ACADIA Pharmaceuticals

Neuren Pharmaceuticals Launches First-Ever Phase 3 Trial for Phelan-McDermid Syndrome

- Neuren Pharmaceuticals has initiated the first investigational site in the United States for its Phase 3 clinical trial of NNZ-2591 for Phelan-McDermid syndrome, marking the first-ever Phase 3 trial for this severe neurodevelopmental disorder. - The randomized, double-blind, placebo-controlled trial will assess 13 weeks of treatment in approximately 160 children aged 3 to 12 with PMS, with all participants eligible for a 12-month open-label extension. - Phelan-McDermid syndrome affects an estimated 1 in 8,000 to 1 in 15,000 people and currently has no approved treatments available. - The trial program is fully funded by Neuren's existing cash reserves, following FDA alignment on the Phase 3 trial design including primary efficacy endpoints.