Accent Therapeutics: What can we learn?, page-18

1. Yes there is no need for a liposomal formulation, but it is covered by our patents anyway.

2. Yes. Not only can the historical data be used for the IND, but we are using the historical data for the IND. We have access to both the Lederle and NCI historical and IND data. The reason we can use it is we have made a lot of effort to keep the formulation and manufacture the same as the original Lederle formulation. The FDA has already stated they consider our drug the same as Lederle’s.

3. Golden Biotech is just another one of the hundreds of biotechs trying to run trials in AML. I had a look at the trial (it is on clinicaltrials.gov) and I am less than impressed. There are about 3 dozen companies that presented at ASH 2020 with better drugs.

4. Yes if we change the formulation we can not rely on the historical data for FDA approval, but this is totally irrelevant as what we are doing in Pillars 1 and 2 has no historical data. It only matters for Pillar 3 and only for extramedullary AML and MRD.

5. I am rather skeptical about claims from management that the FDA changed the goal posts at the last moment. In my opinion what is much more likely is that management didn’t ask the FDA the right questions up front. The FDA does not tell you what to do, they just answer your questions. It is up to you to work out what they want - they are much like that difficult girlfriend (or boyfriend) that won’t tell you what you need to do or say to get back in their good books, you just are expected to work it out on your own.

6. I am not sure what the relevance is in taking out the EM AML patients that responded from the trial??? Every patient with EM also had bone marrow AML too - what was interesting and unexpected about the Israeli trial is that the EM patients responded at all. EM patients are considered so difficult to treat that they are actively excluded from most clinical trials - investigators don’t want them in the trials as they tend to make the trial look bad when they don’t respond to treatment. When you look at the population that was treated in the trial (very late stage, short time to live, heavily pretreated, and with EM) the response rate was amazing.

I will respond to reasonable critics and even outright trolls on occasion if they have something of value to add, but I be more circumspect with the mindless trolls.