Neuren Pharmaceuticals (NEU) has received approval from the US Food and Drug Administration to conduct a Phase 2 clinical trial of a drug to treat patients with Fragile X syndrome (FXS), the company recently announced. The trial is expected to start before the end of 2013, and is expected to involve several sites within the Fragile X Clinical & Research Consortium (FXCRC).

Adolescent and adult males will take part in a double‐blind, placebo‐controlled study, testing two different dose levels of NNZ‐2566. Enrollment is expected to continue through 2014, with initial results announced in the first half of 2015.

The approval of this investigational drug for Fragile X syndrome represents an expansion of NEU’s strategy to determine its effectiveness. The drug is currently in clinical trials for Rett syndrome. Like FXS, Rett syndrome is caused by a mutation of the X chromosome, affects cognition and behavior, and is often misdiagnosed as autism.

NFXF Board Member Elizabeth Berry-Kravis, MD, PhD, is also medical director of the Fragile X Clinic at . She is familiar with the drug and the upcoming trial. “They tried it in the Fragile X mouse and so far it works very well,” she said. “Our clinic will be participating in this trial along with other FXCRC sites.”

In a mouse trial, scientists use genetic engineering to create FXS in laboratory mice and then study the effects of various drugs on their behavior. Mice and humans share about 90 percent of the same genes, so drug research on mice is generally a good—though not exact—predictor of a drug’s effect on humans.

Neuren Pharmaceuticals is a New Zealand-based company with operations in both Australia and the U.S. For more information on the upcoming trial, see:–47740.html.