Agreed
@Moreforme. What makes Revascor unique is the fact that the now publicly discussed and patent pending mechanism of action for Revascor does not overlap with the two other classes of currently available heart drugs mentioned in the excerpt of SI’s presentation on the final results - see my post #75546267 above that homes in on the heart segment of the CC transcript. (I did my own research, just using plain old google and got detailed answers on the MoA for the latter.)
This point was highlighted a couple of times in the company’s own presentation a long time ago. It didn’t garner much attention then because a defined regulatory pathway seemed ages away, and of course that was before the company’s full attention became all riveted by the drama around the potency assay. Both are now ticks IMHO.
Dr. Krause, Dr. Rose and SI, in a moment of brilliance, presented the three heart trials in a bundle to the FDA, fresh on the heels of the potency assay discussion having reached a turning point. I believe that was when the penny dropped, that this was something very different from existing treatments, as SI highlighted again in the recent results presentation. Not only that, because in the 537 patient DREAM trial, it was done in a multi-centre fashion. That makes the approach a valid consideration, in fact one of only 2, for AA (this is stated in black and white in the FDA’s own guidance on AA considerations for RMAT designated products). Based on the admissibility of the data in this large multi-centre trial and the smaller but still well sized LVAD one, the company is then guided to pursue the AA pathway for the latter. I believe that the company would also have managed to put the message across successfully, that there is a unifying underlying theme for the company’s platform technology, which is immuno-modulating (not simply immunosuppressive) - anti-inflammatory among other things, and because it is multi-modal, it can work differently in different disease conditions. It would have then all started to make sense.
The other point that you mentioned,
@Moreforme, and posters including myself have canvassed quite extensively here, is the regulatory stance. The much better resourced Office of Therapeutic Products headed by Dr. Verdun, is tasked to look at biologic products which are a different ball game from small molecule drugs where the mechanism of action ie. understanding how the proposed treatment works, is less important to nail down because small molecule drugs are not from living organisms. MoA helps the determination of the choice of potency tests used in manufacturing control which are more important in cellular therapies by contrast because of the natural variability.
A while back, I looked up the org chart, and inadvertently noticed something very interesting. There are lots and lots of PhDs in biopharma and biology etc manning the new OTP, but the chieftain, Dr. Verdun, is the rare MD, and she is of course at the top. A medical practitioner with a lot of regulatory experience. I think she might even now still be practising medicine, because she was still doing it last time I looked a year ago. I believe this is significant. The meetings between Dr. Verdun and the company in the last 12 months would have been very significantly different from all the previous engagements in one important regard. It would have been among medical doctors like Dr. Rose and Dr. Verdun and others with a first hand understanding of the disease conditions with different patients of all shapes and sizes. These people understand variability but they are also people who are very curious about the how and why treatments work or they don’t. These people ended up where they do developing or regulating drug development, understandably because of their curiosity about translating their practising knowledge into treatments or products. It is likely a very different language that the company found itself talking in during these meetings. Then the significance of the trial and data started to sink in. I still remember in one of the webcasts earlier this year, the sense of positive surprise SI conveyed when talking for the first time about the new AA pathway the FDA started guiding the company to consider.
We know there is still a lot of work ahead, but the dramatic turnaround is not only in prospects for aGVHD but also in heart.
Not advice. DYOR.
(20min delay)