About Ganaxolone

Dedicated to the Development of Ganaxolone

A new mechanism of action designed with selectivity and safety in mind.

Ganaxolone, our lead clinical stage drug candidate, brings a new GABAA receptor modulating mechanism and an extensive safety database with exhibited antiepileptic (anti-seizure) and anxiolytic (anti-anxiety) activity.

Our goal is to maximize the value of ganaxolone as a first-in-class innovative neuropsychiatric therapy with a portfolio of diversified indications to treat drug-resistant seizures and neuropsychiatric disorders.

right treatment

The Right
Treatment

Ganaxolone, a positive allosteric modulator of GABAA, acts on a well-characterized synaptic and extrasynaptic target known for antiepileptic (anti-seizure) and anxiolytic (anti-anxiety) activity.

right formulation

The Right
Formulation

Marinus is developing three different convenient dose forms of ganaxolone—IV, capsule, and liquid—to maximize the therapeutic reach to adult and pediatric patients in both acute and chronic care settings.

cdkl5 deficiency disorder

A Wide Range
of Conditions

Marinus has initiated a pivotal ganaxolone study in children with rare, CDKL5 deficiency disorder and is currently conducting studies in postpartum depression and refractory status epilepticus.

healthy mom happy children

 

Extensive Safety Database

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 two years. In these studies, ganaxolone was generally safe and well tolerated, with the most commonly reported adverse events of somnolence, dizziness, and fatigue.

Find a Clinical Trial

ganaxolone study

Ganaxolone has been studied in more than 1,600 subjects, both pediatric and adult

How Ganaxolone Works

Ganaxolone is a GABAA modulator which has been shown to be an effective anticonvulsant by restoring electrical balance to the seizing brain.

While allopregnanolone’s GABAA modulatory activity is well documented, allopregnanolone has the potential to convert back to its metabolic precursor, progesterone, which could lead to hormonal side effects. Ganaxolone has been designed with an added methyl group that prevents back conversion to an active steroid, which unlocks ganaxolone’s potential for chronic use. In preclinical studies, ganaxolone exhibited potency and efficacy comparable to allopregnanolone.

GABA (gamma-aminobutyric acid) is the chief inhibitory neurotransmitter in the brain. One of the subclasses of receptors that respond to GABA is the GABAA receptor. When activated, these receptors selectively conduct chloride ions through a pore that results in the inhibitory effect of hyperpolarization of the neuron. Synaptic GABAA receptors respond quickly to inhibit neurotransmission, while extrasynaptic GABAA receptors provide ambient tonic inhibition.

Both ganaxolone and allopregnanolone bind to GABAA at the synaptic and extrasynaptic binding sites. Activity with extrasynaptic GABAA receptors is of particular importance for treating patients who developed tolerance to benzodiazepines and barbiturates. Ganaxolone binds to the GABAA receptors, which opens the pore to allow chloride ions to move into the postsynaptic neuron, leading to the inhibition of neurotransmission.

allopregnanolone model

ganaxolone model

ganaxolone mindful innovation

Mindful Innovation to Make a Difference

See how Marinus is developing ganaxolone to improve the lives of patients with epilepsy and neuropsychiatric disorders.

Explore Our Pipeline

Patented Innovation

Years of in-house research at Marinus resulted in the invention of a patented nanoparticulate composition for solid and liquid oral dose forms of ganaxolone that are highly stable and provide good exposure. This discovery resulted in the issuance of U.S. and foreign patents covering use of these complex ganaxolone nanoparticles in solid and liquid dose forms.

The nanoparticulate composition patents for oral liquid and solid dose forms of ganaxolone are expected to provide intellectual property protection through at least 2026. This application identified a novel process using an organometallic methylating agent, which is significantly more cost effective in the manufacture of ganaxolone than previous processes. This synthesis patent is expected to provide intellectual property protection through at least 2030.