What will good data look like? The trial is an open label study...

What will good data look like? The trial is an open label study which potentially could produce early study read outs - how early is early?

It dawned on me that just as ATL1102 targets CD49d, in turn CD49d can also be used as a biomarker for disease progression in DMD. The scientific paper, CD49d is a disease progression biomarker and a potential target for immunotherapy in Duchenne muscular dystrophy provides the relevant outline. One of the key conclusions by researchers including Dr Gillian Butler-Browne is that CD49d can be used as a predictive biomarker for clinical trials. This would appear to be huge.

If ATL1102 is effective in inhibiting CD49d expression on T cells then potentially we could get excellent quantitative data. CD49d has been demonstrated to be a reliable circulating biomarker which monitors both the severity and progression of disease in DMD patients.

If we go to the Trial Review for ATL1102 in DMD we see in fact that a Primary Outcome is lymphocyte modulation assessed by CD4 and CD8 T cells. Does this give us the nod that its been used as a biomarker?

Reading the Outcomes from the Trial, we can see that a baseline is taken and then lymphocyte modulation is assessed during treatment weeks 1, 3, 5 etc. So in fact the trial clinicians would be getting a very early read indeed. There are also secondary outcomes including performance of upper limb assessment but its the primary outcome which I suspect will be of critical importance. Remember the first patient was dosed 29 August. For the first patient, we would be in week 10 with readouts available for weeks 1, 3, 5, 7, 8 and 10.