About Ganaxolone

Ganaxolone, our lead clinical stage drug candidate, possesses enhanced activity at receptors for the neurotransmitter GABA (gamma-aminobutyric acid), the major regulator of excessive activity of neurons in the brain. We believe it may have the potential to modulate the abnormalities that underlie some of the most severe seizure disorders.

Ganaxolone – A Novel GABAA Receptor Modulator

Our goal is to maximize the potential of ganaxolone to address areas of unmet medical need where its mechanism can have the greatest impact, including a range of seizure disorders.

The Right Formulation

Marinus is developing ganaxolone formulations, both IV and oral, to maximize its therapeutic potential in both
acute care and outpatient settings for adult and pediatric patients.  

A Wide Range of Conditions

Marinus has clinical studies with ganaxolone in children with rare genetic epilepsies, including CDKL5 deficiency disorder and tuberous sclerosis complex. The company is also conducting a trial in refractory status epilepticus, a neurologic emergency with prolonged, continuous or near-continuous seizures that have failed to respond to currently available therapies. Additionally, the company plans to conduct a Phase 2 trial of adjuvant use of ganaxolone in established status epilepticus (ESE).

Ganaxolone has been studied in more than 1,800 patients, both children and adults

How Ganaxolone Works

Ganaxolone is a GABAA receptor modulator that acts by regulating brain activity. This process can include inhibiting the abnormal electrical discharges that cause seizures and status epilepticus or restoring balance in disrupted neuronal activity in other CNS disorders.

Because ganaxolone modulates both synaptic and extrasynaptic GABAA receptors and is unique relative to other approved antiepileptic drugs, we believe it has the potential to treat seizures resistant to other available medications. These binding sites, including the extrasynaptic GABAA receptor, may also be important in treating status epilepticus and other brain disorders.


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 chloride ions through a pore on the cell surface. This action changes the positive and negative charges inside and outside the cell, which inhibits the firing of the neuron.

Ganaxolone is an analogue of the naturally occurring neurosteroid, allopregnanolone. Neurosteroids do not possess the metabolic effects of other steroids; rather, they regulate the balance of excitatory and inhibitory brain activity.  Ganaxolone has been designed with an added methyl group to prevent back-conversion to an active steroid intended to better maintain its specific neuromodulatory activity.