Marinus Presents Additional Responder Analysis Data for Ganaxolone in Focal Onset Seizures at the American Epilepsy Society Annual Meeting

Print 07 December 2015

43.9% of Ganaxolone-Treated Patients Achieved ≥ 30% Improvement in Weekly Seizure Frequency

RADNOR, Pa., Dec. 07, 2015 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, presented additional responder analysis data from the Phase 2 clinical trial of ganaxolone as an adjunctive treatment in 147 randomized adults with focal onset seizures at the 69th Annual Meeting of the American Epilepsy Society, December 6, 2015, at the Pennsylvania Convention Center in Philadelphia, PA.

The poster, entitled, "Additional responder analysis on a phase 2 study of ganaxolone in patients with partial onset seizures," highlighted post-hoc analysis data showing that the percent of ganaxolone-treated patients who achieved ≥40% improvement in weekly seizure frequency was nearly twice that of placebo-treated patients (30.5% vs. 16.7%, p=0.07). In addition, significantly more patients treated with ganaxolone achieved ≥30% improvement than the placebo group (43.9% vs. 25%, p=0.027). The study observed no difference in percent change in mean weekly seizure frequency or response rate in patients by gender.

Dr. Albena Patroneva, Chief Medical Officer of Marinus Pharmaceuticals, commented, "Our phase 2 clinical trial evaluating ganaxolone as adjunctive treatment in patients with focal onset seizures was a well-designed, well-conducted study that enrolled highly-refractory patients — those that have been struggling with epilepsy for more than 25 years and taking more than two concomitant anti-epileptic drugs. This is a very difficult patient population to treat. To show a 30% improvement rate in weekly seizure frequency in those patients that responded to ganaxolone, gives us the encouragement that ganaxolone could potentially be an attractive alternative for epilepsy patients and may provide additional treatment options for physicians treating these refectory patients."

In addition, Marinus presented several posters during the scientific exhibit, where the Company unveiled for the first time to the medical community its preclinical data and clinical plans for ganaxolone intravenous (IV) formulation. The presentations included a preclinical animal model of benzodiazepine-resistant status epilepticus (SE), where 12 mg/kg and 15 mg/kg of ganaxolone IV elicited a sustained (≥5 hour), statistically significant block of SE when administered 60 minutes after onset, whereas allopregnanolone produced a partial and short-term block. In another preclinical model of SE, ganaxolone IV blocked behavioral seizures and promoted survival at rates similar to those observed with administration of allopregnanolone as measured at 24 hours after SE onset. GLP toxicology data enabling the IV administration in humans showed that ganaxolone IV was generally well tolerated in rats and rabbits. The new formulation did not cause hemolysis and was found compatible with human plasma in vitro.

About Ganaxolone

Ganaxolone is a CNS-selective GABAA modulator 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 acts on a well-characterized synaptic and extrasynaptic GABAA target known for anti-seizure and anti-anxiety activity. Ganaxolone is currently being studied in a multi-national Phase 3 clinical trial in adults with focal onset seizures, an exploratory Phase 2 proof-of-concept clinical trial in children with PCDH19 female epilepsy, and an exploratory Phase 2 proof-of-concept clinical trial in children with Fragile X syndrome. Ganaxolone has been studied in more than 1,300 subjects, both pediatric and adult, at therapeutically relevant dose levels and treatment regimens for up to two years. In these studies, ganaxolone was generally safe and well tolerated, with the most commonly reported adverse events of somnolence, dizziness and fatigue.

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 GABAA 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 and clinical 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.

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