Marinus Pharmaceuticals Announces Commencement of Phase 2 Trial of Ganaxolone for the Treatment of Posttraumatic Stress Disorder
Published: Apr 21, 2011
BRANFORD, Conn., April 21, 2011 /PRNewswire/ -- Marinus Pharmaceuticals, Inc., the leader in the development of neurosteroids for central nervous system disorders, today announced commencement of a proof-of-concept clinical trial of its lead candidate ganaxolone for the treatment of posttraumatic stress disorder (PTSD). Ganaxolone modulates inhibitory GABA-A receptors, possibly at a specific neurosteroid recognition site. Neurosteroid levels have been implicated in both the severity and treatment outcome in PTSD patients.
"This trial will test the hypothesis that a neurosteroid analog of allopregnanolone will be beneficial in the treatment of PTSD and augment our existing safety database for ganaxolone in humans," stated Gail Farfel, Ph.D., Chief Development and Regulatory Officer of Marinus. "Achieving full remission of PTSD symptoms is difficult with current treatments for PTSD. New options that can increase efficacy are needed for the growing population of PTSD patients worldwide."
This randomized, double-blind, placebo-controlled clinical trial is being conducted by the INTRuST Consortium to evaluate the safety, tolerability and efficacy of oral ganaxolone after six weeks of dosing in patients with PTSD. The INTRuST Consortium is a group of clinical study centers in the United States funded by a Department of Defense award to advance treatments and medical research in PTSD and traumatic brain injury. This public-private collaboration will study ganaxolone under Marinus' Investigational New Drug Application (IND) filed with the Food and Drug Administration (FDA). The trial will take place in the United States and is designed to enroll approximately 120 PTSD patients.
"Ganaxolone has been shown in clinical studies to be efficacious and well tolerated in refractory epilepsy patients, and we expect the INTRuST PTSD trial to expand ganaxolone's profile into neurosteroid-mediated psychiatric disorders," said Kenneth Shaw Ph.D., Senior Vice President, R&D of Marinus. "We are pleased that the INTRuST Consortium is conducting this trial and await the results expected in 2012".
Ganaxolone is a synthetic neurosteroid and a derivative of the naturally occurring neuromodulator, allopregnanolone. Ganaxolone has been administered to more than 950 healthy adult volunteers and patients in Phase 1 and Phase 2 studies. Completed Phase II epilepsy studies have generated data supportive of the efficacy and safety of ganaxolone in the treatment of both children and adults suffering from refractory epilepsy (patients who continue to have seizures despite taking multiple anticonvulsant drugs). Scientific research has suggested that ganaxolone therapy may be useful in the treatment of several other central nervous system disorders including posttraumatic stress disorder (PTSD) and Fragile-X syndrome (FXS). Ganaxolone is being developed as a first in class treatment in epilepsy, PTSD and FXS.
Marinus has successfully developed several proprietary and novel patented formulations of ganaxolone.
About Posttraumatic Stress Disorder (PTSD)
PTSD is a severe anxiety disorder that can develop after exposure to a traumatic event, overwhelming the individual's ability to cope. As an effect of psychological trauma, PTSD is less frequent and more enduring than the more commonly seen acute stress response.
Diagnostic symptoms for PTSD include re-experiencing the original trauma(s) through flashbacks or nightmares, avoidance of stimuli associated with the trauma, and increased arousal such as difficulty falling or staying asleep, anger and hyper-vigilance. PTSD symptoms cause significant impairment in social, occupational or other important areas of functioning.
Lifetime prevalence is at least 1% and may be as high as 15% in the U.S. population. A national comorbidity survey conducted in the early 1990s found that women are twice as likely as men to experience PTSD. In high-risk groups, such as combat veterans and victims of violent crimes, prevalence can range from 20% to over 50%.
About Marinus Pharmaceuticals
Marinus is a specialty pharmaceutical company dedicated to the reformulation, development, and commercialization of novel drugs to treat serious neurological and psychiatric disorders. Marinus is located in Branford, Connecticut. Its investors include Domain Associates, Canaan Partners, Sofinnova Ventures and Foundation Medical Partners. For additional information, please visit the company's Web site at www.marinuspharma.com.
For more information on INTRuST please visit the consortium's website at http://intrust.sdsc.edu/.
SOURCE Marinus Pharmaceuticals, Inc.