Navigation Links
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, 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 Fragile-X Syndrome

The result of a defect on the X chromosome, Fragile-X Syndrome (FXS) is the leading cause of inherited mental disability and the most common single-gene cause of autism. The condition is estimated to affect one in 3,600 males and one in 4,000 females. Approximately one-third of all children diagnosed with Fragile-X Syndrome also have some degree of autism. Fragile-X symptoms include learning and memory impairment, anxiety, hyperactivity and social avoidance.  Up to 30% of people with Fragile-X also develop seizures.

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.


'/>"/>
SOURCE Marinus Pharmaceuticals, Inc.
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. Marinus Pharmaceuticals Issued Patent for Ganaxolone
2. Conatus Pharmaceuticals Completes the Second Closing of its Series B Private Placement Financing
3. MAP Pharmaceuticals to Present Data From LEVADEX® Pharmacokinetics and Pharmacodynamics Safety Studies at the 63rd Annual Meeting of the American Academy of Neurology
4. Avanir Pharmaceuticals Announces Appointment of William Sibold as Chief Commercial Officer
5. Valeant Pharmaceuticals Files Presentation for Cephalon Stockholders
6. Webcast and Conference Call Alert: Isis Pharmaceuticals Holds Webcast to Discuss Recent Advances in its Development Pipeline
7. Auxilium Pharmaceuticals, Inc. Announces Launch of XIAPEX® in EU by Pfizer
8. Vanda Pharmaceuticals Announces Commencement of Clinical Study of Long-Acting Injectable Formulation of Fanapt®
9. Idenix Pharmaceuticals Prices Public Offering of Common Stock
10. Pacira Pharmaceuticals to Present at BioCentury Future Leaders in the Biotech Industry Conference
11. Idenix Pharmaceuticals Announces Proposed Public Offering of Common Stock
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/28/2017)... -- Cohen Veterans Bioscience and Early Signal Foundation announce ... home sensors for real-time monitoring of patients with trauma-related ... organization focused on disruptive health solutions for rare disorders ... to record and integrate behavioral, cognitive, physiological and contextual ... ...
(Date:9/25/2017)... AMSTERDAM , Sept. 25, 2017   ... Trial Master File solutions, today—from the IQPC Trial ... Amsterdam , NL)—announced that EastHORN Clinical Services ... its clinical programs and TMF management. EastHORN, a ... Montrium,s eTMF platform to increase transparency to enable ...
(Date:9/22/2017)... , Sept. 22, 2017  As the latest Obamacare ... Bill Cassidy (R-LA) and Lindsey Graham ... that the medical device industry is in an odd ... tax, the 2.3% excise tax on medical device sales ... also want covered patients, increased visits and hospital customers ...
Breaking Medicine Technology:
(Date:10/13/2017)... (PRWEB) , ... October 13, 2017 , ... “The Journey: ... faced every danger possible to save lost souls in the Philippines. “The Journey: From ... Carole is a dedicated teacher of the Bible. She has taught all ages and ...
(Date:10/12/2017)... New Orleans, LA (PRWEB) , ... October 12, 2017 , ... ... fitness centers in the U.S., announced today its plans to open a flagship location ... club will occupy the former Rooms To Go store next to Office Depot in ...
(Date:10/12/2017)... (PRWEB) , ... October 12, 2017 , ... ... AccentCare, a leader in post-acute health care, have expanded their existing home health ... Home Health. , AccentCare has been operating a joint venture home health company ...
(Date:10/12/2017)... Washington, D.C. (PRWEB) , ... October 12, 2017 , ... ... of their peers in Washington, D.C., for the 49th Congress of the International ... Ph.D ., Vice President of the Center for Cancer and Blood Disorders ...
(Date:10/12/2017)... ... October 12, 2017 , ... ... in property taxes a year. In some states—like New York, New Jersey, Massachusetts, ... contrast, many overseas retirement havens have extremely low property-tax rates, which contributes to ...
Breaking Medicine News(10 mins):