Q4 2023 Geron Corp Earnings Call
[music].
Operator: Good morning, my name is Rob, and I will be your conference operator today. At this time, I would like to welcome everyone to the Geron Corporation's fourth quarter and full year 2023 conference call. All lines have been placed on mute to prevent any background noise.
Good morning, My name is Rob and I will be your conference operator today at this time I would like to welcome everyone to the Geron Corporation fourth quarter and full year 2023 conference call. All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question and answer session if you'd like to ask a question.
Operator: After the speaker's remarks, there will be a question and answer session. If you'd like to ask a question during this time, simply press the star followed by the number one on your telephone keypad. If you would like to withdraw your question again, press star number one.
During this time simply press star followed by the number one on your telephone keypad. If you would like to withdraw your question again press the star one.
Aaron Feingold: Thank you. Aaron Feingold, Vice President of Investor Relations and Corporate Communications. You may begin your conference. Good morning, everyone.
Aaron Feingold, Vice President of Investor Relations and corporate Communications you May begin your conference.
Good morning, everyone welcome to the Geron Corporation fourth quarter and full year 2023 earnings conference call I am Erin angle, Sir I'm, Vice President of Investor Alicia.
Erin Feingold: Welcome to the Geron Corporation fourth quarter and full year 2023 earnings conference call. I am Erin Feingold, Geron's Vice President of Investor Relations and Corporate Chiefs. I'm joined today by several members of Geron's management team. Dr. John Scarlett, Chairman and Chief Executive Officer. Michelle Robertson, Executive Vice President and Chief Financial Officer; Dr. Faye Feller, Executive Vice President and Chief Medical Officer. Anil Kapoor, Executive Vice President of Corporate Strategy and Chief Commercial Officer, and Dr. Andrew Bresline, Executive Vice President and Chief Operating Officer. Before we begin, please note that during the course of this presentation and question and answer session, we will be making forward-looking statements regarding future events, performance, plans, expectations, and other projections, including those relating to the therapeutic potential and potential regulatory approval of an adult's VAT, anticipated clinical and commercial events and Actual events or results could differ materially. Therefore, I refer you to the discussion under the heading risk factors and Geron's most recent periodic report filed with the FCC, which identifies important factors that could cause actual results to differ materially from those contained in the forward-looking statement. Geron undertakes no duty or obligation to update this forward-looking statement.
I'm joined today by several members of their management team.
Doctor, John Scarlett, Chairman and Chief Executive Officer, Michelle Robertson Executive Vice President and Chief Financial Officer, Doctor Bay seller, Executive Vice President and Chief Medical Officer.
Neil <unk> executive Vice President of corporate strategy, and Chief Commercial Officer, and Doctor, Andrew <unk> Executive Vice President and Chief operating Officer.
Before we begin please note that during the course of this presentation.
We will be making forward looking statements regarding future events.
Kirk one that's quite an expectation and other projects.
Including those relating to the therapeutic potential and I'm sort of regulatory.
Regulatory approval.
<unk> anticipated clinical and commercial lives on unrelated timeline, the sufficiency of Jarrod on financial resources and other statements that are not historical fact.
Actual events or results could differ materially.
Before I work or you said the discussion under the heading risk factors in <unk>. Most recent periodic reports filed with the ICC, which identifies important factors that could cause actual results to differ materially from those contained in the forward looking statements <unk> undertakes no duty or obligation to update our forward look.
Chet: With that, I'll turn the call over to Chet. Thanks, Aaron. Good morning, everyone. Thanks for joining us. Geron's progress and execution throughout 2023 has paved the way for a potentially transformational 2024 as we plan for the transition to becoming a commercial company. FDA has assigned a PDUFA date of June 16, 2024 for Imitelstat for the treatment of transfusion-dependent anemia in patients with lower-risk MDS and has also provided notice that it is scheduled an ODAC as part of the Imitelstat MDA review to be held on In addition, a review of our MAA for the same indication is expected to be completed in early 2025. We're focused on and prepared for these critical next steps in the regulatory review process, which we hope will result in approval of what we believe is a highly differentiated and important treatment option for patients with transfusion-dependent lower-risk MDS.
With that I'll turn the call over to Chad.
Thanks, Aaron Good morning, everyone. Thanks for joining us today.
<unk> progress and execution throughout 2023 has paved the way for a potentially transformational 2024, as we plan for the transition to becoming a commercial company.
<unk> has signed a <unk> date of June 16, 2024, <unk> for the treatment of transfusion dependent anemia in patients with lower risk Mds and has also provided notice that it's scheduled an OTA. It's part of the image hopes that NDA review to be held on March 14th 2024.
In addition, a review of our MAA for the same indication is expected to be completed in early 2025.
We're focused on and prepared for these critical next steps in the regulatory review process.
Which we hope will result in approval of what we believe is a highly differentiated and important treatment option for patients with transfusion dependent lower risk Mds.
Chet: The publications from our pivotal eMERGE Phase III clinical trial, including most recently in the Lancet, show a robust response rate and an unprecedented durability of red blood cell transfusion independence with Imitelstat treatment across multiple MDS patient subgroups, addressing areas of high unmet need. Additional unique attributes of Imitelstat include patient-reported outcomes of less fatigue and significant reductions in variant allele frequency in commonly mutated MDMA, with a well-characterized safety profile of generally manageable and short-lived thrombocytopenia and neutrophils, we believe the clinical evidence supporting the benefits of Imitelstat is compelling and that it can become a transformational treatment option for currently underserved populations if approved by regulatory authority.
The publications from our pivotal emerge phase III clinical trial, including most recently in the Lancet go robust response rates and an unprecedented durability of red blood cell transfusion independents with image helps out treatment across multiple mds patient subgroups addressing areas of high unmet need.
Additional unique attributes of Intelsat include patient reported outcomes with less fatigue and significant reductions in variant allele frequency and commonly mutated M. D. S. G.
With a well characterized safety profile generally manageable and short lives thrombocytopenia neutropenia, we believe the clinical evidence supporting the benefits of Intelsat is compelling and that it can become a transformational treatment option for currently underserved populations if approved by regulatory.
Sure.
Chet: Behind our lead indication, lower risk MDS, is a similarly important program, the Phase 3 Impact MS clinical trial in JACI relapsed and refractory myelocybrosis, and interim analysis is expected for this study in the first half of 2025. Impact MF is the first and only MF phase three trial with overall survival as a primary endpoint. If the expected interim analysis in 2025 or the final analysis expected in 2026 is positive, these data could be transformational for patients with JACI relapse and refractory MAP who have dismal survival prognoses. Our two lead indications for Imitelstat represent significant commercial opportunities, with a total addressable market, or TAM, of $3.5 billion for each indication across the U.S. and EU in 2030, thus representing a combined $7 billion TAM for transfusion-dependent lower-risk MDS and relapsed relapsed Rheumatoid arthritis.
Behind our lead indication in low risk Mds is a similarly important program the phase III impact MF clinical trial, and Jack I relapsed and refractory myelofibrosis.
An interim analysis is expected for this study in the first half of 2025 impact that map is the first and only MF phase III trial with overall survival as a primary endpoint.
The expected interim analysis in 2025 or the final analysis is expected in 2026, if positive these data could be transformational for patients with jacquard relapse and refractory MF look dismal survival prognosis today.
Our two lead indications for <unk> that represent significant commercial opportunities with the total addressable market or Tam of $3 $5 billion for each indication across U S and EU and 2031.
Thus, representing a combined $7 billion Tam for transfusion dependent lower risk Mds and relapsed refractory in that.
Chet: Given this very substantial opportunity, amid a deep unmet need for transfusion-dependent lower-risk MDS, we expect to be prepared to launch and self-commercialize Emetelstat in its lead indication upon potential FDA approval in the middle of this year. We have also completed multiple long-lead activities to prepare Geron and Mattelstat and the market for our potential launch in the US with the goal of ensuring broad access and reimbursement for our important medicine. Moreover, if Imitelstat is approved by the European Commission in transfusion-dependent low-risk MDS, we expect commercial launch in Europe would occur in 2025. We're continuing to evaluate our strategic options for European commercialization, including self-commercialization or partnering, and expect to be able to provide an update later this year.
Given this very substantial opportunity amid a deep unmet need in transfusion dependent lower risk Mds, we expect to be prepared to launch and self commercialize intelsat in its lead indication.
Potential FDA approval in the middle of this year.
We have also completed multiple long lead activities to prepare jiron, Intelsat and the market for our potential launch in the U S with the goal of ensuring broad access and reimbursement for our important medicine.
Moreover, if <unk> is approved by the European Commission in transfusion dependent lower risk Mds, we expect commercial launch in Europe would occur in 2025.
We're continuing to evaluate our strategic options for European commercialization, including self commercialization or partnering and expect to be able to provide an update later this year.
Chet: Lastly, we ended 2023 with a strong cash position of approximately $378 million, which, based on our current plans and expected available resources, we expect will enable us to fund a potential successful launch of transfusion-dependent lower-risk MDFs in the U.S. and fund our planned operations into the third quarter of 2025. We believe our differentiated product candidate, the very important commercial opportunities in transfusion-dependent lower-risk MDFs and relapsed refractory MS, the excellence and experience of our employees, and the financial resources to execute on our near-term milestones put us in a strong position for value creation. With that, I'll turn the call over to Faye for a regulatory and clinical update. Faye?
Lastly, we ended 2023 with a strong cash position of approximately $378 million, which based on our current plans and expected available resources we have.
Expect will enable us to fund a potential successful launch in transfusion dependent lower risk Mds in the U S and fund our planned operations into the third quarter of 2025.
We believe our differentiated product candidates the very important commercial opportunities in transfusion dependent lower risk Mds in relapsed refractory MF, the excellence and experience of our employees and the financial resources to execute on our near term milestones puts us in a strong position for value creation.
With that I'll turn the call over to say for a regulatory and clinical update.
Faye: Thanks, Tiff, and good morning to everyone on the call. As Tiff mentioned, we are deeply focused on the regulatory processes for our Imitelstat MDA and MAA, which are currently under review by the FDA and EMA for the treatment of transfusion-dependent anemia in patients with lower-risk MDS who have failed to respond to or have lost response to or are ineligible for ESA. As previously disclosed, the FDA assigned a PDUFA action date of June 16, 2024. On January 30, 2024, the FDA also provided notice in the Federal Register that it has scheduled an ODAC as part of the Imitalstat NDA review to be held virtually on March 14, 2024. We feel very well prepared for the scheduled ODAC.
Thanks, Ken and good morning to everyone on the call.
Mentioned, we are deeply focused on the regulatory processes for her and the top that NDA and MAA, which are currently under review by the FDA and Eni for the treatment of transfusion dependent anemia in patients with lower risk Mds, who have failed to respond or have lost response to or are ineligible for Esa.
As previously disclosed the FDA assigned a <unk> action date of June 16, 2024.
On January 32024, the FDA also provided notice in the Federal Register that it has scheduled and oded as part of the <unk> NDA review.
Be held virtually on March 14th 2024, we feel very well prepared for the scheduled or that they've been working for many months with an expert consultancy group, which complements our in house regulatory experience.
Faye: We've been working for many months with an expert consultancy group, which complements our deep in-house regulatory experience. I am personally excited to have the opportunity to discuss Imatelstat with experts and peers, as we believe Imatelstat could be an important and compelling new medicine for transfusion-dependent, low-risk MDS patients. Our readout of positive top-line results from our pivotal eMERGE trial in January 2023 was followed last year by a number of additional presentations and analyses of the data, including at ASCO and EHA earlier in the year and last quarter at ASH. This growing body of data from the trial continues to give us confidence in what we believe is a meaningful clinical benefit and manageable safety profile of Telstat in patients with transfusion-dependent lower-risk MDS As reported at these conferences, the clinical attributes of Imitalsac were differentiated, particularly with respect to high RBC-PI response rates, durability of response, and the consistency of effect across MDS subgroups that have historically been very difficult to treat.
I am personally excited to have the opportunity to discuss in the couch that with experts and Pierre I believe.
Please note that could be an important and compelling new medicine for transfusion dependent.
Risk Mds patients.
Our readout are positive topline results from our pivotal emerge trial in January 2023, well follow back to you by a number of additional presentations and analyses of the data, including at Opco and <unk> earlier in the year and last quarter at Ash.
This growing body of data from the trial continued to give a confidence in what we believe is a meaningful clinical benefit and manageable safety profile with <unk> in patients with transfusion dependent lower risk Mds.
As reported at these conferences the clinical attributes of <unk> differentiated, particularly with respect to high RBC Ti response rate durability of response and the consistency of effect across Mds subgroups that has historically been very difficult to treat.
Faye: The Lancet published results from our eMERGE Phase 3 trial this past December, a strong validation of the importance of the study within the field, as well as a powerful way to reach hematologists and other providers globally with these potentially practice-changing results that offer the possibility of relief for lower risk MDS patients from chronic reliance on blood cell transfusions. Turning now to our Phase 3 trial of Imitalstat in Relapsed Repractory MS, we were excited to have completed 50% enrollment in the study in November of 2023. We continue to expect an interim analysis in the first half of 2025, which will occur when approximately 35% of the planned enrolled patients have died. A final analysis is expected in the first half of 2026 when over 50% of the planned enrolled patients have died.
The lancet published results from our emerge phase III trial. This past December a strong validation of the importance of the study within the field as well as a powerful way to reach hematologists and other providers globally, but these potentially practice changing results.
And also the possibility of relief for lower risk Mds patients from chronic reliance on blood cell transfusions.
Turning now to our phase III trial of Intelsat in relapsed refractory and that we were excited to have completed 50% enrollment in the study in November of 2023.
We continue to expect an interim analysis in the first half of 2025, which will occur when approximately 35% that the planned enrolled patients have died.
Final analysis is expected in the first half of 2020.
And over 50% of the planned enrolled patients have died.
Faye: We look forward to continuing to keep you updated on this important study. Lastly, we are also evaluating Imitalstat in a Phase I study as a combination therapy with Brexolytinib in patients with frontline myelofibrosis. Our main goal for this combination study, known as IMPROVE-MF, is to determine the safety profile of the combination regimen of ruxolitinib and imitofet, as well as to explore their potential activities in a frontline MF disease setting.
Look forward to continuing to keep you updated on this important study.
Lastly, we are also evaluating the top that in the phase one study as a combination therapy with <unk> in patients with frontline myelofibrosis.
Our main goal for the combination study known as improve enough to.
To determine the safety profile of the combination regimen of <unk> and then it helps that as well as to explore the potential activity in frontline MF disease setting in January 2024, we escalate it to the third of four dose cohorts in the study following a unanimous decision by the study's safety evaluation team or Sac, who will review the second cohort data.
Aneel: In January 2024, we escalated to the third of four dose cohorts in the study, following an unanimous decision by the study's safety evaluation team, or SET, who reviewed the second cohort data. We are very pleased with this progress and look forward to providing future updates. With that, I'll turn the call over to Aneel for a commercial update. Okay?
We are very pleased with this progress and look forward to providing future updates.
That I will turn the call over to Neal for a commercial update aneel.
Thank you and good morning, everyone.
I look forward to discussing first where do we see significant unmet need in the market.
Dan will provide a brief update on our U S launch.
There remains significant unmet need across key transfusion dependent lower risk Mds patient populations.
Aneel: Thank you, Faye, and good morning, everyone. I look forward to discussing first where we see significant unmet needs in the market, and then we'll provide a brief update on our U.S. launch plan. There remains significant unmet need across key transfusion-dependent lower-risk MDS patient populations that are underserved by current available treatment options. Approximately 10% of lower-risk MDS patients are not eligible for ESA and represent a very high unmet need. RS negative patients make up approximately 75 percent of lower risk MDS patients and are a population particularly vulnerable to poor clinical outcomes. There are no treatments indicated for the treatment of anemia in RS-negative patients once they have relapsed or are refractory to ESP.
Underserved by current available treatment options.
Approximately 10% of lower risk Mds patients are not eligible quota Esa and represent a very high unmet need subgroup.
Rs negative patients make up approximately 75% of Nordisk Mds patients are not a population, particularly vulnerable to poor clinical outcomes.
There are no tariff these indicated for the treatment of anemia in Rs negative patients once they are relapsed or refractory to <unk>.
Rs positive patients make up approximately 25% of the lower risk Mds patients and most who had high transfusion burden lack of effective treatment options.
These underserved subgroups are at greater risk for disease progression.
And suboptimal survival and not in the need for more effective treatment options.
Aneel: RS-positive patients make up approximately 25% of the lower-risk MDS patients, and most who are at high transfusion burden lack effective treatment options. These underserved subgroups are at greater risk for disease progression, and suboptimal survival and are in need of more effective treatment options. Moving on to an update on U.S. launch preparation. With our PDUFA date just about three and a half months away, we have completed multiple critical launch readiness activities and plan to be ready to launch a metal start in the U.S. market when it is ready. Long lead time activities, such as securing our global trademark on our brand name and manufacturing of commercial supply, are now complete. In preparation for launch, we have also finalized our distribution network and our patient support provider.
Moving on to an update on U S launch preparations.
Without the new partners, just about three and a half months away. We have completed multiple critical launch readiness activities and plans that will lead to long term. It does start in the U S market upon potential approval.
Long lead time activities, such as securing our global trademark our brand name manufacturing of commercial supply are now complete.
In preparation of launch we have also finalized our distribution network and our patient support providers.
In addition, we have on boarded and fully integrated a highly experienced commercial and medical affairs team and together.
We continue grow foundation geron towards a commercial company.
Aneel: In addition, we have onboarded and fully integrated a highly experienced commercial and medical affairs team into Geron. We continue to transition Geron into a commercial company with the integration and adoption of systems and processes to recognize and report revenues and the continued refinement of engagement plans with marketing, commercial access, payer, and reimbursement stakeholders. With regard to our field team, all regional business directors were onboarded in early January, and key account manager roles are being recruited. We expect to onboard the sales force in the first and second quarter of 2020. I'm very excited by the caliber of talent we have recruited into the commercial team and across the organization.
Integration and adoption of systems and processes to recognize and report revenues and the continued refinement of engagement plans with marketing commercial access and.
And reimbursement stakeholders.
With regards to our field team all regional business directors, but on voted in early January and key account manager roles are being recruited.
We expect to onboard the salesforce in the first and second quarter of 2024.
I'm very excited by the caliber of talent, we have recruited onto their commercial team and across the organization.
The cross functional launch team have deep oncology expertise.
Operational experiences and they have been part of multiple oncology launches.
Michelle: Our cross-functional launch teams have deep oncology expertise and operational experience, and they have been part of multiple oncology launches. We are excited about the opportunity to bring this innovative therapy to patients and are confident in our readiness to launch Imitalstat in the U.S. market upon potential FDA approval.
We are excited about the opportunity to bring this innovative therapy to patients and are confident in our readiness to long term. It does start in the U S market upon potential FDA approval.
With that I'll now pass the call over to Michele for a financial update Michelle.
Thanks, Neil and good morning, everyone.
<unk> Q4, and full year 2023 financials. Please refer to the press release, we issued this morning, which is available on our website.
Michelle: Thanks, Anil, and good morning, everyone. For detailed Q4 and full year 2023 financials, please refer to the press release we issued this morning, which is available on our website. I will now review some highlights from the quarter and full year. At the end of 2023, our cash, cash equivalents, and marketable securities were $378.1 million. There are approximately 2.5 million warrants outstanding, and the potential proceeds from these warrants are $3.2 million.
I will now review some highlights from the quarter and full year.
At the end of 2023, our cash cash equivalents in marketable securities were $378 1 million.
Approximately $2 5 million warrants outstanding and the potential proceeds from the warrant a $3 $2 million.
Total operating expenses for the three and 12 months ended December 31, 2023, or $54 3 million and $194 1 million, respectively, compared to $42 million and $139 1 million for the comparable 2022 periods.
Michelle: Total operating expenses for the 3 and 12 months ended December 31, 2023, were $54.3 million and $194.1 million, respectively, compared to $42 million and $139.1 million for the comparable 2022 period. R&D expenses for the 3- and 12-months ended December 31, 2023, were $32.9 and $125 million, respectively, compared to $28.2 million and $95.5 million for the same period in 2022. Expenses have increased year-over-year, primarily related to supporting our Phase 3 clinical trials, iMERGE and IMPACT-MF. Both personnel and consulting costs have increased to support regulatory submissions and increased investment in manufacturing as we prepare for the potential U.S. commercialization of Imitelstat in transfusion-dependent, lower-risk MDS. G&A expenses were $21.4 million and $69.1 million for the 3 and 12 months ended December 31, 2023, compared to $13.8 million and $43.6 million for the same period in 2022.
R&D expenses for the three and 12 months ended December 31, 2023, with $32 nine and $125 million, respectively, compared to $28 2 million and $95 5 million for the same period in 2022.
Spencers of increase year over year, primarily related to supporting our phase III clinical trials I emerged and impact MF.
Both personnel and consulting costs increased to support regulatory submissions and increased investment in manufacturing as we prepare for the potential U S commercialization of <unk> in transfusion dependent lower risk Mds.
G&A expenses were $21 4 million and $69 1 million for the three and 12 months ended December 31, 2023, compared to $13 8 million and $43 6 million for the same periods in 2022.
The increase in G&A expenses is primarily attributed to headcount and external expenses to support the commercial readiness activities.
At the end of December 31, 2023, the company had 141 employees, which we project will grow to approximately 270 employees by the end of 2024 subject to receiving FDA approval and then they tell us that.
The increase in head count is primarily in the commercial and medical Affairs team.
Our projected full year 2024 operating expenses are expected to be between $270 million to $280 million.
Based on our current operating plan and expectations regarding the timing of a potential approval of our <unk> NDA that is currently under FDA review and subsequent potential U S. Commercial launch we believe that our current cash resources together with projected revenue from U S sales of Intelsat proceeds from the exercise of outstanding warrants.
Michelle: The increase in G&A expense is primarily attributed to headcount and external expenses to support the commercial readiness activities. At the end of December 31st, 2023, the company had 141 employees, which we project will grow to approximately 270 employees by the end of 2024, subject to receiving FDA approval under Telstat. The increase in head count is primarily in the commercial and medical affairs teams. Our projected full year 2024 operating expenses are expected to be between $270 and $280 million.
And funding under our loan facility will be sufficient to support our operations into the third quarter of 2025.
I will now turn the call back over to chip chip.
Kim.
Thanks, Michelle <unk>, our progress in 2023 represented the culmination of a many year multifaceted scientific and drug development journey. The goal of which is to translate the promise of telomerase inhibition into a potentially powerful medicine.
Today, we're just three five months away from the <unk> date for our first in class <unk> inhibitor, which we believe has the potential to offer important and potentially life changing treatment option to patients with transfusion dependent lower risk Mds.
Chip: Based on our current operating plans and expectations regarding the timing of a potential approval of our Immetelstat NDA, which is currently under FDA review, and subsequent potential U.S. commercial launch, we believe that our current cash resources, together with projected revenues from U.S. sales of Immetelstat, proceeds from the exercise of outstanding warrants, and funding under our loan facility will be sufficient to support our operations into the third quarter of 2025. I will now turn the call back over to Chip. Thanks, Michelle.
We believe this is a robust commercial opportunity and we are on track for a successful transition to becoming a commercial company.
In addition, we're excited by the momentum in our phase III impact MF trial, which is 50% enrolled as of November 2023, and for which we expect an interim analysis in the first half of 2025.
We believe these programs carry significant value for patients and shareholders alike, and we look forward to keeping you updated on our progress.
Operator: For Geron, our progress in 2023 represented the culmination of a many-year, multifaceted scientific and drug development journey, the goal of which was to translate the promise of Tuamari's inhibition into a potentially powerful meta. Today, we're just 3.5 months away from the PDUFA date for our first-in-class telomerase inhibitor, which we believe has the potential to offer important and potentially life-changing treatment options to patients with trans We believe this is a robust commercial opportunity, and we're on track for a successful transition to becoming a commercial company. In addition, we're excited by the momentum in our Phase 3 IMPACT-MF trial, which is 50% enrolled as of November 2023, and for which we expect an interim analysis in the first half of 2025. We believe these programs carry significant value for patients and shareholders alike, and we look forward to keeping you updated on our progress. Now, we open the line to questions. Operator?
We will now open the line to questions operator.
At this time I would like to remind everyone in order to ask a question Press Star then the number one on your telephone keypad. Your first question comes from the line of chlorine Johnson from Goldman Sachs. Your line is open.
Hey, good morning, maybe one from us and a follow up to that how would you characterize the likelihood that you could see intelsat approved with either black.
Black box warning and or a Rems program and then perhaps more importantly, what do you view as the implications from a commercial perspective under either scenario.
Well a lot of.
<unk>, let me say first of all.
You're going to hear this.
Okay, everybody well.
I'll just take one so lumpy.
Ongoing conversations with regulators.
And to be opportunistic within very close all the regionals.
Let me just say I don't think its warranty I don't think so.
Operator: At this time, I would like to remind everyone in order to ask a question, press star then the number one on your telephone keypad. Your first question comes from the line of Corinne Johnson from Goldman Sachs. Your line is open. Good morning.
Arkansas.
Either a black box warning.
Maybe one quick one.
Okay.
See how all of that.
What's that.
Thank you.
Great.
We installed in Q.
T.
Chet: Maybe one from us and a follow-up to it. How would you characterize the likelihood that you could see a Mattel set approved with either a black box warning or a RIMS program, and then, perhaps more importantly, what do you view as the implications from a commercial perspective under either scenario? Yeah, a lot of deposition here. Let me say, first of all, you're going to hear this over and over. Everybody will
<unk>.
Please.
Yes.
The population.
Okay. Thanks.
Yes.
Last question.
And if commercial does.
The commercial implications if it was implemented.
Yeah.
John.
Asian people stay out of that.
Chet: So I'll just say once again that we're not going to comment on ongoing conversations with regulators and the opportunities that we're getting very close to all of these milestone events. But let me just say, I don't think it's warranted. I don't think that, from our perspective, either a black box or any part of the wellness program is warranted, but predicting how all of that will go is a little too subjective for us. But we do feel very comfortable with the ability to be used faithfully and effectively in this target population. Okay, thanks. I'm sorry.
Everybody understands that.
Sure.
Either black box warnings of content indication fees at all nuanced ease at all.
Nuance.
One our foods operation.
For Microsoft.
Hello.
But overall, we're focus forward all beyond those hopes of ball.
Sure.
Okay.
Okay. Thank you.
Yes.
Chet: Yeah, the first and second part of that question; I didn't catch it. It's a commercial, the commercial implications if it was to be implemented. Then there's speculation. I think I'll stay out of that speculation. I think everybody understands that it all depends on the, certainly when it comes to either black boxes or warnings or contraindications, these are all nuanced, these are all nuanced issues. Again, I'll refrain from speculation. It's never been done, from my perspective, but again, it kind of depends on how that gets put together.
Your next question comes from the line of <unk> Patel from B Riley Your line is open.
Yeah, Hey, good morning, and thanks for taking the question.
There was a letter submitted to be Oh that members by an independent group regarding the safety for Intelsat.
The conclusion on that letter was positive overall for Intelsat.
But I guess the question is have you requested any other independent assessments for maybe additional topics.
It is evaluating the safety of <unk>.
Operator: But that's not where we're at. We're focused forward beyond those types of issues we have. Okay, thank you. SPEAKER 2 What do you do in the? Your next question comes from the line of Kalpit Patel from B. Reilly. Your line is open.
<unk> is from all of the studies before the AD com.
Well.
Quickly I will qualify for those who aren't aware of it.
<unk> model.
Model.
<unk>.
The members of our board.
Chet: Yeah, hey, good morning, and thanks for taking the question. There was a letter submitted to the ODEP members by an independent group regarding the hepatic safety of Imetelstat. The conclusion in that letter was positive overall for Imetelstat. But I guess the question is, have you requested any other independent assessments for maybe additional topics such as evaluating the safety of the cytopenias from all the studies before the outcome? Well, I think we have to clarify for those who aren't aware of it, that letter was sent by the members of our hepatic safety or hepatic evaluation committee, the support agency. That group of little experts was convened, gosh, almost a decade ago, so very, very well known to many people in the industry for drug-induced liver injury, et cetera.
Got it.
Thank you.
Valuation.
For the agency.
Grupo <unk> will.
<unk>.
Almost a decade ago.
The overall well see all these.
We believe we will see.
Absolutely.
We're going to have your thoughtful on.
We engaged <unk> to oversee all of the helpful thoughts going forward.
So they did a wonderful job.
Quarterly meetings.
Scrap flows inflammation.
Around the world.
While this sort of information that relates to in any way shape perform too.
Liberal part is.
Public safety.
The level of <unk> for people, who need it simply reaffirms our belief.
Chet: And we engaged them to oversee all of the Indian Health Act files going forward. And I have to say, they did a wonderful job. We had quarterly meetings. They looked at every scrap of information in case it's hard to download. They looked at every scrap of information that relates in any way, shape, or form to liver hepatotoxicity or liver safety.
Oh.
<unk>.
The voice.
So that's really where it came from.
Yes.
I think I'll just leave it to Paul.
Bob.
Paul.
Well coupons.
Paul.
Chet: So let us know in the dockets for people to read it. It simply reaffirms their beliefs that this was the very first drug team that was suspected. So that's really where it came from.
Our world.
Looking for especially.
So.
Okay. Thanks for taking the question.
Okay.
Your next question comes from the line of Stephen Willey from Stifel. Your line is open.
Chet: I think we'll just leave it at that. That is kind of a well-confined area of interest that many people are aware of, especially decades ago. Okay, thanks for taking the question. Our next question comes from a line from Steve and Willie from Stiefel. Your line is open.
Yes, thanks for taking the questions.
I guess Ken.
Again, I'm not sure if you'll be able to speak to this but can.
Can you remind us just what the frequency of platelet and cell count monitoring in the emerge trial was and I guess.
Chet: Yeah, thanks for taking the questions. I guess can, Again, I'm not sure if you'll be able to speak to this, but can you remind us just what the frequency of platelet and cell count monitoring in the iMERGE trial was? As you think about a potential product label, would you expect that monitoring requirement to be a bit more stringent than what it was in the trial? I think it's agriculture that was weakly monitored for several cycles, and I think whatever was in the trial is not unlikely to be looked at by regulators as sort of a de facto standard. I won't comment on labeling per se, you know where that will go exactly, but I do think that that makes sense and it's given us the ability to really interrogate the frequency and the sort of course of these cytokines, which, as you know, are indeed short-lived and well-managed. Manageable, I guess I should say.
As you think about the potential.
Product label would you expect that monitoring requirement to be a bit more stringent than what it was in the trial.
Hum.
I think if I recall what was equally.
Our model.
Several cycles.
<unk>.
Thank whatever was in the trial.
Unlikely.
To be looked at by regulator results.
Sandy I won't comment on late this fall.
Where that will go exactly.
<unk>.
That makes sense.
Given us the ability to deal with.
The frequency.
So sort of of course you saw.
<unk>.
Nemo.
Sure.
Launched knowledgeable on what we can say.
Chet: But we only know that because we studied them in that way. So I think that's the de facto standard and what I would expect going forward. Okay. And do you know, I guess, what proportion of these patients, I mean, obviously, they're low-risk MDS and have dysplastic marrow, but do you know what proportion of them are already doing weekly cell counts, whether it's at home, whether it's in a heme office? Well, I don't know the answer to that. It's an interesting question, though.
But normally because southern warmer mothballed, so it's impossible to talk to us.
I would say.
Going forward.
Oh.
Thank you.
Yourself.
Okay.
No I guess, what proportion of these patients I mean, obviously, they are low risk Mds and dysplastic marrow, but do you know what proportion of them are already doing weekly sell accounts, whether it's at home whether it's in the home office.
Well, we need to develop into some questions.
I would say that.
Chet: I would say that it kind of depends on where they are in their journey, and as you say, kind of what the level of their neurodysplasia is and so forth, and what their own experiences are. As you saw in the placebo one, we did have patients who simply also showed meaningful cytokine use even though they were receiving placebo and whatever other background therapy was allowed. So, but on the other hand, I think the regular monitoring probably is more associated with different products that these patients take that are no longer associated with cytokine as well as endocarditis. So my guess is that this would be something that would get started when you start a new drug. Most psychologists would follow very carefully, because it just kind of makes sense. And then, ultimately, it will be up to the physician on whatever the labeling says to decide how often and how that sort of spreads out over the course of a patient's treatment with any drug you include. Okay, maybe this is just a kind of bigger picture question.
Well.
We're holding our own journey and as you say.
What's the level of bull.
No disposal Wilson.
What their own expenses, which you saw on the placebo arm.
<unk>.
We want patients to.
And simply also solid meaningful outside opinions.
Paul.
They're receiving placebo.
Both of these law.
But on longhorn outcome from resolute.
But the regular momentum probably assume associated with Stifel.
Okay.
So that will no longer be associated.
Both.
So my guess is that this would be something solid.
When you say, we need to all most all Paul's would follow those helpful. Just kind of all makes sense.
Ultimately will be up to boardwalk will be globally falls off too.
The solid.
Hi.
At that point.
So.
Amit.
<unk>.
Okay, and then maybe just kind of a bigger picture question I mean theres obviously.
Chet: I mean, there's obviously, you know, kind of this ongoing narrative amongst investors regarding the exclusivity runway and, you know, the development program for Imitalstat here is still not necessarily expanding, but it's being broadened into some additional indications. And so I guess, you know, I'd like to know, do these Phase I studies reflect your internal confidence in the IP, or do you think that these are opportunities you could bring online quickly, maybe carve out some additional orphan drug designations? We'd just be kind of interested in terms of how you're thinking about the longer term R&D program for this, and others. So I think we were referring just to be specific to inclusion law and the AML study. Did I get your question right about that?
This ongoing narrative amongst investors regarding the exclusivity runway and.
The development program for <unk> that here is still.
Not necessarily expanding but it's being brought in into some additional indications and so.
Just curious I guess does.
Do these phase one studies reflect.
Confidence in the IP or do you think that these are opportunities you can bring online quickly maybe carve out some additional orphan drug designations would just be kind of interested in terms of.
How you are thinking about.
The longer term R&D program for this drug.
Sure.
So I think we're.
So just to be.
<unk> two <unk>.
<unk>.
On the AML studies.
Okay.
Chet: Yeah, correct. So the RUX combo study and just some of the others. Yeah, that are on Facebook.
Yes, correct.
So the rux combo study in just some of the other <unk>.
Thanks Mark.
Uh huh.
Chet: So I think it's incumbent on every company developing a drug, regardless of whatever the nuances of the IP positions are. I think we study new combinations, we study different utilities of drugs, and sorry for being a little preachy about this; I don't mean to be preachy. I think we do that on the basis that if we think that there is benefit to be derived for patients, that's when we become really interested. The Rupp chain is a really good example, as you know and have written about.
Sure.
Ultimately humbling.
Every company.
Regardless.
Oliver will nuances.
These are awesome.
In semi.
New combination studies.
<unk>, sorry for being long <unk>.
But perhaps a little bit on the high level.
Meanwhile, on the places where we think.
But also it can be derived.
Patients will.
We'd be comfortable with.
Some of them some room.
For example.
No.
Sure.
Mark.
Chet: There were a whole series of preclinical experiments done a number of years ago now, and they were in a really good lab of scientists that looked at the question of whether or not there were different ways to exploit some of the intrusive activity of the primary stimulus against some of the cells that are relatively transformed and ultimately cause malfibrosis and clinical outcomes. And what we've seen was that if you end up treating with, and that clinical disrupts also, as I recall, but, you know, with the JAK inhibitors, you see damage. And then if you sort of chase that, if you come after that, which you can do in orthopedics fairly easily with autonomous inhibitor muscles in the top shot, you actually get a higher proportion of animals surviving longer.
<unk> preclinical at Swan song on.
A number of municipal law.
One big one.
Rob.
Awesome.
We work with the question of whether along with different ways to slight intrinsic activity at this moment receivables we've done.
Some of the south.
Hum.
Currently.
Cosmo myelofibrosis.
Outcomes on what we've seen most of it should be.
Bob <unk> will.
But what's also what we call the Jackie.
Launch is.
Sort of choice.
Now would you.
Leather goods.
We will apply.
On a possible you also get a higher proportion of animals.
I do want to point also.
Chet: And also, you see a depletion of the living hematopoietic stem cells. So, that was the key for us to start, ultimately, doing all the work necessary to start this study. And it's because, A, we can go up front with this and to front-line patients. And B, we hope to be able to actually have, ultimately, improved efficacy, although this study is predominantly a safety study. I guess that some people are going to do this. If we don't do it, some people somewhere in the world will start to do it on their own.
You'll see a depletion.
The logo.
So.
Bob.
Key for us too so ultimately we do all the work and also save itself right.
We can grow upfronts.
So frontline patients.
We hope to be able to actually have ultimately.
Yes.
<unk> is predominantly.
Yeah.
C J.
Some people are going to do this if we want to let some people somewhere in the world. So if you do it on loan we would.
Chet: And we would think we know the drug, we've touched it better than anybody else, and we're not going to control that. So, sorry to be, again, kind of preachy about it, but I think that's the rationale. The same with AML.
We noted above.
Anybody else.
All of that so sorry to be reaching a bottom.
The thoughts there Rob.
Small <unk> emails a terrible disease.
Chet: AML is a terrible disease, and relapsing clostrid patients have very, very few options. There are other drugs on the horizon that I hope for these patients. They are also being experimented with in this country. But our goal is really the patient. And then, usually, if you do something reasonably innovative, which I think these are innovative, ultimately, you file for actual property protection.
You will have some cost completions of Borgwarner two options.
There are other drugs, one new homes a month.
I would hope for these patients also.
Thank you Brenda.
Paul.
Our goal is really patients we want.
Soon.
We reasonably intermodal volume.
Ultimately.
File.
Absolutely.
Chet: That plays out as a completely separate story in one of those two categories. Okay, makes sense. I appreciate the comments. Thanks. Thank you all. Again, if you'd like to ask a question, press star 1 on your telephone keypad. Your next question comes from the line of Gil Blum from Needham & Company. Your line is open. Yeah, hi, this is Ian Markowski on behalf of Gil.
Novelty, including software.
Right.
<unk>.
Okay. It makes sense I appreciate the comments thanks.
Thanks.
Again, if you'd like to ask a question press star one on your telephone keypad. Your next question comes from the line of Gil Blum from Needham <unk> Company. Your line is open.
Yes, Hi, this is Ian Murkowski Entre Gil Thank you for taking our question.
Operator: Thank you for taking our questions. The first one would be, how much extra effort do you think you'll have to spend on educating community physicians on managing cytopenias versus academic ones, assuming approval? And then, do you have any thoughts on how the recent data from morphosis at ASH could impact the myelofibrosis landscape and any future developments in the space? Thank you. I think there's a killer for Anil Kapoor. Anil?
First one would be how much I know you've touched on this before but how much extra effort do you think you'll have to spend on educating community physicians on managing cytopenia is versus academic ones assuming approval and then do you have any thoughts on how the recent data from morphosis at Ash could impact.
The myelofibrosis landscape and any future development at this stage. Thank you.
I think this is tailor made for annual Kapoor.
Sure.
Anil Kapoor: Sure. We have had extensive discussions with both community and academic providers. And just as a reminder for everyone, low-risk MDS is predominantly treated in the community setting. Physicians tell us they have deep experience with drugs such as LEN and HMAs and are very adept at managing heme-related side effects for patients. And for them, they point to prolonged myelosuppression with HMAs, et cetera, as areas which they have managed successfully for a long period of time. With Imitalstat in particular, what is really important, which they receive really favorably, is the predictability of the cytopenias, the fact that the cytopenias typically are of finite duration and tend to go away by cycles two and cycles three. And also importantly, there are no clinical consequences associated with these cytopenias in terms of infections and hospitalizations, and bleeding events.
So we have had extensive discussions with both community and academic providers and just as a reminder for everyone noticed MBS is predominantly treated within the community setting.
Physicians tell us they have beef experiences with drugs, such as Len HMH and are very adept at managing.
Heme related.
Side effects for patients and for them they point to prolong myeloid suppression with SMA is et cetera.
Areas, which they have managed successfully for a long period of time.
<unk> will start in particular, what is really important base, which made us. He's really favorably is the predictability of decitabine, yes. The fact that the cytopenia typically out of finite duration and tend to go away.
By cycles through and cycled three and also importantly, there are no clinical consequences associated with these type of thing is in terms of infections and hospitalizations.
Bleeding events.
Anil Kapoor: And when we also provide context on the mechanism of action, this is something that's received very favorably by the practicing hematologist. So we do not see many differences between the community versus the academic hematologist in terms of their perceptions and management of hemotoxicities. They are adept with their training and also their practice.
And then we also provide context on the mechanism of action.
This is something that <unk> received very favorably by the practicing hematologist. So we do not see much differentiation between the community versus academic Hematologist in terms of their perceptions and management of <unk> toxicities. They added after that training and also their practice experiences.
Anil Kapoor: With regard to the question on myelofibrosis, I think this is also an area which is fast moving. We will obviously wait to see what happens with the BCL2 and the BET inhibitors in terms of entering the landscape, but a reminder for myelofibrosis is that we are the only Phase 3 study, the largest effort to the best of my knowledge in the world, looking at survival in a patient population that's relapsed and refractory to Jack Eye. This patient population is extremely underserved.
With regards to the question on Myelofibrosis I think this is also an area which is fast moving.
We will obviously wait to see.
What happens <unk>.
The Bcl two and the bet inhibitors in terms of entering the landscape.
But a reminder forward myelofibrosis is that we are the only.
The phase III study the largest export to the best of my knowledge.
The word which is looking at survival in a patient population, that's relapsed and refractory to JAK.
<unk>. This patient population is extremely underserved and every feed.
Anil Kapoor: And every feedback from physicians, academic experts, leading KOLs is that if our myelofibrosis study is positive, it is going to be transformative for patients with myelofibrosis and really will be rapidly well adopted within the space. So I'll just stop here. Thank you. Your next question comes from the line of Joel B. from Baird. Your line is open.
Feedback from physicians academic experts, leading kols is that.
If our myelofibrosis study is positive it is going to be transformative for patients with myelofibrosis and really will be rapidly adopted within the space. So I'll just stop here.
Thank you.
Your next question comes from the line of Joel <unk> from Baird. Your line is open.
Operator: Hi, thanks for taking the questions. For the Early Access Program, can you provide any updates or quantify how many patients are a part of it? Achyut, do you want me to take that?
Hi, Thanks for taking the questions for the early access program to provide any updates or quantify how many patients are a part of it.
Jim do you want me to take that.
Sorry go ahead.
Anil Kapoor: So that's a clinical study, Joel. We do not provide updates on the enrollment, et cetera, projections within that space, but the study is ongoing.
So thats a clinical study to oil we do not provide updates.
On.
The enrollment et cetera projections within that space.
Anil Kapoor: Got it. Thanks. And then for the guidance of the cash runway into Q3 2025, what sales assumptions are going into that guidance?
<unk> ongoing.
Got it thanks and then.
For the guidance of the cash runway into Q3, 2025 sales assumptions are going into that guidance.
Michelle: Oh, sorry Joel, I was on mute. Um, we haven't cleared any... Oops, sorry. We haven't shared any sales guidance or revenue guidance just yet.
Oh, sorry, Joe.
We haven't made any bones.
Oh, sorry, we havent shared any sales guidance on our revenue guidance just yet.
Okay.
Aaron Feingold: Thank you. And we have reached the end of our question and answer period. I will now turn the call back over to Aaron Feingold for some final closing remarks. Thank you everyone so much for joining us today. We appreciate your interest in Geron and look forward to keeping you updated. Be well. This concludes today's conference call. Thank you for your participation. You may now disconnect.
Got it thank you.
And we have reached the end of our question and answer period I will now turn the call back over to Aaron <unk> for some final closing remarks.
Thank you everyone. So much for joining us today, we appreciate your interest in Huron and look forward to keeping you updated.
This concludes today's conference call. Thank you for your participation you may now disconnect.
Okay.
Yeah.
Yeah.
[music].
Yeah.
Okay.