Seaside Therapeutics to move autism drug to Phase 3 trials

By Julie M. Donnelly

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Seaside Therapeutics Inc. has issued positive results for a second Phase 2 clinical trial on its drug candidate to treat autism.

While the first study focused on fragile X syndrome, the most common cause of autism, the second study expanded the indication to a variety of autism spectrum disorders. The company has decided to move forward with Phase 3 trials for the potential therapy, called STX209, in both indications. The Cambridge-based biotechnology company said that the study showed a marked improvement in patients’ symptoms, including irritability and social withdrawal.

“These study results add to a growing body of evidence supporting the potential of STX209 to play an important role in treating neurodevelopment disorders such as fragile X syndrome and autism spectrum disorders,” said Randall L. Carpenter, CEO of Seaside Therapeutics, in a statement. “In addition, we have now observed significant improvement in social interaction across two studies. We believe the reduction in social withdrawal is important as it suggests that STX209 is demonstrating efficacy for a core symptom of both fragile X syndrome and autism.”

The second trial for the broader therapeutic indication was conducted as a so-called open label study, which means there was no control group taking a placebo. This type of study is considered to be less scientifically rigorous. The first study, focusing on fragile X, was a placebo-controlled study.

Thirty-two patients with autism spectrum disorders between the ages of 6 and 17 were enrolled in the study. Two patients withdrew from the study due to a worsening of their symptoms. Another patient reported a serious adverse event, a worsening of aggression, as the patient transitioned off the drug.

Seaside won a U.S. patent in February for the method of treating autism.

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