Q2 2021 Geron Corp Earnings Call
Ladies and gentlemen, this is the operator today's conference call will begin in two minutes until that time your lines will again be placed on hold.
Operator: and gentlemen, this is the operator. So this conference call will begin in two minutes. Until that time, your lines will again be placed on hold. Thank you for your patience. Again, ladies and gentlemen, Ibes is the operator. Today's conference call will begin in two minutes. Until that time, your line will again be placed on hold. Thank you for your patience.
For your patience.
Again, ladies and gentlemen, basically operator today's conference call will begin in two minutes until that time your lines will again be placed on hold thank you for your patience.
unknown: and the, and so on. Thank you.
Operator: Ladies and gentlemen, thank you for standing by, and welcome to the Q2, 2021-Juron earnings conference call. At this time, all participants are in a lesson-only mode.
[music].
Ladies and gentlemen, thank you for standing by and welcome to the key to this conference one you want and you're an earnings conference call. At this time all participants are in a listen only mode. Later, we will conduct a question and answer session and instructions will follow at that time.
Operator: Later, we will conduct a question-and-answer session, and instructions will follow like that. If I want to show your core assistance during the conference, please press star zero on your touchdown telephone. As a reminder, this conference call is being recorded. I would like to turn the conference over to your host, Ms. Olivia Bloom. Please do so. All right, thank you very much, Grace. And good afternoon, everyone.
And how much will get quite assistance during the conference. Please press star zero on your Touchtone telephone as a reminder, this conference call is being recorded.
I would now like to turn the conference over to your House Smith Alesia Blue. Please go ahead.
Alright, Thank you very much Grace and good afternoon, everyone. Welcome to this conference call to discuss updates on our ongoing <unk> phase III clinical trials as well as second quarter financial results.
Olivia Kyusuk Bloom: Welcome to this conference call to discuss updates on our ongoing Emmettelstat Phase 3 clinical trials, as well as second quarter financial results. I am joined today by Dr. John Scarlett, Geron's chairman and chief executive officer, Dr. Alexander Rizzo, Geron's Executive Vice President and Chief Medical Officer, and Anil Kapoor, Geron's Executive Vice President of Corporate Strategy and Chief Commercial Officer. After the market closed today, we announced updates on our Emerged Phase 3 clinical trial and financial results for our second quarter via press release, which is available on our website.
I am joined today by Dr. John Scarlett Geron, Chairman and Chief Executive Officer, Dr. Alexandra Rizzo Jones, Executive Vice President and Chief Medical Officer, and a New Yorker, poor Geron executive Vice President of corporate strategy and Chief commercial officer.
After the market close today, we announced updates on our emerge phase III clinical trial and financial results for our second quarter via press release, which is available on our website.
Olivia Kyusuk Bloom: In addition, an archive of this webcast will be available on our website for 30 days, including the slides being presented today. Before we begin, please note that during the course of this presentation and question-and-answer session, we will make forward-looking statements regarding future events, performance, plans, expectations, and other projections, including those relating to the therapeutic potential of and potential regulatory approval for Imitel-Stat, anticipated clinical and commercial events and related timelines, the sufficiency of Geron's financial resources, and other statements that are not historical facts.
In addition, an archive of this webcast will be available on our website for 30 days, including the slides being presented today.
Before we begin please note that during the course of this presentation and question and answer session. We will make forward looking statements regarding future events performance plans expectations and other projections, including those relating to the therapeutic potential of and potential regulatory approval of mattel's that anticipated clinical and.
Events and related timelines, the sufficiency of Gerrans financial resources and other statements that are not historical fact.
Olivia Kyusuk Bloom: Actual events or results could differ materially. We refer you to the discussions under the heading risk factors in Jerome's quarterly report on Form 10Q for the quarter ended June 30, 2021, which contains and identifies important factors that could cause actual results to differ materially from those contained in the forward-looking statement. We undertake no duty or obligation to update our forward-looking statement.
Actual events or results could differ materially we refer you to the discussions under the heading risk factors in <unk> quarterly report on Form 10-Q for the quarter ended June 32021, which contains identifies important factors that could cause actual results to differ materially from those contained in the forward looking statements.
We undertake no duty or obligation to update our forward looking statements and now I will turn the call over to Dr. Scarlett chip.
John A. Scarlett: And now, I will turn the call over to Dr. Scarlett. Chip? Thanks, Olivia, and good afternoon.
John A. Scarlett: Thanks, Olivia, and good afternoon, everyone. I'm very happy to say we've made a lot of progress since last quarter, particularly with regard to our ongoing phase three trial and lower risk MDF. First, in that trial, we've reached 91% of the planned enrollment, and currently expect to complete enrollment in the fourth quarter of this year. In addition, we've accelerated the timing of top-line results in Emerge Phase 3 by three months. We did this by shortening the follow-up period after the last patient has been enrolled from 15 months to 12 months for the primary analysis.
Thanks, Olivia and good afternoon, everyone.
I'm very happy to say.
We've made a lot of progress since last quarter, particularly with regard to our ongoing emerge phase III trial in lower risk Mds.
First in that trial, we've reached 91% of the planned enrollment.
Currently expect to complete enrollment in the fourth quarter of this year.
In addition, we've accelerated the timing of topline result in emerge phase III by three months. We've done this by shortening the follow up period. After the last patient has been enrolled from 15 months to 12 months for the primary analysis.
John A. Scarlett: As a result, we now expect top-line results in the first quarter of 2023. And finally, we expect that our current and projected financial resources will be sufficient to get us to those top-line results. Olivia will expand on this in her section of the call today, and Alexander, our chief medical officer, will comment on the progress we've made in both of our ongoing phase three trials. And finally, Anil, our Executive Vice President of Corporate Strategy and Chief Commercial Officer, will discuss the market opportunity for lower risk MDI. So let's get started.
As a result, we now expect top line results in the first quarter of 2023.
And finally, we expect that our current and projected financial resources.
Will be sufficient to get us to those topline result.
Olivia will expand on this in her section of the call today.
Alexander our Chief Medical Officer will comment on the progress we've made in both of our ongoing phase three trials and finally, Aneel, our executive Vice President of corporate strategy, and Chief Commercial officer, who will discuss the market opportunity in lower risk Mds.
Let's get started.
Olivia Kyusuk Bloom: Thanks, Chip. The increase in operating expenses for the second quarter and year-to-date periods of 2021 compared to the same periods in 2020 was primarily driven by higher development expenses. This increase in R&D expenses includes higher clinical development costs associated with our two ongoing phase three clinical trials, higher personnel-related costs for additional head count, as well as the conduct of long lead time, manufacturing, and quality activities, such as manufacturing validation batches of Emmettlestat.
Thanks Chip the increase in operating expenses for the second quarter and year to date period of 2021 compared to the same period in 2020 was primarily driven by higher development expenses.
This increase in R&D expenses include higher clinical development cost associated with our two ongoing phase III clinical trial and higher personnel related costs for additional headcount as well as the conduct of long lead time manufacturing and quality activities, such as manufacturing validation batches of Intelsat.
The increase in general and administrative expenses for the second quarter and year to date period of 2021 compared to the same periods in 2020, primarily reflects new costs in connection with pre commercial activities, including modernizing the internal infrastructure to support a commercial launch and higher legal.
Olivia Kyusuk Bloom: The increase in general and administrative expenses for the second quarter and year-to-date periods of 2021, compared to the same periods in 2020, primarily reflects new costs in connection with pre-commercial activities, including modernizing the internal infrastructure to support a commercial launch and higher legal costs. Previously, we provided guidance that our financial resources were sufficient to fund our operations through the end of 2022. As of June 30, 2021, we had $239.1 million in cash, cash equivalence, and marketable security.
Half.
Previously we provided guidance that our financial resources were sufficient to fund our operations through the end of 2022.
As of June 32021, we had $239.1 million in cash cash equivalents and marketable securities.
Olivia Kyusuk Bloom: We now project these financial resources, combined with expected future non-dilutive funding from the second tranche under our current debt facility, will fund our operations through the end of the first quarter of 2023, by which time top-line results from Emerge Phase 3 are expected. With that, I will now turn the call over to Alexandra, who will review the progress in our Phase 3 clinical trials. Thank you, Olivia, and good afternoon, everyone.
We now project these financial resources combined with expected future non dilutive funding from the second tranche under our current debt facility will fund our operations through the end of the first quarter of 2023 by which time topline result from emerge phase III are expected.
With that I will now turn the call over to Alexandra who will review the progress in our phase III clinical trial Alexandra.
Thank you Joey and good afternoon, everyone.
Unknown Attendee: Let me start with two important updates related to our Emerge Phase 3 clinical trial, for which I'm so pleased that we are nearing completion of enrollment. As Chip commented in his introduction, we have achieved 91% of the planned enrollment as of last week, and we now expect to complete enrollment in the fourth quarter of 2021. Moving on to another important update about this study, we have determined that the clinical cut-off date for the primary analysis could occur three months earlier by shortening the follow-up period from 15 months to 12 months after the last patient has been enrolled.
Let me start to be two important updates related to our re emerge phase III clinical trial for which I'm. So pleased that we are nearing completion of enrollment.
As chip commented in his introduction, we have achieved 91% of the planned enrollment as of last week.
And we now expect to complete enrollment in the fourth quarter of 2021.
Yeah.
Moving onto another important update about this study.
We have determined that the clinical kind of date for the primary analysis Kudoka three months earlier by shortening the follow up period from 15 months to 12 months. After the last patient has been enrolled.
Unknown Attendee: This change is due to the significantly longer enrollment period caused by the COVID-19 pandemic, which has allowed enrolled patients in the study to have longer follow-up than initially planned. By shortening the follow-up period, we estimate that the overall median follow-up in the phase three trial will be similar to the overall median follow-up in the phase two trial, which, as you might remember, was 24 months. Thus, even with the shorter follow-up, we continue to expect to have a mature data set that will allow us to assess the safety and efficacy of Imetelstat, including durability of transfusion independence.
This change is due to the significantly longer enrollment period caused by the COVID-19, pandemic, which has allowed enrolled patients under study to have longer follow up than initially planned.
In shortening the follow up period, we estimate that the overall median follow up in the phase III trial will be similar to the overall median for a lot in the phase two trial, which as you might remember was 24 months.
Thus, even with the shores are full up we continue to expect to have mature dataset that will allow us to assess the safety and efficacy from intelsat, including durability of transfusion independence.
Furthermore, since we've already enrolled more than 90% of the patients. We believe the impact of this change on our efficacy results will be minimal.
Unknown Attendee: Furthermore, since we've already enrolled more than 90% of the patients, we believe the impact of this change on our efficacy results will be minimal, if at all. Accordingly, we submitted a protocol amendment to the FDA and have not received any comments on the proposed change.
At all.
Accordingly, we submitted a protocol amendment to the FDA and have not received any comments on the proposed change.
Unknown Attendee: We plan to distribute the final protocol amendment to all clinical sites. With a revised 12-month follow-up period for the primary analysis, we now project that the Top Plan results for Emerge Phase 3 will be available in the first quarter of 2020. Also, in July, a regularly scheduled meeting of the Independent Data Monitoring Committee for this study occurred, and the committee recommended the trial continued without modification. In conclusion, we are making good progress with this study, and I look forward to our announcement when we achieve full enrollment of the trial.
We plan to distribute to final protocol amendment, all clinical sites shortly.
With our revised 12 months for what's been good for the primary analysis. We now project the topline results for emerge phase III will be available in the first quarter of 2023.
Oh, so in July our regularly scheduled meeting of the independent data monitoring Committee for this study occurred.
And the committee recommended the trial continue without modification.
In conclusion, we are making good progress with this study and I look forward to our announcements when we achieve full enrolment of the trial.
Unknown Attendee: Turning to Impact MS, our second ongoing phase three trial, the focus for this trial has been on opening sites, which, as you know, is one of the key factors for patient enrollment as a flash tweet. 55 sites were open for enrollment.
Turning to impact.
Our second ongoing phase III trial.
The focus for this trial has been on opening sites, which as you know is one of the key factors for patient enrollment.
As a fast week 55 sites are open for enrollment.
Anil Kapur: We plan to engage over 180 stakeholders in 30 countries across North, South America, Europe, Australia, and Asia. We continue to expect the interim analysis to occur in 2024 and the final analysis in 2025. Just as a reminder, the number of events required to conduct the interim analysis for this study could occur before enrollment is complete, as these events will accrue throughout the enrollment process. With that, I'll turn the call over to Anil. Anil?
We plan to engage over 180 sites in 30 countries across North and South America, Europe, Australia and Asia.
We continue to expect the interim analysis to occur in 2024, and the final analysis in 2025.
Just as a reminder, the number of events required to conduct the interim analysis for the study could occur before the enrollment is complete as these events will occur throughout the enrollment period.
With that I'll turn the call over to Aneel.
Aneel.
Thanks, Alex and good afternoon, everyone.
Anil Kapur: Thanks Alex and good afternoon, everyone. In my prepared remarks today, I'll provide our perspective regarding two topics, unmet needs and the market dynamics in low-risk MDS that we believe make for exciting commercial potential for imital. Lower risk MDS, as many of you know, represents approximately 70% of the total MDS patient population. This is an attractive market with a large, addressable patient population. There is a significant unmet need for new therapeutics in this setting, as patients are typically elderly and present with chronic anemia, are dependent on frequent red blood cell transfusions, have poor quality of life, heightened risk of transformation to acute myeloid leukemia, and shortened survival
In my prepared remarks today.
I'll provide our perspective regarding two topics.
Unmet needs and the market dynamics in low risk Mds that we believe may put an exciting commercial potential for them at goldstrike.
Lower risk Mds as many of you know.
Good afternoon.
It might be 70% of the total Mds patient population.
This is an attractive market with a large addressable patient opportunity.
It is a significant unmet need.
Our new 10, a few takes in this setting.
<unk> patients are typically annually.
With chronic anemia.
Brendan on frequent red blood cell transfusions have poor quality of life.
Clinical transformation to acute myeloid leukemia and sharpen somebody.
If you look here, we have a schematic of the low risk Mds landscape.
Anil Kapur: If you look at this schematic of the low-risk MDS landscape, you will see that erythropoietin stimulating agents or ESAs are the mainstay of treatment for approximately 90% of patients who have symptomatic anemia and do not have deletion 5-Q. Not all patients respond to or are eligible for ESAs, and among responders, responses typically last for between 18 and 24 months. Treatment options are limited for patients who have failed ESAs or are ineligible for ESAs and may include hypomethalating agents or HMAs. As of recently, this part is set. I would also quickly note that HMAs are not a preferred option given their limited benefits. Also, they are not broadly approved across the EU for this indicator.
You will see that it took voices stimulating agents at ESC.
The main steel treatment for the approximately 90% of patients who have symptomatic anemia and do not have been you shouldnt by SKU.
Not all patients respond to all of our eligible for the USAID and among responders responses typically last for between 18 and 24 months.
Treatment options are limited for patients who have failed Esa.
All are ineligible for Esa and May include HEICO manipulating agents thought hma's.
As of recently missed by the SEC.
Wanted to also quickly note that.
Chimneys agnostic preferred option given their limited benefits.
So they are not broadly approved across the EU for this indication.
Despite the SEC was recently approved in 'twenty 'twenty, four Esa Finn ring <unk> positive patients.
Anil Kapur: Lospatosep was recently approved in 2020 for ESA-Failed, Rined Sideroblast positive patients. This RS positive segment covers only approximately 25% of patients, leaving a significant unmet need for effective therapies for the remaining approximately 75% of lower risk MDS, Qar Ares. The expected broad Imitalstadt opportunities is shown by the orange dotted line on this, Moving on to Immitelstarts expected target product profile, in our recent market surveys, community and academic hematologist, reaffirmed the unmet needs in lower risk MDS and highlighted how the strengths of Imital start can address those, Key aspects of a metal stats profile that resonated most strongly with these hematologists are shown on this slide, in particular, I would like to point out that the hematologist appreciated, having the ability to treat RS negative patients where there is no approved treatment.
This is part of the niche segment covers only approximately 25% locations, leaving a significant unmet need for effective therapies for the remaining approximately 75% of lower risk Mds patients who are Rs negative.
They expected broad emitter start opportunities as shown by the Orange dotted line on this slide.
Moving on to Amazon starts expected target product profile.
In a recent market surveys community and academic Hematologist.
The unmet needs in lower risk Mds and highlighted.
How the strengths okay metal start can address those needs.
Key aspects, okay, Michael starts profiles.
Donated most strongly with these hematologist are shown on this slide.
In particular.
I would like to point out that the Hematologist appreciated.
Having the ability to treat August negative patients, where there is no approved treatment.
In addition, they pointed to the durability of transfusion independence.
Anil Kapur: In addition, they pointed to the durability of transfusion independence. The 24-week and the one-year red blood cell Transcution Independence data from the I-Murge Phase 2 trial resonated particularly well with physicians as they felt these outcomes to be more clinically relevant than 8-week Transfusion Independence. In Europe, both regulators and pairs have requested to see 24-week TI data as, In addition, the potential for disease modification was also very well. We expect to achieve broad label coverage for Emetal Stat in ESA relapse-refractory, non-deletion 5Q, low-risk MDS, that includes not only both RS positive and RS negative patients but also first-line patients who are ESA ineligible.
24 weeks and the one year Red blood cell transfusion independents data from the eye emerge phase II trials.
Resonated, particularly well with physicians as they've tried these outcomes to be more clinically relevant than eight week transfusion independence.
In Europe, both regulators and payers have requested to see 24 week data as well.
In addition, the potential for disease modification was also very well received.
We expect to achieve broad label coverage for <unk> in Esa relapsed refractory non Venetian five SKU Nordisk MBS.
That includes not only both Rs positive and <unk> negative patients, but also first line patients who are Esa ineligible.
Anil Kapur: This sets up ImitalStat well for reimbursement across the broader population. Therefore, we expect a highly differentiated position for a metal start at launch, as well as the ability to significantly penetrate this attractive market, eventually becoming part of the standard of care in lower risk MD. This next slide describes the potential market segmentation in more depth.
This sets up.
Well for reimbursement across the broader population.
Therefore.
We expect a highly differentiated position polymer it'll start at launch.
As well as the EBITDA.
<unk> to significantly penetrate this attractive market.
Eventually becoming part of the standard of care in lower risk Mds.
This next slide.
Describes.
Potential market segmentation in more depth.
Anil Kapur: We expect Immitelstad patients to come from four main groups, all of whom are eligible to be enrolled in our ongoing Imerge phase three trials. The first group, and our key focus, is the ESA relapsed and refractory RF negative. This is the largest opportunity of about 22,000 addressable patients in the U.S. and the five largest European markets. These patients currently do not have effective, approved therapy available to treat them
We expect it'll start patients to come from four main groups.
All of whom are eligible to be enrolled in our ongoing <unk> phase III trial.
The first group and our key focus.
Is the ESC relapsed and refractory Addis negative patients.
This is the largest opportunity off about 22000 addressable patients.
In the U S and the five largest European markets.
These patients currently do not have effective.
Approved therapy available to them.
Anil Kapur: If phase three is positive, we expect ImitalStat will become the standard of care in this setting. The second group is the ESA relapse and refractory RS positive group. This group has an addressable patient opportunity of approximately 7,000 patients in the US and the five largest European markets. Note also that this is where Luspacet is currently approved.
If I have much phase III is positive.
We expect <unk> will become the standard of care in this setting.
The second group.
Is the ESC relapsed and refractory Rs positive patients.
This group has an addressable patient opportunity of approximately 7000 patients in the U S and the five largest European markets.
Note also that this is where this partnership with currently approved.
Anil Kapur: We expect a metal start to compete favorably with Lospatosept in this setting, especially in patients with higher waistline transfusion burdens, such as greater than four units per 8p, which is more than 50% of the population in this segment according to RNA. The third group is drawn from the first line low-risk MDS patients with high endogenous serum EPO levels greater than 500 milliliters per ML who are ineligible for the This group has an addressable patient opportunity of approximately 3,700 patients in the U.S. and the five largest European markets.
We expect it'll start to compete favorably with this partnership in this setting.
Actually in patients with higher baseline transfusion burdens such.
Such as greater than 40 units.
It beats, which is more than 50% of the population in this segment according to our analysis.
The third group.
Is drawn from the first line lower risk Mds patients with high endogenous Epo levels.
Greater than 500 million units, but.
Who are ineligible for the assays.
This group has an addressable patient opportunity of approximately 3700 patients in the U S.
And the five largest European markets.
Anil Kapur: Finally, the fourth group of patients for Imitalstadt will come from those who have been previously treated with spadda. The ultimate size of this segment is yet to be determined, and we expect it to grow over time. Just to also point out, as many of you may know, both Lospatocypt and ESAs stimulate production of the normal red blood cell via the EPO receptor, although at different points in the red blood cell lifecycle.
Finally, the fourth group of patients for whom it goes part will come from those who have been previously treated with cytosorb.
Are you seeing maybe size of this segment is yet to be determined and we expect it to grow over time.
Just to also point out as many of you may know, both Novartis and Esa's stimulated production of the normal red blood cells.
The EPA would accept it.
Although the pinpoints in the Red blood lifecycle.
Anil Kapur: Based on the fact that we have encouraging data in patients who have failed ESAs, we expect that ImitalStat will likely be effective in Lospatocyp treated patients. As a reminder, these patients are eligible to be enrolled in our I-merge phase three trial. Based on our current commercial assumptions and assuming regulatory and fair access to the four patient populations I just described, we expect ImitalStat to exceed $1.2 billion in potential peak revenue across the US and the five largest European markets in lower risk MDA.
Based on the fact that we have encouraging data in patients who have failed <unk>.
We expect.
It'll start we'll likely be effective.
In the <unk> treated patients.
As a reminder, these patients are eligible to be enrolled in our <unk> phase III trial.
Based on our current commercial assumptions and assuming regulatory and fair access to the Covid patient populations I just described.
We expect it'll start to exceed $1.2 billion in potential peak revenue across the U S.
And the five largest European market and lower risk Mds.
Lastly.
Anil Kapur: Lastly, as shown on this slide, I would like to provide a brief update on our commercialization with top line results expected in the first quarter of 2023, and assuming priority review, we could potentially launch in the US market in the first half of 2024, as an early preparation for launch. We recently hired senior, highly experienced industry professionals to lead our medical affairs and market access functions. With this team, we will be refining our value proposition across all stakeholders, building a deep understanding of the customer base, and executing on our medical affairs plans to ensure a comprehensive understanding of the potential for Emetal Start within the lower risk MDS treatment community.
As shown on this slide I would like to provide a brief update on our commercialization efforts.
With topline results expected in the first quarter of 2023.
And assuming priority review, we could potentially launch in the U S market in the first half of 'twenty 'twenty four.
And then early preparation for launch.
We recently hired senior high.
Highly experienced industry professionals to lead our medical affairs and market access functions.
With this team we will be refining our value proposition across all stakeholders.
Building, a deeper understanding of the customer base and executing on our medical affairs plans to ensure a comprehensive understanding of the potential for a minute I will start within the lower risk Mds treatment community.
As we do this.
Anil Kapur: We are applying a stage-gated, milestone-driven investment mindset, which will result in the bulk of our commercial investments coming after talk line results are We believe ImitalStat has the qualities that are strongly differentiated from other drugs currently being marketed or in development today for lower risk, and we are excited about the progress we are making to bring this potentially transformational product to the market. With that, I will now hand over the call to Chip.
We are applying a stage gated milestone driven investment mindset.
Which will result in the bulk of our commercial investments coming after topline results are available.
We believe <unk> has the qualities that strongly differentiated from other drugs currently being marketed.
In development today for lower risk Mds.
And we are excited about the progress we are making to bring this potentially transformational product to the market with that I will now hand over the call to chip chip.
Thanks, very much Neil.
John A. Scarlett: Thanks very much, Anil. When I turned the call over to Olivia, I said we had a lot to cover today. Now, you can see one of the reasons I said that.
When I turn the call over to Olivia I said, we had a lot to cover today now you can see one of the reasons I said that.
And Neal just commented that Intelsat is a potentially transformative product.
John A. Scarlett: And Neil just commented that Imittlestad is a potentially transformative product. I'd like to add that, with Imitelsat, we expect to transform Geron into a dynamic commercial company over the next several years and to become a leader in the treatment of hematologic and lung cancer by modifying the course of these diseases in order to improve and extend the lives of patients. As we conclude our call today, based on this slide, we'd like you to know that we plan to host a virtual event for investors and analysts in November when management and key opinion leaders will discuss several broad topics.
I'd like to have that within <unk>, we expect to transform <unk> into a dynamic commercial company over the next several years.
And to become a leader in the treatment of hematologic malignancies.
Modifying the course of these diseases in order to improve and extend the lives of patients.
As we conclude our call today.
Shown on this slide we'd like you to know that we plan to host a virtual event for investors and analysts in November when management and key opinion leaders, who will discuss several broad topics. The first is <unk> potential for disease modification in lower risk Mds and refractory MF.
The next is our expected path to commercializing Amytal staff.
Third is an expansion of the <unk> development plans, including new studies and additional indications.
Operator: The first is Imitalstatt's potential for disease modification and lower risk MDS and refractory MS. The next is our expected path to commercializing Imital staff. The third is an expansion of Imetelistat's development plans, including new studies and additional indications, and the fourth will at least briefly cover our early discovery program for second generation psalamase inhibitors. So please look for further details about this event that will probably come in October of this year. So that operator, let's please open the call to questions.
In the fourth will at least briefly cover our early discovery program and second generation <unk> inhibitors.
So please look for further details about this event that will probably come in October of this year.
So with that operator, let's please open the call to questions.
Absolutely. Thank you ladies and gentlemen, if you have a question at this time. Please press Star then the number one key on your question on telephone.
My question has been answered or you wish to remove yourself from the queue. Please press the pound key.
Your first question comes from the line of Charles Duncan from Cantor Fitzgerald. Your line is open.
Yes, good afternoon, John and team thanks for taking our questions and congratulations on the.
Operator: Absolutely, ladies and gentlemen, if you have a question at this time, please press the number one key on your touchstone telephone. If your question has been answered or you wish to remove yourself from the queue, please press the counter. Your first question comes from the line of Charles Duncan from Canter Fitzger-Dir Lange Lain's old, Yes,
Enrollment progress Eni emerge.
So I.
Yeah. So.
Just a couple of questions and.
Alexander.
I guess kind of alluded to this but with regard to the primary analysis.
Kipp and FDA response, I think she said no comments from FDA. So you take that as agreement from them.
Charles Duncan: Yes, good afternoon, John and team. Thanks for taking our questions and congratulations on the enrollment progress in IMIRG. So I, Yeah, he had just a couple of questions, and Alexander, I guess, kind of alluded to this, but with regard to the primary analysis, Chip, and FDA response, I think she said no comment from FDA. So you take that as agreement from them that the analysis you've done to allow for a shorter follow-up is acceptable with them.
The analysis, you've done to allow for a shorter follow up is is copacetic for it with them.
Go ahead, Alex sure so and Charles we submitted a protocol amendment to the FDA over 30 days ago and.
You know we have not received any comments on the proposed change.
You know this is kind of a regular procedure. If you don't hear from the agency within 30 days.
No you go ahead and.
Unknown Attendee: Go ahead, Alex. Sure, so, Charles, we submitted a protocol amendment to the FDA over 30 days ago, and, you know, we have not received any comments on the proposed change. As you know, this is kind of a regular procedure. If you don't hear from the agency within 30 days, you go ahead and, you know, issue a protocol amendment and move forward. So we are at that stage. The 30 days have passed, and no comments have been received. So we're moving ahead. Okay?
You know.
Issued a protocol amendment and move forward. So we are at that stage. The 30 days have passed and no comments received so we're moving ahead.
Okay very good and then with regard to the enrollment thus far.
Nearly complete again, congrats any geographic concentration or.
Any any color that you can provide on the enrollment pattern relative to the criteria.
I don't think so I don't think there's any geographic pad pattern here you know we've.
Put so much time and effort in all the enrolment boosting activities.
Unknown Attendee: Okay, very good. And then with regard to the enrollment thus far, yeah, nearly complete, again, congrats. Any geographic concentration or any color that you can provide on the enrollment pattern relative to the criteria?
We also believe that the rollout of the vaccines, especially with all of it for the elderly patients that are enrolled in our trials is has helped so you know with that.
The enrollment has really picked up very well, but no to go back to your question I don't think theres any any pattern there.
Okay, and then regarding the blinded efficacy.
Unknown Attendee: I don't think so. I don't think there's any geographic pattern here.
<unk> interim looks at DMC look in July was there any criteria for trial.
Unknown Attendee: You know, we've put so much time and effort into all the enrollment boosting activities. We also believe that the rollout of the vaccines, especially for the elderly patients that are enrolled in our trials, has helped. So, you know, with that, enrollment has really picked up very well. But, no, to go back to your question, I don't think there's any pattern there. Okay.
You know I guess changes any any efficacy.
Criteria or anything else that really could have come out of that meeting.
<unk> safety.
Right. So so all we are here from D. I B M C.
Pearl if the documents per the charterers is whether the study should continue with or without modifications. So just like last time. They you know at this time, they should continue without Gino without modification. So.
Unknown Attendee: Okay, and then regarding the blinded efficacy or blinded interim looks, the IDMC look in July, was there any criteria for trial? You know, I guess changes, any efficacy criteria, or anything else that really could have come out of that.
That's okay as we know.
Okay last question is and I'll, probably have to wait till November to get.
The answer to this but when you contemplate commercialization of Vim itself stand across the globe do you think that you'll end up working with a partner for ex U S. I mean, what is what is your preferred strategy there.
Unknown Attendee: Right, so all we hear from the IDMC, you know, per all of the documents per the charters, is whether the study should continue with or without modifications. So just like last time, this time, they said continue without, you know, without modification. That's as much as we know.
Chavez chip yes.
Actually deep in.
Consideration of that question doing a lot of analyses a lot of a lot of background work a lot of specific plea with regard to the drug in the markets and so I think today, we're not ready to make that have that comment.
John A. Scarlett: Okay, last question is, and I'll probably have to wait until November to get the answer to this, but when you contemplate commercializing Imital Stad across the globe, do you think that you'll end up working with a partner for XUS? I mean, what is your preferred strategy?
Comment on it but that is our.
That's kind of our plan and stay tuned as they say.
Okay.
Thanks for taking my questions Congrats on the progress.
Thanks, so much thanks, Jeff.
Thank you and your next question comes from the line of Justin Wash from B Riley Securities. Your line is open.
John A. Scarlett: Chess, Chip, yeah, we're actually deep in consideration of that question, doing a lot of analyses, a lot of background work, a lot of work specifically with regard to the drug and the markets. And so I think today we're not ready to make that, you know, have that comment, comment on it, but that is our plan. That is, that is kind of our plan. And stay tuned, as they say. Okay. Thanks for taking my questions. Congratulations on your progress.
Hi, Thanks for taking the questions.
I'm sure we'll hear more details in November, but maybe you could provide some color on why now is a good time to expand and Mattel stats development plans and how the second generation telomerase inhibitors are expected to fit into the Geron story.
I'll, let Alex take the first part and I'll take the second part so.
Additional development plans, who want to just comment about that I think that at this point Justin. We you know we are certainly considering two to expand in areas, where we have a good preclinical data.
John A. Scarlett: Thanks so much. Thanks, Jeff.
We believe in our data we believe our strong data and you know there's always it's always a good time to expand into additional indications. If you have good data.
Justin Walsh: And your next question comes from the line of Justin Walsh from B Riley Securities, your learning Hello, thanks for taking the questions. I'm sure we'll hear more details in November, but maybe you could provide some color on why now is a good time to expand Imitelstats' development plans and how the second
Yeah, I think I'd add to that Justin if you look back at a consistent theme that we've had for quite some time has been the exploration of the effect of <unk> on.
The molecular basis of the disease and in particular on malignant stem and progenitor cells and I think we've put together an amazing.
Unknown Attendee: Generation Pellomerase inhibitors are expected to fit into the geron story. I'll let Alex take the first part, and I'll take the second part. So, additional development plans? Do you want to just comment about that?
Cascade of data, that's really strongly supported that what's going on is that we are selectively targeting and killing malignant stem and progenitor cells in the marrow and these are these are the cells and the clones that are responsible for the disease. So when we see in both of our big phase III programs of really.
Unknown Attendee: I think that at this point, Justin, we are certainly considering expanding in areas where we have good pre-clinical data. We believe in our data, we believe it's strong data. You know, it's always a good time to expand into additional indications if you have good data.
Good evidence that that there is significant.
So and selective killing of these malignant stem and progenitor cells and ultimately the mineral repopulate with with with more normal cells.
I think that really encourages us to look at additional indications in heme malignancies. This is kind of what's really needed right and so now that we've got both of these phase III studies up running one almost completed in terms of enrollment I think it's the right time to look at other opportunities.
John A. Scarlett: Yeah, I think I'd add to that, Justin. If you look back, a consistent theme that we've had for quite some time has been the exploration of the effect of imitelsstat on the molecular basis of disease, and in particular on malignant stem and progenitor cells. And I think we've put together an amazing cascade of data that's really strongly supported that what's going on is that we're selectively targeting and killing malignant stem and progenitor cells in the marrow.
<unk> and of course as Alexander says we have.
Preclinical data non clinical data some of which is public some of which is not yet public that really encourage us. So stay tuned for November I think we're excited to tell you about some of this.
Got it and how about the second generation inhibitors, how do those fit in.
Well.
I think it's a fair statement to say that.
It would tell us that is really a first generation telomerase inhibitor. It's the only one that we're really aware of that is.
John A. Scarlett: And these are the cells and the clones that are responsible for the disease. So when we see in both of our big phase two programs really good evidence that there is significant and selective killing of these malignant stem and progenitor cells, and ultimately the marrow repopulates with... with more normal cells, I think that really encourages us to look at additional indications in these malignancies. This is kind of what's really needed, right?
In the clinic today and is advance this far there are a lot of things we would love to do that you can imagine that would incorporate.
Even more attractive profile and I think we won't get into that today, and we want to save a little bit of the powder for for November but we.
We just.
We decided sometime ago that it was really the right time to go off and begin the early stages of a medicinal chemistry effort.
To identify other scaffolds that would be a potentially attractive.
And we'll go into that in a little more detail, obviously, a lot of it remains proprietary but we did want people to know that we're not just sitting back with Intel status are only.
John A. Scarlett: And so now that we've got both of these phase three studies up and running, one almost completed in terms of enrollment, I think it's the right time to look at other opportunities. And, of course, as Alexander says, we have pre-clinical data, non-clinical data, some of which is public, some of which is not yet public, that really encourage us. So stay tuned for November. I think we're excited to tell you about some of them. Got it. And how about the second generation inhibitors? How do those fit in?
Idea towards telomerase inhibition, we think the data really strongly supports.
Going forward in multiple different areas and having a new drug new indications or new IP, new everything would be very potentially.
Potentially attractive.
Got it and I'm looking forward to hearing more in November.
Next question. So what if anything do you guys think that the the recent acquisition of constellation could tell us about the deal appetite and continued interest in the EMS space.
John A. Scarlett: Well, I think it's a fair statement to say that Emmettelstatt is really a first-generation telomerase inhibitor. It's the only one that we're really aware of that is in the clinic today and is advanced this far. There are a lot of things we would love to do that you can imagine that would involve an even more attractive profile, but I think we won't get into that today and want to save a little bit of the powder for November.
Well whenever you see one of your colleagues who is involved in similar areas that you are get acquired and at a very nice premium I don't think that it makes any of us.
Anything, but good right I mean, that's just a shareholder a broad shareholder perspective.
And one that I.
John A. Scarlett: But we just decided some time ago that it was really the right time to go off and begin the early stages of a medicinal chemistry effort to identify other scaffolds that would be potentially attractive. And we'll go into that in a little more detail. Obviously, a lot of it remains proprietary, but we did want people to know that we're not just sitting back with Imital Stad as our only idea towards telomeree's inhibition. We think that the data really strongly supports going forward in multiple different areas, and having a new drug, new IP, new everything would be very potentially attractive.
Hard for anybody to argue against.
I think that it's an interesting deal we won't go into how we view various elements of the deal itself.
But I will say that it was that it was a healthy price it was still relatively early.
Asset and I, just see more opportunities.
We'll come the way for patients to get better therapy as more resources are put into any of these drugs. So.
Good for them and good for the business and most of all good for patients.
Got it now our last question from me.
Now that we have patients enrolling in impact EMS do you have a sense. If most of these patients have seen treatment with <unk> as well as retrofitting them, then how that could possibly affect any read through from embark.
John A. Scarlett: Got it. Well, I'm looking forward to hearing more in November.
Operator: Next question. So what, if anything, do you guys think about the recent act?
So I can take that question I think that at the moment I mean, it's a randomized study it's meant for F. Four registration and we're really not looking into the data a patient disposition or anything like that so I think it's very early for us to give a comment.
John A. Scarlett: that the recent acquisition of Constellation could tell us about the deal pace and continued interest in the MS space.
John A. Scarlett: Well, whenever you see one of your colleagues who is involved in similar areas that you are, get acquired and at a very nice premium, I don't think that it makes any of us feel anything but good, right? I mean, that's just a shareholder, a broad shareholder perspective, and one that I..., hard for anybody to argue against again. I think that it's an interesting deal, but we won't go into how we view various elements of the deal itself.
On that Justin Yeah, I would agree.
Sorry.
Yes.
I would tell it would make a separate note I would make a separate comment our perception is that for drought and it does not have a huge uptake in the market, but that's not that's really not for us to make comments about.
And as Alex said disappointed big blinded study and Hans hands off.
Most of this.
Non blinded bodies randomize.
In phase III registration.
John A. Scarlett: But I will say that it was a healthy price. It was still a relatively early asset, and I just say more opportunities will come the way for patients to get better therapy as more resources are put into any of these drugs. Good for them and good for business, and most of all, good for them. Now, last question from me. Now that we have patients enrolling in Impact MS, do you have a sense if most of these patients have seen treatment with thadratinid as well as ruxolitinid? Then how could that possibly affect any read-through from Embark?
Exactly.
They should not be looking at any of that exactly.
Got it sounds good thanks for taking the questions.
You bet.
Thank you and your next question comes from the line of Stephen Willey from Stifel. Your line is open.
Yes. Good afternoon, thanks for taking the questions and congrats on the enrollment progress.
I was wondering so I know that you're speaking to.
I guess the subgroup of <unk>.
Potentially frontline patients that are Esa ineligible.
I know that they were I think a fractional component.
Unknown Attendee: So I can take that question. I think that at the moment, I mean, it's a randomized study. It's meant for registration, and we're really not looking into the data, patient disposition, or anything like that. So I think it's very early for us to give a comment on that question. Yeah, I would agree.
The phase two patient population I'm, not sure if you've ever seen activity or or clinical data broken out of them.
That's a function of.
Assay eligibility or eligibility, but just wondering if you've had any regulatory conversations around.
Unknown Attendee: I would agree. However, I would make a separate comment.
Your ability to procure a label that would be in in this subgroup based upon the phase III emerge data specifically in the context of the distribution of Esa in eligible patients looking similar to the phase two experience.
John A. Scarlett: Our perception is that phidratinib does not have a huge uptake in the market, but that's really not for us to make comments about. And as Alex said, this is a blinded, big blinded study, and hands off from this.
Unknown Attendee: Most of this, and not blinded, but it's randomized and face re-registration. Yeah, so it's exactly what they should not be looking at any of that.
Yeah. It's a great question a very thoughtful question. Thank you Alex we will start yes.
Hmm.
This is actually data that's published in the GTO and <unk> seen a couple of our abstracts or conference presentations, where we've been reporting data.
Unknown Attendee: Exactly. Got it. Sounds good. Thanks for taking the questions. Thank you. And your next question comes from the line, Stephen Willie, from Steefall. Your line is both.
With.
In different subgroups of patients with a lower or higher than 500 micro units per ml of Ebola.
And what.
What we've what we've shown you said actually you can achieve transfusion in the eight week transfusion independence or H I E irrespective in which subgroup you belong right. So you're talking specifically for those that aren't the hi E. Pas riser that are refractory to <unk> to Epo patients.
Stephen Douglas Willey: Yeah, good afternoon. Thanks for taking the questions, and congrats on the enrollment progress. I was wondering, so I know that you're speaking to, I guess, this subgroup of potentially frontline patients that are ESA ineligible. I know that they were, I think, a fractional component of the phase two patient population. I'm not sure if we've ever seen activity or clinical data broken out as a function of ESA eligibility or ineligibility. But just wondering if you've had any regulatory conversations around your ability to procure a label that would be in this subgroup based upon phase three. I merge data specifically in the context of the distribution of ESA ineligible patients looking similar to the phase two experience.
That have never seen an EPA before they've never seen any treatment. So far so based on our data.
Hum.
I believe that this patient population.
It can be included in our in our label So and we had we had a good proportion of patients even in our phase II study that we're in D. C are in this bucket I would say.
So I don't know cheap if you want to add something to that so yes, we don't usually get into conversations that we've had or haven't had with regulators and it's always speculative right. How this works out but I think the critical element is that there is a small but meaningful percentage of patients who never respond to.
Unknown Attendee: Yeah, it's a great question, a very thoughtful question, thank you. Alex Wilson.
Unknown Attendee: This is actually data that's published in the JCO and also in a couple of our abstracts or conference presentations, where we've been reporting data in different subgroups, so patients with lower or higher than 500 microunits per ML of EPO. And what we've shown is that actually you can achieve transfusion independence in the... eight-week transfusion independence or HIE, no matter in which subgroup you belong, right? So you're talking specifically about those that are high EPO, right? So the refractory to EPO patients that have never seen an EPO before; they've never had any treatment so far.
<unk> and those patients were included in.
Medalist study as I recall.
And we've seen some patients in our <unk> and our phase two so I think we feel pretty comfortable with the idea that we will be able to access that patient population.
How it actually comes out and whether it gets labeled whether it's via MCC and guidelines. There are a lot of different ways. This can play out but the most important thing is to have data. So we would expect to have data that would bear on your question Steve.
Okay, and then maybe just.
A follow up from one of the earlier questions with respect to Nextgen telomerase inhibitor is and again, maybe this is a.
November Investor day topic, but are are some of these next gen compounds that youre looking at are they also I guess all ago based in terms of.
John A. Scarlett: So based on our data, you know, I believe that this patient population, you know, can be included in our label. And we had, we had a good proportion of patients even in our phase two studies that were in this, in this bucket, I would say. I don't know, Chip, if you want to add something to that, so... Yeah, we don't usually get into conversations that we've had or haven't had with regulators, and it's always speculative, right, how this works out.
In terms of structure or.
Is there a way I guess from Medicare perspective that we should think about Nextgen telomerase inhibitor is being I think a little bit maybe more in the traditional small molecule mode.
Well Youre right.
This.
I'm going to just tease you on this and not tell you anything more than what we will talk about a little bit more.
John A. Scarlett: But I think the critical element is that there is a small but meaningful percentage of patients who never respond to ESAs, and those patients were included in the medalist study, as I recall, and we've seen some patients in... in our phase two. So I think we feel pretty comfortable with the idea that we'll be able to access that patient population. How it actually comes out and whether it gets labeled, whether it's via MCCN guidelines, there are a lot of different ways this can play out, but the most important thing is to have data. So we would expect to have data that would bear on your questions. Okay, and then maybe just I will.
In November we wanted to make sure people had a good reason to come to that call and that was one of them was we thought this would have some real interest.
Let it suffice to say right now that we would like to see.
Whole host of things that obviously, if you startup.
Discovery program. Many years. After your initial discovery was started there are lots of things that you would change and we'll talk about some of those and some of the scaffolds and what they look like.
So it's de Novo medicinal chemistry work that we're doing let's put it that way.
Very interesting thanks for taking the questions.
Thank you.
Yes.
Thank you. This concludes the Q&A session of the call I would now like to turn the conference back to Dr. John Scarlett for any closing remarks.
John A. Scarlett: Okay, and then maybe just, I guess, a follow-up from one of the earlier questions with respect to next-gen telomerates inhibitors, and again, maybe this is a November investor day topic, but are some of these next-gen compounds that you're looking at, are they also, I guess, oligo-based in terms of, in terms of structure or, Is there a way, I guess, from a MedCamp perspective that we should think about next-gen telomerase inhibitors as being, I think, a little bit maybe more in the traditional small molecule mold? Thanks.
Thanks, everybody for joining us today, we really appreciate you taking the time to dial in and participate and we do expect to present at three healthcare conferences in September.
And webcast details for those events will be available at the beginning of next month, so keep an eye out for that and we also obviously look forward to virtually seeing many of you at our Investor day in November. Thank you.
Everybody have a good afternoon.
Thank you presenters, ladies and gentlemen. This concludes today's conference call. Thank all for joining you may now disconnect.
Yes.
John A. Scarlett: Well, you're right. I'm going to just tease you on this and not tell you anything more. We will talk about it a little bit more in November. We wanted to make sure people had a good reason to come to that call, and that was one of them. We thought this would have some real interest. Let it suffice to say right now that we would like to see a whole host of things that, obviously, if you start a discovery program many years after your initial discovery was made, there are lots of things that you would change. And we'll talk about some of those and some of the scaffolds and what they look like. So it's de novo medicinal chemistry work that we're doing. Let's put it that way. Great
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Stephen Douglas Willey: Okay, very interesting. Thanks for taking the question.
John A. Scarlett: Thank you. This concludes the Q&A session of the call. I would like to turn the conference back to Dr. John Scarlett for any closing remarks.
Operator: Thank you everybody for joining us today. We really appreciate you taking the time to dial in and participate. We do expect to present at three healthcare conferences in September, and webcast details for those events will be available at the beginning of next month. So keep an eye out for that. And we also obviously look forward to seeing many of you at our Investor Day in November. Thank you. Everybody, have a good afternoon. Thank you.
Yes.
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Operator: Thank you, presenters, ladies and gentlemen. This concludes today's conference call. Thank you all for joining. I'm going to disconnect.
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