080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

Tackling Fragile X Seaside’s unique funding stream enables progress in treatments for fragile X and autism

Lisa M. Jarvis, pubs.acs.org |

At the annual fragile X conference in Dearborn, Mich., in July, executives from Seaside Therapeutics found themselves confronted by a desperate mother. Her son was enrolled in a clinical study of STX209, a drug the small biotech firm is developing to treat the neurological disorder fragile X, and she had driven across several states to make a direct plea to the company: Please extend the trial, or at least provide medication to kids who participated.

“She didn’t know what she would do if it stopped,” recalls Seaside’s chief executive officer, Randy Carpenter. Indeed, preliminary results from the Phase II trial suggest the drug is having a positive effect on the behavior of kids with fragile X, which is caused by a mutation in a single gene and is also the most common known genetic cause of autism.

Seaside was able to convince the Food & Drug Administration to approve an open-label extension of the trial so that children could continue taking the drug. Nearly all of them have stayed on it. Like the mother at the conference, parents are in despair over the lack of treatments for fragile X, symptoms of which range from learning impairment to mental retardation.

Until recent interest by big pharma, Seaside was pretty much the only company developing drugs for fragile X and autism. Other small start-ups focusing on the diseases have one by one run out of money and closed up shop. Venture capitalists simply haven’t had an appetite to fund such risky drug development.

But Cambridge, Mass.-based Seaside has a secret weapon: an anonymous wealthy family that is committed to finding new treatments for fragile X and autism. With funds from the family and grants from the National Institutes of Health and patient advocacy organizations, Seaside has been able to explore the basic science of the brain disorders in tandem with its drug development efforts. Although unusual, that balanced approach to building up a scientific and clinical rationale looks like it’s working. In just five years, the firm’s two drug candidates have made it to mid-stage trials as a treatment for fragile X, where they show promise, and to early-stage tests on autism.

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