Its lead candidate, ganaxolone, is a drug designed to exhibit antiepileptic, anxiolytic, antidepressant, and sedative activity by virtue of its GABAA receptor modulating properties.

Ganaxolone is being developed in intravenous and oral formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings where there is a mechanistic rationale for ganaxolone to provide a benefit.

In September 2020, the company announced positive Phase III results in children and young adults with CDKL5 deficiency disorder, a rare, genetic epilepsy with refractory seizures.

Marinus stock leapt 70% in after-hours trading following this news. The firm said it would submit to the US regulator for approval in this indication in mid-2021, with a European submission to follow soon after.