Ann: Phase II/III autism trial shows further benefits, page-16

To make it clear Orphan designation for PANDAS/PANS is a good thing but a phase 3 (registrational) trial is the next step before a new drug application can be made. If the NDA is accepted then the drug is considered marketable, and a potential PRV comes into play (for sale to another company potentially). (Only applies for the USA and rare diseases)

Funds for a phase 3 are a concern. I am not certain what their commercial strategy is (or if it has been released) as it seems a bit of a mixture of different things. My perception was that the Autism program was the most advanced and management felt that TGA approval was most likely. It would be a smaller market revenue wise, but any income is a good thing.

OD/Rare diseases are where the big bucks are but these economics apply primarily to the US. The cost of these larger scale trails will reduce more funds.