Ann: PTX-200 AML Trial Progresses to Higher Dose, page-28

Hi psych & all, regarding dose escalation given the dosing schedule between PTX-200 & Cytarabine was changed from the previous cohort in an effort to reduce unwanted drug to drug toxicity issues a few patients experienced. I don't believe the AML trial has yet found the maximum PTX-200 tolerated dose, hence upping the next cohort to 45mg/m2. The Phase 1 trial design allows increments of 10mg/m2 up to maximum 55mg/m2 PTX-200 with enrolment estimate up to 40 patients: -

Experimental: PTX-200 and Cytarabine: PTX-200 administered intravenously over 1 hour Phase I: 4 dose levels: 25 to 55 mg/m2 (with reduction to 15 mg/m2 if needed).

It's been a long time & if memory serves we have 3 pCR's out of 18 patients, however for the AML trial I've never seen any mention of ORR, which also includes PR & SD as we did with the Breast & Ovarian. Totally understand pCR is the holy grail of any trial, in my mind ORR is equally important. As this demonstrates if PR & SD are seen then the treatment in addition to any pCR is making a positive % difference to otherwise heavily pre-treated very ill people with little to no other options available. Maybe this is something needed to be raised with management..... & yep agree comms are not the best vs other ASX bio's either....

Anyway fingers crossed we see additional CR/s in the next cohort!

Cheers,

AMS