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.
The drug bested placebo on two metrics judged by a patient’s caregiver and physician, respectively, called the Rett Syndrome Behaviour Questionnaire and the Clinical Global Impression–Improvement (CGI-I) assessment. Both assessments determined improvements in patients’ symptoms over the 12-week duration of the study.
On the RSBQ assessment, patients’ scores dropped an average of 5.1 points versus a 1.7-point decrease in the control (p=0.0175). Meanwhile, the CGI-I test showed a 3.5-point change for patients in the trofinetide arm versus 3.8 for placebo (p=0.003).
Taking home a late-stage win in Rett syndrome is a good look for Acadia, and investors cheered the news with the company’s stock $ACAD trading at just above $20 early Tuesday, up roughly 5% from Monday’s closing price.
Acadia now plans to take its data in front of the FDA by the middle of next year, with a pre-NDA meeting scheduled for the first quarter, it said. Trofinetide has received fast track designation from the agency and would be eligible for priority review.
Acadia is one of a group of drugmakers looking to secure the first-approved therapy for Rett syndrome in the US. The disease is one of a suite of rare neurological conditions that has stumped drug developers for years, but this late-stage win — and a potential positive review — could put some wind in the sails of Acadia’s competitors in this space.
Those players include Anavex, which also has a fast track designation for its blarcamesine, and gene therapy startup Alcyone, which launched this summer, among others. But the field has also been ripe with failure. In 2020, Newron Pharma dropped its own late-stage Rett program sarizotan after the drug flopped a Phase II/III trial.