It’s a very rewarding trading day for Marinus Pharmaceuticals Inc (NASDAQ:MRNS) investors with shares up nearly 60%, making the stock Wall Street’s bull of the day. The reason? The drug maker announced this morning that top-line data from its Phase 2 open-label study in patients with CDKL5 disorder support advancing ganaxolone, a small molecule, into a definitive late-stage clinical trial.

Oral ganaxolone, in addition to baseline treatment, 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. There are no approved or effective available treatment options. Marinus 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, MD, 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.”

Marinus is a biopharmaceutical company, which engages in the development of neuropsychiatric therapeutics. Its clinical stage drug candidate, ganaxolone, is a novel synthetic small molecule that is an analog of allopregnanolone, a natural occurring neurosteriod produced by the human body, which modulates gamma-aminobutyric acid a neurotransmitter in the brain.