People with Fragile X - Research Studies

Currently Seaside has two compounds in active study for the treatment of Fragile X:
STX209 and STX107.

STX209

Overview
STX209 (arbaclofen) is a selective gamma-amino butyric acid type B (GABA-B) receptor agonist. STX209 inhibits glutamate signaling in the brain and should thereby indirectly inhibit excessive metabotropic glutamate receptor (mGluR) mediated protein synthesis in Fragile X Syndrome. Preclinical studies using STX209 and other prototypic GABA agonists in animal models of Fragile X have demonstrated profound efficacy, suggesting that GABA agonists may provide significant benefits to people with Fragile X Syndrome and other disorders of brain development.

Program Status
Seaside has completed Phase 1 testing in healthy volunteers and initiated a Phase 2 clinical trial in adults and adolescents with Fragile X Syndrome in late 2008. For more information on the Phase 2 study please visit www.clinicaltrials.gov. Assuming an acceptable safety and efficacy profile, the ultimate goal would be to expand the study to include younger children.

STX107

Overview
STX107 is a highly potent, selective mGluR5 antagonist in-licensed from Merck. Seaside Therapeutics' scientific founder Dr. Mark Bear discovered a connection between mGluR5 signaling and Fragile X Syndrome. Specifically, the evidence suggests that most if not all of the neurological and psychiatric consequences of Fragile X can be accounted for by exaggerated signaling through mGluR5 receptors. MGluR5 antagonists provide a compelling opportunity to treat symptoms associated with Fragile X Syndrome, autism and other developmental disorders. Seaside has been awarded translational research grants to support the development of STX107 from the National Institute of Health including the National Institute of Mental Health, the National Institute of Child Health and Human Development, National Institute of Neurological Disorders and Stroke, Autism Speaks, FRAXA and the Best Phamaceuticals for Children Act.

Program Status
In collaboration with Merck and NIH scientists, Seaside has completed investigational new drug (IND) enabling studies. STX107 entered Phase 1 clinical studies in healthy volunteers in October 2009. For more information on the Phase 1 study please visit www.clinicaltrials.gov.