Ann: Kazia's paxalisib obtains Rare Pediatric Disease Designation, page-99

Do you understand your n=30 is a reference to one single trial that was critical to the FDA granting Kazia fast track designation for GBM? That’s a rather conflicting reference which refutes your own argument.

Can we progress paxalasib in DIPG, which has a Rare Pediatric Disease Designation, and receive a potential key PRV until you (qqq) are satisfied with the hundreds of patients you require to be enrolled for data read outs in this indication? It seems you have set a rather high bar to achieve for a rare disease? However, you have given us your key unqualified opinion with a footnote about “honesty and integrity” so you must be right? Side note, I need to add a footnote to my posts so people clearly know what I stand for too.

Hopefully the FDA know what they’re doing giving paxalasib fast track designation for GBM based on the current data. Interestingly, the FDA also supported the GBM AGILE phase 2/3 trial, among other things. That trial was designed by over 130 key opinion leaders in consultation with the FDA. I’m guessing you were not one of the over 130 people (nor do you work for the FDA), as the maximum size in stage 1 is only 150 patients, and stage 2 is only an additional maximum of 50.