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 is currently conducting studies in CDKL5 deficiency disorder, PCDH19-related epilepsy, and refractory status epilepticus.

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

Status Epilepticus
(SE)
Data 1H-2022

SE is an emergency condition of continuous seizures lasting more than 5 minutes. Ganaxolone Phase 3 study initiated in Q3 2020 with topline data expected 1H 2020. Trial info

CDKL5 Deficiency
Disorder (CDD)
NDA Mid-2021
MAA Q3 2021

CDD is a rare genetic disorder resulting in developmental delay, intellectual disability, visual impairment, and severe epilepsy. Positive Phase 3 data reported in September 2020. Open label extension study is on-going. Trial info

Tuberous Sclerosis
Complex (TSC)
Data Mid-2021

TSC is a genetic disorder resulting in benign tumors, cognitive impairment, behavioral problems, and refractory epilepsy. Phase 2 topline data expected mid-2021. Trial Info

PCDH19-Related
Epilepsy (PCDH19)
Data 1H-2021

PCDH19-related epilepsy is a genetic disorder leading to psychiatric problems, neurodevelopmental delays, and refractory seizures often occurring in clusters. Phase 2 placebo-controlled data, stratified by a potential biomarker, are expected 1H-2021. Trial info

Administered
Depressive Disorders

Treatment Resistant
Depression (TRD)*

* Program on hold pending decision to conduct additional trials