BARDA Contract

The Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response within the US Department of Health and Human Services, has awarded Marinus Pharmaceuticals $51 million of an $84 million contract to fund the company’s Phase 3 trial in refractory status epilepticus.

On a successful development, BARDA and Marinus could potentially negotiate a procurement agreement for a supply of ganaxolone for potential response to nerve agent exposure threats.


September 2020 announcement

BARDA and Marinus Partner to Develop New Treatment for Refractory Seizures


FAQs

Q: What is BARDA and why is BARDA working with Marinus?

A: The Biomedical Advanced Research and Development Authority (BARDA), part of the HHS Office of the Assistant Secretary for Preparedness and Response (ASPR), was established to aid in securing our nation from chemical, biological, radiological, and nuclear threats, as well as from pandemic influenza and emerging infectious diseases. BARDA supports the transition of medical countermeasures such as vaccines, drugs, and diagnostics from research through advanced research and development towards consideration for approval by the FDA and inclusion into the Strategic National Stockpile. BARDA’s support includes funding, technical assistance and core services, ranging from a clinical research organization network to Centers for Innovation in Advanced Development and Manufacturing, and a fill-finish manufacturing network.

BARDA is supporting Marinus in the company’s Phase 3 refractory status epilepticus (RSE) trial (RAISE). This trial will investigate the use of ganaxolone in the treatment of RSE caused by medical conditions, as well as a potential indication for benzodiazepine refractory status epilepticus caused by nerve agent exposure.

Q: How is Marinus working with BARDA on the RAISE Trial?

A: BARDA is supporting Marinus in the Phase 3 clinical trial of ganaxolone for the treatment of RSE. Parallel to this Phase 3 clinical trial, Marinus and BARDA are also conducting non-clinical studies of ganaxolone efficacy in a nerve agent exposure model. This trial is expected to have a data readout in the first half of 2022. 

Q: What are the potential outcomes of the RAISE trial for both Marinus and BARDA?

A: If the RAISE trial is positive, Marinus would expect to file a new drug application with the FDA requesting approval for the use of ganaxolone for treatment of RSE. For BARDA, a positive RSE trial, together with additional data in a nerve agent exposure model, would support the potential use of ganaxolone for nerve agent toxicity – a BARDA medical countermeasure priority. 

Q: What is the primary endpoint of the RAISE trial?

A: The co-primary endpoints for the RAISE trial are (1) proportion of patients with status epilepticus cessation within 30 minutes of treatment initiation without other medications for the treatment of status epilepticus, and (2) proportion of patients with no progression to IV anesthesia for 36 hours following treatment initiation without additional medications for acute treatment of refractory status epilepticus (RSE). RSE cessation is determined based on clinical and electroencephalogram (EEG) findings.

Q: How much capital has/will BARDA contribute to the RAISE trial?

A: The agreement between Marinus and BARDA covers a base period during which BARDA will provide subject matter expertise and $21 million in financial support to fund, on a cost share basis, the RAISE trial and to fund the non-clinical studies. Contingent on favorable clinical and pre-clinical outcomes in the base period, the contract includes Options for up to approximately $30 million of additional BARDA funding for manufacturing, supply chain, clinical, regulatory and toxicology activities. Under the current contract, Marinus will be responsible for cost-sharing in the amount of $33 million if all development options are completed.

Q: Is BARDA working with Marinus on the established status epilepticus (ESE) trial and the RAISE II trial in Europe?

A: No, BARDA is not working with Marinus on these trials. 

Q: What is the timeline of the RAISE trial?

A: See Marinus’ pipeline chart.