Phaedrus is right that there are definitely more than enough pigs flying in the air for shareholders to smell bacon.
People should remember how complicated these diseases are, how far the company has come on its journey and how much understanding of its products and processes it has developed, progressed and is now capitalising on, on the back of problems and delays. Not to mention the knack of getting regulatory alignment that it has finally been developing. These are excellent outcomes for long suffering shareholders but remember we are investing in biotechs. We go in with eyes wide open that massive R&D expenditures are required and there are bound to be many misses. However, with people like Australian Super resisting Origin Energy going into the hands of private equity, there are lots of reasons why we should be glad that we are still able to smell the pork.
Just wondering about your thoughts on how something with an RMAT designation can get AA according to the FDA’s own guidance. There is mention on p.10 of that guidance that one of the two legs this type of approval can stand on is a significant number of sites. I was actually curious when I read that. You can google how a biologic with an RMAT can get an AA from the FDA to bring it up. It will be easier, of course, for the company because Dr. Krause is there to give the advice. I have no doubt that the company still needs to design the right primary endpoint for any P4 confirmatory trial, but an attempt to get alignment that such a trial could be approved at warp speed by the FDA would be very interesting if only to test the response of the regulator who was in the room to hear that D. Perin started his work in 2001, as anyone, even the blind, can see the benefit of a therapy which is not already offered by other available drugs in the market in such a condition. Doing it of course in the right way ie. to target success, is still crucial. I am much more confident now, however, that with the understanding it has developed from past failures, the company’s chances of success are good. The options are starting to be in-the-money. Commercialisation is not far, nor is take-off velocity. Execution skills will be key.
(20min delay)