Ann: Ryoncil Highlighted at Tandem Transplantation Meetings, page-150

Well ,, this could actually be good.
"contraindication or intolerance to, Jakafi®"
1: under 12s our cells are first in line.
2: over 12s our cells would still be first in line for those that are high risk. There condition would in the majority of cases be in contodraindication for the safe use of Rux given the substantial list of side effects patients would be exposed too..
for example the child may have a weak liver.
3: If treatment was not allowed till Rux was used in these very sick patients our treatment would still be used as we know Rux is in the majority of cases ineffective or has to be scaled back due to intolerance.
4: Priceing. As it appears that this bunch are trying to cut the indicated population back from what was included in 001 , they would also need to re-evaluate 001 data by cutting out those that were likely to respond to Rux. While survival % wouldn't change substantially the control changes dramatically going from survival around 45% to 15 % .
5: 12 months on and we will be looking at real world data.
head line results with above in place.
70% survival in population that if not treated would have no better than a 15 % chance of survival.
With the FDA approved population
68% survival in a population that left untreated would have a survival of 45% . Still very good but you can see the difference.
So after about 12 months in children one would think momentum of clinical development and off lable would start to be significantly ramped up , I would say that the slightly smaller treatment population would drive growth in sales and development far quicker. Short term pain, long term gain.
GLTAH