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Marinus Pharmaceuticals Announces U.S. Department of Defense Grant to Study Ganaxolone in Fragile-X Syndrome
Date:4/13/2011

NEW HAVEN, Conn., April 13, 2011 /PRNewswire/ -- Marinus Pharmaceuticals, Inc., the leader in the development of neurosteroids for central nervous system disorders, today announced the award of a $3 million grant by the U.S. Department of Defense to study its lead candidate ganaxolone for the treatment of Fragile-X Syndrome (FXS). Ganaxolone modulates GABA-A ion channels by selective binding to the neurosteroid receptor. Early research suggests that normalizing neurosteroid levels with ganaxolone treatment may eliminate the behavioral symptoms associated with FXS.

The $3 million federal grant was awarded to three University of California, Davis (UCD) researchers: Randi Hagerman, M.D., an international authority on Fragile X-related disorders; Michael Rogawski, M.D., Ph.D., a world-renowned epilepsy researcher; and David Hessl, Ph.D., an expert in psychophysiology studies. The group will enroll 60 children between the ages of six and 17 years to study the safety and effectiveness of ganaxolone for treating behaviors and anxiety common with FXS.

"The fact that a study with ganaxolone was awarded this grant is a testament to the potential for neurosteroid augmentation as a new treatment for FXS patients," stated Gail Farfel, Ph.D., Chief Clinicaland Regulatory Officer of Marinus. "The scientific data suggests that ganaxolone may be ideally suited to reverse FXS symptoms related to the down-regulation of the neurosteroid binding site on GABA-A ion channels."

"Over the next few months, we will work with UCD to provide ganaxolone for the FXS study and work on obtaining regulatory approval from the Food and Drug Administration to allow the researchers to conduct this study under Marinus' Investigational New Drug Application," commented Kenneth Shaw, Ph.D., Senior Vice President, R&D at Marinus.

About Ganaxolone

Ganaxolone is a synthetic neurosteroid and a derivative of the naturally occurring neuromodulator, allopregnanolon
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SOURCE Marinus Pharmaceuticals, Inc.
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