Whether you are a prospective investor, physician, or a patient suffering with drug-resistant seizures or neuropsychiatric disorders, you will find answers to the most commonly asked questions about Marinus and our development of ganaxolone below.

Q. How many participants have been involved in ganaxolone trials?

A. Ganaxolone has been studied in over 1600 patients, both adult and pediatric.

Q. What medical conditions are being investigated with ganaxolone?

A. Marinus has studied ganaxolone in patients with CDKL5 deficiency disorder, PCDH19-related epilepsy, Tuberous Sclerosis Complex, and refractory status epilepticus. The company intends to explore other neuropsychiatric areas.

Q. What is the timing for readouts on current clinical trials?

Q. What does the competitive landscape look like for status epilepticus?

A. Many antiepileptic medications used for treatment of status epilepticus (SE) are IV reformulations of oral drugs and are not FDA-approved specifically for the treatment of SE. There have not been rigorous, controlled studies in refractory SE (RSE) and no drugs are approved for that indication.

Q. What does the competitive landscape look like for CDKL5 deficiency disorder?

A. There are currently no disease-specific, approved treatments for CDKL5 deficiency disorder (CDD). Patients typically experience early onset seizures and are often poorly responsive to antiepileptic drugs, with a tendency for treatments to become less effective in controlling seizures over time.

Q. What does the competitive landscape look like for tuberous sclerosis complex?

A. Antiepileptic therapy in tuberous sclerosis complex (TSC) is typically guided by seizure type rather than the underlying disorder. Despite the availability of the anti-cancer drug, everolimus, for treatment of focal seizures in TSC and other medications being studied, there remains a high rate of poorly controlled seizures and considerable unmet need for new therapies.

Q. What does the competitive landscape look like for PCDH19-related epilepsy?

A. Like many other genetically-determined epilepsies, there is not a disease-specific antiseizure treatment for PCDH19-related epilepsy. Seizures occur in clusters and are often poorly responsive to antiepileptic therapy.

Q. Why is Marinus proceeding to a Phase 3 trial in refractory status epilepticus?

A. Findings from the Phase 2 clinical study evaluating IV ganaxolone in 17 patients with RSE met the primary endpoint with no patients progressing to IV anesthetics within 24 hours of treatment initiation. The onset of effect was rapid, with a median time to seizure cessation of 5 minutes, despite patients having failed an average of 2.9 prior treatments for SE. This result and the unmet medical need provided Marinus with confidence to move forward to the Phase 3 trial.

Q. What does the current IP Protection look like?

A. The nanoparticulate formulation composition patents for oral liquid and solid dose forms of ganaxolone are expected to provide intellectual property protection through at least 2026. Many of the indications Marinus is developing for ganaxolone provide regulatory exclusivity periods for 7 years from product approval as orphan drug indications.