"We anticipate potential top-line results from this trial could be presented in H2 2014," analyst Christopher Marai says, referring to news that RNA has dosed the first patient in a Phase 1/2 trial of PRO053.
"The market has broadly overlooked the opportunity for RNA's follow-on compounds," Marai adds, before predicting "approval worthy" Phase 3 data (presumably for drisapersen) in Q4 of this year.
"The big risk to the outcome of the Phase 3 study... is the effect that younger DMD boys (between 5 and 7) and those with greater walking ability at baseline will have on the overall results," Adam Feuerstein said Friday. "What we know now, but what GSK and RNA say they didn't know when the phase 3 study was started, is that DMD kids younger than 7 are generally still capable of improvements in six-minute walk distance without treatment."
Wedbush's price target is $50.