UPDATED: Cash-strapped biotech fails PhIII test for pioneering Fragile X drug

Randall Carpenter, CEO of Seaside Therapeutics

Seaside Therapeutics has revealed its second failed study for its lead drug STX209 this month. In one of two Phase III studies for the compound for the genetic mental disorder Fragile X syndrome, STX209 treatment missed its main goal of reducing social withdrawal. 

The Phase III misfire comes on the heels of a failed Phase IIb study of the compound in autism spectrum disorder, and the setbacks could have financial ramifications for privately held Seaside (a 2012 Fierce 15 company) because Roche ($RHHBY) has options to commercialize the therapies if approved. Seaside's lead drug has aimed to be the first to combat a core molecular trigger of the diseases.  

The small biotech's financial troubles have already been felt in the autism and Fragile X communities. The company has pulled the plug on two extension studies of STX209 for patients with the two related disorders, saying in letters on its website that the company can no longer afford the research because of "resource limitations." On Tuesday Pharmalot reported that advocates have begun a petition to revive Seaside's research.

In a report about Roche's deal with Seaside in June 2012, The New York Times reported that most of the $90 million Seaside had raised was from the Barony Trust, a private investment outfit. Seaside CEO Randy Carpenter has declined numerous questions about the financial details of his company from FierceBiotech.

As of early this month, the company planned to seek further development of STX209 in autism based on evidence of positive impact on patients' social competence in the failed Phase IIb study, Carpenter said in an interview. However, he also sounded concerned about the amount of support for drug development in the autism field.

"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," Carpenter said in the May 1 interview.

Roche and Novartis ($NVS) are among the relatively few pharma companies developing drugs for Fragile X syndrome. The disease, which causes intellectual disabilities, is caused by mutations in the FMR1 gene and affects one in about 3,800 males and approximately 5,000 females, according to the National Fragile X Foundation. 

Seaside plans to complete a second ongoing Phase III study of STX209 (arbaclofen) in Fragile X patients later this year. The program grew from the research of company co-founder Mark Bear at MIT. And the drug aims to restore normal protein synthesis in the brain cells of patients with Fragile X and autism.

- here's Seaside's note to the Fragile X community
- and the note to the autism community
- see Pharmalot's article

Special Report: Seaside Therapeutics – 2012 Fierce 15

Clarification: This article was updated and removed a line that stated that Pharmalot's Tuesday report did not pick up the news about the Phase III trial failure. The Phase III failure was made known on Wednesday on Seaside's website, after Pharmalot's report about the termination of the extension studies of STX209 on Tuesday.