Good afternoon, everyone. Thanks again for joining us at the Goldman Sachs Global Healthcare Conference. We are thrilled to have Michael Metzger and Keith Goldan, Chief Executive and Chief Financial Officer for Syndax, here with us this afternoon. Maybe we will just kick it off with some high-level questions. First, how do you think about the core competencies of Syndax, and how does that inform your strategic priorities for the business over the near term? First of all, thank you, Corinne. Thanks for inviting us here today. Great opportunity to be in front of this audience. Syndax is in a great position as a company. You mentioned our core competencies. We have been able to bring two drugs all the way through development and get them approved and certainly launch them in the last year plus. It has been very successful for the company. That kind of informs our core competencies. I would highlight R&D and the ability to translate early science into products of meaning and bring them all the way through development. The R&D capabilities of Syndax are quite strong, have gotten more robust over time. That is something we lean on significantly to advance our medicines and really differentiate in the marketplace. Then, of course, the commercialization competency within heme today, we think is differentiating for us as we have advanced both of our medicines, and they are both heme medicines, to bring them to as many patients as possible. I think those are core competencies that allow us to advance the business and really drive differentiation over time. I would highlight those as really important. Great. Maybe more temporally, we are currently in the middle of a pretty busy clinical data period between ASCO and EHA. Could you speak to the key themes you hope doctors and investors take away from the presentations you have at those two conferences? It is an exciting time for us as we've been meaningfully represented at all of the conferences, most recently at ASCO and at EHA this coming week, where we have multiple presentations, both oral and poster presentations, for both of our medicines. I'd highlight for Revuforj, the significant data in combination with standard of care agents, venetoclax and INQOVI, also chemotherapy, seven+three. This is both in relapsed refractory and in newly diagnosed patients. We're running pivotal trials in newly diagnosed patients, we have a whole slew of data coming, both real world and controlled trials and single-arm trials that help inform and de-risk these combinations. I think that's extremely important as we continue to build the business, physicians get comfortable with using the drug both on-label and potentially off-label, and really solidify our leadership as the method of choice for many years to come. I'll also highlight the maintenance data that is becoming increasingly important, specifically around the KMT2A population for Revuforj, where patients are getting in higher, increasing amounts to transplant, which is a great outcome for patients. Then with Revuforj, you have the opportunity to go back on in a maintenance capacity, and that has become an important growth driver for the business. The data that we're seeing in the real world and around many of the centers that we're working with suggest that patients can do quite well in maintenance. We saw this at ASCO in an oral presentation where a subset of patients, KMT2A, NPM1, and NUP98, which is another important lesion that is able to be treated with Revuforj. We've seen that patients get maintenance and can stay on, as a landmark analysis in that trial, two years of 90%. That's a sea change versus what you've been able to see before the advent of Revuforj. I think these are the themes, combination data, treating patients earlier, the outcomes there, why it's meaningful to treat them, bring them to transplant, and also give them maintenance. That's what we're hoping to highlight. All right. One more temporal question, we're going to come back to all the clinical and commercial pieces. You recently did this convert, you announced it about a week ago. Maybe talk about that in the context of your overall financial strategy and funding the business. What does that allow you to do? Sure. Maybe I'll start and pass it to Keith. The convert was an opportunistic inbound from fundamental investors we believe are important to our business going forward. Low cost of capital for us relative to anything we've seen. Keith will outline the terms of it. Essentially, it gives us an opportunity to fund beyond our current business, right? We have a pipeline that's emerging. We'll talk about that at our upcoming R&D day. We're excited about these assets. We need to invest in them. We want to invest in them. We want to move them quickly ahead. We have a track record of success, as you mentioned earlier, bringing assets in, developing them optimally, and moving as quickly as we can. I think that is a use of the proceeds as well as for IPF. Beyond this trial that we're running, a positive study at IPF will lead to future development and expense, and we want to have the resources available to us to exploit that opportunity. Those are sort of the uses. Maybe Keith can describe. Yeah. I mean, I begin by saying that it was opportunistic. The deal came to us. It was unsolicited inbound by a fundamental investor that wound up anchoring the deal, that specifically wanted to participate in the growth of the company through a convert. It was historically low cost of capital for us. The deal was done at two and a quarter, up 35%. It's a nice addition to our capital structure, which is right now comprised mainly of just equity, as well as the synthetic royalty that we sold on Niktimvo to Royalty Pharma. It was a nice complement to the overall capital structure and use of proceeds, like Michael said. Great. Maybe digging in on the commercial side, you have REVYFORGE now approved in relapsed/refractory AML in both the KMT2AR and NPM1. If we start with KMT2AR, could you talk about the patient journey, where they see REVYFORGE today, what portion of patients are getting that therapy, and where that could go in terms of going to transplant, coming back to maintenance, et cetera? Yeah. It's a very important advance for patients. I mentioned it earlier, KMT2A patients are generally younger than NPM1 patients, for example. When I say younger, a lot of children, average age in our pivotal trial was about 35. The goal for those patients who hadn't really had a medicine before that was impactful for their disease, certainly not one directed at KMT2A, was to get them to remission, which our drug does. About two-thirds of the patients in our pivotal trial got to a complete remission, and then get them to transplant. You treat them for about two - three months, you bring them to transplant, and then the paradigm is after they've gotten through their engraftment period where their bone marrow repopulates, you can put them back on Revuforj to continue treatment and keep them in remission. Right now, what we're seeing, we had about 25% in our clinical trial going to transplant, which is a big difference versus what they historically had before Revuforj. Now in the real world, as we reported in our last quarter, we're at about 50% of patients going to transplant. About 70% of our patients are treated either second or third line, so that's earlier than they were in the clinical trials, drives them to higher response rates and ability to get to more transplantation. Now what we're seeing, patients coming back, about 45% of the patients who go to transplant come back for maintenance. We expect that number to grow. We expect it to grow because physicians tell us they want to do it universally. They believe that this is an important impact on patients. We also have seen it in the data, starting to see some real-world data suggesting higher rates of maintenance. We expect that to go to somewhere in the neighborhood of 70%-80% of the patients going on to maintenance at steady state. That's the paradigm, and that we expect will be a big driver for the business going forward. Okay. With those in mind, you said that 6 - 12 months should be the expected duration of therapy, that's a pretty big window. Yeah. What do you need to see to narrow that, and where could it go over time? Certainly, it could go 6 - 12 is a wide window. first year on the market, we saw average duration of therapy in the four - six-month range. That was due to the fact that you didn't have the impact of maintenance longer term, right? You only had 12 months on the market. You didn't have the opportunity for patients to go through their engraftment period and go back on and have the impact of maintenance. second year, we said somewhere in the 6- 12-month range. We now know about, call it 50%, 45% of the patients are going on to maintenance. We expect that number to grow meaningfully over time. That will drive the duration of therapy, 6- 12 months. Could it go longer, in the future? Yes. Physicians have told us they intend to give in the neighborhood of 12 - 24 months of maintenance on average. Okay. We need that time to build through the 6- 12 months in the second year, but we do think it's realistic. As you think about where you are, maybe you could mark to market from when you launched this product in KMT2AR to today, how are you tracking in terms of the metrics that you cared about at the time, whether it's market share or percentage going on to transplant or maintenance, et cetera, versus your expectations? Well, we're certainly tracking with expectations, maybe even exceeding expectations. It's a 2,000 patient incidence for KMT2A in the U.S. We penetrated about 50%, approaching 50% in year one, which sounds very high and is very high. There's really nothing indicated for KMT2A, and standard of care is not effective, as I mentioned. That 50%, we believe could grow to 70%, 80% market share. That's about as high as you see for a targeted therapy. That's essentially where we expect it to go. How long it'll take to get there? Probably in the next year to two years, we expect to be there. Okay. The more recent approval for Revuforj was in relapsed/refractory NPM1 AML, and maybe you could just start by characterizing the competitive positioning for Revuforj in that patient population where patients have more options. They do have more options. NPM1 is a larger patient population, roughly 4,500 patients in the relapsed/refractory setting. They have co-mutations, NPM1 is a driver mutation. They are often co-mutated. About 85% of the patients have some other mutation. Some are actionable, some are not. I will mention two. One is IDH, IDH1 or two. There are medicines approved for those two mutations. There is also FLT3, which has two different drugs approved for FLT3. Physicians, when they are thinking about treating NPM1 patients, often will reach for standard of care medicines in addition to Revuforj, that could impact when they actually give a menin inhibitor such as Revuforj in the treatment of a patient with NPM1. We do expect to have a dominant share. The data that we have put out so far is quite significant, stacks up well against any competition. Efficacy is the most important driver of choice for physicians, why they would use our drug over another drug. Our drug has the highest level of efficacy in the category. The drug is very well tolerated. There is a lot of history now, use of the drug. Same physicians are actually treating KMT2A and NPM1, the expertise and the experience, I should say, treating patients with both now, even preceding the approval in NPM1, we had some utilization off-label for NPM1. Physicians are used to using the drug, are comfortable with the profile. The efficacy is strong, as I mentioned. It can be used as monotherapy now in combination, we are seeing about 40% of use in combination with drugs like venetoclax. There is a big body of evidence that the drug is effective, and I would say that the safety is well-chronicled. The drug is easily tolerated. Safety is well-managed. We understand it well. Physicians are, in turn, feeling very comfortable with the profile, compared to what else they have to utilize for these patients. Okay. Between the two, you have put out a $2 billion or so peak sales opportunity estimate. Yeah for the drugs. Now that you're on the market, have you learned anything that would shift that expectation? Well, I think we feel like it's effective, as I said, monotherapy and in combination, and the ability to treat patients in combination will drive utilization earlier. When we mention the total addressable market, we're talking about treating the absolute most patients you can in the category. In order to do that, you need to be able to treat them earlier, and I think using the drug in combination will impact that. I think that is one potential learning, as well as the fact that I would say for NPM1, historically, there have not been a lot of transplants. We don't expect there to be as many transplants as there are in KMT2A. However, we are seeing patients getting transplanted that are NPM1 specifically on Revuforj, and that's sort of an upside. Okay that we see. We're excited about that. Another kind of source of potential upside is the data you've started highlighting with the NUP98 population. Yeah. Can you expand on the size of the opportunity and the role Revuforj could play there? Right. It's another lesion. When we discovered that NPM1 was an area of interest, it was based on the fact that there's an overlapping gene signature with KMT2A, the HOXA9 signature, upregulation of HOXA9. NUP98 is another such lesion where there's upregulation of HOXA9. The drug has been showing up in some of our datasets, and it's exciting to see the impact on that specific lesion. We'll have more data at EHA to highlight that presentation specifically around NUP98. I would look out for that. It's an important area. We're learning that as it used to be thought of as more of a pediatric indication, it's an area where we're seeing adult patients actually show up with NUP98 as well. What we've heard from experts is that it could be as large as five percent of AML overall. No overlap with KMT2A or NPM1. There's no co-mutation relative to NPM1. That's a discrete patient population that could add meaningfully to what we're doing, both in relapsed/refractory disease as well as frontline. Again, it's what we would expect as menin inhibitors. We expect to be discovered to be able to be used in other lesions, not just the three that we've been talking about, but potentially more in the future as we track that HOXA9 signature. In terms of the development strategy to make sure physicians understand they can use it there and that they do, what kind of investment are you talking about in making sure that gets educated, et cetera? We have some investigator-initiated work. We also have some real-world data that we're putting together. We'll continue to put that data out at conferences and potentially have a strategy to get it into guidelines, which would be impactful for physicians. Okay. Beyond the relapsed/refractory patient populations we've just been talking about, you also have clinical efforts in the frontline setting. Maybe as you look at the frontline, how do you think about the pockets of unmet need and how Revuforj kind of might be able to satisfy those? Yeah, certainly. The unmet need is probably the highest in the unfit population. There's unfit and fit within AML. Fit is generally patients who can withstand high-dose chemotherapy, and unfit are generally older patients who do not take the same regimen of chemotherapy. They generally get Venaza. Venaza is approved and is now actually very impactful for patients, and it's thought that maybe even patients who are fit for chemotherapy might be better off with taking Venaza. That's an emerging thesis. Some data at ASH this year that suggests that that could be the case, a lower impact regimen from a chemo perspective. The area of highest unmet need in the frontline is certainly the unfit patient population. Right. Our data so far is the most robust in combination with Venaza. We've been able to show impact on CR rate well beyond the doublet in a phase I/II trial called BEAT-AML trial. We continue to update that data, and we have overall survival as an important readout in that trial toward the end of this year. That's an important supportive dataset. We are running a pivotal trial in the frontline to get the drug to as many patients as possible, as quickly as possible, and it's called the EVOLVE trial. Okay. Maybe you could double-click on that a little bit. Sure In terms of what we expect to see from a clinical data generation perspective over the next 12 - 24 months in the frontline setting. Right. We're enrolling this 400 - 500-patient trial called EVOLVE. It's a randomized trial on top of standard of care, Venaza. It's a dual primary endpoint, independently powered, so you can win on either. CR is the accelerated approval endpoint, OS is the confirmatory endpoint, that trial is up and running. It's a global registration trial, it's up and running now. We expect to open several hundred sites around the world and enroll the patients over the next one year - two years. That's the timeline. We haven't given guidance on when we expect to finish that enrollment exactly. It's something where we started first. We expect to be first to frontline, and I think this is the trial that will likely get there before others. The second trial is the REVEAL trial in the fit patient population, similarly sized with an accelerated approval endpoint, as well as event-free survival as the other endpoint, the confirmatory endpoint. Both of these trials up and running, enrolling patients globally, and on plan to get there first. You mentioned Beat AML, and I think you anticipate having some updates for that program as well this year. How should we think about benchmarks from Beat AML and how they might translate into our confidence in the frontline strategy? The VIALE-A was the approval trial for venetoclax alone, and that is, I think, widely accepted as the benchmark with a CRR rate in a 30% range, 30%-40% range, as well as overall survival in the high 14, around 14 and a half months, that range. What we're looking to do is improve upon both. So far, Beat AML has shown us on a CR where close to 70%, so almost double what we're seeing with venetoclax alone, and overall survival on a very immature look at seven months, we were out to about 15 months. Already pacing a little ahead of where venetoclax was alone. We do expect to do a mature look at the data at the end of the year, which will be many more months of follow-up, bigger data set. That's of interest to us as well. We think will add meaningfully to the overall survival piece of the puzzle here, which should portend well for de-risking the EVOLVE trial. Okay. How should we think about the market opportunity in the frontline setting relative to the other? In the relapsed or refractory? Yeah. The newly diagnosed setting, about 9,000 patients between the fit and unfit patient population. It's about 55/45 split between patients who are fit for chemotherapy versus patients who are not fit for chemotherapy. That opportunity, if you think about it, we like to think about it as a $5 billion-plus setting in the sense that you have 9,000 patients at roughly $40,000-$50,000 a month for the drug, WAC pricing, and patients staying on for likely 12 - 24 months is what we expect in the frontline setting. That's the calculation you get to that kind of total addressable market of about $5 billion. Maybe just last, before we talk about Niktimvo, how do you think about the competitive landscape in frontline versus the competitive landscape that you are currently situated in? Well, we were first to get a drug approved in KMT2A and NPM1. I expect that we'll be first to frontline as well. We started first. We have the most data there. Physicians are extremely enthusiastic. We're working with the best groups in the world to get those trials up and running and enrolled. I think we have a very good setup to be first. I think the profile that we've shown to date with the combinations has really shown itself to be as pretty much as good as you can get. We feel very encouraged by the data. We feel encouraged by our position, and we'll have a dominant share once we get there. I think it's important to be there first, and that's what we're set up to do. Okay. Niktimvo was approved in August 2024, it's a partner product with Incyte. Maybe you could just talk about, again, mark-to-market on the launch to date and how it compares versus your expectations in chronic graft-versus-host disease. Niktimvo was approved in third-line plus chronic GVHD, as you all know. It launched in February of 2025. In the first 11 months on the market, did $152 million in sales. Closest competitor is a product called REZUROCK, which was also approved in third-line GVHD a couple of years prior, we outpaced what they were able to do in their first 12 months by a nice margin in terms of sales, in terms of patients treated. That's usually not how it goes. Usually, order of entry, you have some step down off of the prior drug that has been approved, that was not the case with us. I would say it surpassed our expectations in the first year. The product is very well accepted by physicians at this point, it's very broadly covered, not only by payers, physicians are writing the product, that's because the efficacy is probably the best they've seen in the category for heavily pre-treated patients to see that kind of level of efficacy and tolerability is really encouraging. Physicians are warming to the product. We've seen about 50% penetration in fourth-line, about a third of patients in third-line, growing meaningfully. That has been a nice start for Niktimvo. We do expect it to grow meaningfully from here as we continue to penetrate third-line and waiting on combination trials. We have an important combination trial reading out in the Q4 of this year that could drive utilization, really open up Niktimvo frontline opportunity for additional growth. Okay. Since you just mentioned that, I guess, could you talk about the broader development strategy for Niktimvo in the context of GVHD, including the subcutaneous product that's in development? Sure. You mentioned Sub-Q. Sub-Q, right now the product is an IV, dosed every two weeks or potentially once a month as well. Physicians have the opportunity to do that. The product, we believe, will grow meaningfully once we have combination data with frontline, or I should say other agents that are approved standard of care. Ruxolitinib, Jakafi is approved in second line, steroids are approved in first line. We have trials combining with both. At the end of this year, in the Q4, we'll have data on a very interesting phase II trial where we combine with ruxolitinib. We also have a RUX arm in that trial and a steroid arm in that trial. Seeing the components will be pretty revealing in terms of what we can do. Can we improve upon the standard of care, RUX alone or steroid alone in that population, again, newly diagnosed patients? I think that's a very important trial. In the Q4, we're also running a pivotal trial in combination with steroids. Steroids are effective, but you tend not to want to use them too long, and they have some difficult side effects. Tolerability gets a little challenging as well. You want to be able to use steroids early on in the treatment course, but turn them off and wean them off. The ability to combine with axatilimab or Niktimvo and really keep efficacy at a high rate while safety and tolerability keeps pace, I think that's the name of the game, eliminate steroids from that regimen over time. That pivotal trial will read out in early 2028. These are the key trials that we're launching in order to, or carrying on in order to really solidify our position, build our position in chronic GVHD. You mentioned Sub-Q. Sub-Q is a nice add-on, potentially for GVHD and even for other indications such as IPF. We are running an important trial. Yeah In IPF. The ability to give the drug, perhaps once a month, as a Sub-Q could open up some opportunities for us within that paradigm. Okay. Maybe in the interest of time, want to shift to the IPF trial. Sure. Thank you for the segue. In terms of the data update that we're going to get later this year, I guess, could you remind us of the trial design in terms of patient inclusion, exclusion criteria, and you're just confident that this phase II will be representative and de-risking of a registrational program? Precisely. That's exactly the setup. The setup is a randomized trial, two to one on top of standard of care. Patients can receive pirfenidone or nintedanib as background therapy. We will stratify for those patient segments. It's a 26-week endpoint, so a good test of the medicine over a period of time, which we think translates well into a 52-week potential extrapolation of that data. The trial is, as I said, it's 135 patients, so it's, again, randomized. 135 patients. We actually over-enrolled it a bit, so it's about 145 patients. Well powered to show at least a 40-milliliter difference in decline between the placebo arm and the active arm. This is a FVC, so the endpoint is FVC, and it's the approach you want to be able to extrapolate to that phase III. We believe that this will be a good test. It's well powered. to show a meaningful effect on FVC. We'll look at a variety of secondary measures as well, DLco. and quality of life measures. This has the opportunity, if the drug works well in this category, to be a fantastic add-on as a therapy that doesn't have drug-drug interactions. It's an antibody, so it could combine very nicely with other standard of care agents, and we'll be testing on top of standard of care. For us, we feel like this is a very precise and well-conducted phase II proof of concept trial. One of the questions we get is what the level of confidence is that six months is enough time to see a separation between patients that are on the treatment arm versus patients who are not. What is your level of confidence that you will, and that you've enrolled the right patients to see that separation? Well, 26 weeks, if you look at the trials that have been positive and those products that have gone on to prove themselves on the 52-week endpoint in larger pivotal trials, you look at the curves at 26 weeks, and they separate, and they stay separate. I think based on what we've observed from other trials, we should be able to have a good result and have a durable result by the 26-week time point. We feel quite confident in that. It's not true of trials that were on the 12-week endpoints. That's what drove us to power it up, use a dose that we feel very comfortable with, which is the 0.3 milligram dose, and stick to the 26-week endpoint. We're including patients, as I said, that are stable on background therapy, so they have to have some meaningful respiratory function, but have frank disease. We're looking at adding to what standard of care is and obviously staying on therapy is going to be important. You want to have enough fitness where you can test the medicine in those patients, but not lose patients because they're too sick to participate. Correct. IPF is also a very busy clinical indication right now in terms of competitive agents. How do you think about the fit of this drug and mechanism of action relative to the other ones in the category right now? The mechanism is a CSF1R inhibitor. CSF1, as we know in GVHD, is upregulated and affects the disease-causing macrophage. Similar in IPF, where you see upregulation of CSF1 and actually having high levels of CSF1 is a negative prognostic factor for disease and progression in IPF. I think we feel a commonality of, if you can impact the disease-causing macrophage by downregulating CSF1, you have an opportunity to impact both fibrosis and inflammation. That is a new mechanism, different cell type than anybody else is targeting within IPF. If we have the opportunity to show this in this trial where we impact the disease, it'll be the first time CSF1 has been able to show those types of effects. Excuse me, effects. As I mentioned, we have a potential combination with other standard care agents that impact other cells. Okay. Just remind us, because this is a partnered asset, how do the economics and decision-making work between you and your partner in Incyte? Yeah. Do you want to talk about that? Sure. The asset is partnered with Incyte for all indications. For research and development purposes, for any indication in the U.S., Incyte pays 55%, we pay 45%. We get advantage economics there, then commercially, for any indication, it's a 50/50 profit split, defined as net product revenue, which Incyte books, less cost of sales, less all of our advertising and promotion, gets you to a net product contribution, and that's split 50/50. We pick up the 50% on our P&L as collaboration revenue. In terms of the decision-making around the IPF program, what does that look like pending positive data later this year? Well, both parties will have a decision to make. We do these things together, but we also have independent decisions, and if one party wants to go forward, they can without the other. Most likely, with a positive result, we expect both parties to participate together in funding the trials and finishing the development. Okay. What does a registrational study look like in IPF, and could you give us a sense for timelines there as well? Well, we haven't guided on timelines. A little premature. Trial design will have something to do with the result that we see in phase II, and I mean specifically around the number of patients that we're going to need to include in the trial. It'll likely be one trial. We'll use this trial as a supporting trial for the package for approval. It'll be the standard endpoints, 52 weeks, FVC, secondary endpoints as well. It'll look a lot like what others have done. I don't think we're looking to blaze a new trail on a pivotal trial. Really carry through what we learned and recapitulate a lot of that from the phase II into phase III. Okay. You highlighted the R&D day earlier, I guess. We did. What should we be focused on as we head into that event? Yeah, look, I think the R&D day is our first opportunity to do this. We had announced, it was through the R&D announcement, that we have an early pipeline now that we are excited to talk about. It's getting to that stage, and it'll require some investment. We talked about the converter earlier, and that's one of the uses of proceeds to beyond 2026 to really accelerate the early pipeline, new science on strategy, precision medicine within oncology. Those are the themes. We're very excited about these opportunities. We'll also talk about IPF and GVHD and some of our ongoing work with REZUROCK as well, talk about frontline. So it's an opportunity for us to really put in perspective all of the R&D work, with some emphasis on some of the new projects that we're working on. Obviously all of that is internal, at least already internal. Yes. How do you think about the broader business development opportunity and capital allocation as you have a little bit more cash on the balance sheet shifting to profitability over the intermediate term? Profitability is something we can do with the existing cash that we have on the balance sheet. None of our expense guidance has changed. We feel like we are driving to profitability, that both products really contribute to that significantly, we'll get there reasonably soon. I think the new cash is a really important piece of the puzzle from a standpoint of growth, right? New products in the pipeline, as well as some of these adjacent opportunities, IPF being one of them, where we need to be able to invest in those opportunities alone. I think BD fits in from the standpoint of we're always interested in looking at new opportunities, we're very disciplined, keep a very high bar. We have some additional flexibility, I wouldn't conclude that we're going to go spend a lot of money on new business development. I think we feel very confident in what we're working on today and what we're bringing through, we'll talk a little bit more about that at the R&D day. Beautiful. Well, with that, I guess we'll see you guys in July. Thank you. Thank you so much for joining us, both for those of us who joined us here and online. Great. Thanks, Corinne. Thanks. Good to see you. Thank you.