Ann: Positive Results from CSL112 Phase 2b Trial, page-24

No comments re: CSL specifically but re: this clinical trial:
- it is published in full in Circulation
- it had only 8% power to detect a significant effect for the secondary endpoints
- if you look at the forest plot you will see that most of the hazard ratios (~ relative risk averaged over time, eg. if 1.2 ~ 20% worse, 0.8 ~ 20% better), most of them fall on the wrong side of 1.
- did what it was supposed to do on a preclinical basis, but cannot extrapolate that to clinically improved outcomes.
- in fact the outcomes of most clinical significance, mortality and heart failure, decidedly worse in experimental arm (although only 1 event in control). I suspect if you combined the groups (which is not proper in any way), that you may have seen a significant result in favour of the control arm.

to put this forward into a phase 3 will cost a lot- I believe to be 90% powered based upon data in the supplementary appendix it would cost ~$1B USD people using 30,000 people (intervention 15K plus control 15K) and then using ~35k per participant in cost as inputs.

Am sure they have sophisticated algorithms for working out the chances of a significant result on the basis of the interim results achieved to date, before they make their go no-go decisions, but certainly it is not straightforward from a commercial perspective as far as I can see.