Ann: AGM Presentations, page-39

"Some information that is simply wrong and is very misleading . The more accurate estamate would be around 50% but then when you look at the patients enrolled in your trial they may have been slightly better off than the average population . This would resault in the trial not showing any benifit."


We have had that conversation previously, when you compared CYP-001 to be about as effective as a glass of water and I have suggested to check out the REACH-2 data:
https://hotcopper.com.au/threads/gvhd-orphan-drug-same-drug-clinically-superior-issues-for-cyp-and-msb.6589558/page-111?post_id=60250181

Just to make sure we are talking about the same indication here - Steroid-Refractory acute GvHD:


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


https://www.ema.europa.eu/en/documents/assessment-report/jakavi-epar-public-assessment-report_en.pdf


Now, perhaps looking at our data (Supplementary Table 4) as published in nature:

How many of these patients would have been considered High Risk at Baseline using the following Definitions:

https://www.researchgate.net/figure/GVHD-Risk-Definition-by-Organ-Stage-at-Onset_tbl2_270966544

Just based on that, I would consider A3, A4, A6, A7 and A8 in the lower dose Cohort A and B2, B5 and B6 in the higher dose Cohort B.
And out of these 5 patients in Cohort A, 3 showed a CR on Day 100, and 2 out of 3 showed a CR in Cohort B on Day 100. Unfortunately there is no breakdown of the 60% survival after 2 years. It would definitely be very interesting to see.

On that note, I found this comment regarding Temcell very interesting, "Treatment of Steroid-Refractory Acute Graft-Versus-Host Disease Using Commercial Mesenchymal Stem Cell Products":


https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8417106/

Comparing that with our very small and therefore limited data, I count 3 patients with liver involvement (A4, B2 and B6), two of them not showing a response by Day 28 and all three of them seem to be considered high risk but had a CR Best Response on Day 100. Of course, based on a limited patient size of 15 this could have just been a coincidence. Lets wait for the P2 results I would say.

That was mostly just a repeat of the last exchange we had about the above.

I've been trying to look for data that shows a 2 year survival in SR-aGvHD patients, in adults I should add and ideally not subject to selective recruitment. Since I couldn't find that published by a company here in AU, I had a look at Jakafi again. Since that is also the only FDA approved therapy for SR-aGvHD in adults:
https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-ruxolitinib-acute-graft-versus-host-disease

The P3 REACH-2 trial enrolled 310 patients (12+ years) and listed an Overall Survival (OS) [ Time Frame: Up to 24 months ] Secondary Outcome Measure.

Patient breakdown:

https://www.ema.europa.eu/en/documents/assessment-report/jakavi-epar-public-assessment-report_en.pdf


Obviously, Jakafi had much better results than the 17%, but still only around the 40% mark.

Some may wonder why there's barely a difference between Jakafi and BAT:


As mentioned numerous times before, the data is limited and so far Cynata has only conducted a P1 trial in 15 patients.

Before you start the usual plenty of data is available yet CYP has not even done a proper historical study to deminstate effecacy . That however is still not good enough for the FDA , they require patients to be matched at onset and followed to establish a current controll " argument again...

... Cynata conducted a Phase 1 trial:


https://www.fda.gov/patients/drug-development-process/step-3-clinical-research

The upcoming P2 trial should provide us with a lot more data.
I know, they "shoulda/woulda/coulda...". Well, whilst "Back to the Future" may appear to be a documentary on time travel to some, allowing to bring future knowledge to the present, sadly, it is not. We still have to follow the normal procedures to hopefully learn from the past, draw the right conclusions in the present and benefit from that in the future.