Marinus Pharmaceuticals is dedicated to developing innovative treatments for severe and rare forms of epilepsy, particularly focusing on conditions such as status epilepticus, tuberous sclerosis complex, and CDKL5 deficiency disorder. The company is advancing its clinical pipeline with ganaxolone, a drug that operates through a validated GABA A mechanism, which has potential applications in both chronic and acute care settings. Marinus is committed to conducting high-quality research and clinical trials to ensure the safety and efficacy of its treatments, collaborating with healthcare professionals, patient advocacy organizations, and families to enhance the lives of those affected by seizure disorders.

A significant aspect of Marinus's strategy involves its partnership with the Biomedical Advanced Research and Development Authority (BARDA), which has awarded the company substantial funding to support its Phase 3 trial for refractory status epilepticus (RSE). This collaboration aims to explore the use of ganaxolone not only for RSE caused by medical conditions but also for potential indications related to nerve agent exposure. The trial's primary endpoints focus on the rapid cessation of seizures and the prevention of treatment escalation, highlighting Marinus's commitment to addressing urgent medical needs in the field of epilepsy treatment.

United States

Marinus Pharmaceuticals, Inc., a clinical stage pharmaceutical company, focuses on developing and commercializing therapeutics to treat rare seizure disorders. Its clinical stage product candidate, ganaxolone, is an allosteric modulator of GABAA, developed in formulations for two routes, including intravenous (IV) and oral for adult and pediatric patient populations in acute and chronic care, and in-patient and self-administered settings. The company’s ganaxolone product candidate, acts at synaptic and extrasynaptic GABAA receptors, a target for its anti-seizure, antidepressant, and anxiolytic potential. It is developing ganaxolone for status epilepticus, cyclin dependent kinase like 5 deficiency disorder, PCDH19-related epilepsy, tuberous sclerosis complex, and postpartum depression. Marinus Pharmaceuticals, Inc. has license agreement with Purdue Neuroscience Company and CyDex Pharmaceuticals, Inc. The company was incorporated in 2003 and is headquartered in Radnor, Pennsylvania.

Central Nervous System (CNS)

Genetic Diseases

Nervous System Diseases

Pathological Conditions

Marinus Pharmaceuticals

Cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder

GABA is the chief inhibitory neurotransmitter in the brain. Ganaxolone binds to one of the receptor subtypes, the GABAA receptor. When this receptor is activated, it increases the movement of &nbsp;chloride ions through a pore on the cell surface. This action changes &nbsp;the positive and negative charges inside and outside the cell, which &nbsp;inhibits the firing of the neuron.
Ganaxolone is an analogue of the naturally occurring neurosteroid, &nbsp;allopregnanolone. Neurosteroids do not possess the metabolic effects of &nbsp;other steroids; rather, they regulate the balance of excitatory and &nbsp;inhibitory brain activity.&nbsp;&nbsp;Ganaxolone has been designed with an added &nbsp;methyl group that prevents back-conversion to an active steroid intended &nbsp;to better maintain its specific neuromodulatory activity.
<a href="https://marinuspharma.com/science-pipeline/about-ganaxolone/">Source</a>

Marinus Pharmaceuticals

Company Details