argenx SE

Welcome, everyone. I'm Allie Bratzel, one of the biotech analysts here at Piper Sandler. And it is my pleasure to introduce Argenix. So joining us today, we have CFO Karl Gubitz. The format of this is going to be fireside chat, but first I'm going to let Karl give us kind of an intro to the company, what's going on, and what to look forward to. So Karl, go ahead.

Thank you, Allie. It's a pleasure to be here. Thank you for the opportunity. At Arganix, I think things are going really well. We continue to execute for our shareholders. A few years ago, we laid out our vision for 2025. Happy to report that we've achieved all of those objectives. So that is why a few months ago at our R&D day, we laid out our new vision for 2030. And the headline of that new vision is 5, 10, 50. Starting with 50, 50,000 patients by 2030. I think that's a really ambitious but an achievable target, creating drugs with a differentiated outcome for 50,000 patients. Ten, ten is ten new indications on label, or ten indications on label by 2030, and five is five products, new products under development. I think that's a very ambitious plan and also at the R&D day we showed you some of our science. We showed some of our scientists were given the opportunity to present the science, which I think was all very exciting. To let investors see a little bit under the hood of the company. We also had the Q3 financial results. 573 million of product net sales. Really good quarter, very satisfied with that quarter, but also included the first quarter of our CIDP launch, which is our second indication, and I'm sure we'll talk about that a little bit later. And the last update maybe I'll give is on myositis. We gave you the results of a myositis phase two study. As a little bit of background, it was a seamless phase two, phase three for three subsets of myositis. And when we fully recruited the phase two part of it, we immediately started enrolling the phase three part of that study. And we announced the go, or continue if you like, for all three subsets of myositis. So that was also really exciting news. As we look forward to the end of the year and Christmas, we have no further catalyst to report. I think we're done for the year, but we will, of course, continue to execute for our shareholders on both the commercial front and on the clinical side. Thank you, Ali.

Great. So I think we'll start out digging in a little bit on myositis. You know, we didn't get a ton of data in the top line other than the study minutes endpoint showing a... improvement I mean total improvement score on all six core measures I guess can you kind of talk to what you viewed as the goal hurdle you had in place or what would what do you view as a clinically meaningful benefit in myositis yeah because it's a seamless phase two phase three we cannot and we don't want or we shouldn't from a regulatory point of view and from a competitive point of view

provide you with a lot of data, but what we've seen is a consistent and statistical significant treatment result for patients across all three subtypes using the TIS total improvement score at week 24. Yes, all six parameters of a TIS trended in the right direction were also statistically significant. We get a question a lot, what do we mean statistically significant? We don't share our own data, but we can point you to another study, a president's study, where 20-point difference between the active arm and the placebo was deemed approvable statistically significant by the FDA. As we now look forward, we are recruiting in all three subsets. of myositis, recruitments are going well, and I think that speaks to the unmet need in myositis. We will probably at a later date be more specific in when you can expect results. We're not gonna, it of course depends on how quickly we can recruit patients. In terms of a commercial opportunity across all three subsets, The way I like to look at it is that I think everybody is now very familiar with MG, Myasthenia Gravis, as an opportunity. And across all three subsets, in terms of the size of opportunity, if investors and analysts ask me how would I quantify that, I will, I mean, in simplistic terms, I would just say, think of it as an MG-like opportunity.

Excellent. So like you mentioned at the beginning, a big focus right now for investors is progress on the CIDP launch. I guess any color on what you're seeing in the field now that we're a little bit over halfway through the fourth quarter? And any thoughts, you know, as we're kind of in the midst here of the holiday season, any thoughts on potential seasonal effects in Q4?

Now the launch continues to go really well. We told you at the end of Q3 that we were happy with the launch. We gave you patient numbers, just over 300 patients on treatment at the end of Q3. That's the first quarter of the launch. We continue to, of course, recruit patients and it is going well. I think one of the biggest hurdles we talked about before the launch is market access. And at the end of the quarter, we gave you the data point of 54% of commercial lives or covered lives, commercial covered lives, had payer policies in place and a higher percentage for the government channels. We continue to make progress on that. And I think that really speaks to the value proposition of VIVCard. Payers do understand and accept it. And one of the data points which really resonates with payers is the regain of function. You'll remember from that year's study, over half of the patients who entered the study in wheelchairs walked out of the study. And I think that talks to our message that VivCard is not just a more convenient IG. So I think on the payer front, it's going really well. Our strategies in terms of a commercial interactions with healthcare professionals. We've got a very experienced, very capable team. They were all hired at the beginning of, we expanded the team. The expansion were all hired in Q1 of this year. So those colleagues are in the field on a daily basis and interacting with HCPs. So the launch continues to go really well. In terms of seasonality, I think that Of course, looking forward to Q1, yes, we will see seasonality again, driven by the re-verification of benefits. That's a payer environment in the U.S. I think you see that in all drugs. For December, we do have Thanksgiving. We do have Christmas. That has a little bit of an impact. And also, if you have big winter storms, it does have an impact. More so on the MG business, because the bulk of that is still IV. And if you lose infusion capacity, capability, the logistics in replacing that, of course, is complicated and you typically lose those sales. So I think those are the things we just need to watch out for.

Excellent. Maybe a question on IVIG. You know, I know that's standard of care and there are a number of IG players out there. Can you talk to what you're seeing in terms of counter detailing or, you know, defense of that, the IV or sub-QIG market from those players in the field? And just, you know, is that message resonating with physicians and patients? And just how is that, you know, a barrier for VivGuard uptake and CIDP?

Now, the IG companies, of course, they will and they do speak for themselves on their value proposition. They talk about their safety record, their decades of being in the market for decades. I think we will want to take it back to our value proposition, starting with the efficacy of a drug, talked about the regain of function. We ran the biggest study ever in CIDP The safety and tolerability of VivCard is there for everybody to see. Over 8,000 patient years now of safety data. And we continue to innovate on the patient experience side. We've got a very convenient sub-Q injection, 30 to 90 seconds. In the U.S., it's still HCP administered. Outside of the U.S., it's self-administered. We are waiting for the PFS. We have a PDUFA date, pre-filled syringe. The PDUFA date is in April, subject to the FDA. We're hoping to get that approved and then also have self-administration in the U.S. So I think our value proposition of a drug is very strong. The reality, of course, is when you launch into a new indication, you typically get the more relapsed refractory patients. That is why we talk about the 12,000 patients in the U.S. which are not adequately treated today with standard of care, largely, of course, IG, but also steroids. I think those are the patients we're focusing on today. Those are the patients we're getting today. And over time, we are hoping to move earlier lines, similar to what you've seen with MG. When we launched MG, most of our patients were refractory. And now today, most of our patients are coming from the orals, steroids, three years into the launch. And our thinking is that with CIDP, you will probably see a similar dynamic, but will be clearly not varied.

I know prior to the launch, a big question I was getting was whether there would be a warehousing of patients or bolus of patients coming on therapy. Didn't sound like you saw one in the first quarter of launch. Is that still the case? I think you actually talked about linear uptake in the first quarter launch. Is that something you're still seeing, or just any color there would help us out?

We didn't see a bolus, of course, and we also didn't have patients from the study waiting to become commercial patients because we put the open label extension, and of course we ran global studies. There are not that many patients in the U.S. So no bolus, The patient ads, the scripts, think of the top of the funnel, were consistent and linear every month. Most of the patients actually got on treatment in September because it takes time for those scripts to be converted into patients getting their drug. Now into Q4, the second quarter, we continue to be happy with what we see in the field. adding patients at the top of the funnel, those patients being converted and getting treatment. We continue to make progress on the payer front. As I said earlier, 54% of commercial lives were covered at the end of Q3. Today, that percentage is higher. We continue to add payers, and we're still on track to have broad coverage by the end of this quarter, Q4. So I think the launch continues to go well, but we do also just want to point out cautionary aspects. One of them is utilization. You know the study was done weekly. In the study utilization is done weekly. Injections are done weekly. I think in the real world that will be less than weekly. We know that from the open label extension where patients can dose every second week or every third week. We know that from industry analogs. We also know that physicians who treat CIDP patients, a vast majority of them, over 70%, also have CIDP, sorry, MG patients, and with those patients, they are used to individualized dosing, tapering off patients. So I think utilization question is still out there. The discontinuation question is still out there in stage A of a study. two-thirds of patients, 67% of patients responded. We don't know what the discontinuation or responder rate will be in the real world. We're waiting for that. We talked about the competitive pressures from the IG companies. So those are all headwinds on top of CIDP being a sticky disease, it's a switch. Patients are typically on IG, and they need to switch away from a drug to another drug. That also, of course, is something which we need to work through. But there are also tailwinds, and some of those tailwinds are that, as I already said, the physicians know the drug, they have experience with the drug for MG, the drug is now established, safety profile is established, the market access, which we've got is really good. So we are headwinds and tailwinds, and I think we need to see how that all balances out, but we continue to be encouraged by what we see in the field.

And to your point about this being a switch market, can you just talk to the level of patient-driven demand for VivGuard and CIDP, and is this primarily driven by the doc or the patients themselves? And any qualitative way to kind of talk to the effectiveness of your DTC or your awareness campaign and just any role that continued DTC could play going forward.

Thank you. I'll talk about DTC in a moment. Maybe first. in terms of treatment options, this is the first new innovation into CIDP for like 30 years. So I think there's an unmet need out there. Clearly there's an unmet need. As I already said, about 12,000 patients which are not adequately treated. So I think there's a big opportunity, and I think that opportunity is recognized by the payers. That's why we fairly quickly is getting broad access It's recognized by the physicians, and it's recognized by the patients who have been waiting for new innovation. Of course, it takes time to establish ourselves in terms of DTC, patient activation strategies. We, of course, do a lot of interaction with the patient associations, working with them pre-launch. We had an unbranded TV campaign on disease awareness. We have a website, it's called Shining Through, where patients, creating a patient community for patients to interact. Post-launch, we are not currently on television for CIDP. You might see VivCard ads on television, but it's all for MG, Maristina Graves. We might get back to CIDP DTC at a later date. We're still evaluating. But patient activation, getting the patient to ask for VivGuard to demand more, is something which worked really well for MG, and that's something which we aim to replicate for CIDP.

Excellent. So, yeah, maybe shifting over to MG, you know, I think earlier this year you outlined plans to build out VivGuard's body of clinical care. data to include zero negative in ocular patients. Seems to me that the ocular focus and in particular can really well can cement or even accelerate you know Vivgard's positioning as the early line biologic therapy. I guess is that the rationale or just kind of discuss discuss the thinking there.

I think as VivGuard is establishing itself as the first biologic. I think from an efficacy point of view, the MSE data, minimum symptom expression, getting patients to walk around symptom-free, I think we get 50% of patients in MSE. I haven't seen a competitor getting patients to that level. But there are landing spots for competitors, for example, seronegative. You do have competitors which have that in the label. We don't. So I think it is also important to make VivCard available for those patients. In some territories, for example, Japan, zero negative is on label, and we hear very good feedback. So I think it's only the right thing to do for patients to continue to have programs to help them so they can also access the drugs. And then just at the higher level, the MG launch has been really successful. Consistent growth quarter after quarter. What's driving that launch, yes, is moving into the earlier treatment lines. As I said, most of our patients are now just after the orals. It is continuing to innovate in terms of presentations. We have a sub-Q, the pre-filter ranges around the corner. But we also need other innovation to maintain that growth. And part of that other innovation is, of course, these label expansion studies. And seronegative is an important part of that. It enlarges the total addressable patient population by 15, 1.5%. And then we also want to do it with ocular, which we'll think of it as another enlargement of a patient population, again by another 15%. And I think those are the kind of innovation which will help to maintain the consistent growth we've seen for Vivgard in myasthenia gravis.

Excellent. Maybe on that note, looking forward to next spring when the autoinjector is expected to be approved for self-administration, can you talk to your expectations on what that does to the addressable patient population or the push into earlier line MG patients? Would you anticipate that, the switching dynamic from IV or from Hytrulo to the PFS, you know, for MG and CIDP? Just help us understand what that dynamic could look like.

So remember, in April next year is the BDUFA date for the pre-filled syringe, which we hope would be self-administration. It's not yet for auto-injector. We are working on an auto-injector. It is coming, but that is not next year. Next year is the pre-filterage. In terms of, I think, what we've seen with iTrulo, remember iTrulo is the current version of the sub-Q, is that in the beginning of the launch, it tended to be new patients and new physicians. Now, a few months into the launch, you do see switches from IVs. But what's important is that it opened up new patient segments. And I think with PFS you're going to see a similar dynamic. There are patients out there who are, think of younger patients, active patients, patients who have full-time jobs, take care of their families, who doesn't have a time to go to the infusion centers or hospital or doctors to get these injections or treatment options. If you can have a drug which is self-administered, which you get from a specialty pharmacy, which is literally in your fridge, and you can just take it out and administer it yourself when you need it, whether it's in a fixed dose regimen or individualized dosing regimen, we think it will add more patients. And I think it will add more patients, more physicians. It's going to grow the market. You will also have switches. So I think you're going to see both. And maybe on that point, the other aspect which is really important is that self-administration would be a pharmacy benefit. And for us, having VivCard through a medical benefit and a pharmacy benefit, part B and part D, creating that optionality for patients, for physicians who might prefer to see their patients, also because it might impact their office dynamics in terms of doing buy and pull, and also the payer aspects of either a Part B drug or a Part D for Delta drug. Creating that optionality is something which is unique to Organics and our value proposition in terms of different presentations. And I think that's also something the state should be aware of.

Just on NextGen FGAR, you provided a little bit of color on that this past summer. I think as an IV format. Can you just help us understand what kind of communication we can expect around that asset in coming quarters? And to your point about optionality in dosing formats, would you expect to follow a similar strategy for 213?

Yeah, I mean, just on a high level, we believe we are the FCRN leaders, so you shouldn't be surprised that we have a second FCRN program. We've been busy with it for a while now. We talked about it at the R&D day, but that's also in response to questions which we're getting because Analysts were looking at the IP data and they could see the patent filings. So they asked, what are you filing? What's this? The 213, which we showed you, the commercial organization asked the scientists, can you build an FCRN where we only dose once a month? If you can dose even less, even better, but it would be meaningful from a commercial point of view If we can think of it as the same drug, but just once a month dosing. Can you build it for us, please? And that is the drug which we've showed you, 213. We've only showed you some of the data points. We've shown you IV, but of course, clearly, we also need sub-Q, and all of that is in the works. And I think that is really important if you want to build a franchise. and you really want to make this a big value proposition for Arginix and our shareholders, you need to think about what's next. The IRA clock is ticking. You need to be able to have different options in terms of pricing. You need to be able to switch your patients to a next gen at some stage. So I think that's all part of a longer-term strategic planning the company is doing.

Excellent. And it looks like with that, we are at time. So thanks, everyone.