Why is this man smiling? Or, the thing about good studies
Forum rules
- Comments must be civil and on topic
- Back up claims with evidence/reasoning/sources (posting links is allowed)
- No commercials/harassment/spam
- Comments must be civil and on topic
- Back up claims with evidence/reasoning/sources (posting links is allowed)
- No commercials/harassment/spam
-
biopearl123
- Posts: 1677
- Joined: Fri Jul 20, 2018 5:13 pm
Why is this man smiling? Or, the thing about good studies
Why is JS smiling that confident smile? It’s not because FDA approval for LR MDS is pending. He smiled that smile a long time ago but maybe some people just didn’t notice. Nope. He is smiling because he knows two prominent things (among the many). #1. The AML study was extended by a year a while back. #2. Good studies have an objective panel of caretakers charged with periodic reviews of the data know as the data and safety monitoring board (DSMB or some other similar name). They have a lot of power and with that power comes responsibility. Responsibility to patients who have taken a chance on a yet unapproved therapy. They can 1. Stop a study for futility 2. Stop a study for safety concerns. 3. Allow a study to continue because numbers 1 and 2 don’t apply. They are allowed to know things and they are smart. So while they might not know who exactly is on study drug (in a blinded study for example), they probably have a pretty good idea. For an unblinded study (say comparing drug to a best available therapy arm), there is little question as to who is on study drug and how long they are living or what is happening to their symptoms or if a drugs side effects are problematic. The ongoing Geron studies have an active DSMB. And that is why JS is smiling. Best Wishes, bp
Re: Why is this man smiling? Or, the thing about good studies
I emailed the facility in Australia (QIMR Berghofer) who twice has said that the Impress trial was impressive - I asked how many patients they treated with imetelstat who had AML - they didn't respond - I thought it was worth a chance - they did give me a top fan badge on facebook whatever that means
Re: Why is this man smiling? Or, the thing about good studies
Yes it might be time to acknowledge what has happened since the ides of March…
We finally, finally (finally) have had recognition of what everyone on this board has been screaming from the mountaintops for it looks like about a decade, Imetelstat is something special. It’s next generation ‘siblings’ probably won’t be so bad themselves…
And biopharma also was able to see who leading the scientific team at Geron, Dr. Feller. I think Dr. Scarlett can smile when he knows, she’s got this. And, considering the potential acquirers, what bp executive does not want a rock star on their team?
We finally, finally (finally) have had recognition of what everyone on this board has been screaming from the mountaintops for it looks like about a decade, Imetelstat is something special. It’s next generation ‘siblings’ probably won’t be so bad themselves…
And biopharma also was able to see who leading the scientific team at Geron, Dr. Feller. I think Dr. Scarlett can smile when he knows, she’s got this. And, considering the potential acquirers, what bp executive does not want a rock star on their team?
-
huntingonthebluffs
- Posts: 254
- Joined: Wed Feb 24, 2016 12:00 am
Re: Why is this man smiling? Or, the thing about good studies
At the Needham fireside chat I think Dr. John Scarlett was more upbeat than I have ever seen him. He made several comments that reflected his enthusiasm and confidence that both patients and investors would be rewarded for their patience.
Of course, he also reiterated many of the key points from past presentations as well. Including $7B TAM by 2031 in US/EUA for MDS & MF. Current cash on hand should cover through 2Q26. TI is the ultimate QOL measure for MDS patients.
The following are approximate specific comments that he made:
Exciting time for patients, investors and Geron. Imetelstat would only be the 3rd drug to be approved in 20 years in the MDS space. It is both setup to be very helpful for patients and will be extremely valuable from a ROI perspective.
Subject of buyouts comes up frequently because there are relatively few late-stage assets available in this space.
Overall we remain open minded, our #1 goal is to get this drug to patients and that is why we are preparing to launch this drug and expect to launch with enthusiasm and an awful lot of belief that we are really doing something special for patients.
We take into account the fact that we have many long term and very, very patient and loyal investors we want to see rewarded. There are many ways that reward can come but we look at all that taken together and will adjudicate all the different possibilities as they come, in the interest of all the fund administrators and investors.
No existing convincing readouts of OS in CTs to date. IMpactMF is well powered to provide strong results. it’s pretty easy to say that if readouts (definitely was smiling here) of the IMpactMF CT are positive, it will be transformational for R/R MF patients, and we are pretty eagerly looking forward to that readout.
Of course, he also reiterated many of the key points from past presentations as well. Including $7B TAM by 2031 in US/EUA for MDS & MF. Current cash on hand should cover through 2Q26. TI is the ultimate QOL measure for MDS patients.
The following are approximate specific comments that he made:
Exciting time for patients, investors and Geron. Imetelstat would only be the 3rd drug to be approved in 20 years in the MDS space. It is both setup to be very helpful for patients and will be extremely valuable from a ROI perspective.
Subject of buyouts comes up frequently because there are relatively few late-stage assets available in this space.
Overall we remain open minded, our #1 goal is to get this drug to patients and that is why we are preparing to launch this drug and expect to launch with enthusiasm and an awful lot of belief that we are really doing something special for patients.
We take into account the fact that we have many long term and very, very patient and loyal investors we want to see rewarded. There are many ways that reward can come but we look at all that taken together and will adjudicate all the different possibilities as they come, in the interest of all the fund administrators and investors.
No existing convincing readouts of OS in CTs to date. IMpactMF is well powered to provide strong results. it’s pretty easy to say that if readouts (definitely was smiling here) of the IMpactMF CT are positive, it will be transformational for R/R MF patients, and we are pretty eagerly looking forward to that readout.