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Monday, 04/03/2023 12:27:28 AM

Monday, April 03, 2023 12:27:28 AM

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Good conversation you two are having. Most of it well above my pay grade. However, without the same grasp of details, I was also arriving at a similar big picture scenario. Continue IB with patients with ACL>900 and update results in Lugano in June and then hopefully the data lines up for the Phase 3 registration trial mtg with FDA with fast track Orphan drug status.

CAR-T Gene therapy was mentioned in the prior posts as a possible competing treatment for PTCL. I believe Miller really took a few shots at this line of treatment in one of his previous presentations. If CAR-T does show promise and make it to market, I doubt that this treatment would be priced less than $500,000.


Given the difficulties treating PTCL and the relative rare occurrence rate. I agree w the 8K annual estimate in the US based on the following:
https://acsjournals.onlinelibrary.wiley.com/doi/10.3322/caac.21589
Given this limited US market, I can see the FDA-Orphan drug pathway as reasonable shot. I have no idea how it would be priced. $150K is certainly consistent w other life saving treatments. If trials are successful, I would also assume that the emphasis would shift to front line treatment or combo in order to reach patients before their immune response is degraded by Chemo.

Thanks for the discussion.

FL
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