SNDX - NASDAQ

Good day, everyone, and welcome to the Syndax Second Quarter 2025 Earnings Conference Call. Today's call is being recorded. [Operator Instructions] At this time, I would like to turn the call over to Sharon Klahre, Head of Investor Relations at Syndax Pharmaceuticals. Sharon Klahre Thank you, operator. Welcome, and thank you, all, for joining us today for a review of Syndax's Second Quarter 2025 Financial and Operating Results. I'm Sharon Klahre, and with me today to provide an update on the company's progress and discuss financial results are Michael Metzger, Chief Executive Officer; Steve Closter, Chief Commercial Officer; Dr. Nick Botwood, Head of R&D and Chief Medical Officer; Keith Goldan, Chief Financial Officer. Also joining us on the call today for question-and-answer session are Dr. Peter Ordentlich, Chief Scientific Officer; and Dr. Anjali Ganguli, Chief Strategy Officer. This call is accompanied by a slide deck that has been posted on the Investor page of the company's website. You can now turn to our forward-looking statements on Slide 2. Before we begin, I'd like to remind you that any statements made during this call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities Litigation Reform Act 1995. Actual results may differ materially from those indicated by the statements as a result of various important factors, including those discussed in the Risk Factors section in the company's most recent quarterly report on Form 10-Q as well as other reports filed with the SEC. Any forward-looking statements made represent our views as of today, on August 4, 2025, only. A replay of this call will be available on the company's website, www.syndax.com, following its completion. And with that, I am pleased to turn the call over to Michael Metzger, Chief Executive Officer of Syndax.

Thank you, Sharon, and good afternoon, and thank you, all, for joining us today. Starting with Slide 3. First half of 2025 has been a transformational period for Syndax, marked by excellent commercial and pipeline execution. We are well positioned for rapid growth in the second half of 2025 and beyond with its first -- with 2 first and best-in-class therapies with a combined market opportunity exceeding $10 billion. Revuforj and Niktimvo sales are growing with nearly $100 million in combined net product sales in the first half of the year, significantly exceeding expectations. Notably, Revuforj net revenue increased 43% quarter-over-quarter to $28.6 million, even with approximately 1/3 of patients pausing treatment to receive a stem cell transplant. Importantly, we are on the road to profitability with growing contributions from Revuforj and Niktimvo, a strong balance sheet, and an operating expense base that will remain stable for the next few years, while fully funding our strategic priorities. Looking to the future, our leadership in the menin space positions us to be first to the frontline and meaningfully expand the Revuforj franchise. We have a similarly compelling opportunity to bring Niktimvo into earlier lines of therapy and additional patient populations. Turning to Slide 4. Let's dive into more detail on Revuforj, the first and only FDA-approved treatment for relapsed or refractory acute leukemia with a KMT2A translocation. The continued growth reflects strong uptake, the high unmet medical need and physicians' enthusiasm for Revuforj. It is clear following the recent presentations at ASCO and EHA that revumenib has a best-in-class profile with compelling activity across multiple genetic subtypes, including efficacy data in relapsed/refractory mutant NPM1 AML that surpassed any other results seen in the field. The breadth and strength of our clinical data will be the key to our success in acute leukemia, a market that is efficacy driven, given the severity of the disease. As we look ahead, the outlook is very promising with multiple drivers that will further solidify our leading position and ensure sustained growth for many years to come. I will briefly highlight those drivers, and the team will provide additional details. First, patient identification and uptake has been strong. Since launch, we have already treated over 500 patients with Revuforj with approximately 90% of usage in KMT2A patients. In just 7 months, we have already reached 1/4 of the 2,000 patients diagnosed with relapsed/refractory KMT2A acute leukemia each year. Based on the robust activity we have seen in this population and physician excitement around the drug, we expect the total number of patients treated with Revuforj to grow materially in future quarters, particularly as it is the only approved therapy for these patients. Second, Revuforj is increasingly being used in earlier lines of therapy. Emerging claims data showed that the use in KMT2A as of this quarter is already being concentrated in the second line. This trend is especially important in oncology because as patients are treated earlier, they generally have a higher response rate, a longer duration of response, and a higher chance of proceeding to a potentially curative stem cell transplant. Thus, as Revuforj is used earlier, we expect to see an increase in the average time on drug for all patients. We also expect to see a high rate of patients proceeding to transplant -- a higher rate of patients treating -- proceeding to transplant than was observed in our pivotal trial, which on average enrolled a later-line patient population. In fact, early indicators suggest that we are already seeing a meaningfully higher transplant rate in the commercial setting. Third is the group of patients receiving Revuforj post-transplant increases, it should substantially increase the overall duration of therapy. Notably, prescribing physicians tell us they plan to restart patients on Revuforj post-transplant for 1 to 2 years. Given the high risk of recurrence, both patients and physicians tell us they are eager to restart the therapy that induced remission, especially when the drug has an excellent tolerability profile. These 3 drivers position Revuforj to transform care for KMT2A patients from an acute treatment paradigm with survival measured in a few months to a more chronic disease with the potential to extend survival from months to years. Importantly, relapsed/refractory KMT2A acute leukemia is just the first opportunity for Revuforj. In the near term, we anticipate both the inclusion of Revuforj in the clinical treatment guidelines and the approval of our supplemental new drug application, or sNDA, in relapsed/refractory mutant NPM1 AML. The anticipated approval of our sNDA, which was recently granted priority review and a signed a PDUFA action date of October 25, 2025, would expand our addressable population to over 6,000 patients across both genetic subtypes and increase the relapsed/refractory market opportunity for Revuforj in the U.S. to $2 billion. Importantly, Revuforj is positioned to become the first and only menin inhibitor with a label that expands to mutant NPM1 and KMT2A translocated patients, both adults and children. Based on resounding KOL feedback, the expected breadth of our label will be a major competitive advantage. Looking to the future, we will further extend our leadership into the frontline setting, a U.S. market opportunity exceeding $5 billion. Enrollment is already ongoing in our frontline trial for patients unfit to receive intensive chemotherapy and start-up activities are well underway to initiate our trials in patients able to receive intensive chemotherapy. With Revuforj's best-in-class profile and a multiyear start into the market versus potential me-too competitors, we will maintain our dominant position in this multibillion-dollar market opportunity. Shifting gears to Slide 5 to Niktimvo, our first-in-class therapy for chronic graft-versus-host-disease, or GVHD. I am pleased to highlight a very successful first full quarter for sales with our partner, Incyte, reporting $36.2 million in net revenue. This is up significantly from $13.6 million in the first 2 months of the launch in Q1. The $50 million in net revenue generated in the first 5 months of the launch underscores the substantial opportunity in chronic GVHD. Importantly, Niktimvo is already profitable to Syndax with our 50% share of the Niktimvo product contribution amounting to $9.4 million for the second quarter. As sales continue to ramp, the cash flow contributions to Syndax from Niktimvo will only grow in significance. With initial Niktimvo sales tracking with the early benchmarks set by RE

Thank you, Michael. Let's dive right into our commercial updates on Revuforj and Niktimvo, starting with Revuforj on Slide 6. As Michael said, the launch is going very well with net revenue for the second quarter increasing 43% quarter-over-quarter to nearly $29 million and $56 million generated over the first 7 months of the launch. These impressive results are driven by multiple factors, including: a high unmet patient need; a robust stream of new patient starts over the quarter; expanding breadth and depth of prescribing; excellent formulary coverage; and a product in Revuforj that is delivering for patients. Physicians are observing excellent activity in clinical practice, Revuforj is rapidly becoming a standard of care in our indicated population. Over 1,300 prescriptions for Revuforj have been written for more than 500 patients from launch through the end of June. Just midway through the year, we have already penetrated 25% of the annual 2,000 patient incidents and are on track to penetrate 50% by year's end. Next, I'm excited to share some of the emerging data and customer feedback that provide important insights into the population of patients being treated with Revuforj, and bolster our confidence that the momentum we have seen since launch will continue well into the second half of the year and beyond. First, Revuforj is increasingly being used to treat patients earlier in their treatment journey. Early claims data show that 70% of Revuforj use has been concentrated in the second and third-line settings, with approximately 50% of use coming from that second line, which we can also call first relapse patients alone. Second, we estimate that 1/3 of KMT2A patients treated with Revuforj have proceeded to transplant based on our analysis of medium to large academic institutions using Revuforj commercially. In contrast, one out of 4 KMT2A patients proceeded to transplant after treatment with Revuforj in AUGMENT-101, which enrolled a significant percentage of later line and heavily pretreated patients. It's important to understand that patients who ultimately proceed to transplant are typically treated with Revuforj for 2 to 4 months to ensure complete disease remission before pausing Revuforj for approximately 3 months to ensure engraftment of the transplant. Notably, physicians tell us they expect to put most, if not all, of their patients back on Revuforj post-transplant for 1 to 2 years. In fact, in our clinical trial experience and compassionate-use program, we have already seen transplant patients who have been on Revuforj for 1 to 2 years and we were still on drug at the time of the data cutoff. Encouragingly, in the commercial setting we have started to see the first cohort of patients restart Revuforj. Based on a sampling of our accounts, we estimate that at least 1/3 of transplant patients have already restarted Revuforj, with that percentage expected to grow over time as more patients clear the engraftment period. As Revuforj continues to move earlier in the treatment paradigm, we expect this will translate to a significant increase in the average duration of therapy over time. Specifically, we expect the average treatment duration to build to 4 to 6 months in the first year of launch. According to our assessment of patients who started Revuforj shortly after launch, the average time on therapy is already well within the projected 4 to 6-month range, and we expect this duration to expand to an average of 6 to 12 months as treatment patterns mature in the second year of launch. I'd now like to briefly review some other metrics that underscore the strong position we're in for continued growth in KMT2A, and our anticipated launch into relapsed/refractory mutant NPM1 AML. First, we have a broad and growing prescriber base. From launch through the end of June, we've penetrated 65% of our higher priority Tier 1 and Tier 2 accounts, up from 44% of accounts at the end of last quarter and continuing to grow into the third quarter. These Tier 1 and Tier 2 accounts are the centers of excellence in the medium to large academic institutions, which represent 2/3 of the patient opportunity. Adoption is also increasing across all other tiers too, including in the community setting. Among all accounts that have ordered, the vast majority have ordered multiple times. Second, we have established excellent market access. Formulary coverage is now complete with more than 97% of all lives covered, including commercial, Medicare and Medicaid patients. Nearly all prescriptions are reimbursed with very few patients requiring our patient assistance program. The average time from prescription to first fill is less than 4 days, significantly faster than typical industry benchmarks. The best-in-class customer service we are delivering, will be a key factor for our long-term competitive immunity and brand loyalty. Notably, Revuforj performance is outperforming the early launch benchmarks set by other targeted AML therapies on key metrics, including revenue and prescriptions, patients treated activation accounts, as well as formulary coverage. Further, all indicators give us confidence that Revuforj is delivering for patients, and that we are well positioned to develop this medicine into an industry-leading franchise with a market opportunity exceeding $5 billion across the relapsed/refractory and frontline setting, as outlined on Slide 7. Now turning to key Niktimvo metrics on Slide 8. Since the beginning of the launch, over 4,000 infusions have been administered to an estimated 700 patients, representing approximately 10% of the third-line plus chronic GVHD total market. Of all the patients that had started Niktimvo, approximately 80% to 90% remain on therapy today. More than 80% of all bone marrow transplant centers in the U.S. are using Niktimvo, reflecting solid execution and the strong commercial synergies Niktimvo has with both companies' product portfolios. Importantly, Niktimvo is poised for further growth given the high unmet need in the chronic GVHD space and the positive experience physicians and patients are having with the drug. Physicians are reporting rapid and durable improvements across organ systems, including some of the most difficult-to-treat organs like the lungs and skin. These observations align with the results we demonstrated in our pivotal trial and highlight Niktimvo's unique ability to address both fibrosis and inflammation, hallmarks of the condition. As shown on Slide 9, Niktimvo has a multibillion-dollar market opportunity. Our current indication allows us to target the 6,500 chronic GVHD patients in the U.S. who require 3 or more lines of therapy. This represents a $2 billion total addressable market, assuming an average treatment duration of 12 months, which could be conservative given the chronic nature of the disease and the tolerability of Niktimvo. Notably, in our clinical trial experience, we have seen some patients stay on therapy for more than 3 years. To summarize, we are very pleased with the progress we've made with both Revuforj and Niktimvo. Early indicators for both launches drive our confidence in continued growth and expansion with both products. With that, I'll hand the call over to Nick.

It's a pleasure to be on the call today, and to have the opportunity to build upon the exceptional work that Syndax has done pioneering 2 new therapeutic approaches. Starting on Slide 10 with Revuforj or revumenib, an asset which has the potential to become the menin inhibitor of choice across the breadth of menin-driven acute leukemias. In the second quarter, we advanced our leadership position with a strong presence at EHA and ASCO, including 2 important publications. At EHA, we and our collaborators presented the latest data from AUGMENT-101 and the BEAT AML trial. I'd like to highlight 2 key takeaways from these. First, the AUGMENT-101 data demonstrate the breadth of revumenib activity across relapsed/refractory mutant NPM1, KM2TA and NUP98r acute leukemias. Notably, in the pivotal NPM1 population, nearly half of the patients achieved an overall response. And in a subgroup analysis, a median overall survival of 23 months was observed among these responders. These data, along with the rate of CR/CRh and duration of CR/CRh, are encouraging results that really stand out in this population. The compelling results are particularly relevant as efficacy is paramount in patients with acute leukemia given the severity of disease and the need for improved outcomes. Data from the pivotal NPM1 population were recently published in Blood, an important milestone that makes these landmark results available to the clinical community. Turning now to NUP98r. Phase I data from the AUGMENT-101 trial show an overall response rate of 60% among 5 patients with relapsed/refractory NUP98r AML, which is an aggressive difficult-to-treat form of acute leukemia. While the sample size is small, physicians are encouraged by these data and further trials are underway that will evaluate revumenib in NUP98r as well as other acute leukemias associated with HOX upregulation. The compelling and consistent results observed with revumenib across these genetic subtypes highlights the potential for revumenib to transform the standard of care for potentially 50% or more of all patients with AML. Moving now to the second key takeaway. The BEAT AML data presented at EHA and simultaneously published in the Journal of Clinical Oncology, are encouraging. As a reminder, this is a Phase Ib trial evaluating revumenib in combination with venetoclax and azacitidine in newly diagnosed older patients with mutant NPM1 or KMT2A rearranged AML. The data support the combinability of revumenib with ven/aza in the frontline setting and the potential for the triplet to provide high rates of complete remission and MRD negativity, 2 treatment goals associated with improved clinical outcomes. The overall response rate was 88% and the complete remission rate was 67% in the 43-patient intent-to-treat population. Importantly, MRD negativity was 100% by centralized flow cytometry testing. Both the CR and MRD negativity compare very favorably to the historical rates reported in the VIALE-A trial of ven/aza. Looking ahead, we have revumenib publications and presentations planned at major upcoming medical congresses, including the anticipated presentation of the first real-world evidence before the end of the year. Given the strong clinical interest in real-world evidence, we are thrilled to be working with leading cancer centers and physicians to present outcomes for this new therapeutic class. Turning now to Slide 11, and our further work developing Revuforj and Niktimvo into industry-leading franchises, I want to highlight 3 key points. First, we are laser-focused on extending our leadership position in menin inhibition into the frontline setting. Enrollment is well underway in the pivotal EVOLVE-2 trial of revumenib in combination with ven/aza in newly diagnosed patients with mutant NPM1 or KMT2A rearranged AML, who are ineligible or unfit to receive intensive chemotherapy. EVOLVE-2 is a Phase III randomized, double-blind, placebo-controlled trial. This trial will have dual primary endpoints of complete remission and overall survival to support potential accelerated approval and full approval, respectively. While the trial is open to both NPM1 and KMT2A patients for enrollment, the primary efficacy analysis will be based on the NPM1 population. This is the population that is more commonly ineligible for intensive chemotherapy due to advanced age or other comorbidities, unlike the KMT2A population which tends to be younger and fit enough for intensive chemotherapy. We are conducting this trial in partnership with the HOVON Group, a leading clinical trial cooperative known for executing clinical trials that deliver practice- changing data in hematology. Second, in the newly diagnosed fit population, study start-up activities are well underway for 2 randomized placebo-controlled [indiscernible] studies of revumenib in combination with intensive chemotherapy followed by maintenance. We have named these the REVEAL trials. One trial is designed for patients with an NPM1 mutation and one for patients with KM2T2A (sic) [ KMT2A ]rearrangements. We expect to initiate in the fourth quarter of 2025. In the NPM1 population, the study will have dual primary endpoints of MRD negative CR and event-free survival, as these are important clinical endpoints in this population, and could have the potential to support accelerated approval and full approval, respectively. We expect that high awareness of Revuforj and positive experience in the clinic will drive rapid enrollment across our frontline programs. In support of our trials in the fit population, we are also looking forward to reporting Phase I data in newly diagnosed patients treated with revumenib and intensive chemotherapy in the fourth quarter of the year. Lastly, I want to highlight the work underway to develop Niktimvo, or axatilimab, for additional patient populations. In partnership with Incyte, several important trials are well underway, including a Phase II trial studying axatilimab in combination with ruxolitinib and a Phase III placebo-controlled registration-directed trial investigating axatilimab in combination with steroids. Beyond chronic graft-versus-host disease, we have an ongoing Phase II placebo-controlled trial called MAXPIRe, which is studying axatilimab in idiopathic pulmonary fibrosis, or IPF. Enrollment is proceeding very well, and we are on track to complete enrollment in the fourth quarter of this year, with top line data anticipated in the second half of 2026. We are optimistic about the potential for axatilimab in IPF and beyond, given the strong mechanistic rationale and preclinical evidence, along with the remarkable lung response observed in the AGAVE-201 trial. With that, I will hand the call over to Keith to discuss our financials.

Thank you, Nick. Earlier this afternoon, we reported detailed second quarter 2025 financial results, and I'll touch on a few of these key points on Slide 12. For the second quarter of 2025, we reported Revuforj net revenue of $28.6 million. Quarter-over-quarter sales growth was driven by demand, as inventory levels remained stable to the first quarter at 2 to 3 weeks. We expect quarterly growth to meaningfully accelerate over the next year with the potential approval in NPM1, as well as the benefit of a longer duration of treatment in KMT2Ar acute leukemia. Also in the second quarter, Incyte reported Niktimvo net revenue of $36.2 million with inventory accounting for less than 5% of sales. Syndax reported $9.4 million in Niktimvo collaboration revenue after deducting the cost of sales and commercial expenses. Importantly, Niktimvo is already a positive cash flow contributor to Syndax in just its first full quarter of sales. We expect the Niktimvo margin contribution, defined as collaboration revenue recorded by Syndax, as a percentage of Niktimvo net sales to be in the 20% to 30% range in the near term, and we anticipate this will improve longer term as sales grow and the partnership leverages a largely fixed expense base. We expect continued sales growth given GVHD remains a chronic disease, where there is a high response rate to Niktimvo and the average patient will likely remain on therapy for years. R&D expense was $62.2 million in 2Q with the increase versus the comparable prior year, driven by costs related to ongoing trials and increased activities to support commercialization. SG&A expense was $43.8 million, with the increase versus the comparable prior year driven by costs related to the U.S. commercial launch of Revuforj. With regard to expenses, you can find our guidance for the third quarter of 2025 and full year in the press release we issued today. Notably, we announced today that we expect our operating expenses to remain stable over the next few years. Turning to the balance sheet. We continue to maintain a strong financial position with $518 million in cash, equivalents and short and long-term investments as of June 30th. As I've said in the past, and I reiterate today, we expect Syndax will reach profitability with current funds on hand. In fact, my confidence is higher today given both drugs are outperforming our original forecasts. We are confident we can execute commercially and also deliver on our integrated clinical development plans for both drugs while keeping operating expenses at today's levels. Our combined cash with increasing Revuforj and Niktimvo cash flow contributions alongside a fixed expense base, will drive our path to profitability. Turning the call back over to Michael.

Thank you, Keith. Before we move to Q&A, I want to take a moment on Slide 13 to reiterate how well positioned Syndax is as a company. Revuforj and Niktimvo are outperforming expectations as strong physician enthusiasm drives robust adoption. We have a very sizable cash balance that will allow us to control our destiny and achieve sustained profitability with what we know are 2 dominant products in multibillion-dollar markets. A few key points to recap on Revuforj. Revuforj is the only FDA-approved therapy for KMT2A patients, and we have already identified and treated over 500 patients since launch, with 90% of those being on label. Physicians are treating earlier relapsed/ refractory patients, which portends more favorable outcomes. Revuforj is getting patients to transplant at an even higher rate than what was observed in our clinical trials. Physicians are eager to put their patients back on Revuforj post-transplant, and we have begun to see evidence this is happening. Further, all key indicators of demand remained strong in July, which gives us confidence in the continued momentum of this launch. Ultimately, in the future, KMT2A patients on Revuforj and with the aid of transplant, will likely remain on drug for 1 year or more, with the best hope of improved survival. In the near term, we are poised to expand into relapsed/refractory mutant NPM1 AML, pending the FDA's anticipated approval of our sNDA. Additionally, we are extending our leadership position to the front line with enrollment already under -- well underway in our first pivotal frontline trial. It is important to keep in mind that acute leukemia is an efficacy-driven market, and it is clear that Revuforj is a highly effective therapy with a favorable safety and tolerability profile. Finally, I will also note that we've retained worldwide rights to Revuforj and patent protection continues through at least the late 2030s. Turning to a few key points on Niktimvo. In collaboration with our partners at Incyte, the leaders in GVHD, the Niktimvo launch is off to an exceptional start. It is well positioned for growth in the $2 billion market for third-line chronic GVHD treatments with patent protection extending to the late 2030s. Niktimvo provides a novel option in a market that needs new mechanisms of action. Patients initiating therapy may continue drug for years. We and Incyte continue to advance development programs designed to bring this drug into earlier lines of chronic GVHD therapy and other diseases, starting with IPF. Niktimvo's financial contribution to Syndax is already profitable in its first full quarter. This will grow materially as sales ramp and operating margins continue to expand. Syndax has never been in a stronger position than we are today, and I look forward to sharing additional progress with you in the months ahead. As always, I want to close by thanking everyone who has supported us on this journey, including most importantly, the patients and families who have placed their trust in us, as well as our dedicated Syndax employees and long-term investors. And with that, I would like to open the call for questions. Operator?

[Operator Instructions] Our first question will come from Anupam Rama with JPMorgan.

Just wanted to ask a question about this path to profitability. Keith, I know you mentioned operating expenses staying stable over the next couple of years. But what are you assuming in terms of the top line, in terms of treatment settings for Revuforj and Niktimvo? Can you get to profitability on sort of the refractory settings alone? Is there some sort of assumptions on frontline expansion baked into getting to profitability? How should we be thinking about that?

Anupam, thanks for the question. We have been pretty consistent since November that we expect to get to profitability with the existing resources that we have today. And I would say the only thing that's changed since then is that we have 2 launches that are both outperforming our expectations. So the new disclosure that we provided today, stable operating expenses for the foreseeable future, say, the next 2 to 3 years, we're doing that because we want to give the buy-side and sell-side, we want to give the street the appropriate data for them to model our business better, so you guys can get to the same answer that we're getting to. We're not giving revenue guidance per se, Anupam, but I will say that given the time lines to get to approval in the frontline setting, you can definitely assume that we are getting to profitability on the relapsed/refractory indications alone. And I think, I just want to add, the guidance that we are giving, stable operating expenses, is not to be taken as we are taking our foot off the gas pedal, because we're not. The modeling that we've done allow us to fully invest in the continuing successful launches of 2 products, executing commercially, but additionally, executing our integrated clinical development plan, as Nick talked about, both for Revuforj and Niktimvo. So, I think we're in a pretty unique position to control our own destiny. We have 2 launches that are both outperforming and a stable expense base. And the team has worked extremely hard to put ourselves in this position to specifically reward our shareholders for their investment. So, thanks for the question.

Our next question will come from Corinne Johnson with Goldman Sachs.

This is Kevin Strang on for Corinne. I had a quick question on the patients moving on to transplant. After how many cycles is that typically occurring? And for patients that are going back on drug, you said that it was about 1/3 of patients so far. What are your expectations for the ultimate proportion of patients that will move on to maintenance therapy? And is this something you'll report quarterly?

Kevin, thanks for your questions. So, look, I think we're very encouraged by what we're seeing with patients going to transplant. As we had noted early on when we first started developing this drug, patients do respond very quickly to revumenib, and that's usually within the first few cycles. That generally is preceded by a transplant thereafter. And that transplant can happen very quickly. It can happen within a couple of weeks. That's usually for KMT2A patients, a goal to get them to transplant as quickly as possible. So, a few cycles, certainly 2 to 3, getting into remission, moving to transplant. That's how it generally works. We do see in our trial about 1/3 -- in our commercial experience, rather, about 1/3 of patients getting to transplant. We expect that number to accelerate. And the reason for that is we are treating patients earlier and earlier in the treatment paradigm. Physicians had told us they would put patients on Revuforj in sort of second line or first relapse. We see the vast majority of our patients being treated now in second and third line, which is a great outcome for them, generally means patients do stay on drug longer, do better and have a better chance of going to transplant. So we do think that, that 1/3 number could go up from here, and we expect it too. And ultimately, we will continue to track this over time. It's early days in the launch, and we do expect to report on that metric at some point going forward. And then in terms of maintenance, I think your last question, patients are coming back on maintenance. We know that and we reported that based on the earliest patients -- earliest cohort of patients that we've seen. About 1/3 of the patients have already come back. Again, early days of launch, that's a very good indicator. And physicians have told us repeatedly that they expect to put the vast majority of their patients back on maintenance, and that could range anywhere -- 70%, 80%, 90% of the patients, assuming that they're eligible for maintenance. And so that's, I think, a goal for us, and we'll see that play out over time.

Our next question will come from Kelly Shi with Jefferies.

So, after the second full quarter of launch, could you comment on the latest observation of treatment duration for Revuforj in real- world practice? And also, how do you expect the treatment duration to evolve over time, especially now when you have more earlier second lines of patients on the treatment?

Kelly, thank you for the question. So first question, duration in the real world, what are we seeing? I'm going to hand that to Steve to answer that.

Yes. Thanks, Kelly, for the question. And we'd always predicted this first year would be roughly in the 4 to 6-month range for average duration. Based on data that we've been able to see, we're very confident that's the case. We take a look at the earliest cohort of patients, and they're certainly within that range. That will improve over time. I appreciate the mention of the earlier line patients, which were also in our prepared remarks. And that's a real phenomenon. And this happened very, very quickly. The first patients at launch were not these patients. They were likely more third, fourth line patients, but it moved earlier very, very quickly, and that portends well on terms of treatment duration. So a better chance of success of getting to a transplant and more likely, as Michael just described on the previous question, the concept of returning to drug. So that will build over the course of this year. We would expect in 2026 that, that average treatment duration will be 6 to 12 months and could skew towards the latter end of that as the launch matures and we're able to move patients earlier and physicians gain more experience.

Just one more question, if I may. So on the cost side, how could we expect the change quarter-over-quarter for the rest of the year?

Thanks, Kelly. I'm going to ask Keith.

The cost side?

The cost, just change over quarter-to-quarter.

Yes. Yes. Kelly, thanks for the question. So we gave guidance today that we expect -- We changed the way we gave guidance actually. We used to give guidance with respect to OpEx inclusive of noncash stock comp, but we heard from investors that they are more focused on our cash consumption. So today, we changed the way we gave guidance to focus on our operating expenses less noncash stock comp. We said that for the third quarter, we expected that to be $95 million to $100 million and reiterated our full year guidance that we expect that to be now $370 million to $390 million. We implicitly gave fourth quarter guidance because we have 3 quarters of -- 2 quarters of expenses, gave third quarter guidance. So the fourth quarter guidance that is implicit through the math is almost exactly even with our third quarter guidance for our research and development plus selling, general and administrative expenses less noncash stock comp.

Our next question comes from Phil Nadeau with TD Cowen.

Congrats on the 2 successful launches. A couple of questions from us. First, on the KMT2A launch for Revuforj. You suggested, I think that 25% of patients with KMT2A for 1 year have initiated therapy in the second quarter, which suggests in the incident population the penetration is probably quite high. Can you give us a sense of where you think the penetration is in the incident KMT2A population here today in Q2 of 2025? And kind of where could that go at peak? I guess we're trying to understand how much growth could be left over the next couple of quarters before the label expansion? And then second, on the NPM1 label expansion, any update on inclusion of NPM1 in the NCCN guidelines?

Yes, Phil, thanks for the questions. The first question related to KMT2A and penetration in '25. I think we're going to clarify that. I'm going to ask Steve to clarify that a little bit.

Yes. Phil, thanks for the question. And, yes, I mean, so we've treated over 500 patients since launch. We often measure ourselves versus that overall available market of 2,000 KMT2A relapsed/refractory AML and ALL patients. So since launch, we estimated we've covered about 25% of that population. I think one thing to think about, it's 2,000 patients over the course of the year, not at any one point in time. There's going to be some variability as those patients are identified and diagnosed. We feel great about finding patients. Rev is very early to become the standard of care. Physicians are -- diagnostic testing is prevalent, so they're finding patients. And the -- over the course of the quarter, the number of new patients coming in has been strong and robust. So we expect that to continue for the rest of this year. We'd expect to finish the year roughly at 50% of the identified population. We think that would be a great launch, doing a lot of good for patients, but also really filling the funnel for Revuforj in our initial indication.

And then in terms of your second question, Phil, about label expansion NPM1, you had asked about the guidelines. Comment is, we've submitted to the guidelines. We published in Blood. The data is available, very helpful to our medical team to help educate in advance of launch. Guidelines, we don't have perfect information about when the guidelines will be updated. It could be any day. We do expect it before we get approval in NPM1, and that will help aid our launch even more by having guidelines. I think that's important for payers as well as physicians. So looking forward to that, but everything is all set up and ready for launch. We're just -- we're eager to continue to make progress.

Great. Can I just follow up on the first one? So with the 2,000 patients in the year, it's reasonable to assume 1,000 patients in 6 months. It's been 6 months since launch, 500 patients have started therapy. So that's 50%. You're suggesting 50% of the identified population by the end of the year. Obviously, more and more patients will go on over time, but this is a very sick population, so some are going to fall off. So are you kind of at peak penetration now and therefore, revenue growth over -- in the KMT2A population specifically, revenue growth over the next 6 to 12 months will be basically dependent on patients living longer and the duration of therapy increasing?

No, Phil, there's a lot of upside. So what we're seeing is over the first year, there's 2,000 eligible patients, we're going to get to 1,000 of them over the year, right, through the end of '25. We can go higher than that. So there is definitely some upside. I'd say we're not at peak penetration right now. There are more patients that will ultimately be diagnosed. We have a great deal of momentum executing at a very high level. But there's a lot of upside still on KMT2Ar. And then the next driver of growth on top of that is going to be NPM1, right, which PDUFA date in late October, and that will be the next leg of the stool. And that's obviously a much larger patient population. But that's how I think about growth on the new patient side.

Yes. And I would just add, Phil, obviously, other part of this is duration, as you brought up. The duration of therapy is going to be a key driver for KMT2A. As you treat patients earlier, more patients are going to transplant. We're seeing that evidence in our commercial experience and you're going to be able to put more patients back on therapy, we're seeing that early evidence as well. So we expect those to be major drivers year-over-year as you've added -- you're adding new patients, so the new patient starts plus the compounding effect of duration of therapy for these patients who come back and stay on maintenance. So, I think there's quite a bit of growth left to do.

Our next question comes from Peter Lawson with Barclays.

Congratulations on the quarter. Just as we think about the looming FDA approval, kind of -- what can you tell us around any remaining open items or feedback you've got from the FDA and kind of just -- anything that kind of helps add around the confidence around the FDA approval? And then kind of second question would just be around the maintenance setting and what kind of percentage of patients do you think you can eventually get on the maintenance setting?

Yes, Phil -- sorry, Peter, thank you for the questions. First, on FDA approval, I'm going to turn it over to Nick. Are we learning anything new?

Yes. Thank you. The submission is progressing very well. We have our PDUFA date. We've been working very closely with the FDA. And so it',s a team we know well now, and things are progressing very well according to plan. So we're looking forward to the PDUFA as guided on October 25th.

Yes. And is it -- with regard to your second question, maintenance, what percentage do we think we can put back on? Look, I think we had heard from physicians, we continue to hear from physicians, the vast majority, if not all of their patients, they'd like to put back on maintenance. We know that not every patient will be eligible for maintenance. But with physicians treating patients earlier and the majority of the treatment being concentrated in second line, even at this early stage of launch, that's a very good sign that physicians will drive hard to take more patients to transplant, which give us more opportunity to put them back on once they clear their transplant and graft. So, I can't give you an exact percentage, but it should be a very high percentage of KMT2A patients.

Maybe I could circle back on the first question just around the FDA. I know there's always a level of uncertainty, and it seems to be a heightened level of uncertainty. Have you seen any changes in that dialogue, any moving targets?

Yes -- Peter, no. I mean I think that's -- clearly, that's not what's -- there's nothing to indicate that it's anything other than really very good progress. We have priority review. We're under our tour. We're having consistent quality dialogue with the agency. The feedback has been very good. We have a PDUFA date that's coming close, and we're prepared for launch. So I think everything on the regulatory front is really hitting on all cylinders. No indication that it's anything other than that.

Our next question will come from Michael Schmidt with Guggenheim.

This is Paul on for Michael. I have 2 on the frontline combo opportunities. So first on the recent EHA updates from BEAT AML. It seems pretty clear that revumenib is enhancing the CR and MRD compared to ven/aza alone. But would love to get your thoughts on the degree of OS improvement you're seeing and whether or not you plan to provide another survival update in the study with additional follow-up? And then secondly, just looking ahead to the intensive chemo combo, can you sort of talk about how we should think about key CR and MRD benchmarks for that combo and sort of what to expect for the update later this year?

Paul, thank you so much for the questions. I'm going to turn it over to Nick to touch on the BEAT AML piece of this first.

Yes. Thank you for that. And firstly, very encouraged by the BEAT AML study. I mean, this was an important study. We were able to confirm a dose to take into Phase III and show that the dose was tolerable, and also, as you say, report out some early efficacy measures. And I think the efficacy measures that are probably most in this -- most important in this setting are the complete response rate and MRD negativity because remember, this is a relatively small 43-patient Phase Ib study. So interpretation in that context is quite difficult. And the CR rate and MRD negativity were very high. They were 67% and 100% MRD negativity, which, as I said in the earlier comments, are really a step change above what you expect from historical controls. Now, when you look at the overall survival, you have to remember that the median follow-up is quite short currently. The median follow-up was only around 7 months, and it's over 20 months in VIALE-A. So certainly, we expect as those data mature, you'll see some changes in the median OS. There's a lot of steps in the Kaplan-Meier currently, which suggests the median is quite unstable and it's therefore very difficult to estimate. Having said that, it's already very comparable or somewhat similar to VIALE-A, which you must also recall is a very heterogeneous group of subtypes of AML with a variety of different genetic mutations. And when you actually benchmark against NPM1, you probably find the median overall survival is a little less than was reported and closer to 10 months. So, in summary, we remain extremely confident in the profile of the combination with ven/aza. And I think it gives us a very high level of confidence that the EVOLVE-2 study that we're doing in collaboration with HOVON will read out well in due course. With regard the intensive chemotherapy and the MRD negative CR, we think both CR for unfit and MRD negative CR, both plasma and bone marrow are important endpoints in this setting. They've been shown to predict for improved outcomes around event-free survival and OS and believe that they could serve as surrogates to support accelerated approval. Those are obviously discussions we have had with the agency and have refined those. They are built into the protocols as dual primary endpoints, which means that they are independently powered. So you could have either the surrogate CR endpoint or the time-to-event endpoint, whether that be OS or EF, to give a positive study. And we remain quite confident that both of those should read out favorably. As previously indicated, we will be updating data for our combination with intensive chemotherapy for fit patients, specifically the 7+3 regimen from our own sponsored study, the 708 study. And also, we'll likely hear from the study we're doing in collaboration with the NCI, which is also a combination of intensive chemotherapy in the latter part of this year. And those data should both confirm the dose, tolerability, and also early signs of efficacy to support our Phase IIIs with intensive chemotherapy. So overall, we feel very confident about the programs, and we really have very good momentum going into the latter part of this year.

Our next question comes from Yigal Nochomovitz with Citi.

On the 1/3 of the patients that have restarted after the transplant and then the 2/3 that don't, could you just clarify -- so of those 2/3, are they not expected to restart? Or is it simply that they're not ready to restart and the expectation is that most of them, in fact, will restart?

Yigal, good -- thanks for the questions. It's the latter, right? So, clearly, we said early days, 1/3 have restarted already, which is very encouraging to us, which does leave 2/3 of those patients who could restart. And we expect a very high proportion of patients to restart out of that cohort as well. So it's an ongoing, evolving landscape of patients coming back from transplant and going on to maintenance. So we haven't excluded that 2/3. We actually -- we're waiting for those to come back.

Okay. And then just can you clarify on the mechanics? Do they need to get a reimbursement approval again when they come back after the transplant? Or is it seamless and they just start Revuforj again without a second need to request the reimbursement?

Steve, do you want to address that?

Yes. Our expectation, Yigal, it's pretty seamless. I mean there's typically -- in this industry, I mean, it's 6-month renewals, which are pretty standard. That may be the case here. But typically, if you're within that window, they'll restart without any challenges from payers. And even when there is a restart or a reinitiation of a prescription, we have not heard of any challenges with doing that. Payers covered the formulary coverages over 97%. Claims are being reimbursed on a regular basis. So we're not expecting any challenges with restarting at all.

Okay. And then lastly, I'm just curious if you could speak in a little more detail about this first wave of the real-world evidence that you're going to have at the end of the year? Can you just expand on that a little, please?

Maybe Nick can take that.

Yes, I'd be happy to. So we're obviously working with leading centers across the U.S. and leading thought leaders, and we're at the point now with the drug being used in clinic from commercial supply that we're getting some interesting series of data from physicians' experience in the real world. So we're collaborating closely with them to collect those data and look forward to presenting those real-world experiences from those centers in the latter part of this year. And I think we're uniquely well placed to be able to do that with this new therapeutic classes. We're now available commercially in the clinic and can actually report real-world experience versus just clinical trial experience. So I think those data and how the drug is getting used in the real world will be very insightful, and we're looking forward to those reporting out later in the year.

Our next question will come from Justin

Congrats on the strong quarter. So looking ahead to the October 25th PDUFA date for NPM1 label expansion, could you walk us through how you're preparing the commercial organization for launch readiness? And do you anticipate a meaningful incremental uptake in the population out of the gate or would it be more gradual?

Yes. Thanks, Justin, for the questions. I'm going to turn it over to Steve to touch on the launch readiness, launch maintenance.

Yes. So we're in market, Justin. I appreciate, obviously, with another indication, which would be different from someone who's entering the market. So here's how I would think about this launch. I mean part of the success or the preparation is doing a great job right now, right? So physicians, we're leveraging that market experience in KMT2Ar. Patients understand how to dose the drug. They appreciate the dosing options that they have, how do they initiate treatment, how to manage any AEs that might occur in treatment initiation, getting through them, what do they expect from efficacy and how to bring the drug in, right, how do they -- as a treatment center prefer to bring the drug in. So that's one piece. I think the other piece is we're in the same audience, right, right now. So when we think about treatment centers that treat KMT2Ar, they are the same ones that treat NPM1. So we're already there. We've got a best-in-class customer-facing team. They have excellent relationships. They understand the space. They understand how these treatment centers treat. So they've already got a leg up at once the indication is granted on the sNDA. And the last piece is just a great drug, right? We believe we've got a best-in-class profile with NPM1 data. Any physician would tell you most important across any of these types of agents is does the drug work, and does it work better than anything else that's out there? We believe we've got a winner in Revuforj. The efficacy data really screams, physicians tell us that. I think as Michael may have pointed out in his comments, the fact that we've got multiple indications for menin inhibitor, that is a big deal. That is not something that's minor. And that holds for treaters. It also holds for payers, right? Payers when looking at a second indication, like they are with Revuforj, it's an easy add and that will get us ahead. So those are the things I think about. In terms of uptake, absolutely, we're expecting a bump, right? We know that right now the usage outside of KMT2Ar is small. We maintain -- it's about 10%. We'll call it spontaneous or off-label use. The biggest bang for NPM1 is going to be at the indication granting, right? When promotionally and commercially we can stand behind the drug, we think it will make a big splash. Physicians are ready for it. So we'll expect a decent driver at that time later this year.

Yes. I'll just add that adding NPM1, you take your patient population from about 2,000 patients to 6,000 patients, that's a big difference. And so we expect it to be a really important driver, not only starting at the end of the year, but going into next year and beyond. It's important to be first. It's important to have the best profile. We have both. So we're in good shape.

Our next question will come from Salim Syed with Mizuho.

Congrats on the quarter. Mike, Keith, maybe just a couple from us. One on Niktimvo. So -- and I apologize, this is going to be another math question. But when I look at 2026, consensus currently, I think, is around $240 million or so on the end user sales number. And just kind of like looking at some of the numbers that have been released between yourselves and Incyte, there were $45 million of sales, I think, or so ex inventory, that's our number, I think, 4,500 infusions. I think they mentioned that on their call. I know you guys are saying over 4,000, but they said 4,500. So it looks at about $10,000 net price per infusion, assuming the trial duration of 10.3 months. You start to get to this price of $225,000 for the 22 infusions that would take place, so assuming they talked about having 1,000 patients at the end of this year, perhaps. So you start to get to these numbers of $225,000. And again, that's using the 10.3, not the 12-month duration, no additional inventory impact, no additional penetration, no growth. Is it just me? Or is that number just incredibly light, the [ 26 ] Niktimvo end- user sales number just based on that math? Is there something I'm missing?

Thanks for the question, Salim. So we're trying to track with your math here. But Keith, why don't you comment?

Yes. Trying to track your math. I mean, I think going back to some of the comments that Mohammed and Bill made on the call that Incyte had last week, I think they were asked a question about peak sales, and there was a response that included an estimate of looking at the RE

Okay. Yes. I mean they did talk about the [ $1,000 million ] sort of at the end of the year from the current [ $700 million ] or so. That's sort of the basis for the math. But I understand your point. And just quickly, I guess, on your slides, it looks like you updated your EPi data for the NPM1 from 3,000 to 4,500 just to 4,500. Is there something you guys did on the Epi to make you more confident around the upper end of that range? The one that's $5 billion TAM?

Yes. So thanks for the question. Look, the only thing that -- a lot of things give us confidence. I think when we think about the EPi, I mean, we're treating patients now really much more in the second line. So you're going to capture the upper end of that number if you continue to treat in that capacity. So that's how physicians tell us they want to treat. They want to treat earlier. And so that gives us confidence to kind of capture the upper end of that range. Just making a comment, I want to go back for a second to your Niktimvo question. You mentioned duration at 10 months. I would just point to the fact that this is a drug that physicians intend to keep patients on potentially for years. I mean this is a very efficacious drug. And so, we believe that there's potentially a lot of upside in duration of therapy. And so I would be thinking about your assumptions there, and we'll track with that, obviously, over time. But I think that's a -- one thing that stood out to me in your math that you might want to take a look at.

Yes. And so...

I meant to be conservative, yes.

Yes. No, I'm just -- again, I think it's -- as a conservative estimate, I understand where you're coming from, but I think we are encouraged by duration here and what this category and what specifically this drug and mechanism can bear. So I would just pay attention.

Especially 5 months into launch, Incyte made comments that 80% to 90% of patients that started on therapy are still on therapy.

Very good point.

Our next question will come from David Dai with UBS.

Congrats on the quarter. A couple for me. One, just on the $28 million of Revuforj revenue, could you talk about the percentage of revenue is inventory? And also, how much of that is coming from new patient start versus refill?

Thanks, David.

Yes. So David made comments that I made -- said that only -- well, I'm sorry, let me back up. The demand in the quarter was driven by patient growth, patient demand. Inventory stayed level, 2 to 3 weeks, and we expect that to be the case going forward. It's pretty typical of a drug that's used and distributed using specialty pharmacy specialty distributors, 2 to 3 weeks of inventory. So we don't expect that to grow.

Got it. Okay. And then just another question on the patients on the stem cell transplant. And right now, you have about 33%, 1/4 of it or 1/3. I'm just curious, do you expect this to increase given that you have 50% of patients currently treated in the second line? So how should we think about this going forward in terms of the increase in stem cell transplantation?

David, thanks for the question. We tried to get at this a little earlier. Look, I think the transplant rate at 1/3 -- remember, in AUGMENT-101, we were at about 1/4 of patients going to transplant. Now we're at about 1/3. We're treating patients a lot earlier instead of third and fourth line, we're more second and third line. 70% of our prescriptions are in that second and third line. Transplant rate could go 50% higher. We don't know. But we are expecting it to materially change over time and get better. And what gives us confidence is that we know if we treat patients earlier, they tend to get to a higher level of response, higher rate of response and are more eligible or will be eligible for a transplant. So I think that's what's giving us confidence. We don't have the upper bound of that, but we do expect it to grow over time.

Our next question will come from Jason

A couple from us, if we may, based on your earlier comments. But first being, of the 2,000 or so relapsed/refractory KMT2A patients, can you give us any color on your assumptions regarding the overall peak penetration here? And then similarly, given the comments regarding the overall opportunity in relapsed/refractory, at least as far as your cash runway goes, any color on your insights or assumptions as far as the competitive split in the NPM1 population look like?

Thanks, Jason. Thanks for the question. So first question related to peak penetration, our estimates on peak penetration for the 2,000 KMT2A patients. Steve, do you want to take a shot at that?

Yes. I think we can -- as we've said, we've already covered 25% of the population. We'll get to 50% of that. There's some upside to that. We haven't guided to a number on peak. But I think we'll be at levels at 1,000 that are close to the upper end of models that some of you may have, but we think there's some beyond that. Treating earlier, I think, as Michael said, brings more people in. And just remember, Jason and -- even that in KMT2Ar, there's no one in the near term coming to market. So it is really white space and Rev is already the standard of care after just 7 months on market and physicians are using it that way.

Yes. Jason, I would just -- I would say there's -- penetration into a market never happens within 1 year. It always takes an oncology more than that. And I think we expect, as Steve said, within 1,000 patients this year. So we have more work to do next year and the year beyond. So that's, I think, just in terms of new patient starts. In terms of really building this market, I'll say it again, duration of therapy is going to be key, right? Physicians treating earlier, we know that, that's going to give us the best outcomes. Patients are going back on therapy post-transplant. That will ultimately compound the revenue as it goes forward, patients staying on drug for long periods of time. So, I think those are the real drivers, those 2 things. It's actually pretty simple. But that's -- it's not just about how many patients you can penetrate on KMT2A. It's how long they stay on drug as well. And then overall opportunity, you had mentioned the second question being, what do you -- what's your estimate for competitive split in NPM1. Look, I think we're first to market. We know we have best profile. I think the data speaks for itself. We're feeling very confident about that. We would expect to have a significant percentage of that market and dominating NPM1 as well as KMT2A. So we feel very positive about the fact that we're entering the market first. And that's essentially how we see it. So we're not -- we can't guide exactly to what the split would be, but we would expect to have a big share.

Our next question will come from George Farmer with Scotiabank.

This is Chloe on for George. A couple from us. Can you talk a little bit about the monitoring that's happening in the real world right now for potential cardiac AEs, how they're being managed? And to the extent that you're privy to this information, what percent of these cardiac AEs, like the QT prolongation, is due to revumenib as opposed to any concomitant medications? And how many of those have resulted in discontinuations in the real world? Then number two, if you could give some color on how to model the royalty pharma interest expense in the P&L moving forward, that would be super helpful. And the last one on Niktimvo. In IPF, could you please speak to the unmet need, how big that opportunity is and how you're thinking about positioning in a market that's dominated currently by generics?

Chloe, thanks for your questions. So I counted 3. So let's start with the first one. Nick will take that. How is monitoring being done?

Yes. Thank you for the question. So firstly, we've shown in our extensive clinical trial experience now that management of QT is done very simply. There's clear guidelines in the label now for QTs in the first month. We know that almost all patients that have any sign of prolongation happens early and is monitored and managed appropriately. That's very consistent with our real-world experience. As I said, we'll be reporting on some of that later in this year. We, of course, have very extensive safety surveillance systems in place. But we're really not hearing any evidence of concerns with standard guidelines and management of those patients. So, things are progressing very well. The physicians are very happy with the profile and not flagging any concerns.

Great. And Keith, do you want to talk about the royalty, please?

Yes, Chloe. So with respect to your question about how to model the Niktimvo. So we've already achieved profitability. First full quarter sales, we mentioned that $36 million in net revenue, we reported $9 million in collaboration revenue. So we're already in a range of 25% to 30% from a gross contribution perspective. And like I said in my prepared remarks, we only expect that to grow. With respect to the royalty, it's really easy. It's simply 13.8% of the net revenue that is reported by Incyte. So in second quarter, that represents $5 million in royalties paid on $9.4 million in collaboration revenue. We're using the effective interest rate method. So the cash paid to Royalty Pharma won't exactly match what we report on the P&L.

And then lastly, I think you asked about IPF. So, IPF, very important indication for us. We have a Phase II trial ongoing. We expect to fully enroll that trial this year, data next year. That's a big market opportunity. As you pointed out, there are some entrenched competitors, different mechanism of action brought by this drug, we think a very impactful one, about 150,000 patients into the market in the U.S., 280,000 worldwide. So it's a big market. Patients are still in absolute need of new therapy, and we think we bring that with Niktimvo potentially with this mechanism. So we'll have those trial results, and we think we will meaningfully differentiate over time if those are obviously positive. So more to come on that, but it's a -- we think a very big opportunity for us to exploit as a second indication.

Our last question comes from Mayank Mamtani with B. Riley Securities.

Congrats on the progress. Could you just clarify, and sorry if I missed that, if there's any real-world evidence you're planning to generate as you strengthen the case for Revuforj use as maintenance therapy post-transplant? And what's your expectation for duration of therapy there, say, relative to the 4 to 6 months you're seeing in pre-transplant? And I have a quick follow-up.

Yes. Thank you. Thanks so much for your question. I'm going to let Nick address the real-world part.

Yes, we'll be presenting some relatively preliminary data from the series we've been monitoring towards the latter part of this year, second half of this year, we're looking forward to presenting. That will obviously include patients' demography, how they do on therapy, and importantly, also the proportion of patients that go on to transplant in the real world from these series and also those patients that then start on Revuforj after transplant. So I think those will be important data. Obviously, as time goes by and our commercial experience increases, we'll be able to add to those data. But I think it will be interesting preliminary data, and we'll look forward to reporting those out. And I think they'll shed some light on some important questions.

And I'll just make a comment, I think, as you asked about what our assumptions are on post-transplant maintenance, what percentage of patients are likely to go on to transplant and then, of course, on what time frame. And I think we commented earlier about high percentage of patients going to transplant. I think that's obviously very clear to us now and exciting. And then in terms of staying on therapy, physicians have repeatedly told us, 1 year to 2 years -- is what I said in my prepared remarks, 1 year to 2 years is pretty consistent. Could be longer. Some physicians say that they will keep them on indefinitely without a real compelling reason to take them off. And obviously, these patients are at high risk of relapse. So you want to keep them in remission as long as possible. That's the key goal. So I think an assumption you can make on average for maintenance that physicians are thinking about 1 year to 2-year time frame.

Very helpful. And then on Niktimvo, can you just remind us what development milestones to look out for as you think about the therapy moving into earlier line GVHD? I believe the label is third line, fourth line, but the uptake is in earlier lines. But I was curious if any additional clinical data milestones we should be on the lookout for?

Yes. Thank you. Nick, do you want to take the question?

Yes. And just to recap, and we haven't guided specifically on the time lines. We have 2 important studies in the frontline setting, one in combination with dexamethasone. That's a Phase III with registrational intent. Again, we haven't guidelines specifically on when that study will read out. And an important Phase II study as well in combination with Jakafi. And then obviously, our IPF randomized Phase II study, which is around 135 patients, we're anticipating we'll complete enrollment towards the latter part of this year and data in the second half of next year. So a number of important milestones reading out both in earlier lines of GVHD and in IPF.

This concludes our question-and-answer session. I will now turn the floor over to Mr. Michael Metzger for any additional comments or closing remarks.

Thank you, all. We appreciate you all tuning in today to discuss our recent progress and the exciting milestones ahead. We look forward to seeing many of you at several investor conferences and events in the third quarter. And with that, have a great evening.