Syndax Pharmaceuticals at Goldman Sachs Conference: Strategic Growth and Profitability

On Wednesday, 11 June 2025, Syndax Pharmaceuticals (NASDAQ:SNDX) presented at the Goldman Sachs 46th Annual Global Healthcare Conference, outlining a strategic vision marked by early successes and future growth opportunities. The company highlighted its robust product portfolio, financial stability, and plans for expansion, while acknowledging competitive challenges in the market.

Key Takeaways

• Syndax’s RevuForge and Niktombo drugs show early success in their respective markets, with strong sales and market penetration.
• The company is focused on expanding its indications and maintaining financial stability through profitability.
• Syndax plans to capitalize on its existing products while considering future asset acquisitions.
• The company expects to achieve profitability in the coming years, driven by its current product lineup.

Financial Results

• RevuForge:

- Fourth quarter sales reached $7.7 million, with one-third attributed to stocking.

- First quarter sales surged to $20 million.

- The drug targets KMT2A translocations in acute leukemia, with significant formulary coverage and a growing prescriber base.

• Niktombo:

- Stub quarter sales amounted to $13.6 million.

- The drug, targeting fibrosis and inflammation in chronic GVHD, has a high gross margin.

- Syndax is financially positioned to reach profitability within the next few years.

Operational Updates

• RevuForge:

- Approved for KMT2A translocations, affecting about 10% of AML and ALL cases.

- Clinical trials showed a two-thirds response rate, with many patients proceeding to stem cell transplants.

- The drug is gaining traction among tier 1 and tier 2 institutions, with over 90% formulary coverage.

• Niktombo:

- Launched in January following its approval in August.

- Demonstrates early and durable responses in GVHD patients, with a rapid activation of the transplant center user base.

Future Outlook

• Syndax aims to expand into frontline AML settings with combination trials for unfit populations and phase 3 trials for fit populations.
• The total addressable market for KMT2A and NPM1 is estimated at $2 billion, with a price point of $40,000 per month for approximately 5,000 patients.
• The company anticipates that the combination of RevuForge and Niktombo will drive growth and profitability.

Q&A Highlights

• Syndax’s management expressed confidence in their competitive positioning, noting superior data in the NPM1 pivotal trial compared to competitors.
• They addressed concerns about QTc prolongation, stating it is manageable for physicians.
• The company expects its drugs to be included in NCCN guidelines upon approval.
• While the focus remains on existing products, new acquisitions may be considered to enhance value.

In conclusion, Syndax Pharmaceuticals is poised for growth and profitability, driven by its innovative product offerings and strategic market positioning. For more details, please refer to the full transcript below.

Full transcript - Goldman Sachs 46th Annual Global Healthcare Conference:

Corinne, Goldman Sachs: Good afternoon, everyone. Thanks for joining us here at Goldman Sachs Senior Healthcare Conference. Thrilled to be joined by the team from Maybe I’ll kick it off. I’ll let you guys introduce yourselves and then introduce the company.

Michael Metzger, CEO, Syndax: Sure. Good to see you, Corinne. Thank you. I’m Michael Metzger. I’m the CEO of Syndax, I’ve been with the company about ten years.

And to my left, Steve? Yes. Steve Kloster, our

Steve Kloster, Chief Commercial Officer, Syndax: Chief Commercial Officer at Syndax. Corinne, thanks for having us here. Looking forward to the discussion.

Corinne, Goldman Sachs: All right. So let’s do an overview of the business.

Michael Metzger, CEO, Syndax: Look, I think exciting time for the company. I couldn’t be more excited to be here and to tell you about it. We have two approved products addressing very large markets, mover advantage, capital to execute. So we’re funded through profitability. And profiling both of our drugs is sort of best in class.

So we feel fantastic about our position as we go into their early this is early launch time. So we started with approvals last year and launching the drug late last year with Revuforge, which is our drug for acute AML and ALL and subtypes of those diseases. And then with Nikimbo, which is for chronic GVHD in line plus patients. And so both of those drugs are now launched. We’re in the early phases of that.

And things are proceeding very, very well. We’ve had some early sales data, which we put out, and we expect tremendous growth from here. Great.

Corinne, Goldman Sachs: I think we’ll start with RevuMinav. RevuForge is the brand name, obviously. It was recently approved, as you said, in a subset of acute leukemia, KMT2A translocations, adult pediatrics. Maybe you could just like talk to us about the population and the opportunity there and also help us understand like the clinical data that led to that approval.

Michael Metzger, CEO, Syndax: Sure. So this is an area of very high unmet medical need where patients have a very poor prognosis. KMT2A acute leukemia affects about ten percent of AML and ALL. So it’s a significant portion of an identifiable portion. Patients are tested for KMT2A because they have a poor prognosis, the standard of care treatment that really help them.

So, we are the with a menin inhibitor to be approved. We have really significant profile across the board. The data speaks for itself. I think physicians are excited about the fact that two thirds of the patients in our trial got to a response. What that means is they wiped their tumor, really cleared their tumor, and that allows for these patients in our trial at least 35 years of age.

So younger patient population affects a lot of pediatric patients. We do have, as you mentioned, an indication for adults pediatrics, AML and ALL. So it’s an agnostic across the board indication one of its kind. So, we really cover all of the different populations, which is really, really important. Going back to the data, two thirds of the patients get to a response.

Many of those patients go on to get a stem cell transplant, which is the goal of treatment for these patients. And the ability to actually get a transplant and then go back on a maintenance treatment in a relapsed refractory setting is exactly what physicians want to do with this drug. And it is exactly what they’re starting to do in the commercial setting. We saw it in the pivotal trial. And to be able to transplant twenty five percent to fifty percent, maybe more percent of the patients that we see, that is a game changer for these patients.

So we are well on our way to achieving that, and it’s a really exciting time.

Corinne, Goldman Sachs: Yes. At the early stages of the launch, you, I think, had 7,700,000.0 in the fourth quarter. That was only a couple of weeks, 20,000,000 in the first quarter. Maybe you could characterize the demographics driving the early launch with respect to the prescribing population and like the patients that are coming into therapy?

Steve Kloster, Chief Commercial Officer, Syndax: Yes, absolutely. I think any good launch starts with great execution. That starts with the people. So we hired a great team. Our customer facing team is some of the best in the industry, a lot of experience at launches working in biotechs like this.

They know their customers on the HCP side. Payer side, no different. We’ve been in market for one point years talking to payers and building up a great trade team. Think all that’s paid off. So when we think about fundamentals of a launch, you need an active and growing user base.

We have that. We’ve given some markers over time. We have these Tier one and Tier two institutions, a couple of 100 of the largest academic centers in the country. They represent about two thirds of the opportunity. We’ve got more than 50% of that group prescribing.

There’s also prescribers at smaller academic centers as well as community oncology. So that’s great. On the payer side, we were getting claims paid very early on even before formulary coverage was being established. That’s obviously advanced. Our formulary coverage now is well north of 90%, which is pretty good six, seven months into launch.

And the last piece is a very effective trade team and some great specialty pharmacy partners. These patients are very sick, as Michael said, days matter. So our goal and what we’ve been able to achieve is patients on drug on average less than five days. It all leads to better treatment progression. And when you look at those markers, those metrics targeted AML therapy that we can look at that has numbers as good as that.

And I say that because off to a good start, we expect very good things in the near term, midterm and even the long term that we’ve got to make sure we’re executing.

Corinne, Goldman Sachs: I think on a like week adjusted per week basis, sales were somewhat flattish, a little up in the first quarter, but I imagine there were some dynamics going on in early launch. Can you tell us about anything inventory, bolus, patients transferring over from clinical studies? Like what drove some of those early launch dynamics?

Steve Kloster, Chief Commercial Officer, Syndax: Sure. In terms of the growth, we believe there is growth from quarter to quarter. I think some of that explanation is simply in inventory. So that first quarter of 7.7%, fully onethree of it was stocking. So that stocks the channel.

We’re not going to give exact guidance on inventory changes by quarter, but you can expect it to be in the two to three week range. So the inventory component of Q2 was very small. I think how you have to think about the market, and Michael laid this out really within the clinical data. There are roughly three different pathways of patients. You need to understand each one to see how that evolves and then how does that ultimately contribute to sales.

The is the goal of treatment, right? These are very sick patients. Best chance for them for a cure if they’re fit is to get the transplant so that patient will be on treatment for some period of time, go to transplant. There’s roughly a ninety day window on average. We’ll call that a drug holiday where patients are off treatment.

Mhmm. So the engraftment can take place, and then they’re back on treatment. Right? And we believe that’s a patient that will be on drug for a very long period of time.

Corinne, Goldman Sachs: Mhmm.

Steve Kloster, Chief Commercial Officer, Syndax: That set of patients are ones that may not be fit. They will get some benefit from the drug. They will be on for some period of time. And the last piece of the patients that may not respond, and that’s always going happen. When we think about the dynamics of a launch like this, there are patients in market.

And when a new drug comes out, there’s a lot of excitement. It lasts maybe the six, eight, ten weeks or so where a lot of different patients are put on drug. So you’ve got patients at relapse, which is where the drug is indicated for. That’s where physicians tell us they want to use it. And then you’ve got patients all the way out to a very layer line, which are like the ones in the trials, we’ll call these hospice patients.

So it’s a mix. Over time, it’s going to move earlier. So better quality patient, better chance for them really being going to transplant, ultimately maintenance. And that’s how it’s evolving.

Corinne, Goldman Sachs: So you think about what’s going to drive revenue here, is it going to be about like to what extent is duration versus new patient growth drive sort of like the opportunity?

Steve Kloster, Chief Commercial Officer, Syndax: Great question, and it’s both. You can’t have one without the other. We’ve shown that we can find KNT2A or relapsedrefractory patients. We’ll expect by end of the year, we’ll be above 50% of penetration in that market. That would be a great number.

Each month, there are more patients coming in for treatment, which is great. So that’s one component. That will continue to grow. The piece is the duration piece. So I think about it this way.

In part, where we are in the year, that quality of patient has mentioned, it will go earlier, so you’ll have more success in that patient. But we would expect duration of treatment roughly in the four to six month range in this year, and that’s a blended rate of all patients. And that will move out over time as more patients go to transplant maintenance, it will move closer to nine.

Corinne, Goldman Sachs: Four to six months this year, like, over time, what did you see in the trial? Or, like, how are you thinking about what should guide us our expert guide our expectations for duration over time?

Steve Kloster, Chief Commercial Officer, Syndax: The trial’s really suggested nine overall, and that that is, again, that mix of patients. Transplant maintenance, those having some effect, but may not go to transplant and those that that didn’t have the the benefit from a drug. Okay.

Michael Metzger, CEO, Syndax: And I think it’s just to add on here. I think the efficacy profile of the drug really suggests that they’re going to use this as early as possible in treating KMT two a and and also with m p one. And I think that will drive utilization longer. More patients will get the transplant if you treat them earlier. More patients will go back on therapy post transplant.

Mhmm. I think this really changes the dynamic with a with a great efficacy profile where you really distinguish yourself from others. You can get patients on. You can keep them on. That’s that’s really the name again.

Corinne, Goldman Sachs: In that maintenance setting, the post transplant, they’ve come back on. What triggers, like, people staying on therapy versus coming off therapy? Is there a stopping mechanism?

Michael Metzger, CEO, Syndax: No. I mean, it’s it’s it’s up to the individual treating physician and the Mhmm. Physician. I mean, I mean, patient. I mean, this is a this is a drug that’s really well tolerated.

Mhmm. And physicians typically look at it. This is the drug that got the patient to a response and cleared their tumor. Mhmm. In difficult diseases, that’s really critical, and they wanna keep that response in check.

Mhmm. So they look to the put the patient back on that therapy post in a post maintenance or in a maintenance setting in order to maintain that response. And so physicians are with a drug that is well tolerated. There’s really no reason not to do There’s there’s only upside. And so that’s how they think about it.

Corinne, Goldman Sachs: Is there additional data that they would like to see or that you would like to generate that would help physicians understand how to use this drug in a maintenance setting?

Steve Kloster, Chief Commercial Officer, Syndax: Well Yeah. They as Michael said, they they want to do this, and this is what they’ve done with other agents. We we were just at ASCO. It’s a great opportunity to meet with customers. We conducted an advisory board.

We had 10, mostly hemes, some transplanters, top institutions around the country. And this is what they want to do. So in terms of is there more data, we’ll generate more data over time. Some of it’s going to be real world data. It doesn’t take we talk about competitive immunity and where do you create the muscle memory to use the drug.

Tends to be about two to three patients. And we’re getting there amongst many of our bigger creatures. They’ll gain experience, and we’ll provide it with supplemental data over time.

Michael Metzger, CEO, Syndax: Yeah. There will be a lot of data that will be continue to roll out over time as we we look at you know, we’ve also published some data already Mhmm. In the maintenance setting, which has been helpful to physicians to think about. Understood.

Corinne, Goldman Sachs: Maybe we should talk about NBM one AML. You’ve reported the registrational data here. The supplementary NDA is currently in review. Maybe, like, talk to us about how this gets to market because it’s not necessarily strictly about the regulatory process. So talk to us about how you get this to patients?

Michael Metzger, CEO, Syndax: Right. So key is getting it to patients, and we are excited obviously. We’re the only MEND inhibitor approved. We have KMT2A, and that affords us the opportunity to publish the data and then submit it to guidelines, which we’ve done. And so our expectation is that we’ll have guideline coverage very soon, hopefully, and we expect that before approval.

The drug has been, as noted, submitted as an sNDA for this for the MPM1 indication, and that will be we’ll have our PDUFA date soon. So we expect under priority review to be able to get that drug approved in October. Okay. So the steps here, getting into guidelines, getting the drug approved Mhmm. This year, both should happen, and that is the most expedited way to get the the product Yeah.

To patients. So that’s the that’s the

Corinne, Goldman Sachs: So you’ve got, obviously, the registrational data. There’s a competitor that shared registrational data as well. Could you review the data that you’ve shared with a focus on what some of like the key metrics are and how this compare to the others?

Michael Metzger, CEO, Syndax: Look, we’re thrilled we came out of ASCO. We just finally saw some competitor data in their NPM1 pivotal trial. I think it’s very clear that our FNP profile is the best there is, best in class on all measures. And I would say a couple of things stand out. of all, overall response rate, which is the measure of clearing your tumor, this is what physicians want to see.

They want to see the tumor go away. So in about forty eight percent of our patients, that is that was the that was the case. That compares to our competitors about a third of the patients. So a very different overall response rate. And then you think about, CRC or H, which is a regulatory endpoint, were twenty six percent.

They were twenty three percent. Doesn’t seem numerically different, so numerically different, but it is superior for sure. And I would say, overall, survival is a big measure of this as well. In our patient population, we are at twenty three months, not only in the patients who reach the CR CRH, but the overall response rate. So in the forty eight percent that I mentioned before, that overall survival median overall survival is out to twenty three months compared to our competitor at sixteen months, about a fifty percent difference.

So we’re talking about the most efficacious drug. This is an efficacy driven market. Physicians are most concerned with that. If you have a loved one, you want to get rid of the tumor Drug is very well tolerated across the board, ours is. I would say our competitor showed some additional side effects such as pruritus, which is pretty significant.

That’s itching. When you remove the drug, doesn’t go away. So this is a potentially an ongoing side effect for them to have to deal with. So our profile in terms of safety, tolerability, risk benefit, and the best efficacy, which is what physicians are really driven by, that’s what stands out for us.

Corinne, Goldman Sachs: Obviously, whenever we’re comparing trials, there’s significant caveats. Anything in particular you’d highlight from a baseline patient characteristics perspective or, like the way the trial was executed, where it was executed, anything that we should be keeping in mind as we think about comparing these two?

Michael Metzger, CEO, Syndax: No. I would say just in our patient population, the vast majority of patients, about three quarters of the patients were treated with prior venetoclax, which is a very difficult regimen to treat after. Showed excellent efficacy in the patients that were treated in the trial. That you often sort of look at that as a But overall, I would say this is an international trial. So we did enroll patients all over the world, a lot of them in The U.

S, a lot of in Europe and so So it’s a what we say is a representative patient population, line.

Corinne, Goldman Sachs: Okay. One of the things that we’ve heard spoken about is the QTc prolongation. Maybe you could address that. Just like how I’ll just lob it to you with that. Yes.

Michael Metzger, CEO, Syndax: Nonissue. Okay. QT is a side effect that a lot of drugs in AML encounter, including our competitor

Corinne, Goldman Sachs: who had

Michael Metzger, CEO, Syndax: QT, Grade three QT. So it’s more of a class effect, I would say, of these of these drugs. In our case, easily identifiable, honorable. It goes away. We if you get to a certain level, grade three, we dose adjust.

Patient doesn’t come off drug. They can take you. Very easy for the physician. You give an EKG to assess this. They’re not walking around with QT on an extended basis.

So nonissue, really easily managed for for physicians. And

Corinne, Goldman Sachs: What about monitoring? What are the monitoring requirements?

Michael Metzger, CEO, Syndax: So by label, it’s once a week for the month and then monthly thereafter. Mhmm. So there’s it’s pretty liberal based on what the physician feels is the right thing. These are patients who are in the office. They’re getting EKGs.

They’re getting EKGs for any number of drugs that they may be taking. So this is standard practice, not an onerous practice at all for physicians. Then everybody knows how to do it.

Corinne, Goldman Sachs: Okay. On the October timeline for approval, but guidelines could come anytime. Do we have any sense when the guideline changes will happen? And how quickly do new guidelines get implemented in AML practices? Yeah.

Steve Kloster, Chief Commercial Officer, Syndax: I mean, I think one analog is is simply the, you know, our RevuForge initial indication as well as Nictimbo for chronic GVHD. And those were off cycle and in the guidelines. Nictimbo, I believe, was two weeks. RevuForge was under four weeks. So, it can happen at at any time.

For these particular guidelines, the management came out in May. We immediately submitted to the NCCN guidelines. They did have a meeting on May 19. We’re not sure. They don’t confirm what they talked about at their meetings.

But but I would we would expect it to happen at any time. It could be this month. And and certainly, as I think Michael said earlier, before the approval comes, it will come within this window.

Corinne, Goldman Sachs: And so then once they get on guidelines, in terms of reimbursement, is that sufficient? And in terms of, like, physicians changing their practice, would you expect kind of, like, that to be the trigger for doctors to start using this?

Steve Kloster, Chief Commercial Officer, Syndax: I would say it’s another trigger. It’s another driver. It’s already being used to a smaller extent in MPM1 and other off label indications. Payers are paying claims. We believe from what we can tell they are paying even for off label claims.

That’s not uncommon. The guidelines themselves would be a driver, but it so so that will for payers and for physicians, you know, the larger, medium sized academic centers. I mean, MPM one patients relapse refractory is is as severe patient to treat as a KT two AR relapse refractory as they really have nothing. So physicians over time have used drugs because if they’ve had to, RevuForge is in terms of education on MPM one data. As an organization, you can tap into the SIUU guidelines, which are FDA guidelines on medical affairs sharing information with interested clinicians.

That’s something that you need a peer reviewed journal, which we have. Have a sizable MSL team with great relationships. So that will start, and that impact as well.

Corinne, Goldman Sachs: Okay. There’s obviously been a number of drugs approved in AML and different kind of genetic subsets. So what are the, like precedent launches in other areas of AML tell you about what to expect from a launch perspective as well as like how much does to market advantage matter?

Steve Kloster, Chief Commercial Officer, Syndax: I mean, did mention some of the earlier, penetration to the marketplace, formulary covers getting patients on drug quickly. Those were we’re all ahead of. The obvious questions, you get a new patient and duration. So we’ve kind of already covered that. We feel like we’re in a really good place.

In terms of mover advantage, this isn’t a concept, obviously, we’ve made up. That’s what the marketplace dictates. I did mention a couple of questions ago how long does it take for a physician to gain experience with the drug. And the muscle memory they create, it’s for the physician, it’s the nursing staff, it’s the path lab, it’s formulary, it’s all those aspects of any institution that have to get educated on the drug. So there are hurdles to utilization.

We’ve gone over, you know, many of them with, you know, a large part of our customer base. That will continue. It takes two to three patients to to really gain some experience.

Corinne, Goldman Sachs: Sure.

Steve Kloster, Chief Commercial Officer, Syndax: And that’s very long lasting. So question is when another similar drug comes out, would I use it? I think as Michael laid out, we’ve got a drug that will have two indications, much broader population Yeah. Adults, pediatrics, down to age one. The drug is easy to use, easy to dose.

It’s a high hurdle, you know, for and the payer the payer side as well as is it makes it challenging. So they need a reason to use any new drug. And from what we can tell based on their dataset, there’s just not a lot of reasons.

Michael Metzger, CEO, Syndax: Yeah. And I would add, if it’s the most efficacious drug, it’s even better. Right? Added to what what Steve said, I think that is a the key point. That’s what they’re gonna reach for.

Corinne, Goldman Sachs: Okay. In terms of overlap between the prescriber population that’s already using the drug in KMT 2A versus NPM1, like, you talked about tiering the patients for KMT. Heard me. So why don’t you just tell me a little bit more about like the overlap there and how that will advantage your early launch in NPM1?

Steve Kloster, Chief Commercial Officer, Syndax: Just complete overlap. It’s the same treatment centers. You may see a little more interest and willingness to prescribe for MPM1 patients in the community and smaller academic centers. They just see these patients more. And at diagnosis, there, it’s a positive prognosis and then eventually it’s not.

So they have some additional experience. What we’ve done is we size the team and focus the team on the full audience, really, at our at our initial launch in November, knowing that we were gonna eventually get an indication. So we’re we’re covering the full audience, same treaters. Nothing else, you know, really changes. So we’re already in the places we need to be for the for the future launch.

Michael Metzger, CEO, Syndax: He says we’re everywhere.

Corinne, Goldman Sachs: think you guys have said, like, described the opportunity between the two as about $2,000,000,000 per year estimates. I guess, what does that embed in terms of duration of treatment, annual cost, penetration, etcetera? How should we think about those estimates around the size of this market opportunity?

Michael Metzger, CEO, Syndax: Right. So so $2,000,000,000 is both KMT-two A and NPM1. That’s a TAM that assumes a $40,000 a month price and roughly 5,000 patients from warrant, 2,000 MT2A, 3,000 to 4,000 MPIONE. Duration of treatment, somewhere in the nine month range, as we talked about earlier. That’s that is and and the differences between the amount of patients going to transplant and then how long maintenance goes on for KMP2A versus a component of patients who are going to go to maintenance for NPM1 after their transplant.

That those are some of the variables that will drive it perhaps even beyond that number, but I think it’s a a fair estimate rated based on the the FDA we have.

Corinne, Goldman Sachs: Okay. So duration, are they the same in both populations, KMT2A?

Michael Metzger, CEO, Syndax: Duration. Roughly. Mean, we think about it as sort the time on treatment. It takes a little bit longer to get to a response for m for MPM one. Okay.

The KMT2As or the KMT2A patients respond within a you know, it’s about a cycle cycle and a half or two, and it’s about an extra month for the MPM1 patients. Okay. And then you have to measure duration of response. Duration of response in our KMT2A trial was six point four months. MPM1 trial was four point eight months.

Corinne, Goldman Sachs: So So the net of this The

Michael Metzger, CEO, Syndax: net of this is similar, very similar.

Corinne, Goldman Sachs: Right. One of the things that could expand this market from there is just a frontline opportunity. So talk to us about the development priorities as you look to go into earlier lines of therapy.

Michael Metzger, CEO, Syndax: So the high unmet need is the area that we’re starting which is which is Benaza in the unfit population. We’re doing a combination trial. We actually have data EHA that’s being presented this week in that patient population in beta AML trial. But this is an important population where the unmet medical need is high. The opportunity to add to VEN in that setting, we will be the to get to the frontline in that setting based on the fact that we started our trial.

We’ve enrolled patients. We have the most data presented to date by a long shot in that population. So we’re quite keen to move ahead there. And then the other population, of course, is the unfit population where seven plus three is used, and we’re finishing up our Phase one. We’ll take the dose that we confirm in that trial and start two Phase three trials this year or later this year to get at NPM one and KMT two a separately.

And those are, of course, the the other half of the frontline population. Together, we should have trials, three registration trials going on before the end of this year covering the broad landscape. But the priority is to get this to as many patients as possible as quickly as we can. I think we’ve been able to demonstrate the combinations or in with VEN and VENESA quite well tolerated efficacy and, we can add to the standard of care. Now we’re seeing that in our v to AML trial.

We’ve incorporated things like, complete response, as a as an accelerated approval endpoint in our protocol in order to get to that Yeah. That important point as quickly as possible and then confirm it with OS. So these are Okay. You know, the right designs in order to get to to the best case.

Corinne, Goldman Sachs: So speaking of the beta ML data at EHA, guess, what are the things we should be paying attention to in terms of those results?

Michael Metzger, CEO, Syndax: Right. Great results. We’re thrilled by what we’re seeing in that combination. I think the investigators are really excited about it. We believe it will help drive enrollment in our pivotal trial.

I would pay attention to the CR rate and the MRD rate. So CR is in our trial as of now with forty three patients and sixty seven percent. I’ll just draw your attention to VIALA A, which is the approval trial for VENESA. The CR rate was thirty seven percent. So quite a big delta between what we’re seeing and what was reported again across trial comparison.

But I think that gives us an indication. MRD in our trial was one hundred percent. In the VAIOLA A, it was twenty three percent. So very different. And those endpoints tend to correlate with survival.

So that’s why you can use CR as an accelerated approval endpoint. So that gives us a lot of confidence that we’re going to see a very nice margin of improvement in that trial. I would pay attention to those things. We do report on a very early basis overall survival. In the VIALA trial, they looked at it at twenty months, and they were at 14.7 of overall survival.

I think when you look at our seven month cut, which will, of course, will do additional cuts so the investigators will, we’re beyond what they showed in by LA. We’re fifteen and a half months. So early cut gives us a lot of confidence. We know that CR and MRD correlate with OS. So that’s the idea.

So we expect to really improve on that.

Corinne, Goldman Sachs: What does the registrational program look like or a registrational trial look like in that patient population? How many patients,

Michael Metzger, CEO, Syndax: control arm? Right. So it’s the control is VENESA. Do we do it we add to that, so it’s a triplet. K.

It’s a 410 patient trial. CR is the accelerated endpoint, and then OS is the confirmatory primary endpoint. So these are dual primaries.

Corinne, Goldman Sachs: Sure.

Michael Metzger, CEO, Syndax: We’ll enroll very quickly, we expect. Based on the data we have in hand, people are excited about this trial. So we expect to be there in the next handful of years ahead of competition, and then we’ll confirm it with those.

Corinne, Goldman Sachs: Okay. Maybe there I think you described an overall $4,000,000,000 opportunity once you start layering in these. As you think about, like, what we’re gonna unlock with the h one, can you help us understand, like, proportionality between the different populations you’re going into architectures for and what, like, are kind of the most important.

Michael Metzger, CEO, Syndax: Right. So the the highest unmet need is is what I had mentioned is the unbeaten population. These are older patients. They’re patients that don’t have the fitness to get to get chemotherapy. So they need they need treatment and they need to stay on therapy for an extended period of time.

The tolerability is also very important. It will be very important. We’ve been able to demonstrate that we don’t add any liabilities to the Veneta regimen. So we’re in a great position. It covers about half the market.

Corinne, Goldman Sachs: Mhmm.

Michael Metzger, CEO, Syndax: So that’s again, that’s a a very high priority for us. When it goes to the FIT population, we’re segmenting out KMT2A versus NPM1. KMT2A, again, very high risk. N p m one segments into high risk, medium risk, you know, and and and lower risk population. So there’s a mix of patients with different prognoses.

So we’ll be able to get at that with our trials. But I wouldn’t say there’s one area of priority versus the other. I would say the highest unmet medical need Okay. Is is in the unfit population. That happens to be where we’re we’re deploying our resources, you know, but it’s closely followed by these other two very important populations.

You

Corinne, Goldman Sachs: said this, so I would like for you to just double click on it. In terms of overlapping tolerability, any concerns as you start to think about combinations with Venaza or some of the

Michael Metzger, CEO, Syndax: other drugs that you’re looking at? No. I mean, we’ve seen we’ve seen Venaza in the relapsed refractory setting. We’ve seen VEN and CoV, which is another hypomethylating agent, really not adding toxicity to to what you see with ven alone or Venaza or Ven and CoV, for instance. So we feel great about that.

Patients are able to stay on drug Mhmm. Low rates of discontinuation, and really drive very high So we feel really great about that. With chemotherapy, we we tested in the relapsed refractory setting, and now we’re doing frontline newly diagnosed patients with seven plus three. In the relapsed refractory setting, did flag IDA, which is another regimen of chemo. And drug’s really well tolerated, saw, again, excellent efficacy across the board.

So we expect to see the same sort of Mhmm. You know, risk benefit on all of these combinations, really not adding significant toxin in any particular

Corinne, Goldman Sachs: Great. Maybe we can spend a few minutes on Niktombo. I know we don’t have a ton of time left, but let’s just start with a bit of background, and talk about, like, the launch came in August so we can do a bit of background and how the launch is going.

Steve Kloster, Chief Commercial Officer, Syndax: So I’ll just give a little

Michael Metzger, CEO, Syndax: background and Steve can talk about the launch. The product is CSF-1R antibody, which we enlisted several years ago from UCB and have directed it to line GVHD, so the approval came. It’s a new mechanism of action, and the product is really going after fibrosis and inflammation. So it’s a new paradigm for these patients who really have to deal with intractable disease over many years. In our trial, we saw patients who had been on on drug an average of four years.

Mhmm. And so resolution of symptoms is is is key for these patients. Mhmm. In areas such as the lung and skin where they just can’t get relief. And so we were able to see it quickly with these patients.

They respond quickly. They stay on drug. But I think it’s just so remarkable what we’re seeing is patients are not staying on this for months. They’re staying on it for years. Seeing it through our trial, a very high percentage of patients still taking the drug from our trials.

It’s rather amazing, multiple years. I expect this drug to really expand the category. We’re talking about six thousand five hundred patients in the line plus, talking about 15,000 patients in The US frontline and later. It’s not an insignificant patient population, but if you could take a very significant share and extend this for years, think multiple myeloma, things like that. I mean, it’s really an underappreciated market opportunity.

So we’re thrilled that how the drug is performing.

Steve Kloster, Chief Commercial Officer, Syndax: Steve, can talk a little

Michael Metzger, CEO, Syndax: bit about the launch, but it’s really exciting.

Steve Kloster, Chief Commercial Officer, Syndax: Yeah. I can certainly talk further. Our partners is inside on this product. So they’ve really went pretty much created the chronic GVHD space. So, you know, running start to this.

And then the drug was approved in August, but didn’t hit the market until late January to new vials. It’s really been just a stub quarter. So net sales are about $13,600,000 But like I said earlier, in terms of execution, you look at user base, the transplant audience is small. It’s very focused in the country, maybe 150 transplant centers that capture ninety plus percent of the transplants. All of them, I would say, at this point have prescribed, many of them have reordered.

What we’re hearing early is what we saw in the clinical data, early response, a durable response. I mentioned we were just at ASCO, and folks were coming up to our booth. The new Kimba booth actually sits with an insight. But even at our booth, which was Revu Forge, physicians came up unsolicited and started talking about what they’re seeing in patients.

Michael Metzger, CEO, Syndax: It’s an insidious disease. You know,

Steve Kloster, Chief Commercial Officer, Syndax: patients have have really, suffered through leukemia. They’ve gone through transplant, and then they have this long lasting highly symptomatic condition that questions the will to live, honestly. So the ability to treat patients early has been great. The feedback has been excellent. Payers are paying for the drug.

So a very, very encouraging start. Rezaroc is often the drug we use as the analog, It’s a $05,000,000,000 brand and growing. And we did in two months, what took them about six months to do in terms of activating user base. So really good stuff.

Corinne, Goldman Sachs: Maybe we can spend a minute on, like, cash flow, balance sheet. I think you guys have said that you’re confident that you’re funded to profitability. You almost understand the path to profitability. Like, what point do you do you flip to being cash flow positive?

Michael Metzger, CEO, Syndax: Yeah. Look, I think we control our own destiny. We say we’re gonna be profitable in the next, you know, few years, and I think that’s a really solid statement. The products that are contributing are twofold. Right?

So we have we have Nictimbo, which is a very high margin product, basically profitable in the two months. We think that’ll meaningfully contribute to the profitability of this company. And, RevuForge also hot ramping quickly. And we know that those expenses are kind of fully loaded in the P and L at this point in the next handful of years. So we don’t expect expansion of expenses.

We don’t expect expansion of SG and A expense, R and D expense, SG and A. So we’re kind of where we are today, and we feel very good that with expanding product revenue and very good margins that will have contribution from both and get the profit delivered in that time.

Corinne, Goldman Sachs: Probably just without saying, that include the Phase III programs that you guys have talked about?

Michael Metzger, CEO, Syndax: It includes everything. It includes everything, absolutely.

Corinne, Goldman Sachs: Given that, how would you think about bringing in additional assets from here? Is that something you guys are interested in? And what kind of criteria?

Michael Metzger, CEO, Syndax: Yes. Look, I think we can probably talk about new assets in the future. I think we are so focused on driving value with these two franchises. We’re in a very unique situation where we have two approved products that are best in class and really differentiated in their markets. And our job is to bring it to as many patients as possible and drive value for PersonalX shareholders.

So I think we can do that successfully with these two products in a way that other companies can’t. So all of our energy, all of our resources are really going into achieving that. So new products, we’ll talk about that maybe sometime.

Corinne, Goldman Sachs: Lots to do. Lot of things. Yeah. Absolutely. Lots to do in the existing portfolio.

Wonderful. Well, thank you guys so much for joining us. Thanks to everyone who joined us here and online, and really appreciate the time this afternoon. Thanks so

Michael Metzger, CEO, Syndax: much.

Steve Kloster, Chief Commercial Officer, Syndax: Thanks, Corinthian.

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