Ann: Global Phase 3 Trial of Presendin to Launch in Q4 2021, page-95

cleared up with Tom, thought Id share:

Running another Phase 2 study is unnecessary and will delay things because we know this drug lowers ICP (as it follows the science) and moreover, the regulator (EMA) was ok with a single Phase 3 to register this drug. Running a 100 patient Phase 2 over the next say 2+ years would only re-confirm what we’ve done and not have the statistical power to show a difference in those clinical measures. So it would be a waste of time and money. As we spoke about, we are fully funded to complete, report and file Presendin for approval in these markets. If it fails, we would still retain sufficient funds to consider next steps. Unfortunately, this is biotech – we need to spend the money to find the answer(s). If it works, the payback is enormous, naturally.
An interim analysis will significantly compromise the final analysis and the strong advice from study statisticians was to not consider it.

Interim analyses introduce the chance of falsely rejecting the null hypothesis. In other words, every time you look at the data there is a chance of a type 1 error (false positive). Also the medical fraternity will likely frown on interim analysis ahead of the final planned results.

Interim analyses are more common in diseases like cancer where the intervention may be very effective, and because there is a risk of dying of progressive disease, may be unethical to not offer the intervention to the control arm. In Invex’s case, the treatment period is relatively short (24 weeks) and IIH does not pose a significant risk of death