Syndax Pharmaceuticals at Citi’s Biopharma: Strong Drug Sales and Strategic Growth

On Tuesday, 02 September 2025, Syndax Pharmaceuticals (NASDAQ:SNDX) shared its strategic vision at Citi’s Biopharma Back to School Conference. The company spotlighted robust sales of its flagship drugs, Revuforj and Ictimo, while outlining plans for future growth. Despite the positive trajectory, challenges remain in expanding market penetration and achieving profitability.

Key Takeaways

• Revuforj has generated over $50 million in revenue since its launch, with a notable 43% sales growth from Q1 to Q2.
• Ictimo, in collaboration with Incyte, reported nearly $50 million in sales in its initial quarters.
• Syndax aims to keep operating expenses flat through 2025 and reach profitability without additional financing.
• The company is exploring market expansion for Revuforj in NPM1-mutated AML and developing a subcutaneous version of Ictimo.
• Syndax is preparing for a potential FDA approval of Revuforj for NPM1-mutated AML, with a decision expected by October 25th.

Financial Results

• Revuforj has treated over 500 patients, capturing 25% of the estimated market for KMT2A-rearranged AML.
• Ictimo’s collaboration revenue exceeded $9 million in its first full quarter.
• Syndax secured $350 million through a royalty monetization in November.
• Profit margins on Ictimo are expected to be 25%-30% of Incyte’s reported net sales.

Operational Updates

• Revuforj’s average treatment duration is expected to extend from 4-6 months to 9 months by 2026.
• The company targets treating over 50% of the KMT2A AML patient population by year-end.
• Ictimo has been administered to approximately 700 patients, with 80%-90% remaining on the drug.
• Development of a subcutaneous Ictimo formulation is underway, with potential applications in earlier cGVHD lines and IPF.

Future Outlook

• Clinical trials are planned to combine Revuforj with intensive chemotherapy for AML patients.
• The NPM1-mutated AML market presents a larger opportunity with around 4,500 patients.
• Syndax is focused on expanding Revuforj into frontline AML and moving Ictimo into earlier cGVHD therapy lines.

Q&A Highlights

• Rapid uptake of Revuforj is anticipated with increased market awareness.
• The FDA’s decision on Revuforj for NPM1-mutated AML is expected to accelerate market penetration.
• Syndax’s partnership with Incyte strengthens Ictimo’s market position, differentiated by its unique mechanism.

Readers are encouraged to refer to the full transcript for a comprehensive understanding of Syndax Pharmaceuticals’ strategic plans and financial performance.

Full transcript - Citi’s Biopharma Back to School Conference:

Yigal Nochomovitz, Biotech Analyst, Citi: Welcome, everyone, to the, I believe, the last session of the first day of Citi’s BioPharma Back to School Summit. I’m Yigal Nochomovitz, biotech analyst here at Citi. It’s my pleasure to have with me senior management from Syndax Pharmaceuticals. Before we get started, if you have questions, just raise your hand and we can take questions from the audience. Welcome to everyone also listening on the webcast. It’s my pleasure to introduce Keith Goldan, CFO of Syndax, Steve Kloster, CCO, and Nick Botwood, Head of Research and Development. Welcome all of you. Thank you so much for attending. Keith, maybe you want to just give us a start. Obviously, you have two approved medicines now last year, two rapid approvals in succession.

If you could kind of just give us an overview of the commercial franchise and how those two launches, Revuforj and Ictimo, are going, that would be great.

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, sure. First of all, I want to thank Citi and Yigal. Thanks for having us. Great conference, as always. It is a really exciting time at Syndax. As Yigal mentioned, we have two, we’re in the midst of two really great launches. We are outperforming, which is a good place to be. I think it’s really a testament to the unmet need that we’re serving, both first-in-class products, Revuforj, targeting KMT2A-rearranged acute leukemia, and Ictimo, which is indicated for third-line cGVHD, chronic graft-versus-host disease. Both first-in-class products, we think best in class. Like I said, targeting areas of really high unmet need. I think that’s really complemented by outstanding execution by this team here over the last six to nine months. Both products, really long IP, we’re funded to profitability. It’s a great story. Like I said, we’re excited to be here.

Yigal Nochomovitz, Biotech Analyst, Citi: Awesome. OK, let’s get into a little bit of detail on Revuforj. This is the AML product. You have the approval, obviously, in KMT2A. Can you just talk a bit about the growth drivers? You’ve had a few quarters now of sales. How is it looking? What is the, I don’t know if you’ve given guidance per se, but just in terms of just qualitatively, you know what the momentum looks like. Of course, we’ll talk about NPM1 as well in a moment.

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, sure. It was a great quarter. I guess it was about a month ago we announced earnings through Q2. It was 43% growth in net sales. Q1 into Q2, we’ve generated over $50 million since the launch of the drug. We’ve finally started providing some metrics on the performance of the drug. We’ve been able to treat over 500 patients since the launch. This is through June. That’s on top of about 1,300 prescriptions across that patient panel. Yigal, I’d say there’s two drivers to the performance right now. One is new patients. The other is really duration of treatment. In terms of new patients, as mentioned, we’ve had 500 patients, KMT2A-rearranged patients since launch. That’s about 25% of what we consider the available market. We often talk about a TAM of about 2,000 incident patients. I think to note, that’s 2,000 new patients each and every year.

Treating about a quarter of them through June, we think is a great accomplishment. A lot of momentum, a lot of new patients being found that we’ll expect by the end of the year. We’ll treat over 50% of that population. We’re about 1,000 patients. We think it’s a good start. The other piece to the puzzle is really average duration of treatment. We’ve given previous guidance. Now we have data to confirm it. For this year, we would expect all patients, on average, to be on drug for about four to six months. That’s those patients who perhaps didn’t respond well versus those that were on drug for extended periods of time. Some dynamics that we’re seeing in the marketplace are really interesting. At the immediate launch, you saw much later line patients, much like we saw in the clinical trials.

These were patients that were fourth or fifth line. In the most recent quarter, we’ve seen that migrate to much earlier line patients. About 70% of patients we’ll call either second line or third line. Second line meaning first relapse. Likely, those patients, given that they’re treated earlier, are going to do better. They have a better chance of treatment success and ultimately stay on drug for longer periods of time. One of the dynamics that we see evolving is the percent of patients who go to transplant. It’s about a third from what we can tell from the data sources that we see. That comparison in the clinical trials was about a quarter. We’re performing better in the real world. That could go up even higher than that. We’ll have to see what time tells us. Those patients are likely from transplant.

Physicians tell us they want to go put patients back on as a restart or on maintenance treatment. That roughly takes about 90 to 120 days. That is exactly where we are now. The expectation is that as we move into 2026, the average length of treatment will evolve from four to six months to nine months. Those two things at play just give us a lot of confidence that we’re really building a very good business and doing some great things for patients.

Yigal Nochomovitz, Biotech Analyst, Citi: OK, you made a lot of interesting and important points there. The interesting point, you’re saying from fourth to fifth line to moving to second to third line, what do you attribute that to? Is that just getting your feet wet with some of the more sicker patients and then doctors getting more comfortable with earlier treatment? Of course, the question you could probably anticipate is of the transplant, the ones that go to the transplant, how many of those are coming back? Do you know yet if they’re coming back to Revuforj? That was something that was highlighted, I remember, at ASH and many other times.

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, maybe we could answer the first question. I’ll give my view. I think Nick probably has a view as a clinician on the movement. I think pretty typical. You enter a market at a point in time, and you have that range of patients, those that are newly diagnosed and those that are much later line. We heard of some patients coming off of hospice. I think that’s just a dynamic in the launch. All along, I mean, as we were even developing Revuforj, physicians want to use it at first relapse. That’s exactly what they’re doing. Part of it is finding patients, and part of it is just using the drug and having some success. That’s what’s led them to increasingly do that. Maybe I’ll pause there for Nick. Has any comments?

Yigal Nochomovitz, Biotech Analyst, Citi: No, they’re all important points. I would just add that, you know, two things. One is that physicians like to treat earlier in disease. We’re seeing that. We’ve presented a lot of data now for Revuforj in earlier lines of therapy, whether that be in combination or analyzed our data in the relapsed refractory setting for those patients that had had less prior therapies and shown really quite compelling evidence. It’s kind of typical as you treat earlier in a disease that you get better activity. I think when patients have identified a KMT2A mutation, to actually treat that with Revuforj is a compelling proposition. They want to treat earlier, and when you treat earlier, you have a better chance of a response.

You have a higher likelihood of going on to get a stem cell transplant, which particularly in the KMT2A population is really one of the ambitions of therapy. The other thing that we’ve learned, you know, talking with physicians across academic centers, thought leaders, and also community doctors, is given the precedent after stem cell transplant, you want to maintain those patients in remission. Their experience really dictates that many of those patients, they want to go back on to Revuforj to really maintain that remission after stem cell transplant. Even based on our experience in the clinical trials, we anticipate in clinical practice that more and more patients will, particularly if they have any evidence of minimal residual disease, either before the transplant or immediately after, want to go back on to Revuforj for maybe a year or two to really make sure they maintain that remission.

That’s the expectation of what we’re going to see in terms of a treatment paradigm and what we’re seeing in the clinic. The last point I’d make on this is we are working with a number of leading centers and planning registries actually to collect data in the real world of what the experience for patients are and hope to present some of those data later in this year. That may shed some additional light on the patients that go back onto therapy after transplant. Willing to give a preview, not the data itself, but the types of metrics that you would be collecting on that real-world evidence?

Keith Goldan, CFO, Syndax Pharmaceuticals: It’s preliminary for now. I think when we share those data back into this year, early in 2026, we’ll be able to show both the proportion of patients that actually go on to transplant and then those that go on to therapy subsequently. It’s too soon, I think, to share any insights from that data now. We are in a unique position, being the only approved menin inhibitor in the clinic with quite extensive use commercially now, to be able to work with physicians and other health care providers to collect those data, which we’re actively working to do, and then present the data that look at those important parameters. We’re looking forward to presenting it. I think it’ll be quite insightful when we do.

Yigal Nochomovitz, Biotech Analyst, Citi: Steve, you mentioned the 2,000 incident. You mentioned climbing from the 25% now, which is already a very good start, to 1,000. To get that, to close the gap, to get to even above 1,000, what is required there? Is there another level of investment, or is it just more market awareness, just more experience?

Keith Goldan, CFO, Syndax Pharmaceuticals: I would say it’s just more time. We’ve had enough time to understand how to work closely with treatment providers, and we’ve got a great customer-facing team that does that. We’ve shown success from launch through June, and we’ll expect that to continue. It’s been a robust stream of new patients coming onto trial. It’s hard to predict exactly when the patients are coming in. It’s not equal increments of a 12th of the 2,000 patients every month, so it may go up and down. We keep on course and keep executing, as Keith said, to find patients and make sure Revuforj is an option for them.

Yigal Nochomovitz, Biotech Analyst, Citi: Keith, of course, you have a very important FDA date coming up.

Keith Goldan, CFO, Syndax Pharmaceuticals: October 25th.

Yigal Nochomovitz, Biotech Analyst, Citi: October 25th, I was about to say. Yeah, tell us what that’s for and why that’s so significant for Revuforj.

Keith Goldan, CFO, Syndax Pharmaceuticals: I’ll let Steve talk about the commercial significance. We do have an sNDA being reviewed by the agency under RTOR, Real-Time Oncology Review, with a PDUFA date of, as Yigal said, October 25th, a couple of weeks away.

Yigal Nochomovitz, Biotech Analyst, Citi: Commercial opportunity.

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, so we’re excited. The treatment community is excited. Patients are certainly excited. For us, it’s a big driver. We talk so far, we’ve talked about KMT2A-rearranged patients. NPM1-mutated certainly is another driver. That market is bigger than the KMT2A market. It’s about 4,500 patients versus the 2,000. The patients are a little different. They tend to be a little bit older, more Medicare coverage. Fewer of them go to transplant. I think as an organization, we’re excited because the same treaters that we’ve been calling on, treating, and finding KMT2A-rearranged patients are the same treaters that will find NPM1-mutated patients. We’ve got a leg up. We take competitive immunity seriously and first mover advantage.

Having the ability to share Revuforj and the opportunity for current treaters, by the time we hit our PDUFA date, and if in fact there is another menin in the market, you’ll have well over 1,000 patients that have been treated on Revuforj. That’s meaningful. To gain some muscle memory with a treatment center is important. It’s not just physicians. It’s their staff. It’s the nursing staff. It’s pathology. It’s formulary. It’s how do they access the drug. That’s a lot of experience. Yigal offered it takes not a lot for physicians to gain experience and some loyalty with a drug. Typically, they’ll say it’s two to three patients. For many of the providers out there, they’ll be in that window. We’re really optimistic and excited about the launch of NPM1-mutated. The space possibly will be competitive. We’re more than ready for that.

Yigal Nochomovitz, Biotech Analyst, Citi: For those a little bit less familiar, can you just summarize the pivotal data that supports NPM1?

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, I would be happy to. These are data from our Augment 101 study in NPM1. This was a series of patients with a primary endpoint of CR/CRh. We reported 26% of that in the enlarged phase II cohort of around 77 patients. I think perhaps even more important than CR/CRh is when you look at the overall response rate. That’s really the endpoint physicians are most interested in, because if you can get these patients into response, it gives them the highest probability of then potentially going on to get a stem cell transplant. We reported 48% overall response rate in these patients. That’s really quite unprecedented for nearly half of the patients to have a response. We’re really very excited about that. Importantly, the durability of that is also key.

We did an exploratory analysis looking at overall survival in those patients that respond, in the nearly half of patients that respond, and found the median overall survival to be 23 months, so nearly two years, which again, in a setting where traditionally the expectations of survival for patients with relapsed refractory AML is of the order of two to three months, this is an enormous change in the potential standard of care. That was also coupled, again, I focus on efficacy because these are very unwell patients where you really want to focus on efficacy. I think that’s also coupled with a very predictable and well-established tolerability profile. Very few patients had to have any form of dose reduction or dose interruption as a result of toxicity. It’s a very manageable safety profile. Physicians are now familiar with it from our experience with KMT2A. It’s a compelling profile.

We believe it’s a best-in-class profile. We’re looking forward to the upcoming PDUFA.

Yigal Nochomovitz, Biotech Analyst, Citi: October 25th. Steve, the ramp for NPM1, how do you see that? You already have the drug in the market, and people are already using it. They’re probably either maybe using it a little bit off-label or very much wanting to use it. Is the ramp going to be the same, or is it going to be steeper for NPM1? You do have a competitor coming possibly a month later, so there’s different pushes and pulls there.

Keith Goldan, CFO, Syndax Pharmaceuticals: There are. I think the market is primed. I mean, they know about the compound. There is, as mentioned, some off-label use. Obviously, we don’t promote there. About 10% of the use is outside of the approved indication. There’s some experience. We haven’t commented on ramp. The drug is available. It’s there. The education is high. The awareness is high. The NPM1 data was published in Blood Peer Review Journal back in May. The interest level will be there. I would expect some pretty rapid uptake. I think as it relates to a competitive space, I may reiterate some of the things Nick has said. This is a market that’s really built on efficacy. Physicians want to use the drug that’s going to work the best. These patients are very much at risk. The second thing I point out is physicians like to use drugs that have multiple indications.

We’ll be the only menin on market, assuming the next one makes it, that has two indications. It’s broader. It’ll treat up to roughly 50% of the population. It’s going to be adults and pediatrics, AML and ALL, and all of that. It’s very attractive. The third point I make is just the experience that they have with us, drug and channel, positive experiences with Revuforj. That’s going to be, I think, challenging for a competitor to overcome that. We remain optimistic on the launch.

Yigal Nochomovitz, Biotech Analyst, Citi: Of course, those two relapsed refractory markets, the KMT2A and the NPM1, that’s adding it up, it’s 6,500 or so incident. That’s just a start. I’d love to hear more about the earlier studies. There are two. There’s the Beat AML, the Save AML that are going to try to advance into earlier lines of therapy. I’d love to understand the market for that, as well as the design of the studies and what you’re going to show. I believe there’ll be more data coming at ASH on both of those.

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, I think from a market perspective, it really opens up the opportunity. We’ve often talked about the relapse refractory market as just over $2 billion in potential between the two indications. Overall, when you include frontline, it’s really a $5 billion opportunity. It’s roughly about 9,000 patients that would be affected frontline for KMT2A, as well as NPM1. Duration of treatment is going to be on the longer side. Patients are just earlier in their disease therapy. At the current price point, that’s how you get a TAM that’s as large as that.

Yigal Nochomovitz, Biotech Analyst, Citi: Maybe Nick, you want to comment a little on the studies?

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, I would be happy to. We’re excited to be leading in this space. As leaders in the class, we were the first to have a patient enroll in the frontline setting in a phase III study. This is a study that we have ongoing in collaboration with the Hovon Group. This is a well-established collaborative group that we’re working with for the combination with venetoclax and azacitidine. This is for patients who are unfit for intensive chemotherapy. The premise for this study was really based on the Beat AML data that was presented at EHA this year that really showed compelling activity when you combine Revuforj with venetoclax and azacitidine in terms of the CR rate, but also importantly, the MRD negativity rate.

We reported 100% of the patients, so 37 out of 37 patients had MRD negativity and a really compelling efficacy profile combined with a very tolerable combination regimen at the standard approved dose of 163 mg. Those data are very supportive of the phase III study we have ongoing in collaboration with Hovon. We will be updating those data in due course. It’s a Beat AML sponsored study. The study is continuing to enroll, so there will be more patients in that study in due course. We will update some of those efficacy endpoints. It was a compelling profile. In addition to that, we have two planned studies combined with intensive chemotherapy. We’re thinking about this such that we have one study focused on patients that have KMT2A-rearranged AML and one for NPM1-mutated AML.

We will be presenting later this year phase Ib data that establishes a tolerable dose in combination with intensive chemotherapy. That study is progressing well and also preliminary efficacy that we’re anticipating will be very supportive of those two phase III settings we have with intensive chemotherapy in the frontline setting. Those are, I believe, very smartly designed studies. We’re really driving some innovation in this space as leaders in the menin inhibition field. We’ll reveal more about the designs of those studies later this year. I think they are very well designed. They have the potential for accelerated approval using early surrogate endpoints. We think that’s exciting because that gives us the opportunity to bring combination therapy to patients earlier in the frontline or newly diagnosed setting. We’re hoping to have those studies enrolling. They’ll enroll competitively based on the data we presented.

We think we can continue to lead in that space. It’s a very exciting part of our lifecycle management plans. It’s a priority for us.

Yigal Nochomovitz, Biotech Analyst, Citi: Would that be something on the lines of like a minimal residual disease, MRD negativity type surrogate?

Keith Goldan, CFO, Syndax Pharmaceuticals: It would. In the Venaza unfit, complete response is a well-established surrogate for these patients. It’s been used before for regulatory approvals and is in guidelines. We’ve built complete response rate in as a dual primary endpoint in collaboration with the Hovong study. That would certainly serve as a potential surrogate to support accelerated approval in combination with intensive chemotherapy, the so-called 7 plus 3 regimen for NPM1 patients. We’re actually looking at MRD negative CR. We think that could be a more sensitive and more accurate predictor for correlating with event-free survival and OS subsequently. Our dual primary endpoint there is both based on MRD negative CR. We think that if we can show a sizable difference over the current benchmarks, which trend around 40% if you look at MRD negative CR, clearly a high unmet need remains in those patients even getting treated with intensive chemotherapy.

If we can add to that with the addition of Revuforj, we believe that could also serve as a potential for accelerated approval in due course. That’s something that we have built into the phase III.

Yigal Nochomovitz, Biotech Analyst, Citi: How are things shaping up competitively? There are obviously other companies that are pushing in frontline as well in combination with some of the similar regimens you described. How is it shaking out as far as getting to market in frontline if you have estimates for that?

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, we feel very confident. We have a well-established profile and a well-established support and advocacy for the programs we presented. I think compelling data from Beat AML. As I say, we’ll be presenting more data in the latter part of this year that will support those phase III programs. The designs have been supported by the best academic thought leaders working really closely with leading sites and centers. We think they’re well-designed studies, and we think that working with our partners across the spectrum, those studies will be very competitive and recruit well. Our expectation is to continue to lead in this space.

Yigal Nochomovitz, Biotech Analyst, Citi: OK, maybe we could switch gears a little bit and talk about cGVHD. Of course, there you’re partnered with Incyte, which I’m sure is helping. Just can you kind of characterize the launch? It’s been strong from what I gather. Tell us about the dynamics. I mean, that’s in the third line. Similarly, thematically to AML, you’re also starting to move into the earlier lines. It’s kind of a different drug, but same concept.

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, we had our first full quarter. We do, as Yigal mentioned, we partner with Incyte. Their release in earnings, I think, predated ours by roughly a week. Very strong quarter, almost $50 million in sales between the first partial quarter and the first full quarter. The business is incredibly healthy. I think it speaks to a market that was ready for another drug. Patients are highly symptomatic. Ictimo has a different mechanism than either of the two drugs that are currently on the market. The launch in third line, chronic GVHD has been very strong. We’ve announced about 700 patients have been put on drug from the launch through June. It’s about 4,000 infusions. About 80% to 90% of patients are staying on drug. It’s too early. There is a big component to the commercial story here on duration of treatment, which is often measured in years, not months.

Early to say on that. The fact that so many patients are staying on treatment, moving from one treatment to the next, I think speaks to the tolerability of the drug. In terms of the prescribing audience, about 80% of all transplant centers have used Ictimo. A majority have used it more than once. In terms of payers, and this is a different type of drug than Revuforj. This is a Part B to buy and bill. It’s not necessarily formulary coverage. There is a medical benefit that pays for it. North of 80% of payers already have it in a position to be covered. Really good start. I think more to do. We’re grateful we’re partnered with Incyte. They somewhat created the space with Jakafi.

We do co-promote with them hand to hand in offices, a very efficient call point for us and for them because we’ve got other products in the bag. We’re able to really draft off of their infrastructure. It’s been a great launch so far.

Yigal Nochomovitz, Biotech Analyst, Citi: In the early days, some people were concerned because obviously, you’re an infusion, although you are working on a subcutaneous formulation. We can talk about that. You weren’t oral, and there were several oral options ahead of you. That didn’t seem, at least what we’ve seen in the early innings, doesn’t seem to be a limitation.

Keith Goldan, CFO, Syndax Pharmaceuticals: It’s certainly not. I mean, these patients are highly cared for. They’re complex. Their disease is serious. They’re often seen at a transplant or associated wherever they may be receiving care. It’s certainly not a hindrance. They’re in the offices typically once a month anyway for some type of evaluation. This is every other week. Not an issue at all for patients starting and so far staying on treatment.

Yigal Nochomovitz, Biotech Analyst, Citi: I mentioned the subcutaneous formulation. What is the status of that? There is a plan to produce one that’s being worked on?

Keith Goldan, CFO, Syndax Pharmaceuticals: There is. Yeah, there is as an outline plan. I think there are a number of potential catalysts in the lifecycle management of Ictimo. I mean, development of subcutaneous formulation is one option that provides some benefit to patients to be able to provide the drug subcutaneous as opposed to IV. That is in development. The other catalyst, I would say, and you alluded to this, is the move into earlier lines of therapy. Incyte have two studies that they’re actually sponsoring that we’re partnering on. One is in combination with dexamethasone against dexamethasone. That actually provides quite a compelling rationale just because of the mechanism of action of CSF1R. It’s a very different type of mechanism than steroids. It impacts the monocyte macrophage lineage as opposed to more T cells. There’s a good rationale why you might want to combine with dexamethasone.

Also, a potential steroid-sparing regimen in combination with Jakafi against Jakafi and against steroids. That’s a three-arm phase II. The study with dexamethasone is actually a potential regulatory study. It’s a randomized double-blind placebo-controlled study, well-designed. Those are all important catalysts, I think, in the lifecycle management of Ictimo. We do also have a proof-of-concept study ongoing, which we are sponsoring again in partnership with Incyte in idiopathic pulmonary fibrosis. That’s a very important study for us. It’s a randomized phase II study with a relevant endpoint that would support the design of a phase III study in terms of SFVC. We’re anticipating that study will be fully enrolled by the end of the year, and with data in the second half of next year, then that could really inform a phase III program in idiopathic pulmonary fibrosis.

Given everything that we’ve seen and the fact that actually Ictimo was originally conceived to be a drug that would have activity in idiopathic pulmonary fibrosis based on preclinical models and based on the clinical data we observed in patients with pulmonary symptoms of cGVHD, a syndrome called bronchiolitis obliterans syndrome, we saw compelling improvements in response rate and also symptoms. Also intriguingly, the impact that CSF1R has on inflammatory cytokines like TGF-beta and IL-4. All of those things, I think, predict very well for a positive outcome in that proof-of-concept study. We are hoping for a really kind of compelling clinical benefit that would catapult us into a phase III development program. More on that one to come.

Yigal Nochomovitz, Biotech Analyst, Citi: Was that a controlled study, that one?

Keith Goldan, CFO, Syndax Pharmaceuticals: Yes, it’s a randomized controlled study. It’s about 135 patients randomized two to one, with an FVC primary endpoint.

Yigal Nochomovitz, Biotech Analyst, Citi: What is the control?

Keith Goldan, CFO, Syndax Pharmaceuticals: It’s standard of care.

Yigal Nochomovitz, Biotech Analyst, Citi: It’s a combo of Ictimo plus?

Keith Goldan, CFO, Syndax Pharmaceuticals: It’s Ictimo against standard of care.

Yigal Nochomovitz, Biotech Analyst, Citi: Oh, it’s just axitinib. It’s not Ictimo. OK. Ictimo is in cGVHD.

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, OK.

Yigal Nochomovitz, Biotech Analyst, Citi: Just to clarify. The study with Ictimo, I wasn’t aware that there’s a third arm that’s just steroids. I wasn’t aware. That’s interesting. What is the plan for when that’s going to read out? That’s up to Incyte?

Keith Goldan, CFO, Syndax Pharmaceuticals: Incyte’s sponsoring that study. I don’t think we’ve guided on the timelines for it. It’s enrolling now, and the readout, we haven’t guided on timelines for it yet.

Yigal Nochomovitz, Biotech Analyst, Citi: That’s all coming. Which of those do you consider to be more significant in terms of the opportunity, the steroid combo with axitinib or the Jakafi combo? Are they just?

Keith Goldan, CFO, Syndax Pharmaceuticals: The steroid combination is a powered phase III randomized controlled double-blind study. The combination with Jakafi is a phase II study. It’s powered differently, and they have different endpoints. One’s focused on response rate, the other one is focused on event-free survival.

Yigal Nochomovitz, Biotech Analyst, Citi: In this practice of cGVHD, which is typically first line? Is it steroids or Jakafi?

Keith Goldan, CFO, Syndax Pharmaceuticals: It’s usually steroids.

Yigal Nochomovitz, Biotech Analyst, Citi: OK. All right. You would open the door to that immediately. OK. Assuming the Jackify combo looked good, they would take that forward as well. OK. Keith, since we have the CFO here, can you just comment briefly as well in terms of the P&L dynamics, how you’re thinking about moving towards a profitable enterprise, what that looks like, and what the OPEX looks like? Obviously, we’re talking about going into some larger studies, earlier line studies. That’ll impact the spend.

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, sure. Happy to. We gave a couple of pieces of guidance during the last call, which maybe I’ll repeat now. Since we’re just talking about Ictimo, maybe I’ll just comment on Ictimo. As has been discussed, we do have a 50/50 profit split with Incyte. We co-promote the drug with them, overlapping call point, which is really efficient and great for us, both for Incyte as well as Syndax. We gave guidance on this last call that the margin that we report on our P&L would be 25% to 30% in the near term of what Incyte reports on their P&L in terms of Ictimo net sales. Ictimo, our partner, reports net sales. We present a line on our P&L called collaboration revenue.

That collaboration revenue, to give you a quick example, if they reported $100 million in a quarter of Ictimo net revenue, one should expect our P&L to reflect about $25 million to $30 million of collaboration revenue. That’s important because in the first full quarter of Ictimo’s launch, which was just this past quarter, the product is already profitable on a contribution profit perspective. We reported over $9 million in collaboration revenue, which I think surprised a lot of folks that it became profitable so quickly. I think it just speaks to the unmet need and the impact the product is having on patients. We raised $350 million through a royalty monetization back in November with Royalty Pharma. At that time, we came out on our third quarter call and made the statement that we believed we had cash-to-profitability. We wouldn’t need to finance the company again.

I think that statement wasn’t really appreciated by the street. We went a step further on this last call and actually gave forward-looking OPEX guidance for the next two to three years, stating that we expect to keep our operating expenses flat to 2025 levels. We thought it was important to do that, Yigal, because we wanted to give the street a reason to believe our statement that we do not need to finance this company, that we have enough cash to get to profitability with an adequate cash cushion underneath that. I think based on the reaction, that’s beginning to become appreciated. The one question we’ve been getting often is, how are you going to do that? You have all these frontline studies that you’re funding. Aren’t they expensive? There are puts and takes.

In the last couple of years, we had a couple, I’ll say, one-off expenses that were not insignificant. We filed an NDA, an sNDA, as well as a BLA. Those endeavors are extremely expensive, not just regulatory costs, medical writing, QA, QC, IT, you name it. It puts a big burden on the organization. Those are behind us, as well as launching two products, coming with a big bolus of spend to successfully commercialize those products. Those costs are going away, going to be replaced with additional clinical costs. At the end of the day, we’re going to be able to keep our expenses flat over the next three years.

Yigal Nochomovitz, Biotech Analyst, Citi: OK. Now, I remember last year you got the approval for KMT2A. Didn’t it come a little bit ahead of schedule, if I recall?

Keith Goldan, CFO, Syndax Pharmaceuticals: It did. It did.

Yigal Nochomovitz, Biotech Analyst, Citi: Should we expect something similar for NPM1 or not necessarily?

Keith Goldan, CFO, Syndax Pharmaceuticals: It is a priority review. It’s under our tour. Dialogue with the agency is going well. We don’t comment necessarily on any specific interactions with the agency.

Yigal Nochomovitz, Biotech Analyst, Citi: The conduct with the FDA is going well, given we’ve seen a lot of changes with the FDA.

Keith Goldan, CFO, Syndax Pharmaceuticals: Yeah, the process is working very smoothly. Again, not specifics on the commentary of our regulatory interactions. It is a team we know very well. This is an sNDA for us, which is, of course, much simpler. Many components or modules that you have to provide for an NDA we’ve already covered for KMT2A, such as CMC, et cetera. Yes, it’s progressing very well. We’re very confident in the process and moving very well towards the planned PDUFA date.

Yigal Nochomovitz, Biotech Analyst, Citi: You have the two approved products, which is great. Not many companies can claim two FDA approvals in a six-month period. Are you thinking about anything beyond this in terms of BD, or are you focused now on these two?

Keith Goldan, CFO, Syndax Pharmaceuticals: I would say that from a capital allocation perspective, there’s three primary goals. The first goal is to continue to successfully commercialize both products. I think the results so far speak for themselves, but we need to continue to focus and execute well there. I think secondly, it’s allocating capital to maintain our leadership position in the menin inhibition space. That’s investing in the clinical development of moving these products earlier in lines of therapy so they can serve as many patients as possible. Third is getting to profitability. With those capital allocation goals in mind, I don’t think that necessarily investing heavily in other products right now is our focus. We’re going to get to profitability very soon. When we do, I think we’ve shown a core competency in being able to in-license products, proof-of-concept products, targeted therapies, and quickly develop them and get them to market.

I don’t think we want to go back to our roots and continue to do that, but not until we get to profitability.

Yigal Nochomovitz, Biotech Analyst, Citi: OK, excellent. All right. Thank you all very much. Great discussion. We look forward to good news in a few months.

Keith Goldan, CFO, Syndax Pharmaceuticals: Thanks, Yigal.

Yigal Nochomovitz, Biotech Analyst, Citi: Let’s go. All right.

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