Hello, and welcome to the Geron Corporation Third Quarter 2024 Earnings Call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question-and-answer session. [Operator Instructions]. I would now like to turn the conference over to Aron Feingold, Vice President of Investor Relations and Corporate Communication. You may begin.

Good morning, everyone. Welcome to the Geron Corporation third quarter 2024 earnings conference call. I am Aron Feingold, Geron's Vice President of Investor Relations and Corporate Communication. 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; Jim Ziegler, Executive Vice President and Chief Commercial Officer; Dr. Faye Feller, Executive Vice President, Chief Medical Officer; and Dr. Andrew Grethlein, Executive Vice President, 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 launch, commercial opportunity and therapeutic potential of RYTELO, 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. 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 and our future updates to those risk factors. Geron undertakes no duty or obligation to update our forward-looking statements. With that, I'll turn the call over to Chip. Chip?

Thanks, Aaron. Good morning to everybody on the call. Thanks for joining us today. Following FDA approval and the commercial launch of RYTELO, our first-in-class telomerase inhibitor, this has been a transformative year for Geron. As a result, we believe we're well-positioned to build long-term commercial value with this product. In our first full quarter on the market in the United States, we achieved $28.2 million in RYTELO net product revenue, which exceeded our expectations. The initial quarter of product revenue speaks to our execution as a commercial company, as well as the high unmet need in lower-risk MDS and the compelling value proposition of RYTELO for hematologists and patients. This gives us confidence in future continued demand and momentum for RYTELO. Our strong IP position underlies the long-term commercial value proposition for RYTELO. We believe this IP position, including specific claims in our patents covering the indication that's in our FDA label and buttressed by the FDA's grant of orphan drug exclusivity for lower-risk MDS into June of 2031, will provide exclusivity in the United States through August of 2037. Today, our primary focus is on continuing to deliver on the initial success we achieved in the third quarter, getting RYTELO to more eligible lower-risk MDS patients and maximizing our opportunity in the U.S. market. In Europe, we believe that the CHMP review of our RYTELO marketing authorization application in lower-risk MDS could be completed in late 2024 or early 2025, with potential EU approval in the first half of 2025. Subject to receiving this approval, we're continuing to prepare for the potential launch of RYTELO in the EU and are planning to commercialize RYTELO in select EU markets beginning in 2026. As all of you know, Jim Ziegler joined us as Chief Commercial Officer in early September. Jim…

Thank you, Chip, and good morning, everyone. I am honored to join Geron at this important time for the company. With the U.S. launch of RYTELO, we have an exciting opportunity to improve the lives of patients with lower-risk MDS, with transfusion-dependent anemia. As Chip highlighted, we achieved $28.2 million in RYTELO net product revenues in our first full quarter of U.S. sales. In the first few months of launch, demand has increased month-over-month with Q3 performance exceeding our expectations. Demand from launch through Q3 has come from 388 ordering centers, which represents approximately 45% of our key targeted accounts. This strong start reinforces the high unmet need in RYTELO's clinical profile in first line ESA ineligible and second line plus lower risk MDS. Our market research indicates treating physicians appreciate RYTELO's differentiated clinical profile in 24-week and one year red blood cell transfusion independent rates, median duration of red blood cell transfusion independence and hemoglobin rise. We believe RYTELO's strong clinical data support broad utilization across treatment eligible patient sub-groups in both community and academic settings. Patient access is also critical for adoption and update, and we have achieved significant payer coverage since approval. Payers responsible for approximately 70% of U.S. covered lives have implemented medical coverage policies for RYTELO that are consistent with its FDA label, clinical trials and/or NCCN guidelines. Additionally, our Permanent J-code was issued in October 2024 and becomes effective on 1st January 2025. We believe the Permanent J-code will streamline billing and reimbursement for centers treating patients with RYTELO. I also want to acknowledge questions from investors regarding the trajectory of weekly RYTELO sales as reflected in third-party claims data. We believe that, while these claims data may reflect trends and demand that are directionally consistent with what we see internally, there are caveats…

Thanks, Jim, and good morning, everyone. For detailed Q3 2024 financial results, please refer to the press release we issued this morning, which is available on our website. We are pleased with our commercial performance this quarter and with securing the synthetic royalty and debt financing announced this morning. I'll bring your attention to key Q3 financial results and then discuss the new royalty and senior term loan agreement. As of September 30, 2024, we had approximately $378.9 million in cash, cash equivalents, restricted cash and marketable securities on a pro-forma basis including gross proceeds from the upfront payment under the Royalty Pharma agreement and the first tranche of the Pharmakon loan and after repayment of our existing debt, we had approximately $542.4 million in cash, cash equivalents, restricted cash and marketable securities as of September 30, 2024. Total product revenue net for the three and nine months ended September 30 was $28.2 million and $29 million respectively. Total net revenue for the three months and the nine months ended September 30, 2024 was $28.3 million and $29.5 million respectively, compared to $164,000 and $214,000 for the same period in 2023. The increase in revenue is due to product revenue from U.S. sales of RYTELO, which was available for prescribers to order from specialty distributors as of June 27, 2024. Total operating expenses for the three and nine months ended September 30, were $56.5 million and $183.1 million respectively, compared to $47.8 million and $139.9 million for the same period in 2023. Cost of goods sold was approximately $450,000 and $473,000 for the three and nine months ended September 30, 2024 respectively, which consisted of cost to manufacture and distribute RYTELO. Research and development expenses for the three months and nine months ended September 30, 2024 were $20.2 million and…

Thanks, Michelle. And hello, everyone. I'd like to start by sharing how meaningful it has been for me and my entire team to hear feedback about the impact of RYTELO in the commercial setting. This further motivates our team to develop and deliver imetelstat for patients with myeloid hematologic malignancies. The field medical team has been responding to information requests that support HCPs as they use RYTELO in the commercial setting, in particular around education on cytopenia management and sequencing with other lower-risk MDS treatment. Today, I will focus on our ASH abstracts released earlier this week, which we believe continue to highlight telomerase inhibition with imetelstat as an important and powerful approach to treating myeloid hematologic malignancies. For detailed information on the data abstract, please view the press release we issued on Tuesday, available on our website or visit the ASH website to view the abstract. First, I will cover new analyses in the IMerge clinical trial, suggesting that imetelstat demonstrates clinical activity in patients with lower-risk MDS with transfusion dependent anemia, regardless of cryotherapy. Abstract 352, accepted as an oral presentation, pooled thea from IMerge Phase 2, Phase 3 and the QTc substudy and evaluates the effect of prior treatment, including ESAs, luspatercept, lenalidomide and HMA on the clinical activity across these patients. Although we have small numbers in some cases and limited data on outcomes in later lines of treatment, we believe these data have important clinical implications suggesting that these patients experience an RBC transfusion related clinical benefit and improvements in hemoglobin with imetelstat, regardless of their prior treatment history. Abstract 4590, accepted as a poster presentation, reports the first efficacy and safety results from the ventricular repolarization IMerge QTc substudy conducted per FDA guidance. As of the data cutoff on May 10, 2024, no clinically…

Thanks, Fay. To close, we're obviously very pleased with this quarter's performance and the feedback we're receiving from customers and payers. We're confident in our launch trajectory and opportunity for long-term growth, while recognizing we're only four months into this launch. We have conviction that RYTELO can become part of the standard-of-care for eligible patients in this high unmet-need, lower-risk MDS treatment paradigm and that it can bring differentiated benefits to patients both in the U.S. And subject to regulatory approval in the EU. In addition to lower-risk MDS, we're also looking-forward to the readout of our pivotal Phase 3 IMpactMF trial in relapsed/refractory MF. We believe that approval of RYTELO in lower-risk MDS in the EU and a positive outcome in this MF trial are key milestones that could contribute very significantly to the commercial value proposition for RYTELO in the future. We'll now open the line for questions. Operator?

[Operator Instructions] Your first question comes from the line of Tara Bancroft with T. D. Cowen.

Hi. Good morning and congrats on the great quarter. So given what you have seen this quarter, we were hoping you could give us a better idea of how you think growth cadence could look over the next few quarters or over the next year. But I'm especially curious how your growth assumptions and even the ultimate market for RYTELO factored into the terms of the royalty deal?

Tara, this is Chip. Could you explain a little bit more what you mean by what factored into the royalty due just for the royalty [indiscernible]

Like, when you were negotiating the deal, how were your assumptions for the ultimate market and or growth over the next couple of years kind of factored into that?

Sure. Michelle, would you like to take that?

Sure. Well, I think Jim why don't we have Jim answer her first question, which is just about how we're thinking about growth over the coming quarters, and then I can comment on the structure.

Okay. Good morning, Tara. Jim Ziegler here. So what we're looking at is and expecting is steady consistent growth across all of the patient segments, specifically second line, in both, RS negative and RS positive, first line ESA ineligible, and then, of course, the relapsed refractory third line plus patients. We're not giving guidance at this point, but we are expecting steady and consistent growth going forward.

And I'll take the second one. First of all, I just want to say that this was an extremely competitive process, and we're very happy with the outcome. We used our current internal forecast and, to determine, and negotiate the terms, which we, which we're very, again, just very pleased with.

Your next question is from Faisal Khurshid with Leerink Partners.

Hey, guys. Just two questions, if you don't mind. 1st, just on the royalty percentage rates. Can you just clarify sort of how you think investors should be thinking about the royalty rate as well as the kind of cap multiplier on that? I think the rate seems like it starts out a little bit higher than your typical royalty deals then kind of comes lower. And then if you could just clarify your cash runway expectations?

So I mean, I'm not going to comment on other transactions that have been done with Royalty Pharma. Again, very competitive. We're very happy that it is a capped royalty deal at the 1.65. It's achieved, by a certain date. And so we think that the rate is competitive, from what we've seen. But, again, I think that our focus, is on that cap of 1.65. And based on our current plans and meeting our internal revenue projections, we feel pretty confident, that we can reach that, prior to peak sales.

Got it. Understood.

On the cash runway, as I mentioned in my script, there are scenarios, where we feel that these two transactions, can take us to breakeven. Again, based on our current plan, that would mean meeting our internal revenue, our OpEx projections over the next several years. We feel that, we could reach breakeven without needing additional equity financing. One of our goals of these transactions was to not fall below 12-months of cash, as I said in Q2. Just to clarify, these two transactions allow us to maintain 12-months of cash going forward, again, based on our current revenue expectations, and OpEx projections. So we're not saying, we have 12-months of cash, because I know previously we talked about cash into Q2 of 2026. What we're saying is that, we can maintain that 12-months of cash, which has always been one of our goals not to fall below 12-months.

Yes. It makes sense. And then just to clarify your comments, so you're saying that your kind of internal assumptions are that you kind of tripped the multiple on like on the earlier side?

Yes. If we meet our current internal revenue projections, yes.

Your next question is from Corinne Johnson with Goldman Sachs.

Thanks. Good morning. You did mention that you expect to see kind of steady growth over the coming quarters. But could you clarify a little bit about what that means to you? Is that in terms of like absolute number of patient growth or the growth rate? Just a little clarification would be helpful there. And then, in terms of the patients that you're seeing come on to therapy, are these primarily second line or third line patients? And what portion of them have previously seen Revlimid at this stage?

Go ahead, Jim.

Hi, Corinne. This is Jim Ziegler. Again, we're not providing guidance, but in terms of our growth, what we expect is growth across all patient segments in both the academic and community setting. And what we provided on this earnings call is that, 388 of our targeted accounts have ordered. So that's the breadth. Over time, what we're expecting is, obviously to increase the breadth and depth of prescriptions, our prescribing, RYTELO for patients across these accounts. So, I'll just reinforce that we're seeing, uptake in utilization across all patient segments, including first line ESA ineligible, second line and then third line patients at this point.

Your next question comes from the line of Carter Gould with Barclays.

Great. Good morning. Thanks for taking the questions. May I start-off just one or two housekeeping questions? Can you spell out any impacts from inventory in the quarter, and as well as you're providing patient starts numbers? That would be helpful. And then maybe just a follow-up on the prior question. The breadth of centers with ordering is impressive. I guess what I'm -- but I guess with our back of the [indiscernible], it does suggest that, the depth of prescribing is still very much in its early stages. Can you talk a little bit about what this implies around how centers are sort of experimenting with the product and then adopting and maybe if that argues against bolus and any commentary there would be helpful?

Great. Thanks, Carter. Jim Ziegler again. So in terms of inventory, typically in buy and bill what we see is between 2 and 4 weeks of inventory and we're right in that range. So, that's consistent with what we would expect. In terms of the breadth and depth questions, we're still relatively early in launch. This is our first full quarter. And so, your observations are correct. Early on, we're seeing the breadth, physicians are getting experience. And then over time, we expect to see depth in these accounts and continued breadth going forward. In terms of bolus, the way we think about it is, there's still a high unmet need, RYTELO gives patients another treatment option. We expect consistent steady growth. We within Geron did not model a bolus. We expected steady and consistent growth. And early in launch, that's what we're seeing.

And the patient starts number, are you guys providing that?

Yeah. I'm not, as you know, Carter, on the patient starts, in the buy and bill market, we don't get that hard data, in the way you might with other markets. So the way to think about it is if you take the total milligrams that were sold divided by your assumption on average patient wait times 7.1 starting dose, that would give you the range of patients. But it doesn't account for specifically is new patients versus continuing patients or dose interruptions or, dose reductions. So that's why we're not gonna give that going forward because it's not a clean number, it's a calculated number.

Your next question is from Emily Bodnar with H.C. Wainwright.

Hi. Good morning. Thanks for taking the questions, and congrats on a good quarter. I guess maybe if you can discuss some of the initial real world experience in terms of benefit on transfusion reductions and also on the safety profile. Are you kind of seeing the data in line with the IMerge study? And then curious how luspatercept kind of transitioning more into the first line setting has, impacted the launch, if at all, and if you're seeing more patients previously treated with luspatercept in the first line?

Hi. It's Jim again. I'll take the first part of the question and maybe Faye can also jump in. It's still relatively early in launch and, much of the insights that we get are from the field and from, market research like our patient chart audits. What I would share right now is that, performance and real world sort of experiences are consistent with clinical trials. But again, it's still relatively early on, in launch. And then in terms of, luspatercept in the first line, yes. Based upon their label and the commands, I expect that trial that they will, compete and, compete for that first line patient against ESA as you know, and regardless of whether that first line patient, is on ESA or luspatercept, we expect that our differentiated product profile will allow us to become standard of care in second line and we still have that first line ESA ineligible patient population, which is about 10% that, we expect to, compete for as well.

Yes. And -- Hi this is Faye. Just to add and reinforce what Jim was saying. It's still early, but anecdotally, what we are hearing from the field is that, this the community of providers are comfortable managing, these cytopenias and are -- it's -- overall enthusiastic and excited to use, RYTELO for their and have this option for their patients.

Your next question is from Stephen Willey with Stifel.

Yes. Good morning. Thanks for taking the questions, and congrats on the progress. I was wondering if you could just maybe talk a little bit about the split that you're seeing. I know, it's early in terms of data that you guys are getting, but just in terms of the utilization split between academics and community prescribers. And then, you mentioned your review of chart audits, in terms of informing how current utilization trends are looking. Can you talk about just how the dose management, to address the cytopenias that occurred typically during the first few cycles is playing out, in the real world data, just relative to IMerge. And then I just have a follow-up for Michelle.

Sure. Hi, Steven. Jim Ziegler again. The split between community and academic is 65/35 or roughly two-thirds, one-third. And then right now, what we're hearing from our physicians in both field observations as well as our market research is that, cytopenia management is well understood. There aren't major concerns. I would expect that, the real world cytopenia management is at least as good as it is in the clinical trials. We have a lot of personal, non-personal efforts to help support appropriate cytopenia management associated with RYTELO.

Okay. And then, maybe for Michelle. I know you've kind of given kind of longer-term gross to net guidance in the mid-teens range. Just curious if you could give us a gross-to-net number for the quarter. And then also, obviously, the royalty transaction will impact cost of goods going forward, but just curious, if you can kind of give us, a steady state cost of goods assumption that, excludes the impact of the royalty.

Yes, sure. So, we have continued to guide on the gross-to-net in kind of the mid-double-digits, and that's what it was that's what we ended in the third quarter. I believe it was -- you can do the math, around 14%. So we expect that to slightly go up as we -- the volume increases, and we review the mix, the 340B mix. But we continue to guide on the mid double-digits. And then, on COGS, I mean, obviously, we're not guiding on specific COGS. But, once we get out of sort of this inventory that had already been expensed, we anticipate our fully-loaded COGS to be mid-single-digits going forward.

Next question is from Kalpit Patel with B. Riley Securities.

Yes. Good morning and thanks for taking the questions. Maybe first for Michelle, I guess, can you elaborate on why the royalty and debt structure was chosen over maybe a simpler equity raise, especially given the current market cap of the company? What specific considerations made this approach more preferable?

So again, the transaction, this is a result of an extremely competitive process. We had options across both debt and royalty structures and those ranged in various sizes and terms. Based on the terms that were available to us, we thought carefully about how much debt to take on so that we could pay-off our current debt that we have with Hercules, SVB that had unfavorable terms, which we did pay-off, once we received the funds from Pharmakon. And then just the amount of sales to retain in the early launch period and we felt that this struck the right balance. And again, this reduces our dependency on the equity markets, which was a goal of ours was to not further dilute the stock.

Okay. Got it. And then maybe in the data that you're seeing so far, do we have an early sense of what the month-over-month adherence rates or continuation rates are for these patients who started treatment? How consistent is that with the clinical trials?

Hi, Kalpit, this is Jim again. It's still relatively early. I'm not comfortable given any data at this point, but obviously it's something that we're trying to assess through a number of different mechanisms. But for right now anecdotally what I would say is that we expect month-over-month treatment to be more consistent with clinical trials and that over time, we're obviously aspiring to have better management over time.

The next question comes from Gil Blum with Needham & Company.

Good morning, and thanks for squeezing our question in, and congrats on a very impressive first quarter of sales. So just a couple from us. Now that you have the additional capital, is there a plan on further investing on expanding sales in the U.S., for example or really this is just about keeping that 12-months buffer? And my other question is regarding assumptions on profitability, which you've mentioned. Do you think that includes potential in myelofibrosis?

Michelle, why don't you go ahead and restart?

Sure. Yeah. Thanks, Gil. So the reason why we chose this hybrid structure is for the flexibility. So as you recall, we took down the first $250 million, we still have access to another $125 million, really we looked at our strategy around redundancy and second supplier for RYTELO, and that has always been extremely important to us. This now allows us to invest and get ready for a second supplier and for redundancy on our drug substance. It also allows us to continue to support the U.S. commercialization of RYTELO, and, but all of those costs for commercialization are in our current plans in the U.S. but it allows us to also start spending some capital on preparing for a potential EU launch, and as you mentioned, we retain more than 12 months of cash going forward. And then, Gil maybe your second question?

Regarding, assumptions on profitability and if they include myelofibrosis.

Well, I mean, there are scenarios where the financing takes us to breakeven, with just lower-risk MDS and with myelofibrosis, again, based on our current plans and our internal revenue, and OpEx projections, but, yes, we do include MF in our -- internal revenue projections. [Operator Instructions] This will conclude the question-and-answer session. I will turn the call to Aron for closing remarks.

Thanks so much, everyone, for joining us today. We appreciate your interest in Geron and look forward to keeping you updated during this very exciting time for our company. Thank you.

This concludes today's conference call. We thank you for joining. You may now disconnect.