Its lead product trofinetide is licensed to Acadia Pharmaceuticals for North America, with Neuren retaining all rights elsewhere. Trofinetide has completed Phase II development for Rett syndrome, and is in Phase II development for Fragile X syndrome, moderate to severe traumatic brain injury (TBI) and concussion. Neuren has conducted double-blind placebo-controlled Phase II trials in Rett syndrome and Fragile X syndrome, which both demonstrated clinical benefit from treatment with trofinetide.

Currently there are no drugs approved for any of these conditions and there are few drugs in clinical development.