Ann: Quarterly Appendix 4C and Activities Report, page-209

Ph2 trials are typically done to assess efficacy AND safety. Usually in that order.

Ph1 defines safety, typically in a small cohort.
Ph2 explores efficacy and safety in a larger group.
Ph3 studies efficacy in a larger group again.
Ph4 is typically done post-marketing.

I am expecting the primary outcome to be met, and anything over and above 15% decrease in proteinuria to meet clinicalsignificance. That would warrant a Ph3.

Depends upon how the company releases it. If all patients show a 20% decrease? Or half of them show a 40% decrease and the remainder show a 10% decrease? Stats was never my strong suit.

And a Ph3 company in a FDA-designated orphan disease.... you can figure out the rest...