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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