Complex partial seizures result in impaired consciousness, with symptoms of decreased responsiveness and decreased awareness of self and surroundings. Approximately 1.4 million people in the U.S. have epilepsy in the form of complex partial seizures. Refractory complex partial seizures occur in about 35% of persons with epilepsy. These refractory patients have uncontrolled seizures despite all available drugs and treatment paradigms.

Marinus Pharmaceuticals has completed enrollment its Phase 2a clinical trial of ganaxolone for the treatment of refractory adult complex partial seizures. In this double blinded placebo controlled study with 147 patients, statistically significant reductions in seizure frequency was observed with an excellent safety profile.

Post-Traumatic Stress Disorder, PTSD, is an anxiety disorder that can develop after exposure to a traumatic event including violent personal assaults, natural or human-caused disasters, accidents, or military combat. Post-traumatic stress disorder symptoms are commonly grouped into three types: intrusive memories, avoidance and numbing, and increased anxiety or emotional arousal (hyperarousal). The U.S. lifetime prevalence of PTSD is estimated at >6% of the general population as high as 30% in the military. The consequences of PTSD at the personal, family and societal levels are very significant and new treatments are needed.

Marinus has entered into a public-private relationship with the INTRuST consortium which is a group of PTSD treatment centers in the U.S. that has received government funding (Dept. of Defense) to evaluate promising new treatments for PTSD. Ganaxolone will be tested by the INTRuST consortium in PTSD patients. The trial is starting in February 2011.

Fragile X syndrome (FXS), the most common cause of inherited mental impairment. This impairment can range from learning disabilities to more severe cognitive or intellectual disabilities. Low expression of the neurosteroid GABAergic binding site suggests that neurosteroid replacement treatment with ganaxolone may help normalize the anxiety, hyperactivity and learning disabilities associated with this inherited disorder.

A group of neurologists at the University of California, Davis has recently been awarded a grant to study ganaxolone for the treatment of Fragile-X. This study is planned to start later in 2011.