Marinus Pharmaceuticals Inc. (NASDAQ: MRNS) saw its shares make a huge gain to kick off the week after the firm announced top-line results from its mid-stage study in patients with CDKL5 disorder. Essentially, the Phase 2 study of ganaxolone showed a sizable and durable seizure-frequency reduction in the majority of patients, with some achieving an increase in the number of seizure-free days and reporting behavioral benefits.

For some quick background: CDKL5 disorder is a severe, rare genetic epilepsy that results in early-onset, treatment-refractory seizures, pervasive neuro-developmental delay and disabling behavioral issues.

The median change in 28-day seizure frequency from baseline in the intent-to-treat (ITT) population (primary endpoint) was a decrease of 43%. At the same time, the median change from baseline in seizure-free days in the ITT population (key secondary endpoint) was an increase of 78%.

Ganaxolone was generally safe and well-tolerated with no serious adverse events. To date, there have been no adverse event reports of somnolence or dizziness.

Looking ahead, the company plans to meet with regulatory agencies to obtain agreement on the clinical development plan that would be needed for approval of ganaxolone for CDKL5 disorder.

Orrin Devinsky, M.D., director of the NYU Langone Medical Center’s Comprehensive Epilepsy Center and principal investigator in the current study, commented:

I am impressed with the magnitude of seizure reduction and gain in seizure-free days seen with ganaxolone treatment in children with this highly refractory epilepsy. The durable anti-epileptic effect seen in several children distinguishes ganaxolone’s efficacy from the more than 20 currently available anti-epileptic drugs that provide limited seizure control lasting a few weeks to months. When treating children with the severest forms of epilepsy such as CDKL5 disorder, there is a desperate need for new drugs that are well-tolerated, efficacious and easy to administer with no need for special monitoring. With no other treatment showing this degree of promise, I am excited to lead the effort to advance ganaxolone into potentially the first, late-stage clinical trial in children with CDKL5 disorder.

Shares of Marinus were up 56% at $4.87 early Monday, with a consensus analyst price target of $5.00 and a 52-week range of $0.82 to $5.43.

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