Study 1504 was initially to be conducted only in Europe, but was recently expanded to include sites in the U.S. and Canada due to significant investigator interest as they consider stiripentol an important medication in the available treatment armamentarium for seizure control in Dravet syndrome. Importantly, Study 1504 meets the requirements of a Phase 3 pivotal trial in the U.S."We are pleased to initiate the efficacy and safety portion of this important Phase 3 study," said Professor Rima Nabbout, M.D. Ph.D., Department of Pediatric Neurology, Reference Center for Rare Epilepsies, Necker Enfants Malades Hospital, and Principal Investigator of Study 1504. "There remains a significant unmet need for improved treatments for Dravet syndrome, and based on the data garnered to date, ZX008 has the potential to address this treatment need for refractory patients. Based on the number of patients in Europe currently on a stiripentol regimen, we expect this trial to enroll quickly, and look forward to the availability of data later this year." "The initiation of the efficacy portion of Study 1504 represents another key milestone in Zogenix's ZX008 Dravet syndrome Phase 3 program," said Stephen J. Farr, Ph.D., President and CEO. "Study 1504 is important to our Phase 3 development program for several reasons. Most importantly, because it meets the requirements of a pivotal trial, we have increased optionality around our regulatory strategy for ZX008. In addition, Study 1504 will be used to support orphan exclusivity in Europe." "We are excited to have the opportunity to participate in this study in the U.S.," said Kelly Knupp, M.D., Associate Professor, Pediatrics - Neurology and epileptologist at Children's Hospital Colorado and University of Colorado School of Medicine. "We have seen a significant amount of interest from Dravet families in participating in this study, and look forward to evaluating ZX008 in this underserved patient population. I especially am pleased to assess a potential new medication, ZX008, with the stiripentol regimen that is used in children with Dravet syndrome in our clinic."
About ZogenixZogenix, Inc. (Nasdaq:ZGNX) is a pharmaceutical company committed to developing and commercializing CNS therapies that address specific clinical needs for people living with orphan and other CNS disorders who need innovative treatment alternatives to improve their daily functioning.For more information, visit www.zogenix.com. Forward Looking StatementsZogenix cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "indicates," "will," "intends," "potential," "suggests," "assuming," "designed" and similar expressions are intended to identify forward-looking statements. These statements are based on the company's current beliefs and expectations. These forward-looking statements include statements regarding the structure of the Study 1504 clinical trial, including the number of patients to be enrolled; the ability to use Study 1504 as a pivotal trial for submission to the U.S. Food and Drug Administration, the timing of top-line results from the Phase 3 clinical trial of ZX008 in Dravet syndrome; the ability of ZX008 to treat patients with Dravet syndrome; the potential commercialization of ZX008; and the timing of any submission of a new drug application to the U.S. Food and Drug Administration or comparable market authorization filing in Europe. The inclusion of forward-looking statements should not be regarded as a representation by Zogenix that any of its plans will be achieved. Actual results may differ from those set forth in this release due to the risks and uncertainties inherent in Zogenix's business, including, without limitation: the uncertainties associated with the clinical development and regulatory approval of product candidates such as ZX008, including potential delays in the commencement, enrollment and completion of clinical trials, including Study 1504; the potential that earlier clinical trials and studies may not be predictive of future results; Zogenix's reliance on third parties to conduct its clinical trials, enroll patients, manufacture its preclinical and clinical drug supplies and manufacture commercial supplies of its drug products, if approved; unexpected adverse side effects or inadequate therapeutic efficacy of ZX008 that could limit approval and/or commercialization, or that could result in recalls or product liability claims; Zogenix's ability to fully comply with numerous federal, state and local laws and regulatory requirements, as well as rules and regulations outside the United States, that apply to its product development activities; Fast Track designation may not result in an expedited regulatory review process; the potential for distraction of management related to the transition of management responsibilities; and other risks described in Zogenix's prior press releases as well as in public periodic filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Zogenix undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
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