RE: Unaudited Financial Results in the RNS...7 Sep 2020 19:50
Grace. A comment about Stach's Dec post.
At the end of 2019 we were assuming the target number of patients had been reached (50 anticipated in July 2019). The 2 year extension was supposed to increase the dose for a much larger cohort. Like Stach I was anticipating some good news, but by February was disillusioned by the smaller sample size, and the question why? With 50 patients and some fair efficacy, PIII seemed a reasonable bet. But not now.
By February 2020 it was confirmed the actual number of patients was only 18. We're now back down to 12. Have we wasted 2 years??
Like Stach, I fear the efficacy endpoint will prove wanting, mainly because the sample is back down to 12. In the earlier reported results, I seem to remember comments about the increase of dose from 2mg to 4mg not giving any conclusive evidence for a better response with the larger dose. Some patients fared better on the lower dose.
As a statistician, I would dearly like to work with a sample greater than 12.