There are many ways to think about partnering

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huntingonthebluffs
Posts: 206
Joined: Wed Feb 24, 2016 12:00 am

There are many ways to think about partnering

Post by huntingonthebluffs » Wed Dec 08, 2021 7:34 pm

This from the Geron Website (About Us tab drop down under Partnering)

Partnering

Corporate Development
Since our founding, Geron has focused on strong science and the pursuit of R&D excellence to discover and develop innovative, first-in-class therapies for the treatment of cancer as evidenced through the development of our first-in-class telomerase inhibitor, imetelstat, in hematologic myeloid malignancies.
We enjoy working with like-minded partners who want to join forces to transform the lives of patients with hematologic malignancies by delivering novel therapeutics such as imetelstat that change the course of their disease. We are interested in alliances, partnerships and collaborations that bring complementary capabilities and programs to realize the full potential of imetelstat development and commercialization on a global basis.
To contact our Corporate Development team, please send an e-mail to corpdev@geron.com.

Scientific Collaborators
Share our passion and vision. Academic research has an important role throughout the development of new medicines. Our collaborations with many scientific and clinical experts have led to key contributions to the scientific understanding of telomere and telomerase biology, and our telomerase inhibitor, imetelstat. We invite you to partner with us in our quest to develop this novel therapy and make a difference in patients’ lives.

Please send all inquiries to: info@geron.com.
xxxxxxxxxxx

While there are many pieces to the definitions for alliances, partnerships and collaborations, I chose the following for myself to obtain a better understanding of what Geron is after with partnerships.

Alliances: A close association formed to advance common interests. Often used interms of countries or politics but also an association of people, groups or companies working together for a specific purpose. Aligning or merging of efforts or interests and may involve a formal agreement.

Partnerships: A formal arrangement by two or more parties to manage and operate a business and share its profits. There are several types of partnership arrangements. In particular, in a partnership business, all partners share liabilities and profits equally, while in others, partners may have limited liability. There also is the so-called "silent partner," in which one party is not involved in the day-to-day operations of the business.

Collaborations: Collaboration is a working practice, especially a joint intellectual endeavor between scientists, working together for a common purpose to achieve business benefit. Collaborations consist of two or more entities motivated to solve problems and/or develop solutions, share information, consider alternatives, and engage in advancing knowledge, understanding, solutions, products, organizations, etc. Collaboration relies on openness and knowledge sharing but also some level of focus and accountability on the part of the business organizations.
xxxxxxxxxxxxxx

So with the above definitions in mind or whatever definitions one cares to use, we know Dr. John Scarlett and Geron are looking for partnerships to “bring complementary capabilities and programs to realize the full potential of Imetelstat development and commercialization on a global basis.” There has been a continuous flow of posts predicting big money partnerships and buyouts as the scope, effectiveness, potential of Geron’s company, personnel, drugs, patents, CT results, etc. continue to be quantified.

I personally do not believe this stated intention is to simply reflect a desire to partner with BP in a way that BP buys/controls a piece of the action with and beyond Imetelstat and owns the rights to a portion of the global commercialization. I do not believe JS/Geron are simply looking for another JNJ-like arrangement as their opportunity now is much closer, more certain, more complex and massive. I personally think Geron, if not already, will have dozens of partnerships as the realization of Imetelstat’s potential continues to seep out into the scientific communities within pharmaceuticals, independent labs, universities and hospital settings, ASH, EHA, MPN type information delivery entities, etc.

There are many levels on which “complementary capabilities and programs” are established. Alliances, partnerships and collaborations all reflect a form of partnership. Partnerships like the JNJ partnership reflects a single approach. JS/Geron are much more independent, functional, capable, established, organized, developed, knowledgeable, etc., etc. than in 2014 following the fiction based, likely sinister hold put on by the FDA. I do not think JS/Geron needs to or will simply do a refresh similar to the JNJ deal on some fraction of the global commercialization opportunity. I believe they are and have been thinking outside the box, for quite some time now, in an effort to quantify, maximize and optimize the utilization of partnership components, that will achieve company, patient, investor and future development results AND be represented as the controlling entity in the partnership.

I don’t think Dr. Scarlett is simply thinking he wants to get on with it and go out with a buyout or any other flavor of a feather in his hat. Personally, in all the years I have watched JS/Geron, I have never seen him exhibit a huge ego or a lack of patience, maybe I’m just blind. However, I think a buyout could and typically would have occurred months, even years ago. Now that Geron is very close to incredible success, JS will not move hastily or jeopardize that in any way by acting to quickly to simply cement his legacy beyond what he has already accomplished. Financing will not be a showstopper challenge going forward, it’s just a matter of finding the best way forward and a package partnership with upfront cash is just one way to do that. At this juncture, I personally don’t think a BO is on the table in JS’s mind, as he would lose control of accomplishing company, patient, investor and future development results. And in that vein, any partnership parameters will need to include major factors beyond just financials and paring down the global scope of Geron’s opportunity.

That said, I, along with the rest of us, greatly appreciate kmall’s efforts at quantifying Geron’s value, personnel, connections, relationships, etc. and estimated BO parameters. His efforts and skills are quite incredible and helps us all better realize what we have in Geron and Imetelstat. On the other hand, I personally, believe much of that value, once better quantified by the trading and investment communities, can/will be realized without actually doing a BO once commercialization has commenced and revenue rapidly ramps up. Wall Street will come around just as rapidly and that is not that far away in relation to how long many of us have been following and “invested” in this space.

As a side note, I also think that the “big boys” know how JS/Geron are thinking about big partnerships and BO’s which also has a tendency to keep the share price in check. So as a result, we are likely going to have to wait, as investors to receive our appreciation, just as long as the patients have to wait to receive the benefits of this amazing drug. All in all, that seems fair to me.

bucbeard
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Re: There are many ways to think about partnering

Post by bucbeard » Thu Dec 23, 2021 2:47 am

This is an all-star post. I've come back to it more than a few times, as I regularly ponder the future of this company and my investment. Thank you HOTB!

LWS
Posts: 142
Joined: Thu Jul 14, 2016 2:00 am

Re: There are many ways to think about partnering

Post by LWS » Fri Dec 24, 2021 7:10 pm

Alliances, Partnerships, Collaborations

These types of associations will have to be made to explore and research combinations.

Imetelstat's future strength will be its ability to enhance existing, approved cancer medicines (for example AbbVie's venetoclax for AML---a blood cancer) in all forms of cancer. To proceed, some single-agent approval for MDS or MF will be extremely helpful. Once approved, all sorts of off label uses will be possible. MD Anderson is already exploring some of these possibilities.
-------------------------------------------------------------------------------------------------
"Medications that are being investigated in Phase 3 randomized clinical trials at MD Anderson for possible approval include the following:

• Imetelstat (telomerase inhibitor) is a pioneering medication in treatment of MF because it appears to significantly prolong survival of MF patients who failed ruxolitinib. Currently, the possible survival benefit of imetelstat in MF patients is being evaluated in a pivotal Phase 3 trial (IMpactMF); survival benefit is an unprecedented primary endpoint in clinical trials for investigational MF medications."

LWS
Posts: 142
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Re: There are many ways to think about partnering

Post by LWS » Wed Dec 29, 2021 4:54 pm

The Geron plan seems to be to go it alone for MF and MDS to establish a line of credibility for Imetelstat, and to reinforce Dr. Rizo's "remarkable and compelling" description. The British now are the most likely to grant approval in a timely way, since they accept 'rolling-data'. Then there will be many combinations' partners seeking to enhance their approved (or in the pipeline) medicines. Imetelstat has a bright near-term as a single-agent, and a huge potential long-term in combinations (blood cancers & solid tumor cancers).

LWS
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Re: There are many ways to think about partnering

Post by LWS » Tue Jan 04, 2022 4:42 pm

MD Anderson now is evaluating Imetelstat for MF and MDS in phase 3, where I understand they have had good medical successes, and are pursuing advanced studies in other areas with this medicine. I would like to see additional information.

It seems to me that MD Anderson would not pursue Imetelstat, unless they were very confident about its usefulness and safety. MD Anderson has filled the research gap that Mayo Clinic once filled.

MD Anderson is a collaborator with worldwide recognition in all forms of cancer treatment and cancer research. Their reviews carry both respect and weight.

LWS
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Re: There are many ways to think about partnering

Post by LWS » Fri Jan 07, 2022 12:21 am

My Thoughts for 2022:

Imetelstat is a proven medicine by trials (phase 2s & ongoing phase 3s), science, safety and research. It is needed now on the front lines for MF and MDS and is needed in research for all sorts of combinations from blood cancers to solid cancers. Only some serious safety problem (none known), at this point in time, can stop Imetelstat medically. Calculating hedge funds, dilution, and management, one way or the other, can and have affected Geron's PPS ($1.22). Imetelstat will succeed, partners will develop, and patients will benefit.

huntingonthebluffs
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Re: There are many ways to think about partnering

Post by huntingonthebluffs » Sat Jan 08, 2022 9:19 pm

Thanks for the feedback bucbeard, always nice to hear from you! LWS I appreciated your various cuts at this thread’s thought process and your opinions on the way forward.

I have, as most here, spent time on understanding what the JNJ/Geron partnership offered and what was missing. Much of what went on in steering committee meetings and decision making process will likely never see the public light of day. There are many views and processes for establishing partnerships, etc. which one can easily find via Google searches. I think the major difference we can expect going forward is that Geron will be owning the process initiation and probably defining a considerable amount of the specifics. I personally never felt that Dr. Scarlett had that much say in the JNJ partnership specifics due to Geron’s weak negotiating position following the hold on the Mayo CT. To his credit, the upfront money, Janssen’s contributions and the opportunity to plan for a discontinuation by maintaining control of all the patents and return of all scientific materials, CT data and management were important accomplishments given the lame duck situation.

I think most, myself included, do not fully understand or appreciate what can and can not go into the development of a partnership. However, while there are many books and documents out there if one is interested. One fairly interesting (and reasonably short) cut at it that I found useful can be found at (Strategic Alliances Strategies and Processes Benchmarking Study):

https://www.nasa.gov/sites/default/file ... 0processes

While this is a 2009 document I think it still is quite useful on the framework of an effective strategy for establishing partnerships, alliances and collaborations. If anyone cares to read it, I would appreciate your feedback posts on the parts that you think could resonate regarding what Geron, has or will do regarding partnerships and why. Maybe one take away we all could appreciate more is the effort that must go into this process to make it worthwhile for everyone involved, including patients and investors. Also, you might find it interesting to see the names of the companies participating listed in an appendix.

Hoping everyone has a blessed and prosperous 2022, I think it will be one to remember!

huntingonthebluffs
Posts: 206
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Re: There are many ways to think about partnering

Post by huntingonthebluffs » Tue Jan 11, 2022 6:26 am

While it may not always sound like it, I do believe partnerships have great value and are coming. However, put me in the “be cautious” column regarding partnering. I actually hope the Ex-US partnerships come sooner and are even more significant in value than we expect but I think it is helpful to understand the pro’s and con’s. I also don’t think there is an immediate need to partner as Geron’s value grows with each day closer to commercial approval, as well as a stronger negotiating position regarding the terms of the partnership. While the right partner will have an initial drag on Geron’s internal workings as it rolls into place, it could also have potential to speed somethings up. However I believe the share price impact of new partnerships might be temporary until the CT data readouts and approval.

It would be naive to say at this juncture Dr. Scarlett and company do not know where they want to go regarding any partnership, so this is all likely mute discussion while we wait for Mr. Koval and others (including a gaggle of lawyers) to carry out their partnering missions. I doubt they have had anything like a blank check offered to them but I believe they have moved into the negotiating driver’s seat on most types of partnerships. Any company wanting to partner with Geron likely has an obvious desire to move into or expand into a restricted market littered with unmet needs. So it would also be naïve to say they don’t understand the level of interest in Imetelstat, as well as, the risks in partnering, especially regarding a strategic alliance.

Most of us who have been here for a while continue to average further into our Geron investment when the price dips as it has recently, knowing the short cadre are the primary cause versus changes in the data or potential or impending successes. Regardless the size, value and quality of the partnership(s), terms will have very little, if any, relationship to the current stock price. IMHO, when the time comes, any major Ex-US partnership terms will include a significant upfront financial transaction but likely be weighted towards things like opportunity scope, sharing of revenue and profit, leveraging complementary resources and capabilities, leveraging joint combination studies, avenues to grow more quickly and efficiently, channels of distribution, marketing and sales responsibilities, brand expansion, geographic expansion, cost reduction, manufacturing, and other supply-chain synergies, access to intellectual property and even employee talent, as well as countering competition, future product research / development and future expansion of the relationship.

Maybe it is just my cautious nature here, but I think an important risk to their partnership agreements is being able to avoid partners that have questionable principles like trust, honesty, fairness, open and frequent communications, commitment follow through and executive management support and involvement; which I continue to believe did not sufficiently exist with the JNJ partnership. Also, it matters if their corporate cultures are reasonably compatible and believe in and visibly support win-win partnerships. Again, I think it’s useful to reference the link in my previous post and review: 4.0 Key Findings, Critical Success Factors, and Best Practices. Risks and challenges are described in many documents, a few of these include:
• Partners may misrepresent what they bring to the table.
• Partners may unilaterally dissolve partnerships.
• Partners may fail to commit resources and capabilities to the other partners.
• Partners may have key employees with inconsistent strategies and objectives to other partners.
• Partners may not openly and frequently communicate to the other partners.
• Partners may fail or choose not to use their complementary resources effectively.
• Partners may mistakenly or even overtly fail to fairly define:
o The duties and responsibilities of each party
o Ability to maintain or shift strategies if needed
o Confidentiality and noncompetition
o Payment terms
o Scientific or technical milestones
o Ownership of intellectual property
o Remedies for breach; and
o Termination

Another very good source of information on strategic alliances can be found at:
https://www.management4all.org/2013/01/ ... ce_20.html

Lastly, if one only cares about partnerships because it will impact the stock price, maybe take a look at the price before and after an announcement of a significant partnership between a BP and a pre-commercial biotech and share your findings. If you look at GERN or LTCX, the price isn’t that much different or even lower after a partnership announcement. Basically, I just see a spike shortly before, during and shortly after the announcement, then it comes back down. If one is a buy and hold biotech investor, that phenomena is very frustrating as we know the short cadre is killing it again. IMHO, given a spike or two along the way, it seems likely to me that the only development that will move this stock persistently higher is CT data and commercial approval, followed by effective rollout and revenue/profit generation. Which is also where we will likely see a persistent value accruing as the result of having a quality partnership. We may be on the launch pad, but the ignition is likely a few months or even a year away unless Wallstreet miraculously comes to their senses. Although everyone knows partnerships with significant financial provisions certainly could come before public CT data or approval and will have a significant impact on the stock price, at least for a “while unless the CT data and/or approval, etc. occur concurrently or there abouts with partnership announcements, then Katie bar the door.

LWS
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Re: There are many ways to think about partnering

Post by LWS » Thu Jan 13, 2022 7:37 pm

The most important topic and only topic is Imetelstat, that can be broken down:

1. Effectiveness and safety as a single agent (phase 2 successes and ongoing phase 3)
2. Potential in combinations (blood cancers and solid tumor cancers)
3. Collaborators (MD Anderson)
4. Potential partners (Novartis, JNJ, BMY, AbbVie, etc.)
5. Approvals (England (rolling data), USA, European Union)

We all know that Geron's PPS (for whatever reason) is ridiculously low. We also know that there will be an EVENT (more than one), that will drive the PPS upward.

LWS
Posts: 142
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Re: There are many ways to think about partnering

Post by LWS » Sun Jan 16, 2022 1:08 am

Partners are the most important consideration now. Imetelstat has cleared all phase 2 hurdles, and the phase 3 finish line is clearly in sight. Imetelstat does what no other cancer medicine can do (disease modification, transfusion improvement, overall survival time records, combination potential).

The right partner (or partners) gives Imetelstat both the deep pockets and credibility that it needs to overcome the ability of some undefined entities to firmly control Geron's PPS at the low-end. With the UK's equivalent to the FDA (MHRA) now accepting 'rolling-data', Imetelstat may finally get the recognition that it has earned and deserves.

Patient's needs will be fully addressed, but the timeline is still uncertain.

LWS
Posts: 142
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Re: There are many ways to think about partnering

Post by LWS » Mon Jan 17, 2022 9:16 pm

Collaborator

MD Anderson has replaced Mayo Clinic as a research collaborator. Their reputation is world class.
-------------------------------------------------------------------------------------------
Srdan Verstovsek, MD, PhD, United Energy Resources, Inc. Professor of Medicine, director, Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms, chief, Section for Myeloproliferative Neoplasms, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, discusses the key objective of the phase 3 IMpactMF trial (NCT04576156) examining imetelstat in patients with myelofibrosis.

The trial is comparing the use of intravenous imetelstat vs best available therapy in the second-line setting, Verstovsek says. To be eligible for enrollment, patients must be nonresponsive or refractory to JAK inhibitors. The key objective of the study is to improve overall survival (OS) in this patient population, according to Verstovsek.

This trial marks the first time that investigators have had the ability to consider prolonging life for patients with myelofibrosis within the context of a clinical study, Verstovsek adds. Using OS as a primary end point has raised the bar for the field in that the focus has moved beyond the spleen or symptoms such as anemia, Verstovsek explains. Imetelstat is an exciting new approach in development for patients with myeloproliferative neoplasms, Verstovsek concludes.

From David (Yahoo Board) ---MD Anderson update

LWS
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Re: There are many ways to think about partnering

Post by LWS » Thu Jan 20, 2022 4:27 pm

More about MD Anderson and Imetelstat.

The MD Anderson/Imetelstat story continues. Their independent observations will be recognized by all (researchers, doctors, patients, investors).
------------------------------------------------
From Kmall

Also remember that Geron and Imetelstat are CURRENTLY involved in Pre-Clinical studies of Lymphoid Malignancies at MD Anderson Cancer Center in Houston, TX led by Dr. Swaminathan Iyer, Professor, Department of Lymphoma/Myeloma, Division of Cancer Medicine.

Lymphoid Malignancies encompass multiple indications affecting a 1,00,000+ patients worldwide.

Dr. Aleksandra Rizo was involved with multiple studies of Lymphoma while at Janssen. Targeting these indications commercially could yield Billions of $$ in additional Global annual revenue stream for Geron. Results of this Pre-Clinical study are expected by the end of this year.

See: "A Purple Squirrel & Venetoclax Combo - AML & Lymphoid Market Potential" on Imetelchat for a more detailed look at indications and market potential. -Kmall

Ryan
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Re: There are many ways to think about partnering

Post by Ryan » Thu Jan 20, 2022 8:24 pm

I'm not able to respond to KMall's message re: MD Anderson on Yahoo!, so adding this basic thought here:

Even more impactful than these 'petri dish' studies will be the Clinical trials with Imet in Combo, which will soon be initiated as Sponsored trials.
(Sponsors to trail are also Geron Partners)

LWS
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Re: There are many ways to think about partnering

Post by LWS » Sun Jan 23, 2022 7:21 pm

ILAP

This is a full explanation of ILAP (from Fusion Pharma Limited), which will be very important in forming new alliances and partnerships.
-----------------------------------------------
Today we are focusing on the Innovative Licensing and Access Pathway (ILAP).

What is the ILAP?

The ILAP is a new pathway supporting innovative approaches to the safe, timely and efficient development of medicines to improve patient access. It aims to accelerate the time to market, facilitating patient access to medicines. Horizon scanning and regulatory science aim to ensure the pathway is at the forefront of cutting-edge developments and has the framework to develop evidence-based practice as new technologies and methods emerge.

What types of medicines does it include?

new chemical entities
biological medicines
new indications
repurposed medicines

Who is the ILAP open to and what does it involve?

Available to both commercial and non-commercial developers of medicines (UK based and or global), the ILAP comprises of:

an Innovation Passport designation
a Target Development Profile (TDP)
access to a toolkit to support all stages of the design, development and approvals process for applicants
opportunities for enhanced regulatory and other stakeholder input

Which partners are involved in the ILAP?

Permanent partners:

The Medicines and Healthcare products Regulatory Agency (MHRA)
National Institute for Health and Care Excellence (NICE)
Scottish Medicines Consortium (SMC)

Supporting partners:

NHS England and NHS Improvement
Health Research Authority (HRA)
National Institute for Health Research (NIHR)

How does the Innovation Passport work?

The Innovation Passport application is the first step in the ILAP. It is the mandated entry point to the ILAP and is open to developers at the pre-clinical trial stage through to the mid-development programme point. The passport includes a broad and inclusive definition of innovation and both new and repurposed medicines are within scope. This designation is linked to a portfolio of activities through the creation of a product-specific Target Development Profile. The evidence required for a product to fulfil the criteria will depend on where in the development pathway the product is and can be based on non-clinical data. The Innovation Passport does not replace the Promising Innovative Medicine (PIM) Designation of the EAMS and you can apply for both initiatives. You must submit an Innovation Passport application for each separate medicinal product (different active substances). However, a single Innovation Passport can cover multiple indications for the same medicine (active substance).

What are the criteria for the passport?

There are 3 categories which make up the criteria for the Innovation Passport:

Criteria 1: Details of the condition, patient or public health area:
the condition is life-threatening or seriously debilitating
there is a significant patient or public health need

Criteria 2: The medicinal product fulfils one or more of a specific area:
a) innovation – such as an advanced therapy medicinal product (ATMP) or new chemical or biological entity or novel drug device combination
b) clinically significant new indication for an approved medicine
c) rare disease and/or other special populations such as neonates and children, elderly and pregnant women
d) development aligning with the objectives for UK public health priorities such as the Chief Medical Officer, Department of Health and Social Care (DHSC) or Life Sciences Sector Deal (including those in Devolved Administrations, where appropriate)

Criteria 3: the medicinal product has the potential to offer benefits to patients
You must provide a summary of how patients are likely to benefit from the product or indication coming to market.

Link to Innovation Passport application form.

When can you enter the pathway?

The ILAP enables multiple entry points depending on:

the stage of development of the product
the data available
the ambition of the applicant to engage with UK stakeholders
the applicant’s appetite for new innovative ways of working
Therefore, the pathway will allow entry very early, based on non-clinical data, where all the tools described below might be options, as well as catering for products with mid-development ‘global’ dossiers. However, to maximise the benefits, applicants are encouraged to apply early in the development of their products. Products that are towards the end of their development programme are generally not suitable for the ILAP unless there are one or more indications still under active investigation.
The ILAP does not replace the Early Access to Medicines Scheme (EAMS), which remains an important flexibility for earlier patient access towards the end of the development programme in areas of unmet medical need and where major advantage over existing therapies can be demonstrated. The ILAP is broader in scope and is open to all innovative products.

What is the Target Development Profile (TDP)?

A product-specific team of experts will help define the target development profile (TDP) based on your product’s characteristics. The TDP will:

define key regulatory and development features
identify potential pitfalls
create a road map for delivering early patient access

What will the TDP include?

It is anticipated that the TDP will be a living document, updated along the development programme timelines and milestones as new knowledge is generated.
The TDP will include details about how to work with other UK stakeholders for coordinated and efficient evidence generation and evaluation and address commercial and managed access considerations.

Need more information?

Contact innovationpassport@mhra.gov.uk and TDP@mhra.gov.uk

To receive your Complimentary BREXIT LICENCE REVIEW from Fusion Pharma Limited:
Contact us at hello@fusion-pharma-limited.com or submit the form on our Brexit page:
https://fusion-pharma-limited.com/brexit-ready/

biopearl123
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Re: There are many ways to think about partnering

Post by biopearl123 » Sun Jan 23, 2022 9:45 pm

Hi LWS, I and other board members have reviewed this. Thank you for posting it. Even with all of this it is still murky, at least to me, as to whether one must apply separately for an MAA.

kmall
Posts: 297
Joined: Thu Mar 21, 2019 3:57 pm

Re: There are many ways to think about partnering

Post by kmall » Sun Jan 23, 2022 10:25 pm

I emailed the MHRA earlier today, to hopefully get some clarity on the situation and specifically with Geron. I'm doubtful I get a direct response concerning Geron's status, but I will post any reply here as soon as I get one.


1:17 PM (2 hours ago)
to newscentre@mhra.gov.uk

Hello,

I am a former patient caregiver of someone with multiple sarcomas/hematological disorders. As a result, I became aware of Imetelstat and I’m currently an investor in Geron, a publicly traded company on the NYSE. On 10/25/2021 a Press Release from the company informed investors of your “Innovation Passport Designation Awarded to Imetelstat for the Treatment of Lower Risk Myelodysplastic Syndromes.” Within the PR mentions: “Key benefits of being within ILAP include a 150-day accelerated assessment and rolling review of a Marketing Authorization Application (MAA).”

My question to you is; has the MAA officially begun with this PR announcement – which would include the 150-day accelerated assessment, or does Geron still need to formally submit an application at a later date to generate the 150-day accelerated assessment? Thank you for any clarification you are able to assist me with on this subject.

Kind Regards,
Kmall


*I did get an automated reply 1min later:


News Centre
1:18 PM (2 hours ago)
to me

Thank you for emailing the MHRA News Centre. Please note this inbox is monitored between 08:30-17:00 Monday-Friday.

Therefore if you are emailing outside of these working hours or during the weekend and you have an urgent media enquiry, please call our out-of-hours News Centre number on +447770 446 189.

Kind regards**


**Funny that they used the same closing "Kind Regards" - perhaps I was channeling my inner Englishman? (spent 2yrs there in my youth in grade school) :) -Kmall

kmall
Posts: 297
Joined: Thu Mar 21, 2019 3:57 pm

Re: There are many ways to think about partnering

Post by kmall » Sun Jan 23, 2022 11:06 pm

From the 11/9/2021 Quarterly:

Pg 20:

In October 2021, we completed enrollment for IMerge Phase 3. Based on current planning assumptions, we expect top-line results from this trial to be available at the beginning of January 2023. Assuming the results of IMerge Phase 3 support regulatory submissions, we plan to submit a New Drug Application, or NDA, in the United States in the first half of 2023, and a marketing authorization application, or MAA, in Europe in the second half of 2023, for imetelstat in lower risk MDS. Under a either priority or standard review for the NDA and, upon potential approval by the United States Food and Drug Administration, or the FDA, we expect that commercial launch of imetelstat in lower risk MDS in the United States could occur in the first half of 2024. In Europe, we anticipate review of the MAA by the European Medicines Agency, or EMA, could take approximately 12 months and that commercial launch of imetelstat in lower risk MDS in Europe could occur in the second half of 2024.

If imetelstat is approved for marketing by regulatory authorities, we plan to commercialize imetelstat independently in the United States and may seek potential commercialization partners for territories outside of the United States.

In October 2021, we gained access to the Innovative Licensing and Access Pathway, or ILAP, through the receipt of an Innovation Passport for imetelstat to treat lower risk MDS. The ILAP is a new program sponsored by the Medicines and Healthcare products Regulatory Agency, or MHRA, in the United Kingdom, or the U.K., post-Brexit. The objective of this new licensing and access pathway is to reduce the time to market and enable earlier patient access for innovative medicines. The Innovation Passport is the first prescribed entry point in the ILAP process. Key benefits of being within ILAP include a 150-day accelerated assessment and rolling review of an MAA, as well as opportunities for frequent interactions with the review staff at the MHRA and its partner agencies to discuss imetelstat’s development, regulatory and reimbursement plans.



Pg 44 - under: RISKS RELATED TO REGULATORY COMPLIANCE MATTERS AND COMMERCIALIZATION OF IMETELSTAT
Our inability to maintain regulatory clearances and approvals to continue the clinical development of, and to potentially commercialize, imetelstat, would severely and adversely affect imetelstat’s future value, and our business and business prospects, and might cause us to cease operations.*

Pg 48 -

The Innovation Passport designation from the United Kingdom regulatory authorities does not guarantee marketing approval and may not lead to a faster development, regulatory review or approval process. *

In October 2021, we gained access to the Innovative Licensing and Access Pathway, or ILAP, through the receipt of an Innovation Passport for imetelstat to treat lower risk MDS. The ILAP is a new program sponsored by the Medicines and Healthcare products Regulatory Agency, or MHRA, in the United Kingdom, post-Brexit. The objective of this new licensing and access pathway is to reduce the time to market and enable earlier patient access for innovative medicines. The Innovation Passport is the first prescribed entry point in the ILAP process. Key benefits of being within ILAP include a potential 150-day accelerated assessment and rolling review of a Marketing Authorization Application, or MAA, as well as opportunities for frequent interactions with the review staff at the MHRA and its partner agencies to discuss imetelstat’s development, regulatory and reimbursement plans.

*****(Disclaimer - not on Pg 20)*****

Although the goal of ILAP and the Innovation Passport is to reduce the time to market and enable earlier patient access, it does not accelerate conduct of clinical trials or mean that the regulatory requirements are less stringent, nor does it ensure that any imetelstat MAA will be approved or that any approval will be granted within any particular timeframe. Despite receiving Innovation Passport designation, we may decide to delay or forego the commercialization of imetelstat in the U.K.

https://d18rn0p25nwr6d.cloudfront.net/C ... bac689.pdf

The wording, just as in the 10/25/2021 is somewhat murky. They are specific in a time-frame for NDA with the FDA and MAA with the EMA, but no clarity on UK MAA submission as far as a time-frame is concerned, or if the ILAP acceptance automatically triggers the 150-day accelerated assessment. -Kmall

LWS
Posts: 142
Joined: Thu Jul 14, 2016 2:00 am

Re: There are many ways to think about partnering

Post by LWS » Tue Jan 25, 2022 12:42 am

Directly from Geron (ILAP & MAA)

For what it is worth--Why would Geron not apply for MAA (Marketing Authorization Application)?

"We are pleased imetelstat met the three qualifying criteria for the Innovation Passport----", said Sharon McBain, Geron’s Vice President, Global Regulatory Affairs.

It seems to me that Imetelstat meets every criteria (and then some) --- safe + "remarkable & compelling"-- and is the perfect medicine for this accelerated process. I do not know if the (ILAP & MAA) automatically go together, and as someone said--"This is Geron"-- so everything has to be double-checked. I understand that some are trying to get confirming information and will post here.
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Repeating the 3 criteria:

Criteria 1: Details of the condition, patient or public health area:
the condition is life-threatening or seriously debilitating
there is a significant patient or public health need

Criteria 2: The medicinal product fulfils one or more of a specific area:
a) innovation – such as an advanced therapy medicinal product (ATMP) or new chemical or biological entity or novel drug device combination
b) clinically significant new indication for an approved medicine
c) rare disease and/or other special populations such as neonates and children, elderly and pregnant women
d) development aligning with the objectives for UK public health priorities such as the Chief Medical Officer, Department of Health and Social Care (DHSC) or Life Sciences Sector Deal (including those in Devolved Administrations, where appropriate)

Criteria 3: the medicinal product has the potential to offer benefits to patients
You must provide a summary of how patients are likely to benefit from the product or indication coming to market.
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FOSTER CITY, Calif., October 25, 2021--(BUSINESS WIRE)--Geron Corporation (Nasdaq: GERN), a late-stage biopharmaceutical company focused on the development and commercialization of treatments for hematologic malignancies, today announced that imetelstat, the Company’s first in class telomerase inhibitor, has been granted an Innovation Passport, which is the first prescribed entry point to the Innovative Licensing and Access Pathway (ILAP) launched in the United Kingdom (UK) by the Medicines and Healthcare products Regulatory Agency (MHRA) in January 2021, post-Brexit. The objective of this new licensing and access pathway is to reduce the time to market for innovative medicines. Key benefits of being within ILAP include a 150-day accelerated assessment and rolling review of a Marketing Authorization Application (MAA), as well as opportunities for frequent interactions with the review staff at the MHRA and its partner agencies, including the National Institute for Health and Care Excellence (NICE), to discuss imetelstat’s development, regulatory plans and reimbursement plans.

"We are pleased to participate in this new expedited review pathway established by MHRA and look forward to working with them and their partner agencies as we collaborate to bring imetelstat to UK patients," said Sharon McBain, Geron’s Vice President, Global Regulatory Affairs. "We are pleased imetelstat met the three qualifying criteria for the Innovation Passport and believe that because of the durable transfusion independence across different patient subgroups and strong evidence of disease-modifying activity observed in our IMerge Phase 2 study, imetelstat’s novel telomerase inhibitor approach has the potential to be an important drug in this patient population."

LWS
Posts: 142
Joined: Thu Jul 14, 2016 2:00 am

Re: There are many ways to think about partnering

Post by LWS » Mon Jan 31, 2022 7:40 pm

Geron/Imetelstat has always been a speculation with Nobel Prize science, a very complete medical history and numerous trials (successful, ongoing and being formed). I believe this all ends well for patients and patience investors. The time of the next important event is unknown (except perhaps to JS and Dr. Rizo), but it is likely to involve UK approvals (ILAP & MAA). This extends from Mayo Clinic to MD Anderson.

LWS
Posts: 142
Joined: Thu Jul 14, 2016 2:00 am

Re: There are many ways to think about partnering

Post by LWS » Thu Feb 03, 2022 7:01 pm

Cancer Moonshot and Partners

The revival of Biden's Cancer Moonshot (from the time that he was VP) is perfect timing for Imetelstat----- new approvals, successful & new trials, and new partners. COVID research (mRNA, protein snippets, etc.) has been phenomenally successful, but has taken the spotlight off of cancer research. Imetelstat, with UK approvals likely, is in the right place at the right time, especially with ongoing rolling data (from phase 3s). Going forward combinations research will be in focus.

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