Good Vibes

[LWS]

The "good vibes" from the ASH2024 abstracts continue as we await the conference starting December 7. However, all biotech stocks (PPS) are being affected by the "bad vibes" coming from a potential dysfunctional FDA in 2025.


3 Oral Presentations (ASH2024):

Abstract #52:-- Oral presentation on Saturday, December 7, 2024 at 10:15 a.m. PT by Claudia Bruedigam, Team Head, (AML)

Abstract #352: --- Oral presentation on Saturday, December 7, 2024 at 4:45 p.m. PT by Uwe Platzbecker, M.D., Department of Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases, Leipzig University Hospital, Leipzig, Germany (MDS)

Abstract #998 -- Oral presentation on Monday, December 9, 2024 at 4:45 p.m. PT by John Mascarenhas, M.D. (MF)

3 Poster Presentations:

Abstract #3222: --- Poster presentation on Sunday, December 8, 2024 from 6:00 p.m. - 8 p.m. PT by Uwe Platzbecker, M.D. (AML)

Abstract #3210: --- Poster presentation on Sunday, December 8, 2024 from 6:00 p.m. - 8 p.m. PT by Mikkael Sekeres, MD, University of Miami Health System and Sylvester Comprehensive Cancer Center (MDS)

Abstract #4590: -- Poster presentation on Monday, December 9, 2024 from 6:00 p.m. - 8 p.m. PT by Rami S. Komrokji, M.D., (MDS)

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Abstract #352: "Effect of Prior Treatments on the Clinical Activity of Imetelstat in Transfusion-Dependent Patients with Erythropoiesis-Stimulating Agent, Relapsed or Refractory/Ineligible Lower-Risk Myelodysplastic Syndromes"

Oral presentation on Saturday, December 7, 2024 at 4:45 p.m. PT by Uwe Platzbecker, M.D., Department of Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases, Leipzig University Hospital, Leipzig, Germany

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Abstract #4590: "Initial Results from the QTc Substudy of the IMerge Phase 3 Trial Demonstrate Clinically Meaningful Efficacy, Manageable Safety, and Absence of Proarrhythmic Risk in Patients with Lower-Risk Myelodysplastic Syndromes Who Received Prior Therapies Beyond Erythropoiesis Stimulating Agents"

Poster presentation on Monday, December 9, 2024 from 6:00 p.m. - 8 p.m. PT by Rami S. Komrokji, M.D., Vice Chair, Malignant Hematology Department, Moffitt Cancer Center
=================
Abstract #3210: "Correlation of Patient-Reported Outcomes with Red Blood Cell Transfusion Reduction and Rise in Hemoglobin in Patients with Lower-Risk Myelodysplastic Syndromes in the IMerge Trial"

Poster presentation on Sunday, December 8, 2024 from 6:00 p.m. - 8 p.m. PT by Mikkael Sekeres, MD, University of Miami Health System and Sylvester Comprehensive Cancer Center
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Abstract #998: "Trial Update from IMproveMF, an Ongoing, Open-Label, Dose-Escalation and -Expansion, Phase 1/1B Trial to Evaluate the Safety, Pharmacokinetics, and Clinical Activity of the Novel Combination of Imetelstat with Ruxolitinib in Patients with Intermediate-1, Intermediate-2, or High-Risk Myelofibrosis (MF)"

Oral presentation on Monday, December 9, 2024 at 4:45 p.m. PT by John Mascarenhas, M.D., Professor of Medicine at the Icahn School of Medicine at Mount Sinai
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Abstract #3222: "A Phase II Study Evaluating the Efficacy and Safety of Imetelstat in Patients with Advanced Myelodysplastic Neoplasms or AML Failing HMA-Based Therapy - Interim Analysis Results of the IMpress Study"

Poster presentation on Sunday, December 8, 2024 from 6:00 p.m. - 8 p.m. PT by Uwe Platzbecker, M.D.
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Abstract #52: "Overcoming Ven/Aza Resistance Through Imetelstat-Mediated Lipophagy in Acute Myeloid Leukemia"

Oral presentation on Saturday, December 7, 2024 at 10:15 a.m. PT by Claudia Bruedigam, Team Head, Leukaemia Metabolism Laboratory, QIMR Berghofer Medical Research Institute, Queensland, Australia

[LWS]

"Good Vibes" --- Rytelo is an exceptional blood cancer medicine, which will be reenforced by 6 presentations on December 7-9 at ASH2024 (AML, MF, MDS ---3 oral, 3 posters).

"Bad Vibes" --- Geron's PPS is now being influenced by the unknown future of the FDA, which now appears to be rather grim in these days of anti-science, disruption and political hacks taking charge.

"Unknown Vibes" -- Still, the importance of Rytelo cannot be ignored and European approvals are near. Fortunately, Rytelo is already FDA approved. 2025 is unpredictable and will be a year for the history books.

[LWS]

Considering the quality of the presenters, and their prestige as KOLs, I see only "good vibes" coming from ASH2024 for Rytelo. Perhaps there will be some "super vibe" that we are not expecting.


PT by Uwe Platzbecker, M.D., Department of Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases, Leipzig University Hospital, Leipzig, Germany (0ral)

PT by Rami S. Komrokji, M.D., Vice Chair, Malignant Hematology Department, Moffitt Cancer Center (Poster)

PT by Mikkael Sekeres, MD, University of Miami Health System and Sylvester Comprehensive Cancer Center (Poster)

PT by John Mascarenhas, M.D., Professor of Medicine at the Icahn School of Medicine at Mount Sinai (Oral)

PT by Uwe Platzbecker, M.D. (Poster)

PT by Claudia Bruedigam, Team Head, Leukaemia Metabolism Laboratory, QIMR Berghofer Medical Research Institute, Queensland, Australia (Oral)

[LWS]

If there are some "super vibes" for Rytelo at ASH2024 they are likely to come from ferroptosis, longevity and AML.

The AML presentations of Bruedigam (Dec. 7 - oral), Platzbecker (Dec.8 - poster), and Stevens (Dec. 9 - extra) will be closely watched.

========

Extra Review, AML

Forward Looking---- Monday, December 9, 2024, 6:00 PM-8:00 PM, Presented by Dr. Alexandra McLean Stevens (AML, Children)

Imetelstat is a first-in-class telomerase inhibitor approved by the FDA in 2024 for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia who are r/r/ineligible for erythropoiesis-stimulating agents. Imetelstat has also been shown to drive AML cells into ferroptosis (Bruedigam Nature Cancer 2024).

[LWS]

As far as I know, the "good vibes" continue.

I have not been able to find the fireside chat (Dec. 4) or the full ASH2024 presentations (Dec. 7-9) on the Geron Website. Either they have not been posted yet, or I am looking in the wrong place. From what I have read, the vibes are still all good from both the fireside chat and ASH2024.

We are waiting for the recommendations from the EMA and the MHRA/UK. Apparently, we will know something soon (perhaps tomorrow). These European recommendations need to lineup favorably with the FDA approval, especially for the long investment community. The PPS is still under the pressure of tax loss selling and hedge fund games. The next few weeks will be very interesting.

[LWS]

"Good Vibes" from CHMP & EMA for Rytelo.
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Geron Announces Positive CHMP Opinion for RYTELO™ (imetelstat) for the Treatment of Adults with Transfusion-Dependent Anemia due to Lower-Risk MDS
December 13, 2024

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of RYTELO (imetelstat) for the treatment of adult patients with transfusion-dependent (TD) anemia due to very low, low or intermediate risk myelodysplastic syndromes (LR-MDS) without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. The European Commission (EC), which has the authority to approve medicines in the European Union (EU), will review the CHMP’s recommendation and is expected to make a final decision on the marketing authorization application (MAA) in the following months.

“The positive CHMP opinion is an important step towards our goal to optimize value and patient access to RYTELO in the European Union, where we look forward to the opportunity to make this important new treatment option for LR-MDS patients available in select markets. If approved, RYTELO would be the first and only telomerase inhibitor available in Europe,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “We are deeply appreciative to the patients, caregivers, advocates and investigators across the EU who contributed to the clinical development of RYTELO.”

As part of its review of the marketing authorization application (MAA), the CHMP looked at the results from the IMerge Phase 3 clinical trial and assessed that the benefit of RYTELO in patients with transfusion-dependent anemia due to very low, low or intermediate risk MDS is a reduction in the need for red blood cell transfusions in the first 24 weeks of treatment compared to placebo, as observed in the double-blind controlled study. The most common side effects were thrombocytopenia, leukopenia, neutropenia, increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), increased alkaline phosphatase (ALP), asthenia and headache.

[LWS]

The "Good Vibes" are real and documented, but, at this moment in time, seem to be present primarily on this board only. Most of the investment world does not get their information from this board, where most of the information is honestly posted. Even so, many investment houses realize Rytelo's potential and have buy recommendations.

The medical successes are real, with the clinical TELOMERE trail (now getting off the ground) likely to support Rytelo (AML combination treatments with very significant synergies shown in preclinical results). Only a rejection by the EMA can hold Geron's PPS down (very unlikely IMO). We can do nothing about market forces (some likely not legal, but there is no one looking that has any control).

[LWS]

NEWS FOR GERON

Geron’s Promising Market Growth: Strong Sales Performance and Optimistic Financial Guidance
Jan. 13, 2025, 07:09 PM

Stifel Nicolaus analyst Stephen Willey has maintained their bullish stance on GERN stock, giving a Buy rating yesterday.

Stephen Willey has given his Buy rating due to a combination of factors including Geron’s strong preliminary fourth-quarter 2024 Rytelo sales, which exceeded consensus estimates and demonstrated significant sequential growth. This positive sales performance helps to counteract previous concerns about underperformance, driven by inaccurate third-party vendor data.

In addition, Geron has provided optimistic guidance for fiscal year 2025 operating expenses, which are lower than expected, indicating a more favorable financial position. The company’s management has expressed confidence in achieving profitability without further financing, supported by anticipated regulatory approval in Europe and subsequent commercial activities. These elements collectively highlight Geron’s promising growth potential in the market.

In another report released yesterday, Needham also maintained a Buy rating on the stock with a $7.00 price target.

[LWS]

NEWS FOR GERON

Strong Growth and Strategic Financial Management Bolster Buy Rating for Geron’s Promising Rytelo Drug

Jan. 15, 2025, 01:11 AM

Leerink Partners analyst Faisal Khurshid has reiterated their bullish stance on GERN stock, giving a Buy rating on January 13.

Faisal Khurshid has given his Buy rating due to a combination of factors that suggest strong potential for Geron’s Rytelo drug. The pre-announced fourth-quarter 2024 revenues for Rytelo are in line with expectations, showing substantial growth from the previous quarter, which indicates a robust early launch phase. This growth supports confidence in Rytelo’s potential to become a blockbuster drug for lower-risk myelodysplastic syndromes, backed by significant sales figures and positive survey data.

Furthermore, the disciplined operational expenditure guidance for 2024 and 2025, which is lower than previous estimates and consensus, points to Geron’s potential pathway to profitability. This financial prudence, combined with the promising market opportunity and strategic moves such as growing prescriber base and payer coverage, underpins the optimism for Rytelo’s continued success and solidifies the Buy rating recommendation.


Read More on GERN:
Geron to Showcase RYTELO Success at Healthcare Conference
Geron (GERN) Stock Surges on Game-Changing New Treatment
Geron announces positive CHMP opinion for Rytelo
Geron announces result from IMproveMF study at ASH

Note:43rd Annual J.P. Morgan Healthcare Conference (now ongoing). Geron does not appear to be there. What major health conference comes next?

[LWS]

Barclays Buy rating for Geron (Target $9)

Jan. 15, 2025, 06:15 AM

In a report released today, Peter Lawson from Barclays maintained a Buy rating on Geron (GERN – Research Report), with a price target of $9.00. The company’s shares closed yesterday at $2.94.

The word on The Street in general, suggests a Strong Buy analyst consensus rating for Geron with a $8.20 average price target, which is a 178.91% upside from current levels.

Based on Geron’s latest earnings release for the quarter ending September 30, the company reported a quarterly revenue of $28.27 million and a GAAP net loss of $26.45 million. In comparison, last year the company earned a revenue of $164 thousand and had a GAAP net loss of $44.81 million.

[LWS]

Rytelo has "good vibes" based on everything we know about presented papers, completed trials, ongoing trials and upcoming clinical trials involving combinations. Geron's very poor PPS is controlled by market forces that seem to always have the upper hand.

The next hurdle is approval by the EMA based upon strong recommendations. Hopefully, these approvals will push the PPS higher, as Barclays and many others are indicating. From all indications, we should hear from the EMA very soon.
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Previous:

“The positive CHMP opinion is an important step towards our goal to optimize value and patient access to RYTELO in the European Union, where we look forward to the opportunity to make this important new treatment option for LR-MDS patients available in select markets. If approved, RYTELO would be the first and only telomerase inhibitor available in Europe,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “We are deeply appreciative to the patients, caregivers, advocates and investigators across the EU who contributed to the clinical development of RYTELO.”

[LWS]

The news that cancer research has been stopped at NIH with the changing of the guard is not good news. The future of the FDA is very uncertain as the anti-vax, anti-science, non-expertise crowd takes charge. In hindsight, Rytelo was very fortunate to obtain FDA approval in 2024. 2025 will be a tough year for cancer research in the USA.

[LWS]

The discussion of vaccines in general, and cancer vaccines in particular is ongoing. Rytelo has been suggested as part of a cancer vaccine and as a cancer preventative (most recently as a supplement to chemotherapy). There have been numerous references to Rytelo, over the years on this board, as part of a cancer vaccine. These are early days, with the future and funding of cancer research a topic now very much in focus.
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A historic leap in cancer vaccines – here’s what you need to know
Nov 22, 2024

Preventive cancer vaccines exist that target viruses known to cause certain types of cancer.
Image:National Cancer Institute
Michelle Meineke
Senior Writer, Forum Stories

OUR IMPACT
What's the World Economic Forum doing to accelerate action on Health and Healthcare Systems?

This article is part of:
Centre for Health and Healthcare
This article has been updated.

The UK’s National Health Service has launched the world’s first trial for a cancer vaccine.
One in five people worldwide will get cancer in their lifetime and 20 million new cases were identified in 2022 alone.

Advancements in healthcare puts the spotlight on how to narrow the gap in diagnosis and treatment between high and low-income nations – also a key focus for the World Economic Forum’s Global Health Equity Network.
The launch of the world’s first trial for a cancer vaccine is not just groundbreaking in the field of medicine – but for the future of hundreds of millions of people in the 21st Century. This first-of-its kind vaccine is personalized to each patient and the culmination of many decades of research.

The new NHS Cancer Vaccine Launch Pad (CVLP) is a platform that will speed up access to a tailored cancer vaccine clinical trial for people who have been diagnosed with cancer – and accelerate the development of cancer vaccines.

[biopearl123]

LWS, to my knowledge Rytello has never been suggested as part of a cancer vaccine.

[kmall]

The Land Of Make Believe continues on I-Chat

[LWS]

There was interest in Imetelstat (now Rytelo) and cancer vaccines. I believe LCTX was spun off from Geron. Rytelo is now newly approved as a single medicine. These are early days, with combination possibilities just getting off of the ground. I suspect there will be renewed interest in Rytelo (and new chemistry) as possible parts of vaccines or preventatives. I do not know what happened to LCTX.
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Lineage Cell Therapeutics

CARLSBAD, Calif., May 12, 2022--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today reported financial and operating results for the first quarter of 2022.

We are working to advance OPC1 and VAC2 into their next phases of clinical testing, in spinal cord injury and oncology, respectively, as well as advancing our auditory neuron and photoreceptor programs through preclinical development and toward pre-IND meetings with FDA. We believe the combination of our disciplined use of capital and current balance sheet will support multiple years of progress, during which we anticipate reaching significant events with each of our clinical and preclinical programs."

Announced completion of patient enrollment in Phase 1 clinical study of VAC2 for the treatment of non-small cell lung cancer (NSCLC) by Cancer Research UK; Lineage has now assumed responsibility for further clinical development of VAC2 and any future development opportunities derived from the VAC platform; and
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Note: VAC2 is an allogeneic, or non-patient specific, cancer vaccine candidate designed to stimulate patient immune responses to telomerase which is commonly expressed in cancerous cells but not in normal adult cells. VAC2, which is produced from our pluripotent cell technology using a directed differentiation method, is comprised of a population of mature dendritic cells. As the most potent type of antigen presenting cell in the body, dendritic cells instruct our body’s immune system to attack and eliminate harmful pathogens and unwanted cells. To target cancerous cells, VAC2 is engineered to express the tumor-selective antigen telomerase, which is found in over 85% of all cancers. Because the tumor antigen is loaded exogenously into the dendritic cells prior to administration, VAC2 is a platform technology that can be modified to carry any antigen, including patient-specific tumor neo-antigens.

[biopearl123]

You have conflated Rytello with a vaccine approach. They are very different. It is not early days but decades. Lineage has written a “trust us, we can’t disclose everything” to its shareholders. Scarlett spun them off for good reason and at least now has an important drug on the market almost 10 years after he promised to. Give it a rest.

[LWS]

bp -- Perhaps, I have misunderstood. The premise was that Rytelo (in some form) was suggested as part of a vaccine approach (VAC2 ???). Your comment was that was a bad idea then and it still is. There have been suggestions to use Rytelo as a supplement to chemotherapy as a preventative (killing remaining cancer stem cells in the blood stream). Also, there have been suggestions to develop it into an oral form and use it as a blood cancer preventative (like baby aspirin to prevent a stroke). A vaccine and a preventative seem to me to be cousins. At this point in time, this is only chatting about possibilities.

The immediate hurdles in front of us are EMA and EU approvals. This appears to as easy hurdle to pass in the very near future. However, there seems to be a great deal of nervousness and disbelief from parts of the investment community, reflected in Geron's PPS (under $3 today). The truth is that we do not know how good Rytelo and its upgrades can be in all sorts of clinical combinations. There are many years of research ahead. So far, so good.

[biopearl123]

You did misunderstand. Rytello was not suggested as part of a vaccine approach. It has been suggested as potentially useful to prevent recurrence or at least delay recurrence after initial chemo treatment. You cannot manifest something by thinking it.

[LWS]

bp --- In order to improve my understanding of what has been attempted, could you please explain VAC2? I believe it can be classified as a vaccine attempt. I don't know if it was a complete failure or if it is still being studied. Also, I had assumed that in some way VAC2 involved Imetelstat or something similar. Was that also a wrong assumption?

I believe that there are some approved cancer vaccines that actually work, such as the vaccine for cervical cancer. However, this is a special class of cancer vaccines that target viruses. Blood cancers, as far as I know, do not involve viruses.