Seaside Therapeutics reports positive data from STX209 Phase 2 study in fragile X syndrome

Seaside Therapeutics, Inc. announced today data from the largest randomized, placebo-controlled study conducted to date in individuals with fragile X syndrome. In a Phase 2 study of STX209, clinically meaningful improvements on global and specific neurobehavioral outcomes were observed in the general study population. The improvements were statistically significant in pediatric patients with more severe impairments in sociability -- a core symptom of fragile X syndrome. STX209 is a selective gamma-amino butyric acid type B (GABA-B) receptor agonist. The results were presented in a podium presentation at the National Fragile X Foundation's 12th International Fragile X Conference in Michigan on Saturday, July 24, 2010 by investigators Elizabeth Berry-Kravis, MD, PhD, Professor of Pediatrics, Neurological Sciences and Biochemistry at Rush University Medical Center in Chicago, Illinois and Randi Hagerman, MD, Medical Director, M.I.N.D. Institute, Professor, Endowed Chair in Fragile X Research, School of Medicine, University of California, Davis, in Sacramento, California. Seaside continues to conduct additional analyses of other outcome measures and biomarker data sets and intends to submit full results for publication.

“Seaside Therapeutics conducted one of the most comprehensive studies to date in a neurodevelopmental disorder and achieved our goal of increasing knowledge regarding potential efficacy measures in patients with fragile X syndrome”

"A majority of the patients enrolled in the STX209 study are participating in the ongoing open-label extension study and are continuing to benefit from treatment with STX209," said Dr. Randi Hagerman. "Physicians and parents are reporting increased sociability and communication and decreased outbursts and tantrums. In several cases, patients have been successfully withdrawn from other medications, including mood stabilizers, anti-depressants and, most importantly, anti-psychotics—a significant benefit for patients given the severe side effects associated with this particular class of drug. It is my hope that, with further study, STX209 may be able to play a much needed role in improving the symptoms of fragile X syndrome and help patients and their families achieve an improved quality of life."

"Seaside Therapeutics conducted one of the most comprehensive studies to date in a neurodevelopmental disorder and achieved our goal of increasing knowledge regarding potential efficacy measures in patients with fragile X syndrome," said Randall L. Carpenter, MD, President and Chief Executive Officer of Seaside Therapeutics. "The study assessed a broad range of behavioral and cognitive outcomes and, as would be expected in a study of this scope, we observed significant benefit in some key areas but not in others. We believe the depth of data coming from this study will be of tremendous benefit for the field and will inform ongoing discussions with both clinicians and the FDA to confirm the most appropriate outcomes to measure efficacy in individuals with fragile X syndrome. We look forward to initiating late-stage clinical trials of STX209 later this year after discussions with the FDA."