SOURCE: Neuren Pharmaceuticals

Neuren Pharmaceuticals

November 12, 2014 13:24 ET

Neuren's NNZ-2566 Successful in Demonstrating Clinical Benefit in Rett Syndrome Phase 2 Trial

BETHESDA, MD--(Marketwired - Nov 12, 2014) - Highlights:

  • Achieved primary endpoint -- both dose levels of NNZ-2566 were well tolerated after 28 days of treatment and no safety concerns were identified
  • Higher dose (70mg/kg twice daily) exceeded the pre-specified criteria for improvement in core efficacy measures compared with placebo
  • The clinical benefit in the trial encompassed core symptoms of Rett syndrome and was observed in both clinician and caregiver assessments
  • Meeting with FDA expected in Q1 2015 to discuss further development in Rett syndrome
  • Results will enable applications for both Orphan Drug and Breakthrough Therapy designation

Australia-based Neuren Pharmaceuticals (ASX: NEU) today announced top-line results from its US Phase 2 clinical trial in Rett syndrome, which successfully demonstrated clinical benefit from treatment with NNZ-2566. 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 intends to publish and present full analyses of results from the trial in the first half of 2015. Details of the top-line results can be seen in Neuren's Australian Securities Exchange announcement at the following link: http://www.neurenpharma.com/IRM/Company/ShowPage.aspx/PDFs/1448-10000000/NeurensuccessfulinRettsyndromePhase2trial

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."

About Rett syndrome

Rett syndrome is a post-natal neurological disorder that occurs almost exclusively in females following apparently normal development for the first six months of life. Typically, between 6 to 18 months of age, patients experience a period of rapid clinical decline that stabilizes later in life. Rett syndrome is caused by mutations on the X chromosome on a gene called MECP2. There are more than 200 different mutations found on the MECP2 gene. Rett syndrome strikes all racial and ethnic groups and occurs worldwide in approximately 1 in every 10,000 live female births. There are approximately 20,000 females with Rett syndrome in the United States and more than 50,000 worldwide. 

The young women enrolled in Neuren's trial were in the late stage of the disease which is typically characterized by an inability to speak, walk and use one's hands. Additional signs and symptoms in young women with Rett syndrome include autonomic dysfunction (which can affect the rate and rhythm of the heart), breathing abnormalities, seizures, abnormal curvature of the spine, irritability, unusual behaviors such as prolonged bouts of crying and screaming, and muscle rigidity or spasticity. On the whole, older individuals with Rett syndrome tend to have more severe symptoms and a greater degree of overall debilitation relative to children with this disorder.

About NNZ-2566

NNZ-2566 is a synthetic analogue of a naturally occurring neurotrophic peptide derived from IGF-1, a growth factor produced by brain cells. In animal models, NNZ-2566 exhibits a wide range of important effects including inhibiting neuroinflammation, normalizing the role of microglia and correcting deficits in synaptic function. NNZ-2566 is being developed both in intravenous and oral formulations for a range of acute and chronic conditions. The intravenous form of NNZ-2566 is presently in a Phase 2 clinical trial in patients with moderate to severe traumatic brain injury. The oral form of NNZ-2566 is in Phase 2 development in Rett syndrome, Fragile X syndrome and mild traumatic brain injury (concussion). Three programs have received Fast Track designation from the US FDA and the Fragile X syndrome program has also received Orphan Drug designation.

About Neuren

Neuren Pharmaceuticals Limited (Neuren) is a biopharmaceutical company developing new therapies for brain injury, neurodevelopmental and neurodegenerative disorders. The novel drugs target chronic conditions as well as acute neurological injuries. Neuren presently has a clinical stage molecule, NNZ-2566 in Phase 2 clinical trials as well as NNZ-2591 in pre-clinical development.

Forward-looking Statements
This announcement contains forward-looking statements that are subject to risks and uncertainties. Such statements involve known and unknown risks and important factors that may cause the actual results, performance or achievements of Neuren to be materially different from the statements in this announcement.

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