The related statements in the PR are not very clear. The lack of reproduciblity of the earlier data in 19 patients: is it related to a change in the clinical results (thus N is now less than 19) or a change in the number of patients with short telomere length. I read the PR statements several times and they are not clear. But, it appears to me that GERN may put more immediate focus in imetelstat in hematologic disorders rather than solid tumors.
I do not think the company has enough money/time (which would amount to many more years in phase 2) to do anymore exploratory survival studies in solid tumors.
In the case of AML and myelofibrosis and other types of non-solid cancer types, I think GERN will have enough money/time to get some NDAs here. The burn rate with the current approach to NDAs (physician sponsored trials rolling into company sponsored phase 2b and then phase 3) would be very low. The time span is also short.
GERN needs to show the world that they can get drugs approved by the FDA.
I do read their latest statement on the short telomere NSCLC trial patients as putting the nail in the coffin lid for solid tumor efficacy hopes.
I agree that hematologic malignancies seem strong contenders based on clinical results so far and resources available to get through at least Phase II trials.