Neuren Pharmaceuticals (ASX: NEU) has completed enrolment for its Phase 2 clinical trial of NNZ-2566 treatment for Rett Syndrome.
This will enable top-line results from the trial to be released by Neuren in the 4th quarter of 2014, as planned.
The double-blind, placebo-controlled trial, initiated in April 2013, is the first commercial multi-site clinical trial in Rett Syndrome, for which currently there is no approved therapy available.
The trial has been supported by the International Rett Syndrome Foundation and is being featured at the current Rett Syndrome Symposium in the US.
Two dose levels of orally administered NNZ-2566 are being tested in female subjects aged 16 to 45 years with a trial duration for each subject of ten weeks, including the screening and follow-up periods.
To date, 47 subjects have completed the trial, with 7 more in progress. The total number of subjects completing the trial therefore may be up to 54.
As well as the primary endpoint of safety and tolerability, a number of different measures will be analysed for signs of clinical efficacy. To date no safety concerns have been identified.
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.
NNZ-2566 is a synthetic analogue of a naturally occurring neuropeptide derived from a growth factor produced by brain cells.
In animal models, NNZ-2566 inhibits neuroinflammation and normalises the function of microglia with consequent improvements in molecular, cellular, anatomic and behavioural outcomes.
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