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Autism Speaks Announces Results Reported for the Study of Fluoxetine in Autism (SOFIA)

First Industry-Sponsored Trial for the Autism Clinical Trials Network (ACTN)

NEW YORK, N.Y. (February 18, 2009) – Autism Speaks today announced initial results reported for the first industry-sponsored trial of the Autism Clinical Trials Network (ACTN), the Study of Fluoxetine in Autism (SOFIA). These results showed that fluoxetine was not effective for reducing repetitive behaviors in children and adolescents with Autistic Disorder as compared to placebo (non-medicinal sugar pill). The study was carried out in collaboration with study sponsor, Neuropharm Group Plc., a specialty pharmaceutical company focused on neurodevelopmental disorders.

The study evaluated the efficacy and safety of Neuropharm's new low-dose form of fluoxetine, known as NPL-2008, which is designed specifically for the treatment of Autistic Disorders in children and adolescents. NPL-2008 uses the Zydis® melt-in-the-mouth formula, intended to be an easier form of the medication for children. The study showed that repetitive behaviors were reduced when children were given either NPL-2008 or a placebo, but no differences between groups were found in terms of the level of reduction of repetitive behaviors. Thus, it was concluded that NPL-2008 was not more efficacious than the placebo. The new formulation was generally well tolerated by patients and no serious adverse events were reported.

Geraldine Dawson, Ph.D., Autism Speaks' Chief Science Officer remarked, “The development of effective drugs that are easily taken by individuals with autism is an important goal. While it is disappointing that NPL-2008 was not found to be effective for reducing repetitive behaviors, this study does highlight the importance of high quality, rigorously controlled clinical trials. Studies like this help build the evidence-base and offer parents guidance in making more informed decisions about their child's clinical care.”

The Autism Speaks ACTN was launched in 2005 to address the need for more thoroughly tested biological treatments for autism. The ACTN provides a platform to rigorously test the safety and efficacy of existing compounds, most of which do not have specific indications or specific dosing instructions for their use in autism. These treatments are prescribed by medical practitioners for “off-label” use.

Fluoxetine is one of the most widely prescribed central nervous system (CNS) drugs for disorders such as anxiety, depression, and obsessive-compulsive disorder. It has been prescribed “off-label” by physicians to alleviate repetitive behavioral symptoms in autism. Smaller studies in children and adults showed promising results, but there had not been a large scale study of this type to systematically confirm the efficacy of the drug for reducing these behaviors in autism. Currently, there is only one treatment with a specific FDA-approved indication for autism, but it does not target the core symptoms of autism.

The SOFIA study is the largest trial ever conducted in patients with Autistic Disorder. The study was carried out across 19 ACTN medical centers and clinical sites throughout the US. A total of 158 patients between the ages of 5 and 17 were enrolled into the SOFIA study in which patients received either NPL-2008 or placebo during a 14 week treatment period. The full analysis of the primary and secondary data from the study is ongoing.

Peter Bell, Executive Vice President for Programs and Services commented, “Finding safe and effective medications that treat the core symptoms of autism is one of the greatest challenges facing our community. One of our primary goals at Autism Speaks is to help identify effective treatments that lead to improved quality of life for all those affected by autism.”

Robert Mansfield, Neuropharm's CEO, stated “These initial results are both unexpected and disappointing, and we await the results of the full analysis which is currently ongoing. We would like to thank Autism Speaks' Autism Clinical Trials Network who facilitated the timely completion of this study and to thank the teams at the sites involved in SOFIA.”

For US media inquiries:
Rubenstein Communications, Inc. - Autism Speaks
Jane Rubinstein 212.843.8287

Weber Shandwick-Neuropharm Group Plc.
Traci Mogil
212.445.8113

For UK and European media inquiries:
Buchanan Communications-Neuropharm Group Plc.
Mark Court, Mary-Jane Johnson, Catherine Breen 44 (0) 20 7466 5000 About Autism
Autism is a complex brain disorder that inhibits a person's ability to communicate and develop social relationships, and is often accompanied by behavioral challenges. Autism spectrum disorders are diagnosed in one in 150 children in the United States, affecting four times as many boys as girls. The prevalence of autism has increased tenfold in the last decade. The Centers for Disease Control and Prevention have called autism a national public health crisis whose cause and cure remain unknown.

About Autism Speaks
Autism Speaks is dedicated to increasing awareness of autism spectrum disorders, to funding research into the causes, prevention and treatments for autism, and to advocating for the needs of individuals with autism and their families. It was founded in February 2005 by Suzanne and Bob Wright, the grandparents of a child with autism. Bob Wright is Senior Advisor at Lee Equity Partners and served as vice chairman, General Electric, and chief executive officer of NBC and NBC Universal for more than twenty years. Autism Speaks merged with the Autism Coalition for Research and Education (ACRE), the National Alliance for Autism Research (NAAR) and Cure Autism Now (CAN), bringing together the nation's leading autism advocacy organizations. To learn more about Autism Speaks, please visit www.autismspeaks.org

About the Autism Clinical Trials Network
The Autism Clinical Trials Network (ACTN) is a collaboration of treatment and research centers dedicated to accelerating clinical trials of investigational treatments for autism and to increasing the number of biological treatments available to families and clinicians. For more information, please visit www.autismspeaks.org/ctn

About Neuropharm Group Plc
Neuropharm Group Plc is a specialty pharmaceutical company focused on the development of products for the treatment of disorders of the central nervous system. For more information, please visit www.neuropharm.co.uk. The Neuropharm press release is found at http://www.neuropharm.co.uk/media_centre/news_release/?id=3542.