We've discussed what the lack of efficacy concerns actually means in these DSMB reports and the majority view (though not necessarily the correct view) is that apabetalone is not increasing MACE rate compared to placebo, which would be bad. Just because the trial wasn't stopped for efficacy concerns does not necessarily mean that apabetalone is performing superior to placebo. Keep in mind that the previously planned futility and sample size re-estimation analyses were never performed, which would have been a chance to officially assess futility or make an informed adjustment to the trial size. There is a reason to run clinical trials. Hypotheses and post-hoc results are great, but proving this in a well designed trial is essential. There are recent examples of CVOTs going to completion but not achieving a robust and/or statistically significant difference. A primary outcome in BETonMACE that fails to achieve statistical significance is seen as no effect.  It is irresponsible to assume BETonMACE will succeed. One must not blind themselves from the not so rosy potential outcomes.

BearDownAZ