Here's what I'm looking forward to hearing more about at the AGM...

Here's what I'm looking forward to hearing more about at the AGM this month. Another opportunity for Neuren to raise the level of awareness and strength of the science behind NNZ-2566.

1. Re Rett Program: the latest NEU report highlighted that a DSMC review will be conducted on completion of the lower dose cohort.

2. We should receive a recruitment update.

3. Initiation of higher dose once the low dose cohort is completed.

4. Fragile X: - Full results have already been presented at the neuropsychiatry meeting in April. The company mentioned that they are presently considering the implications of these results and options (note plural) for possible development.

5. Hopefully we will find out when Neuren will initiate the trial.

6. Will it be in advance of results from the Rett trial, study design?

7. Hopefully not too long now re news on Orphan Drug, Fast Track designation & Breakthrough Therapy designation.

8. Are Neuren considering laying off part of the risk through an early licensing deal so early in the game with such incredibly exciting results from Fragile X obtained with any molecule in a validated model that has been posited to predict the outcome of clinical studies in human patients? Will this project be the trigger? One cannot discount this as a possibilty. Not that we're going to hear anything re this at the AGM, however we will gain a better understanding of the company's plans.

9. I would not take this comment from the company below lightly (and yes it's only mouse model but certainly encouraging) - We believe that NNZ-2566 has the potential to represent a MAJOR breakthrough in this therapeutic area.

Points taken from the Conference:

* Treatment with NNZ-2566 significantly ameliorated all of these aberrant features of the fmr1KO mouse phenotype.

* Significantly, a complete reversal of macro-orchidism was also seen following NNZ-2566, indicative of a potential for disease-modifying effects involving not just the CNS but also other tissues that are affected adversely in Fragile X Syndrome. Taken together, these data suggest that the novel small molecule, NNZ-2566, may represent a potentially important treatment for Fragile X Syndrome.

10. And finally on Fragile X - In January 2013 Neuren entered a collaborative research and development agreement with the neurosciences group at Walter Reed Army Institute of Research.

We now know that Neuren is testing NNZ-2591 in the same fmr1 mouse model of Fragile x Syndrome in which NNZ-2566 was tested. Two separate drugs testing the same indications!

11. As I have previously mentioned, if two separate drugs are testing the same indications (and they're being assessed as a clinical candidate for the treatment of a number of chronic neurological disorders), there's no reason why Neuren couldn't potentially license one drug out to Pharma (NNZ-2591) and take the other (NNZ-2566) all the way. Still more work to do, but I certainly wouldn't discount this as another possibility.

12. More importantly, how long have both (Neuren and WRAIR) known that there was sound reason/opportunity to consider this as another option for the same indications?

I believe we're going to hear a lot more about Rett Syndrome, Fragile X and Neuren's plans moving forward at the AGM.

Looking forward to it.

All the best.

Regards,
Tony