This morning Marinus Pharmaceuticals Inc. (MRNS:NASDAQ), a pharmaceutical firm dedicated to the development of innovative therapeutics to treat epilepsy, depression and other neuropsychiatric disorders, announced that it has completed the top-line analysis of the open-label, dose-finding Phase 2 study evaluating intravenous (IV) ganaxolone in patients with refractory status epilepticus (RSE).
The company reported that ganaxolone met the primary endpoint in the study with none of the 17 enrolled patients progressing to IV anesthetics within 24 hours of treatment initiation and that ganaxolone had an acceptable safety and tolerability profile for the RSE patient population in all dose groups. Marinus further advised that the data has been accepted for an oral platform presentation at the Neurocritical Care Society meeting in Vancouver, Canada, scheduled for October 16, 2019.
Lawrence Hirsch, M.D., professor of neurology, chief, Division of Epilepsy and EEG, and co-director of the Yale Comprehensive Epilepsy Center and Critical Care EEG Monitoring Program, commented, "Maintaining control of status epilepticus without progressing to additional IV anesthetics or antiepileptic drugs (AEDs) is a remarkable clinical response to IV ganaxolone treatment. This outcome is particularly impressive given all study participants, with diverse disease etiology, had failed up to four prior AEDs. The rapid onset of activity is equally important in this patient population as a number of published studies support that stopping status earlier is a key factor in improving both short and long-term patient outcomes."
Dr. Scott Braunstein, CEO of Marinus, added, "All patients who received the target dose of ganaxolone, regardless of underlying cause of status, experienced status cessation without progression to additional second line AEDs or third line anesthesia...These data give us confidence in our IV dose selection as we make preparations for advancing into Phase 3 with our first hospital-based ganaxolone indication."
Marinus advised that it is making preparations for an End-of-Phase 2 meeting with the FDA to discuss next steps towards a potential Phase 3 pivotal study and it is also evaluating a new ready-to-use dose formulation that does not require compounding, mixing or diluting, which would enable immediate administration at the bedside and ensure patients receive the benefits of treatment as rapidly as possible. The new formulation will be evaluated in a few additional patients and the data will be included in the End-of-Phase 2 meeting anticipated to take place in Q1/20.
The firm explains that 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. SE is believed to affect approximately 75,000 patients yearly in the U.S. and 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.
Marinus Pharmaceuticals is headquartered in Radnor, Penn., and describes itself as a pharmaceutical company dedicated to improving the lives of patients suffering from epilepsy and depression. The firm is developing ganaxolone, which it claims offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing. The firm indicates that 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 RSE.
MRNS shares opened much higher today at $1.94 (+$0.57, +41.61%) over yesterday's $1.37 closing price. The stock has traded today between $1.49 and $2.00/share and closed the day at $1.69 (+$0.32, +23.36%) on more than 25 times average daily volume.[NLINSERT]
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