Cathy's questions

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biopearl123
Posts: 1665
Joined: Fri Jul 20, 2018 5:13 pm

Cathy's questions

Post by biopearl123 » Mon Jun 08, 2020 12:09 am

Please see previous post re Cathy's questions. To be fair, regarding value creating events, continuing and completed enrollment in both PIII studies certainly are value creating. The loss of the AML study is not. But unless there is a partner, a positive change in approval status or a buyout, it is tough to see short to mid term stock appreciation unless the importance of the new EHA data is fully appreciated by the market at large. Frankly, I think this will depend on public relations more than anything since most of the data is already out there with the possible exception of actual bone marrow data showing actual disease modification (OS as a marker for disease modification is already in the public domaine but not largely appreciated.) AML remains a mystery. Does Imetelstat modify transformation from MDS (even low risk can transform) and MF? One would think so since the malignant clone is destroyed or at least modified leaving fewer malignant cell the statical likelihood of transformation. I am trying to sort out however and would appreciate some help as to the importance of clonal drift and "pressure" on the malignant clone. My limited understanding is that as Imetelstat works to limit the CSC and malignant clone, more resistant cells (and more disturbed mutations that are harder to treat) may emerge. So if patients live longer, have fewer symptoms (spleen that shrink and anemias that are easier to treat) is the ultimate price more complex mutations and a tendency toward AML development. I am basing this though on next to nothing but seek clarification. Could this be why front line MF (a logical place to go) is not vigorously pursued? I would like to see these issues addressed in a scientific forum. Re the MDS study. Dr. Scarlett has been extremely good at managing expectations and is projecting approval in early 2024. Its not that great a leap however to hope that the data/safety monitoring board might see such a profound statistical effect early on ( early unequivocal achievement of primary and safety end point) that the study could be stopped early for efficacy. This is pure speculation but there is a hard press to enroll a lot of patients in a significant number of newly added centers. The timelines have been well outlined by Ryan: 8 weeks median for drug effect. We will have a much more stringent control group than the Lus study. The overall study seems to be designed to made a very profound statement and it might make it before mid 2022 even with the new hurdles levied by a 3 micron virus. bp

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