Seaside Therapeutics, a stealthy biotech startup in Cambridge, MA, has raised $30 million to further develop research from MIT neuroscientist Mark Bear that has the potential to create the first drugs that treat the underlying neurological disorder at work in patients with Fragile X syndrome and autism.
Seaside raised the money from a private, family investment firm that wishes to remain anonymous, says CEO Randy Carpenter. The company, founded in 2005, has now raised a total of $66 million from the family, the National Institutes of Health, and disease foundations like Autism Speaks and the Fragile X Research Foundation, Carpenter says. It hasn’t raised venture capital, and it hasn’t needed any, he says.
“We’re thrilled,” Carpenter says. “The end game for us is not to sell the company for a profit. The end game for us is to develop effective new therapies.”
Autism has been surging in public awareness in recent years and is now estimated to affect one out of every 150 children in the U.S. Yet autism has stumped scientists for generations, because they don’t really know what causes it, or how to classify all the various symptoms like social isolation, obsessions like staring at ceiling fans, or having trouble with language. That’s made it hard for pharmaceutical and biotech companies to even know where to begin with strategies to develop new therapies. But that’s beginning to change, partly because of work done by Bear, a co-founder of Seaside, Carpenter says.
Carpenter and Bear collaborated together earlier this decade at Sention, a Providence, RI-based company that eventually went out of business. But Carpenter says Sention had a “skunk works” project on Fragile X and autism that eventually gave birth to Seaside. The newer company is built on the idea of treating Fragile X syndrome—a genetic abnormality that leads to mental retardation and about 5 percent of autism cases—by designing drugs to restore normal function of a molecular pathway called mGluR5. This target has long been on the radar screens of major drug companies like Novartis, Roche, and AstraZeneca, but usually as a potential target for treating chronic heartburn, pain, anxiety, or other neurological disorders, Carpenter says.
Researchers will soon get an idea of whether Seaside is on the right track. It has an oral pill already in a pair of mid-stage clinical trials—one in adults and adolescents with Fragile X syndrome, and the second in adolescents with a spectrum of autism disorders. That drug, STX209, is expected to show results by the end of March 2010, Carpenter says.
A second drug, STX107, was licensed from Merck, and is poised to enter its first trial in healthy volunteers next month, Carpenter says.
Seaside has 22 employees now, and Carpenter says it will look to use some of its new cash to hire “a few” more with expertise in clinical development and regulatory affairs. The company plans to continue building up in-house expertise for drug development, frankly, because it doesn’t see any other way.
That’s part of the challenge when a drug company tries to blaze an entirely new trail. Seaside has had to do a lot of difficult labor in establishing proper goals for clinical trials, objective scientific measurements of progress with regard to behavior or language—the kind of things that would convince doctors that the clinical trial was valid, and not some fluke. Common diseases like cancer have well-established endpoints, but with autism, Seaside will have to work with the FDA and leading physicians to craft the best ways to assess progress. “We’ve been training a lot of people,” Carpenter says.
Seaside may seek help from pharmaceutical and biotech partners down the road, especially if it gets to the point where it needs to distribute an effective new autism drug beyond just the wealthy nations of North America and Europe. Seaside feels it has an obligation to bring its work to a broader group of patients who need its therapy, and is willing to find partners who can help it do that, Carpenter says.
“We have the resources we need now, and in the future, but if a partner allows us to get to market sooner, we’d consider that,” Carpenter says.
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