BioCentury
ARTICLE | Clinical News

Seaside Therapeutics, Roche preclinical data

September 24, 2012 7:00 AM UTC

In vitro application of STX209 to the hippocampus of mice lacking the fragile X mental retardation 1 (FMR1) gene significantly reduced levels of local protein synthesis to levels found in the hippocampus of normal mice (p<0.001), but showed no significant effect in wild-type mice. In FMR1-knockout mouse neurons in culture, STX209 significantly reduced AMPA receptor internalization, a known functional consequence of increased protein synthesis according to Seaside, to levels found in normal mice (p<0.001).

In a mouse model of fragile X syndrome, STX209 significantly reduced seizure frequency compared to vehicle-treated controls (p<0.0001). Furthermore, STX209 corrected alterations of neuronal morphology specific to FMR1-deficient mice and patients with fragile X syndrome, but did not alter neuronal morphology in normal mice. Based on the results, Seaside said that STX209 has the potential to be disease modifying in fragile X syndrome patients. Data were published in Science Translational Medicine. ...