Ann: Contract to Commence Phase 2 Extramedullary AML Trial, page-143

This series of questions is almost as bad as those I get from @Mason14

Extramedullary AML is a subtype of AML. It is typically treated the same as normal AML as there are no specific treatments for it. Patients with extramedullary AML tend to respond worse than normal AML patients. It is not known why, but it is likely linked to the extramedullary tumours behaving more like a solid tumour and the fact that the drugs used in treating leukemias not working very well on solid tumours. In someways you can think of patients with extramedullary AML as having two seperate cancers - a leukemia in the bone marrow, and a solid tumour somewhere else.

Azacitidine is a standard of care drug for AML in general (i.e. it is used on all AML types including extramedullary AML)

There are lots of reasons why a patient might be considered fit or unfit for high intensity chemo. It is difficult to say at this point which Stratum will respond better to treatment as we don’t yet have any clinical data on the Stratum 2 combination - all we know is it works well in mouse models of AML.

Actually most of the patients that were treated in the Sheba trial would not be given intense chemotherapy here in Australia. This is one of the most underappreciated aspects of the first trial - the patients were almost all unfit patients at the end of life. Just to give you an example, one of the patients in the Israeli trial was 80 years old - here in Australia doctors rarely give AML patients old than 65 intense chemo. Let’s just say that Israeli doctors are not afraid of risk.

It is worth remembering that for Stratum 2 we are exploring a new treatment approach (FTO). If we are really lucky the patients will do a lot better than those that just get Inqovi, but even if they don’t we will have learned a lot about what dose to use, what are the side effects to expect, and what to do next. We will be running other trials in parallel targeting FTO in different cancers. Of course none of the FTO trials may work, but that risk is one of the reasons the MC of RAC is $500 million, not $5 billion (just picking a number at random here).