Marinus Pharma (MRNS) is trading sharply higher today (+50%) after the company announced positive clinical data. Since you're probably not familiar with Marinus Pharma, a little background would help. It's a development stage biopharma focused on the development of ganaxolone, which offers a new mechanism to improve the lives of patients suffering from epilepsy and neuropsychiatric disorders. Ganaxolone is a positive allosteric modulator of GABAA that acts on a well-characterized target in the brain known to have both anti-seizure and anti-anxiety effects.
Ganaxolone is being developed in three different dose forms (IV, capsule and liquid). Marinus is currently evaluating ganaxolone in women with postpartum depression and in orphan pediatric indications for the treatment of genetic seizure and behavior disorders, and preparing to initiate a Phase 2 study in status epilepticus, an orphan indication. MRNS does not generate revenue at this point.
Turning to today's news, Marinus announced that top-line data from its Phase 2 study in patients with CDKL5 disorder support advancing ganaxolone 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.
CDKL5 disorder is a severe, rare genetic epilepsy that results in early-onset, treatment-refractory seizures, pervasive neuro-developmental delay and disabling behavioral issues. There are no approved or effective available treatment options. Orrin Devinsky, MD, Director of the NYU Langone Medical Center's Comprehensive Epilepsy Center and Principal Investigator says that 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.
Earlier this year, Marinus received Orphan Drug Designation from the FDA to develop ganaxolone for the treatment of CDKL5 Disorder. MRNS says the tolerability and complete lack of dose regimen-limiting safety concerns, as compared to the most widely used anti-epileptic drugs for this condition, could make ganaxolone an exceptional option for patients with CDKL5 disorder.
In sum, investors are clearly excited with today's news as you can see in the stock reaction. Looking ahead, Marinus is planning to submit the full CDKL5 disorder data set for publication or presentation at a medical conference. The next step is to meet with regulatory agencies to obtain agreement on the clinical development plan that would be needed for approval of ganaxolone for CDKL5 disorder.
After trading mostly sideways for much of the past year, the stock has started to pick up steam since mid-July. In late June, the FDA granted Orphan Drug Designation to ganaxolone for the treatment of CDKL5 Disorder. Then a few week later, Deerfield Mgmt (9.98%) and Granite Point Capital (9.75%) disclosed sizeable passive stakes in MRNS and now today's news is moving the stock sharply higher.