It would seem they have gone the full year follow-up from treatment for just patient 1. Perhaps this is simply due to that patient's time of enrollment, as the follow-up time gets shorter as the a patient number increases.
It would also seem, these were all recruited based on having their platelet count being below the threshold needed for recruitment.
If we look purely at the table provided, then all patients see increases in hemoglobin (red cells) and ANC (white cells), while just Patient 1 see an increase (though it is a dramatic increase) in platelets.
Patient 1 clearly sees the most improvement with consistent positive trend.
If we look at the graphs themselves.
We did see all patients have the minimum threshold or below the minimum threshold for white blood cells, but then all of them recover to be significantly above the minimum threshold (1500 cells per microlitre)
All patients were significantly below the minimum platelet count 150,000, (10,000-30,000 mark), with only Patient 1 seeing significant increases and even reaching minimum normal healthy range. The other 2 patients not getting much benefit here.
We see all patients starting at low levels of hemoglobin, defined at less than 12g/dl (men is 13-14g/dl), but Patient 1 going into normal range, Patient 2 on a positive increase trend and presently hitting 10g/dl, but given the positive trend, could go higher with more time. Patient 3, no real increase but potentially avoiding things getting worse?
Therefore it would seem that we are seeing activity for White cells, and Red Cells, and then some activity for Platelets.
Also it should be noted that we are looking at data from the lowest dose cohort, at just 1 million cells/kg. The next cohort is 2 million and then the highest in this trial is 4 million.
Technically, we would expect to not see much at all in efficacy from the 1 million cell cohort, as the level of dosing would mainly be to start at a low dose to check from any safety issues. Again this is Phase I where safety is the key thing we are investigating.
So it is very much a good sign that at this low dose level were are not only seeing 0 treatment related AEs but also that we are seeing potential for efficacy, albeit from a small sample.
Also one needs to remember that with the ARS NHP data it was the 4 million dose that had the second highest efficacy on ARS survival (83% vs 86% at 10 million), so it will be particularly interesting to see what efficacy we might see as we go up to the 2 million, but most of all for the 4 million cohort.
In logical dose escalating study design we see that we have 3 samples for the 1 million cohort, 6 samples for the 2 million and 15 for the 4 million. So by the time they reach the 4 million sample there should hopefully be enough data to have a decent indication of efficacy even though 15 will probably not be enough for a significance check.
In either case, it's again positive that continued human dose shows 0 treatment related AEs.
On a final thought, it will also be interesting to see what the disease type might have on efficacy, Patient 1 having AML and having received allogeneic HCT. But that we'll just have to see based on new patient recruitment.
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