zu MMP7:
Definitely not a once in a lifetime type of drug, as there are many fantastic targets for RNAi. I will say, however, that this a unique and very valuable opportunity. Even though IPF is an orphan indication (under 200k patients), all 100k patients in the US are known, as it requires a diagnosis - usually because of some symptoms. Once being diagnosed, life expectancy is about 3 years, as there are no decent treatments - so it's a horrible disease. Another horrible aspect of IPF is that there are approximately 30k new patients diagnosed in the US every year.
MMP7 is also known as "matrilysin" and it has been shown in research as far back as 20 years ago that it is a key regulator of pulmonary fibrosis, except that nobody has been able to knock this gene down - that is, until now.
ARO-MMP7 is now beginning to dose its very first patients, and the hope is that it will work and save lives. If it does work, my guess is that IPF will eventually be no longer classified as an orphan disease, as it is very possible that Arrowhead's drug will grow the IPF population (by keeping them alive) to over 200k patients.
So why is this drug so unique?
1) There will be no difficulty in identifying patients, like it will be for AAT. IPF patients are known, so sales and marketing will be a breeze. AAT, however, only around 10-15% of the patients are known (out of 120k), so the challenge for AAT will be to try and identify more prospective patients that are afflicted with AAT.
2) IPF patients will need to take this drug for the rest of their lives, and every year you can add another 30k patients in the US alone. The amount of patients that can be added on a yearly basis for this orphan indication will easily rival the number of patients that get added on larger, non-orphan indications, as typical orphan indication increases are around 5% per year (like it is for AAT).
3) Since this is such a serious condition, and an orphan indication, the cost of this drug should easily surpass $75k per year (I'm guessing it could easily go well into the $200k range). Crunch those numbers and you'll see that you'll easily bring in $10B+ in yearly revenue after 3-5 years on the market. And if the drug works, Accelerated Approval after phase 2 is a no-brainer. At that point, analysts would be giving price targets in the $300+/share range based on this one drug alone. That's right, $300+ based on this one drug alone.
So even though it's an orphan indication, it will theoretically take advantage of a larger, non-orphan population, but also benefit at orphan drug pricing levels, especially since there's currently no competition and it's for an incredibly grave condition.
Realize that all has to go right in order for this to happen. We still don't have proof-of-concept for lung, let alone what the knock-down would be for this drug. I'll tell you this, given these circumstances, it is incredibly exciting.
https://finance.yahoo.com/quote/ARWR/community?p=ARWR
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