Gamida Cell Ltd (fka GMDAQ)

[Spideyboy]

Apologies for the long post, but I decided to go into Gamida M/C in a bit more detail. Thanks in advance for reading if you go through the whole thing.

Gamida presentation mentions:

Of 13,000 eligible patients for allogeneic transplant in the USA, there are 8,000 Stem Cell Transplants per year, which means 60% of the eligible population gets a transplant, indicating the 40% of the 13,000 don't get a transplant.

Additionally, we need to take into consideration that even among the 8,000 that do get a transplant, there is delay to engraftment increasing likelihood of graft failure, sickness and worst case with poor graft success or graft failure that these patient succumb to their cancer, for which HSCT is recognised as the only opportunity for cure of MDS, as well as being used for all the other cancer malignancies AML, ALL, CML, Lymphoma (HL & NHL) mentioned in the ppt.

Hence I believe why Gamida comes to the understanding of omidubicel being relevant for up to 80% of eligible patients,not just the 40% that don't get a transplant, as except for perfect HLA match patients Gamida can help all patients achieve higher engraftment potential and in a shorter space of time will be important for their success rate & survival.

To look at the total USA/EMA eligible population. 13,000 patients in the USA. Also in Europe (50 countries) there are 41,000HCST patients receiving HSCT transplants in 2017. But let's half that to arrive at something which would better reflect the situation in the 28 member states that the EMA covers. So let's go with 20,000 Patients in EU covered by EMA, receiving HSCT transplants. Now let's assume that number of patients receiving HSCT also only accounts for a similar 60% of eligible patients (simply due to matching issues not financial issues), then the total eligible EU patients would be 33,333 or 33,000, and 40% (13,333) currently don't get any transplant.
https://www.nature.com/articles/s41409-019-0465-9

From the Abstract of the article above: 42% of European HSCT patients receive allogeneic HSCT = 33,000 * 0.42 = 13,860 eligible european HSCT allogeneic patients. Putting USA and EU on similar number of eligible patients, which I think makes sense.

So the Phase III clinical trial would cover geographies for 13,000 + 13,860 = above 26,000 eligible patients.

Within HSCT transplants
- 82.5% of allogeneic HSCT are covered by Gamidas indications
https://www.nature.com/articles/s41409-019-0465-9
https://www.nature.com/articles/s41409-019-0465-9/figures/1

So 26,000 * 82.5% = we have above 21,450 eligible allogeneic HSCT patients for omidubicel in USA/EU28 per annum.

Say we use omidubicel in not the 80%, but 50% of the eligible population. That would mean 10,725 or 11,000 annual patients.

Average cost of stem cell transplant is 500,000USD in USA. And in Europe, it would seem perhaps about of 100,000USD.
(http://www.hurriyetdailynews.com/turkey-rising-star-of-europe-in-stem-cell-cancer-treatment-expert-141910). Let's say Gamida charges just 40,000 extra, which might seem alright given this can increase your HSCT success and chance at life, and cost less than 2 UCB units. Also why would you go through a normal Dual (2) UCB HSCT, have it potentially fail and then try again with Gamida? (I base my valuation on the below) source:

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5580430/
"In general, the ex vivo expansion studies require more specialized techniques available in only a few centers. However, the cost of procurement of each UCB unit is $30,000 to $45,000, which may make ex vivo expansion of 1 UCB unit more cost-effective than using 2 UCB units." Could mean about 60-90,000USD for normal 2 UCB units. Gamida could price at 80,000, but let's say they go with a low 40,000 and a mid 60,000.

11,000 Patients * 40,000 USD = 440M Revenue per year
OR
11,000 Patients * 60,000 USD = 660M,

In both cases, where Gamida should take a high profit margin.

A BLA provides 12 years exclusivity in USA, and EMA is about 10 years.

A 10x revenue (and with no other costs but a GDA-201 sequential Phase II and III trial over years) = 4B - 7B market cap.

Given that Gamida is already running the study in 45 centers across US, Europe, Israel and Asia (Singapore). For which 21 are of the 71 key USA transplant centres in USA, responsible for ¬80% of HSCTs. 16 centres in the EU (France, Italy, Netherlands, Spain, UK). Seems they should have a clear path to commercialisation with many key centres/physicians on board via the trial.

Then we tag on the prospect of approaching filing in China or India within the first 2-3 years of US/EMA launch, and we should definitely hit the 5 Billion mark?

Additionally when looking at the competition. There was Mesoblast with MSC co-culture, but that seems to have gone the way of the dodo.
https://clinicaltrials.gov/ct2/show/NCT01854567?term=mesoblast&draw=2&rank=6

And then there is also the Fred Hutchinson Cancer Research Center (FHCRC)/Bernstein Lab which appears that it in Phase II trials and thus years away.

So it would seem like there would be no competition for the ex-vivo expanded UCB market.


How do you feel about all this and feel free to point out anything I may have missed.