Steve Davis, Acadia CEO

Aca­dia touts late-stage win for its Rett syn­drome drug, edg­ing out a lead against its com­peti­tors

Rare neu­ro­log­i­cal dis­or­ders have long proved dif­fi­cult for drug de­vel­op­ers, with huge R&D bud­gets and com­mer­cial plans of­ten go­ing bust at the very last minute. Now, Aca­dia Phar­ma is tout­ing a win for its late-stage can­di­date in Rett syn­drome, which could help juice its com­peti­tors’ ef­forts as well.

Aca­dia’s trofine­tide hit its co-pri­ma­ry end­points in Phase III study in 187 girls and young women with the rare neu­ro­log­i­cal dis­ease Rett syn­drome, which im­pacts be­tween 6,000 and 9,000 US pa­tients and is of­ten mis­di­ag­nosed as autism, cere­bral pal­sy or non-spe­cif­ic de­vel­op­men­tal de­lay, the com­pa­ny said Tues­day.

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