But all that I read says we wont get news until mid next year, is this right?????
If by 'news until mid next year' you mean Phase 2 clinical trials results for Rett Syndrome, yes you are correct. Phase 2 clinical trials in 4 indications will report results from mid-2014 with Rett Syndrome being the first.
However, there's a lot more pieces to this puzzle that I am looking forward to, way before Rett Syndrome results are reported next year.
Here's a recap of the items that I've taken notes from company announcements over the past few months that I'm waiting to hear news on. Quite a few items on the list now :-) The only new item that I neglected to add to my list is #23. This is getting tighter and tighter and these items could come in at any time now and in succession.
Waiting patiently :-)
Tony
ITEMS
The three big items that I am waiting for NEAR TERM will be an update on Perseis in vivo results (or anything from left field as highlighted in point #16 below), 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 another patient enrollment update as we now know recruitment at the University of Alabama site has begun.
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. Already received.
7. Fragile X - Orphan Drug Designation for Fragile X Syndrome. Already received.
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.
As I have previously highlighted, 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 soon.
10. Fragile X - Army update on NNZ-2591 for testing Fragile X in the same mouse model as NNZ-2566.
11. 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.
12. 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.
13. 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.
14. 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 (hopefully news on this front soon) 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.
15. 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. Hopefully news on this front soon as we approach 2014.
16. 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.
17. 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 that are well overdue? Or do Neuren/Noble have a bigger plan?
18. 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.
19. 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.
20. 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.
21. Funding alternatives - Additional funding from the US Army, other sources for non dilutive grants and the possibility of introducing strategic investors.
22. Risk Evaluation Mitigation Strategies (REMS) - I'm wondering whether this could be a new item to add to our list? Note: as highlighted in the video I posted titled 'Orphan Drugs - Small Market Big Opportunity' and the link to the FDA website on the subject.
Here's the video
http://www.youtube.com/watch?v=1knkGIZrf0o
As I mentioned, having Risk Evaluation Mitigation Strategies (REMS) in place before a drug is approved may very well increase the chances that it will be approved, however it will depend on a number of things. Please refer to the link below:
Here's the link to the FDA website on REMS. Click on the PDF under 'Related Information'.
23. Rett Syndrome - In the TAP Conference presentation (see link below) I also noted that Neuren highlighted that a Phase II trial in pediatric Rett Syndrome patients was planned for 2013.
(20min delay)