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On Thursday, 13 March 2025, Syndax Pharmaceuticals (NASDAQ: SNDX) presented at the Barclays 27th Annual Global Healthcare Conference. The company outlined its strategic vision to dominate the menin inhibitor market, highlighting the promising launch of Revuforged and Niktimbo. While the company remains optimistic about future growth, it also addressed challenges such as managed care coverage and clinical trial progress.
Key Takeaways
- Syndax’s initial launch of Revuforged showed strong results, with 53% managed care coverage within three months.
- The company plans to expand into the MPM1 market, leveraging its first-mover advantage in the KMT2A-rearranged AML segment.
- Financial results revealed $7.7 million in net sales for Revuforged in the stub quarter.
- Syndax is actively pursuing guideline inclusion and regulatory approvals to drive future growth.
- Clinical trials, including the HOVON study, are progressing, with first patient enrollment expected in the second quarter.
Financial Results
During the stub quarter ending December, Syndax reported net sales of $7.7 million for Revuforged. Approximately one-third of these sales were attributed to inventory in the channel, while the remaining two-thirds represented actual demand. Inventory levels are anticipated to stabilize at around two weeks, a typical duration for targeted therapies. Managed care coverage has reached 53% and continues to grow.
Operational Updates
Syndax has launched two products, marking its transition to a fully integrated commercial company. The company is engaging with approximately 2,000 treatment centers, with a focus on the top 200. Notably, one-third of these prioritized institutions have already prescribed Revuforged, and most have issued multiple prescriptions. The company is also witnessing a shift in patient treatment paradigms, with earlier diagnoses leading to improved outcomes.
Future Outlook
Syndax aims to maintain its leadership in the menin inhibitor space, with plans to transition Revuforged to frontline treatment indications. The company anticipates significant growth from both the KMT2A-rearranged and MPM1 markets, the latter being larger in relapsed/refractory settings. Syndax expects to publish clinical trial data in the second quarter, aiming for guideline inclusion and regulatory approval by the end of the year. Additionally, multiple FIT trials are scheduled for the second half of the year.
Q&A Highlights
During the Q&A session, Syndax noted that the initial launch of Revuforged exceeded expectations, with minimal early access program patients and no warehousing issues. The company reported that claims are being processed and paid by various payers, including Medicare Part D, commercial, and Medicaid, with most patients accessing the drug within days. Off-label use of Revuforged in MPM1 patients is being monitored, and Syndax is poised to leverage any accelerated approval endpoints available.
For a deeper dive into the details, readers are encouraged to refer to the full transcript below.
Full transcript - Barclays 27th Annual Global Healthcare Conference:
Peter Lawson, Biotech Analyst, Barclays: My name is Peter Lawson. I’m one of the biotech analysts at Barclays and had the pleasure of covering Syndax since I’ve been at Barclays and a little bit beyond that as well or before that. So really pleased to have on stage with me, Michael Metzger, CEO and Steve Colster, Chief Commercial Officer. And I’ll be moderating this conversation. And first set of questions I’ve been asking just to see if there’s any kind of impact of the current administration, say on supply chain, tariffs.
Is there any worries there from importexport? Is there any worries from I guess you’ve got prepared from COVID, but just any concerns we should be thinking about APIs, etcetera?
Michael Metzger, CEO, Syndax: Yes. Good question. First of all, let me just say thank you for having me in Florida and be speaking with you. Obviously, your coverage is very good and fantastic and has been for a long time. So, thank you for all you’re doing for us.
Thank you. So, just to answer your question, I think it’s a timely question relative to tariffs and changes and so forth. No, I think we’re pretty well situated today. I think in terms of we’re now a commercial stage company. We have products that we’re marketing in The U.
S. So, I think we make our products in The U. S, and we’re in pretty good position relative to what we know today, right? So I don’t think there’s anything in particular that we could point to that is particularly troubling that is going on with the administration that would impact us to the extent we know.
Peter Lawson, Biotech Analyst, Barclays: Okay. And then the impact on the FDA, have you seen any kind of changes in communication?
Michael Metzger, CEO, Syndax: We haven’t. We’ve had a great relationship with the FDA. We just went through the NDA process and the BLA process for that matter last year with Revuforged and Actimbo, both went well. Drugs are both approved, which is a good place to be. So, we have experience with them and have a good relationship with them.
I think the changes that are being talked about, at least since most recently, I think some of those changes have been kind of rolled back, potential changes. So, in terms of one thing to keep in mind, we do pay PDUFA fees. So, user fees actually help to employ FDA people and allow us to have ongoing review work done. We have an sNDA that we’ll be filing this second quarter and hope to get the drug approved in the second indication, Revuforged, by the end of the year. And so I think that we expect to be able to keep to our timelines.
We haven’t seen any impact relative to what at least we know today from FDA.
Peter Lawson, Biotech Analyst, Barclays: Got you. Thank you. And then I know it’s a long term impact that NIH budgetary cuts does does that have any role and effects with clinical trial sites?
Michael Metzger, CEO, Syndax: Again, I think it could potentially could, but we don’t we haven’t seen any impact just yet. I think we’re kind of status moving. Woah. Yes. So, I think we’re moving ahead.
We are standing up number of trials and doing a lot of work globally with our clinical trial sites. And so, I think that plan is intact as far as we know that any kind of cuts have not had a direct impact on that.
Peter Lawson, Biotech Analyst, Barclays: Got you. Thank you. And then how should we think about revenue launch and kind of the puts and takes for 2025, so coming back to
Michael Metzger, CEO, Syndax: Right. So we did launch our two products. And so it’s just an exciting time for the company as we are now a fully integrated commercial company. And maybe I’ll we did report our first stub quarter. So in the fourth quarter earnings, we talked about revenue for Revufor is not yet for NIktimbo.
But maybe I’ll give Steve the floor and let him kind of give some details here.
Steve Colster, Chief Commercial Officer, Syndax: Yes. Happy to. As Michael said, we reported earnings, I think it was last Monday. So, we had 7.7% in net sales through December. About a third of that was inventory, ultimately be about two weeks in the channel moving forward from inventory.
Most of the sales will the other two thirds were of demand. Very few patients rolled off of VAP. How I would characterize the launch and we had provided some commentary on our earnings call last Monday around a few things. It’s finding patients and activating the user base. It’s also getting the drug on formulary managed care payer plans and also getting patients on drug quickly.
So, we’ve been able to a very good start. This is data really through February. We call on about 2,000 treatment centers. We prioritize the top 200 centers. These are largely medium large size academic institutions.
About a third of those institutions have written, which we think is a great start. Majority of those accounts have written more than once, which we believe is a really good start. Certainly, a lot more upside as we roll forward. On the managed care front, we’ve activated against payers well before the approval. Our formulary coverage is at 53% and growing every day.
We think that’s great at the third month mark. More importantly though, claims are being adjudicated and paid by payers and claims are rolling through managed care formularies, Medicare Part D plans, commercial as well as Medicaid. And I think the last piece that’s relevant is how do we get patients on drug quickly. We know the earlier diagnosis, getting patients on drug fast leads to really better outcomes. So, we’ve got some great trade partners and some really talented internal folks.
Many patients are getting on drug within a couple of days. So, from what we see at the launch, it’s good. We’ll provide more data and more updates and perspective as we roll over the coming months. We’ve got our next earnings is coming up in early May.
Michael Metzger, CEO, Syndax: Yes. I think just to cap it off, I think that we’re in such a good position as a company to really dominate this space. We’re the only menin inhibitor to have a KMT2A broad label. We probably will for several years be in that position. I think that market in particular has really good setup for us to bring a very important medicine to patients and really drive utilization and then the idea is that we would get the second indication in 2020, first hopefully with guidelines in place and then ultimately with through the second the sNDA process.
So, I think we’re in a really good position to really dominate the men in space over the next several years. And then ultimately, we’ll transition to moving more towards frontline.
Peter Lawson, Biotech Analyst, Barclays: Got you. How important is the first to market
Michael Metzger, CEO, Syndax: movement? Super important. I mean, I think our point of view is and I think this has been proven over time with oncology drugs. I think being first with a potential best in class agent is a game changer for physicians, right, in terms of getting the right organization lined up to meet the needs of physicians and patients immediately. As Steve described, we’re doing right out of the gate.
And having that relationship with a physician, giving them the service on an ongoing basis, making sure that they understand how to use the drug and ultimately getting it paid for and following their patients, helping them to manage through the utilization of the drug. I think all of that is the type of service that you need to provide, but we can do it as first mover without any competition in the market, helps us really build competitive immunity for what we think will be several years.
Peter Lawson, Biotech Analyst, Barclays: Got you. Thank you. And then how should we think about the new patients coming on? Is that kind of a steady process? Is there seasonality there?
Do you get a kind of initial bolus? And just
Steve Colster, Chief Commercial Officer, Syndax: talk us through your thoughts. Yes. Good question. And we’re not going to provide strict numbers yet, but we’ll see. We need more data to see how things play out.
I think when you think about the initial patients that come on drug, I mentioned there wasn’t a whole lot of EEP patients, but and there’s not a lot of warehousing or bolus. Patients are just too sick, too advanced in their disease. So, what we saw and some of this is anecdotal, we don’t have all the data on specific patients, but you had patients that were close to first relapse and you had patients that were brought off the hospice. So, we believe from what physicians tell us, physicians will move patients earlier. There’s better treatment outcomes if you identify those patients earlier.
So that will certainly change over time. I mentioned the one third of our priority accounts that I’ve written. The restful, right? I mean, there’s urgent unmet need in the space. Physicians really like what they see in the profile of Revuforged.
It’s not a matter of if those treatment centers and those associated physicians will prescribe. It’s just a matter of when. We’ve got a very experienced customer engagement sales team. We’ve got support in the field to make sure that physicians and associated nursing staff understand how to use the drug successfully and onboard patients. So, we think it will be steady over the course of the year.
We just need more time to activate the user base.
Peter Lawson, Biotech Analyst, Barclays: Got you. Okay. And then, so we what should we think about gross to net inventory for a couple of weeks or something like that? Is that a sensible number to think about? And should we think about any more patients coming off of the extended access program?
So, there are no more patients off of
Steve Colster, Chief Commercial Officer, Syndax: our expanded access program or EAP. They’ve already been transitioned for KMT to AR patients. The inventory assumption is it’ll be about two weeks and that’s typical for targeted therapies like this. So the inventory that we built through December, the only change is inventory expansion as we move from quarter to quarter. Obviously, as demand grows, that inventory will build up and we’ll try to give some guidance on that over time.
Got you.
Peter Lawson, Biotech Analyst, Barclays: And then you talked about these Tier one and Tier two accounts and what I think 33% penetrated. How quickly can you penetrate the rest? What’s the most addressable kind of population you should think about as regards to those Tier one and Tier two sites?
Steve Colster, Chief Commercial Officer, Syndax: Yes. As I mentioned, I mean, it’s not a matter of if they’ll ultimately convert it to when. And they’ll all go at their own pace. I mean, we’re doing obviously everything we can. We’ve got great relationships with institutions.
I think what’s interesting in the user base right now, there’s no clear pattern, meaning there are treatment centers that were involved in the clinical trials for revumetab, those that were not and they’re writing. I think that the base of prescribers and beyond the Tier one, Tier two users, there’s a much larger group, right, that are, we’ll call them Tier three, meaning they’re smaller. So smaller academic centers have begun to prescribe. And I think also interestingly in this launch, community oncology tends to play a more substantive role later, year one, year two, year three. There’s not a ton of business in community oncology right now, but there’s more than one would suspect.
And I think that speaks to their ability to identify patients through diagnostic testing. I think it also speaks to the how they view the profile and the ability to use the drug and use it successfully. Got
Peter Lawson, Biotech Analyst, Barclays: you. Is there as we think about those T1 and T2 accounts, is there kind of a goal by the quarter by quarter? Or is that kind of a fluctuating thing?
Steve Colster, Chief Commercial Officer, Syndax: A goal that we have for converting? Sure. I mean, we’ll look at our deployment activity levels. We prioritize those accounts because they just they have more patients available to be treated. The Tier one, Tier two account for about two thirds of the opportunity.
So, we’ll be excited to share progress over time. But and of course, we have some internal goals on what we want to accomplish. It’s not ready to share those out externally.
Peter Lawson, Biotech Analyst, Barclays: Quarter by quarter, what metrics will you be sharing with the street?
Steve Colster, Chief Commercial Officer, Syndax: Certainly, the ones we’ve already covered users, a sense of obviously repeat prescribers, managed care formulary coverage. In fact, I mean, the numbers are already provided here through February are outdated because they have gone up. And we’ll look towards providing more substantive numbers over time. I think for us, it’s still early three months in. While we do have a limited distribution network, we don’t have visibility to all of the data, meaning prescriptions that go through the specialty distributor side.
We just know the customer the product goes to, but we don’t know number of patients. We don’t know if it was a new patient or refill or mutational status. For the specialty pharmacy side, which is about half of our business, we get more information. So as that coalesces over time, we get more usage, we’ll be able to be more accurate and then we’ll be able to report more.
Peter Lawson, Biotech Analyst, Barclays: Got you. Has there been anything that surprised you in the launch in the first few weeks?
Steve Colster, Chief Commercial Officer, Syndax: Yes. Some of the things I’ve already mentioned, I think the role of community oncology is certainly interesting. The makeup of who’s been using, I mean, that we likely could have predicted. The base is where we expected, perhaps on even on the bigger side. Traction on the drug is high.
I think the setup Michael may have referenced this a bit earlier, but just the setup for the data. We had MPN one top line data back in November, ’2 weeks later was the approval and then ASH. And I think all the interest in the menin space accrues to the benefit of Revuforged because it’s the only drug on the market. So, I think that’s uniquely created some momentum for us as we roll through the early launch period.
Michael Metzger, CEO, Syndax: Not that it’s a surprise, but I think the broad excitement about the profile and the ability to use the drug and use it well on a continual basis is really exciting to base. So, I think it’s not a surprise, but it’s nice to see.
Peter Lawson, Biotech Analyst, Barclays: Yes. Encouraging. And then as we think about the MPN one side of the story kind of the inclusion in the guidelines, do you think that kind of helps bolster sales or is do you expect a change there in the pace?
Michael Metzger, CEO, Syndax: Yes. No, I think look, I think guidelines what we had said is we’ll look to publish the data in the second quarter, get it into the guidelines soon thereafter, hopefully around mid year. The drug will hopefully get approved before the end of the year. So, there’s a a little gap in time. So, the ability to get guidelines, physician’s practice to guidelines, and I think that’s a very important aspect of the launch.
And so, yes, I think getting through guidelines and ultimately approval, I think that will be additive to what we’re seeing in KMT2A. I mean, Steve mentioned script base is primarily KMT2A. We are seeing some additional or at least hearing about some additional off label use. We don’t promote it that way, but that’s certainly how physicians have the opportunity to use the drug in other capacities. So, I think ultimately when you see guideline inclusion and an approval that should really lead to the next leg of our growth for sure.
Got you.
Peter Lawson, Biotech Analyst, Barclays: Okay. Have you been seeing it used off label, so in the NPN one percent of patients?
Michael Metzger, CEO, Syndax: We’ve heard yes, we have heard that. It’s hard to identify in the data today. We know that that’s happening.
Peter Lawson, Biotech Analyst, Barclays: Would you be able to break that out going forward, so MPN1 versus KMT280?
Michael Metzger, CEO, Syndax: Yes. You want to comment on that?
Steve Colster, Chief Commercial Officer, Syndax: Yes. As I mentioned, some of the data we get through our specialty pharmacy and our hub, which is about half our business, we don’t get all mutational status, severity of patient. We get some basic information. So, it’s going to take us a little bit of time to piece this together as more patients are put on drug and there’s more experience from an HCP standpoint. We’ll be able to do more work and some claims work and some actual market research really understand.
But now as Michael said, it’s anecdotal, but we hear it from multiple sources.
Peter Lawson, Biotech Analyst, Barclays: Okay. And will that improve over time? Or do you care at the end of the day? Or is that an important metric internally?
Michael Metzger, CEO, Syndax: Well, no, not necessarily. I mean, I think we know that the growth we have tremendous opportunity in KMT2A and we need to really work through that and just drive growth that way. We think that can really build the business, a substantial business. And ultimately, MPM One will come when it does and physicians are going to utilize it and we think that’s a big piece of business as well. So, they’re kind of unrelated in a way, because we think that they’re both big drivers of upside.
So, it doesn’t necessarily mean that we have to break it out and have to show it, but we will do our best to actually track it at some level. Yes.
Peter Lawson, Biotech Analyst, Barclays: Because what initially you want to track to just make sure there’s nothing going wrong with it not being used in NPM one page?
Michael Metzger, CEO, Syndax: Sure, sure. No, I think we’ll have a better feel for it. It’s just in terms of importance, I think there’s to your point, there’s some importance of understanding how the market’s evolving and how we’re penetrating. But ultimately, we think it’s going to flow very well.
Peter Lawson, Biotech Analyst, Barclays: Got you. Do you have kind of internal goals of where you think that would be for like 2025, the distribution of MPM one versus?
Michael Metzger, CEO, Syndax: We do. I think it’s premature to share anything like that. We know that the MPM one market is in terms of number of patients for in relapsedrefractory, at least is bigger than KMT2A. But we haven’t shared what we think the breakdown is and how much in terms of penetration over what time.
Peter Lawson, Biotech Analyst, Barclays: Got you. And just on to some of the clinical trial readouts and initiation, so the Benazir and unfit to the HOVON study. Yes. Kind of remind us where you are in initiating and kind of the gating steps for the first patients.
Michael Metzger, CEO, Syndax: Right. So we are in the initiation phase of that trial. This is our, as you mentioned, our benaza combo with Rev frontline newly diagnosed patients. It’s a global registration trial. We will be the first to frontline through this through this as the start up of this trial, highest unmet needs.
So, really important new opportunity for us to be first to market. And this trial is initiating now, and so we’re getting sites up and running. And then first patient, we haven’t said exactly when, but certainly in the second quarter, we’ll have that.
Peter Lawson, Biotech Analyst, Barclays: Got you. Okay. So initiated second quarter, you get the first patient? Yes. And then what’s the split you think is going to be U.
S. Versus ex U. S. For that trial?
Michael Metzger, CEO, Syndax: So I’d say predominantly it’s I would say ex U. S. I mean we have U. S. Sites as well, but a lot of our sites will enroll ex will stand up outside The U.
S. So it’s a global trial and we’ll look to get it drug registered globally.
Peter Lawson, Biotech Analyst, Barclays: Got you. Is there just going back to the kind of the funding questions, I guess, there’s no is there any worry European funding or external funding? No, no, I don’t think so. Any worries around the ability to control that study just because it’s HOVON run study?
Michael Metzger, CEO, Syndax: We have a very close collaboration with HOVON. So it’s collaborative in that sense, and we have a lot of involvement in that, both in the stand up of trials and how we do that. They are the sponsor, but we are working actively in The U. S. And outside The U.
S. With them to do that. So, it’s a very close relationship. They’re expert in this. They’re probably the best in the world, but executing on these trials.
They’ve done it many times. And they have the premier centers, the premier physicians involved. They have things like centralized patient identification, all the things that you would want, and they’re very proactive group. So I think we feel very confident that they’ll be excellent to work with and have been so far.
Peter Lawson, Biotech Analyst, Barclays: Okay. And are there any what are the kind of the pros and cons of working with Hovland? Clearly, you highlight some of the benefits of they’re up and running and they kind of know what they’re doing, but any time delays in getting the data or just
Michael Metzger, CEO, Syndax: No, quite the contrary. I think they’re excellent at they have a centralized system for collecting data and they’re very good at and experience at doing that. And we’re closely collaborating with them. So, there’s no issue with timing of data transfer or any of that sharing. So, we no, I don’t think there’s any downside.
I think we actually see all the upside of working with them. I mean, the alternative, of course, is to do it solely on our own and stand up a global trial. We’ll do that also, right? We’re doing that on the fit in the fit population as we start to get those trials up and running in 2025. But we’re taking an approach where it’s not just one size fits all.
We’re doing different trials and working with different people to get the best of the working environment, people that had a lot of experience doing these things.
Peter Lawson, Biotech Analyst, Barclays: Got you. Thank you. And then should we is there an interim read that we should be thinking about for the HOVALM study?
Michael Metzger, CEO, Syndax: So, we haven’t been completely transparent with all of our plans. I think we’ve announced the trial design. It’s on ct.gov, so you can see it, but it’s a randomized trial with an OS endpoint that is the gold standard as we know. A CR endpoint an interim CR endpoint is something that I think others have talked about as an accelerated approval endpoint and something that we haven’t expressly said out publicly, but I think it’s a potential option for us as well. Any kind of accelerated endpoint, I think we should assume whether it’s in the HOVON trial and some of our FIT trials that we’ll be working through and getting up and running this year, I think you should assume that we’ll avail ourselves of any accelerated approval endpoint that is available, right?
So, we have conversations with regulators. We don’t usually talk about our regulatory interactions, but I think we feel quite confident that we’ll be in a good position to leverage some of these
Peter Lawson, Biotech Analyst, Barclays: as well. Is that a seamless process where you can kind of make those amendments to have an accelerated endpoint integrated into the trial? Yes. Perfect. That’s okay.
Thank you. And then just expectations around CR8s and OS that we should be thinking about for the control arm at least?
Michael Metzger, CEO, Syndax: For the HOVON trial?
Peter Lawson, Biotech Analyst, Barclays: Yes.
Michael Metzger, CEO, Syndax: Yes. I mean, again, we haven’t released the statistics. I think you can think about the VYALA A trial as a kind of model for some of this, I think in that trial, the CR rate was forty three percent. If I remember, OS was 14 point seven months. So, I think those are the hurdles for the Viala A trial.
Again, we haven’t talked about what we would need to achieve relative to an increment on top of what standard of care is, but I think you should kind of think about those metrics as at least the baseline of what we need to do better than.
Peter Lawson, Biotech Analyst, Barclays: Yes. Should and should we use BEAT AML as a kind of a proxy for what’s being run now?
Michael Metzger, CEO, Syndax: Same population. We’ll take a broad population. So BEAT AML enrolled and is enrolling both MPM1 and KMT2A patients. And that’ll be also the case in our trial. The trial will be really powered for MPM1.
That’s the majority it’s a much bigger patient population, especially in the unfit population than KMT2A is, because most of the KMT2A patients are younger and fit for chemo. So, I think that’s the that is the population that has been enrolling in beta ML. So, it’s very much similar. So, I would certainly think about that as a proxy for what we might be able to accomplish in our trial, right? So, that’s why we’re so encouraged.
That’s why the physicians are so encouraged seeing in the beta AML trial one hundred percent response rate, ninety five percent MRD negativity, just fantastic data on top of VENAZA. So that’s what we’re gearing up for. Okay.
Peter Lawson, Biotech Analyst, Barclays: And then as we think about first line FIT trials, it sounds like there’s other trials being planned and worked through kind of what should we expect in the second half as regards to those?
Michael Metzger, CEO, Syndax: Right. So we’re excited about our plans there. We haven’t been as explicit on the FIT trials yet. We will be finishing our Phase one dose ranging. We already feel pretty confident in the dose.
We’re exploring two doses that are both active as monotherapy. We’ve also explored those doses in a chemo combo in our AUGMENT 102 trial. So, we have a very good understanding of what the dose is likely to be and we’re enrolling some additional patients there to just confirm that. We’re in the at the same time looking to stand up, I’d say, more than a few trials this year. And they’re going to be some will be registration directed, some will be more guideline directed.
And the populations there are several populations within the FIT setting that we need to address. And so, our strategy, which will keep private for the time being, really looks to address the broadest and most important populations there, get the drug approved and also build a base of data that physicians can relate to as they start to use our drug as an approved
Peter Lawson, Biotech Analyst, Barclays: agent. It is we’ve hit the bottom of the hour. But thank you so much. Always a pleasure speaking to you. Thank you.
Thank you.
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