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Results from Phase 2 Exploratory Clinical Study Support Continued Development of Ganaxolone in Fragile X Syndrome

Study Investigator to Participate in Conference Call and Webcast Today at 9:00 a.m. ET

RADNOR, Pa., June 28, 2016 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (MRNS), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced top-line data from a Phase 2 exploratory, investigator-sponsored study to evaluate the safety, tolerability and efficacy of ganaxolone for the treatment of anxiety and attention in children with Fragile X Syndrome (FXS). The investigator selected Clinical Global Impression of Improvement (CGI-I), which is a broad scale to assess the overall improvement across the entire Fragile X Syndrome, as the primary endpoint of the study. While this primary endpoint was not met, consistent with Marinus’ expectations and ganaxolone’s mechanism of action, treatment with ganaxolone improved anxiety and hyperactivity across multiple measures in FXS patients with high baseline anxiety. The results from this study support the anxiolytic effect of ganaxolone and provide a strong rationale for Marinus to advance the clinical development of ganaxolone in anxious FXS patients.

Christopher M. Cashman, chief executive officer of Marinus Pharmaceuticals, commented, “Children with FXS suffer from a wide spectrum of behavior problems impacting their quality of life, including anxiety, attention deficits, hyperactivity and autism spectrum disorders. Our goal for this study was to measure improvement in these behaviors based upon the anxiolytic activity of ganaxolone and use the results from this exploratory study to inform enrollment criteria and endpoint selection in future studies. The data we observed in several analyses related to patient anxiety and attention are encouraging and clearly warrant further investigation. We will be working closely with our advisors and the FDA to finalize our clinical and regulatory strategy for this indication and intend to provide more details on our plans later this year.”

The Phase 2 study enrolled 59 children between 6 and 17 years of age with a diverse range and severity of FXS-related symptoms. The trial utilized multiple clinician- and parent-administered rating scales designed to identify a signal among a variety of symptoms common to FXS children. Twenty-nine of the treated patients had a high level of anxiety at baseline (defined as baseline Pediatric Anxiety Rating Scale (PARS) ≥13) and showed improvements in anxiety, attention and hyperactivity as measured by scales designed to detect these symptomatic changes (Visual Analog Scale (VAS), Anxiety, Depression & Mood Scale (ADAMS), and Aberrant Behavior Checklist (ABC)).

Mean Percent Improvement Seen with Ganaxolone vs. Placebo in Patients with PARS ≥ 13

VAS ADAMS ABC
Severity of Anxiety Severity of Attention General Anxiety Manic/Hyperactive Behavior Hyperactivity Social Withdrawal
Ganaxolone 291.9 % 110.4 % 23.0 % 12.3 % 23.5 % 26.6 %
Placebo 89.7 % 38.4 % -14.1 % -15.4 % 1.9 % 5.0 %
p-value 0.055 0.030 0.030 0.017 0.045 0.267

Frank Kooy, Ph.D., Department of Medical Genetics, and investigator in the Phase 2 study from Antwerp University in Belgium, commented, “Consistent with the mechanism of action and preclinical studies, anxious FXS patients treated with ganaxolone showed clinically relevant reductions in anxiety across several scales and subscales sensitive to measuring changes in anxious behavior. These results are encouraging and suggest that ganaxolone may improve anxiety in children with FXS, which could...


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