CAMBRIDGE, Mass., (Embargoed until September 19, 2012, 2:00 PM US Eastern Time) Seaside Therapeutics today announced the publication of two papers in Science Translational Medicine, supporting its lead candidate, STX209 (arbaclofen), for the treatment of fragile X syndrome (FXS). The works presented highlight STX209 as a potential disease-modifying drug in preclinical studies, with improvement in social function in a clinical trial of patients with FXS.
"In preclinical results, we demonstrate that STX209 treatment corrects core aspects of FXS pathophysiology in the fragile X mouse model, leading us to conclude that STX209 is a disease-modifying drug with the potential to significantly improve the lives of patients with fragile X," said Randall Carpenter, MD, President and Chief Executive Officer of Seaside Therapeutics. "In fact, in our Phase 2 clinical trial, we observed statistically significant improvements in social avoidance, a core symptom of fragile X syndrome that is characterized by behaviors such as preference to be alone and being withdrawn or isolated. Social avoidance is also a core symptom of autism spectrum disorders, suggesting STX209 could have a positive effect in this larger patient population. Together, these results support our continued development of STX209, which is currently enrolling patients in a Phase 3 study in fragile X syndrome and recently completed enrollment in a Phase 2b study in autism spectrum disorders."
"There are currently no FDA approved therapeutics that address the core symptoms of fragile X syndrome, leaving patients and their caregivers with limited treatment options," said Elizabeth Berry-Kravis, MD, PhD, Professor of Pediatrics, Neurology, and Biochemistry at Rush University Medical Center in Chicago, and the lead author of the paper on the Phase 2 study results. "We are very excited about the clinically meaningful improvements in social impairment observed to date in patients receiving
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