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No negative insinuation; I think it's fabulous that they are on an FDA published list of vendors that may produce a product that needs FDA regulation. Nothing bad about that at all. Just pointing out that i'm not willing to take the leap that the date on that page correlates to some recent action/filing by PMCB with the FDA...for reasons stated. It could, and once again, I hope it does.
Just did a random sampling of their SRO list, and every one of them is showing an updated date of 01/03/2020. So, the fresh updated date does not look specific to PMCB...although I really wish it was.
Wolfy you've been hitting on a couple things today that I am very sensitive to. You are aboslutely correct. In the real world these "misses" (and i'm using that term broadly) would never be tolerated; there would be real penalties/incentives/ramifications/plans in place to mitigate all of this. I don't really care what the root cause is, be it poor planing by PMCB/AN or other partners...at the end of the day it all sits at the feet of KW. For all those that say that he is working hard, doing the best he can, learning, biologics are hard, etc...well that's all fine except this isn't really a nice guy business where you get to learn on the fly on our dime...he is getting compensated as someone who does not make mistakes, has the i's dotted and t's crossed, knows the details and should have mitigation plans in place accordingly; he is getting compensated as if he knows what he's doing. And that includes the leadership to make sure AN and other partners deliver as properly contractually obligated. Nobody said trying to take an emerging biologic to market is easy, but if someone is going to get compensated as a big boy, then they have to produce...simple as that.
I agree a big company like a Merck would have the proper infrastructure in place to take this up a notch. Problem with that is, what are we worth to a big pharma before proving something? So, although that may be the technically correct approach to move this to fruition, it may not reward the longs here who have and still do believe in the actual technology.
For all the serial haters here, you can kiss my aXX...this is not intended for your little hating world...like it's your job...because it is. Same for the pumpers. Y'all are not that transparent, in spite of what you may think.
Check the managerial incompetence at the door and get this in the clinic to prove or disprove this technology one way or the other.
This may be of interest to you purple:
Thank you for sharing this info.
Also, because such studies are being done by other companies for us, we cannot control the way such studies are done and the timing of them because they may be involved with studies for other companies as well.
You may remember that in 2017 we lost essentially 6 months from our timeline simply because we could not obtain the proper growth medium for the cells that we use for the population of our Master Cell Bank and then used for encapsulation because the single supplier in the world (a major company) was unable to produce it according to specifications
As for the involvement of TD2 – no, they will not be involved in conducting the trial. This was essentially their decision because they did not believe that they had enough infrastructure to coordinate the conduct of a trial like the one we will be doing. TD2 is more suited to coordinating the conduct of Phase 1 and early Phase 2 trials.
I agree. I'm also expecting the dreaded "Phase2b IND delayed due to trial redesign" announcement.
If that happens, I am going to call complete BS on this. How much time have we already spent designing, redesigning, re-redesigning a Phase2b trial??? I'm going to guesstimate 1-2 years. Just dig out the design we took into the FDA pre-IND...that's what we walked into that meeting with. Remember, Hidalgo, Lohr and Von Hoff were involved in that Phase 2b design... So, imo, if they come out with that excuse (and i'm expecting it), then it is nothing but the latest fabricated way to drag it out, diluting the hell out of this to raise money for their ongoing huge salaries. Remember, they get paid as this goes along, while we indirectly lose value.
Yes, that we know for sure. A lot of assumptions here, but...
Best case is that we are 5 weeks into testing right now, and worst case we are 1 week into testing.
Sterility and Mycoplasm should be completed first, taking on the order of 3 weeks (assuming they are run in parallel). Worst case is in 2 weeks, those tests should be done and cells shipped to AN for further testing and encapsulation. Best case is they have already shipped 2 weeks ago.
Assuming parallel testing at Eurofins and the subcontractor, the total testing duration for non-AN tests should be about 7 weeks. So, strictly from a Eurofins/subcontractor perspective we have anywhere from 2 to 6 weeks left.
AN has to repeat the Sterility and Mycoplasm tests (3 weeks). So, giving a week to ship to AN, their testing/encapsulation should end at roughly the same time as Eurofins. Worst case late March.
I'm assuming a lot of parallel processing going on here. I would NEVER have used the term parallel processing and PMCB in the same sentence before...drove me nuts how they seemed to do everything serially. But, the fact that they have stated Eurofins and AN testing will take place concurrently is a welcome change, and tells me the tone has changed and they are showing a sense of urgency now...for the first time I have witnessed. Why is that?
Would be curious to get Wolfy's thoughts on this.
Regarding diabetes retrieval, I asked the specific question: "Does the CIAB technology meet the retrieval requirements of the FDA for diabetes?" The answer I received: "If the capsules need to be retrieved they can be, depending in where they are placed in the body. If they are placed in a location where there might be a possibility of their migration, they can be contained in some way so as to make their retrieval (along with the cells inside them) possible. Please always keep in mind that the genetically transformed cells cannot escape from the CIAB capsules."
So, they are talking in terms of "if", "depending", etc. To me, that isn't really a plan. Like you said, after years of working on diabetes, and especially with the consortium experts, you would certainly think they would have something more concrete with regarding to solving the retrievability problem. Why they don't...well your guess is as good as mine. But if we are guessing about possibilities, I wouldn't sleep on Novo Nordisk. Very late 2016 their senior management admitted that finding a viable encapsulation platform was the only roadblock for their stem cell diabetes treatment. He also admitted they "are collaborating with certain groups on technology to encapsulate the transplanted cells to help protect them". But he wouldn't name any names.
I had an email exchange last week with Investor Relations on the topic of "cell retrieval".
Their answer matched yours regarding PC: "The retrievability issue is only relevant for chronic conditions like diabetes where the capsules may reside in the body for a long time."
That said, there was another recent post here that referenced a Lohr presentation from ASCO 2017 as saying that in the pre-IND the FDA said they didn't care what happened to them after activation. That does not match with the response I received from Investor Relation. I asked them specifically whether it was discussed with the FDA at the pre-IND, and they mentioned that it was not discussed, and has not been discussed since. So, based on the information I received, it seems that they think it is a non-issue for PC, but it was not discussed at the pre-IND or since. That does not mean it wasn't discussed a long time ago with the FDA, which is what I think Lohr was referencing in that ASCO presentation. (Listening to the clip, I did not hear him say pre-IND), but he certainly did mention that the FDA said...when, where...who knows.
So, if I take that information and use the thing between my ears, I would lean towards it really not being an issue for PC because think about it, does cell retrieval really matter for a death sentence? Investor Relations did say that the cells could be removed if the tumor was being surgically removed. If the tumor is being surgically removed, it's a really good thing for us (well, for those who really want the treatment to succeed...not necessarily everybody on this board). Ideally, it would have been confirmed with the FDA, and maybe it has at some point, but IR says it was not during pre-IND or after.
Since Austrianova had that statement on their website that cells can be retrieved if needed, I did reach out to their contact information, but have not received any response...nor have I ever received a response when reaching out to Austrianova. I'll give it to PMCB, at least they took the time to communicate with me.
I think diabetes is another issue altogether...
I'll go out on a limb and say that retrieving post mortem would not satisfy any FDA minimum safety requirements :)
It is encouraging that the AN site does say they can be retrieved if necessary.
I did go back and re-read the post pre-IND company PR's and it was said that the single most important rate limiting factor was the MCB and WCB (at that time). No mention of retrieval at all.
I emailed the company yesterday. I'll post if I get a reply.
Thanks for the reply.
Yes, I think it would be an excellent point for the company to clarify. I will email them...may not get to that today though.
I guess my opinion is cautiously optimistic for the reasons I stated regarding the pre-IND. I mean, this question would be a poster child for what that meeting is intended for...imho. So I really hope we would have heard something if we had a problem. Could the requirements for PC and Diabetes be different? Possibly, but I think that's not a large possibility...doesn't quite make sense to me...why one and not the other. I'll reply here if I hear anything back.
Thanks.
I'm a longtime (mostly) reader of this message board and really appreciate your posts and knowledge.
Regarding "The cells need to be retrievable for the fda to approve trials"... I am honestly a little confused on this. I know that on at least 2 previous occasions you wrote that our encapsulated cells are "retrievable". Has something changed recently to change your opinion on that? Also, I know the JDRF is looking for "retrievable" solutions, but I wasn't aware it was a hard FDA requirement. And lastly, I would have thought that something like this would have been talked about at the pre-IND we had with the FDA as being a show-stopper, yet in any PR's or conference calls we heard nothing about this at all...yet other things were mentioned that they needed to finish up. So, for something so major...it would basically be back to the drawing board with little chance for any kind of a quick path to IND, I would've thought we would have heard about it in the company's post pre-IND messaging if it was indeed a problem?
Again...thanks for the info and knowledge you share. I'm a long, i've been in this for 4+ years and have a decent position.
Great info Wolfy3. I appreciate your insight!
No problem at all. I was just verifying that I hadn't missed something that changed along the way.
Wolfy3 I really appreciate your posts. You obviously have a wealth of knowledge...thanks for sharing. A small thing, but I thought the number of patients was at least 250 (reason for not going with TD2)?
Well, the PR said "recent" data, and I could make a strong argument that "recent" could and should mean the current prescribing instructions for Abraxane/Gemcitabane. The Celgene website matches the PR stats.
However if you do want to cite that retrospective study, then I believe it's only fair to point out significant stats such as the fact the dosing reducing negatively impacted the median progressive-free survival from 5.5 months to 4.8 months. A step backwards, at least in one regard, from the study results which were the basis for FDA approval.
I actually thought the PR showed great restraint. As an example, in the Abraxane/Gemcitabene trials which were the basis for FDA approval, median for the number of cycles of treatment given were ~4. The PMCB studies outlined in the early PMCB trials were self-limited to 2. Limited because at the time the survival characteristics of the encapsulated p450 cells were unknown. That is not the case now, and for the planned trial, the CIAB treatment will now be on a level playing field where treatment will be repeated until disease progression (as was the case with the Abraxane/Gem study). Basically, the cuffs are off...so (hopefully) the results improve. We'll see...
I'm not a pumper at all, I just want to make money. That's the reason I looked further into what you had said...just in case there was some legitimacy to it.