- Annual Meeting
CEO: Christopher M. Cashman
Marinus Pharmaceuticals, Inc. (NASDAQ: MRNS) is a biopharmaceutical 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 neuropsychiatric disorders. Ganaxolone is a positive allosteric modulator of GABAA that acts on a well-characterized target in the brain known to have both anti-seizure and anti-anxiety effects. Ganaxolone is being developed in three different dose forms (IV, capsule and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Marinus is preparing to initiate a pivotal study in children with CDKL5 deficiency disorder (CDD), a rare form of epilepsy, and currently conducting studies in patients with postpartum depression and refractory status epilepticus.
In clinical studies, ganaxolone has shown both anti-seizure as well as anti-anxiety activity. This dual benefit has the potential to address both the seizures and behavioral co-morbidities associated with a variety of orphan, genetic disorders. Top-line data from a Phase 2 open-label study in patients with CDD showed that oral ganaxolone, in addition to background treatment, provided a reduction in seizures and increase in seizure free days in the majority of patients, with some reporting behavioral benefits. In a Phase 2 study in children with Fragile-X syndrome that had a high level of anxiety at baseline, ganaxolone showed benefits over placebo in anxiety, attention and hyperactivity. The anti-anxiety benefit is being furthered explored in the Phase 2 studies in women with severe and moderate postpartum depression.
Ganaxolone is generally safe and well-tolerated. More than 1,600 children and adults have received treatment with ganaxolone ranging in duration from one day to more than two years. The most common side effects are somnolence, dizziness and fatigue.
Lisa M. Caperelli
Executive Director, Investor & Strategic Relations