SNDX

Good day, everyone, welcome to the Syndax Second Quarter 2026 Earnings Conference Call. Today's call is being recorded. If you'd like to ask a question following the company's prepared remarks, please press star five during the call. At this time, I would like to turn the call over to Sharon Klahre, Head of Investor Relations at Syndax Pharmaceuticals.

Great. Thank you, operator. Welcome, thank you all for joining us today for a review of Syndax's second quarter 2026 financial and operating results. I'm Sharon Klahre, and with me this afternoon 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, and Keith Goldan, Chief Financial 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 two. 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 of 1995.

Actual results may differ materially from those indicated by these 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, August 4th, 2026 only. A replay of this call will be available on the company's website, www.syndax.com, following its completion. With that, I'm pleased to turn the call over to Michael Metzger, Chief Executive Officer of Syndax.

Thank you, Sharon. Good afternoon, everyone, thank you for joining us. Starting with Slide three. The Syndax team delivered another quarter of solid commercial results and progress across our growing pipeline of programs targeting areas of high unmet need and substantial commercial opportunity. The business fundamentals are strong. Sales of Revuforj and Niktimvo grew to a combined total of $115 million in the second quarter. Both medicines are now annualizing at well over $200 million each, and we have just started to unlock their multibillion-dollar potential. Revuforj continued to grow by double digits, with net revenue totaling $55 million in the second quarter, up 91% year-over-year and 12% quarter-over-quarter.

These results highlight our continued leadership in menin inhibition, robust demand in both NPM1 and KMT2A, and the positive impact of an increasing average treatment duration driven by multiple factors, including a growing number of patients on therapy for an extended period after receiving a stem cell transplant. As the number of patients on therapy post-transplant continues to stack, this reoccurring base will be an important driver of long-term compounding growth. Encouragingly, the average duration of treatment is also increasing among patients who are not proceeding to a transplant. As Steve will describe shortly, the positive impact of an extending average treatment duration, which was partially offset this quarter by fluctuation in the number of new patients initiating therapy, one of several drivers of our business. Today, we are even more confident in the forward trajectory and substantial commercial opportunity with Revuforj.

Our conviction is underpinned by the multiple drivers for continued growth, including a best-in-class profile valued by physicians, an increasing average duration of therapy, a broad and expanding prescriber base, and ample opportunity in NPM1, KMT2A, and other menin-dependent acute leukemias. We are well-positioned to extend our leadership in menin inhibition into the future and continue driving innovation for patients. Building on a long history of landmark firsts, we are positioned to be first to frontline AML, driven by strong global site initiation and patient enrollment in our pivotal trials. With future anticipated indications in frontline AML, we expect that Revuforj could reach in excess of $2 billion in peak annual net revenue in the U.S. alone. We have another data-rich period ahead for revumenib.

In the second half of the year, we will report additional practice-informing evidence from multiple trials at major medical meetings, expanding on the prominent presence we had at ASCO and EHA in June. We also expect to publish data in relapse refractory NUP98-rearranged acute leukemia in the fourth quarter. These results could inform clinical practice and guidelines in a patient population similarly sized to KMT2A. Patients with NUP98 urgently need new treatment options, including therapies that may reduce the risk of relapse after transplant, as is the case with KMT2A. Physician feedback indicates that NUP98 rearrangements are more common than once thought, occurring in perhaps 5% or more of AML cases, translating into potentially 1,000-2,000 pediatric and adult patients with NUP98 annually.

As the first and only company to report clinical data showing activity with a menin inhibitor in this subtype, we have a unique opportunity to pursue a guideline listing that could meaningfully expand the patient population treated with revumenib. Switching gears to Niktimvo and chronic GVHD. Niktimvo net revenue grew to $60 million in the second quarter, up 67% year-over-year. This performance reflects robust demand and Niktimvo's unique ability to address inflammation and fibrosis. Niktimvo is positioned for further growth with strong adoption in the fourth line and increasing uptake in the third line, driven by a broad base of prescribers who are enthusiastic about the results they've seen in their patients. We also have multiple expansion opportunities and important upcoming catalysts for axatilimab with phase II data in IPF and frontline chronic GVHD expected in the fourth quarter, poised to unlock new multi-billion-dollar opportunities.

Turning to our pipeline assets. At our R&D event last month, we unveiled two innovative assets we are advancing into the clinic, leveraging our world-class R&D capabilities and experience taking revumenib and axatilimab from IND to FDA approval in about five years. SNDX-4321 is a novel mutant-selective CNS penetrant allosteric EGFR inhibitor for non-small cell lung cancer that offers a new approach to addressing patient populations with high unmet medical needs, such as those with L858R mutations and CNS metastases. SNDX-62122 is a next-generation menin inhibitor we are developing for myelofibrosis, or MF, building on our extensive experience pioneering menin inhibition in hematology. It is the first molecule from our internally developed and wholly owned library of next-generation menin inhibitors, which we intend to advance into promising new areas.

We are entering into another exciting chapter for the company as we build Revuforj and Niktimvo into major commercial franchises and leverage our proven R&D engine to bring new treatment options to even more patients. We are fully funded to execute on our commercial and R&D priorities and continue to advance towards profitability with growing revenue from the first two medicines from our pipeline. I will now turn the call over to Steve to discuss our commercial results in more detail. Steve?

Thank you, Michael. Starting with Revuforj on slide four. We delivered our sixth consecutive quarter of double-digit Revuforj net revenue and prescription growth and continue to track well above launch benchmarks set by other mutation-directed AML therapies. Net revenue totaled $55 million, up 12% from the prior quarter. Total prescriptions were approximately 1,500, up 15% from the prior quarter. These results reflect robust demand and an increasing average duration of therapy, one of several important drivers of our business. We have dominant share of the overall menin business today, having treated over 1,600 patients commercially since launch, including 250 new patients added in the second quarter.

We saw some fluctuation in new patient starts in Q2 relative to prior quarters, reflecting typical variation quarter to quarter in the number of available patients with a rare disease and market dynamics when physicians have more than one drug in class they can consider using, depending on the patient's mutational profile. To ensure we leave no appropriate patient behind, we have optimized our established customer footprint and targeting, expanded our ability to leverage lab data to engage physicians when they have a suitable patient in their care, and increased our promotional efforts and educational activities. We remain confident we will continue to lead this market with the strongest efficacy profile in an efficacy-driven market and multiple drivers supporting long-term growth. Our business in KMT2A and NPM1 is strong, and it is growing.

Revuforj is the standard of care for relapsed/refractory KMT2A translocated acute leukemia and remains the only targeted therapy for an aggressive cancer with no other effective treatment options. We continue to expand into our second indication, with NPM1 accounting for at least 40% of new patients in the second quarter and more than 30% of the $55 million in net revenue. All indicators suggest Revuforj will continue to be the menin inhibitor of choice for all menin-dependent acute leukemias. Physicians value having one efficacious and well-tolerated drug they can use across multiple acute leukemia subtypes in both adults as well as children. They appreciate that the efficacy they see with Revuforj in the real world is consistent with, or in fact, even better than the clinical trial results.

They value individualized dosing, not having to worry about reduced efficacy when their patients are taking commonly prescribed gastric acid-reducing agents like PPIs and H2 blockers, and the lack of any clinically meaningful pruritus, an adverse event that can be impactful for patients and very difficult for clinicians to manage. This combination of efficacy, tolerability, and dosing flexibility is why Revuforj is and will remain the drug of choice for physicians. Turning to slide five. There are two fundamental drivers of our business, new patients and average treatment duration, and both are building. The unique breadth of our indication provides us with the opportunity to target approximately 2,000 patients diagnosed annually with relapsed/refractory KMT2A translocated acute leukemia, plus 4,500 with relapsed/refractory NPM1 mutated AML.

We've made excellent progress reaching this population, with more than 1,600 patients treated with commercial drug since launching in KMT2A in the fourth quarter of 2024 and NPM1 in the fourth quarter of last year. Importantly, there's still plenty of room to reach more patients each quarter. For instance, of the annual 4,500 relapsed/refractory NPM1 patients, we estimate that less than 15% have received a menin inhibitor, highlighting the substantial opportunity for further growth. Compared to KMT2A, where we saw a steep uptake curve due to the lack of other approved or impactful therapies, we expect our NPM1 business will build over time due to other options that physicians may consider for this population, depending on their co-mutations or other factors.

The second fundamental driver is average treatment duration, which is increasing due to evolving clinical practice and a product profile that is conducive to patients staying on therapy for extended periods of time. Physicians are reaching for Revuforj early in the relapse refractory treatment paradigm, and are often choosing to use it in combination with other therapies, with the goal of driving responses and extending the duration of effect. Claims data shows 75% of use in the second and third line, and approximately 40% of use in combination. Encouragingly, a significant proportion of patients are proceeding to stem cell transplant after receiving Revuforj, which is the goal in the relapse refractory setting for both KMT2A and NPM1 patients who are fit enough to receive a transplant. We continue to observe approximately 50% of KMT2A patients proceeding to transplant.

About 50% of those patients have resumed therapy thus far, after pausing for three to six months, up from an estimated 45% last quarter. We expect this % will continue to increase as our colleagues in medical affairs report additional evidence from the post-transplant setting in collaboration with leading treatment centers, building on the encouraging data MD Anderson presented at ASCO and EHA this past June. Over time, we expect that up to 70%-80% of transplant patients will ultimately return to therapy for one to two years based on feedback from physicians and clinical trial and real-world experience. These evolving treatment patterns are increasing the average treatment duration, especially the growing number of patients on therapy post-transplant. This group is already averaging at least nine months of therapy, with this duration expected to steadily increase as we continue to follow patients over time.

Among patients who do not receive a transplant, over half are still staying on therapy for a significant period, with an average treatment duration that is already over seven months and building. With a significant addressable patient population and an increasing average treatment duration, we are confident in our ability to build a sustainable business with our first two indications for Revuforj. Moving to slide six. We have a solid commercial foundation in place to support the success of Revuforj, including a highly accomplished team with deep and strong customer relationships. Our already robust prescriber base has continued to expand quarter-over-quarter, including our activation of Tier 1 and Tier 2 accounts, the highest volume centers in the U.S., who treat two-thirds of our target population. Nearly 90% of these accounts have ordered, up from 70% prior to the approval of Revuforj in NPM1.

Overall, more than 580 accounts have ordered Revuforj, up 11% from the prior quarter, reflecting growing adoption from centers of all sizes, including community practices. Our growing prescriber base reflects physicians' enthusiasm for Revuforj and positions us to drive further penetration for both indications. We have excellent payer coverage, and physicians can access the menin inhibitor they prefer. As of the end of Q2, Revuforj's formulary coverage was 98% of all covered lives for both indications, a coverage position that leads the class. In addition to having nearly 100% formulary coverage, Revuforj has preferential coverage on plans representing 17% of all covered lives versus less than 2% of lives for the other menin inhibitor. Turning to Niktimvo on slide seven. Niktimvo net revenue totaled $60 million in the second quarter, up 67% year-over-year and 9% quarter-over-quarter.

This result reflects strong and consistent new patient starts and solid persistency. More than 300 new patients were added, and about 5,750 infusions were administered in the second quarter. Niktimvo is annualizing at $240 million and continues to track with the launch of REZUROCK, a drug that reached $500 million in annual U.S. net sales within the first four years of launch in the same indication. Moving to slide eight. The fundamentals of our Niktimvo business are strong, with multiple drivers for continued growth. The first is continued adoption in the fourth line and steadily increasing uptake in the third line as clinicians gain experience with Niktimvo. Within one and a half years of launch, Niktimvo has captured approximately one-third of the third-line-plus chronic GVHD market. As the patient mix shifts more towards patients with less advanced disease, we expect this will extend the average treatment duration.

This is a chronic disease with the potential for patients to stay on therapy for long periods. We've observed solid persistency in the commercial setting, with 60%-70% of patients staying on Niktimvo for at least 12 months. Our clinical trial experience suggests the duration of therapy could be measured in years for a meaningful proportion of patients. Our Niktimvo business benefits from a broad and productive prescriber base and commercial synergies for both Syndax and Incyte. Nearly every bone marrow transplant center in the U.S. has prescribed Niktimvo and become a repeat customer. Physicians continue to report impressive activity in multiple organs, with particularly notable responses in the lungs and skin, some of the most difficult to treat organs. All these drivers position us to expand our impact in third-line-plus chronic GVHD, a $2 billion U.S. market opportunity.

Looking ahead, the ongoing trials in front-line chronic GVHD and IPF could unlock additional multi-billion dollar opportunities. With that, I'll hand the call over to Nick to talk about our development programs.

Thank you, Steve. Turning to slide nine, I'd like to highlight the progress we've made advancing our scientific leadership in menin inhibition with a strong presence in medical meetings and two landmark publications. At ASCO, we had four revumenib abstracts, including an oral presentation of post-transplant maintenance data from a cohort of heavily pretreated patients with KMT2A/NPM1 or NUP98 alterations who resumed revumenib post-transplant. This analysis showed a two-year overall survival rate of 90%, which is nearly double the historical rate observed prior to the introduction of revumenib. Moving on to EHA, we had 12 revumenib abstracts highlighting the clinical activity with revumenib in multiple acute leukemia subtypes and settings, including in combination with standard of care therapies in the frontline and relapsed/refractory setting. In June, data from the relapsed/refractory cohort in the Phase I/II SAVE trial were published in the "Journal of Clinical Oncology".

The results observed with the all-oral combination of revumenib, venetoclax, and decitabine/azacitidine in a heavily pre-treated population of patients with NPM1, KMT2A, or NUP98 AML are impressive. 88% achieved an overall response. 80% of evaluable CRc responders achieved MRD negativity, and 45% of patients proceeded to transplant, with 63% resuming revumenib post-transplant. These results are similar to outcomes observed in real-world cohorts treated with revumenib-based combinations, underscoring the potential to reproduce these results in clinical practice. In July, we and our collaborators published the results of groundbreaking preclinical studies with revumenib, which showed menin is a novel dependency in a proliferative megakaryocytes, major drivers of myelofibrosis. These data provide the basis for our innovative clinical development program in myelofibrosis.

Looking ahead to the second half of the year, we will deepen our scientific leadership with new revumenib data from across the acute leukemia treatment continuum, including from the maintenance and real-world setting. We also look forward to presenting more mature frontline data in the fourth quarter, with updates expected from SAVE and BEAT AML trials of revumenib with low-intensity chemotherapy and from the 708 trial with intensive chemotherapy. We expect these data will be impactful for the clinical community and provide strong support for our ongoing pivotal frontline combination trials. We also expect to publish data in relapsed/refractory NUP98-rearranged acute leukemia in the fourth quarter, followed by submission of this important publication to the clinical guidelines for consideration.

Patients with NUP98 rearrangements have poor outcomes. There is an urgent need for new treatment options, with many parallels to the unmet needs in KMT2A prior to the introduction of revumenib. Physicians are positive about the potential for revumenib to provide a new treatment option for this disease subtype based on the data reported. At EHA, we presented data from 25 heavily pre-treated relapsed/refractory NUP98 patients showing a 28% overall response rate, with 43% of responders proceeding to a transplant and all resuming revumenib post-transplant. Turning to our pipeline on slide 10, I'd like to highlight just a few key points. First, we are rapidly advancing an integrated evidence generation plan designed to establish revumenib as the menin inhibitor of choice across the acute leukemia treatment continuum. Importantly, we maintain good momentum to be the first to deliver pivotal frontline data for a menin inhibitor.

Global site activations and patient enrollment is well established in our pivotal trials, informed by an extensive body of clinical evidence, allowing for optimization of endpoint assumptions and other aspects of the study design. Second, we are nearing two important axatilimab readouts. We are on track to report top-line data from the phase II MAXPIRe IPF trial in the fourth quarter. Positive data would open a transformative opportunity for axatilimab and provide a strong biological rationale for its mechanism in several other diseases where the pathology is underpinned by fibrosis and inflammation. We also anticipate top-line results from the phase II trial of axatilimab in combination with ruxolitinib in frontline chronic GVHD in the fourth quarter. Positive outcomes with the novel combination could begin to unlock the potential for a steroid-sparing regimen in newly diagnosed chronic GVHD.

Third, as highlighted at our recent R&D event, we announced two new pipeline assets, SNDX-4321 and 62122. In line with our focused R&D strategy, these are targeted molecules both with first and best-in-class potential, supported by compelling preclinical data, mechanistic insights, and advocacy of leading researchers and clinicians. With both revumenib and axatilimab, we have demonstrated our ability to efficiently generate clinical data that validates new therapeutic targets, leading to new approvals, a strength we will leverage again as we advance the next chapter of our R&D strategy. Turning to slide 11, 4321 is a novel mutant-selective, CNS-penetrant allosteric EGFR inhibitor. We're developing it for non-small cell lung cancer patients with high unmet needs, such as those with the L858R mutations, CNS metastases, and atypical activating mutations or resistance to current therapies.

In contrast to ATP site-directed third and fourth-generation EGFR inhibitors, 4321 binds at a pocket adjacent to the ATP site that is only accessible in the presence of L858R and certain other EGFR mutations. The allosteric approach allows for high selectivity and a double lock approach to inactivating the receptor. 4321 and ATP site-directed therapies combine EGFR at the same time at different sites on the receptor, potentially enhancing efficacy and delaying resistance. We expect to submit an IND for 4321 by the end of 2026, with an assessment of monotherapy activity expected in early 2028. 62122 is our next-generation menin inhibitor in development for myelofibrosis that could offer a novel and potentially disease-modifying approach. It is the first candidate from our library of internally developed and wholly owned next-generation menin inhibitors that we plan to advance into new areas.

We expect to submit an IND and initiate Phase I trial of 62122 in 2027. This program will be informed by a proof-of-principle trial of revumenib in myelofibrosis that will be conducted in partnership with world-leading experts in myelofibrosis. We expect this trial to initiate in the fourth quarter, with initial data anticipated in the second half of next year. In summary, we've made enormous progress advancing our late-stage trials and expanding our pipeline of differentiated assets. We are nearing several important data readouts and have multiple opportunities to transform the standard of care for some of the most difficult-to-treat diseases. With that, I'll turn the call to Keith to discuss our financials.

Thank you, Nick. Pardon me. Earlier this afternoon, we reported detailed second quarter 2026 financial results, and I'll highlight a few key points on slide 12. Total revenue for the second quarter of 2026 was $72.8 million, up 92% over the same period last year. This consisted of $54.7 million of Revuforj net revenue and $18.1 million in Niktimvo collaboration revenue, which equated to 30% of the Niktimvo net revenue reported by our partner, Incyte, in the quarter. We expect Niktimvo margin contribution, defined as a collaboration revenue recorded by Syndax as a percentage of Niktimvo net sales, to continue to be in the 25%-30% range in the near term and increase longer term as sales grow. We expect revenue from both Revuforj and Niktimvo to continue growing and advancing the company towards profitability.

Our guidance for R&D plus SG&A expenses in 2026 remains approximately $400 million, excluding the impact of $550 million in estimated non-cash stock compensation expense. We ended the second quarter with $575 million in cash equivalents and investments. This includes $244 million in net proceeds from the issuance in June of $250 million of 2.25% convertible notes. That deal provided the lowest cost of capital that Syndax has ever accessed and positions us well to build shareholder value over the long term. We're fully funded to execute our commercial and R&D priorities, including our late-stage trials of revumenib and axatilimab, and the development of our pipeline assets. With relatively modest investments and proof-of-principle trials of 4321 and 62122, we believe we can quickly generate early clinical data that creates significant value for the company and its shareholders. With that, I'll hand the call to Michael for closing remarks.

Thank you, Keith. Syndax is well-positioned for long-term growth and success, with multiple blockbuster opportunities and upcoming milestones as highlighted on slides 13 and 14. We have a best-in-class menin inhibitor that is positioned to be the first to frontline AML and deliver peak annual net revenue in excess of $2 billion in the U.S. alone. We are nearing data readouts in the fourth quarter that could unlock multibillion-dollar upside for Niktimvo in IPF and frontline chronic GVHD. We have a proven track record of successfully developing and commercializing novel medicines and are bringing forward two new pipeline assets with best-in-class and blockbuster potential in EGFR-mutated lung cancer and myelofibrosis. With $575 million on the balance sheet and growing revenue from two products, we are funded through profitability and have the capital to realize our pipeline opportunities and drive significant long-term value.

I will close by thanking our dedicated employees and the many patients, clinicians, and scientists who support our work and inspire us to pioneer bold, new approaches to some of the most devastating diseases. With that, I would like to open the call for questions. Operator?

At this time, I would like to remind everyone in order to ask a question, press star, then the number five on your telephone keypad. If you'd like to withdraw your question, press star and the number five once again. We'll pause for just a moment to compile the Q&A roster. The first question is from Anupam Rama with JPMorgan. Your line is now open.

Hey, guys. This is Joyce on for Anupam. Thanks for taking our question. Could you discuss the physician feedback you received at ASCO and EHA on the Revuforj data that you guys presented there, especially the post-transplant maintenance data? How soon could those data have positive pull-through to the launch in terms of what you're seeing with the proportion of patients going on maintenance therapy? Thank you.

Joyce, thanks so much for the question. I'll direct the first question to Nick.

Thank you for the question. I think the data was very well received. I think it's a remarkable outcome when you look at the proportion of patients alive at two years, 90%. It's obviously not randomized, but MD Anderson did a very nice job comparing it to the current standard of care, which is considerably less than that. I think they're very encouraged by that. We have significant research efforts ongoing to further elucidate the benefits of giving revumenib in the post-transplant setting. It's really important, I think, to optimize the dose. We're learning a lot about that, and we have a dedicated phase I study ongoing to optimize dose, and we hope to report on that later this year.

We also actually have the first prospectively randomized study called the MenTain Study that was recently announced on ct.gov in collaboration with Dana-Farber and other collaborators, which will hopefully further elucidate the benefits of maintenance

Which will be extremely helpful, and it's an important study to do. Generally, however, we are seeing the uptake of maintenance very much in clinical practice, and that will be evidenced by the real-world series we've already reported upon, where you see high rates of both transplant and then patients going back onto therapy after transplant. We'll be updating on those further in the year with more data, which I think support and perhaps even exceed what we've seen to-date.

Our next question is from Brad Canino with Guggenheim. Brad, you may unmute yourself and ask a question.

Hey, afternoon. Thanks for the questions. Maybe two for me. One, just any quantification you can provide on the duration of treatment increasing, either numerical or some estimation of a relative increase the DoT saw? I'm just trying to judge the magnitude of increase. Two, have you seen any indication of an inflection in the maintenance rate? Sounds like it was 50% is what you're up to for this past 2Q, but any inflection in July after showing the maintenance data at ASCO and EHA as you've been going out and talking with physicians? Thank you.

Brad, thanks so much for the question. First of all, in terms of the duration of treatment, we're very much encouraged by what we're seeing. I think for patients who are actually going on to maintenance, we know that cohort of patients is out beyond nine months. That's an increase from what we've seen previously, and we feel quite encouraged by that. You might have picked up in my remarks, what's also encouraging are patients who don't go to transplant, who remain on therapy for an extended period of time, are well beyond seven months at this point and building. That's new news and very encouraging for what we think will continue to build the recurring revenue within the franchise. Thank you. In terms of inflection in maintenance rates in July, that was a pretty specific question.

I'll just say that we are seeing continued buildup in maintenance. I don't think we've quantified it quite for July, but we feel very encouraged by what we've seen coming out of last quarter and into this quarter. We believe that will continue to build. We've talked about reaching 70%-80% of patients getting on maintenance. We believe that assumption will hold as we build beyond the 50% that we saw this quarter. When exactly that will get to the 70%-80% will take a little bit of time, but we do feel quite encouraged by the momentum we're seeing coming out of last quarter.

The next question is from Faisal Khurshid with Jefferies. Your line is now open.

Hey, guys. How are you doing? This is Faisal from Jefferies. I just wanted to ask, this looks like the first quarter that you actually took a step down in new starts. Can you give any more specificity on the reasons for this? Also, in terms of modeling this going forward, what are the forward trends that we should expect on new starts, each in KMT2A and NPM1? Thank you.

Thanks so much for the question. Maybe I'll turn it over to Steve to make some comments on new starts for this quarter.

Yeah. Thanks for the question. Appreciate you pitching it to me, Michael. Overall, good performance. It's a sixth consecutive quarter of double-digit revenue and demand growth. I think interestingly, we grew 15% on TRX demand, and that was in the face of what you notice is a falling number of patient starts from Q1 into Q2. The reasons for that there are just multiple drivers to the business. A lot of our prepared comments around the KMT2A business, how it's advancing. It's heading exactly in the direction that we predicted it would. Higher transplant rates, higher restart rates relative to the clinical trials, which is really driving the DOT. I'd say for NPM1, it's still early days, but we know that the business is growing. It's growing nicely. Our revenue and our patients on drug from Q1 into Q2 advanced as well.

New patient starts are simply just a part of the growth story, but not the only part. Couple things I hit at in the prepared comments, I'll maybe add a different flavor to it as well. This is normal. You're going to see, at least in the larger targeted AML therapy class, new starts do jump around. There's typical variability. We see it month-to-month. You're going to see it quarter-to-quarter. We've been largely immune to that, right? Rev has existed in this space, and we've either had the same number of new starts or grown them quarter after quarter. This is a fundamental aspect of the market that is typically there. Other things that we've mentioned, physicians have more than one drug class to consider, right? There is a big focus on the NPM1 patient.

Based on that patient's mutational profile, physicians are going to make a choice. I think they're still figuring out where domedans fit. Is it before a FLT3, is it during a FLT3, or perhaps after? That will play its way out, but ultimately, patients will relapse, and Rev will play a role. The last piece are just clinical trials. When you sign up to be in this business, oncology, hematology, you're advancing drugs through the clinic, and you're also commercializing them. We've done that successfully since the launch of Rev, this isn't something that's entirely new. There are new trials that pop up from time to time. As they get established, patient flow will ultimately work its way through, and opportunities for commercial patients are going to stabilize.

Many of these are placebo-controlled trials, we expect to still treat many of these patients, either when they relapse. Not all patients are obviously eligible for clinical trials. We made a conscious decision about a year ago to go to a much broader audience. As we updated in this call, we're approaching 600 accounts that have prescribed. Many of those are medium-sized to smaller accounts, less impacted by clinical trials, and we find meaningful patient build there as well. These factors can cause some lumpiness and fluctuation quarter-to-quarter, but we are able to flex and change our business as needed. We're confident in the forward. I think one of the questions was, what is the trend moving forward? We look at this quarter

As an anomaly, we feel confident about finding patients and getting back to levels that we've seen historically.

Got it. Steve, if you don't mind, just a quick follow-up. Am I to take your comments to mean that new starts on commercial drug in relapsed AML as a whole were down quarter-over-quarter? If so, are you able to quantify that at all?

Probably don't have the full data set to look across all of AML. We're limited in what we can see on our drug. Perhaps that is the case. I would bet that it is. Again, it'll jump around from quarter-to-quarter.

Great. Thank you.

The next question is from Phil Nadeau with TD Cowen. Your line is now open.

Good afternoon. Congrats on the progress and thanks for taking our questions. A few commercial questions from us. First, in NPM1, I apologize if I missed this, did you say what you estimate your share of NPM new patient starts was this quarter and how that compared to last quarter? That's first. Second, I think you said 15% of NPM1 patients have been on a menin. That's after approximately three quarters of your launch. Is that trend that we should continue into the future? In another three quarters, should we expect maybe 30% of NPM1 patients will be on a menin? Or is there any reason to think that would either accelerate or decelerate? Finally, on maintenance, can you tell us where maintenance is being used today in terms of what centers and where's the growth going to come from?

What new centers could come online over the next several quarters to drive increased use of maintenance across the market? Thank you.

Great, Phil. Thanks for the question. First question I took as what's our NPM1 share, and I think as we stated in our prepared remarks, we're about two-thirds of the business right now. If you think about where we were last quarter is roughly about the same, two-thirds or more of the overall business. We expect that to continue to build over time. We do have a dominant position. I would also say in terms of relapse refractory business, assuming our competitor gets close to their numbers, we're probably 85+% of relapse refractory. We are quite dominant in the space and expect to continue to build that.

We did make the comment about 15% of the patients, this is your second question, 15% of the NPM1 patients have seen a menin inhibitor, and that's not only our drug, but our understanding of how our competitor contributes as well. That's 15% total. We do expect that to expand meaningfully and from quarter to quarter, we expect that to grow, accelerate. In a year from now, we'll be at 30%. We would hope that it would be even greater than that, and it's supported by all the data that we're generating. Nick mentioned the presence at our congresses and what we've provided in terms of monotherapy and combinations, and that has shown Revuforj to be a very useful drug in a number of ways.

We expect that we'll be treating NPM1 patients and continuing to penetrate that market very meaningfully, hopefully well beyond 30% in a year. Then maintenance, I'll turn to Nick on this one. Maintenance. Where is it being done? What are those centers? We won't list them all. Academic centers for sure. Nick can make a comment there. Where is the growth going to come from in terms of maintenance? Nick?

I think the growth will come from a number of drivers. Number 1 is patients getting treated earlier on. They're getting treated increasingly in combination. That's just driving response rates higher. We know that if a patient gets to response, they're eligible for transplant. Having had a transplant, the likelihood of them going on to post-transplant maintenance. As we gather more data and present more data there, I think physicians are feeling more confident. They understand how to manage the dose better. These patients after transplant are particularly prone to cytopenias generally, so you really do need to manage the dose, and that's something we'll be presenting updated data on later this year, and I think they're getting confidence to do that. Really just the real-world experience from academic centers. We've seen extremely high rates. They're feeling more confident doing it.

Their intent is to treat out to 1 to 2 years. Most of our clinical trials include therapy out to 2 years. Given the high rates of relapse after transplant without any active therapy, they really want to just do their best for the patients and think that revumenib gives them the best chance of a durable remission. We're seeing uptake increase. From a research perspective, it's our efforts to further confirm that benefit, who's most likely to benefit, and how we can make sure that the drug is well-tolerated. I think we're very encouraged by what we're seeing in the uptake, and we'll be presenting more data on that again later this year with a big focus on this whole post-transplant maintenance area.

That's very helpful. Thank you.

Thanks, Phil.

The next question is from Stephen Willey with Stifel. The line is now open.

Good afternoon. Thanks for taking the questions and congrats on the progress. Just curious how you're thinking about the near-term growth opportunity for this franchise prior to potential label expansion. Was also just wondering if you can confirm whether the definition of event-free survival, which is being used in the phase II frontline trial that reads out later this year, plus Rox, is the same as the phase III frontline trial that's looking at Niktimvo in combination with steroids. Thanks.

Stephen, thanks for the question. Maybe I'll take the first with maybe a comment on the second from Nick. Niktimvo, look, I don't agree with the characterization that it's flat, first of all. Niktimvo is not flat, it's growing, and we see it tracking very nicely to what REZUROCK has done. We expect it to continue to grow meaningfully. What I'm talking about is third and fourth line. We've penetrated well into the fourth line. third line, we have about a third of the third line population at this point, which is very meaningful about a year post-launch. I think we will continue to build that and be meaningful in third and fourth line. You should expect continued growth there. Of course, we have expansion opportunity with the new data coming at the end of the year with combination with ruxolitinib.

We feel like that's a obvious expansion opportunity as we're on our way to the front line. We'll also have steroid combination in early 2028. A lot of data to come. I think physicians are eager to see the combinations and how our drug combines with Jakafi. In the meantime, we'll have considerable growth, we believe, over the near term with third and fourth line. Maybe I'll turn to Nick on definition for event-free survival.

Steve, the event-free survival endpoint is in the, for the frontline steroid combination phase III. That does have an event-free survival primary endpoint. For the phase II three-arm study, we're actually going to look at overall response rate at six months. As you recall, this is steroids versus Rux versus the combination of axatilimab and Rux. We expect the benchmark from that based on a publication in 2022 to be about 40% response rate for steroids alone at six months. We're looking for a meaningful improvement over that. I think for the combination, it could be considerably higher than that. It's not formally a comparative study. It's randomized one-to-one-to-one, about 120 patients, you'll recall.

Yeah, we'll look at overall response rate, but I think also the duration of response will be very important to look at in that study to see whether the combination, and Rux alone can offer a meaningful alternative to steroids. That's what we will report on and looking forward to seeing that readout, because I think it could be very informative and potentially practice and guideline informing readout if either or both of those combinations beat steroids alone.

All right. Thanks for taking the questions.

Thanks, Steven.

The next question is from Etzer Darout with Barclays. Your line is now open.

Great. Thanks for taking the question. A quick one for me. Just wondering, for the proof of principle trial of revumenib in myelofibrosis, what is the primary analysis? What will that entail, and what endpoints will you be evaluating there? Just as a potential, obviously read through to the next gen menin inhibitor. Thank you.

Great, Etzer. Thanks for your question. Maybe I'll turn to Nick on the proof of concept trial.

Yeah, this is a study we're doing with the MPN Research Foundation with John Mascarenhas and collaborators. It's not under our sponsorship. It was recently posted on clinicaltrials.gov. It's quite a full posting if you want to look at some of the details of the study there, but I'll just summarize it for you. Cohort 1, which is primarily the safety assessment, will look at just dose-limiting toxicities. That's going to be relatively few patients treated, around six. In Cohort 2, where we will look for the combination of rev in combination with Jakafi, we'll be looking at standard response criteria. We'll be using the ELN response criteria, looking at anemia, spleen response, and symptom benefit. We'll be looking at SVR less than 35%.

In patients that have a suboptimal response on Jakafi alone and are stable on Jakafi for 12 weeks, we'll add in revumenib, and then we will be looking for using standard ELN response criteria, to generate proof of principle data, which will be incredibly informative to our development program and really catalyze, I think, when we go into phase I in 2027 with our next gen menin inhibitor in myelofibrosis.

Great. Thank you.

Thanks, Etzer.

Our next question is from Yigal Nochomovitz with Citigroup. Your line is now open.

Hi, this is Juan Kim on for Yigal. Thanks for taking our questions. I was wondering with regards to your expanded use of lab data, I believe that you had mentioned, to engage physicians when they have a suitable patient. To what extent can that help smooth new start variability, particularly in NPM1, where the population is larger, but co-mutations can help influence the treatment choice? Also with regards to the MAXPIRe trial, just wondering, patients can be on pirfenidone or no background anti-fibrotic. If the study is positive, how should we think about the extent to which the background therapy could help define axatilimab's role, whether it's add-on or potentially for patients not getting much benefit from current treatments? Thanks.

Great. Thanks for the question. First, with the use of lab data, maybe Steve, you want to make some comments on how we identify patients?

Yeah, sure. We do use lab data, as pointed out in the question. It enables us to find diagnosed patients. We buy lab data. We can identify patients that may be suitable. It's obviously de-identified. We can target accounts that we know patients exist. It's largely worked since launch. I think that really speaks to why we've been so successful, particularly at the CAN 2-2a launch. We've modified our approach over time. We've been able to bring in new lab data sets. We've been able to apply some applied artificial intelligence and machine learning principles. It still holds. I think specifically the question was, can you use that to smooth out new starts? The market's the market. Patients are going to come in at often a random pace. Over a year, you know what it is, but month to month, it's going to be different.

We will find every possible patient that we can, right? We've shown we've been able to do that, and we're going to get better simply over time. That's something we remain committed to.

Maybe Nick, do you want to talk about MAXPIRe and the background therapy impact?

MAXPIRe has three strata. We stratified by nintedanib, pirfenidone, and then no anti-fibrotic, as you would expect, consistent with previous studies that looked at IPF. By far, the majority of the patients were on a background anti-fibrotic. We will obviously look at subtypes in those strata to ensure there isn't an imbalance between the arms. The study is really not powered. Recall it's two to one randomized. It's not powered to detect differences between the different types of anti-fibrotic. That's something we will look at. In terms of phase III planning, we would plan to include axatilimab on a background of standard of care anti-fibrotics, and potentially other standards of care.

One of the attractive things I think about the mechanism of action of axatilimab is that it really does treat what we think is the underpinning pathology by targeting specific, these monocyte-derived macrophages, so inflammatory and fibrotic components of the disease. We really think that that could be a very important differentiator and particularly suitable for combination with current standards of care.

Understood. Thank you.

Thank you.

Our next question is from David Dai with UBS. Your line is now open.

Great. Thanks for taking my questions. Just thinking about the Revuforj, new patient starts, and KMT2A penetration. Last quarter, you mentioned that you were 50% penetrating to the KMT2A market. How did the number look like this quarter? How do you envision the peak penetration will look like? How long do you think that's going to take? That's the same question we'll apply to NPM1. What percentage of NPM1 market have you penetrated so far, and what would the peak penetration look like?

David, thanks for the question. Maybe I'll address KMT2A first. First of all, we own the KMT2A market. This is a part of a business that where we are firmly established as standard of care. NPM1 is building. We firmly acknowledge that we have best in class profile for both broadest set of opportunities there. For KMT2A, we have said that we were about 50% penetrated, and that continues to build. Peak penetration's likely to get to roughly 80% or maybe even more. We've seen examples in the market of companies launching products into targeted areas where they have dominant position, and they get to those levels. KMT2A should reach a very deep penetration over the reasonably near term. NPM1, I would say is a slower build, mainly because of some of the things that Steve said.

It is a bigger patient population for sure. More patients are able to be treated, more patients are being treated. They just happen to be on other therapies. Our job is to introduce Revuforj to those patients. It's monotherapy and perhaps combination as physicians want to use the product, and we'll continue to penetrate there. Again, I think Revuforj can have a very high penetration. We already own, as of today, about two-thirds or more of the business in NPM1, and that should continue to build. Our penetration will get to a high percentage over time. Maybe not quite as high as KMT2A, but we do think that we'll have a dominant position in both with best offering as we've talked about. I think there are different kinetics of build between KMT2 and NPM1 as described, but high penetration is the name of the game here.

That's helpful. Then just a question on inventory stocking. What does the inventory stock look like this quarter? Any dynamics there?

I'll give this one to Keith. Inventory.

Thanks, David. The guidance that we've been pretty consistent with, very consistent with since launch, still holds. Again, not just for Syndax, but really for any rare disease-targeted oncology product. We're about two to three weeks. It's been remarkably consistent since we've launched the product. We'll let you know if there's any changes, but you can assume that we have about two to three weeks of Revuforj in the channel.

Thank you so much.

Thanks, David.

Our next question comes from Salim Syed with Mizuho. Your line is now open.

Great. Congrats on the progress, guys. Thanks for the question. One from us on the $2 billion revenue expected in the U.S. alone. Mike or Keith, could you maybe I think this is the first time we're formally seeing it in a slide written like that, and I noticed also that I think this is the first time that the bar chart for the TAM has been taken out of the deck. Wondering if you guys are starting to think here that the $5 billion TAM is a conservative number, you're sort of reworking your numbers internally, or what your assumptions were exactly going into $2 billion, how much first-line is in there versus second-line, et cetera. Then one clarification. I think you guys mentioned, I think it was Mike, 85% of relapse is what you guys are getting.

I presume that's also inclusive of KMT2A rearrange. What % of MPM1 starts are you guys seeing? I think Kura on their side, they've said 40% is their shares, which would imply you guys are at 60. Is that ballpark-ish correct in line? Thank you.

Salim, lots of parts of your question. Let me see if I can tackle them. First of all, just because I see it on the page here, 85% of what I had mentioned, 85% of the relapse refractory business is KMT2A and NPM1 combined. That's our calculation. If you look at the numbers for the quarter relative to our competitor, we're at least 85% of the business. That's one. When we talk about the estimate of $2 billion plus in revenue peak potential, when we include the frontline, you're right. That is the first time we're talking about this in this way, and I think it's important. Why now? We're more confident than ever, and this is clear to us that this is a very large market opportunity.

When you look at the data that we've published over the last quarter or two, all things point to growing relapse, I would say, overall survival, days of therapy. The response rates are higher than we've seen previously. The data is all very positive and gives us confidence that we will, once we get to the frontline, have a very supportable market position and dominant position in frontline. We will be the first to get there. With the profile that we have today, we feel quite confident that we will have a very meaningful share, a dominant share of frontline. Thinking probably the most important driver here is days of therapy or time on therapy. What we've said today is that we see that elongating.

We're quite encouraged by that, which gives us a lot of confidence because it's a very important indicator, a very important component of the calculation for the market, how long patients stay on therapy. So far, even in relapse refractory patients, we're seeing this exceed our expectations. We are quite confident, again, that we can reach that level of sales. When you talk about the breakdown between frontline and relapse refractory, as we've said in our bar chart, nothing's really changed in terms of our evaluation of the overall total addressable market. $5 billion does assume the $2 billion is part of that. We've always been clear about it, that $5 billion is the overall market when you assume that you get to frontline. $2 billion is really our assessment of the relapse refractory opportunity.

Okay, thanks so much.

You're welcome. Thank you.

The next question is from Andres Maldonado with H.C. Wainwright. Your line is now open.

Hi, guys. Thanks for taking the questions and congrats on the progress. First question is a question on slide five. You talk about 40% of Revuforj uses in combination. Curious if, are physicians mainly adding Revuforj to a failing venetoclax-based regimen, or are they beginning a new combination at relapse? How should we be thinking of those variants and approaches, their potential to produce different treatment durations or transplant rates? This is the first question, then I have a follow-up.

Great. Andres, thank you for the question. Maybe I'll turn it to Nick to talk a little bit about combination regimens.

Yeah, what we're observing in the real world is multiple combinations. I think you're right that ven is a common desire, either ven or ven as a combination. Sometimes patients have been exposed to prior ven. There's some interesting data that revumenib may actually synergize with BCL-2 and may even be an opportunity to re-challenge. That's one of the more common combinations, but we do also see other combinations in use. We have, for example, ongoing studies, which I think is important with FLT3 inhibitors. It's not something we hear that it's a high priority amongst the physician community, but we do want to generate data to confirm both tolerability and efficacy with a FLT3 inhibitor, and then potentially other single agent therapies as well.

Certainly ven is one of the more common ones, and what we have observed in our real-world data today is that it does drive response rates up significantly from what you observe with revumenib alone. If a patient's able to tolerate it and they want to treat, we're obviously not promoting in that indication. It's not within our label to treat in combination. We do see, in some cases, 50%-80% of patients actually treated in combination because we know it drives a 60%-80% response rate, which gives a patient a much better chance for durable response and potentially a transplant.

Andres, you have a follow-up question?

Great. Then a quick one on that.

Yeah, go ahead.

Sure. A quick one on MAXPIRe. I think you guys have highlighted in the past some of the expectations of the scenarios, whether you expect the FVC curves to separate earlier or maybe potentially later. In the scenario that the curves maybe gives a modest 26-week FVC result with a longer later slope or biomarker effect, how should we interpret that influence on the potential market there?

Yeah, nice question, Andres. Maybe I'll turn to Nick.

Yeah, we're pretty confident, I have to say, in that 26-week endpoint. It's been a very good predictor in other studies of IPF. We model that out to 52 weeks because that's the endpoint we would use in a pivotal phase III and is the FDA's preferred endpoint. For a proof of concept study like MAXPIRe, 26 weeks is pretty robust. Some other studies have used 12. We're not anticipating any delayed separation of those curves. We saw in our experience in GVHD, very early onset. Certainly symptoms were responding within a month, and we were observing responses within the first month or two. We are expecting by week 26, the evidence of activity will be very much in evidence. That's what we'll be using. We'll be using the model. We're using a mixed linear regression model out to 52 weeks.

That's what we will be using if it's positive to inform and power the phase III study. Obviously, the phase III study size and dimensions will be influenced by what we observe in the phase II. We're feeling very confident, I have to say, in everything we've observed to date that would suggest that study will read out well. Obviously, it remains a double-blind, placebo, ongoing study. We don't know what the study will show, but given all of the preclinical and clinical data we've generated and the mechanism of action, we're feeling quite positive, and we're looking forward to that study reading out in the fourth quarter. We think it'll be a very robust proof of concept given its design.

Thank you very much.

Thank you.

The next question is from Jason Zemansky with Bank of America. Your line is now open.

Hi, this is Jackie on for Jason. Thanks so much for taking our question. You previously characterized the decline in new Revuforj starts as an anomaly, but can you quantify how starts change sequentially within NPM1M and KMT2A-R? Clarify whether the decline primarily reflected clinical trial enrollment, competition, or underlying patient availability. Also, could you maybe tell us whether July starts have returned to prior quarter levels? Thank you.

Jackie, thanks for your question. First maybe I'll turn it to Steve to talk a little bit about what the decline in new patient starts, what it reflected. That's it.

I think we talked about the potential reasons why it's difficult to piece out each one and determine the contribution factor from each. I'd say it was an overall drop in all new patient starts. I think there was an earlier question on AML in general. We didn't see a differing change in NPM1 relative to KMT2A. We do believe it's a temporary effect, and we're going to return back to where we were previously. I think there was the question also just-

About July. Yeah.

We're not going to comment on July, at least in the quarter forward. We feel good. We feel confident in the business plan and in everything that we have going against the brands as shared on this call.

Yeah. Absolutely. I think we expect to return to growth as Steve mentioned. Look, in terms of the dynamics for NPM1 and some of these other I think there are different things that impact from quarter to quarter, but I think we feel confident we have a plan to make sure that we get back to that positive growth there. Thank you.

Great. Thank you so much.

The final question today is from Mayank Mamtani with B. Riley Securities. Your line is now open.

Yes, good afternoon. Thanks for squeezing me in. Appreciate it. I will keep it very tight. Did you say the NPM1 relapse segment, how is the duration of therapy tracking, sorry if I missed that, relative to what you had in the trials, given the context of earlier line use in co-mutation patients? Was also curious if there's a year-end exit rate you expect to have in terms of how the split between NPM1 and KMT2A patients you expect to have at the end of the year?

Mayank, thanks for the question. I don't think we gave a specific number for NPM1 tracking, I'd just say that duration, most of these patients don't go to transplant. As we commented, that very encouraging that the duration for patients who haven't gone to transplant is averaging well over seven months at this point early on. A lot of those patients, of course, are NPM1 or I'd say the disproportionate amount of NPM1 patients don't go to transplant. I think that's a potential indicator of how things are going. We're quite encouraged by the overall, and without breaking it out, that's perhaps an indicator. In terms of the number of NPM1 patients at the end of the year, I don't think we've said or guided to that. All I'd say is that we do have a dominant position in the market.

We expect that to build over time. We'll continue to use all of our resources to identify patients and build that business, we feel encouraged by what we see both as monotherapy and in combination. That's been what the themes have been at our medical congresses. We're quite in a good position to continue to build and feel good about the forward.

Understood. Maybe just lastly, we are coming off a busy conference season, obviously you have your biggest conference at the end of the year. If you could just quickly comment on what to expect there, including from the three frontline setting trials we have ongoing, and if there's any enrollment update we can expect to have on EVOLVE-2 or REVEAL-ND would be good to know. Thanks for taking my question.

Yeah, thanks. Thanks for the follow-up. End of the year, Nick, do you want-

Yeah, very data rich end of year. Looking forward to the second half of the year. We hope to carry the momentum. I outlined briefly what we saw at ASCO and EHA. The teams have been working very hard. We're going to see multiple data sets. I would expect to see updates to all of our frontline studies. There's several, two studies in combination with BEN and HMA combinations, and then obviously our combination study with intensive chemotherapy as well. That'll be very informative to our ongoing pivotal phase IIIs, which remains a focus for us. Expect to also see, as we've talked a lot about, further data on the maintenance after transplant, informing practice there, combination, and of course, real-world evidence. That will be very important.

I think it'll be also important that I know there'll be a lot of interest in looking at how some of the time to event things are coming from our frontline studies, like event-free survival and particularly overall survival. I think we should have sufficient maturity to update on those as well. As I say, very data rich period coming up in the second half of the year we're looking forward to.

Thanks so much. No problem.

Thank you.

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

Thank you all. We really appreciate everyone tuning in today to discuss our recent progress and the exciting milestones that we have ahead. We look forward to seeing many of you at the upcoming investor conferences in the third quarter. Have a great evening, everyone.

Good day, everyone, welcome to the Syndax first quarter 2026 earnings conference call. Today's call is being recorded. If you would like to ask a question following the company's prepared remarks, please press star five during the call. At this time, I would like to turn the call over to Sharon Klahre, Head of Investor Relations at Syndax Pharmaceuticals.

Great. Thank you, operator. Welcome, thank you all for joining us today for a view of Syndax's first quarter 2026 financial and operating results. I'm Sharon Klahre, with me this afternoon 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; and Keith Goldan, Chief Financial 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 two. 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 of 1995.

Actual results may differ materially from those indicated by these 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, April 30th, 2026, only. A replay of this call will be available on the company's website, www.syndax.com, following its completion. With that, I'm pleased to turn the call over to Michael Metzger, Chief Executive Officer of Syndax.

Thank you, Sharon. Good afternoon, everyone, and thank you for joining us. Starting with slide three. In the first quarter, we continued to make strong progress advancing our commercial objectives and pipeline programs designed to unlock the full potential of our first two medicines. On the commercial front, we delivered over $100 million in combined sales of Revuforj and Niktimvo, underscoring robust demand for both medicines and advancing the company towards profitability. Revuforj net revenue totaled $49 million in the first quarter, highlighting our leadership position in menin inhibition and strong adoption across both indications. Revuforj net revenue grew by double digits quarter-over-quarter, primarily driven by new NPM1 patients.

Adoption in NPM1 has been strong and growing steadily since Revuforj was added to the NCCN guidelines for relapsed/refractory NPM1 mutated AML in September of 2025, followed by the FDA approval of our expanded label in October of 2025. We are the leaders in the relapsed/refractory space in both NPM1 and KMT2A, and well-positioned to drive further growth and unmatched efficacy data and expanding prescriber base, excellent payer coverage, and multiple evolving treatment dynamics that should ultimately extend the average duration of therapy, including a growing number of KMT2A patients proceeding to transplant after receiving Revuforj. Notably, recent analysis indicates that Revuforj is enabling nearly half of KMT2A patients to receive a potentially curative stem cell transplant, a significant increase from our prior estimates of 33%.

This growing transplant rate gives patients the best chance for durable remissions, ultimately driving longer treatment durations as an increasing number of patients return to therapy post-transplant. This step change, compared to the 25% transplant rate we observed in our clinical trial, aligns with what we have expected as Revuforj is used earlier in the treatment paradigm and often in combination with other therapies in the real world. The pool of patients on Revuforj post-transplant is expanding each quarter, although that growth continues to be obscured by the large number going to transplant. While the robust transplant rate seen with Revuforj has a short-term impact on the business, it is first and foremost a very positive outcome for patients and will translate into durable and sustainable growth as an increasing number of patients return to therapy.

This is an important and unique growth driver of our KMT2A business. Turning to Niktimvo and chronic graft-versus-host disease, or GVHD. Niktimvo delivered $55 million in net revenue in the first quarter. This result reflects consistent new patient starts, partially offset by natural attrition among the large cohort of predominantly later-line patients who started Niktimvo during the first quarter of launch last year. Notably, the same dynamic was also seen during the same period of the launch of Rezurock, as well as other rare disease therapies. Looking ahead, we expect strong growth with multiple drivers supporting the business, including a broad prescriber base and increasing uptake in the third line, which should extend the average duration of therapy. Turning to our pipeline. We've made excellent progress advancing the programs that will fuel the next phase of growth for the company, including our pivotal frontline trials of revumenib.

We are well-positioned to be the first to get a menin inhibitor approved in frontline AML with strong global site activation and patient enrollment underway in our pivotal trials. We will continue to expand the body of evidence supporting our medicines with multiple important data readouts anticipated in the second quarter and throughout the year. Similar to past years, we will have a major presence at ASCO, EHA, and ASH with new data from multiple studies of Revuforj across the acute leukemia treatment continuum, including new maintenance data, which will be highlighted in an oral session at ASCO. These upcoming datasets will continue to advance our leadership in menin inhibition and highlight Revuforj's best-in-class profile across multiple acute leukemia subtypes and settings.

We are also nearing two potentially transformative Niktimvo readouts in the fourth quarter. Including phase II data in idiopathic pulmonary fibrosis or IPF and in newly diagnosed chronic GVHD patients treated with Niktimvo plus Jakafi. Data from these trials could further unlock Niktimvo's multi-billion dollar potential in chronic GVHD, IPF, and beyond. From a position of strength, we are delivering long-term and sustainable growth and advancing innovative treatments that can significantly improve the lives of patients. We have a world-class R&D and commercial organization with a proven track record of delivering breakthrough medicines to patients. We have two products poised for future label expansion, important upcoming data readouts, and the expertise necessary to continue delivering innovative medicines. We are well-funded to invest in the successful commercialization and further development of Revuforj and Niktimvo, and on our way to reaching profitability with growing revenue and a stable expense outlook.

I'll now turn the call over to Steve to discuss our commercial results in more detail. Steve?

Thank you, Michael. Starting with Revuforj on slide four. Revuforj is now a little over a year into launch, and by any measure, it has been an exceptional commercial success, with net revenue now annualizing at nearly $200 million. We continue to track well above launch benchmarks set by other AML therapies and expect to continue to outpace other drugs and redefine success in this space. Two factors underpin our conviction, our ability to target a substantially larger population than other mutation-directed AML therapies and multiple treatment patterns that are expected to extend the average treatment. In the first quarter, we delivered approximately $49 million in Revuforj net revenue and double-digit quarter-over-quarter growth across all key metrics, including net revenue, total prescriptions, and new patient starts. Two drivers of our business, new patient starts and patients returning to therapy post-transplant, and both are building nicely.

We added about 330 new patients in the first quarter, up approximately 10% compared to last quarter, with the growth driven by new NPM1 patients. New patient starts have increased even more significantly compared to the 200-250 patients we were adding per quarter prior to the expansion of the Revuforj label to include patients one year and older with relapsed/refractory NPM1-mutated AML. We've continued to gain momentum and reach new highs, demonstrating the durability of Revuforj's strong market position, even with the launch of a second menin inhibitor approved for adult NPM1 patients. Early indicators suggest at least 40% of new starts in the first quarter were NPM1 patients, up significantly from 10% of new patients prior to the NCCN guideline update in September 2025.

Between the NPM1 patients who started in the first quarter and those continuing on therapy from prior months, we estimate NPM1 accounted for at least 30% of the $49 million in net revenue. We're positioned to be the market leader in NPM1 with the strongest clinical profile. In recent market research, HCPs who treat our target population ranked efficacy as the most important factor in their treatment decision, followed by safety and tolerability, availability of long-term efficacy data, personal experience with the drug, and inclusion in clinical guidelines. Notably, more HCPs strongly favored Revuforj over the other approved menin inhibitor on the top five most important factors, as well as nearly all other factors, including ease of access, drug-to-drug interactions, peer or KOL endorsement, ease of administration, flexible dosing, to name a few. Turning to slide five.

We have a strong foundation to support the continued expansion of our KMT2A and NPM1 business, including a world-class team with excellent customer relationships and favorable listings in the NCCN guidelines, the most influential guidelines for clinicians as well as payers. Our already robust prescriber base has continued to expand following our approval in NPM1, including our activation of Tier 1 and Tier 2 accounts, the highest volume centers in the U.S. who treat 2/3 of our target population. Over 85% of these accounts have now ordered, up from 70% prior to the label expansion. The total number of accounts that have ordered has also increased quarter-over-quarter and is now well over 500 accounts, reflecting growing adoption in centers of all sizes, including community practices.

Our growing prescriber base reflects physicians' enthusiasm to use Revuforj for their NPM1 patients and positions us to drive further penetration in both indications. We also have nearly perfect payer coverage, and physicians can access the menin inhibitor they prefer without any meaningful barriers. Revuforj's formulary coverage stands at 97% of all covered lives, including all commercially covered lives, Medicare, and Medicaid, a coverage position that leads the class. We have engaged and educated payers extensively, and they recognize the value of Revuforj and its status as the only menin inhibitor approved for multiple acute leukemia subtypes. While the class leader in NPM1 will be determined by HCP preference, not price or recommendations from payers, I will note that payers understand the two menin inhibitors are priced at parity, with Revuforj actually costing less for the large percentage of patients on CYP3A4 inhibitors.

This was made clear in a recently updated IPD Analytics monograph, which no longer suggests use of one menin inhibitor over another based on price alone, clearing up a point that had led to temporary confusion among a small handful of plans representing less than 1% of all covered lives. Turning to slide six and the multiple factors that will drive further Revuforj growth. The first is our continued expansion into relapsed/refractory NPM1-mutated AML. Of the 4,500 patient annual incident population, we estimate that less than 10% of patients have received a menin inhibitor, highlighting the substantial opportunity for further growth. Compared to KMT2A, where we saw a steep uptake curve due to the lack of other approved therapies, we expect our NPM1 business will build over time because there are other options that physicians may consider for this population, depending on their co-mutations, for example.

With 2x to 2.5x as many NPM1 patients as KMT2A patients, we expect NPM1 will become a major component of our business over time. With strong physician support and unmatched efficacy data in a disease where efficacy is the most important attribute to physicians, we expect to have dominant market share in NPM1 and KMT2A. The second factor is the growing number of KMT2A patients who are proceeding to transplant after receiving Revuforj and then returning to therapy post-transplant. As Michael noted, the transplant rate has been building over time, with recent analysis indicating that nearly half of KMT2A patients are proceeding to transplant after receiving Revuforj. Around 45% have restarted after pausing treatment for one to two quarters, a percentage we expect will grow over time.

While the pool of patients in Revuforj post-transplant is expanding, that growth continues to be obscured by the large number going to transplant each quarter. Importantly, we expect to observe a meaningful step up in post-transplant use after leading institutions report on their experience using revumenib as maintenance in the second quarter. Long-term, we expect up to 70%-80% of transplanted patients will ultimately be put back on therapy for one to two years based on our clinical trial experience and feedback directly from physicians. The third factor is use of Revuforj in early lines of treatment and in combination with other therapies. Claims data show about 70% of use in the second and third line, even as we've added more NPM1 patients to our mix.

This is encouraging because when patients are treated earlier, you expect to see higher response rates, longer durations of response, and more patients proceeding to transplant, as we now see playing out. Claims data also show about 40% combination use. While Revuforj is approved and promoted as a monotherapy, the significant combination use highlights physicians' comfort with the Revuforj profile, and that could extend treatment durations. All these evolving treatment patterns will drive an increase in the average treatment duration, especially the growing number of KMT2A patients proceeding to transplant and then returning to therapy. This group of patients is already approaching nine months of therapy on average, with this duration expected to meaningfully increase over time.

We are confident in our ability to continue building a sustainable business with our first two indications for Revuforj, which together represent a $2 billion-plus market opportunity, as shown in slide seven. Turning to Niktimvo on slide eight. Building on an excellent launch year, Niktimvo delivered $55 million in net revenue in the first quarter of 2026 and is now annualizing at over $200 million. In the first quarter, about 5,000 infusions were administered and approximately 300 new patients started, even with severe weather impacting patient access to infusion drugs in the first quarter. Performance this quarter reflects strong and consistent new patient starts and solid persistency, partially offset by natural attrition among the large cohort of predominantly later line patients who started Niktimvo during the first quarter of launch last year.

This dynamic was also seen at a similar point in the Rezurock launch, a drug which reached $500 million in annual U.S. net sales within the first four years of launch in the same indication. All indicators of demand suggest we will resume growth next quarter as we continue to steadily add new, less advanced patients. Turning to slide nine. The fundamentals of our Niktimvo business remain strong with multiple drivers for continued growth. The first is continued adoption in the fourth line and growing usage in the third line. Within one year of launch, Niktimvo has captured 32% the third line plus market with the majority of use in the fourth line and increasing uptake in the third line as providers gain experience. As the patient mix shifts more towards third-line patients with less advanced disease, we expect this will extend the average treatment duration.

Second, this is a chronic disease with the potential for patients to stay on therapy for long periods. Of the patients who started Niktimvo at launch in the first quarter of last year, 60%-70% remained on therapy in the first quarter of this year, highlighting the potential for extended durations of therapy, especially as our patient mix shifts more towards third-line patients. Our clinical trial experience shows the duration of therapy can be measured in years for a meaningful proportion of patients. Third, Niktimvo has a broad and productive prescriber base and strong commercial synergies for both Syndax and Incyte. Virtually every bone marrow transplant center in the U.S. has prescribed Niktimvo, and all have become repeat customers. Physician feedback is very positive.

They continue to report strong activity in multiple organs, with particularly notable results observed in patients with lung and skin fibrosis, some of the most challenging to treat manifestations of the disease. All these drivers put us in a strong position to expand our impact third line plus chronic GVHD, the $2 billion U.S. market opportunity, as we can see on slide 10. In summary, our quarterly results underscore the strong demand for Revuforj and Niktimvo and the substantial commercial opportunities we have with both products. We have the right medicines, the right team, and the right strategies to continue delivering for patients and driving strong and sustainable growth. With that, I'll hand the call to Nick to discuss our development programs.

Thank you, Steve. Starting with revumenib on slide 11, we've made significant progress advancing our pivotal frontline trials and our integrated evidence generation plan designed to establish revumenib as the menin inhibitor of choice across the acute leukemia treatment continuum. I'd like to highlight a few key points. First, we're focused on advancing enrollment in our pivotal frontline trials, which have the potential to support additional FDA approvals and the registration of revumenib outside the U.S. We are well-positioned to be the first to deliver pivotal frontline data for a menin inhibitor. Global site activations and patient enrollment are well underway in these trials, which were informed by a robust body of clinical evidence, allowing optimization of endpoint assumptions and other aspects of the study design.

We've also been able to leverage the strong relationships we've built with leading investigators and patient advocacy groups around the world through our work pioneering this new class of therapy. To support these collaborations and rapid enrollment, we have a team of highly qualified and well-connected MSLs located in key geographies, including Asia, fully focused on revumenib. As a reminder, EVOLVE-2 is the phase III trial of revumenib plus venetoclax and azacitidine in newly diagnosed unfit NPM1 or KMT2A AML patients. This trial, conducted in partnership with the highly regarded HOVON Network, was the first pivotal frontline trial of a menin inhibitor to begin enrolling patients. EVOLVE-2 has dual primary endpoints of complete remission and overall survival, both in the NPM1 population to support the potential for accelerated and full approval respectively.

REVEAL is the phase III trial of revumenib plus intensive chemotherapy in newly diagnosed fit NPM1 patients, including adults and children 12 years of age and older. REVEAL also has dual primary endpoints of MRD negative CR and event-free survival to support the potential for accelerated and full approval respectively. In the fit KMT2A population, we are pursuing a novel and differentiated approach. In addition to generating further data in combination with intensive chemotherapy in partnership with the National Cancer Institute, we are progressing the RAVEN trial with leading clinical researchers. RAVEN is a phase II trial of revumenib plus Ven/Aza in newly diagnosed KMT2A patients who would be considered fit for intensive chemotherapy.

The design of RAVEN is supported by the strong activity observed with revumenib plus ven-HMA in KMT2A patients and growing physician interest in moving from 7+3 intensive chemotherapy backbones to more tolerable ven-HMA backbones. The second point I would like to highlight is that we will continue to advance our scientific leadership in menin inhibition with a major presence at key medical meetings, including at least 15 revumenib abstracts accepted for presentation at ASCO or EHA. The volume and breadth of the data we will present underscore physicians' interest and commitment to revumenib with its differentiated profile and strong activity across multiple genetic subtypes. I'd like to highlight now some of the key datasets we expect to report in the second quarter of 2026. First, we expect new real-world evidence with revumenib.

This dataset will build on the first real-world evidence that another group, Moffitt Cancer Center, presented for revumenib and the therapeutic class at ASH last year. Moffitt reported a 77% overall response rate and 75% MRD negativity rate among relapsed/refractory NPM1, KMT2A, and NUP98 acute leukemia patients who primarily received revumenib as part of combination therapy. In addition to showing compelling clinical activity, revumenib was well-tolerated as a monotherapy and in combination with standard-of-care therapies. We also expect new post-transplant maintenance data, including a dataset accepted for oral presentation at ASCO. These will be important datasets given physicians' growing interest in using revumenib post-transplant. The upcoming data will build on retrospective data presented by MD Anderson at ASH last year from 10 pediatric KMT2A or NUP98-rearranged patients. They reported that revumenib was well-tolerated in the post-transplant setting with encouraging early efficacy.

Notably, all patients were alive and 90% were relapse-free at a median follow-up of 19 months. Additionally, we expect updated data from our phase I trial of revumenib plus intensive chemotherapy in newly diagnosed NPM1, KMT2A, or NUP98 AML. You will first see an abstract with a data cutoff that is similar to the preliminary seven or eight data we presented at ASH last year, showing high rates of response and MRD negativity along with a favorable safety profile. Data with longer follow will be presented at the meeting. We also anticipate updated data from the relapsed/refractory cohort in the SAVE trial of revumenib plus venetoclax and decitabine as well as azacitidine in NPM1, KMT2A, or NUP98 acute leukemias.

This update will build on the compelling data previously reported from SAVE, which shows overall response and MRD negativity rates above 80% in heavily pretreated patients. Finally, we expect additional relapsed/refractory NUP98 rearranged data from our AUGMENT-101 trial and expanded access program. NUP98 rearrangements, which are found in up to 5% of AML cases, are associated with a poor prognosis and high unmet need with no approved targeted therapies. Last year at EHA, we presented the first and only data to our knowledge showing compelling activity with a menin inhibitor in NUP98 patients.

This data was met with strong enthusiasm by clinicians. We are already seeing academic centers treating relapsed/refractory NUP98 patients with revumenib, underscoring the benefit of having one menin inhibitor with activity across multiple subtypes. NUP98 is one of several subtypes associated with acute leukemias associated with upregulation of HOX genes that may be sensitive to revumenib. Altogether, more than 50% of AML patients may have a genetic alteration which is susceptible to revumenib. The body of evidence supporting revumenib will continue to grow throughout 2026, with additional data expected in the second half of the year, including an update from the BEAT AML trial of revumenib plus Ven/Aza in newly diagnosed NPM1 or KMT2A AML. We also expect an update from a phase I trial of revumenib plus gilteritinib in relapsed/refractory patients with a FLT3 mutation and an alteration associated with HOX overexpression.

Early data presented at ASH last year showed the combination appeared tolerable with early signs of efficacy, supporting continued evaluation. For the subset of relapsed/refractory NPM1 patients with FLT3 co-mutations, a menin plus FLT3 inhibitor combo is one of several potential options that physicians may consider depending on patient-specific factors. I'd now like to turn to axatilimab development on slide 12. This will be an important year with two upcoming data readouts. We are now expecting top-line data from the phase II trial of axatilimab plus ruxolitinib in the fourth quarter, a timeline which pulled in due to faster than anticipated accrual. Data from this trial could inform the potential for axatilimab in earlier lines of chronic GVHD and potentially pave the way to a steroid-sparing approach. We also expect top-line data from our phase II MAXPIRe IPF trial in the fourth quarter.

Earlier this year, we completed enrollment and actually exceeded our original enrollment target of 135 patients due to the number of patients put into screening as we neared the end of enrollment. This speaks to investigator enthusiasm for axatilimab and the desire for new treatment options in IPF. Given a high interest in this program, I will highlight the evidence support in the scientific rational for CSF1R inhibitions and outline the trial design. Moving to slide 13. Significant evidence points to CSF1-dependent monocyte-derived alveolar macrophages as a promising new target in IPF. These cells are believed to play a key role in driving lung fibrosis. Multiple studies implicate the CSF1R pathway in IPF and provide strong mechanistic rationale for our program.

For instance, high CSF1R levels are observed in IPF patients versus healthy controls, and higher levels of CSF1R and monocytes predict shorter survival among IPF patients. Turning to slide 14. Axatilimab is an IgG4 monoclonal antibody which is optimized for diseases with an inflammatory and fibrotic component. It binds to the CSF1 receptor on the surface of monocytes and macrophages, preventing their activation by CSF1 and IL-34. This reduces the levels of circulating pro-fibrotic and pro-inflammatory monocytes and monocyte-derived macrophages and inhibits the activity of these pathogenic macrophages in tissues. Based on these observations, axatilimab has the potential to make a more pronounced impact on the fibrotic process by targeting pathways that sit upstream of the pathways engaged by currently available IPF therapies. The IPF program is supported by the remarkable activity observed with axatilimab in chronic graft-versus-host disease.

Responses were observed across all organ studies, including organs with fibrotic manifestations of the disease, such as the lung and skin. Among patients with lung involvement who received axatilimab at the dose we are studying in MAXPIRe, nearly 50% achieved a lung response and over 90% reported improvements in shortness of breath at rest. These data, together with the strong mechanistic rationale, are driving strong physician support for axatilimab's potential in IPF. Slide 15 shows the design of MAXPIRe, a phase II randomized, double-blind, placebo-controlled trial of axatilimab in approximately 135 IPF patients. This is a well-designed trial that has the potential to provide robust proof-of-concept data for axatilimab in IPF to inform a registrational program. The inclusion criteria is similar to other recent IPF trials.

Background anti-fibrotic therapy is allowed but not required, and the primary endpoint is the annualized rate of decline in forced vital capacity, or FVC, measured at 26 weeks. In summary, we have a data-rich period ahead and are laser-focused on executing our pivotal programs. We are just starting to demonstrate our ability to translate promising science into novel medicines for patients with hard-to-treat diseases. With that, I will hand the call to Keith to discuss our financials.

Thank you, Nick. Earlier this afternoon, we reported detailed first quarter 2026 financial results on our Form 10-Q, and I'll now highlight a few key points on slide 16. Total revenue for the first quarter was $64.9 million, up 224% over the same period last year. Demand for Revuforj remains strong with $48.9 million in net revenue, up 144% from the same period last year. We achieved these results even with typical first quarter dynamics, including severe winter storms, which had a transient impact on both products in February, and a growing number of KMT2A patients temporarily pausing Revuforj to proceed to transplant. Inventory levels remain within the two to three-week range we have previously guided.

We expect continued growth over the coming quarters as adoption in NPM1 increases and the average duration of therapy extends as Revuforj is used earlier in the treatment journey and the number of patients on therapy post-transplant continues to build. Now I want to turn to Niktimvo, which continues to be an important cash flow contributor to Syndax with our 50% share of the net commercial profit totaling $15.9 million in collaboration revenue in the first quarter. The collaboration revenue that we reported was 29% of the Niktimvo product revenue reported by Incyte within the range of 25%-30% that we've been guiding to. Our collaboration revenue for the first quarter is a significant step-up from the $0.2 million in collaboration loss we reported from the first two months of sales of Niktimvo in the first quarter of 2025.

We continue to expect our Niktimvo margin contribution, defined as a collaboration revenue recorded by the company as a percentage of Niktimvo net sales, to be in the 25%-30% range in the near term and increase longer term as sales grow while much of the expense base stays largely fixed. We anticipate continued growth throughout the year as Niktimvo is increasingly used in the third line and duration of therapy extends. With regard to expenses, guidance remains at total R&D plus SG&A expenses in 2026 of approximately $400 million, excluding the impact of $50 million in estimated non-cash stock compensation expense. We are well-funded to continue investing in our commercial and development priorities with $352.1 million in cash equivalents, and marketable securities as of March 31st, 2026.

With this robust balance sheet, growing revenue from two medicines, and stable expenses, we are on our way to reaching profitability. With that, I will hand the call to Michael for closing remarks.

Thank you, Keith. Looking ahead, we are focused on driving revenue growth and delivering on the milestones shown on slide 17, which will fuel further innovation and support value creation. We are in a strong position with multiple near and long-term growth drivers supporting both of our medicines. Revuforj is poised for further growth as we expand into NPM1 and continue to penetrate the KMT2A market, a segment where Revuforj is the only approved therapy. A growing number of KMT2A patients are proceeding to transplant, outpacing our near-term expectations and returning to therapy post-transplant, which will extend the average treatment duration and drive durable and sustainable growth.

With the broadest label and best efficacy profile of the menin class, we have the unique opportunity to cement Revuforj as the menin inhibitor of choice in relapse refractory disease and ultimately be the first to frontline, unlocking a $5 billion-plus market opportunity. Niktimvo continues to be a meaningful contributor to our bottom line. We have ample opportunity for further growth in our first indication as our patient mix evolves and important rate readouts later this year in frontline chronic GVHD and IPF that could open transformational multibillion-dollar markets in the near term. I will close by thanking everyone who has made it possible for us to deliver breakthroughs for patients, especially the individuals and clinicians who have chosen to participate in our clinical trials, as well as our dedicated Syndax team and the long-term investors.

With that, I would like to open the call for questions. Operator?

At this time, I would like to remind everyone that in order to ask a question, press star and then the five on your telephone keypad. If you would like to withdraw your question, press star and the five once again. We'll pause just a moment to compile the Q&A roster. Okay, the first question is from Anupam Rama from JPMorgan. The line is open.

Hey, guys. Thanks so much for taking the question. Just a quick one on Revuforj. I know that later this quarter you're gonna have data from that multi-center real-world study that you highlighted in your opening comments. What should we be looking for in that presentation beyond what we learned in that Moffitt study that you highlighted? Looking for data points that might help us understand how physicians are using the product and how we should think about extrapolating to the revenue trajectory of the product moving forward. Will this real-world study include both the KMT2A and NPM1 experience?

Anupam, thanks for the question. Yeah, very important new data coming, so we're excited about that, and I'll pass it to Nick to give you a little bit more detail.

Yeah. Thank you. I'm not gonna talk about the data specifically, but I'll give you a flavor of the things to look for as you would anticipate in these types of dataset. I mean, one is we're expecting, you know, broad coverage across the genetic subtypes, including NUP98. As previously seen, what we're seeing in the real world is extensive use in combinations of therapy. That's clearly not part of our current indication, but given the data we've generated, we know that physicians like to use it in combination in early lines of therapy. I think the other things I would say to look out for is, you know, the number of patients that are progressing to transplant because of those catalysts and factors for that, the number of patients that are able to get to transplant.

I think that'll be an important data point to look for. Of course, the ability to get patients onto maintenance. Now, that's not, again, something we're promoting on, but it's an important part of patient care. Physicians are wanting to do it. We wanna provide data, we are observing it in the real world. Those are the things I'd be looking out for as we generate more real-world evidence.

Thanks so much for taking our question.

Thanks, Anupam.

The next question is from Corinne Jenkins from Goldman Sachs. The line is open.

Sorry, I was muted. Good afternoon, guys. Maybe quickly from us, is there a way that you can kind of help us quantify the headwinds to revenue or patients you're facing in a given quarter if 50% of the KMT2A population is going to transplant, recognizing that that normalizes once you reach a steady state of KMT2A penetration? Maybe on that note as well, I think you commented at the end of four Q, you were at about 50% penetration in the KMT2A market. Is there an update on that front as well? Thanks.

Thanks, Corinne, for the question. I'm gonna hand it over to Steve. Maybe I'll handle the second part of the question first, which is sort of the state of the KMT2A market, which is certainly growing. We had gotten to, roughly, you know, approaching 50% penetrated on an incidence population of about 2,000 patients, last year, and we continue to see that progressing. Certainly the impact on maintenance is a big growth driver for that business. We do think we'll get, you know, nicely north of 50% this year, but I do believe that the maintenance piece is such a you know, considerable part of what grows in the future as more patients are now going to transplant.

We have about 50% of patients going to transplant and right around that number coming back. I think that's likely to grow considerably in the coming year or so, and that's driven by what we hear from physicians and all the data that will be coming out. Nick mentioned some should help that with that growth rate. That's, that's KMT2A, very healthy piece of our business. Maybe I'll turn it over to Steve for the other.

Yeah. I think it was the same question around KMT2A. I think the first part of the question was just headwinds, and maybe that's around bringing patients back in that post-transplant setting. I think what we've looked, and some of the numbers we've updated this quarter are using claims data, and we look back and we realize there's a greater percentage going to transplant and this increasing number coming back post-transplant. The way to think about it is average is probably three to four months to get patients back, but the longer run market, we realize there's patients that may be six months or more off treatment from that transplant to when they get back on. The timing's gonna be important. We've seen that grow as an important piece of our business.

It's a little slower to build than it would be just bringing new patients in, which is, you know, driving the business right now on the NPM1 side. You know, in terms of that, because it all lands on duration of treatment, which is something we've seen extend, and I think we had this in our notes. The patients that do come back, particularly those that start in the first seven months of launch, they're already at nine months and longer in terms of duration of treatment. That, that will extend, and there'll be more folks in that group over time, which is why we expect the duration of treatment to grow considerably over 2026.

Thanks.

Thanks, Corinne.

The next question is from Phil Nadeau from TD Cowen. Phil, your line is open.

Good afternoon. Thanks for taking our questions. Two from us, one commercial and one on the pipeline. In terms of the NPM1 penetration, what do you think is gating for increasing that penetration? Is this simply time on market, or are there datasets that could be important, such as the FLT3 combo data that's expected later this year? That's first. Second, on the pipeline, the data you present from lung involvement in cGVHD is very impressive. What is the time course of the responses in GVHD? Is 26-week duration likely to be sufficient in IPF? Thanks.

Great. Thanks, Phil, for the question. The first question related to sort of what's gating relative to the growth of NPM1, maybe I'll turn it over to Steve to make a comment there and maybe to Nick for the pipeline question.

Yeah. NPM1, it's, you know, clearly we're at the front end of that population, Phil, there's a lot more patients to grab. I think when you think about our own business, there's been step-ups in the business, it really began for us in that Q3 timeframe, right? At the end of September, we got NCCN guidelines then obviously the full indication for the fourth quarter. We've seen big step-ups in our own business, roughly in the 250 range in Q3 of new patients. A big step up in Q4, another step up in Q1. Our average is, you know, we're looking at 330 new patients in the quarter. That'll build. It's a different market. You know, KMT2A was obviously our launch. Lots of patients came in. Rev is already the standard of care.

Patients had no other options. We know for NPM1 they do. There's FLT3 mutation. It's a little bit more complex. The patient tends to be older. We're not gonna see as much likely transplant and then restarts in that population. We're gonna give it time, and the time to peak will just be a little bit longer in the NPM1 population relative to KMT2A.

I'll just add that the profile that we're showing for NPM1 is best in class. I think we feel very confident. Physicians tell us that time and again that we have, you know, as Steve said in his remarks, broad, you know, broad advantages across the profile. We feel very confident that we'll continue to build that market, build that business and have dominant share over time. Now the next question was related to IPF, and I'll turn it over to Nick to answer that.

Thank you, Phil. Happy to take that question. First thing I'd say, this is a very robust and statistically rigorous proof of concept study in IPF, and we're very confident in the 26-week endpoint. There have actually been some other previous POCs that have even looked at 12 weeks. We think 26 weeks is a very good compromise in terms of it being too short, but enough time to be able to detect a difference. We actually annualize that. We'll report annualized rates of FVC decline using, you know, standard FDA-approved models for modeling that out to 52 weeks. This will be a very rigorous proof of concept to inform a phase III program.

If you look at previous phase III studies, if you actually look at the graphs for separation, you see very clear separation by week 26. We're confident that 26 weeks will be sufficient to detect a difference and inform a phase III.

Perfect. Thank you.

Thanks, Phil.

The next question is for Brad Canino from Guggenheim. Brad, your line is open.

Just following on the previous question of the transplant maintenance dynamic continuing to be more of a temporary headwind for now. My question is really simply when will this start to flip to a tailwind? It sounds like there's two levers now, and you're pointing to flat quarterly patient starts, so you're refilling the population. How much higher than a 50% transplant rate can even be possible in this population? Are you topped out there, and now you can grow that maintenance restart? Are you confident in that six-ish month now wait to the restart for maintenance, or could that even extend too to be, you know, patients starting seven, eight, nine months later and push out the time to that start of the tailwind as well?

Separately, it looks like you lost about 14 points of year-over-year growth due to gross to net. Is there a chance that reverses back later this year? Can it get worse, or should we think about that being a neutral effect for the rest of the year? Thank you.

Great, Brad. Thanks for the questions. I think you're right about the maintenance dynamic is, as today is a bit of a headwind. I mean, obviously a great opportunity for patients, many more going to transplant. It's far exceeded our expectations. You've probably heard us talk about the fact that maintenance could or rather transplant could get as high as 50%, that's essentially where we're at. I think that happened a little bit faster than we expected, but that's probably where it'll be. I don't expect it to be much higher than 50%, I do think that has sort of topped out. We will ultimately see, but that's point number one.

Point number two is the effect on restarting and how long we're gonna need to wait until that really becomes a big factor. We have talked to many physicians. Obviously, the work that we've done seems to indicate that we'll get to 70%-80% of patients restarting maintenance, and we've seen that accelerate as every quarter has gone by. I think that's an important milestone or important bogey for us. Whether that takes another quarter, two quarters, a year, we'll see. We ultimately think that's where we'll be at steady state, so that'll be an important driver of growth as we go forward here, and that's the dynamic we see playing out.

We do see patients, as Steve mentioned, that over a six-month period of time, as you wait to come back at engraftment for maintenance, it's a little bit longer than we expected. We are picking up patients now in the claims that, it's not three, four months of engraftment and restart. It's more like five, six months, maybe even longer. That, again, that is a, you know, a piece of this. I don't think that's the vast majority of patients. I think that's some of the patients. Again, we do expect in future quarters to start to see some of the compounding effect as we sort of peaked at our 50% transplant rate, and then we'll have more patients coming back. That's the dynamic to look forward to. Maybe, Keith, do you wanna talk about, gross to net?

Yeah. Brad, with respect to gross to net, I would just say, not sure exactly of your math, but I would say that, you know, we've consistently guided since we launched that we expect gross to nets to settle in the 20%-25% range. We're still squarely within that range, and you know, I'm not changing our guidance at this time. We did see, you know, what I would call typical 1Q seasonality impacts on the gross to net just due to the high deductible, mostly due to the high deductible commercial resets and the Part D donut hole. The, you know, the impact was still landed us within the guided range.

Thank you. The next question is from Stephen Willey from Stifel. Stephen, your line is open.

Hey, good afternoon. This is Josh on for Steve. Thanks for taking our question. Do you think that the data from the phase II Axa-Rux combo trial would be sufficient to potentially support a compendial listing for frontline GVHD? How do you think data from this trial will serve to inform or de-risk, if at all, the phase III trial looking at AXA in combo with steroids in the frontline setting?

Thanks, Josh. Good question. Let me turn it over to Nick to handle both of those, I think.

I think it's a well-designed study and a very interesting hypothesis that you might be able to avoid steroids in the frontline setting, so this is a potential steroid-setting approach. It's 120 patients, about 40 patients per arm, and is basically I mean, it's a noncomparative randomized study, but really the benchmark for this is about a 40% response rate using standard NIH criteria at around six months. If you see a meaningful improvement on that, let's say 60%, which is kind of aligned to where the study would be, I think that could not only inform clinical practice, but potentially also compendial listing just because of the morbidity associated with long-term dexamethasone.

We will also look at a number of other clinically meaningful endpoints in that study, like the time to actually have to reintroduce steroids, and if you can really delay that, again, I think clinically that's significant. Duration of response and time to events or other endpoints, and I think when we look at the totality of those, it'll be very informative both to practice and compendia and could inform future registrational studies. The dexamethasone combination phase III study that you mentioned is a different hypothesis. This is where the axatilimab can actually add to dexamethasone. So it's a slightly different hypothesis, and that could offer an alternative standard of care. They have quite complementary mechanisms of actions, and that is a pivotal phase III study.

Thank you. The next question is from Faisal Khurshid from Jefferies. Faisal, your line is open.

Hello, this is Ananchon for Faisal. I wanted to follow up on the progress in the first-line AML phase III trials. Where do you see yourself relative to the competition there? Maybe a little more on EVOLVE-2 and how you see that stacking up in the end. Thank you.

Thanks, Anan. Let me first comment. I'll let Nick make the other comments. The progress on the trials, we're doing quite well. I think as Nick remarked, or in his written remarks, you know, this is a period of standing up our trial, standing up sites, enrolling patients. Everything is on track and going quite well. We feel we are, you know, going to be first at frontline. We feel confident in everything that we're doing to execute against our timelines. Everything's on track. I'll turn to Nick, maybe more about EVOLVE-2.

Yeah. No, thanks. I mean, simply this is one of the highest focuses for my teams. It's a very high priority, and we're spending a lot of time on this, and we have two different approaches. EVOLVE-2, again, this was the first study to start enrolling, so it's been enrolling now for nearly a year. We're working with HOVON, which is giving us a fantastic network of sites across U.S., and we are also now entering sites in the U.S., making a lot of progress with site activations and indeed patient enrollment. In fact, some of the metrics around patients per site per month are actually in somewhat in excess of what we modeled, and that's quite exciting because I think it speaks to the physicians' enthusiasm and the support of the HOVON group to enroll this study.

Because we've generated such a body of evidence with Ven/Aza, we actually feel very confident in the design and some of the statistical assumptions of that study. It's moving along very nicely, and our expectation is to be first with the readout for that trial. Likewise, REVEAL, we've made a lot of progress. As you know, we recently confirmed the dose at 162.70 in combination with intensive chemotherapy, which is great. We're working with a leading CRO internationally. We're busy setting up sites in Asia and activating sites, and again, we're running very much to track. We're feeling very confident about that study as well, and more on that to come. No, the teams are feeling confident, and we're in a good place.

Thank you so much, guys. Appreciate the call.

Thank you.

Thanks, Anan.

The next question is from Mayank Mamtani from B. Riley. Mayank, your line is open.

Yes. Good afternoon. Thanks for taking our question and congrats. A lot's going on for you guys. Just any chance you are able to share, you know, the absolute number of patients launched to date that have returned post-transplant? On the 300 new patients added in track quarter, you know, if you can give any color on the mix NPM1 and KMT2A versus what we saw in the prior quarter and if anything on the NPM1 you can share on duration and co-mutation use, you know, relative to what you had maybe initially expected last quarter. That would be great to hear. I will follow up.

Yeah, Mayank, thank you for the question. On the absolute number of patients, I think, maybe I'll ask Steve to make a comment if we have, if we have that data.

We can estimate. We don't have exact numbers.

Yeah.

that I would share right now. We know we've treated 1,400 patients roughly from launch date through now.

Yeah.

Break it down by KMT2A and NPM1 patients. It's probably at least a few hundred at this point. More.

Yeah. Yeah. I mean.

We can come back with.

Part of what the challenge of breaking down KMT2A versus NPM1 is it doesn't work like that in claims, so you have to make some certain estimates. You know, that's a little bit trickier. Your question about co-mutations was, you know, what % of patients have co-mutations. I think that was my interpretation of your question. I think, you know, broadly speaking, NPM1, you know, a high degree, 85% of patients have co-mutations. You know, about half of those have FLT3 mutations. We're running trials to look at combinations and things like that in terms of the FLT3 population. Other patients have IDH mutations as well.

It breaks down as a, you know, multifaceted population of patients that you have to deal with combinations or, with, in some cases, we use venetoclax and azacitidine to combine our drug with in order to handle all of that. I think that's, you know, that's the extent of, you know, the co-mutation question. We'll leave it there for now.

Duration. Thank you, Mike. Duration in NPM1, could you comment on that?

Yeah, thanks. Duration, I mean, we had talked about duration of NPM1, you know, over time being in the roughly seven to nine-month range. You know, I think it's early days to look at, you know, and have a duration calculation specifically for NPM1. We had talked a little bit more about the KMT2A population and how that's kinda coming together with the impact of more patients going to transplant and returning for maintenance. As Steve mentioned in his remarks, patients who have actually received a transplant and have gone on to maintenance are on average out to nine months and counting. That's a very positive forward indicator of where that business is going. I can think that, you know, NPM1 is certainly tracking with patients staying on drug for multiple months.

Some will get transplant, most will not. That'll be an impact, you know, a factor in how duration ultimately plays out. We expect patients to do quite well and, you know, be on drug for months.

Got it. On the azacitidine plus drug study, the phase II, you know, timeline got pushed up. Is the phase III steroid study that, you know, Nick, you just mentioned, is that also tracking ahead of plan? I believe you said early 2028 readout. If you can clarify that. Thanks for taking our questions.

Yeah, I know. That timeline's the same. Nothing's moved out. They're independent of one another, as you know, Nick mentioned, the trials are different trials. Everything's different about them. No, I wouldn't assume that timeline has changed.

Gotcha. Thank you.

Thank you.

The next question is from Etzer Darout from Barclays. Etzer, your line is open.

Great. thanks for taking a question. maybe if you could just provide any color commentary or even guideposts on 2026 revenue guidance, and then any color as well or commentary on commercial dynamics with just different NPM1 call points with Revuforj on the market. Anything you can provide there would be great as well. Thank you.

Sure. Thanks for the question. First, maybe I'll turn to Keith on how we're approaching revenue guidance, which is gonna be easy to answer.

Easy to answer. Sorry. Etzer, we don't provide revenue guidance. You know, we're early in the launch of NPM1. There's competition out there, so probably wouldn't be responsible for us to go out and give guidance at this point.

Right. Then anything on the commercial dynamics between us and our competitor? I think that was the genesis of the question there. Steve?

Yeah, I mean, dynamics we can share. Competition's always good for us to be at our best. I think importantly, menins are exciting. You know, lots of physicians are interested. Awareness is there. It's an obvious path in KMT2A. We've done very well and met and probably exceeded physician and patient expectations. The NPM1 patient's different. I think you can see by the nature of these calls, there's co-mutations. It'll build differently over time. We think it's a good thing that there's another menin player in the market, raising awareness, finding a place for menins. We've shared, and Michael's shared thoughts on just our profile, how physicians think about it. We've got a better profile in NPM1, so anything that's done on behalf of the class will have benefit to us as the market leader in NPM1 and in KMT2A.

We'll see how it rolls over the coming months, but we think it's a good thing, and we think ultimately we'll benefit from it.

Yeah. I would just say that, you know, NPM1 is a very considerable piece of our business, it's growing. We feel very good about our competitive position. Steve's organization is, you know, essentially, you know, built us into the market leader, and we expect to continue to build that piece of the business, competition or no competition.

Okay. Thank you.

Thank you.

The next question is from David Dai from UBS. David, your line is open.

Great. Thanks for taking my questions. Just on the Niktimvo IPF, what levels of investigator or payer interest are you seeing today in Niktimvo's anti-fibrotic potential? How might positive IPF data change the commercial opportunity long term?

Thanks for your questions, David. Two questions related to Niktimvo. One, Excuse me, payer interest around IPF. I think that was the first question. Steve, do you have a comment on that?

I really don't. I mean, this is handled. We know there's interest. There's clearly unmet need. The drugs that are there don't work incredibly well, so you're gonna see payer interest serving that population. Work is generally done by Incyte.

I mean.

To handle that piece.

Yeah.

What I would just add is that, I mean, this remains an area of high unmet need. It's poorly served by currently approved therapies. I mean, there are three, maybe four therapies approved. None of them are very satisfactory. The survival and prognosis of these patients is very poor, and no drug to date has really impacted the natural history of the disease. There really is, like, a great need for a drug that could potentially impact the natural history of the disease. You know, that's the hope with axatilimab, that it might be able to do that.

You know, from an investigator and physician perspective, the support we've had from investigators and their desire to contribute in a potential phase III is very high, which I think speaks to their interest in a novel and differentiated approach to the treatment of IPF. We're feeling very good about that.

As you know, the market opportunity here is quite large. We're talking about 150,000 patients or thereabouts in the U.S. This would be a new mechanism for this disease. If we're able to show in this trial meaningful impact on the efficacy endpoints and safety as well, we'll, I think, have a very interesting and compelling proposition in IPF to serve patients. It could be a, you know, sort of a, I'd call it a game changer relative to market size. I mean, GVHD is considerable as we've laid out. We think we have a very compelling proposition in GVHD alone. Once you add in IPF, it's a, you know, obviously a completely different size of opportunity in the U.S. for us to get involved. Excited about that.

Thank you so much.

Thank you, David.

The next question is from Yigal Nochomovitz from Citi. Yigal, your line is open.

Hi. Great, thank you for taking the question. On RAVEN, I had a question. If that study, that phase II looks good, would you then consider a phase III, given the regulatory pathway is a little less well worked out relative to what you're doing with REVEAL-ND? That's my first question. I was also interested, you know, you mentioned that you're guiding to 50% to transplant, up from the 33%. You're still restarting about 70%-80% on Revuforj. I was just wondering if, you know, why that number was staying the same. If you're increasing the number going to transplant, I thought perhaps maybe you'd get even more restarting, but maybe that's not the right logic.

Just curious to how you think about that part too. Thank you.

Thanks, Yigal. Let me just clarify on the second question. We had said that we're getting to about 50% patients transplanted, that was correct. What we expect as the target is 70%-80% of patients to come back. Not everybody will come back for maintenance, but the vast majority we expect to get to that point. Right now, we're not there yet, right? We're at kinda approaching half of the patients coming back for maintenance. That number will grow. It'll grow based on our confidence from the data that we've put together, some of which will be presented in the coming weeks at some of the medical conferences. We've heard from physicians, this is what they expect. Expect to put patients back on maintenance. They feel very strongly about that.

This is what we expect will happen, and we feel very confident in that over time, but we're not quite there yet. That's the dynamic we're experiencing. It's great for patients. Many more going to transplant. The funnel, the actual funnel of patients who are available for maintenance is expanding, and so that's all very positive driver of business going forward. The second question related to RAVEN, and I'll turn it to Nick.

Yeah, happy to take that question, and we're excited about this hypothesis because this is a bit of innovation in an area where, you know, patients could potentially get an alternative and a better-tolerated approach. You know, these patients would normally get intensive chemotherapy in order to try to get them to transplant, and the hypothesis here is by giving them venet and revumenib, you can get these, you know, fit KMT2A patients, many of them quite young, to transplant without all of the morbidities. Now, in terms of its intent, clearly that's a high, you know, an area of high unmet need. We haven't talked about the specifics of the study design yet.

Clearly a compelling result that you get high rates of transplant and good outcomes with a much better tolerated regimen would certainly be, you know, informing a clinical practice, potentially guidelines, and we would even be thinking about, you know, how would that support a registrational approach given the totality of the data we're gonna be generating in the frontline setting. More on that to come, but we think it's a very differentiated and innovative approach to this patient population.

Thanks.

Thanks, Yigal.

The next question is from Jason Zemansky of Bank of America. Jason, your line is open.

Good afternoon. Congrats on the progress, and thanks for taking our questions. Two, if we may. Wanted to return to the subject of Revuforj access. There was some discussion last year in the community that some plans were requiring step edits through one menin inhibitor. You mentioned there was some confusion over the relative cost of Revuforj. Was curious as to whether or not this was related and, can you speak to whether or not you're seeing any step edits throughout the universe? I guess secondarily, you mentioned that about 45% of KMT2A patients were resuming therapy post-transplant. I think last quarter you cited a range of 40%-45% of patients. Just curious, what kind of gets you through that barrier of, I guess, 45%?

Is this a case of sort of fast adopters and very cautious adopters? You know, really what's it take to get more of those docs on board? Thanks.

Thanks, Jason. Good question. Let me start with your question about access, which I'll turn to Steve, and he can address.

Yeah. As you know, the access is great. You know, 97% across both indications. Not only a high number, it did it very quickly. Plans have been reimbursing Revuforj since the launch. Your point about step edits, and I did mention this in my comments, there's literally three plans that have step edits in place, and it's really restricted just to the 270 mg dose. It's not a lot of patients. It's less than 1% of the overall. Some of that came about on a monograph, which I also referenced in my prepared comments.

A monograph that came out later part of 2025 and just had some incorrect information and made some assumptions just based on dosing, that if you were to exclusively prescribe the 270 mg dose, you would have a higher WAC than the other menin inhibitor. We know that that is not the case. In fact, most patients, 75%-80% of them are not on that dose. The WAC ends up being lower, about 15%-20% lower than our competitor at the one dose that they have. Since the monograph has been adjusted, they've corrected the fact base, they've removed the guidance on a step through our competitor at that dose, and the recommendation is no longer there. Plus, they updated it for other reasons, clinical, non-clinical, so the information is there.

The step that is in place is practically ineffective, and physicians get to choose what they want to. According to our information, there's not one patient that has had to walk through a step for Revuforj to date, and we don't expect that to continue moving forward.

Great. Thanks, Steve. I guess the second question relates to maintenance and how do you increase maintenance beyond the 45%? I'll just remind you that we've actually increased the use of maintenance throughout last year. You saw it kind of go from about, you know, a third of patients all the way up to 45%. It is steadily increasing. We do, as I said earlier, that, you know, data and real-world evidence will ultimately drive it. We have a robust strategy in place to deliver to get to this steady state, 70%-80% point, which will happen at some point in the future. We can't predict exactly when. I was a little off on my 50% prediction.

I thought that would happen later, and it happened more quickly. We are very, you know, bullish on the fact that we will get there. All the data that we have coming out, and we're very focused on this, should help physicians think through how best to dose patients and at what interval. You know, they're just. Some are very cautious about patients coming back from transplant, as you can expect. Their engraftment period varies by patient, and physicians want to make sure that count recovery is well underway and that they're managing patients really effectively. We help them with that, where there's lots of data that's coming out, and ultimately, getting to that 70%-80% should be quite achievable. Thank you.

Great. Thanks for the color.

Thank you.

Once again, if you have a question, you may press star five on your telephone keypad. The next question is from Salim Syed from Mizuho. Salim, your line is open.

Hey, great. Congrats on the quarter, guys, and progress. I guess two from us. One on just MAXPIRe, and then just the other one on the Revuforj chart you provided on slide four. On MAXPIRe, Nick, could you maybe remind us, I understand it's a robust study, but could you remind us what your power to show the 26-week time frame? I presume the powering was done on the 26-week and not the 52-week that you plan to model to. And then what the variability or standard deviation you're assuming on the primary endpoint? That's question one. And then question two, just on Revuforj.

I guess, like, one of the dynamics here, it looks like from this chart, which I thought was super helpful here, it looks like on KMT2A, you guys actually, like, maxed out sort of at this 200 new patients per quarter pretty quick. Are you anticipating the same sort of dynamic on NPM1 to max out with, you know, new patients per quarter within, I guess, like, maybe three quarters here or so, four quarters? What would be the underlying dynamic why you would max out so quickly? Thank you.

Wanna answer the, Nick?

Yes, Salim, thank you for the question on MAXPIRe. It's a good question. What I would say is, I don't want to go into the specifics of the statistical assumptions, but I would say it's a very well-powered study. We plan to recruit 135 patients. We actually slightly over-enrolled because of the physician support, we over-enrolled by about 10 patients. It's a 2:1 randomized study with an FVC primary endpoint. It is well-powered, and we'll look at an annualized rate, and we'll use the standard statistical modeling to report out the annualized rate. You know, rather than what it's powered for, what I would suggest to you is, you know, what we would want to see, because we're actually looking for a fairly significant difference, you know, in order to inform a phase III design.

We're not looking for small incremental differences. You know, in terms of the rate of decline, somewhere in the region of a 40% improvement in terms of the reduction in the rate of decline would be really meaningful. That's the kind of, you know, based on historical controls, the sort of figures looking at the totality of the data we'd wanna be looking at to inform a phase III. The study is well-powered to be able to show that type of difference. We'll let it read out and look forward to reporting those data in the fourth quarter.

Salim, I think your second question about dynamics on Revuforj. You know, I'll just simply say that when you think about KMT2A and NPM1, we have not maxed out. I think KMT2A is continuing to grow, as I mentioned earlier, incidence population of about 2,000 patients. It's a rare disease. You need to find the patients, I think we are, you know, getting as many as are available. We're the only menin in that space. We have the best profile, physicians are recognizing that the drug works very well for these patients. We expect to continue to build that market share well beyond what we're at. You know, every quarter is going to be a little bit different, I feel very confident about that.

NPM1, same thing. You know, there's no. It's a much bigger market, as you know. It's about 2x to 2.5x the size of KMT2A, and the opportunity there is large. We expect to, you know, cover the whole universe and get as many patients as possible. This should continue to grow. In NPM1, it's all about new patients. In KMT2A, it's about adding incremental patients quarter-over-quarter, new patient starts. I would say the increase in maintenance is in transplanted, as we talked about in maintenance, is going to be a big driver of growth for that segment as well.

I guess my question is more at the rate that they're coming in. It looks like that's what's maxed out, not the number of total patients. The rate that they're coming into, it seems pretty consistent here.

We've talked about.

You know?

Yeah, we've talked about consistent, a consistent rate of new adds, right? I wouldn't say we've, you know, we'll continue to penetrate and build. It will be a steady new rate of patients coming for KMT2A. NPM1 should be, you know, slightly different, larger market, and new patient starts will be more, certainly in the early stages of launch, be, you know, more dramatic. I think the, you know, the businesses and the way the business actually accrue revenue are slightly different, as we talked about.

Sure. Okay. Got it. Super helpful. Thanks so much, guys.

Thank you.

Our final question today is from the line of Andres Maldonado from H.C. Wainwright. Andres, the line is yours.

Hi, guys. Thanks for taking my questions, and congrats on the progress. Just two quick ones from me. First, a quick follow-up on axatilimab and IPF. I guess, could you provide a little bit more color on how we should be thinking about the phase II readout as primarily an all-comer FVC study? Or is there a biological subgroup, such that patients with maybe more monocyte/maybe macrophage-driven inflammation, where you expect to break out just based upon the fact that the CSF1R mechanism is stronger there? Maybe an underlying question for Revuforj.

I guess, you know, as we look at, you know, the NUP98 kind of opportunity, I guess, what would you need to see clinically to treat this as distinct, you know, expansion opportunity rather than just a scientifically interesting but commercially smaller subset? Thank you very much.

Hi, Andres, thank you very much for the questions. I'm gonna let Nick get into the IPF question.

Yeah, it's a very interesting and, you know, relevant question. Of course, we'll analyze the intent to treat population. Our expectation is that we'll see a benefit, you know, across all comers. These patients are treated on a background of antifibrotics, so we have stratified for by the type of antifibrotic they're on, whether it's nintedanib, pirfenidone or, you know, no anti-fibrotic. We will look at that, and we'll look at those subgroups. Given that we know that patients that have high levels of monocytes and high levels of CSF1R, we are expecting that and that that is correlated with a poor prognosis in IPF. We are expecting that the benefit would be seen across the intent-to-treat population. That would be our expectation and what we'd be planning to do with the phase III.

Certainly we will look at subgroups excepting in a smallish phase II, it's difficult to tease out differences in subgroups. I'm happy to talk a little bit about NUP98-

Sure, please.

...as well, just to start. Just from a clinical perspective, we have, as you already know, presented data on a small subset of NUP98, where we showed that three out of five patients had a form of morphological remission, which was very encouraging. We are planning to present additional data this year. I mean, biologically, it makes complete sense for these patients with NUP98R that they should benefit from Revuforj. You know, we've seen that across the spectrum. As we, you know, we plan to publish further data this year. Certainly, considerations for submitting for the consideration of NCCN guidelines would be something we would be thinking about. You know, that's the plan based on the data we've seen for this subset.

We probably estimate it to be somewhere around 5% of the baseline for, of AML. It's not an insignificant subset and clearly an area of higher met need because these patients tend not to do very well.

Yeah, I think that's, you know, as Nick said, 5%, it seems to be a somewhat underdiagnosed area. We hear this from physicians regularly now that Revuforj could be a good option for them. We've been following the HOX gene signature, which has, you know, kind of yielded NPM1, and now NUP98, and there may be other subsets. The ability to expand even beyond, call it 50% of AML is potentially possible. You know, we feel like we're, you know, on the cutting edge to make this part of, hopefully part of the portfolio. We're, we'll follow up on that. Thanks for the question.

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, operator. Thank you all. We appreciate everyone tuning in today to discuss our recent progress and the exciting milestones ahead. We look forward to seeing many of you at the upcoming ASCO and EHA medical conferences and, of course, several investor conferences in the second quarter as well. With that, have a great evening, everyone.