Ovid's lead drug shows promis­ing ef­fi­ca­cy sig­nal in Frag­ile X syn­drome study, but op­ti­mal dos­ing reg­i­men is un­clear for path for­ward

Take­da and Lund­beck-part­nered rare neu­ro­log­i­cal dis­or­der com­pa­ny Ovid Ther­a­peu­tics has some en­cour­ag­ing mid-stage da­ta from a Frag­ile X study test­ing its lead drug, but there re­main ques­tions about the op­ti­mal dose to take for­ward in­to a piv­otal tri­al.

Three reg­i­mens of the drug, OV101, (5 mg once-dai­ly, twice-dai­ly and thrice-dai­ly) were ran­dom­ized in 23 male pa­tients with a con­firmed di­ag­no­sis of Frag­ile X syn­drome (FXS), the most com­mon in­her­it­ed form of in­tel­lec­tu­al dis­abil­i­ty and autism and is caused by a mu­ta­tion in the FMR1 gene. The con­di­tion has no ap­proved ther­a­pies so far.

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