Zogenix Submits New Drug Application for FINTEPLA® (Fenfluramine) in Japan for the Treatment of Epileptic Seizures Associated with Dravet Syndrome

EMERYVILLE, Calif., Dec. 21, 2021 (GLOBE NEWSWIRE) -- Zogenix, a global biopharmaceutical company specializing in rare disease therapies, announced that it has submitted a New Drug Application (J-NDA) to the Japanese Ministry of Health, Labour & Welfare (MHLW) for the marketing approval of FINTEPLA® (fenfluramine) for the treatment of epileptic seizures associated with Dravet syndrome in Japan. FINTEPLA received Orphan Drug Designation from Japan's Ministry of Health, Labour & Welfare (MHLW) in August 2021.

"There remains a substantial unmet need in the Dravet syndrome treatment landscape globally, and Japan is no exception where patients continue to experience refractory seizures that negatively impact quality of life," said Stephen J. Farr, Ph.D., President and CEO of Zogenix. "We are committed to advancing FINTEPLA as a potential treatment option to ensure that patients in Japan living with this rare, highly refractory form of childhood-onset epilepsy have access to this therapy."

The submission is supported by the results of Study 3, a multi-national randomized, double-blind, placebo-controlled, Phase 3 study, of 143 children and young adults with Dravet syndrome, including trial participants from Japan, whose seizures were not adequately controlled by existing anti-epileptic drugs. Study 3 met its primary objective in demonstrating that patients in the FINTEPLA 0.7 mg/kg/day group achieved a 64.8% greater reduction in mean monthly convulsive seizures compared to the placebo group (p<0.0001). FINTEPLA was generally well-tolerated in this study, with adverse events consistent with those observed in previous Phase 3 studies, Study 1 and Study 2, and with the known safety profile of fenfluramine.

Zogenix retains responsibility for completing its global clinical development programs and seeking regulatory approval for FINTEPLA in Japan for Dravet syndrome and Lennox Gastaut syndrome. If approved in Japan, FINTEPLA will be commercialized in Japan through Zogenix's exclusive distribution agreement with Nippon Shinyaku, Co., Ltd., a leading Japanese pharmaceutical product developer and distributor.

About Dravet Syndrome

Dravet syndrome is a rare, devastating and life-long form of epilepsy that generally begins in infancy or early childhood and is marked by frequent treatment-resistant seizures, frequent resulting hospitalizations and medical emergencies, significant developmental, motor, and behavioural impairments, and an increased risk of sudden unexpected death in epilepsy (SUDEP). There are an estimated 3,000 Dravet syndrome patients in Japan based on the MHLW Patient Survey. Most patients follow a course of developmental delay with cognitive, motor, and behavioural deficits that persist into adulthood. Dravet syndrome severely impacts the quality of life for patients, families, and caregivers due to the high physical, emotional, caregiving, and financial burden associated with the disease.

About FINTEPLA^®

FINTEPLA^® (fenfluramine) oral solution is a prescription medication used to treat seizures associated with Dravet Syndrome in patients two years of age and older. FINTEPLA possesses dual activities to inhibit seizures: as a serotonergic agent, acting as a potent 5-HT releaser with agonist activity at 5-HT1D, 2A, and 2C receptors, and as a positive modulator of Sigma1R. FINTEPLA is approved in the U.S. and Europe for the treatment of seizures associated with Dravet syndrome. The U.S. Food and Drug Administration (FDA) has accepted for filing the company's supplemental New Drug Application (sNDA) and granted Priority Review for the use of FINTEPLA for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS).

Across multiple clinical studies, FINTEPLA demonstrated significant and sustained reduction of convulsive seizures associated with Dravet Syndrome. In three pivotal Phase 3 clinical trials, the reduction in convulsive seizure frequency per 28 days was statistically different for all dose groups of fenfluramine compared to placebo.

About Zogenix

Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases. The company's first rare disease therapy, FINTEPLA^® (fenfluramine) oral solution, has been approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency and is in development in Japan for the treatment of seizures associated with Dravet syndrome, a rare, severe lifelong epilepsy. The U.S. FDA recently accepted for filing Zogenix's supplemental New Drug Application (sNDA) and granted Priority Review for the use of FINTEPLA for the treatment of seizures associated with an additional rare epilepsy, Lennox-Gastaut syndrome (LGS). Zogenix also plans to initiate a study of FINTEPLA in a genetic epilepsy called CDKL5 Deficiency Disorder (CDD) and is collaborating with Tevard Biosciences to identify and develop potential next-generation gene therapies for Dravet syndrome and other genetic epilepsies. The company has an additional late-stage development program, MT-1621, in a mitochondrial disease called TK2 deficiency.

About Nippon Shinyaku

Our mission is to help people lead healthier and happier lives. Through creating unique medicines that will bring hope to patients and families struggling with illness, we aim to be an organization trusted by the community. Please visit our website (http://www.nippon-shinyaku.co.jp/english/) for products or detailed information.

Forward-Looking Statement

Zogenix 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 include: the timing and ability of Zogenix to complete regulatory submission in the EU and Japan for its product candidates; the expected timing of reporting data from clinical trials; the expected timing of review of Zogenix's regulatory submissions including the sNDA for the treatment of seizures associated with LGS; Zogenix's commercialization plans in the U.S. and Europe; and Zogenix's plans with respect to its development programs. 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 due to the risks and uncertainties inherent in Zogenix's business, including, without limitation: FINTEPLA may not achieve broad market acceptance as a treatment option of seizures associated with LGS or Dravet syndrome which would limit Zogenix's ability to general revenues; Zogenix may not be successful in executing its sales and marketing strategy for the commercialization of FINTEPLA in the U.S. and Europe, including due to the costs and procedures related to the REMS certification process or controlled access program; the COVID-19 pandemic may continue to disrupt Zogenix's business operations, impairing the ability to commercialize FINTEPLA in the U.S. and Europe and Zogenix's ability to generate product revenue in the U.S. and Europe and conduct its development programs; unexpected adverse side effects or inadequate therapeutic efficacy of fenfluramine that could limit regulatory approval or commercialization, or that could result in recalls or product liability claims; later developments with FDA that may be inconsistent with the already completed meetings; additional data from Zogenix's ongoing studies may contradict or undermine the data previously reported; the potential for the FDA to delay timing of review of the sNDA due to the FDA's internal resource constraints or other reasons; and other risks described in Zogenix's prior press releases as well as in public periodic filings with the U.S. Securities & 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.

CONTACTS:

Zogenix

[email protected]

Investor

Brian Ritchie

Managing Director, LifeSci Advisors LLC

+1 (212) 915-2578 | [email protected]

Japan

Fleishman-Hillard Japan K.K.

Tomomi Sasaki

+81 080-1010-7812 | [email protected]