Going forward..., page-1579

"risk getting placebo"

Important to note, the P2 trial is treating patients with "High-Risk Acute Graft Versus Host Disease" vs. patients with "Steroid-Refractory Acute Graft-Versus-Host" (P1).
P2: https://clinicaltrials.gov/ct2/show/NCT05643638?term=cynata&draw=2&rank=1
P1: https://clinicaltrials.gov/ct2/show/NCT02923375?term=cynata&draw=2&rank=4


Whilst this might sound the same, it isn't:


https://www.nature.com/articles/s41375-020-0804-2


The Mayo Clinic lists the following (dated 09/10/20):


https://www.mayoclinic.org/medical-professionals/cancer/news/consensus-reached-on-initial-treatment-of-acute-graft-versus-host-disease-in-patients-who-receive-bone-marrow-transplant-nothing-definitive-yet-for-secondary-treatment/mac-20502381#:~:text=First%20line%20treatment%20of%20acute,gradually%20tapered%20off%20over%20time.

You can see that this is in line with the patients at "risk getting placebo":



If we were talking about second line treatment, then I'd agree with you (and Kurtzberg and MSB for that matter), but this is a first line treatment.


As discussed many times on here, the previous P1 trial aimed at safety, with signs of efficacy. You cannot expact a P1 trial to be designed the same way as a P3 (or even P2). Advancements here have been taken into account for the P2 trial, which can be seen here:



MAGIC guidelines used for assessing severity.



High-Risk only.


vs.




https://www.fda.gov/media/141131/download

Comparable results.

Also worth meantioning is the follow-up period:

"During the Follow-Up Period, subjects will have study visits up to 24 months."