Neuren Pharmaceuticals is pleased to inform you of an opportunity to participate in a clinical trial for adolescents and adults with Fragile X syndrome.

This clinical trial is testing an investigational medication, NNZ-2566, as a targeted treatment for Fragile X syndrome (FXS).  The purpose of this study is to assess the safety and efficacy of two dose levels of NNZ-2566 when compared to placebo in adolescent and adult males (ages 14-40) who have been diagnosed with FXS.NNZ-2566 has an entirely novel mechanism of action compared with other drugs that have been tested for Fragile X syndrome. NNZ-2566 is a modified version of a biologically active part of Insulin-like Growth Factor (IGF-1). IGF-1 is a protein in the human body that helps cells grow and respond to stress or damage. In the brain, IGF-1 contributes to the growth of brain cells and the connections between them (synapses). The biologically active part of IGF-1 is called glypromate or GPE and supports the function of brain cells in a different way than IGF-1.  GPE occurs naturally in the brain. NNZ-2566 is a version of GPE modified to make it available to take as an oral medication. NNZ-2566 may help improve brain function by enhancing functions normally performed by GPE and by increasing IGF-1 levels in the brain.  NNZ-2566 has been shown in animal models of Fragile X to improve performance in tests of behavior and cognition.

FDA has granted two special designations for Neuren’s development of NNZ-2566 in Fragile X: Fast Track Designation and Orphan Drug Designation. Both designations are important milestones in accelerating the development of viable treatments for Fragile X, and demonstrate a recognition of the critical unmet needs of individuals with Fragile X.

Participating sites include: Rush University (Chicago), Icahn School of Medicine at Mt. Sinai (New York), University of Massachusetts Medical School (Worcester/Boston), Suburban Research Associates (Philadelphia), Emory University (Atlanta), and Children’s Hospital Colorado (Denver).  Other sites will be added soon.

This trial will include 60 participants between the ages of 14-40. Taking part in this study will involve 8 visits to the doctor’s office over a 10-week (about 2 ½ months) period. The study medication and all study-related evaluations will be provided at no cost to participants. Some support for travel will also be available.

Please see our listing on for more information and the contact information for the participating sites at this link: Neuren Fragile X Study.

More centers will be added in the near future. For more information about Neuren, please see our website: