Cambridge, MA-based Seaside Therapeutics is emerging from stealth mode this morning with an announcement that it has raised $30 million to pursue mid-stage clinical trial development on new therapies for Fragile X Syndrome and autism. Founded back in 2005 and built around an early-stage program licensed in from the defunct Sention, Seaside's 22 staffers are pursuing one of the most puzzling diseases on the planet with an approach that they believe has the potential to significantly improve the lives of autistic patients and their families.
Seaside isn't your average biotech, as CEO Randall L. Carpenter, M.D., made clear in an interview with FierceBiotech. This new venture money was put up by an unnamed family investment firm that has provided the bulk of the $66 million raised to date. And the firm has committed to fund the company through to profitability, "if necessary."
"It allows us to decouple ourselves from the market," says Carpenter, who has been working with Mark Bear, a neuroscience professor at MIT, on developing these new therapies. Seaside is currently enrolling patients in two trials for Fragile X and autism. To date, says Carpenter, only one drug--the antipsychotic risperidone--has been approved for autism symptoms. And the initial efficacy endpoint that they'll be studying is the same: to improve irritability in children and adolescents who suffer from autism. But Carpenter says Seaside's therapeutic approach has the potential to do much more.
"It may allow individuals to speak, to learn normally," he explains. "It may enhance their ability to relate to the environment, be more calm and less anxious and potentially more interactive. We're seeing profound effects in our animal models; how that translates to humans is what we'll find out in the next year or two."
The company's lead therapy, STX209--which inhibits glutamate signaling in the brain--entered a Phase II clinical study in adults and adolescents with Fragile X in December 2008 and a second trial in adolescents with autism spectrum disorders was launched in March 2009. Seaside intends to expand both studies to include children as young as six years old during 2009. Data from both Phase II studies is expected in early 2010.
- check out Seaside's release