re: Ann: Letter to Shareholders from the Boar......

re: Ann: Letter to Shareholders from the Boar... Worthind,

Anything thing that is a serious unmet clinical need (ie life threatening) has a chance of getting up. It must also affect less than 200,000 people in the US (or the EU, if that is where you want to apply.

Generally, orphan indications are more clear cut than the one you are suggesting, but that doesn't mean it won't work.

Issues you need to think through include whether patients of that nature are or can be routinely identified, what the prevalence of patients of that nature are, whether the disease is in fact serious in terms of orphan designation and, probably most importantly, whether another drug has been approved that already covers those individuals, even broadly. Patients must really have no other option.

Also, orphan status is great, but it must be in a niche that lends itself to off-label use. That is, the orphan indication it is approved for must give doctors hope it is worth trying in other situations. Without off label use or a very high price you probably won't have a commercially viable product.

To be frank, I would be very surprised if Chick and Coates haven't already scanned the landscape for an orphan indications. Coates, in particular, knows HIV back to front.

If I were you, my first course of action would be to speak with Drona and find out what orphan indications they have looked at. If he can give a good answer to that, it is probably time to take the overs on cash backing and look elsewhere. If he can't, however, then it is time to poke the hornet's nest a little bit harder.

In terms of an expanded access program, I suspect you need more data. Just because patients continue to use the drug, does not mean it is working. Also expanded access programs are limited to cost recovery. They don't let you make money out of them. So, overall, I think it is only worth pursuing an expanded access program if the data to justify it can be collected easily and, importantly, it fits with a broader commercially viable strategy for regulatory approval.

Bob