Ann: Impressive Bisantrene Phase 2 AML Clinical Results, page-91

Reply to @rfctiger43

There are a couple, some more important than others.

Firstly, the small sample size of n=20 is not sufficient to draw a firm conclusion in the eyes of physicians and regulators that this drug is safe and effective. It does however indicate that on the balance of probabilities, it will be. As with all drugs, a large confirmatory P3 trial that is statistically powered to prove safety and efficacy beyond a chance occurence will still be required. I and many other holders view this trial as yet another confirmatory indicator of this drugs safety and efficacy.

Secondly, this trial was conducted using an older formulation of our drug, RC110. RAC intends to develop the newer formulation (which has significant commercial and clinical benefits) RC220, and use it for subsequent trials. The risk of there being a meaningful difference between the effectiveness and safety of the two formulations is minimal, but not zero.

Thirdly, the dosage used in this trial is different to what we are expecting to use in future trials. Similar point as above, we believe the different dosing to be more effective at the other indications RAC is targeting for commercialisation. Even though the doses are different, this result strongly indicates success with alternative dosing regimens.

This trial was never intended to lead to registration of bisantrene. It was investigator-sponsored so RAC had no input in the design of operation of the trial beyond supplying bisantrene. So for any existing holder of RAC these results are not new, nor are they unexpected (perhaps the results are slightly better than we expected if anything).

What RAC have with bisantrene, with the Sheba trials and historical data, is the most de-risked preclinical asset I have ever seen, with its sights set on one of the biggest markets in the pharmaceutical industry, and a disease that almost everyone has been personally touched by. In my mind the only risk to this company is management execution risk, which may or may not be resolved in the near future. I'm still a hold until the new strategy is released but I've never been more confident in the science.