Ann: Investor Presentation, 8 January 2015, page-22

Though the Investor Presentation (08/01/2015) and this Bell Potter Report (29/07/2014) are great research aids when assessing Neuren's future potential - the more independent qualified sources of information the better.

The below video has Rettsyndrome.org CSO Steve Kaminsky talking about the recent Neuren drug trial results for Retts. Also, below are a couple of significant (edited) quotes from the video that bode well for NNZ-2566 getting FDA Orphan Drug Status and Breakthrough Therapy Status with the FDA:

2.43sec quote: "...So in these behavioral scales, what was very interesting is that behaviors changed, and they changed significantly from the placebo. So, for all of us who were essentially observers of this trial we're very excited that: 1/- 2566 is safe, it's tolerable, and NOW it actually also has an effect on behavior."

3.19sec quote: "...with only 28 days administration, MAJOR BEHAVIORAL CHANGES have taken place in the girls who are in the high dose group of this trial..."

I also note towards the end of the video Steve Kaminsky uses the plural "we" when discussing the next stages for NNZ-2566 getting FDA Orphan Drug Status and Breakthrough Therapy Status. I take that to mean Neuren may have an Allie in Rettsyndrome.org with the FDA (certainly publicly anyway).
ie - 4.00 sec quote: "... and with those, (he's referring to FDA Orphan Drug Status and Breakthrough Therapy Status), hopefully WE'LL be able to move even faster through the regulatory process..."

Video blurb: Published on Nov 24, 2014
Neuren Pharmaceuticals’ human clinical trial using NNZ-2566 as a potential treatment for Rett syndrome (a trial supported by Rettsyndrome.org) was completed in September of this year. Neuren has announced the results of this phase 2 placebo-controlled study, which investigated the dosing of NNZ-2566 for safety and tolerability with efficacy being studied as well. The study was carried out at Baylor College of Medicine (Drs. Daniel Glaze and Jeffery Neul), University of Alabama Birmingham (Dr. Alan Percy) and the Gillette Specialty Healthcare (Drs. Tim Feyma and Art Beisang).

NNZ-2566 was shown to be well tolerated at the dose levels tested after 28 days of treatment, and no significant safety concerns were identified. The data indicated dose-response and improvement over time in the study. The high dose used in this study showed benefit vs placebo both in the group-level analysis as well as in the individual subject analysis. For the details of the results please refer to Neuren’s announcement.

This is the first step needed to develop a drug for treatment of Rett syndrome. The next steps for Neuren are to meet with the FDA and to apply for Orphan Drug Status and Breakthrough Therapy Status. This is exciting for all of us involved with Rett syndrome. At Rettsyndrome.org, we continue to support Neuren in their goal to develop NNZ-2566 as a drug designated to treat Rett syndrome.