News Release

Autism Speaks $2 million grant to DELSIA/Seaside Therapeutics to develop autism biomarkers

DELSIA's First Award funded by Autism Speaks Supports Development of Arbaclofen

Grant and Award Announcement

Autism Speaks

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Credit: DELSIA

New York, N.Y. (December 3, 2012) – Autism Speaks and Delivering Scientific Innovation for Autism, LLC (DELSIA), its non-profit affiliate, announced today a partnership between DELSIA and Seaside Therapeutics (Cambridge, Mass.) that aims to discover biomarkers that can be used to facilitate the development of safe, effective and personalized treatments of autism spectrum disorders (ASD).

DELSIA's partnership with Seaside will direct funding allocated by Autism Speaks to an innovative program aimed at identifying genetic and protein biomarkers that can be used to identify patients most likely to benefit from treatment with arbaclofen (STX209). Under the terms of the agreement, DELSIA will provide $2 million in funding to support the biomarkers discovery program at Seaside Therapeutics.

"Autism Speaks is extremely pleased to be funding DELSIA's first award of $2 million to Seaside Therapeutics, a company that has shown a long-standing commitment to developing treatments for neurodevelopmental disorders, including autism spectrum disorder," stated Geraldine Dawson, Ph.D., chair of DELSIA's Board of Managers and chief science officer of Autism Speaks. The partnership with Seaside represents the inaugural award for Autism Speaks' newly established venture philanthropy affiliate DELSIA.

"We are enormously excited to be partnering with Seaside, a company at the forefront of autism medicines development in the biotechnology industry," said Robert Ring, Ph.D., president of DELSIA and vice president of translational research at Autism Speaks. "Our partnership will help generate critical information that can be used to pinpoint which individuals with autism are most likely to respond to arbaclofen, one of the first treatments that has the potential to address the core social impairment associated with autism. This knowledge will have broader implications down the road for developing effective treatments for people with autism."

"Recent scientific discoveries have identified several potential treatments for the underlying causes of autism spectrum disorders. However, the broad heterogeneity of ASD suggests that optimal treatment will need to be personalized," said Randy Carpenter, CEO of Seaside Therapeutics. "Identification of biomarkers would enable development of targeted treatments and is a first step toward realizing personalized medicine for individuals with ASD."

"Arbaclofen is the most advanced program in the autism clinical pipeline, and if successful, could deliver the first medical therapy for a core symptom of autism," says Ring. Seaside Therapeutics is developing arbaclofen (STX209), an oral selective gamma-amino butyric acid type B (GABA-B) receptor agonist, as a treatment to improve social and communication functions in autism and Fragile X syndrome (FXS). Earlier this year, Seaside entered into an alliance with the Swiss pharmaceutical company Roche (SIX: RO, ROG; OTCQX: RHHBY) to expand its efforts to develop disease modifying treatments for FXS and ASD.

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About Autism

Autism is a general term used to describe a group of complex developmental brain disorders – autism spectrum disorders (ASD) – caused by a combination of genes and environmental influences. These disorders are characterized, in varying degrees, by communication difficulties, social and behavioral challenges, as well as repetitive behaviors. An estimated 1 in 88 children in the U.S. is on the autism spectrum – a 78 percent increase in six years that is only partly explained by improved diagnosis.

About DELSIA

Delivering Scientific Innovation for Autism LLC (DELSIA), a not for profit venture philanthropy affiliate of Autism Speaks, is focused on transforming outcomes for individuals with autism by ensuring that scientific breakthroughs are developed into products that deliver meaningful improvements in the quality of life and health. DELSIA was established in October of 2012 and works with partners in the for-profit sector to fund development activities best poised to deliver products aligned with unmet medical and behavioral health needs of the autism community. For more information please visit www.delsia.org

About Autism Speaks

Autism Speaks is the world's leading autism science and advocacy organization. It is dedicated to funding research into the causes, prevention, treatments and a cure for autism; increasing awareness of autism spectrum disorders; and advocating for the needs of individuals with autism and their families. Autism Speaks was founded in February 2005 by Suzanne and Bob Wright, the grandparents of a child with autism. Mr. Wright is the former vice chairman of General Electric and chief executive officer of NBC and NBC Universal. Since its inception, Autism Speaks has committed over $180 million to research and developing innovative resources for families. Each year Walk Now for Autism Speaks events are held in more than 95 cities across North America. On the global front, Autism Speaks has established partnerships and related activities in over 40 countries on 5 continents to foster international research, services and awareness. To learn more about Autism Speaks, please visit AutismSpeaks.org.

About Seaside Therapeutics

Seaside Therapeutics is working to correct or improve the course of autism, fragile X syndrome and other neurodevelopmental disorders by translating breakthrough discoveries in neurobiology into therapeutics that will improve the lives of patients and their families. While there are treatments that alleviate some symptoms of neurodevelopmental disorders, there are currently none that address the underlying causes. Seaside Therapeutics is using a scientific approach to address this unmet need and is changing the landscape of drug development by focusing on single-gene disorders with a high prevalence of autism, such as fragile X syndrome. www.seasidetherapeutics.com


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