Seaside Therapeutics Secures $30 Million Financing

Thu Sep 17, 2009 8:00am EDT

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Funding to Support Clinical Studies in Autism and Fragile X Syndrome 

CAMBRIDGE, Mass.--(Business Wire)--
Seaside Therapeutics LLC today announced 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. 

About Seaside Therapeutics

Seaside Therapeutics is creating new drug treatments to correct or improve the
course of Fragile X Syndrome, autism and other disorders of brain development.
We are dedicated to translating breakthrough discoveries in neurobiology into
therapeutics that improve the lives of patients and their families.

MacDougall Biomedical Communications
Sarah Cavanaugh/Kari Watson, 781-235-3060 

Copyright Business Wire 2009