Licensees and MSCs

The first part of this post is along the lines of vinns words yesterday morning;
“if you have the science the money will eventually come, the trick is to survive in between”

We all love the bigger picture, the block busters but they are still a little way off for producing income, but of course we cannot predict precisely when the partnerships will start, might be Monday might be December or later.

But MSB needs to start bringing in income beyond rebates sooner rather than later.

JCR Pharmaceuticals with their partner Medipal will probably ramp up in the sales and royalties. Their existing pipeline does not include any new MSC based products so far as I can see.  There is another product being researched, being driven by a researcher outside of JCR it appears.

However the licensee I find most interesting is TiGenix for a number of reasons, some of them I’ve already covered but now I can add a few more in relation to Cx601 or Alofisel.  A few days ago while wandering around I found this;

“TiGenix considers price points between $40,000 to $50,000 as conservative, and indicates that optimal pricing would fall between $60,000 and $120,000 per dose 5 January 2018”

The same location has lots of other interesting stuff, please DYOR.  While you are at it perhaps have a look at what TiGenix and also Takeda have to say.

OK, that’s one part of a guesstimation, next we already know the royalty MSB is to receive is a single digit percentage from the MSB ASX notification of 15 December 2017.  Then the bigger unknown is just how people in the EU countries have complex perianal fistulas as a result of Crohn’s disease.  I found various figures for the US and the UK but not across the EU.  As a figure I settled on 2000 across the current EU.   If anyone can point to some real figures that would be much better.

Takeda has to negotiate with each country in the EU with regards to pricing, for the purpose of this exercise I picked a conservative $50,000 for a treatment, 2000 people and a 3% royalty, that returns US $3,000,000 in royalty payments.

Make it 5% with the other numbers and it becomes $5,000,000.  Pick the median price TiGenix would like, $90k, with those numbers and it becomes $9,000,000. Maybe there are more or less than 2000 afflicted people, maybe the royalty percentage is less, maybe it’s 9%, I don’t know.

Takeda do want to get a move on, going to the member states in an effort to get this treatment out over the coming months.

Not a massive amount in the scheme of MSB expenses, there has already been €5 million up front  as part of license agreement and probably another mile stone €5 million payment is due shortly based on current approvals.  There is another €10 million falling due for other milestones that will also be coming.

Some of that income could, for instance, help finish a second tier MSB program with 28 day primary and secondary endpoints.  This is a trial about a disease with an Orphan designation in the US and other places, the Crohn’s Disease trial in Phase 3.

Why would MSB bother to continue with a Crohn’s Disease treatment when Takeda have one about to be released across the EU?  Because the targets of the treatment are different.  I have bolded some bits for clarification.
----------------------------------------------------------
8 December 2017
The most recent update on CinicalTrials.gov for the Mesoblast trial titled - Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's Disease
A Phase III, Multicenter, Placebo-controlled, Randomized, Double-blind Study to Evaluate the Safety and Efficacy of PROCHYMAL® (ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Intravenous Infusion for the Induction of Remission in Subjects Experiencing Treatment-refractory Moderate-to-severe Crohn's Disease
Recruitment Status : Active, not recruiting.

	Column 1	Column 2
0	Estimated Primary Completion Date :​	July 2018
1	Estimated Study Completion Date :	July 2020

https://www.clinicaltrials.gov/ct2/show/record/NCT00482092
----------------------------------------------------------
15 December 2017
- Mesoblast grants TiGenix an exclusive global patent license to use adipose-derived mesenchymal stem cells in the local treatment of fistulae.
----------------------------------------------------------
Takeda have a license only for the local treatment of fistulae. It appears a single treatment package will have 4 vials of 30 million cells each, to carry out four injections to treat up to three fistula tracts.  A total of 120 million cells.

Mesoblast is aiming for the Induction of Remission of Crohn’s Disease.  The trial uses an intravenous infusion of suspension of adult human mesenchymal stem cells, total of 1200 million (high dose) or 600 million (low dose) cells infused in four visits over two weeks.

A trial with remission as the endpoint, perhaps that explains the 2 year period from the end of primary data collection to the end of the trial, to give time to observe whether or not remission is brief.  If Takeda is interested in a local fix maybe someone will be interested in remission.

The actual time line of events around TiGenix is interesting to look at, TiGenix documents it well.
One of those events was;

20 December 2017
- TiGenix confirms strategic focus on Cx601 and its adipose derived stem cell (eASC) platform

Their next announcement is where Takeda announces their intentions toward TiGenix, but the platform announcement above brings me to sepsis, I think that affects more people again.

The TiGenix sepsis solution, Cx611 is based on their eASC platform like Cx601 is.  Cx601 was the subject of the license agreement with MSB, I suspect that Cx611 will be too if it gets closer to commercialisation.  TiGenix is a member of the SEPCELL Consortium looking at cell based solutions to Sepsis.   Currently in a phase Ib/IIa clinical trial.