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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.
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