Aquazul, It's not a side track. It's a great exercise to analyze potential competitors...
I'm happy to have this thread take deep dives into clinical trials and to compare/analyze the competitive landscape.... True competitors are the most likely at some point down the road to become potential suitors and one way or another to shape ultimate product value up or down - as medications butt heads in the clinic and companies jostle for position vying for market share and sales growth. Xpovio is a small molecule, which in therapeutics might be considered the opposite end of the spectrum from stem cells. It means small molecule companies employ very different technologies and talent (so less likely to become partners/suitors, but you never know). Karyopharm's Xpovio had $30M in oncology sales in 2019 and it's bogged down in phase 2 dose finding - even if they've enrolled about the same number of C-19 patients as Mesoblast and even if already approved for MM, I see them "well behind" in terms of getting to the C-19 finish line or ever becoming a threat in the ICU.
However, the exercise in analyzing them in some detail serves as a template for a more important competitor, namely Jakafi, which is on my list above. Jakafi is also a small molecule, but this one is a blockbuster despite its even longer list of side effects. It's projected to generate $1.6B in annual sales in 2020, growing at over a 20% clip, picking up more indications... and now coming into the C-19 space. I have no doubt remestemcel-L and Jakafi are going to butt heads in GvHD, C-19 and maybe elsewhere. That may start as soon as rem-L gets approval for children, depending on overlap on our age range high end and their low end. That competition will only increase as rem-L expands geographically and applies for adult GvHD indications. Jakafi is said to represent some 90% of annual revenues for Incyte. Even if GvHD/C-19 represents only X percent of total Jakafi sales, how might a $20B company like Incyte respond to the new intruder with a highly disruptive treatment sapping their market share? They may not be passive, so it's worth keeping an eye on them. For anyone who feels shy about making a contribution to the board, there are opportunities like that. Keep a close watch on what's happening in the GvHD/C-19/Incyte space and let us know if you find something interesting. I watch them loosely, things can slip by easily. Keep a watch on their C-19 trial, we already see some differences between Karyopharm and Incyte. What is the FDA telling us?
Regarding Europe, I'm wondering if anyone posted this paper with the positive call-to-arms title, "Mesenchymal Stem Cell Therapy Can Transcend Perianal Crohn’s Disease: How Colorectal Surgeons Can Help in the COVID-19 Crisis"
It appeared on April 4 and was authored by Dr. Amy Lightner of the Cleveland Clinic and Dr. Damien Garcia-Olmo of Jimenez Diaz University in Madrid. My apologies if already discussed on the board, here is a link to the paper:
Despite their appeal for colo-rectal surgeons to heed a higher calling and to get involved with stem cells during C-19, we might note that both of the good doctors are consultants to Takeda and both were involved in the studies that brought Alofisel to EMA approval. It almost sounds like they are proposing Alofisel be re-purposed to C-19 in the paper. However, Takeda has made no public pronouncement to that effect that I can find. We do have this interesting clinical trial that was registered two weeks after the above paper on April 16:
So 100 patients phase 2 at 7 different University Hospitals in Spain. To test: "Allogeneic and expanded adipose tissue-derivedmesenchymalstromal cells"
That would be Alofisel, would it not? However, the brand name Alofisel is curiously missing from the trial description. The trial was registered April 16, remains in "not recruiting" status and has not been updated. Takeda remains silent, however we do have this interesting help wanted ad that appeared on May 7 for a position in Massachusetts:
Job Description
OBJECTIVE:
The Alofisel Launch Lead is responsible for all strategic launch planning and tactical execution for the US launch of Alofisel, a first-in-class stem-cell based therapeutic. This will require broad, cross-functional leadership across Commercial and non-Commercial stakeholders. The role also acts as the main point of contact between the Takeda’s GI Business Unit and the New Product Planning team in developing and managing a portfolio of pre-phase III GI assets.
So, it would appear Takeda plans to launch Alofisel in the US - for fistulae, not C-19. Good for royalties, any thoughts?
In any event, some of the white papers I have reviewed do show that they are getting good results with the product... "life-changing results" for young adults who suffer from fistulae.
Meanwhile, Mesoblast results on the phase 3 Crohn's disease trial are pending... Perhaps we may not hear anything until after the PDUFA date, thoughts on that?
Regarding the interesting discussion on the thread being led by Nydem, I'll just add that getting patients to accept a live-saving treatment is one thing. Getting them to enroll in clinical trials can sometimes be a challenge. Yes, patients and families want a chance at a life-saving treatment... but when you throw in the little proviso "50% chance you'll get a placebo", that can dampen enthusiasm... Imagine enrolling in the Pluristem trial for example and realizing you may get 30 IM injections of placebo. Ouch. That would try my altruism. Our hats should go off to all 300 patients who agree to participate in the Mesoblast trial. We all benefit from those who came before us; we all have a moral imperative to help advance science if possible. But it's still asking a lot and no one is obliged. Many thanks to all who participate. IMO, Left-e
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