Seaside Therapeutics fails to achieve main goal in major autism trial

Seaside Therapeutics missed the main target of a midstage trial of its lead drug STX209 in patients with autism spectrum disorder (ASD), yet benefits of treatment observed in the Phase IIb study have driven the company to plan another major clinical trial in patients with the intellectual disorder.

Randall Carpenter, CEO of Seaside Therapeutics

Cambridge, MA-based Seaside, a 2012 Fierce 15 company, has been one of the few biotech outfits to focus on new therapies for autism, which affects millions of Americans who have very few treatment options. Last year Seaside jumped into the spotlight as the drug giant Roche ($RHHBY) licensed compounds from the biotech and gained options to commercialize STX209.

With the exception of Roche and others, pharma companies have largely shunned the autism field because of the complexity of the illness and the perceived risks of embarking on drug development. Seaside has found most of its support from deep-pocketed advocates, pioneering research on drugs such as STX209 that offer the possibility of combating the core behavioral symptoms of the disease.

The 150-patient Phase IIb study of STX209 in ASD is one of the largest clinical trials conducted in autism, Seaside CEO Randy Carpenter told FierceBiotech. And though the drug failed to beat placebo on the main endpoint of social withdrawal, patients on the drug showed significant improvements in other measures, such as one that gauges social IQs called the Vineland-II Socialization scale.

The company aims to use the measure of social function in a planned Phase II/III trial for STX209 in ASD, with a potential start of the study late in 2013, Carpenter said. It expects to reveal results from late-stage work on the drug in a related disorder called fragile X syndrome later this year.

Asked for his overall feelings about the failed Phase IIb study, Carpenter said: "My biggest disappointment is the lack of investment by others in this field and that we're out there by ourselves hacking our way through the jungle. I thought that other people would be actively engaged in advancing this field to a greater extent than they are at this point."

- here's the press release

Special Report: Seaside Therapeutics – 2012 Fierce 15