Updates from Marinus on Ganaxolone Trials for 3 Forms of Epilepsy

Marinus Pharmaceuticals has recently announced updates on its ganaxolone programs for tuberous sclerosis complex (TSC), PCDH19-related epilepsy (PCDH19-RE), and CDKL5 deficiency disorder (CDD).


Ganaxolone is a positive allosteric modulator of GABAA receptors. It has been found to produce anti-seizure activity and anti-anxiety activity. So far, the formulation has been investigated in 1,600 pediatric and adult patients. There is an oral and an intravenous form of the therapy, which are being investigated in different doses. So far, the therapy has shown to be both safe for patients and well-tolerated.

The longest any patient has been on the drug so far is four years.

The most common AEs experienced by patients are dizziness, fatigue, and somnolence.

Tuberous Sclerosis Complex

After a new biomarker for epilepsy called Allo-S was discovered in a Phase 2 clinical trial for PCDH19-RE, researchers began inquiring about this biomarker in TSC. They uncovered that in fact, TSC may be impacted by Allo-S levels in the same way. Epilepsy patients with lower levels of Allo-S have been shown to have improved responses with ganaxolone.

With this new knowledge, Marinus will be conducting a Phase 2 clinical trial of ganaxolone for TSC within the first half of next year.  It will evaluate the safety of the treatment and its tolerability for patients. Multiple trial sites across the United States will be initiated.

The trial will enroll between 20 and 40 patients who are age 2 to age 65. The primary endpoint of the investigation is the percent change in 28-day primary seizure frequency. This endpoint will be compared between the 12 week treatment period and the 4 week baseline period.

CDKL5 Deficiency Disorder 

The EMA has just provided Orphan Drug Designation for ganaxolone as a therapy for CDD. With this new approval, Marinus will initiate a Phase 3 clinical trial for the therapy. They are still currently recruiting for this trial, but if things go according to plan, data will be reported within the third quarter of 2020. This trial is called the Marigold study.

This investigation will examine the effect of ganaxolone taken orally in 100 patients age 2 to 21 with CDD. This form of epilepsy has yet to have an approved treatment.

The trial will be global, placebo-controlled, and double-blind.

PCDH19-related Epilepsy 

The Phase 3 Violet study is continuing enrollment. It is investigating oral ganaxolone for 70 children age 1 to 17 who are diagnosed with PCDH19-RE. International trial sites are still being initiated. Patients in this trial will be stratified into two groups: biomarker positive or biomarker negative.

Researchers believe this investigation could provide the very first diagnostic blood test for epilepsy which could effectively predict the likelihood that a patient will respond to treatment.

Data from this trial will be reported in 2021.

You can read more about these updates here.

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