Pipeline

Ganaxolone, through its validated GABAA mechanism, has opportunities in orphan indications and in chronic and acute care settings.

Clinical Trial Information

Ganaxolone is being developed in intravenous (IV) and oral formulations intended to maximize therapeutic reach to patient populations in both acute and chronic care settings.

Marinus completed the first ever Phase 3 pivotal trial in children with CDKL5 deficiency disorder in 2020, is planning to conduct a Phase 3 trial in tuberous sclerosis complex, and a Phase 3 trial in refractory status epilepticus is ongoing.

GANAXOLONE
Phase 1
Phase 2
Phase 3
Anticipated Milestones
Administered
Seizure Disorders

CDKL5 Deficiency Disorder 
Marigold Study

NDA submitted July 2021
MAA submission Q3 2021

CDD is a rare genetic disorder resulting in developmental delay, intellectual disability, visual impairment, and severe refractory epilepsy. Positive Phase 3 data reported in September 2020. Trial info

Tuberous Sclerosis Complex 
TrustTSC

Ph3 patient enrollment
Q4 2021

TSC is a genetic disorder resulting in benign tumors, cognitive impairment, behavioral problems, and refractory epilepsy. Trial Info

Refractory Status Epilepticus
RAISE

Data 2H 2022

RSE is a neurological emergency describing patients commonly managed in ICU’s with continuous or prolonged seizures that have failed to respond to 1st and 2nd line anti-seizure medications. Trial info

Refractory Status Epilepticus
RAISE II

Ph3 patient enrollment
1H 2022

RSE is a neurological emergency describing patients commonly managed in ICU’s with continuous or prolonged seizures that have failed to respond to 1st and 2nd line anti-seizure medications.

Established Status Epilepticus
RESET Trial

Ph2 patient enrollment 1H 2022
Data mid-2023

ESE is a neurological emergency describing patients commonly managed in Emergency Departments with continuous (>5 minutes) clinical seizures that have failed to respond to 1st line anti-seizure medications (i.e., benzodiazepines).

Lennox-Gastaut Syndrome

Ph2 trial to begin
2H 2022

Ongoing TrialProposed Future Trial

Oral SuspensionIntravenous Clinical DevelopmentOral Suspension (Second Generation Formulation)