ITEMS TO EXPECT AN UPDATE ON THIS YEAR...
The three big items that I am waiting for NEAR TERM will be an update on Perseis in vivo results, Orphan Drug Designation for Rett Syndrome and the peer-reviewed paper for Fragile X. Everything else will just keep things ticking along nicely.
1. Rett Syndrome - DSMC review on low dose cohort, before moving to higher dose for Rett Syndrome.
2. Rett Syndrome - Possibly a patient enrolment update and new University of Alabama site recruitment to begin.
3. Rett Syndrome - Pediatric Exclusivity.
4. Rett Syndrome - Orphan Drug Designation for Rett Syndrome. This will be significant news.
5. Fragile X - Initiation of Phase II trial.
6. Fragile X - Fast Track Designation to the FDA.
7. Fragile X - Orphan Drug Designation . This will be significant news.
8. Fragile X - Pediatric Exclusivity.
9. Fragile X - Peer-reviewed paper for Fragile X. This will be an extremely important document for the company going forward.
This will not be a lengthy process to get published. In my experience, it typically takes between 4-5 months. If the peer-reviewed paper was submitted end of May 2013, there may be a chance that the paper might be ready in time for the FRAXA Investigators Meeting in Boston at the end of September. Now wouldn't this be something worth having in your hands at that time?
10. Fragile X - Army update on NNZ-2591 for testing Fragile X in the same mouse model as NNZ-2566.
11. Fragile X - FRAXA Investigators Meeting - September 29-October 2, 2013. Neuren's announcement on the 29th July 2013 re Fragile X results for NNZ-2591 highlighted that the company intends to formally present the results from the study at the FRAXA Investigators Meeting in Boston at the end of September. I'm wondering whether the company will be providing the market a follow up presentation of the meeting, similar to how they've done with previous meetings/conferences?
12. Fragile X - Rush University Medical Center already selected as the first site, now just waiting on up to 4 other Fragile X Centers to be announced.
13. Concussion Study - Neuren/Army to determine if it is feasible to conduct the study at the Army Training Centres. Considering Fort Bragg has been selected as the first site, there's a good chance that it is feasible and if so, will make the study more cost effective and capable of being completed more quickly.
14. Concussion Study - Finalising plan to conduct trial in military population for concussion and will be updated on when and where the study will be conducted. Fort Bragg has been selected as the first military centre with possibly a few more to be announced.
15. Intrepid Trial - EFIC process 5 new sites for DOD to approve. In addition, 10 new sites are in the process of being activated and are expected to receive DOD approval in the 3rd quarter this year (only 12 trading days left) which will mean that 18 sites will be actively screening and enrolling, 10 under EFIC protocol. As EFIC protocal is rolled out, expect significant increase in enrolment by participating sites.
Possible update re subjects enrolled as Neuren move forward.
16. Perseis - Antibodies have been screened in vitro for biological activity and binding to TFF and are undergoing final characterisation prior to progressing into an in vivo cancer model. Five antibodies are being evaluated rather than two now. Results from the in vivo experiments are now expected in Q3-2013.
Note: Only 12 trading days left in Q3-2013 for an update on Persies in vivo results, all going to plan :-) Could arrive any day as AGM Presentation highlighted results from the in vivo experiments are now expected in Q3-2013.
17. Perseis - Don't forget this passage from the recent Preliminary Report :-)
Reflecting our recalibrated strategic plan and focus on chronic indications for NNZ-2566, the Company is evaluating all strategic options in relation to the further development and commercialisation of the Trefoil Factor programme. This endeavour is currently being supported by Noble Life Sciences.
I wonder what update we'll receive in regards to this passage?
18. Perseis Patents - I wonder whether we'll hear news on patents that are pending in the USA, Europe, Japan, China, Australia and New Zealand this month in line with in vivo results?
19. Patents NNZ-2566 - Waiting on news for 6 pending applications covering composition of matter, oral formulations and methods of use for NNZ-2566 across a wide range of possible uses.
20. Patents NNZ-2591 - Waiting on news for 2 pending applications covering composition of matter, formulations and methods of use with remaining patent life (without extensions) between 11 and 15 years.
21. Motiva - If you have a read of the 2013 AGM presentation, you'll note that it highlights that enrolment has been slow as approximately 60% of eligible subjects decline to participate. Also an Interim analysis will be conducted for Motiva when 20 patients have completed follow-up which Neuren believe will occur in the 2H 2013.
22. Funding alternatives - Additional funding from the US Army, other sources for non dilutive grants and the possibility of introducing strategic investors.
Regards,
Tony
(20min delay)