Seaside Therapeutics Inc. has announced positive results from a Phase 2 trial for a drug candidate to treat fragile X, the most common form of autism.
Officials at the Cambridge-based company say it is the largest study of its kind on the syndrome and produced improvements on specific neurobehavioral metrics.
Researchers said that the improvements were statistically significant in children with more severe impairments in sociability -- a core symptom of fragile X syndrome. The potential drug , called STX209, is designed to correct a molecular pathway that is disrupted in patients with fragile X.
According to the Centers for Disease Control, an estimated one in 4,000 males and one in 6,000 to 8,000 females have fragile X syndrome.
“We believe the depth of data coming from this study will be of tremendous benefit for the field and will inform ongoing discussions with both clinicians and the FDA to confirm the most appropriate outcomes to measure efficacy in individuals with fragile X syndrome,” Randall L. Carpenter, president and CEO of Seaside Therapeutics, said in a statement.
The company plans to launch a late-stage trial of the drug target later this year after discussions with the Food and Drug Administration.