphs, here.
About Hemophilia A
Hemophilia is 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. People living with hemophilia are at risk of excessive and recurrent bleeding spontaneously and from modest injuries, which have the potential to be life threatening. There are approximately 15,000 people with hemophilia A in the U.S. and 19,000 in the five major European countries. Hemophilia A is about four times as common as hemophilia B. Hemophilia A is characterized by mutations in the factor VIII gene (F8), which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. The current standard of care for hemophilia A requires recurrent intravenous infusions of either plasma-derived or recombinant factor VIII to control and prevent bleeding episodes. There exists a significant need for novel therapeutics to treat people living with hemophilia.
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. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘predict,’’ ‘‘will,’’ ‘‘would,’’ ‘‘could,’’ ‘‘should,’’ ‘‘continue’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. 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): the factor VIII expression levels seen in our trial participants and the reduction in ABR and AIR may not be maintained; (ii) our lead SPK-FVIII product candidate, SPK-8011, may not produce sufficient data in our Phase 1/2 clinical trial to warrant further development; and (iii) any one or more of our product candidates in preclinical or clinical development will not successfully be developed and commercialized. 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 Securities and Exchange Commission. All information in this press release is as of the date of the release, and Spark undertakes no duty to update this information unless required by law.
Source: Spark Therapeutics, Inc.