Massachusetts company receives $30 million to research autism, Fragile X Syndrome

From BusinessWire:

CAMBRIDGE, Mass. -- Seaside Therapeutics LLC announced Sept. 17 that it has secured $30 million in financing from a private, family investment firm which is committed to advancing research in the field of autism and Fragile X Syndrome. The financing will be used to fund the Company`s pipeline of novel therapeutic candidates to correct or improve the course of those who suffer these disorders. The financing brings the total capital raised by Seaside to $66 million.

"Seaside understands the toll that brain development disorders take on individuals and their families and shares in the frustration over the lack of effective therapeutics for these devastating disorders," said Randall L. Carpenter, M.D., President and Chief Executive Officer of Seaside Therapeutics. "Seaside was founded to fill this void by translating breakthrough discoveries in neurobiology into therapeutics that improve the lives of patients and their families. This significant financial commitment will support our ongoing Phase 2 studies of STX209 in both autism and Fragile X and support the initiation of a second novel clinical candidate, STX107, into Phase 1 studies in Fragile X in early October."

Historically, drug discovery in disorders of brain development has been unproductive largely due to the lack of mechanistic understanding of these disorders, as well as the absence of predictive animal models. Seaside Therapeutics is changing this paradigm through scientific exploration that focuses on identifying the fundamental pathophysiology of brain development disorders and applying this knowledge to develop targeted therapeutics. Recent discoveries by the Company's scientific founder, Mark Bear, Ph.D., Howard Hughes Medical Institute Investigator and Professor of Neuroscience at M.I.T., have revealed a molecular pathway, the mGluR5 signaling cascade, that is disrupted in a specific disorder of brain development - Fragile X Syndrome. With this knowledge, further research has provided insights for developing novel medications to normalize the function of this pathway, which Seaside believes may extend beyond Fragile X into a number of other developmental disorders, including autism.

STX209 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 the excessive metabotropic glutamate receptor (mGluR) mediated protein synthesis implicated in Fragile X Syndrome. Preclinical studies using STX209 and other prototypic GABA agonists have demonstrated efficacy in animal models of Fragile X, suggesting that GABA agonists may provide significant benefits to people with Fragile X Syndrome and other disorders of brain development. STX209 entered a Phase 2 clinical study in adults and adolescents with Fragile X in December 2008 and a second trial in adolescents with autism spectrum disorders was initiated in March 2009. Seaside intends to expand both studies to include children as young as 6 years old during 2009. Data from both Phase 2 studies is expected in the first quarter of 2010.

STX107 is a highly potent, selective mGluR5 antagonist that was licensed from Merck & Company, Inc. STX107 was selected for development based on Dr. Bear`s discovery of the 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. Preclinical research indicates that normalizing this exaggerated mGluR5 signaling reverses most of the anatomic, behavioral and synaptic abnormalities associated with Fragile X. By directly inhibiting exaggerated mGluR5 signaling, STX107 provides a compelling opportunity to treat core symptoms and disabilities of 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 Mental Health, the National Institute of Child Health and Human Development,the National Institute of Neurological Disorders and Stroke, Autism Speaks,FRAXA and the Best Pharmaceuticals for Children Act. STX107 is expected to enter Phase 1 clinical studies in healthy volunteers in October 2009.