Neuren Pharmaceuticals gains grant for Phase 2 trials of Neuren Rett Syndrome treatment

Friday, July 06, 2012 by Angela Kean

Neuren Pharmaceuticals (ASX: NEU) will move forward with Phase 2 clinical trials of its lead drug, NNZ-2566, in patients with Neuren Rett Syndrome following the award of a US$600,000 grant from the International Rett Syndrome Foundation.

The International Rett Syndrome Foundation has approved funding of $1.3 million to support new grants for translational research.

The Phase II clinical study will be spearheaded by Dr Daniel Glaze and Jeffrey Neul from the Blue Bird Circle Rett Center at Texas Children’s Hospital and Baylor College of Medicine, in conjunction with Neuren Pharmaceuticals.

In mid-May Neuren held a pre-Investigational New Drug (IND) meeting with the FDA Division of Neurology Products to discuss clinical development plans for the study of NNZ-2566 in the treatment of Rett Syndrome.

With IRSF funding, Baylor College of Medicine and Neuren are well positioned to move forward with this clinical study upon FDA approval of the IND.

Pending FDA review of the IND and completion of the ongoing Phase 1 trial of the NNZ-2566 oral formulation, Neuron is expecting the first Rett Syndrome trial to begin later this year.

The initial trial will be designed to assess the safety and tolerability of NNZ-2566 in adolescent and adult Rett Syndrome patients and to obtain preliminary evidence of efficacy.

NNZ-2566 – multiple application potential

NNZ-2566 has the potential to be the first neuroprotection drug to gain FDA approval. The drug is initially being advanced through Phase II trials for the treatment of moderate to severe traumatic brain injury.

Importantly, these trials have the funding backing of the U.S. Army to the tune of around US$23 million.

Success in this field will give Neuren access to a potential US$2 billion market opportunity.

Adding to the market opportunities, there is the potential for NNZ-2566 to be used in the treatment of Rett Syndrome.


Rett Syndrome

Rett Syndrome is a postnatal neurological disorder which occurs almost exclusively in females following apparently normal development for the first six months of life.

Typically, between six to 18 months of age, patients experience a period of rapid decline with loss of purposeful hand use and spoken communication. Many patients have recurrent seizures.

Rett Syndrome occurs globally in one in every 10,000 female births, and affects some 15,000 girls and women in the U.S. alone.


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Neuren Pharmaceuticals gains grant for Phase 2 trials of Neuren Rett Syndrome treatment