Searching for the X factor

One of the challenges of working in many Orphan diseases is the lack of validated endpoints. Thus when Seaside Therapeutics Inc. set out to run a Phase II trial in fragile X syndrome, it sought to study potential assessment tools as well as its compound.

Last week, the company reported that STX209 arbaclofen showed clinically meaningful but not statistically significant improvements on some global and neurobehavioral outcomes vs. placebo. But even though the GABA B receptor agonist missed the primary endpoint measuring irritability, Seaside thinks it now has enough information to devise a pivotal trial by year end...