With the development of the drug ganaxolone (GNX), Marinus Pharmaceuticals and BARDA hope to create an anticonvulsant capable of halting damaging seizures in their tracks, even those caused by deadly nerve agents like the odorless, colorless liquid, Sarin.
Some seizures have proven resilient to even first-line anticonvulsants like benzodiazepines. These are refractory seizures, and given that continuous or near-continuous seizures can cause permanent brain damage and disabilities, health experts could greatly benefit from a fix. Sarin is one of those agents that can cause these ongoing, life-threatening effects, owing to its toxic compounds causing overstimulation of the body’s central and peripheral nervous system.
Enter Marinus, which has recently completed a phase two clinical study of GNX for us on refractory seizures. Of the 17 patients on whom the drug was tested, none needed to progress to treatment with IV anesthesia, and 16 of the 17 patients remained free of seizures for a full 24 hours after beginning treatment. With BARDA’s assistance, the company now intends to proceed into a large-scale phase three clinical trial, along with non-clinical studies of nerve agent-caused refractory seizures.
As part of this collaboration, Marinus will aim to produce GNX in the U.S. If approved by the Food and Drug Administration, the drug would become the standard of care for treating refractory seizures.