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New Alliance for Developing Autism Medicines

June 20, 2012


This week, two major developers of autism medicines announced a partnership to speed the development of their treatments. The alliance involves the global pharmaceutical company Roche and the privately held Seaside Therapeutics. Seaside specializes in developing medicines for neurodevelopmental disorders.

With this deal, the companies combine forces to develop medicines to relieve the core symptoms of autism and fragile X syndrome. Fragile X is the most commonly inherited cause of autism.

“This strategic partnership is a healthy sign that we have a rapidly growing commitment to the development of autism medicines,” says Autism Speaks Vice President of Translational Research Rob Ring, Ph.D. “It brings together intellectual property and know-how from both companies in a way that ensures a more rapid development of an important class of experimental compounds called mGluR5 antagonists. That’s good for autism science and encouraging for families waiting for evidence-based treatments.”

This deal also provides Seaside with financial support to complete two late-stage clinical trials for a separate type of autism medicine. This agent is STX209, also known as arbaclofen. It comes from a class of compounds called GABA-B receptor agonists. These compounds help normalize levels of glutamate, an important brain chemical associated with autism symptoms. Seaside’s clinical trials for STX209 are scheduled to complete within the next year. If positive, STX209 could become the first evidence-based treatment for a core symptom of autism – social withdrawal.

“All eyes are watching the STX209 trials,” Ring adds.

Through its Translational Research Program, Autism Speaks is playing a growing role in medicine development. “It’s important that we ensure that the medical products being developed by pharmaceutical companies are squarely aligned with the unmet needs of our community,” Dr. Ring says.

These efforts include Autism Speaks’ participation in EU-AIMS, the world’s largest study autism medicines in development. In addition, Dr. Ring and Autism Speaks Chief Science Officer Geri Dawson, Ph.D., recently participated in a global neuroscience symposium cosponsored by Roche and the journal Nature Medicine. (Read more in Dr. Dawson’s recent blog.)

Prior to joining Autism Speaks, Dr. Ring was actively involved in groundbreaking research on mGluR5 antagonists for the treatment of autism. This work was recently featured in the journal Science Translational Medicine.

Autism Speaks is currently funding a number of research studies on the brain chemistry of autism and related treatments. You can explore these and other research using our Grant Search