Challenge, page-22

@whytee

The results of the expanded access programme for graft vs host disease are pretty impressive.

Generally for rare conditions or those with few therapeutic options in general such as this, the FDA does not require a hard endpoint or randomized studies for approval.

In terms of the other question re: endpoints changing. Typically early phase studies are about safety first, then efficacy then only late phase or registration studies are for harder more clinically meaningful endpoints.

I haven't looked into the other studies in depth- but a cursory look at the website and the back pain data suggests this is very promising from he randomized phase 2 data alone. Given this is a very large addressable market a hint of success here would also be enormous.

If I was an investor here I would be more worried about the continual problem with Australian biotech- you take all the risk on development and then someone comes in afterwards at a late stage to actually get the reward.