Zogenix Announces New Efficacy and Safety Data on ZX008 for Treatment of Seizures in Dravet Syndrome
"In this new cohort of patients, we continue to achieve meaningful seizure control in Dravet syndrome patients using low-dose fenfluramine as adjunctive treatment," said Professor
"The improvement in seizures and cardiovascular-related safety data from this ongoing open-label study continues to be quite compelling," said
The data presented highlight the initial results from a new cohort of 7 Dravet syndrome patients who began add-on treatment with low-dose fenfluramine (5 mg to 15 mg per day) at various starting points between 2010 and 2014. Median treatment duration was 0.9 years (range 0.2 to 3.9 years). During the 90-day run-in period prior to initiating low-dose fenfluramine treatment, the median frequency of tonic-clonic seizures was 3.0 per month (range 0.4 to 39.7). At the 6-month evaluation after starting low-dose fenfluramine treatment, the median frequency of tonic-clonic seizures was 1.2 per month, and the median decrease was 73% (range 48-100%). Over the entire observation period, the median frequency of tonic-clonic seizures was 0.9 per month, and the median decrease was 84% (range 55-100%).
A separate poster reported that during this observation period from 2010 to 2015, treatment with low-dose fenfluramine was generally well-tolerated, and in this new cohort of patients, treatment for periods of 0.9 to 3.9 years did not result in any echocardiographic or clinical signs of cardiac valve abnormalities, pulmonary hypertension or any other cardiovascular abnormalities. The most common treatment-related adverse events were mild-to-moderate somnolence (n=6) and anorexia (n=4). There were no fenfluramine discontinuations due to adverse events or lack of effect.
The observed effectiveness, tolerability and cardiovascular-related safety with add-on, low-dose fenfluramine in this new cohort of Dravet syndrome patients extends the findings previously reported in the original cohort in 2012¹.
In addition, a separate, recently published study (see study data here) evaluated the mechanism of action for fenfluramine as a treatment for Dravet syndrome using a gene knockout zebrafish model. As a result of this study, certain 5-HTsubtype receptors that appear to be involved in the mechanism-of-action of fenfluramine were identified. Specifically, the elevation of serotonin levels and interaction with three 5-HT receptor subtypes, 5-HT1D, 5-HT2A and 5-HT2C, were found to be responsible for reducing both abnormal motor behavior and brain activity in this model of Dravet syndrome.
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About Dravet Syndrome
Dravet syndrome (also known as Severe Myoclonic Epilepsy of Infancy) is a rare, severe and therapy-resistant form of epilepsy most often caused by an identifiable gene defect that results in abnormal functioning of a sodium channel in the brain. Children with Dravet syndrome experience severe, long-lasting, fever-related seizures in the first year of life. Other seizures typically arise later, including myoclonus (involuntary muscle spasms) and status epilepticus (prolonged seizures), which often result in severe cognitive and developmental impairment. Episodes of status epilepticus require immediate emergency care and can be fatal.
Individuals with Dravet syndrome face a higher incidence of SUDEP (sudden unexplained death in epilepsy) and have associated conditions, which also require proper treatment and management. Children with Dravet syndrome do not outgrow this condition and it affects every aspect of their daily lives.
Forward Looking Statements
1Ceulemans, Berten, et al. Successful use of fenfluramine as an add-on treatment for Dravet syndrome. EPILEPSIA. July 2012; 53(7):1131-1139.
Andrew McDonaldFounding Partner, LifeSci Advisors LLC646-597-6987 | Andrew@lifesciadvisors.com