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Roche, Seaside Therapeutics to Partner on Autism, Fragile X

Children in a Tel Aviv park (A. Kotok)

(A. Kotok)

Seaside Therapeutics in Cambridge, Massachusetts and the Swiss pharmaceutical company Roche will collaborate to develop treatments for autism spectrum disorders and fragile X syndrome. Financial terms of the agreement were not disclosed.

Autism spectrum disorders and fragile X syndrome are neurodevelopmental disorders for which there are no effective treatments yet that address core symptoms. Autism is a complex developmental disability that causes problems for children, usually before age 3, with social interaction and communication. The disorder can cause delays or problems with many different skills that extend into adulthood. Autism spectrum disorder is a group of similar conditions, including Asperger syndrome and related pervasive developmental disorders.

Fragile x syndrome is the most common form of inherited intellectual disability in boys. The condition involves changes in part of the X chromosome caused by a change in a gene called FMR1 that makes a protein needed for the brain to grow properly. A defect in the gene makes the body produce too little of the protein, or none at all.

The Roche/Seaside partnership aims to develop treatments that target the molecular basis of these disorders, and thus relieve their core symptoms. Both companies have been developing therapies for treating the overactive nerve-cell receptors in the brain that believed to be at the root of the conditions. Seaside will license to Roche exclusive rights to its patents for compounds called antagonists aimed at treating these overactive receptors that are similar to therapies developed by Roche.

Seaside is developing other treatments taking a different approach to treating these conditions. One of Seaside’s therapy candidates, known as STX209, completed enrollment on a phase 2 trial for autism spectrum disorders and has begun enrollment for a phase 3 trial to treat fragile X syndrome. While Seaside will continue to lead the clinical development of these programs, Roche will have the option to commercialize STX209 after certain clinical stages are met.

“Recent discoveries in genetics,” says Luca Santarelli, Global Head of Roche Neuroscience, “have shed light on the biological underpinnings of these conditions thus providing a basis for mechanistic drug discovery.” Randy Carpenter, president of Seaside Therapeutics adds that the collaboration “provides Seaside with additional resources to complete late-stage clinical development of STX209, which we believe has the potential to change the treatment paradigm for fragile X and autism and thereby help patients and their families achieve an improved quality of life.”

Updated, with fixed and new links, 9 July 2013

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