Marinus Pharmaceuticals, Inc. a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy, depression and other neuropsychiatric disorders, announced additional data from its open-label, dose-finding Phase 2 study evaluating intravenous (IV) ganaxolone in patients with refractory status epilepticus (RSE). The results were presented at the American Epilepsy Society (AES) annual meeting by Eugene Ramsay, M.D., Director Emeritus, Ochsner Comprehensive Epilepsy Center in New Orleans, and investigator in the study.
Key Presentation Highlights:
- Previously announced study results demonstrated that 100% of patients achieved the primary endpoint of preventing progression to IV anesthetics within 24 hours of treatment initiation. Additional long-term data, presented for the first time at AES, demonstrate that all evaluable patients (n=6) in the target dose cohort did not experience status relapse during the four-week follow up period.
- Efficacy and safety were consistent across disease etiologies and both nonconvulsive status epilepticus (NSCE) and convulsive status epilepticus (CSE) patients.
- Baseline patient characteristics showed that in the 17 patients enrolled in the study, 7 had RSE resulting from a vascular condition, 4 cases were caused by tumors, 2 by autoimmune disease and 2 by drug overdose/withdrawal. The underlying cause in one patient was unknown. Seven of the patients had a prior history of epilepsy.
- Patients failed a mean of 2.1 second-line IV AED with all patients failing both levetiracetam and lacosamide. All prior AEDs were given at therapeutically relevant doses, administered within recommended dosing guidelines and mean time since addition of most recent AED was 4 hours.
Status epilepticus (SE) is a rare epileptic seizure of prolonged duration, lasting more than five minutes for convulsive seizures or 30 minutes for non-convulsive. It is believed to affect approximately 75,000 patients in the U.S. each year. SE is a true medical emergency that can cause permanent damage to the brain and even death if not quickly brought under control. SE is first treated with IV benzodiazepines then IV anti-epileptic drugs. Patients that fail second line treatment with IV AEDs, are considered to be in refractory status epilepticus (RSE). There are currently no indicated treatments for refractory RSE. The primary treatment goals of clinicians treating SE are; to stop the patient from seizing immediately, assess and resolve the underlying medical condition, and move the patient towards discharge from the hospital as quickly as possible.
Ganaxolone, a positive allosteric modulator of GABAA, is being developed in three different dose forms (intravenous, capsule and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Unlike benzodiazepines, ganaxolone exhibits anti-seizure and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors. Ganaxolone has been studied in more than 1,600 subjects, both pediatric and adult, at therapeutically relevant dose levels and treatment regimens for up to four years. In these studies, ganaxolone was generally safe and well-tolerated. The most commonly reported adverse events were somnolence, dizziness and fatigue.
About Marinus Pharmaceuticals
Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA that acts on a well-characterized target in the brain known to have anti-seizure, anti-depressant and anti-anxiety effects. Ganaxolone is being developed in IV and oral dose forms intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Marinus is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy and has recently released top-line data from Phase 2 studies in women with postpartum depression and patients with refractory status epilepticus. For more information visit www.marinuspharma.com.