Rather than estimate TAM, I am going to compare the relative sizes of the different markets using roughlyestimated US patient populations as the common measure.
ADOA - 10,000
Rett syndrome (Neuren) – 10,000
Phelan McDermid syndrome - 20-25,000
DM1 – 40,000
AATD - 100,000
So, roughly, one might say that the potential market value of Entrada’s DM1 asset, with an est. US population of 40,000 is 4x that of PYC’s ADOA asset and 2x that of its Phelan McDermid asset.
Obviously, pricing then comes into play and that will be influenced by factors such as whether the asset is first to market; if not, how many competitors are there and what is the benefit of the drug v. competitors’ drugs.
When Vertex did the deal for ENTR-701 in DM1, there were 4 therapies ahead of it in development, including 2 in Phase 3, so it’s highly unlikely to be the first to market.
AATD represents a much larger market again; 10x that of ADOA. However, there seems to be 5 or 6 already-approved drugs for this condition and another 5 or 6 in advanced development, some designed to address liver issues and some designed to address lung issues.
A further factor to consider is the likely cost of development. Larger patient population diseases will require larger trials which will also take longer and be more expensive.
For example, one current Phase 3 trial in AATD (NCT01983241), which targets the lungs, has an enrolment of 341 patients, commenced in 2013 and is expected to complete in 2026!
I know I haven’t exactly answered your question but hope this is of some use.
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