Geron Q4 2023 Earnings Call Transcript

Quartr
Provided by Quartr
February 28, 2024

There are 12 speakers on the call.

Operator

Good morning.

Speaker 1

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 4th 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 Thank you. Erin Feingold, Vice President of Investor Relations and Corporate Communications, you may begin your conference.

Speaker 2

Good morning, everyone. Welcome to the Geron Corporation 4th Quarter and Full Year 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 Michelle Robertson, Executive Vice President and Chief Financial Officer Doctor.

Speaker 2

Faye Fowler, Executive Vice President and Chief Medical Officer Anil Kapoor, Executive Vice President of Corporate Strategy and Chief Commercial Officer and Doctor. Andrew Brethlein, 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 facts. Actual events or results could differ materially.

Speaker 3

Therefore, I refer you to the discussion under the heading Risk Factors in Geron's most recent periodic report filed with the SEC, 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. With that, I'll turn the call over to Chip. Chip?

Speaker 4

Thanks, Aaron. Good morning, everyone. Thanks for joining us today. 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 imetelstat for the treatment of transfusion dependent anemia in patients with lower risk MDS and has also provided notice that it's scheduled in ODAC as part of the imetelstat NDA review to be held on March 14, 2024.

Speaker 4

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. The publications from our pivotal EMERGE Phase 3 clinical trial, including most recently in The Lancet, drove a robust response rate and an unprecedented durability of red blood cell transfusion independence with imetelstat treatment across multiple MDS patient subgroups addressing areas of high unmet need. Additional unique attributes of imetelstat include patient reported outcomes of less fatigue and significant reductions in variant allele frequency in commonly mutated MDSGs. With a well characterized safety profile of generally manageable and short lived thrombocytopenia and neutropenia, We believe the clinical evidence supporting the benefits of imetelstat is compelling and that it can become a transformational treatment option for currently underserved populations if approved by regulatory authorities.

Speaker 4

Behind our lead indication in lower risk MDS is a similarly important program, the Phase 3 IMPACT MF clinical trial in JAKI relapsed and refractory myelofibrosis. An interim analysis is expected for this study in the first half of twenty twenty five. IMPACT MF is the 1st and only MF Phase 3 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 JAKI relapsed and refractory MF who have dismal survival prognosis today. Our 2 lead indications for imetelstat represent significant commercial opportunities with a total addressable market or TAM of $3,500,000,000 for each indication across the U.

Speaker 4

S. And EU in 2,031, thus representing a combined $7,000,000,000 TAM for transfusion dependent lower risk MDS and relapsedrefractory event. Given this very substantial opportunity, amid a deep unmet need and transfusion dependent lower risk MDS, we expect to be prepared to launch and self commercialize imetelstat 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, Imetelstat and the market for our potential launch in the U. S.

Speaker 4

With the goal of ensuring broad access and reimbursement for our important medicine. Moreover, if imetelstat 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. Lastly, we ended 2023 with a strong cash position of approximately $378,000,000 which based on our current plans and expected available resources, we expect will enable us to fund a potential successful launch in transfusion dependent lower risk MDS in the U. S.

Speaker 4

And fund our planned operations into the Q3 of 2025. We believe our differentiated product candidate, the very important commercial opportunities in transfusion dependent lower risk MDS and relapsedrefractory MF, the excellence and experience of our employees and the financial resources to execute on our near term milestones

Speaker 5

puts us

Speaker 4

in a strong position for value creation. With that, I'll turn the call over to Faye for a regulatory and clinical update. Faye?

Speaker 3

Thanks, Chip, and good morning to everyone on the call. As Chip mentioned, we are deeply focused on the regulatory processes for our imetelstat NDA 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 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 Imetelstat NDA review to be held virtually on March 14, 2024. We feel very well prepared for the scheduled ODAC.

Speaker 3

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 Imetelstat with experts and peers, as we believe imetelstat 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 trial continue to give us confidence in what we believe is a meaningful clinical benefit and manageable safety profile with imetelstat in patients with transfusion dependent lower risk MDS. As reported at these conferences, the clinical attributes of imetelstat were differentiated, particularly with respect to high RBCTI response rate, durability of response and the consistency of effect across MDS subgroups that have historically been very difficult to treat.

Speaker 3

The Lancet published results from our IMerge 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 III trial of imetelstat in relapsedrefractory MF. 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 twenty twenty five, which will occur when approximately 35% of planned enrolled patients have died. A final analysis is expected in the first half of twenty twenty six, when over 50% of planned enrolled patients have died.

Speaker 3

We look forward to continuing to keep you updated on this important study. Lastly, we are also evaluating imetelstat in a Phase 1 study as a combination with ruxolitinib 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 imetelstat, as well as to explore the potential activity in a frontline MF disease setting. In January 2024, we escalated to the 3rd of 4 dose cohorts in the study following a unanimous decision by the study's safety evaluation team or SAC, who reviewed the 2nd cohort data. We are very pleased with this progress and look forward to providing future updates.

Speaker 3

With that, I'll turn the call over to Anil for a commercial update. Anil?

Speaker 6

Thank you, Faye, and good morning, everyone. I look forward to discussing first where we see significant unmet need 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.

Speaker 6

Approximately 10% of lower risk MDS patients are not eligible for ESAs and represent a very high unmet need subgroup. RS negative patients make up approximately 75% of lower risk MDS patients and RF population particularly vulnerable to poor clinical outcomes. There are no therapies indicated for the treatment of anemia in RS negative patients once they are relapsed or refractory to ESAs. RS positive patients make up approximately 25 percent of the lower risk MDS patients and most who are high transfusion burden lack effective treatment options. These underserved subgroups are at a greater risk for disease progression and suboptimal survival and are in the need for more effective treatment options.

Speaker 6

Moving on to an update on U. S. Launch preparations, with our PDUFA date just about 3.5 months away, we have completed multiple critical launch readiness activities and plan to be ready to launch Imetelstat in the U. S. Market upon potential approval.

Speaker 6

Long lead time activities such as securing our global trademark on 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 onboarded and fully integrated a highly experienced commercial and medical affairs team into Geron. We continue to transition Geron towards 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 regards to our field team, all regional business directors were onboarded in early January and key account manager roles are being recruited.

Speaker 6

We expect to onboard the sales force in the 1st and second quarter of 2024. I'm very excited by the caliber of talent we have recruited onto the commercial team and across the organization. Our cross functional launch teams have deep oncology expertise and operational experiences 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 Imetelstat in the U. S.

Speaker 6

Market upon potential FDA approval. With that, I'll now pass the call over to a financial update. Michelle?

Operator

Thanks, Anil, and good morning, everyone. For detailed Q4 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 $378,100,000 There are approximately $2,500,000 warrants outstanding and the potential proceeds from these warrants is $3,200,000 Total operating expenses for the 3 12 months ended December 31, 2023 were $54,300,000 $194,100,000 respectively, compared to $42,000,000 $139,100,000 for the comparable 2022 period. R and D expenses for the 3 12 months ended December 31, 2023, were $32,900,000 125,000,000 dollars respectively compared to $28,200,000 $95,500,000 for the same period in 2022.

Operator

Expenses have increased year over year, primarily related to supporting our Phase 3 clinical trials, IMerge 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 imetelstat in transfusion dependent lower risk MDS. G and A expenses were $21,400,000 $69,100,000 for the 3 12 months ended December 31, 2023, compared to $13,800,000 $43,600,000 for the same period in 2022.

Operator

The increase in G and A expense 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 of imetelstat. The increase in headcount is primarily in the commercial and medical affairs teams. Our projected full year 2024 operating expenses are expected to be between $270,000,000 280,000,000 Based on our current operating plans and expectations regarding the timing of a potential approval of our Imetelstat NDA that is currently under FDA review and subsequent potential U. S.

Operator

Commercial launch, we believe that our current cash resources together with projected revenues from U. S. Sales of the natalstat, proceeds from the exercise of outstanding warrants and funding under our loan facility will be sufficient to support our operations into the Q3 of 2025. I will now turn the call back over to Chip. Chip?

Speaker 4

Thanks, Michelle. For Geron, our progress in 2023 represented culmination of a many year multifaceted scientific and drug development journey. The goal of which is to translate the promise of squamorase inhibition into a potentially powerful medicine. Today, we're just 3.5 months away from the PDUFA date for our 1st in class swammerase inhibitor, which we believe has the potential to offer important and potentially life changing treatment option to patients with transfusion dependent lower risk MDS. We believe this is a robust commercial opportunity and we're on track for a successful transition to becoming a commercial company.

Speaker 4

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 twenty twenty five. We believe these programs carry significant value patients and shareholders alike, and we look forward to keeping you updated

Speaker 5

on our progress. We'll now open

Speaker 4

the line to questions. Operator?

Speaker 1

Your first question comes from the line of Corrine Johnson from Goldman Sachs. Your line is open.

Speaker 7

Good morning. Maybe one from us and a follow-up to it. How would you characterize the likelihood that you could see imetelstat approved with either black box warning and or REMS program? And then perhaps more importantly, what do you view as the implications from a commercial perspective under either scenario?

Speaker 5

A lot of the suppositions there Karim. Let me say, first of all, and you're going to hear this over and over, everybody will. So I'll just say once we're not going to comment on ongoing conversations with regulators and for the opportunities that we're getting very close to all of these milestone events.

Operator

Let me just say, I

Speaker 5

don't think it's warranted. I don't think that from our perspective, either Black Box or any of the rolling program is warranted, but predicting how all of that will go is a little too speculative for us that we feel very confident in its ability to be used safely and effectively in this target population.

Speaker 3

Okay, thanks.

Speaker 5

Sorry, the second part of that question, I've been talking about

Speaker 7

the commercial implications, if it was to be implemented?

Speaker 5

From speculation, I think I'll stay out of that speculation. I think everybody understands that it all depends on the it certainly when it comes to either black boxes or warnings or counter indications, these are all nuanced issues and then I'll refrain from circulation. Ramesh has never been done from my perspective, but again, kind of depends on how that gets put together. That's not where we're focused forward beyond those types of those issues we hope.

Speaker 3

Okay. Thank you.

Speaker 1

Your next question comes from the line of Kalpit Patel from B. Riley. Your line is open.

Speaker 8

Yes. Hey, good morning and thanks for taking the question. There was a letter submitted to the ODAC members by an independent group regarding the hepatic safety for imetelstat. The conclusion on 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 of the studies before the outcome?

Speaker 5

Well, I think we ought to clarify for those who aren't aware of it. At those, that letter was sent by the members of our hepatic safety or hepatic valuation committee with support agency. That group of little experts were convened, gosh, almost a decade ago. They're very, very well known to many people in industry, experts on drug induced liver injury, etcetera. And we engaged them to oversee all of the Intelsat trials going forward.

Speaker 5

And I have to say they did a wonderful job. We had quarterly meetings that have looked at every scrap of information in the case as far as I'm aware, they look at every scrap of information that relates in any way, shape or form to liver hepatotoxicity or liver safety. So let us say on the topic for people to need it. It simply reaffirms their beliefs that there is a very safe drug dealer perspective. So that's really where it came from.

Speaker 5

And 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 growth of interest, especially a decade ago.

Speaker 8

Okay. Thanks for taking the question.

Speaker 1

Your next question comes from the line of Stephen Willey from Stifel. Your line is open.

Speaker 9

Yes. 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? And I guess, 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?

Speaker 5

I think the evivecalsu that was weekly 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 for some, 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 the course of these cytokines, which as you know are indeed, sure rise and well manageable, I guess I should say.

Speaker 5

But we only know that because we study them in that way. So I think that's the de facto standard and what I would expect going forward from an equity perspective.

Speaker 9

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?

Speaker 5

Well, let me answer to that. It's an interesting question. I would say that it kind of depends on what where they are in the journey and as you say, kind of where the what the level of the neurodisclosure is and so forth and what their own experience is. As you saw in the placebo line, we have we don't have patients who simply also showed meaningful cytokineas even though they were receiving placebo and whatever other background therapy is around. So, but on the other hand, I think the regular the regular monitoring probably is associated with different, different products that these patients take that are normally associated with safety results and the heart status.

Speaker 5

So my guess is that this would be something that would get started. We start a new drug. Most psychologists would follow very carefully. Just kind of all makes sense. And then ultimately, it will be up to the physician on whatever the label itself to decide how the frequency and how that sort of plays out over the course of patient treatment with any drug including the first time.

Speaker 9

Okay. And then maybe just a kind of a bigger picture question. I mean, there's obviously kind of this ongoing narrative amongst investors regarding the exclusivity runway. And the development program for Imetelstat here is still not necessarily expanding, but it's being broadened into some additional indications. And so just curious, I guess, does do these Phase 1 studies reflect your internal confidence in the IP?

Speaker 9

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're thinking about the longer term R and D program for this drug?

Speaker 5

Sure. So I think we're referring just to be clear to improve on that and then the AML study, did I get your question about that?

Speaker 9

Yes, correct. So the rux combo study and just some of the other Phase 1?

Speaker 5

Yes. Sure. So I think it's incumbent on every company developing a drug regardless of what's going to be willing, whatever the nuances of the IP positions are, I think we study new combinations and study different utilities of drugs and sorry for being about this, I don't mean to be, but you have to leave it at kind of a high level. I think we could do that on the basis that we think if we think that there is benefit to be derived for patients, that's when we become really interested. The reciting is a really good example.

Speaker 5

It's you know and have given about, to these people know. There were a whole series of preclinical exams a number of years ago now. And they were a really good lab of some that we looked at the question of whether or not there were different ways to exploit some of the intrinsic activity that's monitoring against some of the cells that are regulated and transformed and ultimately prophylaxis in its clinical outcomes. And what we've seen was that if we end up treating with that thing from Swaxalta as I recall, but the jacket did it, you just see any damage. And then if you sort of chase that, if you come after that, which you can do in a laboratory experiment pretty easily with autonomous input in this case in a top shot, you actually get a higher proportion of animals surviving longer and also you see a depletion of the millennial and outright suns out.

Speaker 5

So that was the key for us to start ultimately to do all the work necessary to start the study. And it's because, A, we can go upfront with this and see frontline patients. B, we hope to be able to actually have ultimately improved efficacy, although this study is predominantly a safety study. C, 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 and we would if we know the drug, we touched that better than anybody else and we want to control that.

Speaker 5

So sorry to be again kind of appreciate about it, but I think that that's the rationale. The same with AML. AML is a terrible disease and relapsedrefractory patients have very, very few options. There are other drugs on the horizon that I hope for these patients take also for telematics will get into. But our goal is really patience.

Speaker 5

And then, usually, if you do something reasonably innovative, which obviously these are innovative, ultimately you file for intellectual property protection that plays out as a completely separate story and one that's not as interesting.

Speaker 9

Okay, makes sense. I appreciate the comments. Thanks.

Speaker 5

Sure.

Speaker 1

Thanks, question comes from the line of Gil Blum from Needham and Company. Your line is open. Yes.

Speaker 10

Hi. This is Ian Markowski on for Gil. Thank you for taking our question. 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 recent data from MorphoSys at ASH could impact how the recent data from MorphoSys at ASH could impact the myelofibrosis landscape and any future development in this space? Thank you.

Speaker 4

I think that's a killer made for Anil Kapoor. Anil?

Speaker 6

Sure. So we have had extensive discussions with both community and academic providers. And just as a reminder for everyone, low risk MDS is predominantly treated within the community setting. Physicians tell us they have deep experiences with drugs such as LEN HMAS and are very adept at managing heme related side effects for patients. And for them, they point to prolonged myelosuppression with HMAs, etcetera as areas which they have managed successfully for a long period of time.

Speaker 6

With Imetelstat in particular, what is really important is 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 2 and cycles 3. And also importantly, there are no clinical consequences associated with these cytopenias in terms of infections and hospitalizations and bleeding events. And when we also provide context on the mechanism of action, this is something that's received very favorably by the practicing hematologists. So we do not see much differences between the community versus the academic hematologists in terms of their perceptions and management of heme toxicities. They are adept with their training and also their practice experiences.

Speaker 6

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 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, which is looking at survival in a patient population that's relapsed and refractory to Jack eyes. This patient population is extremely underserved 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.

Speaker 10

Thank you.

Speaker 1

Your next question comes from the line of Joel Bee from Baird. Your line is open.

Speaker 11

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 that?

Speaker 6

Achim, do you want me to take that?

Speaker 5

Sorry, yes, go ahead.

Speaker 6

So that's a clinical study, Joel. We do not provide updates on the enrollment, etcetera, projections within that space. The study is on good. Okay.

Speaker 11

Got it. Thanks. And then for the guidance of the cash runway into Q3 2025, what sales assumptions are going into that guidance?

Operator

Sorry, Joel, I was on mute. We haven't shared any sales guidance or revenue guidance

Speaker 5

just yet. Got it. Thank you.

Speaker 1

And we have reached the end of our question and answer period. I will now turn the call back over to Erin Feingold for some final closing remarks.

Speaker 2

Thank you everyone so much for joining us today. We appreciate your interest in Geron and look forward to keeping you updated. Be well.

Speaker 1

This concludes today's conference call. Thank you for your participation. You may now disconnect.

Powered by Quartr
Earnings Conference Call
Geron Q4 2023
00:00 / 00:00