NEU (ASX) A little caution...

Originally posted by supertramp ↑

"The talk of FTD enabling this to go direct to market "avoiding phase 3 trials" is very much NOT the case."

The FDA is a stickler for safety and health.

This is often the bane of the biotech investor, but in nnz2566's case it is our friend.

Retts is a serious condition. Young people are dying early because of it. There is no treatment.

nnz2566 has demonstrated a positive effect for Retts sufferers. nnz2566 has been proven to be safe, and has undergone many safety studies and trials.

Hence for the fda it's quite an easy decision. No treatment means suffering and probable early death. nnz2566 is safe and offers a possible treatment.

The fda created the breakthrough therapy designation because it is ethical to offer a possible treatment for a serious condition where no treatment in available as soon as possible. nnz2566 is unusual for BT candidates as it has such a good proven safety record, hence there is no strong valid reason not to make it available to sufferers as soon as possible.

As somebody else mentioned, the retts lobby groups are very vocal and active in the US too.

All imho.

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Interesting.

You will also find that Dr Walter & Dr Percy have been a part of and play a significant role in approvals. You will note that it is not by chance that the ASX announcement included their names to clearly state that they were backing this up.

Dr Percy

Since 1998, Dr. Percy has served as Director of the American Board of Psychiatry and Neurology. He is a Fellow in both the American Academy of Pediatrics and American Academy of Neurology and is actively involved in many national and international pediatric and neurology societies. Dr. Percy is also active in many areas of research including the UAB Mental Retardation Research Center (MRRC), where he serves as an MRRC Scientist.

Dr. Percy was recently named one of America's Best Doctors on the www.bestdoctors.com web site. Only 4% of America's doctors are recognized in these listings based on surveys sent to leading specialists around the world

Dr Kaufmann

Director of Rett Syndrome Program; Senior Associate in Neurology; Co-Director of Fragile X Syndrome Program; Co-Director of Down Syndrome Program; Clinical Co-Director, Translational Neuroscience CenterProfessor of Neurology, Harvard Medical School

An Interesting paper was published by them including Daniel Glaze and others regarding Rett. It was published in Journal of neurodevelopment on 22 July 2014.

I am beginning to believe that this study would have formed the base or at least a benchmark to make comparison with. Statistically significant improvements would have been observed and hence a succesful trial.

Moving forward with NNZ-2566 here is where NEU is headed

The primary focus is on the pathogenesis of neurodevelopmental disorders including autism, Fragile X Syndrome, tuberous sclerosis, Turner Syndrome, 22q Deletion Syndrome, Prader-Willi Angelman Syndrome, Williams Syndrome, Lysosomal Storage Diseases, dyslexia, specific language impairment and Fetal Alcohol Syndrome.