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Zogenix Announces Top-Line Results From Third Pivotal Phase 3 Trial Of FINTEPLA In Dravet Syndrome; Says 'Results corroborate highly statistically significant convulsive seizure reductions seen in earlier multinational Phase 3 studies of FINTEPLA'

Results corroborate highly statistically significant convulsive seizure reductions seen in earlier multinational Phase 3 studies of FINTEPLA in Dravet syndrome FINTEPLA at 0.7 mg/kg/day achieved

· 09/10/2020 08:02
  • Results corroborate highly statistically significant convulsive seizure reductions seen in earlier multinational Phase 3 studies of FINTEPLA in Dravet syndrome
  • FINTEPLA at 0.7 mg/kg/day achieved a 64.8% greater reduction in mean monthly convulsive seizures compared to placebo (p<0.0001) and FINTEPLA at a lower dose of 0.2 mg/kg/day achieved a 49.9% greater reduction in mean monthly convulsive seizures compared to placebo (p<0.0001)
  • Positive study will support Japanese new drug application (J-NDA) submission, planned for 2021

EMERYVILLE, Calif., Sept. 10, 2020 (GLOBE NEWSWIRE) -- Zogenix (NASDAQ:ZGNX), a global biopharmaceutical company developing and commercializing rare disease therapies, today reported positive top-line results from its third Phase 3 study (Study 3) of FINTEPLA® (fenfluramine) oral solution for the treatment of seizures associated with Dravet syndrome. The study corroborates the substantial impact of FINTEPLA on convulsive seizure reduction previously demonstrated in two earlier Phase 3 trials (Studies 1 and 2) in patients with this severe, rare and often debilitating form of infant-onset epilepsy. It also expands the countries where FINTEPLA has been evaluated to include Japan and Study 3 will be the pivotal study included in the Company's planned submission of a new drug application (J-NDA) in that country, expected to occur in 2021.

"There remains a substantial unmet need in the Dravet treatment landscape globally and these compelling results corroborate the substantial levels of seizure control provided by FINTEPLA in Dravet syndrome that was also demonstrated in Studies 1 and 2," said Stephen J. Farr, Ph.D., President & CEO of Zogenix. "Importantly, Study 3 included subjects from Japan and, based on our prior discussions with Japan's Pharmaceuticals and Medical Devices Agency (PMDA), should meet the requirements to serve as the pivotal study for a J-NDA submission. We are excited to work with our commercialization partner in Japan, Nippon Shinyaku, to leverage their expertise and commitment to rare diseases to provide FINTEPLA, if approved, as a potential new treatment option for patients and their families. On behalf of everyone at Zogenix, I would like to extend sincere gratitude to the patients, families and investigators involved in this study. With FINTEPLA now commercially available in the U.S. and under regulatory review in Europe, we are excited to continue our work to bring FINTEPLA to patients in additional countries over time."

Study 3 was a multi-national, randomized, double-blind, placebo-controlled, Phase 3 study enrolling 143 children and young adults with Dravet syndrome, whose seizures were not adequately controlled by existing anti-epileptic drugs. The median age of patients was 9 years (range, 2-18 years) and the average baseline convulsive seizure frequency across the study groups was approximately 63 seizures per month.

Following a six-week baseline observation period, patients were randomized to one of three treatment groups: FINTEPLA 0.7 mg/kg/day (26 mg maximum daily dose; n=49), FINTEPLA 0.2 mg/kg/day (n=46) or placebo (n=48), in which FINTEPLA or placebo was added to each patient's current treatment regimen of anti-epileptic drugs. Patients were titrated to their target dose of FINTEPLA over two weeks and then remained at that fixed dose for 12 weeks.

The study 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). The median percent reduction in monthly convulsive seizure frequency was 73.7% among FINTEPLA 0.7 mg/kg/day patients compared to 7.6% in placebo patients.

The same analyses comparing FINTEPLA at a lower dose of 0.2 mg/kg/day versus placebo was a key secondary objective and demonstrated that patients in the lower dose group achieved a 49.9% greater reduction in mean monthly convulsive seizures compared to placebo (p<0.0001). Collectively, these top-line data are highly consistent with the results of Study 1 in demonstrating a dose-response relationship for FINTEPLA in the treatment of convulsive seizures in Dravet syndrome.

"Dravet syndrome is a rare, highly refractory form of childhood onset epilepsy marked by frequent and often prolonged seizures that are difficult to control with existing medications," said Joseph Sullivan, M.D., Professor of Neurology & Pediatrics and Director of the Pediatric Epilepsy Center of Excellence at the UCSF Benioff Children's Hospitals, and the Principal Investigator for FINTEPLA in Dravet syndrome."Given the profound reductions in convulsive seizure frequency seen across clinical studies, combined with the ongoing, robust safety monitoring that will be part of this medicine's use, I continue to believe that it will offer an extremely important treatment option for Dravet syndrome patients, and bring new hope to families around the world living with the severe effects of this disease."

Additional key secondary objectives of the study were to compare FINTEPLA 0.7 mg/kg/day and 0.2 mg/kg/day (independently) with placebo in terms of (1) the proportion of patients who achieved ≥50% reductions in monthly convulsive seizures and (2) the median of the longest convulsive seizure-free interval. These results are shown in the following table. The proportion of patients who achieved ≥75% seizure reductions, a secondary efficacy measure, is also presented.