Anavex Life Sciences Corp (AVXL)

[Doc328]

There was not a primary endpoint but rather 2 co-primary endpoints. The lack of data values or a p value implies a complete miss (p> 0.10). High placebo responses are the scourge of many trials. Did Missling run an underpowered study to try to save money? Was the 2:1 randomization in a small trial a smart or stupid idea? Was a 16% total dropout rate (probably 18-20% among treated-same as trofinetide) due to tolerability to blame? Was there too much hype in the Reverse Rett community leading to over optimistic RSBQ reporting in both placebo and treated groups? I think the subgroup analysis (genetic markers, age, severity) will be interesting and help to design the next A273 trial if Missling goes in that direction. Or do they wait for 3-71? In MMRM/LSM, there would already have been some adjustment for subgroups.

From the PR
" The other co-primary endpoint, the Clinical Global Impression – Improvement scale (CGI-I), which represents a less granular assessment by the site investigators using a seven-point scoring (one=“very much improved” to seven=“very much worse”), was not met."