Hi Reg, "...Also in that article is price per vial - with Si...

Hi Reg,

"...Also in that article is price per vial - with Si indicating pricing over 500k in pediatric SR aGvHD...."

IMO that''s conservative.

Not sure if I've mentioned this previously, but I understand from recent Mesoblast' shareholder briefings, including the AGM and Feb 29 2024 Earnings call:

1. the reason that we are in the USA (and not in EU) with Remestemcel-l seeking initial marketing approval is principally the compelling economics of USA reference pricing; and

2. the pricing for Remestemcel-l for paediatric SR-aGVHD would be:

- only comparable to that of a curative paediatric drug (rated in terms of assessed Qualty-Adjusted-Life-Year benefit). Note that Amtagvi (Iovance), approved in Feb 2024 uses TIL cell therapy, is presently priced at USD515,000 (not including hospital fees and side-effect addressing drugs), has very significant side effects and is not considered generally curative,

- priced in a range having, as a lower limit i.e. a minimum under any circumstances, the current CAR T-cell therapies pricing i.e. USD375,000 to USD500,000

- in MSB execs estimation, significantly in excess of USD500k per treatment. CEO Itescu's exact words at the February 29 shareholder call were, in part:

" ...And I think when you -- some of the gene therapies that are providing that kind of curative outcome charge some pretty remarkable prices.
I would say that we would see that pricing for this product is somewhere between the CAR-T products and the curative gene therapy products... "

What are those curative gene therapy prices? Here's a list as at 25 March 2024 of the most prominent of the approved cell and gene therapy products available in the US atm. I think everyone will be able to tell which ones are considered the curative gene therapy products:


Link HERE. The article had this example to give of ICER's cost-effective pricing calculation in the US at the moment:

" Assuming a five-year event-free survival rate of 35% with second-line Yescarta and 10% with standard of care, Yescarta was cost effective at a willingness to pay ratio of $150,000 per QALY, as DrugTopics summarized. Yet Yescarta was no longer cost-effective if its 5-year event-free survival was less than or equal to 26.4% or if it cost more than $972, 061 at a willingness-to-pay threshold of $150,000."

The article goes on to point out that cost-effectiveness assumptions are still being made about the long-term benefits associated with Zolgensma, for example. The authors comments seeméd to me to highlight Mesoblast' products relative cost-effectiveness advantages, viz:

" Ultimately, whether cell and gene therapies can deliver on their clinical promise while justifying their frequently high costs will become clearer in the coming years as more long-term data become available.

The durability of treatment benefits remains a critical consideration; a frequent argument for the therapy class is that one-time therapies with lifelong effects are far more cost-effective than those requiring repeat administration.

The size of the treatable patient population is another important dimension, with ultra-rare disease therapies facing a steeper challenge in recouping development costs than treatments for more prevalent conditions.

Finally, the availability of alternative treatments, even if less effective, can raise the cost-effectiveness bar for new gene therapies.": (emphasis added)

I think Mesoblast' management are very focussed, and I expect that MSB's products will be viewed as realtively curative compared with these therapies. Those 4yr-plus long-term survival statistics for Remestemcel-l will be critical IMO.

Cheers & have a good one.
GLTA(LT)H