argenx - Earnings Call - Q4 2024

Transcript

Rob Harris (Head of Quality Training)

Good morning. My name is Rob, and I will be your conference operator today. I would like to welcome everyone to the call. At this time, all lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question-and-answer session. Thank you. I'd now like to introduce Beth DelGiacco, Vice President, Corporate Communications and Investor Relations. You may begin your conference.

Beth DelGiacco (VP of Corporate Communications and Investor Relations)

Thank you. A press release was issued earlier today with our Q4 and full-year 2024 financial results and business updates. This can be found on our website along with the presentation for today's webcast. Before we begin on slide two, I'd like to remind you that forward-looking statements may be presented during this call. These may include statements about our future expectations, clinical developments, regulatory timelines, the potential success of our product candidates, financial projections, and upcoming milestones.

Actual results may differ materially from those indicated by these statements. Argenx is not under any obligation to update statements regarding the future or to conform these statements in relation to actual results unless required by law. I'm joined on the call today by Tim Van Hauwermeiren, Chief Executive Officer, Karl Gubitz, Chief Financial Officer, and Karen Massey, Chief Operating Officer. I will now turn the call over to Tim.

Tim Van Hauwermeiren (CEO)

Thank you, Beth, and welcome everyone. I'll begin on slide number three. 2024 was a phenomenal year for Argenx. We expanded our reach to over 10,000 patients globally across three approved indications and delivered significant impact to the CIDP community early into launch. We also achieved several key clinical milestones and passed the proper adjoint ranks of empasiprubart as a phase III asset following impressive MMN data, and we successfully advanced empasiprubart into larger registrational studies following go/no-go decisions in Sjögren's disease and three subsets of myositis.

Lastly, we continue to drive innovation, identifying additional novel targets and nominating four pipeline molecules. These accomplishments have laid a robust foundation for our continued momentum into 2025. As we execute across the priorities highlighted on this slide towards our vision 2030, we remain committed to maximizing the growth opportunities ahead of us in a data-driven way, always prioritizing transformational impacts for patients.

Slide number four. Let's begin with a commercial opportunity. Vyvgart has meaningfully shaped the gMG treatment landscape by setting these standards for rapid, deep, and sustained efficacy with favorable safety and without trade-offs on convenience. We look forward to continuing the growth momentum with multiple regulatory milestones and continued data generation supporting earlier use across the treatment paradigm. The prefilled syringe, which opens the door for self-administration in the U.S. is expected to be a key driver of this growth in 2025 for both MG and CIDP. It is incredible to see the strong early adoption of Vyvgart Hytrulo in CIDP patients.

Underpinning the real unmet need that still exists in bringing the first major innovation in treatment to CIDP patients in over 30 years, we not only have a significant opportunity before us to set a new treatment standard, but also an important responsibility with the long-term commitment we made to the CIDP community. We will work to continue delivering transformative impact with efgartigimod by executing on our launch strategies while generating new evidence to support physician treatment decisions and advance our understanding of the underlying biology of this complex disease. This commitment informed our decision to run the IVIG switch study soon after launch to help inform treatment decisions, especially with recognition that over 80% of patients have IVIG experience. It was also the impetus to advance our first-in-class C2 inhibitors into a registrational study in CIDP.

We are confident that we have a unique opportunity to drive meaningful impact across two molecules. Slide five. Our disciplined approach to scaling will be critical to navigating the full opportunity across our pipeline. This year, we look forward to advancing 10 phase III studies and 10 proof-of-concept studies across efgartigimod and Empasiprubart and ARGX-119. We are leading a new field of medicine in FcRn, pushing the boundaries of its potential with the broadest development plan.

Our registrational studies in myositis, CIDP, Sjögren's, and ITP are built on established proof-of-concept, with additional proof-of-concept studies in systemic sclerosis, AMR, and lupus nephritis running in parallel. Each of these is grounded in solid biology with the potential to drive meaningful impact in high unmet need areas. Our first-in-class C2 inhibitor and Empasiprubart is following the same innovation playbook as efgartigimod.

The phase II ARDA data in MMN were impressive, with 94% of patients reporting improvement on empasiprubart compared to IVIG. We see an opportunity to disrupt a blockbuster market, bringing transformative benefits to patients with MMN and now CIDP as well, which is why we are exploring both indications in registrational head-to-head studies versus IVIG. And this is just scratching the surface of the broad potential opportunity ahead for empasiprubart to bring a safe treatment option forward in diseases where the lectin and classical pathways are at play. Lastly, we look forward to the first proof-of-concept data from our third molecule, ARGX-119, in CMS. By co-creating with the world experts in MuSK biology, we have built a molecule that has the potential to alleviate diseases all marked by impaired neuromuscular synaptic function.

The CMS data in the second half of the year is the first opportunity to assess whether ARGX-119 is doing what it is supposed to do clinically, which will further help us assess the opportunity ahead in SMA and ALS. Slide six. We had a robust year of activity in our IIP, nominating four new molecules that we are now advancing into phase one development. These molecules, including a second FcRn inhibitor, ARGX-213, a sweeping IgA antibody, and an anti-IL-6, all have significant therapeutic potential across new autoimmune indications. Our IIP is an incredible productive innovation engine, and we have nearly 20 active programs in the discovery stages across many relevant disease areas that will continue to feed our future pipeline.

2025 will be our first year as a profitable company, which is an important achievement and a reflection of our commercial success, our relentless execution, and our commitment to scale innovation in a disciplined way. We have a long-term growth vision that is directly influenced by our ability to continue to innovate, to stay ahead of competition, and to leverage our financial strength by investing in novel targets and pipeline programs that will be the most impactful to patients. This is the future we are building with Vision 2030 and well beyond. I will now turn the call over to Karl to discuss our financial position in greater detail.

Karl Gubitz (CFO)

Thank you, Tim. Slide seven. The Q4 and full-year 2024 financial results are detailed in the press release of this morning. Product net sales are consistent with our pre-announcement in January at $737 million for Q4 and $2.2 billion for the full year. This brings total operating income in the Q4 to $761 million and $2.3 billion for the full year. The product net sales represents 29% quarter-over-quarter growth and 98% growth compared to the same quarter from the prior year. The product revenue breaks down by region to $649 million in the U.S., $27 million in Japan, $49 million in the rest of the world, and $12 million in product supply to Zai Lab in China. Gross to net in the U.S. continues to be around 12%.

In 2025, we expect the dynamics will change due to self-administration, resulting in an increase in gross to net, which will be offset by increased patient numbers. Next slide. Cost of sales are $73 million in Q4 and $227 million for the full year, representing a gross margin of 90%. This is consistent with prior quarters because supply chain efficiencies are offset by growth from efgartigimod, which has a higher cost due to Halozyme royalties. The combined R&D and SG&A expenses total $2 billion for the full year. This is aligned with our financial guidance for 2024. Total operating expenses in Q4 are $658 million, an increase of $83 million compared with Q3 2024. The increase is primarily due to a $61 million increase in R&D, reflecting our capital allocation strategy of investing in innovation.

This results in an operating profit for Q4 of $103 million and an operating loss for the full year of $22 million. The quarterly financial income is $39 million, and in the quarter, we incurred exchange losses of $55 million, mainly related to unrealized effects on our non-U.S. denominated cash balances. Income tax is a benefit of $688 million in Q4 and a benefit of $748 million for the full year. This is due to the recognition of a deferred tax benefit of $802 million for the full year ended December 31st, 2024, of which $725 million relates to a one-time non-recurring recognition of previously unrecognized deferred tax assets existing as of December 31st, 2023. We made the decision to recognize the deferred tax asset because of our assessment that it's probable that future taxable profits will be available.

This results in profit in Q4 of $774 million and profit for the full year of $833 million. Our cash balance, represented by cash, cash equivalents, and current financial assets, is $3.4 billion at year-end. The balance increased by $200 million in 2024. Our OpEx guidance for 2025 is approximately $2.5 billion of combined R&D and SG&A expenses. This is a 25% increase year over year and reflects our commitment to invest in our R&D engine and pipeline growth. Zooming out, we are well positioned to prioritize innovation that will support our sustainable future with our strong balance sheet and transition to profitability in 2025. I will now turn the call over to Karen, who will provide details on the commercial front.

Karen Massey (COO)

Thank you, Karl. Slide nine. I want to start by building on what both Tim and Karl have shared, which is the importance of innovation to Argenx. It's the driving force behind everything we do, and we all agree that innovation only matters if it reaches patients and provides meaningful benefit. In looking back at the Q4 and all of last year, I'm pleased with the continued momentum we saw from the team in bringing meaningful innovation to patients. Our growth was fueled by both MG and CIDP, and it's encouraging to see how Vyvgart continues to transform outcomes as we broaden and deepen our reach in the MG community, and at just two quarters into the CIDP launch, we're already raising the bar on what patients can demand from their treatment.

On today's call, I'll take a deeper dive into our performance, highlighting the drivers that will support our continued growth in patient impact. Next slide. We continue to deliver growth in the MG in the Q4, driven by consistent patient adds and new prescribers. Vyvgart Hytrulo played a key role in reaching patients earlier in the treatment paradigm and predominantly in patients brand new to Vyvgart rather than switches from IV. Some additional growth was supported by a halo effect from the CIDP launch, where we saw neurologists who first wrote scripts in CIDP now also prescribing in MG. To maintain our leadership as the number one prescribed branded biologic in MG, we are prioritizing the anticipated launch of the prefilled syringe and continuing to invest in generating new evidence, including through label extension studies.

We have a long-term strategy to enable continued adoption of Vyvgart in earlier treatment lines, and this remains our key focus. Vyvgart's consistently strong safety and efficacy profile supports this goal, particularly as physicians gain more experience with the treatment and will further build this confidence by investing in evidence generation. Real-world data efforts are underway to evaluate reduction in steroid use, safety in new patient populations, and dosing through our ADAPT NXT study, which we plan to share at upcoming medical conferences. Innovation on route of administration will also support our shift earlier in the treatment paradigm, and the prefilled syringe is a key step towards expanding our patient reach. The opportunity to self-inject at home is a significant innovation for patients and provides an added level of freedom in their treatment regimen.

We were thrilled to receive our first global approval of Vyvgart prefilled syringe this month in the EU for a gMG patient, and we're now looking ahead to the FDA PDUFA date in April for both MG and CIDP. Lastly, we see additional opportunity to address the unmet need in the MG community by expanding our label into seronegative and ocular MG. ADAPT SERON is the first of our phase III studies to read out this year, and we have ample real-world and clinical data sets supporting our ability to drive responses in this population. Ocular MG presents another exciting opportunity, and with 80% of patients progressing to generalized MG, we have the potential to introduce innovation earlier in the treatment paradigm. Next slide. We're very encouraged by the continued momentum of the Vyvgart Hytrulo launch in CIDP.

It's abundantly clear that our data are resonating across patients and physicians, with the initial demand highlighting the unmet need for safe and effective treatment alternatives. One patient shared that she had tried nearly all available CIDP treatments, and they all failed, so she was left with a heavy treatment burden of plasmapheresis every seven to 14 days. Her physician was dedicated to improving this burden and suggested a switch to Vyvgart Hytrulo. She was initially very hesitant, but after seeing the improvement, she realized she had massively underestimated the disease burden she had still been facing. Even as functional as she was, it wasn't until Vyvgart Hytrulo that she felt the enormous boulder lifted off her back. While this patient's improvement was dramatic, this is just one of the many inspiring stories from patients and their caregivers who are empowered to demand more from their treatment.

I'm incredibly proud of the team for building a solid foundation to get us off to this strong start to ultimately reach our target addressable population of 12,000 patients. Our market access team has done a phenomenal job securing broad access to support patients getting on treatment quickly. The sales force expansion further enabled us to successfully reach new prescribers deeper in the community setting, and it paid off. 25% of prescribers over the last quarter were first-time Vyvgart users.

As of year-end, we had approximately 1,000 patients on treatment, with the majority of these falling within our initial addressable population, those who are not sufficiently controlled or who experience side effects on IVIG or steroids. Most neurologists will treat patients for 12 weeks to assess the response to Vyvgart, so we look forward to gaining more insight on response rates and utilization in the coming quarters.

We're just at the beginning. Our priority this year will be to reach more patients and more prescribers and repeat the MG playbook to consistently generate data to build physician-supported confidence while also empowering patients to ask for more from their CIDP treatment. Next slide. We also look forward to additional expansion opportunities outside the U.S. this year, particularly as we plan to launch the prefilled syringe and CIDP in multiple regions. In MG, we have launched in most of the major markets ex-U.S. and expect consistent steady growth over time as market access dynamics fall into place. We're now reimbursed in 13 countries in Europe, including four out of the five major markets, and we're pleased with the recent MG approvals in South Korea and one that is personally very close to my heart, Australia.

Earlier, I highlighted the positive CHMP opinion on PFS enabling sales in the EU, and this is just the first of four regulatory decisions on approval this year. We are just at the beginning of our global CIDP launch, with additional decisions on approval expected in China, Europe, and Canada. Feedback from Japan, the first approval following the U.S. has been positive across patients and physicians in the CIDP community. Next slide. Our expansive pipeline supports our next wave of growth, and we're committed to addressing the unique challenges and gaps in treatment for autoimmune patients across multiple indications, where there is often a lack of innovation and high barriers to access. We've built a robust network of relationships in the neurology community with over 3,500 Vyvgart prescribers, which we'll leverage as we advance into our next launch wave with seronegative and ocular MG.

The impressive data from the ARDA study in MMN for empasiprubart have already drawn attention from our prescriber base, where there is a lot of overlap with CIDP and MG. I shared Tim's excitement about empasiprubart and about MMN and our opportunity to transform the treatment paradigm. We see MMN as a nascent market ripe for innovation, similar to the initial dynamics we saw with MG. Patients continue to be discouraged with the symptom burden and the lack of treatment options, so the phase III data in 2026 will be a big moment for us to potentially introduce the first precision treatment to this community. Looking ahead, we see an opportunity with efgartigimod in myositis to serve as a bridge from neurology into rheumatology with subtypes across both therapeutic areas.

We aim to leverage our neurology playbook as we expand into rheumatology, building on the traction we've already gained in the community, following our decision to advance Sjögren into a registrational study. All in all, this is an exciting time for Argenx across our entire business as we progress towards our vision 2030. Tim?

Tim Van Hauwermeiren (CEO)

Thank you, Karen. Slide 14. I want to extend my sincere appreciation to the Argenx team, including our board, for their outstanding work last year and their unwavering dedication to improving the lives of patients. We've deliberately set a very high bar, and as we move forward, it's critical that our continued innovation in the autoimmune space maintains this best-in-class standard. 2025 will be a year of significant growth for us, expanding our commercial reach, introducing new products, entering new markets, and advancing our late-stage pipeline. Importantly, we never lose sight of our mission, harnessing innovation to develop transformative medicines for the patients we serve. Thank you for your time today. I would now like to open the call to your questions.

Operator (participant)

Thank you. We will now begin the question-and-answer session. If you would like to ask a question, please press star one in your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply press star one again. We ask that you please limit yourself to one question and one follow-up. Your first question comes from Tazeen Ahmad from Bank of America. Your line is open.

Tazeen Ahmad (Managing Director)

Hi, good morning. Thanks for taking my questions. Mine are going to be on the PFS upcoming PDUFA, so maybe for Karen, can you talk about how you're seeing what pent-up demand there could be for PFS? Are there patients that have not gone on to therapy knowing that there potentially could be this more convenient option? And if you're looking for switches to occur, would that be gradual, or would that be a bolus effect that we should see upon approval? And then I have a follow-up.

Karen Massey (COO)

Yeah, thanks for the question, Tazeen Ahmad. For the interest, we're really excited for the upcoming PFS PDUFA date in April and potential approval. So here's what I would say. I don't think we're seeing pent-up demand. In fact, I'm quite pleased with the momentum we've seen on MG and CIDP in terms of new patient starts. It's pretty consistent. But what we do expect is that prefilled syringe for self-injection will open up both the prescriber base and the patients that will consider efgartigimod as an option for either MG or CIDP.

So we think what it'll enable us to do is maintain that consistent momentum and that consistent growth, which is pretty incredible as we get 13 quarters out from launch, and I think that's what the innovation allows us to do. It's not specifically a switch strategy that we're pursuing. To the second part of the question that you asked. Rather, we want to focus on expanding our reach to prescribers and to patients. Thanks for the question. I think you had a follow-up.

Tazeen Ahmad (Managing Director)

Yeah, either for you or Tim, how should we be thinking about just generally pricing for PFS? Would it be similar to the current options available, or should we expect any kind of difference?

Karl Gubitz (CFO)

Hi, Tazeen it's Karl here. Thank you for your question. Consistent with prior launches, we will provide more color on the price at the time of a launch, but let's provide some framework on how we think about it. We'll be bringing important innovation to patients, and our primary goal is to provide broad access and optionality to patients and physicians to choose on what's best for them, and of course, we will aim to price in a responsible and sustainable way for Argenx. Thank you for your question.

Operator (participant)

Your next question comes from Alex Thompson from Stifel. Your line is open.

Alex Thompson (Managing Director)

Hey, great. Thanks for taking our questions. Congrats on the quarter. I guess as a follow-up to that question for Karl, you've talked about sort of net price impact with a PFS. I guess can you talk a little bit more about potential magnitude there and also the rate of some kind of an impact like that, what you would expect to see upon the launch this year? Thanks.

Tim Van Hauwermeiren (CEO)

Thank you for the question, Alex. Yes, the dynamics for self-administration, and this is subject to FDA approval of self-administration, of course, will be different, and gross to net will increase. Currently, it's around 12% with a majority of patients currently in medical benefit Part B for bravo. With self-administration, the patients will transition to pharmacy benefit or Medicare Part D for delta.

You will then get the typical rebates for pharmacy benefit, which will be higher than what we see today. And of course, we will be subject to IRA redesign where the manufacturer, us, get 20% of the cost post-catastrophic on Medicare Part D patients. All of that will result in a higher gross to net. We, of course, expect that increased patient volumes will offset the increase in growth to net, and over time, you will also see it is likely that the VBAs will be phasing out. Thank you for your question.

Operator (participant)

Your next question comes from Derek Archila from Wells Fargo. Your line is open.

Derek Archila (Managing Director and Co-Head of Therapeutics Research)

Hey, good morning, and thanks for taking the questions. Just one from us. I guess, how should we think about the quarter-over-quarter growth cadence in MG in 2025 relative to what we saw in 2024? And then ultimately in 1Q, should we expect some seasonal impacts? Thanks.

Karen Massey (COO)

Yeah, thanks for the question, Derek. This is Karen. I'm happy to take it. So how I think about it is what I was mentioning before in MG: continued momentum and continued strong uptake in line with the strategy that we have of moving earlier lines in the treatment paradigm and broadening the prescriber base. I would expect that those underlying dynamics will continue. Specific to your question on Q1, look, Q1 seasonality is an effect that is seen across the industry. And don't forget, last year we had our Q1 growth rate of 6%. So we are seeing the benefit verifications. That is an industry-wide phenomenon. So take that into consideration perhaps for Q1. But overall, what we're seeing is continued momentum, and our underlying dynamics are good.

Derek Archila (Managing Director and Co-Head of Therapeutics Research)

Thank you.

Operator (participant)

Your next question comes from Yaron Werber from TD Cowen. Your line is open.

Yaron Werber (Managing Director and Senior Research Analyst)

Great. Maybe a couple of questions. One for Karl: when the VBAs expire because of Part D, how does that work? I assume access stays the same, or do you need to renegotiate? And then for Karen, for the first 12 weeks on CIDP, are physicians actually doing INCATs? And are they actually looking at—are they actually assessing formally whether patients are improving, or I imagine they're just keeping them on therapy, and if they're really not benefiting, they'll take them off? Thank you.

Karl Gubitz (CFO)

Yaron, thank you for your question. On the VBAs, with PFS, of course, we will have to go back, and the payer agreements will have to be established. We've done that now a couple of times, and we need to do it again, and as it is a pharmacy benefit, our expectation is that VBAs will not be part of those contracts, and so therefore it will phase out over time. Thank you for your question.

Karen Massey (COO)

Thanks, Karl. Yeah, on the question of so we are seeing with CIDP that generally doctors are giving more like a 12-week trial, as you mentioned. We're not hearing about a lot of use of INCAT and those types of scales. They're more used in clinical trials than in clinical practice. So more generally, the doctors will have a conversation with their patient. They're using more simple assessments to determine how their disease is being controlled by efgartigimod, and then making an assessment on response.

Operator (participant)

Your next question comes from Victor Floc'h from BNP Paribas. Your line is open.

Victor Floc'h (Equity Research)

Hi, thanks so much for taking my question, Victor Floc'h from BNP Paribas. So my first question relates to your 2025 news flow, which is broadly seen, I guess, as lighter than what you're expecting for 2026. And I mean, it's fair to say that 2026 is going to be quite strong. But in the meantime, would you say that investors are overlooking the potential for the seronegative MG trial to significantly expand MG addressable opportunity as well as PFS ability to further drive your line penetration in both MG and CIDP?

And my follow-up on the PFS is I'm just wondering if you could help us understand how important self-administration has been for Vyvgart Hytrulo's penetration ex-US. I know you don't report any sales breakdown and that there are structural differences between U.S. market and ex-US, but just trying to understand how self-administration on label in U.S. could meaningfully drive sales. Thanks so much.

Tim Van Hauwermeiren (CEO)

Yeah, Victor, thanks, and great to hear from you on the call. You're right to call out that we do have self-administration already for Vyvgart Hytrulo outside of the United States. And I will let Karl comment on why that is important for physicians and patients. On news flow, this is an incredibly busy year from an execution point of view. I mean, pushing 10 phase III clinical trials and 10 phase II clinical trials forward at this speed, I think, is a very serious task, but in the first half of the year, I think PFS is important, with hopefully self-administration, as Karen will explain in a minute. And then you're correct to call out the significance of hopefully a positive readout in seronegative MG patients. There's a significant volume of patients out there in high medical need.

We have seen in Japan how important the patient population actually is and how successful Vyvgart actually is in treating these patients, so that could be a very important force or driver in maintaining the momentum we were alluding to, and then I would also like to call out an important proof of concept study in lupus nephritis in the second half of this year, so Karen, you want to continue with the importance of self-administration?

Karen Massey (COO)

Yeah, absolutely. And just to reinforce, we do have self-administration on label in both the EU as well as in Japan. And what we see in those markets is that when patients and physicians have a choice between the IV option and the self-administration subcutaneous option, not all patients move to the self-administration. There are some patients that choose to stay with IV. That's what fits with their lifestyle. Perhaps they prefer not to do the self-administration because of needle phobia or something like that. But a good percentage of patients do have the preference, and it fits into their lifestyle to take the self-administration option.

So what we see is that there's real benefit to both patients and prescribers to having multiple routes of administration so that the patient and the neurologist can really make a decision together about what makes most sense for that patient based on their disease and their lifestyle. And so we think that that same experience will come true in the U.S. if we get approval for self-injection on April 10. Thanks for the question.

Operator (participant)

Your next question comes from Akash Tewari from Jefferies. Your line is open.

Amy Li (SVP of Equity Research)

Hey, this is Amy for Akash. Thanks so much for taking your questions. Starting with the myositis trial, are you planning on making any changes to the trial design after your go or no-go decision? And how are you thinking about the probability of success across each of the subsets now? And then finally, wanted to get your thoughts on the obinutuzumab data and how that reads across to your lupus nephritis trial.

Tim Van Hauwermeiren (CEO)

So in myositis, we announced when we made the go or no-go decision to go forward in all three of the subtypes with no changes to the protocol. You're correct to call out that we have some degrees of freedom to make these changes, but based on the phase II data, we did not see any need to make changes. And you can assume that because we gave the go-ahead for all three subsets, that there's an equal belief in all three subsets for success in phase III. Look, in lupus nephritis, we see a number of mechanisms being tested.

Whatever the data which we see, I think there's ample room for improvement. There's a significant unmet medical need. There's a need for a toolbox to treat these patients, and I don't think there's going to be one-size-fits-all. So let's focus on our data. We have strong conviction in the biology, and we're testing that mechanism of action now in phase II, so let the data speak, but Amy, thank you for the questions.

Operator (participant)

Due to time constraints, we ask that you please limit yourself to one question only. Your next question comes from Rajan Sharma from Goldman Sachs. Your line is open.

Rajan Sharma (Executive Director of Pharma and Biotech Equity Research)

Hi. Thanks for taking my question. I'll keep it to just one. So just to understand some of the underlying dynamics in myasthenia gravis. So we know that back at launch of Vyvgart, you said that the addressable market was about 17,000 patients, and you think there's an additional 25,000 that will come from growth in the biologics market by 2030. So I'd just be interested, back in 2021, when you initially saw that 17,000 market, how do you think that biologic share has progressed now, i.e., how much through that additional 25,000 are we already? Thank you.

Karen Massey (COO)

Yeah. Thanks for the question around MG. And certainly, as we lay out our strategy for MG, we think that we are very much on the beginning still of the growth curve. And you talked about the market expansion that we're already seeing from the 17,000 since the launch of Vyvgart. So we do think there has been market expansion, and the way that we see that is that since launch, we're no longer just being used or advanced biologics are no longer just being used in the most refractory patients. Rather, the advanced biologics are being used earlier line, and Vyvgart is, let's say, leading the charge on that as the number one prescribed biologic.

We shared the statistic that 60% of patients are actually coming directly to Vyvgart from orals. That demonstrates that we're starting to penetrate that 25,000, but we believe that there's quite a long way to go and that we can maintain that consistent and steady growth. Certainly, prefilling for self-injection will help us with that. Thanks for the question.

Operator (participant)

Your next question comes from the line of Suzanne van Voorthuizen from Kempen. Your line is open.

Suzanne van Voorthuizen (Head of Life Sciences Research)

Hi, team. This is Suzanne from Kempen. Thanks for taking my question. I was wondering if you can elaborate a bit on your longer-term thinking of the FcRn franchise. I guess you're getting to the point where investing in more phase III trials for efgartigimod may not always make sense, and you perhaps look towards the next generation ARGX-213. So on 213's clinical development, do you see possibilities for shorter development timelines? What are elements that you can leverage from having developed efgartigimod? Thank you.

Tim Van Hauwermeiren (CEO)

Yeah, Suzanne, I think you're right in calling out that [FcRn] is going to be a franchise. It's an incredible opportunity. I think the size of the opportunity exceeds the ability to serve that with one molecule given the patent life and the IRA life of a molecule like efgartigimod. So I think developing a molecule like ARGX-213 gives us optionality, and we said this is the first of successful molecules. It basically unfolds optionality. I mean, either you will go and try to roll up existing indications, and then you're spot on. I mean, there's so much we know about MG.

There's so much we know about CIDP, ITP, and the other indications to come that actually you could leverage that know-how and leapfrog with molecules like 213, or you basically decide to go into open up new opportunity where you have, for example, a whole new game plan from a positioning and pricing point of view. So that optionality is in front of us, and we will basically unpack it while we go based on data. We're really focused now on generating the phase I data second half of this year, which will tell us a lot about the potential of this molecule going forward. Thanks for the question.

Operator (participant)

Your next question comes from the line of Vikram Purohit from Morgan Stanley. Your line is open.

Vikram Purohit (Research Analyst)

Hi, good morning. Thank you for taking our question. We'll keep our question focused on the auto injector. I believe your release mentioned that you'll be looking to move this forward in 2027. So we just wanted to see what the next update could be for the auto injector and how you're thinking about how this form of efgartigimod potentially could expand the opportunity beyond the IV, the subsc, and the PFS. Thanks.

Tim Van Hauwermeiren (CEO)

Yeah, thank you. Vikram, the significance of an auto injector or the differentiation of the auto injector from a prefilled syringe is actually having a device where the needle is invisible and does not need to be manipulated by the patient. So basically, now the only job left is to hold the device against your belly. You press the button, and then the device is manipulating the needle in a way that you don't see it. And that is significant for a subset of patients. So in an attempt to continue to innovate in our core market, this is a logical next step building from the prefilled syringe. From this moment, actually, we've completely focused in our communication on the prefilled syringe.

It is a very innovative product thanks to the technologies we leverage in the formulation and the technologies we leverage for the container. It's significant innovation, and I think a significant driver for the business. Stay tuned on the auto injector. We're working very hard on that in the background, but it's too early to commit to a next specific update. Okay? So 2027 is the year we're all aiming for, and I would say stay tuned. Thank you.

Operator (participant)

Your next question comes from the line of Andy Chen from Wolfe Research. Your line is open.

Alina Cellura (SVP of Research Management)

Hi, this is Alina for Andy. Thanks for taking our question. A question on CIDP and maybe just some metrics you're seeing early on in its launch. It was mentioned the majority of patients are IVIG experienced. Is IVIG fixated in early lines? Are there signs of early standard of care transformation, maybe docs preferring efgartigimod over IVIG? Just any insight would be great. Thank you.

Karen Massey (COO)

Yeah, thanks for the question about CIDP launch. As I shared, I'm really pleased with where we're at with the launch. We're clearly seeing early success, and there is a clear unmet need that you can see, and what we consistently hear, the feedback from the community, whether it be neurologists, patients, caregivers, is that there is a higher treatment burden and a higher disease burden than they had realized, so bringing the first innovation in 30 years is making a big difference, and I do think, to your question, over time, we will transform this market and reshape what is standard of care. Right now, we're very early in the launch. We're two quarters in. I think we're doing very well. 85% of our patients are switched from IVIG, and that's exactly where we thought we would be, so we're pleased with the momentum so far.

Operator (participant)

Your next question comes from Yatin Suneja from Guggenheim. Your line is open.

Hi, this is Selma for Yatin. Thanks for taking our question. So for the seronegative ocular MG trials, what's the dosing regimen in those studies? And based on the off-label use of Vyvgart in this population, would you anticipate that these patients will need a similar number of unmarked cycles as seropositive gMG, or will they need more frequent dosing? And finally, how should we think about pricing in these indications? Thank you.

Tim Van Hauwermeiren (CEO)

Yeah, I think the answer to this question, thank you for the question, is very simple. We don't design, and we don't anticipate any difference dosing in seronegative or ocular MG patients as compared to the generalized MG patients, which are acetylcholine receptor antibody positive, which we have currently on label in the States. And we also don't anticipate any real pricing difference. The idea is that this is label expanding and is just broadening the offering to the MG community. So you can assume for the models exactly the same parameters. Thank you for the question.

Operator (participant)

Your next question comes from the line of Samantha Semenkow from Citi. Your line is open. I apologize. We lost the connection. Your next question comes from the line of Matt Phipps from William Blair. Your line is open.

Matt Phipps (Group Head of Biotechnology)

Hi, thanks for taking my question. I wanted to follow up on the myositis transition to phase III, and just wondering if you're enrolling a set number of patients for each of the three subsets so as to power each subset individually, or will the primary endpoint be looked across all patients? Thank you.

Tim Van Hauwermeiren (CEO)

Yeah, Matt, I think you're spot on. You will want to see a minimum representation of every subset in that phase III registration trial in order to draw data-based conclusions. You can then also discuss with the FDA. So I think your underlying assumption is correct. Thank you.

Operator (participant)

Your next question comes from the line of Gavin Clark-Gartner from Evercore ISI. Your line is open.

Gavin Clark-Gartner (Managing Director of Biotechnology Equity Research)

Hey, guys. For the prefilled syringe and the VBAs with the managed care organizations phasing out, I just wanted to clarify. In CIDP specifically, are you planning to not have the same cap on use in those contracts?

Tim Van Hauwermeiren (CEO)

So I think, Gavin, it's all here. I think these are price negotiations, which still needs to take place for the PFS. But our expectation is that for pharmacy benefit, that you would give incremental discounts because that's what you typically do, but incremental base rebates, and that the VBAs would not be asked for by the payers. So therefore, it will phase out. So therefore, yeah, there won't be caps. Thank you for your question.

Operator (participant)

Your next question comes from the line of Samantha Semenkow from Citi. Your line is open.

Samantha Semenkow (VP of SMid Biotech and Equity Research Analyst)

Hi, good morning. Are you able to hear me?

Tim Van Hauwermeiren (CEO)

Yes, we can hear you.

Samantha Semenkow (VP of SMid Biotech and Equity Research Analyst)

Perfect. Thanks very much for taking the question. I'm wondering, can you speak to your confidence in FDA granting the self-administration for the PFS? And perhaps you can just remind us on FDA's concerns surrounding self-administration for the butterfly needle process and how that's factored into your strategy as you look to secure self-administration for the PFS. Thank you.

Tim Van Hauwermeiren (CEO)

Yeah, so taking a step back, we feel we submitted a very strong dataset to the FDA with regards to the prefilled syringe, including, I think, a very solid human factor study, which is taking into account the typical questions a regulator may have on how patients can reliably and robustly manipulate the device. So we feel we're starting from a very strong dataset. I think the review is on track based on.

We think we're on track for the date of April 10. And I think the type of questions which we're getting from the FDA signal that actually we're making good progress with the review of the file and that actually we are where we should be in light of the total review timeline. So we are confident based on the dataset and the currently ongoing process. Ultimately, of course, this is the final call to be made by the FDA, and we try to collaborate as much as we can. Thank you for the question.

Operator (participant)

Your next question comes from the line of Thomas Smith from Leerink Partners. Your line is open.

Thomas Smith (Senior Research Analyst)

Hey, guys. Good morning. Thanks for taking our questions. Tim, you called out LN as an important proof of concept readout in the second half of this year. I know that's a study that's being conducted by your partners, Zai in China, and they just recently completed enrollment. I was just wondering if you have a sense of sort of the baseline characteristics and disease severity of these patients and how they compare to other contemporary Western LN studies, and maybe you could just remind us how you're thinking about the bar for success with this readout. Thanks.

Tim Van Hauwermeiren (CEO)

Yeah, Thomas, thank you for the question. First thing I want to do is applaud Zai as a reliable and high-quality development partner that are not only involved in these phase II proof of concept studies, but are also involved in a lot of the phase III global registration trials with speed and high quality. So a very strong partnership. And specifically to LN, the patient population which we are recruiting in China is, of course, perfectly in line with protocol and the inclusion-exclusion criteria, which is representative, I think, for that LN patient population with significant unmet medical needs. There are some variations in the type of treatments these patients undergo. But for us, this phase II, and it's a true phase II, is a proof of concept.

The first question we need to answer in this phase II trial is, are we spot on with this mechanism of action? When we dramatically reduce pathogenic IgGs, do we have the right to move the needle in this type of patients? That's the question we're going to answer. Once we have a positive answer to this question, we will flip it into a global phase III registration trial where we will take into account some of the global treatment regimens which are typically being used. We take it step by step. For the proof of concept question, I think Zai is perfectly equipped to help us with those questions. We're very much looking forward to the data. Thank you.

Operator (participant)

Your next question comes from the line of Leland Gershell from Oppenheimer. Your line is open.

Leland Gershell (Managing Director and Senior Analyst)

Hey, good morning. Thanks for taking our questions. Just a question on 119. As we look forward to the proof of concept data in CMS later this year, just wondering if you could touch on what you are looking to see and could this data impact the ongoing development of 119 and its other indications? Thank you.

Tim Van Hauwermeiren (CEO)

Yeah, I like the question about 119. Thank you for that. As an indication, congenital myasthenic syndrome is really in the bull's eye of the biology of this target. Remember, we have been publishing and presenting spectacular data in the DOK7 animal model, which is perfectly mimicking the DOK7 mutation in human beings. And that's exactly where we're testing for CMS. Think of a genetic form of MG, which has overlapped with autoimmune MG. It's fatigable. It's typically limb girdle, which is being affected, but also the eyes.

So we're basically looking for a strong signal that we move the needle in these patients. So I think it is a very important program from a proof of biology point of view. But remember, we're running in parallel also a proof of concept study in ALS, and we're bringing online now a study in SMA. I would say that all these three indications are very close to the mechanism of action of the molecule, but CMS, I think, is really close. So that is the significance of this first indication for ARGX-119. We're very much looking forward to the data, by the way. Thank you for the question.

Operator (participant)

Your next question comes from a line of Joon Lee from Truist Securities. Your line is open.

Mehdi Goudarzi (Biotech Equity Research)

Hi, good morning. Congrats on the quarter. This is Mehdi Goudarzi for Joon. A question related to your early pipeline asset. We appreciate more color on your plans for ARGX-109 anti-IL-6 based on the importance of the target, but also given its interesting history of about 15 years and its 200 patients phase II in RA in Brazil, and also, it's finally getting back to you. We appreciate the color there.

Tim Van Hauwermeiren (CEO)

I think you're calling out the strength of the molecule. I think this is a best-in-class IL-6 blocker with picomolar potency, a 60-80 days half-life, and already a proven safety profile in two phase ones, actually, one in Brazil and one in China. Happy to finally get this molecule back, and there's a whole new angle which we could take in the meantime on the biology of IL-6 and how it is involved in some real interesting autoimmune diseases, so we decided not to talk too much about these indications yet, but I think we have a unique and novel angle to the biology, and we're progressing the molecule this year at high speed through phase one. We're expecting the phase one data second half of this year, so stay tuned. More to be told on 109 soon. Thank you.

Operator (participant)

Your next question comes from a line of Joel Beatty from Baird. Your line is open.

Chris Chen (Research Analyst)

Good morning. Thanks for taking our question. This is Chris on for Joel. Just a quick one on PFS. If they're approved in April, what are your expectations for when they can reach patients? Thank you.

Karen Massey (COO)

Yeah, thanks for the question. So we're very excited, obviously, for the PFS for self-injection potential approval in April, as you said. We'll be ready to launch as soon as after the PDUFA date in the same way that we have for prior launches. So we expect it to be within days that we'll be out there with PFS for self-injection.

Operator (participant)

Your next question comes from a line of Douglas Tsao from H.C. Wainwright. Your line is open.

Douglas Tsao (Managing Director and Senior Healthcare Analyst)

Hi, good morning. Thanks for taking the questions. Nikki, Karen, I think it would be helpful to hear you talk a little bit about how you think potential approval on ocular MG might affect sort of the treatment paradigm for MG and whether you sort of might see that push earlier, obviously, because some of the patients present with ocular symptoms first, but sort of really whether clinicians might view it as sort of disease-modifying therapy at that stage. Thank you.

Karen Massey (COO)

Yeah, thank you for the question. And I like how you frame it up as disease-modifying. We'll have to let the data speak. But the way that I think about it is very much aligned with how you're thinking about it. Our strategy is that we believe that treatment with Vyvgart in earlier lines results in better outcomes for patients. And that's why we also wanted to move all the way into the MGFA classification 1 with ocular MG, and we would be the first and only that would have that indication.

We know that 80% of patients with ocular MG generalize. And as you say, potentially over time, not with this first data readout, but maybe over time, we can imagine that you could see data in the real world where we're able to delay that generalization. And I think that's really exciting for MG patients. Certainly, that's our vision for how we would transform the market. Let's see how the data plays out, but we're relatively confident and have really strong conviction in the strategy. Thanks for the question.

Operator (participant)

Your next question comes from a line of Manos Mastorakis from Deutsche Bank. Your line is open.

Manos Mastorakis (Pharma and Biotech Equity Research)

Hi, thank you for taking my question. Manos Mastorakis from Deutsche Bank. So how do you think about the potential for competitors to further improve upon clinical outcomes in CIDP above efficacy-based outcomes? And do you believe complement inhibitors have a role to play in this space? Thank you.

Tim Van Hauwermeiren (CEO)

Yeah, thank you for the question. I think in CIDP, we have written history because we have shown for the first time that actually this is an IgG-driven disease in the majority of patients. And I think we have shown a response rate, which is the highest ever reported in this type of clinical trial. So with a 70% response rate, a safety profile which is very much in line with the known safety profile, which we all recognize is pretty unique in this world. And then, of course, we're advancing fast with the dosing optionality, which we have been unpacking in this call. So we've put the bar very high. We need to see data, of course, from competing molecules in the class where they take it. But we have certainly not seen that in myasthenia. We were also first to market. Is there a role for complement?

I think there is. That's also why we're advancing empasiprubart in CIDP, despite the fact that we printed the highest response rate ever with 70%. There is a 30% of patients which have medical needs and were not served in the ADHERE trial. We do know there are some clues from a biology point of view, for example, some patients having these pathogenic IgM autoantibodies, which do recruit complement and which do not recycle through FcRn. I think we're unraveling the answer to your question. I think empasiprubart really deserves the shot on goal. We're very keen to start this study and look at complement data. There's a lot to be unpacked and a lot to be developed in CIDP. Thank you.

Operator (participant)

And we have reached the end of our question and answer session. This concludes today's conference call. Thank you for your participation. You may now disconnect.