Neuren Pharmaceuticals (ASX:NEU) has been given the nod by the U.S. Food and Drug Administration to carry out a Phase II clinical trial of its NNZ-2566 drug for Rett Syndrome.
NNZ-2566 is a oral solution for adolescents and adults with Rett Syndrome, a postnatal neurological disorder which occurs almost exclusively in females following apparently normal development for the first six months of life.
The drug is a synthetic analogue of a naturally occurring neuroprotective and neurotrophic molecule derived from IGF-1, a growth factor produced by brain cells as well as in other parts of the body.
The intravenous form of NNZ-2566 is already in a Phase II clinical trial in patients with moderate to severe traumatic brain injury and has received Fast Track designation from the U.S. FDA.
The company is currently undertaking final preparations to initiate two additional Phase II trials with the oral form of NNZ-2566 – one in patients with concussion or mild traumatic brain injury and one in patients with Rett Syndrome.
The dosing period for this initial study in patients with Rett Syndrome will be 14 days.
The study will utilise a randomised, double-blind, placebo- controlled design. There will be a low dose and a high dose NNZ-2566 cohort in the study.
A total of 60 subjects will be randomised 2:1 to NNZ-2566 and placebo.
The trials will assess pharmacokinetics, safety and tolerability, objective physiological measurements such as EEG and cardiac and respiratory function and standardised evaluations of symptom severity and global functional status.
The study is expected to be completed within about one year. Patient screening and enrolment are likely to begin in January 2013.
Rett Syndrome is a post-natal neurological disorder caused by mutation on the X-chromosome. Typically, patients lose motor functions and spoken communication with many affected by recurrent seizures.
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