Marinus Pharmaceuticals, a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, announced it has entered into a five-year development contract with the Biomedical Advanced Research and Development Authority (BARDA) to support the development of cyclodextrin-enabled IV ganaxolone for the treatment of refractory status epilepticus (RSE), a life-threatening condition in which a significant number of patients do not respond to first- and second-line anticonvulsant drugs.
RSE can occur as a result of a variety of serious, acute medical conditions or after exposure to nerve agents. The agreement covers a base period during which BARDA will provide subject matter expertise and $21 million to fund, on a cost share basis, the company’s planned Phase 3 clinical trial of ganaxolone for the treatment of RSE (as a result of an underlying medical condition) and will fund preclinical studies of ganaxolone in nerve agent exposure animal models. Contingent on favorable clinical and preclinical outcomes in the base period, the contract includes up to approximately $30 million of additional BARDA funding spanning three options in support of manufacturing, supply chain, clinical, regulatory and toxicology activities. Under the contract, Marinus will be responsible for cost-sharing in the amount of $33 million if all development options are completed.
Organophosphate nerve agents (chemical warfare agents and organophosphate-based pesticides) are highly toxic compounds, which may cause prolonged seizures that become more difficult to treat as they progress. Ganaxolone has a complementary and potentially synergistic mechanism to benzodiazepines, which are currently used to treat nerve agent exposure-induced seizures and may help to stop unremitting seizures when other drugs fail.
On a successful development, BARDA and Marinus may negotiate a procurement agreement for a supply of ganaxolone for potential response to nerve gas exposure threats.
Marinus plans to pursue further discussions with BARDA to evaluate additional routes of administration for ganaxolone that would support field-based rapid response treatment in the event of a nerve gas attack.