Neuren’s NNZ-2566 successful in showing clinical benefit in Rett Syndrome phase II trial

Neuren intends to submit applications to the US Food and Drug Administration (FDA) for both Orphan Drug and Breakthrough Therapy designation.

Neuren expects to meet with the FDA in the first quarter of 2015 to discuss the trial results and the requirements for the further development of NNZ-2566 in Rett syndrome.

There are currently no approved medicines for the treatment of Rett syndrome, which is a severe neurological disorder caused by mutations of the MECP2 gene on the X chromosome. The disorder has an onset in early childhood and is often progressive into adolescence and adulthood.

Neuren’s trial was conducted at Baylor College of Medicine (Daniel Glaze, MD, and Jeffrey Neul, MD), University of Alabama at Birmingham (Alan Percy, MD) and Gillette Children’s Specialty Healthcare (Tim Feyma, MD, and Art Beisang, MD). This was the first multi-site, sponsor-led clinical trial in Rett syndrome and was the first trial in an adolescent and adult population.

Walter Kaufmann, MD, Professor of Neurology at Harvard Medical School and Director of the Rett Syndrome Program at the Boston Children’s Hospital, who was not involved in the trial, commented: "The outcome of this trial is very promising in terms of both safety and clinical improvement. It was a challenging study since the older age of the cohort and the short duration of the trial made it less likely to show a positive effect. It opens not only the possibility of successful treatment of adults with Rett syndrome, but also of early interventions modifying the course of the disease."

Alan Percy, MD, Professor of Pediatric Neurology at the University of Alabama, was one of the trial investigators. He commented: "The results of this trial suggest a very promising proof of concept as we continue on the pathway to develop a disease-altering treatment for girls and women with Rett syndrome. Not only was this short-term trial managed successfully, but also the data analyses were conducted in a very robust fashion."

The trial was supported by the International Rett Syndrome Foundation (IRSF). Steven Kaminsky, PhD, IRSF Chief Science Officer, commented: "These are exciting times for Rett syndrome and this trial firmly sets our rudder in the water for the near future. The results will enable engagement with the FDA on the further development of NNZ-2566. This is what we, as the Rett community, have been hoping for."

Neuren Executive Chairman, Richard Treagus, commented: "We are very grateful to the patients and families affected by Rett syndrome, as well as the support provided by IRSF, that have made this ground-breaking clinical trial possible. The trial results have exceeded our expectations and we look forward to discussing with the FDA the remaining requirements to develop NNZ-2566 for the treatment of Rett syndrome."