Neuren Pharmaceuticals - announces positive Rett syndrome results

The neurological focused drug developer Neuren Pharmaceuticals (ASX Code: NEU) announced that it has successfully completed a Phase 2 clinical study in Rett syndrome, with its 70 mg dose of NNZ-2566 encouragingly showing some efficacy in Rett syndrome.

The company commented they were delighted, with the clinical results exceeding their expectations.  The clinical study was conducted in 56 women aged 16 to 46 years which is an older patient set than typically studied in Rett syndrome while the duration of the study was just 28 treatment days, which is pretty short.

Its believed NEU were targeting older patients because of NNZ-2566 is closely aligned to IGF-1 which is a growth hormone and they didn't want any confounding factors from younger subjects who are still growing. 

The higher dose or 70 mg taken twice daily version of NNZ-2566 showed improvements in motor behaviour, clinical assessment and caregivers feedback, while showed little benefit in sleep apnea, behavioral assessments and clinical severity.

The anecdotal sweet spots for this drug appear to be around alertness, attentiveness, improved communication, brightness, while researchers also saw some improvements around gastro health and some better movement.  The full clinical results are expected to be presented around March 2015.

The company reported some efficacy in their lower dose or 35mg of NNZ-2566, however this was not enough to meet the company's pre-specified analysis plan.  The company also encouragingly reported that both doses showed trends of increasing effect with the duration of treatment, remembering this was just a 28 day study.  The study didn't have any serious adverse events attributed to the drug, which is also pretty helpful.  

The NNZ-2566 therapy already has fast track status with the FDA with NEU hopeful to soon add Orphan Drug and Breakthrough Status with the FDA.  The positive study probably bodes well for NEU's 60 patient Fragile X syndrome study, with clinical results due in the June 2015 quarter.   In addition we are expecting clinical results from NEU's traumatic brain injury study in the same quarter, so there is quite allot of action ahead for investors.

Australian investors pushed the stock up 54 percent giving the company a market capitalization of $194 million AUD or $169 million USD. It will be interesting to see how US investors who really understand biotech react to this news.

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