First of all congratulations to all MSB holders (I know you are on here reading every post) I can't say I'm surprised with the outcome. Interesting though how even LT holders suddenly became very quiet after the ODAC briefing papers were published. I really enjoyed the presentions, especially SI's one. Saying he "aced it" is an understatement. If I was a MSB holder with a decent size holding, no doubt my first born would be named after him (even if it was a girl!).
Having said that, the ODAC hearing made it very clear what the FDA is looking for: manufacturing at commercial scale.
With Gen1 manufacturing methods, there are limitations - fact.
Having to rely on multiple donors does leave room for batch-to-batch inconsistencies as adult stem cells can't be expanded indefinitely.
In the first hour it was mentioned that MSB can currently treat about 400 patients from one donation. Sadly, these patients are very sick children, with the majority of them having lost a lot of weight by the time they receive treatment.
Manufacturing at commercial scale for an orphan indication such as SR-aGvHD means producing treatments for less than 200,000 patients per year in the US.
Given remestemcel-L's impressive stats over currently approved treatments for these patients, 500 donors per year doesn't sound too much of a risk. That's what the PMDA thought 5 years ago as well (with numbers even lower than that).
If we now convert these numbers to adults with COVID-19 ARDS, you are looking at 200-250 treatments per donation?
What if you want to use these cells in a non-life-threatening indication such as OA?
In my opinion, a lot of rubbish is being posted about "there can only be one" etc. This is not a remake of Highlander. With some posts, you can't help but wonder if the person posting it is running around with a tin hat foil all day, "because THEY can read your mind"...
There is always a market for a product that could solve problems of an already approved product, that is cheaper, maybe works better in certain indications etc. A lot of data is still required though until such assessment can be done. 15 patients won't cut it! It is a start - and a very good one I might add, but nothing more.
We will get our trial one way or another: if the situation worses, we have MEND to start recruiting in NSW, otherwise we will have our world's largest iPSC P3 trial commencing in addition to FujiFilm's P2 in SR-aGvHD in Japan.
Yes, FujiFilm. So much doubt for no reason in my opinion.
Yes, there are other companies out there manufacturing iPSC-derived MSCs.
Citius is one that has been mentioned on here recently.
I listed to their Chairman Leonard Mazur recently, and he was talking about something revolutionary on the CEORoadshow: They have the ability (in theory as not tested yet, not to mentioned having completed a P1 clinical trial in humans) to manufacture all cells ever needed from one donor Wow... We have been doing that in Australia since 2012... Just saying. https://www.youtube.com/watch?v=7AlwOa90B4c
Whilst they are US based and have therefore access to more funding, it will still take them a while to go through all the pre-clinical and clinical studies.
I am looking forward to competition in this space, as it may also give us also more exposure.
On a side note, the US market loved their announcement that the FDA told them to please complete a few animal studies first before coming back:
Yes, there are even companies out there already SELLING iPSC-derived MSCs.
FCDI of course doesn't do that for free. I assume that they want to be paid based on the cells we manufacture using the iPSC starting material.
The more indications we license out, the more cells that need to be manufactured using our Cymerus technology, the more FujiFilm will earn - even if it is not in GvHD.
FujiFilm's recent advances, establishing them as one of the main forces in the CDMO sector, make this partnership even more valuable. As we know, the technology transfer is currently ongoing (hence FujiFilm had to seperatelly enter into an agreement to have us manufacturing their P2 cells at Waisman). Once that is completed, knowing that they will make money of our cells anyway, I wouldn't be surprised if their well connected global sales network won't be used to also offer our products.
Interesting to see their research collaborations: “Pro-angiogenic scaffold-free Bio three-dimensional conduit developed from human induced pluripotent stem cell-derived mesenchymal stem cells promotes peripheral nerve regeneration” https://www.nature.com/articles/s41598-020-68745-1
FujiFilm's J-TEC being the CDMO and CRO for Cyfuse K.K. plus "a grant from Takeda Pharmaceutical Company Limited."
Other companies investing millions to have a global sales network set-up, we may have gotten it for free because our licensee is also our customer
That brings us back to the delay last year and a recent FujiFilm announcement may have shed some more light into that: "FUJIFILM Irvine Scientific to Sell FUJIFILM Cellular Dynamics iCell Products" "FUJIFILM Irvine Scientific creates the FUJIFILM “Discovery Research” organization to enable speedy, efficient delivery of FUJIFILM Cellular Dynamics iPSC-derived products through their worldwide distribution channels." https://fujifilmcdi.com/news-events/news-item/fujifilm-irvine-scientific-to-sell-fujifilm-cellular-dynamics-icell-products/
IF FujiFilm is planning to establish one entity to be responsible for their worldwide distribution, based on the original agreement with Cynata that would have not been possible due to the sub-sub-licensing issue. FujiFilm didn't complete the Irvine Scientific purchase until mid 2018. In order to streamline their operations, obviosuly changes had to be made, allowing them to sub-sub-license certain rights to these "new" companies. That is now possible.
Also, has anyone noticed that despite their being a ~30% fee payable to WARF on licensing agreements (see Apceth's payment for more information), no payment has been made... yet? Is that maybe also an adjustment that was made as part of the WARF-Cynata-FujiFilm re-negotiations last year?
Anyways, in my opinion we don't need MSB's success to rub off on us, we can get there ourselves and and on our own merits. Enough said for one day. Enjoy the ride (now that the placement stock is just about dried up)!
CYP Price at posting:
70.0¢ Sentiment: Buy Disclosure: Held
I can't say I'm surprised with the outcome. Interesting though how even LT holders suddenly became very quiet after the ODAC briefing papers were published. I really enjoyed the presentions, especially SI's one. Saying he "aced it" is an understatement. If I was a MSB holder with a decent size holding, no doubt my first born would be named after him (even if it was a girl!).
Wow... We have been doing that in Australia since 2012... Just saying.
(20min delay)