Marinus Pharmaceuticals’ Ganaxolone IV Demonstrates Robust Efficacy in Benzodiazepine-Resistant Model of Status Epilepticus

RADNOR, Pa., April 19, 2016 (GLOBE NEWSWIRE) --  Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, presented preclinical data of ganaxolone IV, the Company’s intravenous formulation of its CNS-selective GABA_A modulator,  showing robust activity in a well-accepted and clinically translatable animal model of status epilepticus (SE). The data were presented during an oral and poster presentations at the 68^th American Academy of Neurology (AAN) Annual Meeting in Vancouver, BC, Canada, April 15-21, 2016.

Albena Patroneva, M.D., Chief Medical Officer of Marinus Pharmaceuticals, commented, “Status epilepticus is a life threatening medical emergency with no FDA approved treatment. Typically, single or combination IV antiepileptic drugs are used in an attempt to break the seizures, however many patients do not respond to treatment, which increases the risk of neuronal damage, cognitive impairment and death. The preclinical data presented at AAN shows that ganaxolone IV could be a promising therapeutic for this vulnerable patient group and serves as the foundation for our clinical development plan, which is on-track to initiate a Phase 1 clinical trial this quarter.”

The first abstract, entitled, “Ganaxolone administered intravenously prevents behavioral seizures and promotes survival in the rat lithium-pilocarpine model of status epilepticus,” was presented by Michael S. Saporito, Ph.D., during dual oral and poster presentations. The poster highlighted a study on ganaxolone IV in a clinically translatable rodent model of SE, in which convulsive SE was induced by the administration of lithium chloride and pilocarpine in male rats. Ganaxolone IV (6, 9, or 12 mg/kg) or allopregnanolone (15 mg/kg) were administered at the time of SE onset or 15, 30 or 60 minutes after SE onset. In this rodent model, ganaxolone IV produced a dose-dependent anticonvulsant effect at all time points, even when administration was delayed 30 to 60 minutes, which are models of benzodiazepine-resistant SE. In addition, ganaxolone promoted survival and showed activity similar to that observed with allopregnanolone.

Dr. Saporito also provided a poster presentation, entitled, “Intravenous administration of ganaxolone attenuates electroencephalographic seizures in diazepam-resistant model of status epilepticus.” The poster reported the results from a study evaluating the potential of ganaxolone IV in a benzodiazepine-resistant model of SE where seizure response was measured by electroencephalographic recordings for five hours. Ganaxolone IV (12 or 15 mg/kg) or allopregnanolone (15 mg/kg) were administered 15 or 60 minutes after SE onset. In this study, both doses of ganaxolone IV elicited a complete block of SE at all time points, with antiepileptic activity observed for at least five hours.

About Marinus Pharmaceuticals

Marinus Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety and convenient dosing, to improve the lives of patients suffering from epilepsy and neuropsychiatric disorders.  Ganaxolone is a CNS-selective GABA_A modulator 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) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings.  Ganaxolone IV is planned to enter clinical trials in 2016 and is being developed to treat status epilepticus. Ganaxolone IV is complemented by its oral dose forms, providing the potential for IV-to-oral continuation therapy for patients transitioning from acute care to outpatient settings.  Ganaxolone capsule is being evaluated in a Phase 3 multi-national clinical trial as adjunctive treatment of focal onset seizures in adults.  Ganaxolone capsule and liquid are being studied in orphan pediatric indications with comorbidities in seizures and behavior disorders – PCDH19 epilepsy and Fragile X Syndrome. For more information visit www.marinuspharma.com.

Forward-Looking Statements

To the extent that statements contained in this press release are not descriptions of historical facts regarding Marinus, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.  Words such as “may”, “will”, “expect”, “anticipate”, “estimate”, “intend”, “believe”, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements.  Examples of forward looking statements contained in this press release include, among others, statements regarding our interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical trial testing schedule and milestones, the ability to complete enrollment in our clinical trials, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this release involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements.  Such risks and uncertainties include, among others, the uncertainties inherent in the conduct of future clinical trials, the timing of the clinical trials, enrollment in clinical trials, availability of data from ongoing clinical trials, expectations for regulatory approvals, and other matters, including the development of formulations of ganaxolone, that could affect the availability or commercial potential of our drug candidates.  Marinus undertakes no obligation to update or revise any forward-looking statements.  For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the Company in general, see filings Marinus has made with the Securities and Exchange Commission.