Pfizer Initiates Pivotal Phase 3 Program for Investigational Hemophilia B Gene Therapy
Phase 3 lead-in study initiated following completion of the transfer of Spark Therapeutics’ hemophilia B gene therapy program to Pfizer
The Phase 3 program was initiated following the transfer of the responsibility for Spark Therapeutics’ hemophilia B gene therapy program to Pfizer. Fidanacogene elaparvovec is a novel, investigational vector that contains a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity human coagulation factor IX gene. It is hoped that, once treated, patients will be able to produce factor IX themselves, rather than having to regularly inject factor IX.
"With the lead-in study now open and actively recruiting patients, we are excited to begin our Phase 3 program evaluating fidanacogene elaparvovec for the treatment of hemophilia B,” said
“We are pleased to have transitioned fidanacogene elaparvovec to Pfizer following the positive results of the ongoing Phase 1/2 clinical trial,” said
About the Pfizer and Spark Therapeutics Agreement
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, or rarely into other critical closed spaces such as the intracranial space, where bleeding can be fatal. 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.
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Spark Therapeutics Cautionary note on forward-looking statements
This press 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) SPK-9001 may not free patients with hemophilia B of the need for regular infusions, while eliminating spontaneous bleeding 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
DISCLOSURE NOTICE: The information contained in this release is as of
This release contains forward-looking information about fidnacogene elparvovec, the fidnacogene elparvovec program and the License Agreement between Pfizer and Spark, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical study commencement and completion dates as well as the possibility of unfavorable study results, including unfavorable new clinical data and additional analyses of existing clinical data; risks associated with initial data, including the risk that the final results of the fidnacogene elparvovec lead-in study and the subsequent Phase 3 study and/or additional clinical trials may be different from (including less favorable than) the initial data results and may not support further clinical development; the risk that clinical trial data are subject to differing interpretations, and, even when we view data as sufficient to support the safety and/or effectiveness of a product candidate, regulatory authorities may not share our views and may require additional data or may deny approval altogether; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any applications may be filed with regulatory authorities for fidnacogene elparvovec; whether and when regulatory authorities may approve any such applications, which will depend on the assessment by such regulatory authorities of the benefit-risk profile suggested by the totality of the efficacy and safety information submitted and, if approved, whether fidnacogene elparvovec will be commercially successful; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of fidnacogene elparvovec; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended