Conference Transcript - ARGX 2026-05-12

Tizina Maad, Analyst — Bank of America

Hello, and welcome back to the Bank of America Healthcare Conference. I'm Tizina Maad. I'm one of the biotech analysts here at the bank. Our next presenting company is Argenics. Sitting up here on stage with me is the new CEO, Karen Massey. Congratulations, Karen, on the recent promotion. We're going to be spending time, I think, focused a little bit on the pipeline because there's a lot going on. but maybe we can start off by talking about what your vision is for the company now that you're helming all the major decisions and how you think about strategy going forward. Yeah, thank you so much.

Karen Massey, CEO

It's great to be here and a great conference. So and appreciate the opportunity. I mean, I would say we have laid out our growth strategy for Argenics and that's Vision 2030. And so that gets us to, you know, 50,000 patients by the end of the decade, 10 labeled indications and five molecules in late stage development so we have a i would say we've had that plan in place and we continue to execute on that plan but as i think about my strategy and and and where we want to take argenics um over the the decade even beyond that you know the the vision for argenics has always been to be an immunology innovation company and so uh we're the first leg of that was building vivgut um and establishing vivgut and and we've uh and i think we've demonstrated the ability to do that. We want to continue that leadership in FCRN. So the first leg of the next part of the strategy is extending our FCRN leadership for decades to come. So we have two next generation FCRNs and we're investing in an aural. So that's a big part of the vision for the company. But the second part is how do we also build a non-FCRN portfolio that is as meaningfully contributing to Argenix as the FCRN portfolio. And that's really all about hopefully what we can talk about today, the pipeline beyond VivGuard.

Tizina Maad, Analyst — Bank of America

Okay. So since you mentioned them, let's maybe spend a couple minutes on those next generation assets that you're looking at for the GMG and CIDP indications, as well as the oral of CRM. So until recently, you were heading up the commercial organization. So you can talk to this in real time. What do you think theoretically would be the benefit of, let's say, having an oral FCRN versus the various different methods that you have of delivery now, everything from IV to Hytrulo to pre-fill syringe?

Karen Massey, CEO

Yeah. I mean, our strategy from the beginning with VivGuard and we'll continue with FCRN is that we want to transform these diseases that have significant unmet need. And with VivGuard, what we believe we can deliver in MG, what we're seeing we deliver also in CIDP is really strong efficacy but importantly safety across the broad indications and then as you said the convenience for patients so we launched with infusion we brought subcutaneous and then pre-filled syringe that taking that playbook across all of the indications allows us to start to think about patients being treated earlier in their disease so we're really expanding these indications expanding these markets it's a big part of our strategy and will be a big part of our strategy for future indications and the oral helps us to do that so as well as our next gen so 213 one of the next gen fcrns moves to every four week dosing so it's a step forward in convenience and potentially with the oral you can imagine that's another step forward in convenience but still maintaining that efficacy and safety profile of fcrn when you think about fcrn from an MOA, we know that there are many, many indications beyond what we already have approved in MG, CIDP, and ITP. And so we think by having multiple next generation options, we have the opportunity, yes, to provide more convenience in the indications that will already be approved in. But we can also start to continue to push the boundaries of the biology of FCRN and even further expand those indications beyond.

Tizina Maad, Analyst — Bank of America

Yeah. So it seems though that you've done a good job of moving patients into frontline. I remember, I think, our first version of the model when the drug first launched had a much lower peak sales penetration. And I think part of the reason for the constant revision upward is that the company has been able to move higher in lines of therapy. So as you think about, you know, in a world where, let's say, auto-injectors are much more commonly used because of, let's say, the GLP-1s, the company is developing an auto-injector pen. So as you think about the incremental benefit from moving to a pre-filled syringe, to an auto-injector, to an oral, how much of a meaningful impact do you think you're going to be seeing with each of those iterations?

Karen Massey, CEO

Yeah, I mean, I think for each of these innovations, we do see it as a step forward and driving the majority of our growth. I mean, if you think about the step that we took going from subcutaneous to pre-filled syringe and, as you said, continually increasing the revenue and the projections, that's being driven by pre-filled syringe because what you see is that we've been able to expand the prescriber base significantly beyond where we thought it would be when we launched VivGut, significantly expand the addressable market because of the simplicity of being able to use a pre-filled syringe at home rather than having to go for an infusion. So it is a step forward. I think the auto-injector is less of a step change because the big shift was going from HCP-administered to patient-administered with the pre-filled syringe. The auto-injector will still, assuming approval and we get through the regulatory framework, will still be patient-administered. It helps because it's easier to handle. It helps because if patients are needle-phobic, but it won't be as much of a step forward. But it is, but it will continue to be a differentiator for us. And then you can imagine the oral, as you say, I mean, we'll see how it plays out with the GLP-1s, but I think you can imagine that going from an injectable to an oral opens up a different pool of patients for whichever indication you're looking at.

Tizina Maad, Analyst — Bank of America

So maybe let's talk about a couple of the near-term data catalysts that you have upcoming late stage. Let's start with myositis. So just remind us, when is that phase three reading out? And you were looking at three different subtypes. I think there's been some questions about, you know, what the company thinks is going to be the biggest value driver. So maybe talk to us about those items.

Karen Massey, CEO

Thank you. It's exciting. We have the data reading out in Q3. And yeah, so we see myositis as our next growth frontier, our next growth opportunity. And we see the addressable market as around 70,000 patients. So to put that in context, that's similar to where we see the MG market, addressable market right now. So you can think of it as an MG-like opportunity from that perspective. We designed a clinical trial that allows us to explore three of the different subtypes. So we have IM&M, we have DM, dermatomyositis, and then PM. And from my perspective, you know, each of the subtypes has a clear biology rationale, and we can go through what that is. I would say we're very disciplined at Argenics, always starting with the biology and the science. So we have a clear biology rationale for each. And we designed a clinical trial that is very thoughtful in that it allows us to look at the overall population, but also the subsets so that we can really explore and we have multiple paths to success depending on the data that we see. In terms of the value driver, from my perspective, and certainly as a commercial person, I'm really excited about IM&M, and that's what we've been talking about. As we've been doing our homework to really understand the commercial opportunity, what you see in IM&M, there's nothing else approved. This is a completely white space indication. We think there's around 20,000 patients, so that's bigger than the TAM for CIDP. it's bigger than the addressable market when we launched mg um and and and you we you'll remember that with myositis um it's weekly dosing so cidp like dosing so being able to go to a space where there is a huge unmet need um there there is no competition including no competition on the horizon and there's also quite a bit of overlap in imnm it's if you think about the treatment the treating physicians it's more neurology and rheumatology um and obviously we have strength in rheumatology. So the overall opportunity is a big value driver for us. We have a lot of belief in the biology for all of the different subtypes. But if you had to ask me to prioritize and where I'm most excited about, because of the patient unmet need, I think IM&M is going to be really exciting. Okay.

Tizina Maad, Analyst — Bank of America

And to say that you're most excited about is not the same thing as less bullish about DM, correct?

Karen Massey, CEO

That's exactly right. I would not say that. I mean, I think In DM, it's just a different dynamic. I'm excited about the overall myositis opportunity. In DM, we have a strong biology rationale. The difference from a commercial perspective is the dynamics. It's more heterogeneous, so we know that there's more at play in driving the disease, and there is more competition. But it's a larger patient population. We think that that's a 40,000 TAM. So, look, I think that's also an exciting opportunity. It's another one where it's bigger than the CIDP TAM at launch. So, yeah, it's certainly a big value driver. There's a good biology rationale. And I look forward to seeing actually the data play out for each of the different subtypes because I think they each have their own unique opportunity.

Tizina Maad, Analyst — Bank of America

So when it comes time to showing the data and the press release, give us a sense of what level of granularity we're expected to see.

Karen Massey, CEO

Yeah, I mean, at Argenics and the style that we have is that we like to be transparent and we want to share as much data as possible. This is our first entry into rheumatology. And some of the medical congresses and conferences with rheumatology are a little more strict. And we want to make sure that we have the opportunity to make a big splash with this data when it reads out. So we'll definitely share the top line and as much detail on the subtypes as we can. but we'll just have to, once we see the data, we'll have to determine what the right path forward is there. But we like to be able to give transparency and we'll be able to give a good sense of where we think we're going with this indication.

Tizina Maad, Analyst — Bank of America

Okay. Now, because of the different prevalences of the three different subtypes, how should we be thinking about the balance of each of the subtypes that finally enrolled into the study?

Karen Massey, CEO

Yeah. So the way that we designed the study, if we maybe just take a step back, it's a seamless phase two, phase three. And we did not cap any of the subtypes. So we let the enrollment run both for phase two and then also for phase three. So you can imagine that it's not an equal distribution across all of the different subtypes, but it enrolled based on, I would say, unmet need and as well as the disease characteristics. characteristics. So we haven't shared the underlying sort of the number of patients across subtypes, but we'll be able to do that, I would say, at top line. Okay.

Tizina Maad, Analyst — Bank of America

But there isn't a concern internally that you enrolled too few people in one of the subtypes and therefore it wouldn't be strong enough to support an application?

Karen Massey, CEO

No. I mean, it's a basket design. So we're able to look at, from a statistical analysis plan perspective, the total population as well as the subtypes. Okay.

Tizina Maad, Analyst — Bank of America

And one other common question we're getting lately is, what are your discussions with FDA on the subtypes? Like, is there flexibility to look at the data and then decide if you, you know, want to apply for a full label for all three or a subset of them?

Karen Massey, CEO

Yeah, I mean, as always, we're engaged very closely with the FDA, especially on such an important study. And as always, it will come down to let the data speak, and then it'll be a review decision. But I think we have multiple paths forward. Okay. And then in terms of a commercial organization, assuming that you move forward in some capacity with this indication, what changes would you need to make if any um to to the field force yeah so this is our potentially uh our first step into rheumatology um and so we with with this step and then of course we have on the horizon Sjogren's uh another exciting potential rheumatology indication so we do start to think about expand expansion so that we can cover more of the rheumatology it's a different footprint Also potentially different support services for patients so that you can compete in rheumatology versus neurology. So we can leverage a lot of the commercial infrastructure that we have with, let's say, some expansion so that we can make sure to have the reach that is needed to succeed.

Tizina Maad, Analyst — Bank of America

So if this becomes, let's say, another potential vertical for the company, you just mentioned Sjogren, so maybe let's talk about that.

Karen Massey, CEO

You've got a study underway, which I believe reads out sometime in 2027. um so you wouldn't be alone in looking at Sjogren's um so maybe mechanistically can you just talk us through why it made sense to to invest the you know the money and the time into looking into this yeah um certainly I mean I think one sort of takeaway that I think about with Sjogren's is and some of these rheumatology indications is there was always uh the auto antibodies were generally known but it was always thought that they were more of a bystander or an outcome of the disease. And I think what the real shift has been is that no, in these rheumatology indications, actually, these IgGs could be driving the disease. And I think that's what we've seen in Sjogren's. So the way we went about Sjogren's is the same sort of disciplined approach that we go about to choose any indication, look at the biology, and that's what we can look at, and look at the development path, and then what is the unmet need. The way our development plan I think was quite innovative and agile and reflects the Argenix way. Our competitor did a big phase two study, so we were able to leverage the data from that competitor in FCRN that really demonstrated that the biology rationale was there. And what we did was a small, very focused phase two study where we went deep to really understand the drivers of the disease and really understand how to run a clinical trial so that we were able to execute. And I would say leapfrog ahead. And as you say, we have data coming out next year. And it's certainly an exciting indication. In terms of the third category that we always look at, Sjogren's, I mean, it's a big patient population, 300,000 patients. But what we see is, especially in the sort of around 100,000 more severe patients, there's a real serious unmet need and patient need. It goes beyond just dry eyes. There's systemic manifestations of the disease, and that's where we think we'll have the biggest benefit.

Tizina Maad, Analyst — Bank of America

Okay. And the investments that you would theoretically be making now into rheumatology would be for all indications, right? That's right.

Karen Massey, CEO

That's right.

Tizina Maad, Analyst — Bank of America

And with the targeted physicians, we've talked about the overlapping, let's say, of neurologists who treat CIDP and GMG. So when you make the decision into moving into various rheumatology indications, is it too simplistic to say that any rheumatology indication would be something that, um, the community physician of community, um, rheumatologist would be treating, or are there specializations within that?

Karen Massey, CEO

I think it will, we still need to do the workup. Um, but, but it'll be, I would say it's more special. It'll be more specialized than that. Even when you look at neurology, um, when, you know, we have over 5,000 neurologists that are writing, uh, VivGuard and, and, and so there are a lot of breadth. That's not all of neurology. You know, there are some neurologists that mostly treat migraine. So as an example, so we're not targeting them. It'll be the same in rheumatology. And so what we'll be able to do once we have the data and once we, as part of our launch preparation is really target what's the, where do we need to focus our energy to get the best return on investment? Okay.

Tizina Maad, Analyst — Bank of America

Let's maybe move on to MMN, which is another pivotal study that's going to read out on the fourth quarter, I believe. And, um, it's going to be your first foray of a, of a late stage study. That's not FGAR-Tikamad. It's going to be with AMPA. So maybe talk to us about, um, you know, the excitement that you guys have internally about this particular program, because it could open the door to many other indications for this different drug. Yeah, absolutely.

Karen Massey, CEO

We're very excited about Impasa Pribut. Um, it, you know, when I talked earlier about the long-term strategy for the company, it is to become a leader in immunology. And so this is our next medicine, C2 as the target and MMN as the first indication. And when you look at MMN, I think it's a very Argenix-like indication. Very clear biology rationale for C2 with MMN. You know, a really strong development path that we can unpack a little bit more in terms of a head-to-head versus IVIG based on very strong phase two data and importantly, a very high unmet need. I mean, it's another place that there hasn't been innovation for decades. And in MMM, it's a progressive disease. IVIG is the only option and patients continue to progress even with very high doses of IVIG. So this is a great first indication, but impassive rubate is a pipeline in a product. So we see more indications coming. And, you know, one of them that we've announced is obviously CIDP, but there's even more beyond that.

Tizina Maad, Analyst — Bank of America

Can you maybe just talk to us about the trial design for MMN and what we should be looking at in particular?

Karen Massey, CEO

Yeah, absolutely. So the trial design is a head-to-head versus IVIG. And the reason for that is because we have such confidence based on the phase two data that we can design a phase two study, sorry, a phase three study, non-inferiority versus IVIG. The primary, it's a 24-week endpoint, and the primary endpoint is actually is on grip strength. And that's important because what we've learned in CIDP is that grip strength is a really meaningful endpoint to prescribers and to patients, mostly because it's very easy to understand. I mean, if you're losing the ability, you know, losing nerve function, and you can't, you know, pick up your coffee cup in the morning, if you can, you can understand as a patient, as a layperson, oh, my grip strength is improving. And what we actually saw in the phase two study is grip strength was able to improve over time, not just slowing the decline. So it's an important endpoint that's meaningful to doctors, understandable to patients, and I think really shows functional improvement potential for impassive rubat.

Tizina Maad, Analyst — Bank of America

And then what about quality of life? Like maybe talk to us about, you know, what patients feel they need to have that they don't have right now.

Karen Massey, CEO

Yeah, I mean, this is another indication where at the very simplest level, a patient's immune system is attacking their nervous system. So they lose the ability to use their hands, to walk, to, you know, they lose strength. So from a quality of life perspective, grip strength is a good endpoint. We also have an endpoint, MMM rods, which is more holistically looking at various quality of life measures to be able to assess the impact on the disease. One of the most important data points from phase two that we saw was when you asked patients to compare how they felt on impassal prubart versus at their peak on IVIG, nine out of 10 patients said they felt better on impassal prubart. So there's a lot of secondary endpoints, also more of those qualitative quality of life outcomes.

Tizina Maad, Analyst — Bank of America

And which of those quality of life measures are you looking at? or will you be looking at?

Karen Massey, CEO

We have a very long list of secondary endpoints that look at physician-rated quality of life, patient-rated quality of life, M&M and Rods, as I say. So it's a deep study.

Tizina Maad, Analyst — Bank of America

And then maybe just one more question on mechanism. C2 versus, let's say, C1. On the complement cascade, some might think, are those really that different from each other? But on the work that Argenix has done, maybe explain why C2 might make more sense.

Karen Massey, CEO

Yeah, absolutely. I mean, whenever we look at and identify targets for our genetics, we always like to work with world-leading experts to really understand what's the best target from an efficacy and safety perspective that's going to be able to open up this pipeline in a product. And for us, C2 is that in the complement cascade. And why is that? First of all, it's at the intersection between the classic and the lectin pathway. So you're able to capture indications that are driven by both of those pathways while leaving the alternate pathway free, if you will, which has an important safety benefit. You can still mount an immune response to a bacterial infection. But there's also the other reason for C2, in addition to having the indications opened up for the classic and lectin pathway, is that we think that it's the safest place to intervene in that complement cascade. and we've seen that with the strong safety and the data that we've generated in phase two as well.

Tizina Maad, Analyst — Bank of America

Yeah. So you've focused in on safety, which was going to be my next question. I guess in particular, is there any reason to think baseline assumption that, you know, any kind of black box warning would be part of what a label could theoretically look like?

Karen Massey, CEO

Yeah. I mean, with complement inhibitors, obviously there's always the consideration of a black box warning around vaccinations and considerations. So in our clinical trials, yes, we do run the clinical trials with vaccination, but we're also working very hard and developing data packages alongside that to demonstrate that you can still mount that immune response so that we can start to work towards. And I think we're relatively confident that we should be able to get to a good place in terms of the vaccination requirement.

Tizina Maad, Analyst — Bank of America

Okay. And then same question as before, what to expect at the top line level disclosure?

Karen Massey, CEO

Yeah, I mean, here, again, we will be as transparent as we can. And we'll balance that need for making sure that we can, especially in this indication, I mean, we are first in class in this indication, first innovation in decades, you can imagine how much excitement there will be in the medical community. And so we want to make sure that we get a good, are able to have a good presence at Congress, as well as share the top line results when we get them. So we'll be as transparent as we can.

Tizina Maad, Analyst — Bank of America

Okay. And what is the specialty that treats MMM? It's neurology.

Karen Massey, CEO

So it's also a strong overlap with our current footprint.

Tizina Maad, Analyst — Bank of America

Okay. So maybe in the couple of minutes we have left, I did want to touch upon IGANS because you will be beginning a program for 1-2-1. And I think some people find it interesting that the company wants to invest time and money into this, just considering the view that it's becoming a quickly crowded space given the the april and april baths that um have launched will be launching so just quickly mechanistically why do you think there is still room for a new um entrant like this yeah absolutely so um two things one uh when we looked at mg uh whenever it was and i remember i was like uh when others looked at mg they also felt like it was a competitive space and there wasn't enough room and look how much that's transformed and so i think there's plenty of space in IGAN if we do the same thing as we have in MG, which is provide transformative outcomes.

Karen Massey, CEO

And I think we have the opportunity to do that because our IGA sweeper, it's differentiated because it targets only IGA and it drops them rapidly and sustained. And if you think of kind of this idea of time is nephron, then we think we can provide more rapid efficacy and that's going to be important for these patients.

Tizina Maad, Analyst — Bank of America

And what about on safety? There's some questions about risk of infection for the April BAFs. And just theoretically, what do you think that profile could look like for 1-2-1?

Karen Massey, CEO

Yeah, I mean, we'll have to see it play out. But, I mean, you have people that are walking around and, from a genetic perspective, don't have IgA and there's no risk infection. So we think actually targeting IGA is going to actually produce a good safety profile and will allow us to compete there.

Tizina Maad, Analyst — Bank of America

What is the status of the program? It's enrolling?

Karen Massey, CEO

No, we're starting the program shortly. So not necessarily data this year then?

Tizina Maad, Analyst — Bank of America

No data this year. Okay, great. So with that, we're just about out of time. So I'll say thank you for making the trip all the way to Las Vegas. I know you travel a lot, so I'm not even sure where you might have traveled from to get here, but thank you. And thanks, everybody, for sitting and listening to the session.

Karen Massey, CEO

Thank you.