Cell Therapy News/Articles, page-8246

It's my understanding that these biomarkers are very important and have statical revelents of over 90%. The patients that have these high levels are very sick even if they don't know it. Refer to the announcement in early December. GVHD.
What we don't know is just how much remestemcell would be required in the early stages of GVHD.

We know that for every 100 patients with GVHD about 30 become steroid refractory. That would require around 240 infusions of our therapy and would save 20 + patients.
Now consider also including high levels of biomarkers
100 GVHD patients with high levels of biomarkers, perhaps 50 would become steroid refractory, they would need 400 infusions.
Knowing that we get a better response early if we were to treat this group with just 2 infusions that aren't steroid refractory we could get a complete response in 80 of them leaving just 20. Some of these 20 would be none responders and would probably be best to leave and go onto standard of care drugs. However those that have shown a incomplete response could continue for another round .
As you can see on both patient outcomes and cost basis it could be way better to start treatments early even though the number of patients receiving remestemcell is much greater.
It's my view that we should design the P3/4 trial that is planed In a similar manner as children could be included as they still have a alternative steroid treatment available if ours were to fail. Remember that the control group could possible have 50 srGVHD patients that could go on to receive remestemcell or Jakafi.

Good luck all