RYTELO

[huntingonthebluffs]

Many of you may have known that RYTELO is the proposed trade name for Imetelstat. Anyway it was included in the FDA’s briefing document for the ODAC.

“ Executive Summary
Purpose/Objective of the AC Meeting
The Applicant is seeking approval of imetelstat (proposed trade name RYTELO) for the treatment of
transfusion-dependent anemia in adult patients with low- to intermediate-1 risk myelodysplastic
syndromes (MDS) who have failed to respond, or have lost response to, or are ineligible for
erythropoiesis stimulating agents (ESA).”

Might take some getting used to RYTELO versus Imetelstat.

[LWS]

MIAMI, FLORIDA (March 14, 2024) – Dr. Mikkael Sekeres is an expert in blood cancers at Sylvester Comprehensive Cancer Center - University of Miami Health System. He served on the Oncologic Drugs Advisory Committee (ODAC) for the trial that led to imetelstat vote of support at the FDA today. Imetelstat, also known as RYTELO, is telomerase inhibitor for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS). It will be used for the treatment of certain adult patients with low- to intermediate-1 risk MDS who have not responded to, or have lost response to, or are ineligible for erythropoietin stimulating agents (ESA) used to aid blood transfusions.

“I believe the benefits associated with the drug justify the risks. The benefits are real. When you have 40% of the patients who previously depended on red blood cell transfusions who no longer need transfusions, and this outcome lasts for an average of one year, I think that’s a significant benefit,” Sekeres said.

He added:

Some 20,000 Americans are diagnosed with MDS annually.
Anemia typically affects people 70 years or older.
No treatment options are available for anemia after ESA’s lose their effectiveness.

Dr. Sekeres is chief of the Division of Hematology at Sylvester and his specialty focuses on leukemia and myeloid disorders. He has served on the editorial boards for several medical journals and chairs scientific advisory boards for patient advocacy groups and research funding agencies. He has been the primary study investigator on dozens of phase I, II and III clinical trials, and has authored over 450 scientific articles. He is also an expert on CAR-T cell therapy causing t-cell malignancies issues, topic of ODAC’s meeting on Friday.

[LWS]

There is no medicine that does what Rytelo (imetelstat) can do as a stand alone medicine (transfusion independence, improved quality of life, longer life, disease modification, remissions, etc.). On approval (now a formality) off label uses will be suggested and tested. High on the list will be chemotherapy combinations. Other combinations are already in trials.

It now appears that Rytelo will be an important factor in the treatment of all blood cancers. It is too early to talk about cures, but some are expected.

[LWS]

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, announced that John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer, is scheduled to present at the following virtual investor conferences:

Stifel’s 2024 Virtual Targeted Oncology Days
Wednesday, April 17th at 11:30 a.m. ET (Corporate Presentation)

A webcast of each fireside chat and corporate presentation will be available through the Investor Relations section of the Company’s website under Events. An archive of the webcast will be available on the Company’s website for 30 days.

[LWS]

Stifel Oncology Forum today very positive (replay now available). #1 take away (beyond transfusion independence) --- "Dynamic Increase of Hemoglobin".

[LWS]

Rytelo --- in trials and off-label

Rytelo, as I understand it, is available for off-label uses now, as an approved FDA medicine, alone or with other FDA approved medicines. While ongoing trials will continue and new combination trials will be formed, there will be opportunities for some patients to get ahead of the curve in several areas, including various blood cancers and as an addition to chemotherapy (all cancers).

Rytelo took way too long to get their first FDA approval. Now is the time to speed-up the process for new applications, as Geron brings onboard new partners (manufacturing, distribution, combinations, research, longevity and beyond).

ODAC (all the blood cancer hematologists) recommended Rytelo (12 yes votes) and the FDA approved. The only serious discussion was over the safety profile, which was very good for such a powerful medicine (short term and reversible side effects).
===================

Previous (for reference)

There is no medicine that does what Rytelo (imetelstat) can do as a stand alone medicine (transfusion independence, improved quality of life, longer life, disease modification, remissions, etc.). On approval (now a formality) off label uses will be suggested and tested. High on the list will be chemotherapy combinations. Other combinations are already in trials.

It now appears that Rytelo will be an important factor in the treatment of all blood cancers. It is too early to talk about cures, but some are expected. ---LWS » Thu Apr 11, 2024

[LWS]

Rytelo has been observed to produce complete remissions in MF by Dr. Tefferi at Mayo Clinic. That was the key element that brought us (after many long years) to an FDA approval. There have been some strong indications about MF treatment successes. Where are the updates?

==========

bp --- Dr. T (at Mayo Clinic) apparently believed that he saw "complete remissions" (MF).

That needs to be verified and duplicated now, with Rytelo FDA approved. "Partial remissions", "complete remissions" and "disease modification" are the primary considerations and objectives. Combinations are now in focus (a major step beyond Dr. T's efforts).

************************************

Some History (Complete Remissions in MF):

“Imetelstat is capable of producing complete remission in some patients with myelofibrosis, suggesting disease-modifying activity,” said Ayalew Tefferi, MD, a hematologist at the Mayo Clinic in Rochester, Minnesota. “Imetelstat reverses bone marrow fibrosis and induces morphologic and molecular remissions in some patients with myelofibrosis.”

Note: Source of: "Tantamount to a Cure".

IMpactMF, our pivotal Phase 3 clinical trial in JAK inhibitor relapsed/refractory MF, is an open label, 2:1 randomized, controlled clinical trial of imetelstat to evaluate a potential improvement in overall survival compared to best available therapy. The trial is expected to enroll approximately 320 patients with intermediate-2 or high-risk MF who are relapsed or refractory to treatment with a Janus associated kinase (JAK) inhibitor. IMpactMF is open for screening and enrollment.

[biopearl123]

LWS, what Dr. T believed he saw along with Mayo pathologists who examined pertinent bone marrow samples have remarkably not been reaffirmed to date. Therefore it might be best to carve it in wet clay rather than stone. He has never explained the apparent discrepancies between his original report nor his fractured relationship with Geron. It would be wonderful if true, but we need to continue to wait for confirmation at which time we may be able to back fill some of the uncertainties.

[LWS]

Dr. Rizo, Rytelo, Geron and Vividion have much in common (Rytelo and combinations). We will see how this plays out.

Dr. Rizo, when she became the CEO of Vividion, she also was a senior consultant to Geron for a time, and, I believe to this day, has kept her Geron stock options. She saw an opportuntity:

" ---precision therapeutics for devastating cancers and immune disorders. The company’s platform has enabled it to identify hundreds of previously unknown functional pockets on well-validated protein targets implicated in a wide range of diseases, while simultaneously identifying compounds from its proprietary covalent chemistry library that interact in a highly selective manner with those pockets."


=========================================

I was simply pointing out that because a former CMO becomes CEO of a subsidiary of a major Big Pharma doesn't necessarily equate to a partnership or BO. Again, I'll be the first to admit I'm probably missing more than I hit, so my batting average isn't going to get me into the major leagues anytime soon. Always up for a debate and I sincerely appreciate all of your contributions here, even ones I don't align myself with. Just trying to keep things in perspective. -Kmall
************
About Vividion

Vividion Therapeutics, Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a clinical stage biopharmaceutical company utilizing novel discovery technologies to unlock high value, traditionally undruggable targets with precision therapeutics for devastating cancers and immune disorders. The company’s platform has enabled it to identify hundreds of previously unknown functional pockets on well-validated protein targets implicated in a wide range of diseases, while simultaneously identifying compounds from its proprietary covalent chemistry library that interact in a highly selective manner with those pockets. The company is leveraging its proprietary chemoproteomic platform to advance a diversified pipeline of highly selective small molecule therapeutics targeting high value, traditionally undruggable targets in oncology and immunology. For more information, please visit www.vividion.com.

[kmall]

LWS - Can you please stop copying and pasting my words on this forum. -Kmall

[LWS]

I suspect Dr. Rizo will be very much involved with Rytelo's future, having said that Imetelstat is, "compelling, remarkable and durable."

Dr. Rizo, as much as anyone, is responsible for Rytelo's current successes, ongoing potential, and bright future in combinations. Her position at Vividion will allow her to combine Vividion's novel molecules to Rytelo in new ways to fight cancer.

I suspect that she had that in mind when she made the change. Also, I suspect that she and John Scarlett stay in touch with each other more now, since Rytelo finally is an FDA approved blood cancer medicine. I believe that both still have common objectives to advance the use of Rytelo. We will see.

[bucbeard]

I'm sorry. I'm struggling to handle uniblab on every single thread. it's creating an unpleasant user experience.

feel free to delete this, cause I certainly do understand, but...i just needed to get that off my chest.

[kmall]

Bucbeard - Ditto

[biopearl123]

Ah yes, the famous “Department of Redundency Department.” I am hopeful that posters will take into consideration the sentiments expressed by fellow posters and take their suggestions and guidance to limit redundant posts. This approach would be far preferable to me than having to arbitrarily do so as moderator.

[LWS]

Hi bp, kmall and others --- My recent chats have to do with the relevance of Dr. Tefferi (past history) and Dr. Rizo (current history) as major parts of Rytelo's successes to date and potential going forward. My idea of a chatroom are conversations (threads) that evolve over time, with appropriate references.

If there are rules to be followed here, please post them. I will be happy to comply.

[Ryan]

I’ll volunteer here to try to set up some rules, or maybe just guidelines, so we can reconfigure and move forward rather than fall apart here… it’s too exciting a time for Imet for the Chat lose momentum.

So do your best to mind these guidelines:
- Don’t copy and paste other people’s words. People are specifically appealing for this.
- Don’t respond to yourself. As a main rule breaker here, I will use the computer rather than phone, cause phone is tougher on me;)
- Definitely don’t respond to yourself over and over. Seems like direct logic from the prior guideline
- Try to have something new or noteworthy, whether it be a fresh take or a fresh news, in one way or another. No rehashes really necessary at this point and seems to be more of a chafe rather than a refresh. This one I recognize is a grey area but LWS I would recommend to err on the side of silence here. I think we’re all on the same side here but rehashed information is not really sought out here.
- Try to have a little fun out here. But mainly stfu.

[kmall]

Ryan - great suggestions.

We all appreciate and value free speech as well as the discourse here focusing on all topics surrounding Geron/Biotech/Pharma. Speculation is great, but that's all it is. No one knows for certainty what's going on, except the true insiders - and they most likely are restricted from divulging sensitive information anywhere - especially a message board. Take most things here in stride and please offer concrete, factual based info with links to support posts - it helps. Simply posting a company's overview ad nauseum isn't exactly original. Once is just fine - but 2,3,4x.....we get it. We've all been here way past closing time. -Kmall

[LWS]

Ryan, bp and Kmall --- We all seem to have a difference of opinion on what the guidelines should be. The #1 guideline (IMHO) appears to be commonsense. Reference material, by its very nature, is repeated material. Silence makes little sense for a chatroom. Continuing a thought with adjacent posts is often necessary to keep a thread alive.

Certainly, there are differences of opinion about what is relevant and what is not. This, in part, is derived from if you are focused more on the investment or more on the science and potential. I am interested in both. I don't believe that there should be hard and fast rules.

Bp once said that I was "hyperbolic" about Rytelo. I don't think so. I believe that Rytelo has an important future in treating many cancers and perhaps longevity. I also believe that what Dr. Tefferi and others saw at Mayo Clinic were real complete remissions (to be verified). Afterall, without Dr. Tefferi's very positive inputs, Imetelstat would have died on the spot. There would be no Rytelo.

[huntingonthebluffs]

I think it is amazing and quite admirable of our membership that this board was able to be free of rules and discipline administered between members and on occasion the moderator for 10 years.

I agree with the ideas given by Ryan and kmall. This has only become a significant problem in the last year. I'm still chuckling over bucbeard's term "uniblabber", had to look it up for sure! Obviously we don't have to read everyone's posts but they have actually become cumbersome of late.

For my part, common sense is important but I have found over the years just how subjective that can be.

I would also recommend leaving national politics out, and avoiding all opinions/comments regarding religion, cults and national political or medical figureheads that we align with or not. We all seem to have an opinion regarding if the social and cultural fabric in this country/world is going in an unsustainable direction but like everything else there is 50% of us that believe we are going over the cliff but the other 50% think we will go over the cliff otherwise. In my experience we can only remain a united board if we steer clear of the world at large.

[FC4364]

I have benefitted from Information gleamed from this board; it is invaluable to be able to read comments from folks who are on the healthcare frontline as well as educated comments by an incredible board of learned folks in regard to where the next door will open for Rytelo.

I believe there is an army of sleuths out there researching, listening, reading and digging for the next morsel related to Rytelo. Sherlock Holmes would be green with envy.

A significant number of times, information shared on this board outshines any information provided by those supposedly in the know (Wall Street Analyst) and is usually right on and before they and the real world catches on. As, detective Joe Friday (Dragnet) said, "All we want are the facts, ma'am".

In my opinion, contributions from Kmall, Huntington, Hoosier, Ryan, Jayfish, rccola, KTArsenal, Jingledassy and Lacour set this board far above and beyond all others.

I also believe Bp has a tough job in keeping things civil and an extra shout out to him for keeping imetelchat the "Go To Place" for the real scoop.

FC