Sorry you missed the AGM
@Titans19 as it would have been good to have met you in person. I can’t answer all your questions, but I can answer some of them.
1. Unfortunately we don’t have details on other patients who got bisantrene in the 1980s and 1990s. This sort of information is just not accessible to us and many of the doctors are retired, etc. Most adult AML patients are old so it is unlikely that any adult patients would be alive from this time even if they were cured of AML. Looking at the Phase III breast cancer trial the longest lived patients were all those given bisantrene. The trial ended before all the breast cancer patients died so it is possible that some are still alive, but we just don’t know.
2. Yes we are in discussions with multiple cancer centres both in the USA and elsewhere. Having said this talk is cheap and it is actions that matter.
3. Paths 1 - 3 are most advanced. Pathways 4 & 5 need additional preclinical data, but we have started the process of collecting this data. We have thought very carefully at what we can accomplish within the resources we have. One of the huge advantages of having multiple independent pathways is that if we become too busy we can slow down on some of the pathways for a time and still be making good progress.
4. For the PRV to be awarded the FDA has to grant approval for the rare paediatric disease (paediatric AML in our case). You typically have to show improvements in overall survival, but given the life expectancy of these children is not great that doesn’t take long. The bigger issue is recruitment since paediatric AML is so rare it is hard to find the patients. A lot of work needs to go into ensuring enough sites are enrolled so that it doesn’t take a long time to recruit. Thankfully the FDA recognises this and so the trial sizes needed are modest (~30-40 patients). While we will need to wait and see how our trials run, if we get a results even half as good as the historical trials then we would have an approvable drug.
Hopefully the recording will be out next week once we have finished cutting out all the boring parts
(20min delay)