Preface
Global Blood Therapeutics (GBT) released HOPE part A data in June that was met with mixed feelings. Part of the mixed feelings was associated with a lack of insight into a key secondary endpoint: vaso-occlusive crises [VOC].
I have argued before that "numerical reductions" in VOC was the best-case scenario for part A. This is because "statistical significance" would've been an unreasonable goal to achieve with such a small sample size. Knowing this, HOPE part A was never designed to provide insight on whether or not secondary endpoints met statistical significance or