timeline question, page-2

Hi Kmsmith,

Hope this helps.

Page 4, 6 & 11 of the May 2013 CEO Address AGM highlighted these three passages...

Page 4:

Eligible for regulatory advantages which come with Fast Track, Orphan Disease or Breakthrough designation that can significantly reduce the cost and time of bringing a new medicine to the market and provide additional clarity around the path to approval.

Page 6:

All of the indications that we've selected are expected to be eligible for fast track designation (which has already been issued for NNZ-2566 in TBI) and both Rett Syndrome and Fragile X Syndrome are potentially eligible for Orphan Drug Designation and possibly Pediatric Exclusivity. With strongly positive Phase 2 results, all would be expected for Breakthrough Therapy designation as well.

Page 11:

If the results of the Phase 2 trial are positive, we intend to request an end of Phase 2 meeting with the FDA to discuss requirements for a pivotal trial and registration under provisions of Fast Track and potentially Breakthrough Therapy designation. We are expecting that a pivotal trial would be the same size as the Phase 2 trial and could be completed within one year.

...............

As you are aware, Neuren have already been granted Fast Track for all indications (TBI, Rett and Fragile X Syndrome) as well as Orphan Drug Designation for Fragile X Syndrome. We are still waiting on Rett Syndrome Orphan Designation status.

Here are a few links on the above...

Breakthrough Therapy

http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDASIA/ucm341027.htm

Pivotal trial

If you look at the May 2012 AGM CEO Presentation it highlights this passage...

A second study in pediatric patients is planned for 2013. If the results of the Phase II trials are positive, we believe that a single pivotal trial in adolescents and adults and one in children would be required to submit an NDA for approval.

From Wikipedia, the free encyclopedia:

A pivotal trial can refer to a clinical trial or study intended to provide evidence for a drug marketing approval,[1] eg by the US FDA. Phase III trials are assumed to be pivotal so the phrase is often used for the rare pivotal phase II trials.

http://en.wikipedia.org/wiki/Pivotal_trial

http://www.lillytrials.com/docs/terminology.html#P

Here's an excellent video well worth watching...

Orphan Drugs - small market/big opportunity / Craig Kephart, President, Centric Health Resources

http://www.youtube.com/watch?v=1knkGIZrf0o

Regards,
Tony