What have we learned from VERONA?

[biopearl123]

1. We learned that several studies along with VERONA have yielded disappointing results in improving OS and affecting disease modification in AML.

2. We learned that a large and important study can be stopped before its anticipated final analysis for failure to reach the primary end point. VERONA stopped at least a year early.

3. We know that IMbark continues on (with ongoing evaluations by the DSMB) and we can imply that the issues related to evaluating failure to reach primary end point, as in VERONA have not applied to IMbark thus far. Clearly though, not looking at the same disease but same standard of following for futility vs. efficacy.

4. We know that VERONA did not show meaningful alteration in the overall course of the disease (AML).

5. We know that Geron has plans to evaluate Imetelstat in combination with other drugs looking for similar end points (that's a guess) via the anticipated IMAGINE study.

6. We know that very few (if any) other drugs in the clinic or in development have a (semi) selective effect on the malignant stem cell and the malignant clone. That's a big one. Proving disease modification to the FDA (and the world) would be an amazing thing.

7. We know that Imetelstat has been shown to reduce and in some cases take to zero (!) VAFs.

8. We know of strong preclinical work from Dr. Bruedigam et. al. that extraordinary reductions in circulating blast cells in AML PDX models is possible.

9. We know that Geron plans to be around for a while given the strong hand of Harout.

10. We have a year or less to wait for the interim analysis of IMbark. (Dr. Mascarenhas has used the words Holy Grail if results very positive.)

11. We know that treatment earlier in hematologic malignancies does not appear to encourage the emergence of other malignant clones and might really change the course of these terrible diseases (yet to be proven.)

[huntingonthebluffs]

biopearl123, awesome analysis and thoughts as usual! Our approval and expectations are rising for our CEO as we recognize his confidence and knowledge, and willingness to act!

It has to help for him to know that RYTELO is something very special and at this point, "Holy Grail" might just be the challenge of working its way through the regulatory mesh. Getting there as quickly as possible will require his laser focus and a streamlined operation/organization, which has taken a major step forward today.