I believe the efficacy was that their were 36% less MACE events than the control. No particular improvement in function, but it seems to stop further deterioration. This is what MSB found in their phase 2 trial from memory. The left ventricle function was not improved (which was what they were looking at and hoping for) but it was noticed that their was a decrease in MACA events. This was criticized as data mining when it was really noticing the outcome of the trial of a new science in which one does not know exactly what to expect.
I think that if a treatment gave you a 36% less chance of dropping dead by arresting the progression of the disease then I think that doctors would prescribe that, even though the insurance companies may wish you would just drop dead and be done with it.
To prevent further deterioration of a condition is a step in the right direction and perhaps something to hang the hat on ethically and economically while the science is further developed in its ability to perhaps reverse the condition which may be a cell programing issue or increased or repeat dosage. No one knows for sure yet but there is a general consensus that there is fire under the currently seen smoke.
I find it a matter of consistency that their product produced quite similar results to MSB's phase 2 trial, and there for expect the phase 3 interim results should deliver similar and perhaps better.
So is that enough, to reduce the probability of dropping dead 36%, to have a space in the market. I think so and it seems the FDA does as well, to offer fast track.
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