Revisiting ASH

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biopearl123
Posts: 533
Joined: Fri Jul 20, 2018 5:13 pm

Revisiting ASH

Post by biopearl123 » Sun Dec 13, 2020 10:41 pm

Now that the actual slides are posted on Geron's site (why the site makes it so difficult to find I can't explain), I had a chance to review each presentation and would be interested in anyone else's take away's. The MDS trial is now 50% enrolled even at the height of COVID and is moving along. The Platzbecker MDS presentation confirms marrow improvement and TI's. Old data and it was disappointing not to see this updated. It could have been since the cut off was Feb of 2020 (contrast with Constellation data cut off of Sept 2020 in their MF presentation), it could have been done. As my old neuroanatomy prof used to say "Why?, we do not know." But the data itself stands alone and impressive as is, and the PIII should lead to registration for approval if confirmed. In the mean time there was the Mascarenhas data. Recall that Dr. Mascarenhas was only granted poster status at EHA, yet he had a lot to say at ASH as the value of the MF data becomes recognized. Evidence for fibrosis regression, symptom improvement, spleen improvement and identification of the genetic fingerprints of those likely to respond. It is particularly worth spending some time on the slide with all the little colored squares. These represent genetic mutations some of which disappeared completely. There are many genetic mutations that can be present in MF and picking away at some of them seems to me to be giant step toward picking away at more of them. (Read combination therapy.) Clearly if you reduce the malignant clones you alter the clinical manifestations of the disease and improve not only QOL but longevity. Proof of direct activity in shortening telomeres and direct effects on h-TERT have been conclusively demonstrated. Now all that remains is confirmation in PIIIs. I have not seen a drug with the same MOA in clinicals. It is remarkable that the value of this agent seems unrecognized, perhaps because of the timeline that lies between now and approval. A large Pharma seems unlikely to wait for actual approval or even PIII confirmation especially given the translational and patient safety data. So far it appears there is no partner in the wings. Why? We do not know. bp

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