Additional comments to FDA

[biopearl123]

Filed after the “to be made public deadline, but will be still considered by ODAC” was a filing from “ Institute for Clinical and Economic Review”. This entity plans to compare metadata from the Luspatercept studies with IMerge with plans for this evaluation to take place by July 2024. Should be interesting. Wonder if the timing is such that any EHA submissions will be taken into account. I am sure the economists are scouring data for evidence of improvements in longevity which would certainly have an economic impact if proven. Not to mention the costs of transfusions, hospitalizations etc. There are a lot of treatments out there that cost money and don’t prolong life. Perhaps an agent that results in rarely seen but highly sought after disease modification, will prolong (and improve) life while reducing overall economic burdens. Wonder if any of this will be covered next week. Regards, to all, bp.

[Ryan]

Calm before the storm, it seems. I welcome it.
Seems I have one hand and a peace sign left to count down. Should be a good day.

[mistergern]

"Should Be" - words written with warranted trepidation. Hopefully, this time the chute opens.

[biopearl123]

https://www.regulations.gov/comment/FDA ... -0016-0012

Another heartfelt comment

[jingledsassy]

Rates right up there with Irish Trader 52.....We are all a part helping get Imetelstat to the Blood Cancer Patients.....Our real long wait is almost at an end.......Land Ho!!! Bp and Kmall are leading the effort with the rest of us.......Bottom Floor of the Start to Cure Cancer.......just thinking out loud I think I'll have a drink Sharon gave me goose bumps.....Really looking forward to this coming week...Hmmm that drink went fast I better have another......

[rccola335]

I know the push for the Luspatercept is largely based on convenience of not needing an infusion - we know the results are not comparable in terms of HgB increase - and the most important thing is QOL - I think this might end up being a much bigger factor then we have been thinking - Most people would be willing to sit through an infusion every three weeks if they can enjoy life again

I wonder if imetelstat gets first line for high transfusion burden

[biopearl123]

RC, waaay too sensible. FDA and ADCOM will probably limit approval to submitted data for the applied for indication. What oncologists do after (if) approval is a different story but still think they at least for now will require Lus failure even in high transfusion burden patients before reaching for Imetelstat. It would probably save time, money and capitalize on the potential to halt, slow or reverse disease progression if they just took your advice!

[LWS]

There really is no more to say until ODAC and the KOLs speak on Thursday. All recommendations (good, bad or indifferent) will come from ODAC on March 14th concerning Imetelstat. Everything that I have viewed is in the good (or better) category. This is a powerful, unique blood cancer medicine. The side effects are manageable. The safety factor is high. An ODAC positive review and an NDA (Transfusion Independence) approval is very likely.

[Bigfun]

Hi Folks,

Thanks to BP for giving me access a while back! I have followed this board for sometime and appreciate you all for keeping the discussions going.

Not sure if anyone has mentioned this but there is a waiver listed for the ODAC regarding a conflict of interest for a doctor who is involved in a different study in low-risk MDS sponsored by Novartis. Not sure that it is anything to be truly concerned about but I would be interested to see what his comments look like. Hopefully nobody will try to put their finger on the scale.

https://www.fda.gov/media/176605/download

[biopearl123]

Big fun, nice to see you posting thanks for your thoughts. First off we are in the big time now, everyone on that panel has a reputation and a level of expertise that allows the FDA to know they are getting very high quality input. Dr. Hunter is on the board by invitation, you can be sure he didnt’ self appoint. This indicates a level of trust and confidence by the other reviewers on the ODAC as well as the FDA. Of all the professions medicine is still highly regarded because of the integrity most doctors have. Reputation (a deserved one) is all important and not likely to be influenced by a “pay off” or conflict of interest that can not be overcome. If you look (not to hard even) you will see many prominent clinicians with relationships with companies. Someone with expertise has to run the clinical trials and oversee safety and data collection. The companies pay for the trials so it’s hard to be squeaky clean and unbiased. But I think most Doc’s really try to. Bringing in an outside consultant tells us that there is a reason for this, perhaps to bring an alternative argument for or against approval, or perhaps this person has expertise in Imetelstat induced cytopenias. You can listen to his comments on Thursday and make up your own mind. For my part I will go with the integrity part. The future of Geron depends on a careful examination of study design, data collection, outcomes and adverse reaction. This is a job for the experts. If you look over Dr. Hunter’s CV you will see that he is one of them.

[CKTC]

Dr. Hunter is the perfect addition to the ORDAC committee. The current roster includes two hematologists, but neither has a focus on myeloid malignancies. More importantly, Dr. Hunter's primary research focuses on understanding myeloid malignancies' molecular drivers. So, he should be especially interested in imetelstat, a drug that provides molecular evidence of disease modification.