Recap of what we think we know

Alot of discussion on ymb today and figuring out the next steps....here's a recap-

1) Orphan drug status = 7 years of exclusivity after approval, 50% reduction in taxes, reduced application fees.
2) Orphan drugs have increased odds of being approved (82% v. 35%).
3) the orphan drug typically gets to market faster than non-orphan drugs
4) once its on the market, consideration for expansion to other indications is possible (i.e. AD)
5) once orphan status is achieved, one of four pathways can be utiltized to get the drug to market.
6) we think that Prana will use one of these two paths:

a) the priority review process to get PBT2 to market initiating the process with an NDA. If the NDA is approved for priority review, further clinical studies would not be required. This pathway relies upon the strength of the data and requires submittal of the REACH2HD data we think in the form of a peer reviewed paper. Once the NDA is submitted FDA has 45 days to reply.
or b) if the data are decided by FDA not to be strong enough, they would likely approve with post approval study streamlined to reduce cost, get the answers quickly, designed with FDA input.

Alternatively, if the data are not strong enough the FDA may require additional study but the process will be streamlined with FDA input prior to approval possible under breakthrough therapy designation or other accelerated approval process.

Note: either of these ways gets PBT2 to market quicker than without orphan status - and provides revenue. Once its on the market, off-label use for AD will be tremendous as well as other use. As of the last filing they had 34M in cash more than enough to do any of these. There is only one drug approved for HD and that is not disease-modifying, therefore there is a huge unmet need and it is safe. It would be nearly criminal not to get it to patients quickly.