ADCOM
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- Comments must be civil and on topic
- Back up claims with evidence/reasoning/sources (posting links is allowed)
- No commercials/harassment/spam
Re: ADCOM
=================================================================Anyway, I believe in Imetelstat now (alone) and look forward to off label uses, combinations, synergies, oral Imetelstat, and IMET2.0 (new formulations). from LWS
KTA --- Let's keep this board focused on Imetelstat and its progress with ODAC and the FDA. Without ODAC positive recommendations, there is no future for Imetelstat.
Re: ADCOM
Material for ODAC (Oncology ADCOM)
Imetelstat is listed as one of the "Top 10 Most Anticipated Drug Launches Of 2024"
Release date：2024/1/9 14:13:59
===================
NO. 10. Imetelstat
▶ Company: Geron
▶ Indication: Lower risk myelodysplastic syndromes (MDS)
▶ Status: PDUFA date of 16 June 2024
Imetelstat is a first-in class telomerase inhibitor for the treatment of transfusion-dependent anemia in patients with lower-risk myelodysplastic syndromes (MDS).
The NDA submission is based on results from IMerge Phase 3, in which the primary endpoint of 8-week transfusion independence (TI) was significantly higher with imetelstat vs. placebo (p<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders. Mean hemoglobin levels in imetelstat-treated patients increased significantly (p<0.001) over time compared to placebo patients. Statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category. Patient-reported outcomes (PRO) data reported a sustained meaningful improvement in fatigue for imetelstat-treated patients vs. placebo. Safety results were consistent with prior imetelstat clinical experience.
The Center for Drug Evaluation and Research (CDER)
===================================================
Imetelstat-PHASE-3
Note the fatigue factor. Beyond longer survival times, transfusion independence and disease modification this factor deals with the quality of life.
Imetelstat is listed as one of the "Top 10 Most Anticipated Drug Launches Of 2024"
Release date：2024/1/9 14:13:59
===================
NO. 10. Imetelstat
▶ Company: Geron
▶ Indication: Lower risk myelodysplastic syndromes (MDS)
▶ Status: PDUFA date of 16 June 2024
Imetelstat is a first-in class telomerase inhibitor for the treatment of transfusion-dependent anemia in patients with lower-risk myelodysplastic syndromes (MDS).
The NDA submission is based on results from IMerge Phase 3, in which the primary endpoint of 8-week transfusion independence (TI) was significantly higher with imetelstat vs. placebo (p<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders. Mean hemoglobin levels in imetelstat-treated patients increased significantly (p<0.001) over time compared to placebo patients. Statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category. Patient-reported outcomes (PRO) data reported a sustained meaningful improvement in fatigue for imetelstat-treated patients vs. placebo. Safety results were consistent with prior imetelstat clinical experience.
The Center for Drug Evaluation and Research (CDER)
===================================================
Imetelstat-PHASE-3
Note the fatigue factor. Beyond longer survival times, transfusion independence and disease modification this factor deals with the quality of life.
Re: ADCOM
ODAC and CDER are both parts of the FDA. IMHO, with the EAP in place (can be considered a phase 4 -- John Scarlett) the NDA Imetelstat approval is a done deal. CDER considers Imetelstat to be one of the top 10 medicines of 2024.
=================
The Center for Drug Evaluation and Research (CDER) is a division of the U.S. Food and Drug Administration (FDA) that regulates over-the-counter and prescription drugs, including biological therapeutics and generic drugs . CDER evaluates new drugs before they can be sold. CDER is the lead Center for regulation of human drugs that are regulated by FDA under the authority of the Federal Food Drug and Cosmetic (FD&C) Act. Biological products are covered by the Center for Biologics Evaluation and Research.
Regarding communication between CDER and FDA, the FDA’s CDER Small Business and Industry Assistance (SBIA) can be contacted via phone at (866) 405-5367 or (301) 796-6707 or via email at CDERSBIA@fda.hhs.gov . The FDA has published a guidance document for industry and review staff on best practices for communication between IND sponsors and FDA during drug development. The guidance document outlines the scope of interactions between the sponsor and the review team, types of advice that are appropriate for sponsors to seek, general expectations for timing of communications, and best practices and communication methods. The guidance document suggests that formal meetings between FDA and sponsors, written correspondence from FDA, submissions from sponsors, acknowledging receipt of communications, email between FDA and sponsors, general telephone calls between FDA and sponsors, faxes between FDA and sponsors, and use of out-of-office messages by FDA and sponsors are all acceptable communication methods. The guidance document also provides resources for sponsors, including FDA guidances, policy and procedures, basics for industry, interactive media, presentations, labeling and approvals, and rules and regulations.
============
Note: A PDUFA date is the date by which the Food and Drug Administration must respond to a New Drug Application or a Biologics License Application. The PDUFA date is set by the Prescription Drug User Fee Act, which requires the FDA to review new drug applications within a certain period of time, typically 10 months. The PDUFA date is important for drug developers and investors, as it indicates the potential approval or denial of a new drug.
=====================
=================
The Center for Drug Evaluation and Research (CDER) is a division of the U.S. Food and Drug Administration (FDA) that regulates over-the-counter and prescription drugs, including biological therapeutics and generic drugs . CDER evaluates new drugs before they can be sold. CDER is the lead Center for regulation of human drugs that are regulated by FDA under the authority of the Federal Food Drug and Cosmetic (FD&C) Act. Biological products are covered by the Center for Biologics Evaluation and Research.
Regarding communication between CDER and FDA, the FDA’s CDER Small Business and Industry Assistance (SBIA) can be contacted via phone at (866) 405-5367 or (301) 796-6707 or via email at CDERSBIA@fda.hhs.gov . The FDA has published a guidance document for industry and review staff on best practices for communication between IND sponsors and FDA during drug development. The guidance document outlines the scope of interactions between the sponsor and the review team, types of advice that are appropriate for sponsors to seek, general expectations for timing of communications, and best practices and communication methods. The guidance document suggests that formal meetings between FDA and sponsors, written correspondence from FDA, submissions from sponsors, acknowledging receipt of communications, email between FDA and sponsors, general telephone calls between FDA and sponsors, faxes between FDA and sponsors, and use of out-of-office messages by FDA and sponsors are all acceptable communication methods. The guidance document also provides resources for sponsors, including FDA guidances, policy and procedures, basics for industry, interactive media, presentations, labeling and approvals, and rules and regulations.
============
Note: A PDUFA date is the date by which the Food and Drug Administration must respond to a New Drug Application or a Biologics License Application. The PDUFA date is set by the Prescription Drug User Fee Act, which requires the FDA to review new drug applications within a certain period of time, typically 10 months. The PDUFA date is important for drug developers and investors, as it indicates the potential approval or denial of a new drug.
=====================
Re: ADCOM
Comparing the Stifel fireside chat (Nov. 14, 2023) & the B. Riley chat (Jan. 18, 2024 -- when available)
The chatroom today at B. Riley will add new information. All interested parties need to listen.
=====================
The chatroom today at B. Riley will add new information. All interested parties need to listen.
=====================
by LWS » Tue Nov 14, 2023 10:15 pm
Here is an outline of the fireside chat (Nov. 14) at the Stifel Healthcare Conference with John Scarlett. I suggest listening to it to get the full, very positive inputs. There is much to expand upon.
1/ Preparing for commercialization at time of approval (very close)
2/ Nature of ODAC changing with opportunity to expose Imetelstat to the world through KOLs and other experts
3/ In middle of ODAC review must be very careful what he says
4/ 20 investors or so at conference
5/ Not clear if a ODAC meeting is necessary; referred to Vertex/CRISPR arrangement ***
6/ Talked about ESAs and luspatercept
7/ Connection between transfusion independence, disease modification, and bone marrow reversals
8/ MOA; unprecedented results
9/ EAP a transition and a community service with an eye on full approval
10/ Will update partnering in 2024
11/ Discussion of Overall Survival Time and the redefinition based upon long survival times with Imetelstat
12/ Salesforce in training
=================================================================
***Vertex, CRISPR therapy for sickle cell passes FDA panel test
Expert advisers to the agency appeared generally comfortable with the testing Vertex has done to show the safety of the treatment, called exa-cel and now up for U.S. approval.
Published Oct. 31, 2023
Re: ADCOM
I hope Geron posts this fireside chat, so we can all listen and update.
===========================
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 participate in a fireside chat at the B. Riley Securities Virtual Oncology Conference on Thursday, January 18th, 2024, at 10:30am ET.
A webcast of the presentation will be available through the Investors and Media section of Geron’s website under Events following the presentation. The webcast will be archived and available for replay for a period of 30 days.
===========================
=================================================================Thank you hunter (YMB)
In case you didn’t catch the conference yesterday, it was actually kinda interesting as the analyst firm did their own survey with doctors active in the field that geron is seeking approval, these were my takeaways.
B Riley analyst did a survey with doctors and said 76% of them said the phase three results either met or exceeded their expectations with 12% saying they weren’t familiar with the data.
2/3 of the doctors sited moderate concerns with incidents of cytopenias and 1/3 said they were not concerned, chip stated that they are reversible within weeks and very few of the incidences resulted in needing to see a hospital. He also stated they have very extensive feed back from hematologists that they are used to managing cytopenias becuase agents used currently not infrequently caused prolonged mylosupression.
3/4 doctors said that despite the cytopenias they would prescribe Imet to more then 20% of their lower risk MDs patients which included patients not even tested in the trial which the analyst said would translate to 15% of the lower risk MDs population for the first year of sales
===================================Thank you RockyVll (YMB)
" Imetelstat was designed to reverse the immortality of cancer cells – but this new research shows that it also actively kills cancer cells through a unique process called ‘ferroptosis.’ It turns out that AML cells are particularly sensitive to ferroptosis, so the door is now open for researchers to develop highly-targeted new drugs that are designed to kill AML cells by ferroptosis."
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 participate in a fireside chat at the B. Riley Securities Virtual Oncology Conference on Thursday, January 18th, 2024, at 10:30am ET.
A webcast of the presentation will be available through the Investors and Media section of Geron’s website under Events following the presentation. The webcast will be archived and available for replay for a period of 30 days.
Last edited by LWS on Sun Jan 21, 2024 5:13 pm, edited 2 times in total.
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biopearl123
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Re: ADCOM
Yup just five more months but AML data any day.
Re: ADCOM
There is not much to do until the Oncology ADCOM (ODAC) speaks about Imetelstat in one format or another. We know that the EAP continues, and the NDA is under review. Also, John Scarlett has told us that this is his 'quiet-time'. Medically, as far as I can tell, everything looks very positive, with patients' needs the #1 concern.
===============================
Previous thoughts:
Imetelstat is looking more and more like a miracle blood cancer medicine. The MOA is just beginning to be understood with two mechanisms to kill the cancer stem cells, while not harming normal cells. Everyone is waiting for the NDA approval. Then there will be years of research for fine turning the science and treating many cancer types in combinations. Imetelstat is the 'tip of the iceberg'. --- from LWS (Nov. 15, 2023)
+++++++++++++++++++++++++++++++++
From Stifel fireside chat outline:
7/ Connection between transfusion independence, disease modification, and bone marrow reversals
8/ MOA; unprecedented results
++++++++++++++++++++++++++++++++
" Imetelstat was designed to reverse the immortality of cancer cells – but this new research shows that it also actively kills cancer cells through a unique process called ‘ferroptosis.’ It turns out that AML cells are particularly sensitive to ferroptosis, so the door is now open for researchers to develop highly-targeted new drugs that are designed to kill AML cells by ferroptosis." --- from Rocky (Jan. 19, 2024)
Re: ADCOM
ODAC has awakened (The meeting will be held on March 14, 2024, from 9:30 a.m. to 3 p.m. eastern time.)
This is the last hurdle to get over before NDA (Imetelstat) approval, that we have all been waiting for. This meeting, as I understand it, is open to the public. The KOLs, patients, and other professionals will all be heard.
This is the last hurdle to get over before NDA (Imetelstat) approval, that we have all been waiting for. This meeting, as I understand it, is open to the public. The KOLs, patients, and other professionals will all be heard.
=========================
Previous (Dec. 16, 2023)
JS said, that the ODAC may be changing the way it does business, based on the Vertex/CRISPR arrangement and outcome. That is, if the data and outcomes are sufficiently 'remarkable, compelling and durable' (like Imetelstat, for example), formal hearings can be waved with a direct and timely recommendation to the FDA.
He also talked about the unique mechanism of action (MOA) based upon two methods to kill cancer stem cells (1/ Ferroptosis, 2/ Telomerase & Telomeres) that is just beginning to be understood and put into proper perspective.
Perhaps, the most important observation that JS made concerned the connection between transfusion independence, disease modification, and bone marrow reversals --- that is these are not independent occurrences but are closely related to each other on a molecular level.
ODAC has all the data it needs now, with completed trials and new data being constantly supplied by the EAP.
Re: ADCOM
Imetelstat in focus and subject of Oncology ADCOM (ODAC) meeting on March 14, 2024
===================
SUPPLEMENTARY INFORMATION:
Agenda: The meeting presentations will be heard, viewed, captioned, and recorded
through an online teleconferencing and/or video conferencing platform. The Committee will
discuss new drug application (NDA) 217779 for imetelstat for injection, submitted by Geron
Corporation. The proposed indication for this product is for the treatment of transfusion-
dependent anemia in adult patients with low- to intermediate-1 risk myelodysplastic syndromes
who have failed to respond or have lost response to or are ineligible for erythropoiesis-
stimulating agents.
FDA intends to make background material available to the public no later than 2 business
days before the meeting. If FDA is unable to post the background material on its website prior to
the meeting, the background material will be made publicly available on FDA’s website at the
time of the advisory committee meeting. Background material and the link to the online
teleconference and/or video conference meeting will be available at
https://www.fda.gov/AdvisoryCommittees/ ... efault.htm.
Scroll down to the appropriate advisory committee meeting link. The meeting will include slide presentations with audio and video components to allow the presentation of materials in a manner that most closely resembles
an in-person advisory committee meeting.
===================
SUPPLEMENTARY INFORMATION:
Agenda: The meeting presentations will be heard, viewed, captioned, and recorded
through an online teleconferencing and/or video conferencing platform. The Committee will
discuss new drug application (NDA) 217779 for imetelstat for injection, submitted by Geron
Corporation. The proposed indication for this product is for the treatment of transfusion-
dependent anemia in adult patients with low- to intermediate-1 risk myelodysplastic syndromes
who have failed to respond or have lost response to or are ineligible for erythropoiesis-
stimulating agents.
FDA intends to make background material available to the public no later than 2 business
days before the meeting. If FDA is unable to post the background material on its website prior to
the meeting, the background material will be made publicly available on FDA’s website at the
time of the advisory committee meeting. Background material and the link to the online
teleconference and/or video conference meeting will be available at
https://www.fda.gov/AdvisoryCommittees/ ... efault.htm.
Scroll down to the appropriate advisory committee meeting link. The meeting will include slide presentations with audio and video components to allow the presentation of materials in a manner that most closely resembles
an in-person advisory committee meeting.
Re: ADCOM
ODAC & CDER ---- Webcast of March 14th ODAC (Imetelstat) meeting
===========================
Webcast Information
CDER plans to provide a free of charge, live webcast of the upcoming advisory committee meeting (ODAC, March 14). If there are instances where the webcast transmission is not successful, staff will work to re-establish the transmission as soon as possible. Further information regarding the webcast, including the web address for the webcast, will be made available no later than two (2) business days before the meeting in the Event Materials section of this web page.
CDER plans to post archived webcasts after the meeting, however, in cases where transmission was not successful, archived webcasts will not be available.
Contact Information
LaToya Bonner, PharmD, MBA
Center for Drug Evaluation and Research
Food and Drug Administration
10903 New Hampshire Avenue
WO31-2417
Silver Spring, MD 20993-0002
Phone: 301-796-2894
Email: ODAC@fda.hhs.gov
================================
FROM CDER :
NO. 10. Imetelstat (most important new drug of 2024)
▶ Company: Geron
▶ Indication: Lower risk myelodysplastic syndromes (MDS)
▶ Status: PDUFA date of 16 June 2024
+++++++++++++++++++++++++++++++++++++++++++++++++
===========================
Webcast Information
CDER plans to provide a free of charge, live webcast of the upcoming advisory committee meeting (ODAC, March 14). If there are instances where the webcast transmission is not successful, staff will work to re-establish the transmission as soon as possible. Further information regarding the webcast, including the web address for the webcast, will be made available no later than two (2) business days before the meeting in the Event Materials section of this web page.
CDER plans to post archived webcasts after the meeting, however, in cases where transmission was not successful, archived webcasts will not be available.
Contact Information
LaToya Bonner, PharmD, MBA
Center for Drug Evaluation and Research
Food and Drug Administration
10903 New Hampshire Avenue
WO31-2417
Silver Spring, MD 20993-0002
Phone: 301-796-2894
Email: ODAC@fda.hhs.gov
================================
FROM CDER :
NO. 10. Imetelstat (most important new drug of 2024)
▶ Company: Geron
▶ Indication: Lower risk myelodysplastic syndromes (MDS)
▶ Status: PDUFA date of 16 June 2024
+++++++++++++++++++++++++++++++++++++++++++++++++
The Center for Drug Evaluation and Research (CDER) is a division of the U.S. Food and Drug Administration (FDA) that regulates over-the-counter and prescription drugs, including biological therapeutics and generic drugs . CDER evaluates new drugs before they can be sold. CDER is the lead Center for regulation of human drugs that are regulated by FDA under the authority of the Federal Food Drug and Cosmetic (FD&C) Act. Biological products are covered by the Center for Biologics Evaluation and Research
Re: ADCOM
The FDA (CDER & ODAC) both are very familiar with Imetelstat (successes & potential).
With the EAP in place, and no significant medical negatives, there is nothing (that I am aware of) that would prevent NDA (Imetelstat) approval. Together CDER and ODAC are bringing us the March 14th webcast. CDER has done its homework and has already accepted Imetelstat as an important blood cancer medicine that meets the standards for approval.
========================
The Top 10 from CDER (2024):
1. KarXT
2. Donanemab
3. Resmetirom
4. Sotatercept
5. Datopotamab Deruxtecan
6. Acoramidis
7. mRNA-1345
8. Anktiva
9. Ensifentrine
10. Imetelstat
Imetelstat is an investigational telomerase inhibitor that binds to and inhibits the activity of telomerase, designed to block the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies such as myelodysplastic syndromes (MDS) and myelofibrosis (MF).
In August of this year, Geron Corporation announced that the FDA had accepted the New Drug Application (NDA) for Imetelstat for the treatment of transfusion-dependent anemia in low-risk MDS patients. Geron has requested FDA approval for priority review of the application. Additionally, the company expects to submit a Marketing Authorization Application (MAA) to the European Union (EU) in the fourth quarter of 2023.
With the EAP in place, and no significant medical negatives, there is nothing (that I am aware of) that would prevent NDA (Imetelstat) approval. Together CDER and ODAC are bringing us the March 14th webcast. CDER has done its homework and has already accepted Imetelstat as an important blood cancer medicine that meets the standards for approval.
========================
The Top 10 from CDER (2024):
1. KarXT
2. Donanemab
3. Resmetirom
4. Sotatercept
5. Datopotamab Deruxtecan
6. Acoramidis
7. mRNA-1345
8. Anktiva
9. Ensifentrine
10. Imetelstat
Imetelstat is an investigational telomerase inhibitor that binds to and inhibits the activity of telomerase, designed to block the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies such as myelodysplastic syndromes (MDS) and myelofibrosis (MF).
In August of this year, Geron Corporation announced that the FDA had accepted the New Drug Application (NDA) for Imetelstat for the treatment of transfusion-dependent anemia in low-risk MDS patients. Geron has requested FDA approval for priority review of the application. Additionally, the company expects to submit a Marketing Authorization Application (MAA) to the European Union (EU) in the fourth quarter of 2023.
=================================
FROM CDER :
NO. 10. Imetelstat (most anticipated & important new drugs of 2024)
▶ Company: Geron
▶ Indication: Lower risk myelodysplastic syndromes (MDS)
▶ Status: PDUFA date of 16 June 2024
+++++++++++++++++++++++++++++++++++++++++++++++++
The Center for Drug Evaluation and Research (CDER) is a division of the U.S. Food and Drug Administration (FDA) that regulates over-the-counter and prescription drugs, including biological therapeutics and generic drugs . CDER evaluates new drugs before they can be sold. CDER is the lead Center for regulation of human drugs that are regulated by FDA under the authority of the Federal Food Drug and Cosmetic (FD&C) Act. Biological products are covered by the Center for Biologics Evaluation and Research
Re: ADCOM
ODAC Composition
We know that ODAC (Oncology Data Advisory Committee to the FDA) will meet on March 14th, 2024 and the subject will be Imetelstat. This is their first meeting since Nov. 16, 2023. John Scarlett suggested that the current ODAC group is more flexible than prior groups (fireside chat). He also suggested that he plans to use the public statements (from KOLs, patients, etc.) as part of Geron's program to get Imetelstat recognized and popularized as a needed and unique blood cancer treatment (on several levels).
1/ Who serves on ODAC (names)?
2/ Are there permanent members?
3/ Who appoints new members?
4/ Can ODAC wave formal meetings with overwhelming positive data?
==========================
Contact Information
LaToya Bonner, PharmD, MBA
Center for Drug Evaluation and Research
Food and Drug Administration
10903 New Hampshire Avenue
WO31-2417
Silver Spring, MD 20993-0002
Phone: 301-796-2894
Email: ODAC@fda.hhs.gov
We know that ODAC (Oncology Data Advisory Committee to the FDA) will meet on March 14th, 2024 and the subject will be Imetelstat. This is their first meeting since Nov. 16, 2023. John Scarlett suggested that the current ODAC group is more flexible than prior groups (fireside chat). He also suggested that he plans to use the public statements (from KOLs, patients, etc.) as part of Geron's program to get Imetelstat recognized and popularized as a needed and unique blood cancer treatment (on several levels).
1/ Who serves on ODAC (names)?
2/ Are there permanent members?
3/ Who appoints new members?
4/ Can ODAC wave formal meetings with overwhelming positive data?
==========================
++++++++++++++++++++++++++Agenda: The meeting presentations will be heard, viewed, captioned, and recorded
through an online teleconferencing and/or video conferencing platform. The Committee will
discuss new drug application (NDA) 217779 for imetelstat for injection, submitted by Geron
Corporation.
Contact Information
LaToya Bonner, PharmD, MBA
Center for Drug Evaluation and Research
Food and Drug Administration
10903 New Hampshire Avenue
WO31-2417
Silver Spring, MD 20993-0002
Phone: 301-796-2894
Email: ODAC@fda.hhs.gov