Geron Q2 2023 Earnings Call Transcript

Quartr
Provided by Quartr
August 3, 2023

There are 10 speakers on the call.

Operator

Afternoon, everyone. Welcome to the Geron Corporation Second Quarter 2023 Earnings Conference Call. I am Erin Feingold, Geron's Vice President of Investor Relations and Corporate Communications. I'm joined today by several members of Geron's management team: Doctor. John Scarlett, Chairman and Chief Executive Officer Olivia Bloom, Executive Vice President and Chief Financial Officer Doctor.

Operator

Feiseller, Executive Vice President and Chief Medical Officer Anil Kapoor, Executive Vice President of Corporate Strategy and Chief Commercial Officer and Doctor. Andrew Greflein, 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 imetelstat, anticipated clinical and commercial events and related timelines, the sufficiency of Geron's financial resources and other statements that are not historical fact, Actual events or results could differ materially. Therefore, I refer you to the discussion under the heading Risk Factors in Geron's quarterly report on Form 10 Q for the quarter ended June 30, 2023, which identifies important factors that could cause actual results to differ materially from those contained in the forward looking statements. Geron undertakes no duty or obligation to update our forward looking statements.

Operator

With that, I'll turn the call over to Chip. Chip?

Speaker 1

Thanks, Aaron. Good afternoon, everyone. Thanks for joining us today. Turing's most recent quarter was punctuated by important achievements, which support our evolution from a late stage clinical development company Toward 1 with future substantial commercial capabilities. Foremost among these achievements was the submission in mid June Of a new drug application for imetelstat in lower risk MDS.

Speaker 1

This was the first NDA ever submitted for telomerase inhibitor And reflects our team's dedication, commitment and focus on groundbreaking and innovative drug development over many years. Other important milestones included presentations at both ASCO and EHA of new data and analyses from the IMerge Phase 3 lower risk MDS trial. These data contributed to the evidence for compelling imetelstat efficacy profile, including durable, continuous transfusion independence, As well as substantial increases in serum inulin. Broad responses across MDS subtypes, including in ring seroplast positive and negative patients as well as in high transfusion burdened patients were also reported. Further, the presentations reflected strong evidence for potential disease modifying activity as well as patient reported outcomes of improved fatigue And imetelstat treated patients.

Speaker 1

Faye will comment in more detail on these clinical data updates later on this call. Collectively, these data distinguish imetelstat from other treatments for patients with lower risk MDS that are available commercially Or that are in development today. Based on our market research, including perspectives gained from both academic and community hematologists, We believe the broad hematology community considers Imetelstat an important potential treatment option for patients with lower risk MDS. Our market insights show that Imetelstat is positioned to become a new standard of care in lower risk MDS, particularly for difficult to treat subgroups who have very limited options today. As a result, we believe the total addressable market and lower risk MDS for Imetelstat is approximately $3,500,000,000 In 2,033.

Speaker 1

In order to execute on that large of an opportunity, we're in a full court press to push forward our launch readiness and expect to be ready for a U. S. Commercial launch upon potential approval in early 2024. Aneel will reprise The latest Imetelstat market research and lower risk MDS and launch readiness update in more detail later on this call today. Another important element of Geron's value proposition is IMPACT MF, our pivotal trial evaluating imetelstat and myelofibrosis patients For relapsed or refractory to JAK inhibitors.

Speaker 1

This is the only Phase 3 clinical trial in myelofibrosis using overall survival as a primary endpoint. We believe a positive outcome could transform the treatment landscape for these MF patients who have limited treatment options and a dismal survival outlook. Faye will be providing further detail on enrollment in the IMPACT MF trial later on the call. Based on our current planning assumptions For both enrollment and death rates in the trial, today we're updating our guidance for the interim analysis to be expected in the first half of twenty twenty five And for the final analysis to be expected in the first half of twenty twenty six. From a financial resource perspective, We have approximately $400,000,000 in the balance sheet as of the 2nd quarter close.

Speaker 1

This gives us the financial wherewithal to find potential Successful launch in lower risk MDS and also to support operations through the 1st year of launch. Olivia will comment during the call on the status of warrant exercises and our expectations regarding our cash runway based on these current financial resources. Before I turn this call over to Faye, I'm very pleased to announce Today, the appointment of Scott Samuels as Geron's new Chief Legal Officer following Stephen Rosenfield's retirement at the beginning of this month. Stevenson, the Chief Legal Officer of Geron since shortly after I came to the company more than a decade ago, and both I and the Board are deeply grateful for Steven's partnership and contributions to Geron since then. I'm personally very pleased for Steven that he'll now be able to enjoy his much deserved retirement.

Speaker 1

Prior to joining Geron as our new Executive Vice President, Chief Legal Officer and Corporate Secretary, Scott Samuels recently served as the General Counsel of Beijing, We built a large global legal and compliance team, oversaw launches of 3 internally developed drug products in the U. S, Europe and China, Developed a global healthcare compliance program and led key strategic transactions with Amgen, Novartis and Celgene, which of course is now BMS. Prior to BeiGene, Scott was the Assistant General Counsel and then Acting General Counsel at ARIAD, where he managed the company's legal affairs, including SEC compliance and corporate governance and key licensing and distribution agreements prior to ARIAD's acquisition by Takeda. I believe Scott's experience, technical expertise and history of success in building legal and compliance organizations to meet the needs of a commercial company It epitomizes what our current and our many new employees are focused on as we pursue the future evolution and growth of Geron. Both the Board and I greatly look forward to working with Scott.

Speaker 1

With that, I'll turn the call over to Faye for a regulatory and clinical development update. Faye?

Speaker 2

Thank you, Chip, and good afternoon to everyone on the call. As Chip mentioned, we are thrilled to have submitted our Imetelstat new drug application in June 2023 for the treatment of transfusion dependent anemia in adult patients with low to intermediate one risk MDS who have failed to respond or have lost response to or ineligible for erythropoiesis stimulating agents or ESAs. As permitted under Imetelstat's Fast Track Designation. We have requested that the FDA grant priority review of the NDA. We expect FDA will communicate potential acceptance of the NDA within 60 days of submission, that is sometime in mid August, and reveal the PDUFA date for such a review.

Speaker 2

Under a priority review scenario, we would expect potential NDA approval timing in the Q1 of next year. Under standard review, we would expect potential approval timing in the Q2 of 2024. To expand Imetelstat's potential reach outside of the U. S, We remain on track to submit a marketing authorization application in the European Union for lower risk MDS in the Q4 of 2023. As we await potential commercialization in the U.

Speaker 2

S, we initiated an expanded access program or EAP in June 2023. This is a program that enables us to make imetelstat available to clinicians and patients prior to FDA approval. Treatment of lower risk MDS patients in this program is based on a protocol approved by FDA, which requires each treating physician to apply for access for their patients. We have heard physicians in both academic and community settings express the need for new treatment options for their lower risk MDS patients, and we are pleased to be able to offer this expanded access program to the low risk MDF community. Patients treated with imetelstat and the expanded access program are expected to ultimately be transitioned to commercial supply within 3 months for potential future FDA approval of the drug.

Speaker 2

As Chip described, at ASCO and EHA, we presented new data and analyses from IMerge. These data further differentiate Imetelstat from existing treatments and support its role as a potential new standard of care in lower risk MDS. The content of these presentations were also reported on June 14 during the Geron hosted investor event. We encourage investors to access the archived webcast, which is available on the Investor portion of our website under Events. There you can hear hematologic malignancy key opinion leaders and EMERGE investigators, doctors Uvett Plotsbecker and Rami Kumrokji present these data in detail.

Speaker 2

For the purposes of our call today, I will provide an overview of the key points of Imetelstat differentiation highlighted in our ASCO and EHA presentations. First, the clinically meaningful and durable transfusion independence or TI experienced by metallstat treated patients in IMerge is unprecedented for patients with lower risk MDS. We observed a highly statistically significant improvement in TI rates in Imetelstat treated patients for 8 week, 16 week and 24 week PIs compared with placebo. Even more exciting with 3 months of additional follow-up, nearly 20% of Imetelstat treated patients experienced a 1 year TI, which represents approximately 60% of Imetelstat 24 week responders. Additionally, hemalignancy KOLs at APTO and EHA noted the statistically significant improvement of anemia with a median hemoglobin rise of 3.6 grams per deciliter for Imetelstat treated 8 week TI responders as a very important point of differentiation.

Speaker 2

2nd, a breadth of clinically meaningful responses was observed across key MDS subgroups, including difficult to treat populations such as those without ring sideroblasts or RS negative patients, as well as high transfusion burden patients and those with high serum EPO levels. These patient populations have not been studied with most other agents used to treat the anemia of lower risk MDS, nor have such results been seen with other treatments. At EHA, we presented important new subgroup analysis showing that the rate and durability of TI 24 week TI responders is similar across key lower risk MDS subgroups regardless of ring sitteroblast status, prior transfusion burden, IPSS risk category for baseline serum EPO levels. 3rd, we have presented robust evidence of Imetelstat's potential to alter the underlying biology of lower risk MDS by reducing or eliminating molybdenum clones. In Imetelstat treated patients, we saw a reduction in mutation burden as measured by variant allele frequency or VAS.

Speaker 2

Furthermore, new data on cytogenetic responses and reductions in bone marrow RS cells reported imetelstat's mechanism of action. In totality, these data indicate that imetelstat may have disease modifying potential in patients with lower risk MDS. 4th, data on patient reported outcomes presented at EHA were also very encouraging. These data describe a sustained meaningful improvement in fatigue for imetal fat treated patients versus placebo. This specific patient reported outcome is of particular importance because lower risk MDS patients experience fatigue that is not fully alleviated by red blood cell transfusions.

Speaker 2

Additionally, many of the current treatments for lower risk MDS are associated with an increase in fatigue. Imetelstat is the first treatment we are aware of to show an improvement in patient reported fatigue in lower risk MDS patients. In EMERGE Phase 3 and consistent with prior clinical experience, Grade 3, 4 thrombocytopenias and neutropenias were the most frequently reported adverse events. Unlike several of the treatments in hematologist armamentarium, Such as HMA is melanalidomide, which may cause prolonged myelosuppression, the severe cytopenias associated with imetelstat were short lived, resolving to grade 2 or lower in less than 4 weeks in most cases, and most importantly, only rarely resulted in severe clinical consequences such as bleeding or infection. In total, the IMerge clinical data support a profile for imetelstat that if approved, we believe will serve as a very impactful option for the treatment of transfusion dependent anemia in lower risk MDS patients.

Speaker 2

Next, I'd like to discuss IMPACT MS, our 2nd Phase III trial of imetelstat in patients with JAK inhibitor relapsed refractory myelofibrosis. Today, treatment in myelofibrosis is dominated by JAK inhibitors or therapies with other mechanisms of action in combination with JAK inhibitors. The currently available therapies have been approved based on their Ability to improve symptoms and reduce splenomegaly. Approximately 75% of patients discontinue JAK inhibitor after 5 years and once they do so, they face a dismal overall survival of approximately 11 months to 16 months. We believe that imetelstat could be transformational for these patients.

Speaker 2

In the IMbark Phase 2 study in JAK inhibitor relapsed refractory in mass patients, the overall survival in imetelstat treated patients was 30 months We're nearly double compared to historical controls. Additionally, a comparison of the IMbark Phase 2 data to real world data from a closely matched cohort of patients confirmed improvement in overall survival and lower risk of death for imetelstat treated patients compared with Patients treated with best available therapy. Importantly, in Embark, there was strong evidence of the disease modifying potential of imetelstat in relapsedrefractory MF. With improvements in bone marrow fibrosis and reduction in key MF driver mutations and these reductions correlated to improved survival and other clinical outcomes. These data were the basis for the design and initiation of the IMPACT MS Phase 3 study in JAK inhibitor relapsedrefractory MF patients with overall survival as the primary endpoint.

Speaker 2

The IMPACT MF protocol calls for a planned interim analysis when approximately 35% of the planned enrolled patients have died And a final analysis when over 50% of the planned enrolled patients have died. As an OS Feingold. The timeline for the interim and final analyses depends not only on enrollment rate, but also on death rate. Today, based on achieving over 40% enrollment in IMPACT MF and our planning assumptions for enrollment and death rates in the trial, We are updating our guidance for the interim analysis to be projected in the first half of twenty twenty five and for the final analysis to be in the first half of twenty twenty six. Because these analyses are event driven and it is uncertain whether actual rates for enrollment and events Reflect current planning assumptions, the results may be available at different times than currently projected.

Speaker 2

As can be expected for a study and an indication For which there are multiple ongoing trials, constraints on clinical site personnel resources due to the COVID-nineteen pandemic and other competing trials in MF have led to some challenges in recruitment and enrollment. We are working closely with the MF community and our clinical trial sites on recruitment for the trial and continue to plan to announce when the trial is 50% enrolled. As Chip mentioned, this is the 1st and only myelofibrosis trial with overall survival as a primary endpoint. Our MS investigators remain very excited about the study and the potential of a new treatment that could improve survival for these patients who currently have few treatment options We are also pleased to report that the first patient was dosed this June in the investigator led Phase 2 IMPRESS trial that is evaluating imetelstat in patients with relapsedrefractoryacute myeloid leukemia or high risk MDS. This trial is based on preclinical publications that describe the role of telomerase in AML disease progression and which have reported that inhibiting telomerase in both mouse and human derived AML models targets and potentially depletes leukemic stem cells, thus impairing leukemic progression.

Speaker 2

Relapsedrefractory AML and higher risk MDS are high unmet need areas, and we look forward to understanding more about the potential efficacy of imetelstat in this patient population. With that, let me turn the call over to Anil to provide a commercial update. Camille.

Speaker 3

Thank you, Faye, and good afternoon, everyone. We are very pleased with the status of our commercial readiness activities and are on track to achieve launch readiness by early 2024. Faye has already described several significant efforts that support a potential Imetelstat launch, including important regulatory progress as well as unprecedented IMerge data presented at medical meetings, which deeply support Imetelstat's value proposition messaging. In particular, the PRO data reporting improvement in Pratik with the Imetelstat is an important differentiator and will be a critical element for payer interactions. This quarter, We also advanced critical work by obtaining significant insights from our market research efforts based on the latest Lower risk MDS data presented at ASCO and EHA by Geron and competitors.

Speaker 3

For a full review of this market research, We refer you to the investor event webcast that Fay mentioned earlier. For purposes of today's call, I will highlight key takeaways from that 1st, in alignment with informal feedback on the grounds at ASCO and EHA, Our latest market research from practicing academic and community hematologists across U. S. And key European markets confirmed That our IMerge Phase 3 data has been received favorably, particularly the compelling TI rates across subgroups, Reduction in transfusion burden, hemoglobin rises, meaningful durability of response, balanced with the predictable adverse event profile with manageable cytopenias. 2nd, continued hematologist feedback supports the Imetelstat opportunity Across ESA, relapsed refractory RS negative and RS positive subtypes, as well as high transfusion burden patients.

Speaker 3

3rd, these hematologists indicated that emetelstat is likely to become a new standard of care in post ESA experience and luspatercept experienced frontline patients or second line lower risk MDS patients as well as an important new option for frontline ESA ineligible patients whose serum equal level is greater than 500. All of these insights are instrumental to our understanding of Imetelstat's potential place in the market and our engagement strategy with physicians. With that, I'll turn the call over to Olivia for a full financial update. Olivia?

Speaker 2

Thanks, Anil, and thanks to everyone on the call for joining us today. Please refer to the press release we issued this afternoon, which is available on our website for detailed financial results. As expected, operating expenses were higher for the 3 6 months ended June 30, 2023 compared to the prior periods. The increase in R and D expenses for the 3 6 month periods of 2023 compared to the same period in 2022 primarily reflects higher clinical trial costs for increased activity for both Phase III trial and the Phase I trial in frontline MF, Increased personnel related costs for additional headcount, higher consulting costs to support regulatory submissions and greater imetelstat manufacturing costs in preparation for potential commercialization in the first half of twenty twenty four in lower risk MDS. The increase in general and administrative expenses for the 3 6 months ended June 30, 2023 compared to the same period in 2022 primarily reflects higher personnel related expenses for additional headcount and increased costs for new commercial preparatory activities.

Speaker 2

We continue to expect non GAAP total operating expenses of up to $210,000,000 for the full year of 2023. This financial guidance may be revised in the future upon notification from the FDA of the potential approval timing for imetelstat in low risk MDS. Turning to our financial resources. As of June 30, 2023, we had approximately $400,200,000 in cash and marketable securities. This balance includes approximately $17,800,000 in proceeds from warrant exercises that we received in the Q2 of 2023.

Speaker 2

In the first half of twenty twenty three, in total, we have received approximately $77,600,000 in warrant proceeds, which leaves approximately $32,000,000 in potential future warrant proceeds remaining to be exercised. Based on our current operating plans and our expectations regarding the timing of the submission and potential acceptance and approval of our NDA by the FDA for imetelstat in low risk MDS and the subsequent potential U. S. Commercial launch in the first half of twenty twenty four as well as the revised guidance on timing of the interim and final analyses for Impact. MF.

Speaker 2

We believe that our existing financial resources and estimated revenues will be sufficient to fund our projected operating requirements through the end of Q3 of 2025. If projected exercise proceeds of approximately $32,000,000 from remaining outstanding warrants are added to our current financial resources and estimated revenues. Then we believe such aggregated financial resources will be sufficient to fund our projected operating requirements through the end of 2025. With that, I will now hand the call back to Chip for closing remarks. Chip?

Speaker 1

Thanks, Olivia. I'd like to close by reiterating what an exciting time this is for Geron, for Imetelstat and for the MDS community. Based on our unprecedented Phase 3 data in lower risk MDS and an outpouring of enthusiasm from the medical community, we believe that imetelstat Transform the standard of care in lower risk MDS. We're also very proud to be pioneering the first study in myelofibrosis with overall survival as a primary endpoint. We believe that a potentially disease modifying treatment that improves survival Could be transformational for these patients who have failed JAK inhibitors and have limited other options.

Speaker 1

As a result, We'll stay the course with this SAGE III trial, especially given its immense potential for value creation for patients and shareholders alike. Finally, we look forward to the expected momentum in the months to come as we continue to execute on our commercial readiness plan and ultimately to a potential U. S. Launch of Imetelstat in the first half of twenty twenty four. So operator, with that, let's open the call to Q and A.

Speaker 4

Perfect. Thank you. And we do have our first person in the queue, Kalket Patel. Please go ahead.

Speaker 5

Yes. Hey, good afternoon and congrats on the NDA submission. So maybe starting with one question for Anil. For low risk MDS, assuming that we get approval here in the near future, What early launch metrics do you believe will be important to showcase and highlight the initial stages of launch?

Speaker 3

Thank you, Kulpit. I think for us, it's really important that we have Full coverage across reimbursement policies or both academic and community centers. That's really critical for a new drug at the time of launch. And it's our expectation that we are going to broadly inform the market And make sure that the policy reimbursement blocks, things that are really critical for new drugs get addressed well. We are also going to measure uptake across both academic and community channels for physicians and the type of patients that are on Imetel staff.

Speaker 3

And since these will be early days, we will obviously continue to monitor for durability of response And questions that may come up around reimbursement and ensuring broadest possible access in the very early days of launch. And from a supply chain perspective, making sure that the drug to specialty distributors is going to patients properly. We also will have our patient hub activated. And so this is

Speaker 5

Okay, perfect. And then for the IMPACT MF study, Just curious, is the enrollment rate being impacted because of any potential competition from other clinical studies? And can you maybe add additional clinical trial sites to streamline the enrollment there? It's not

Speaker 2

necessarily for these Trials is more the resources and the staffing. Given that myelofibrosis is an orphan and rare disease, there's limited, like research staff that can be devoted to studies. So I think that is part of the So that is part of the delay in enrollment. Regardless, we continue to believe in the study and the likelihood of positive readout.

Speaker 5

In frontline myelofibrosis, assuming that this study hits its endpoint, Does that in any sense impact your development strategy in myelofibrosis and post drug stations?

Operator

Sure.

Speaker 2

It doesn't necessarily It doesn't change the current status of our Phase 3 study, which is looking at patients who have failed JAK inhibitor and are not eligible for JAK inhibitor. So patients, Whether they were treated on a clinical trial or as part of standard of care in the community will still be eligible for the IMPACT MF trial. And I think Regardless of the readout form of FOCUS, there is still an unmet need for patients after they fail JAK inhibitor.

Speaker 5

Okay. Great. Thanks very much

Speaker 3

for taking the questions.

Speaker 5

Thanks, Kelvin.

Speaker 4

All right. Our next question comes from the line of Robert Driscoll. Robert, please go ahead.

Speaker 3

The feedback we have received multiple times over the last few years has been that the focus from physicians remains on the timing of cytopenias, The resolution data and more specifically on the clinical consequences, especially bleeding infections and hospitalizations. And they have also compared the Imetelstat arm to the placebo arm in the trial. Overall, we see very similar conclusions Both academic and community doctors, and we expect that the AE profile as per them is not a barrier at the time of launch. All site very extensive experiences with managing team toxicities in this setting, especially given long term familiarity And they also state that they'll obviously manage their first patients as they build So our expectation is the year, mechanistic rationale for why these psychopenia occurs, The fact that predictable and the fact there are few clinical consequences, this should be really good. And it could be our medical affairs teams, Our entire teams and we'll continue to focus on education and the linkage to the mechanism of action, so they have a clear What we expect for Emetelstat patients and insurance success.

Speaker 3

Rae, if you would like to add anything clinically from yourself.

Speaker 2

Sure. I completely agree and echo with what Emile said. And the feedback that we hear really is from hematologists. They are comfortable at managing cytopenias and understanding them and knowing what measures to take in order to support their patients. Once we explain the timing, the reversibility and most importantly, the lack of You know, prolonged cytopenias and lack of severe infections or severe bleeding, hematologist I feel quite comfortable managing these cytopenias.

Speaker 2

And also in the upcoming months, as Anil mentioned, we'll have training from Thank you.

Speaker 4

That's helpful. And then just one last one here. Just kind of wondering if there are any key differences between the value proposition for Telsted in the U. S. Versus the EU, just based on the current treatment paradigms for each region?

Speaker 3

I think let me take that yes, sir. So let me take that question, Robert, with EU first. As you can imagine, the most important thing in Europe is to establish broad based reimbursement. Each of the countries is very different. And what's really encouraging about Imetelstat and low risk MDS is our efficacy data sets are all Showing the data that payers have repeatedly asked for.

Speaker 3

This includes PRO data. It includes Feingold. 24 week PR data, which they have insisted that they would really like to see. And that is very positive news for us because the durability of PR And the PRO value proposition is very strong. So our goal there is to establish pricing In all the key markets in sequence, and I think we'll be favorably received within Europe.

Speaker 3

In U. S, again, the goal here is we have a concentrated set of our physician universe, And our aim here is to make sure that Emetelstat is launched really well and these positions have first exceeded success. In terms of commercial value, obviously, as you know, it's price independent. So the U. S.

Speaker 3

Market is Commercially more dollar value higher than the European market, but the adoption and the need for a metal stack comes Pretty equally across both U. S. And Europe.

Speaker 4

Thank you very much.

Speaker 6

What demand you're seeing for that program? And Where are the pockets of that demand coming from and how are you managing here that program ahead of the launch?

Speaker 2

Sure. I'd take that, this is Faye. So the expanded access protocol was recently opened and approved and really is a bridge for or a mechanism for patients to receive Imetelstat prior to approval and commercialization. The The way that the EAP protocol is structured is that the patient care providers and physicians place a request On an individual patient basis, any other type of clinical trial protocol. So it's still early on in Our opening of it and we don't yet have a sense

Speaker 3

of what

Speaker 2

we the kinetics of the enrollment.

Speaker 6

Okay, understood. And then maybe separately, I think I saw in the press release you expect the growth in terms of employees to be up to about 160 by the end of the year. At that point, how much additional hiring will you need to do to be prepared for the launch? Corinne, this is Olivia. I'll take a question.

Speaker 6

So that Total does not include the sales force that is going to need to be hired for launch, because that is the timing necessary.

Speaker 3

So as we have previously guided Corin on that answer, our sales force field expansion will be very PDUFA aligned, And our expectation is national competitive coverage. And as we previously said, we expect our commercial I'd like to be somewhere in the 100 to 100 and 20 full time people including sales and the commercial team supporting Across medical affairs as well as across the commercial side of it.

Speaker 7

Okay. Thank you.

Speaker 4

All right. Our next question comes from the line of Gil Blum. Gil, please go ahead.

Speaker 8

Hi, good afternoon. And thanks for taking our question. Maybe a simple one first. So, we expect News on some FDA feedback soon in August. Will we know also about an advisory committee at that point or could that take a little longer?

Speaker 1

I'll take that one, Gil. Ordinarily, you wouldn't know about an advisory committee either plus or minus at that time. The agency is usually very focused on an acceptance of the NDA for review And their lingo, the filing of the NDA, that's the one thing that we can count on. Something further about the actual PDUFA date, Assuming that it's accepted, which would give an indication of whether it was a priority or a standard review. If and when there would be an advisory committee meeting in ODAC, quite uncertain when that would be Announced or requested or posted, it can come quite late in the review cycle.

Speaker 1

It can come a little bit earlier, but there's no Specific timing for that and I don't think we would have any feel for that right now.

Speaker 8

Thank you for that clarification. Maybe a bit of a broader question, a clinical one. Is there any understanding regarding the non responders in low risk MDS? Is this just because the primary disease is too far longer? What are your insights on patients who seem to not respond?

Speaker 2

That's a great question, Gail. So you're asking about the patients who probably don't achieve TI. A lot of those patients, Though they may not achieve 100% transfusion independence, they may have reductions in transfusion burden. They may have increases in hemoglobin, improvement in symptoms or other quite possibly like intangible benefits or benefits that we're not routinely assessed on the study. So we continue to look into that and we continue to look into if there Are any predictors of patients who will achieve PI in terms of mutation or pathology or anything like that and have not yet seen anything notable distinguishing patients who achieve TI from those who don't.

Speaker 8

Okay. Thanks for taking our questions.

Speaker 3

Thanks, Aotyel. Thanks, Aotyel.

Speaker 4

Our next question comes from the line of Tully, please go ahead.

Speaker 7

Hi. This is Tully on for Heating. It's Stifel. I just have one quick question regarding IMPACT MF. So with the 40% if I remember correctly, with 40% Of patients involved in this study now.

Speaker 7

Can you guys maybe talk a little bit about how So basically, the weather the early event rate that you have observed to date is in Line with your expectation. And also, can you also give us a little bit of color on Maybe the efforts or anything that you're doing to expedite enrollment in Impact MF? Thank you.

Speaker 2

Sure. This is Faye. I'll take that question. Regarding the event rate, given that this is a blinded study, although the patients know which arm they're randomized to, we are blinded to the data and to what patients are taking when we look at the analysis as a whole. So we cannot comment on the event rate until basically either the interim or the final analysis when the study is unblinded.

Speaker 2

But just to say also that the 40% enrollment to contrast that with 35% of the enrolled patients Having died is when the interim is triggered. So and then to move to your second question about Efforts to expedite or to boost enrollment, we have had ongoing efforts since end of last year, early this year, which included site visits and investigator engagements and additional resourcing to encourage enrollment and boost enrollment. We continue to receive enthusiastic feedback from MS investigators for the study and for enrolling patients in the study. And we hope that the efforts that we've put in today will Continue to bear fruit and we'll see that helps and maintains our enrollment.

Speaker 7

That's helpful. Thank you very much.

Speaker 4

Perfect. Our next question comes from Joel Beatty. Joel, please go ahead.

Speaker 9

Hi. Thanks for taking the question. The first one is a follow-up on the previous question on impact MF. So with that guidance now being for interim readout in First half of twenty twenty five, is that timing driven solely by the enrollment rate or does it also take into consideration The event rate on a blinded basis?

Speaker 2

Thanks for the question. So the, it takes into account the Current enrollment rate and what we've seen in enrollment now that the study has been open for some time, we have a better or more of a sense now for the pace of enrollment. But the event rate is based on the initial ones and the assumptions built into the study. And at this time, we haven't recalibrated the event, right?

Speaker 9

Got it. Thanks. And then On MDS, now that ASCO has passed, can you discuss what you see as the opportunity to capture market share And RS negative patients, which is, as I understand it, the majority of the market and also where luspatercept showed a negative trend on the primary endpoint.

Speaker 1

Anil?

Speaker 3

Sure. I can take that question, Bill. So I think the data It's very clear. What we are seeing in terms of sequencing post ASCO and EHA update On all the data that came out at Clarus MDS. In RS negative patients, the strong preference for physician Remains Emmental side, especially in patients who are previously ESA treated.

Speaker 3

And that is pretty overwhelming both from U. S. Perspective as well as from European perspective, so that's very consistent. And I think that is in line with what we know previously from the PACE study And also obviously from the COMAND style in the frontline setting. So we feel that Imetelstat is likely to become the standard of care And RS negative patients, especially the ESA treated patient population, which will be the vast majority and that is Feingold.

Speaker 3

So that is good for patients and it is a validation for the data that came out from IMRC.

Speaker 9

Terrific. And one last question is on operating expenses that looked up a bit From about $40,000,000 in Q1 to about $52,000,000 now in Q2, could you help put that increase into context and Give a sense of what the trajectory and expenses might look like over the next quarter or 2?

Speaker 2

Yes, Joel, it's Solovey.

Speaker 6

I'll take the question. So as I mentioned, we're still maintaining our overall annual guidance up to dollars 210,000,000 of non GAAP expense. And as I think I've said on previous calls that I did anticipate getting towards the second half of the year, the ramp in expenses, especially not only as the more intensified commercial prep efforts, but also as we are manufacturing commercial upgrade inventory, getting ready for launch as well. And so that's where you're seeing the increase happening here in the second Quarter in comparison to the Q1 is that there were increased expenses related to manufacturing costs as well as I would say increased costs for consulting expenses as we submitted all of the regulatory filings related to the NDA and then now in the heart of getting everything ready for the MAA filing here in the Q4 of 2023.

Speaker 3

Great. Thank you. Thanks, Charles.

Speaker 4

Okay. And there are no further questions. So at this time, I would like to turn Back over to Erin Feingold.

Speaker 2

Thank you so much

Operator

for joining us today. We appreciate you taking the time to listen and participate and look forward to keeping you updated on our progress.

Speaker 4

That concludes today's call. You may disconnect.

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Earnings Conference Call
Geron Q2 2023
00:00 / 00:00