Key trial of Seaside autism drug fails to show benefit

Wed May 1, 2013 11:59am EDT

* Drug missed main goal of improving social withdrawal

* Company says trial offered better idea of who would benefit

* Plans for new trial of drug already under way

By Julie Steenhuysen

CHICAGO, May 1 (Reuters) - The first-ever drug designed to treat social impairments associated with autism failed to show a benefit in a midstage trial, representing a blow to families and to privately held drugmaker Seaside Therapeutics.

Results of the study, presented on Wednesday at the International Meeting for Autism Research in Spain, showed the drug known as STX209 failed to improve symptoms of social withdrawal in a 12-month study of 150 individuals with a wide range of symptoms and impairment.

Although the drug missed its main goal, Dr. Randall Carpenter, president and chief executive officer of Cambridge, Massachusetts-based Seaside, said in an interview the study did show a benefit in some patients, especially among individuals with higher IQ.

Carpenter said Seaside has already met with the U.S. Food and Drug Administration to devise a new study based on the current trial's findings that he believes may yet lead to the drug's approval.

"We believe our drug works at a molecular level that corrects signaling pathway abnormalities, circuit abnormalities and even anatomical abnormalities," Carpenter said.

"So the real question for us is if you have a disease-modifying therapeutic, how would you demonstrate that in a short term clinical trial," Carpenter said.

Seaside's drug STX209, or arbaclofen, works to control an overabundance of signaling or "noise" at brain synapses by reducing the amount of the neurotransmitter glutamate that is available in the brain.

The drug is the first of a handful of treatments in development that are expressly designed to correct the genetically induced signaling problems in the brain that result in autism.

Other companies in the space include Swiss drugmakers Novartis AG and Roche Holding AG, which last year licensed patents from Seaside for the use of drugs known as mGluR5 antagonists that attack the signaling problem in a different way.

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