Zogenix Announces Initial Quality of Life and Cognitive Function Data From Phase 3 Trial of ZX008 in Dravet Syndrome

ZX008 Patients Experienced Significant Improvements on Select Measures of Quality of Life and Executive Function Compared to Those on Placebo

Data Presented During Emerging Therapies Plenary Session at 2018 American Academy of Neurology Annual Meeting

EMERYVILLE, Calif., April 27, 2018 (GLOBE NEWSWIRE) -- Zogenix, Inc. ZGNX, a pharmaceutical company developing therapies for the treatment of rare central nervous system (CNS) disorders, today announced that Kelly Knupp, M.D., Associate Professor, Pediatrics-Neurology, Children's Hospital Colorado, presented new encouraging preliminary quality of life (QoL) and cognitive function data from the Company's first Phase 3 trial (Study 1) of its investigational drug, ZX008 (low-dose fenfluramine), for the adjunctive treatment of seizures associated with Dravet syndrome. The data were presented at the American Academy of Neurology Annual Meeting, as part of a broader session entitled: "Neurology Year in Review: Emerging Therapies Plenary Session."

As previously reported, Study 1 randomized patients to one of three treatment groups: ZX008 0.8 mg/kg/day (30 mg maximum daily dose; n=40), ZX008 0.2 mg/kg/day (n=39) and placebo (n=40). Patient quality of life was assessed at baseline and during the study using two scales: the Pediatric Quality of Life Inventory (PEDS-QL) and the Quality of Life in Childhood Epilepsy (QOLCE). For patients 5 years and older, (n=32, 31 and 30 for the ZX008 0.8 mg/kg/day, ZX008 0.2 mg/kg/day and placebo groups, respectively) cognitive function was assessed using the Behavior Rating Inventory of Executive Function (BRIEF).

Both ZX008 treatment groups experienced positive effects on physical and psychosocial health resulting in a significant improvement as compared to placebo in health-related quality of life based on PEDS-QL Total Score (p<0.05). The PEDS-QL is an evaluation of health-related QoL, completed by the parent/caregiver and measuring physical, emotional, social, and school functioning of the child.   

The BRIEF measures cognitive processes, such as anticipation, judgement, self-awareness and decision making, which are also referred to as executive functions. In Study 1, following 14 weeks of treatment (titration plus maintenance periods), patients in the ZX008 0.8 mg/kg/day treatment group experienced significant improvement on the BRIEF Global Executive Composite Index Score (p<0.05), a summary score of all eight BRIEF clinical scales, useful as a measure in evaluating overall executive function.

There were no significant differences noted on QOLCE, an assessment completed by a parent or caregiver that evaluates how epilepsy affects day-to-day functioning of their child in various life areas, including physical activities, well-being, cognition, social activities, behavior, and general health.

"Neurological comorbidities, such as developmental delay, cognitive impairment and behavioral problems, are common significant issues for patients with Dravet syndrome and their caregivers," said Dr. Knupp. "Measuring these non-seizure endpoints and understanding how treatments affect them is important when assessing new treatment interventions.  Any improvements in quality of life and cognitive function would provide further benefit to these patients and their caregivers. The reduction in seizure frequency demonstrated by ZX008 in Study 1 was compelling, while these initial improvements in some quality of life and cognitive function measures are encouraging and warrant further evaluation."

"We believe that these initial data showing improvements in quality of life and executive function are promising and, when further results are available, could enhance the already encouraging efficacy profile of ZX008," said Gail Farfel, Ph.D., Executive Vice President and Chief Development Officer of Zogenix.  "We look forward to having the full Phase 3 dataset to better understand the potential for ZX008 to impact endpoints that go beyond reducing seizures in Dravet syndrome."

About Study 1
The randomized, double blind, placebo controlled, Phase 3 study enrolled 119 patients across sites in the U.S., Canada, Europe and Australia. The median age of patients was 8 years (range, 2-18 years). Following a six-week baseline observation period, patients were randomized to one of three treatment groups: ZX008 0.8 mg/kg/day (30 mg maximum daily dose; n=40), ZX008 0.2 mg/kg/day (n=39) and placebo (n=40) in which ZX008 or placebo was added to current regimens of antiepileptic drugs. Patients were titrated to their target dose over two weeks and then remained at that fixed dose for 12 weeks. The mean baseline convulsive seizure frequency across the study groups was approximately 40 seizures per month.

As previously reported, Study 1 met its primary objective demonstrating that ZX008, at a dose of 0.8 mg/kg/day, is superior to placebo as adjunctive therapy in the treatment of Dravet syndrome in children and young adults based on change in the frequency of convulsive seizures between the 6-week baseline observation period and the 14-week treatment period (p<0.001). The randomized, double blind, placebo controlled, Phase 3 study enrolled 119 patients.  Following a six-week baseline observation period, patients were randomized to one of three treatment groups: ZX008 0.8 mg/kg/day (30 mg maximum daily dose; n=40), ZX008 0.2 mg/kg/day (n=39) and placebo (n=40) in which ZX008 or placebo was added to current regimens of antiepileptic drugs.

About Zogenix
Zogenix ZGNX is focused on developing therapies for patients with rare central nervous system (CNS) conditions that have limited or no treatment options but face a critical need. For more information, visit www.zogenix.com.

Forward Looking Statements
Zogenix cautions you that statements included in this press release or in the poster presentations 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 forward-looking statements include statements regarding: ZX008's potential as a treatment for seizures associated with Dravet syndrome; and the potential for quality of life and cognitive function data to impact the efficacy profile of ZX008. These statements are based on Zogenix's current beliefs and expectations. 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 or in any poster presentation due to the risks and uncertainties inherent in Zogenix's business, including, without limitation: the FDA may not agree with Zogenix's interpretation of the results of the Study 1 and other data; the uncertainties associated with the clinical development and regulatory approval of product candidates such as ZX008; 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; 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 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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