The initial trials are for safety but they still monitor for any signs of efficacy. The first patient with an improvement was kind of unexpected or at least a pleasant surprise at this early stage of the trial. We are hoping there may be some more reports from the higher dose. At least on the surface that seems possible. Personally I feel it is reasonably likely.
The earliest we could have hoped for an announcement on more reports of efficacy was at the time of the prior recent announcement on Safety on the SAD. I feel that was a little early and end of July or early August is more realistic as from memory although the efficacious patient self reported earlier they didn’t announce it until after the 8 week check when they were able to combine her subjective reporting with their objective microperimetry. It could potentially happen any day from now.
Then we will be waiting for signs of efficacy in the MAD this could be as early as mid to late September.
The aim in the end is to prove significance. This is a statistical term. Basically you need the event to happen enough times to show that it wasn’t just a fluke. We also need to know what proportion of people are likely to have a response and how large the response might be.
Hope that’s helpful and please anyone who disagrees or has more experience feel free to correct.
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