I did a little digging and need to correct myself on GvHD off label assumption use.
First, my calcs ( and I think everyone's calcs ) are based on the company stating it's rollout target, which is the hospitals that treat approximately 50% of all pediatric GvHD cases. This equates to around 300 possible patients from those hospitals ( but 300 other patients elsewhere ) so around 600 total pediatric.
The adult SR a GvHD population that is refractory to second line treatment, is approximately 5 x larger than the child population. This would place adult cases around the 3,000 mark, making the toal population ( child and adult ), that are SR to be approximately 3600.
From that we could say that of a total of around 11,000 patients given a transplant, that are on baseline immunosuppression as part of the transplant, about 50% develop aGvHD and then move on to have " first line " therapy , which is optimised immunosuppression ( steroids ). This is the 5500 number. There are no "official" approved second line therapies, which is why statistics around how succesful they are do not exist, but bnased on MSB's numbers, when used with the above article, it looks like around 1900 respond, leaving 3600 non responders out of the 5,000.
Great - but what does that mean for on / off label use market size?
On Label Just the 300 child pediatric patients in the hospitals the therapy will be marketed, at $400,000 per course - weight adjusted. 120M USD in sales There are another 300 pediatric patients at other hospitals that could potentially be sold to on label. 240M USD total on label.
Off Label The Adult SR + second line refractory market is 5x larger, and the dosage more expensive since it is weight adjusted, I used $600,000 per course and 3,000 patients 1.8B USD total Off label for Adult SR + failed second line
If Adults are treated first line ( when aGvHD develops), rather than as a last resort after pumping full of steroids. I used the 4300 number here ( total cases of 5500 minus 1200 children, leaves 4300 adults ) 2.58B USD total off label for Adult First line aGvHD
If the treatment moves into the preventative space, and is administered as a standard part of the transplant procedure instead of / in combination with the baseline immunosuppressants. We are back to the 11000 total transplant number. For ease of maths I'll use 500,000 per course since this includes children and adults. 5.5B USD.
Notes. Potentially the range of sales just for the aGvHD indication should be somewhere between targetted on label figure and preventative use case. From 120 Million USD to 5.5B USD per year depending purely on off label use. During COVID ARDS MSB estimated the maximum possible throughput from 2D growth reactors, was 10,000 treatments I believe stated for 6 month output from memory ( so annualized I believe it was 20,000 ARDS courses)
Note that an ARDS course is at least 1/4 of the GvHD course , but the GvHD course can also be extended by 4 weeks ( to be double ) This means 20,000 ARDS course is the equivalent of only 2,500 up to 5,000 GvHD course depending on weather the patient recieves the second 4 weeks worth. The other assumption was that MSB would require to use ( I think at the time ) all of the 2D suites known in USA to produce that figure.
This means, before the company can treat more than 2,500 patients they will need to transition over to bioreactors. That could equate to somewhere approaching the 500M to 1B USD in sales as I think it is unlikely in the normal course that MSB would persue every suite available when it could be much easier to transition to 3D bioreactors anytime the product can pass the characterisation and potency tests.
MSB Price at posting:
78.3¢ Sentiment: Buy Disclosure: Held
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