Marinus Pharmaceuticals, a pharmaceutical company 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) SBECD-enabled ganaxolone in patients with refractory status epilepticus (RSE). Ganaxolone met the primary endpoint in the study with no patients (n=17) progressing to IV anesthetics within 24 hours of treatment initiation. Ganaxolone had an acceptable safety and tolerability profile for the RSE patient population in all dose groups. 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.
Marinus is making preparations for an End-of-Phase 2 meeting with the FDA to discuss next steps towards a potential Phase 3 pivotal study. In parallel, the Company is evaluating a new ready-to-use dose formulation that does not require compounding, mixing or diluting. This will enable immediate administration at the bedside and ensure patients receive the benefits of treatment as rapidly as possible. This new formulation will be evaluated in a few additional patients with data to be included in the End-of-Phase 2 meeting, anticipated in Q1 2020.
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 release 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.