ImetelChat

Is it true this trial is ongoing and they added more patients?

rob: can you re-post his post here?

I wanted to but ymb won't let me copy paste but it is mentioned 40 patients added total 81 with completion date May 2017 .

robtmsstt1 wrote:I wanted to but ymb won't let me copy paste but it is mentioned 40 patients added total 81 with completion date May 2017 .

Also last updated Dec 2016 I believe its the Mayo study its 4 years old that will be presented to FDA.

Rob, this study was updated several months ago. The updates reflect the presence of a DMB, a total of 81 patients in the study and a May analysis date. Its all there on the clinical trials site. This study is the Tefferi original pilot study from Mayo reassigned to Janssen. bp

Thanks, not sure if anyone else finds this encouraging but for 4 years of data that some are still aLive is encouraging none the less.

Rob, well technically I don't think you can conclude that patients are still alive in the pilot study. One of the study conclusion criterion (among others, including drug approval) is that no patients remain alive. We know from Irish that John has done spectacularly well and ligas3 has contributed information about other patients so we might anecdotally conclude that yes patients are still alive. Remember that the Mayo pilot study looked at a different population of MF patients. If you look at the Geron corporate presentation there is one slide that shows a green eclipse, the tip of which just pokes into the R/R population (so just a few patients there) with the rest in the intermediate to high risk category. Nonetheless, yes, assuming some patients have survived to this point there seems to be a good chance of demonstrating survival benefit which would blow Jakafi out of the water if true. Since it appears that 81 patients will be evaluable (alive or not) by May, there may, and this is a stretch, be enough data for the FDA to conclude that Imetelstat is worthy of approval (this possibly independent of the FDA evaluation of the R/R patients which are much sicker.) In other words perhaps the agency will have data they consider adequate for evaluation and hopefully approval in intermediate/high risk (Mayo study) and R/R patients (Jansen study, currently suspended) to warrant approval for these indications. Its a long shot. But the whole deal has been a long shot. About twenty years to be exact. Regards, bp

Thanks, for your informative response I'm still to this day not sure why J&J took on r/r MF and I personally don't expect good results from this 2nd interim review based on these patients. I'm hoping that they will realize a longer study is needed to see results from imetelstat

Rob, I disagree, there has been nothing to suggest the study is not going well. The discontinuation of the low dose arm means nothing and the continuation of the high dose arm with suspension of enrollment could be looked at two ways. One, there is so much uncertainty that it is not worth the resources to continue to enroll or two, that they have enrolled enough patients (maybe fifty or so in the high dose R/R group) to make a conclusive decision and submit for approval. I subscribe to the latter more optimistic view especially since "encouraging" trends were noted early. The wording was quite careful about the suspension being related to being able to choose the appropriate dose, not about evaluating efficacy. This is just my own interpretation. We will know soon. bp

My problem is why did Janessan want to do a trial in MF r/r why not do similar study as the Mayo? It just seems to me uncharted waters however i can't see Janessan walking away from imetelstat there is so much facts in the science of the drug that i think can be used in other indications with the combo effect. i guess time will tell.

The path for accelerated approval goes through relapse and refractory. Just as imbruvica and darzelex. This is the mo for Janssen and fast track approval.

The 12 September Geron PR says about the 9.4: '../.. this arm warrants further investigation because encouraging trends in the efficacy data were observed'. 'Efficacy' means they saw the drug actually improve certain elements of the disease. We can only guess what they exactly saw, because nothing was disclosed. Based on these 'trends' they let the 4.7 patients go to the 9.4 arm (upon doctors decision), which would have been nonsense if that would not provide these very sick patients any chance on benefit. So I also subscribe to bio's 2nd option.