argenx SE (ARGX) Earnings Call Transcript & Summary

Good afternoon, everyone. Thank you for joining. My name is Rajan Sharma. I'm part of the European Health Care Research Team at Goldman Sachs, covering Pharma & Biotech, including argenx. I'm very pleased to have Karen and Beth with us. Thank you both for joining. Maybe Karen, just to kind of hand it over to you if you want to make some introductory remarks.

Yes. Thanks for having us, and thanks for being here. We're really excited to be able to talk about argenx this afternoon. I guess maybe just the opening comments that I would make, we have a busy year this year at argenx as we do every single year. And early on in the year, we set out an ambitious plan with some clear deliverables. And right now, midway through the year, I would say we're on track with that ambitious plan. And we're happy to talk about all of those, and I know we will as we go through. One of those big catalysts for us this year is the potential CIDP approval. We have the PDUFA date coming up June 21. I think it's just 8 working days away. So I'm sure there will be some questions about that. We're really excited about that. But we also had some really important catalysts coming up in terms of data readouts through the end of the year as well. So looking forward to having the conversation.

Okay. Perfect. I guess that's a good segue, CIDP maybe we'll start there, given, as you said, kind of PDUFA is relatively imminent. So conscious that there's not a huge amount that you can say, but just if you could just talk us through your label expectations ahead of PDUFA, what would be a best case scenario, what's your base case?

Yes. No, I appreciate the question, and it's an important question. As you can imagine, so our PDUFA date is June 21. So it's imminent, and we are on track for that. And what that means is that we are in discussions as would be expected around our label. So we really don't feel comfortable sharing much or anything really on the assumptions on the label or anything like that. But we are happy to talk about if we move forward and sort of make the assumption that there is a potential approval, we can talk about launch readiness and our thoughts on the market and that type of thing.

Okay. Maybe we'll do it then. I guess the first thing, assuming the approval comes through, it's an innovative trial design that you've talked to previously. I guess everyone's kind of familiar with that trial design. How do you think that will be implemented in clinical practice?

Yes. I mean I think it is an innovative clinical trial design, and it's a meaningful clinical trial design. I mean let's take a step back and first of all, say that if approved, this will be the first new mechanism of action in CIDP in over -- in 3 decades. So it's a really important step forward for patients. I think the clinical design did a few things in terms of -- and which will reflect clinical practice. So in Stage A, what we were able to look at is -- so first of all, the patients that came into the clinical trial reflect the reality in the market today, which is, some of them were treatment naive, which means they haven't been on any therapy for the last 6 months, they might have been before that, some on corticosteroids, some on IVIg, that came into the clinical trial. And Stage A, we looked at what is the response rate? How many patients can we expect to respond, CIDP patients can we expect to respond to VYVGART. And we saw, let's call it, 69%, 70% response rate. So broad response rate, which I think is a good signal for the real-world clinical practice because it means, in general, if we try -- if you try VYVGART in a CIDP patient, they have a good chance of responding regardless of their prior treatment. And then Stage B, obviously, we looked at the reduction and risk of relapse, 61% reduction versus placebo. So we -- our physicians can have confidence that there is a risk reduction. We saw an early response. We saw a sustained response, and we saw that response regardless of what therapy those patients came into the treatment on. So I think it gives physicians if approved, real confidence that VYVGART will work in a variety of patients and will have that sustained impact.

Okay. And I realize it's kind of a competitive setting, both with the incumbent as well as kind of forthcoming competition from an FcRn perspective. But it would be very helpful to just kind of get a sense of your expectations on positioning into IDP ahead of the launch. It's a question that comes up a lot with investors.

Yes. Yes, happy to. I mean the way that we think about the CIDP market, we said there's 24,000 patients, the majority of patients in CIDP have, at some point, been exposed to IVIg. And so -- so generally, you will think about certainly at launch that patients that will be tried on VYVGART will likely be coming from IVIg, so some IVIg experience. I think the key will be how we work with the neurologists to determine which is the right patient profiles of VYVGART and where can we provide the most value. Certainly, what we're seeing in market research when we speak to physicians is similar to what actually I've been hearing here today through the meetings similar to what it sounds like the investors are hearing, which is when you show the clinical profile of VYVGART what they see is the value that it's going to create for those patients. And there's a broad range of patients that would be suitable for VYVGART. And so we're confident in that. The key is going to be the payer policies that we get in place. And I'm sure this question is on price, but we're working hard on making sure that we have broad access for patients so that when urologists see a patient they want to put on VYVGART, then they have access.

Okay. Well, that sets up the pricing question quite nicely. So obviously, there's a different dose in general in MG where we know the pricing, CIDP based on the trial, it's very different. So how should we think about pricing in CIDP specifically?

Yes. Absolutely. Happy to answer that. Maybe we can take a few moments on this because I think it's a pretty important one. Maybe I'll talk a little bit about the some of the underlying fundamentals around pricing. But then, Beth, maybe you wouldn't mind providing some of the background that I think has been helpful on how we thought about it for MG and how that might impact inform what we'll be communicating in CIDP. But just to put the fundamentals in place so that we all know the fact, so the price per vial for VYVGART will be the same for CIDP as it is for MG. And so -- so that won't change. And so the dosing for CIDP that was studied in the clinical trial and that we submitted for the label is weekly dosing. We have an open-label extension study that does look at biweekly dosing and even triweekly dosing and so -- as well. So those are for pricing. Now remember, pricing is different than access. So what our goal is as a company is that we want to provide broad access to patients so that they have access to our innovative treatments of VYVGART. And what we're doing, and we'll continue to do through, if we get approval, is work with payers in a similar way that we have for MG to demonstrate the value that VYVGART creates for the health care system and through -- and partnering with them through value-based agreements to make sure that we are creating value for the health care system, which in turn means that patients will have broad access to VYVGART. So our goals are similar in CIDP as for MG. On the pricing point, we will provide more details assuming approval. And maybe, Beth, that's where you can provide what people can expect.

Yes, so with MG, the number that we provided was $225,000 per year, and that was revenue back to the company. And that's based on the weight of the patient. This is for IV. So whether they were 2 vials or 3 vials, it was also based on the median number of treatment cycles per year. So for MG, that was approximately 5, which was determined from the data out of our open-label extension study. Also to get from a gross price down to a net of $225,000, we took into account government rebates and discounts and the implementation of our value-based agreements. We will take a similar approach and will provide a similar level of transparency with the CIDP numbers. So it's not weight based, that's a fixed dose with Hytrulo, but you will understand what we expect utilization to look like in clinical practice, and then how we'll also factor in those government discounts and rebates, so you'll expect a similar communication around that price at launch of CIDP.

Okay. Okay. And then just in terms of kind of indicators that you'll be tracking in the initial launch. And obviously, we'll get sales data. I guess first question there, do you plan to kind of provide sales data by indication?

We have a commitment to transparency in the way that we communicate, we understand that you want to understand the launch dynamics that are specific to CIDP, we're not going to provide revenue by indication. We will just provide a VYVGART revenue. So it's up to us to accurately portray what we're seeing in the launch from a prescriber perspective, patient and also payers. Some of the metrics that may make sense, and we'll have to see how the launch actually progresses, could be the number of prescribers or breadth of depth associated with prescribers. We have shown a number of patients before and also how the payer policies are progressing and the percent that are in place and favorable.

Okay. And obviously, kind of the market has had a lot of time to focus on CIDP and one of the consistent messages that we've been getting from the company is don't extrapolate the MG launch and don't expect an MG like launch in CIDP. Could you just remind us as to why?

Yes. Absolutely. I'm happy to touch on this. I mean I think to start with, just to say that we're incredibly excited about the potential -- the transformative potential of VYVGART in CIDP. I think the clinical profile and the clinical differentiation is clear. And having said that, we are launching potentially, let's say, let's get the approval for it first, that if we get the approval. We're launching into a market that is well established. There are 3 companies with IVIg or subcutaneous Ig that have done a great job of educating physicians around CIDP of getting patients on to IVIg. And certainly, they're very focused on keeping their patients on IVIg. We'll be entering into that market. And along with that, come with all those competitive dynamics of what is, in reality, the sort of the vast majority of patients are on IVIg, and so this market is a little bit different than the MG market. This is more of a switch dynamic market. And what that means is that I think the launch trajectory will take -- will be a little slower than it was for MG. I'm actually -- I'm pretty bullish and pretty confident on the mid- to long-term trajectory. I think for CIDP, it's -- these patients -- there is an unmet need. It's a progressive disease. This is the first innovation in terms of MOA for 30 years. So the value is there, but entering into a pretty well-established market with 3 competitors or 3 companies that are there, that takes -- that's a little bit of a different dynamic. And then, of course, what we talked about earlier, which is the payer policies. I mean, it always takes time to get payer policies in place, we just saw it with VYVGART, Hytrulo. It took about 2 quarters to get our Hytrulo payer policies in place. We expect that it will take about the same to get the payer policies in place for CIDP, so certainly, if we assume approval, then over the next couple of quarters, we'll start to see some uptake and then I think it will start to accelerate after that.

Okay. And in terms of launch preparedness, where are you with that?

We're ready. We're ready, and we're excited. And certainly, from an argenx perspective, I mean, we always like to plan for success, and we want to invest for success. So what we're thinking about is how do we -- from a commercial perspective, how do we get ready to do 2 things. Number one is, what we're really seeing is in the MG market, the opportunity is clearly bigger than we thought. And so we need to be able to maximize that MG opportunity and at the same time, launching CIDP. And so what we've been doing over the last couple of months is getting ready for that. So you will have seen in Q1, and we talked about the fact that we expanded our field team, and that includes not just our sales representatives, but our reimbursement managers and all of those support, we expanded our capacity in terms of our nurse case managers and our patient support program, so that as patients come in, we can fully support both the larger MG as well as the CIDP opportunity. We've been in market with disease awareness and education, both for HCP, so unbranded, but HCP education as well as patient awareness to start to activate patients, and we've obviously already started some of the discussions, the early discussions with payers that can appropriately happen before approval. So we're ready to go. We've been getting everything ready. And if we get the approval, then we'll be ready on day 1.

Okay. And then just maybe finally on CIDP. To what extent can you leverage the commercial infrastructure that you have in place with myasthenia gravis?

Yes, it's a great question. I should have touched on that. I mean -- to a large extent, we can leverage the infrastructure for MG. And I'll talk about it in a couple of different ways that I think are important. One is the patient support program. So My VYVGART Path, which is what we developed and sort of all the, let's call it, all the back end, all of that support everything that we do for patients for MG. We need to adapt it a little bit for CIDP, but all of the infrastructure we can leverage. And that's consistent across with a lot of the different infrastructure that you look at. And then in terms of our field teams, obviously, there's an 85% to 90% overlap in terms of the prescribers, the neurologists for MG and CIDP. So we've expanded our field team because we -- because of that need for more people out there, but certainly, we'll be able to leverage the relationships, and certainly with the neurologists, the knowledge that they have with VYVGART, and the experience that they have with VYVGART, it will be advantageous for us.

Okay. And do you -- is the sales force kind of rightsized now?

I would say, yes, we're confident that we -- as I said, we looked at it from the perspective of how do we make sure we can maximize MG given the bigger size of the opportunity and CIDP. And I think it's fair to say that across the board, we made the investment, we want to make it once, and then we want to be able to execute. So we feel it's rightsized.

Okay. Perfect. Maybe we'll switch gears to MG, obviously, kind of you're in the market there, kind of a more mature market. What are the key drivers from here to continue to grow VYVGART in myasthenia gravis.

Yes. I mean I think our strategy is really clear in MG, and that is that we believe that VYVGART should be used in earlier line treatment in -- for patients with MG. And we're seeing that strategy play out. And what that means is that immediately after mestinon or after the orals, we think you should -- when a patient is uncontrolled, the first -- and so we're executing on that in a number of different ways, around early line treatment. We have 2,700 prescribers, neurologists, so we continue to expand our prescriber base and that's helped with Hytrulo. So with our subcutaneous option for -- with VYVGART, we've already seen that we've been able to reach different prescribers and new patients. So our strategy in MG is really to be the first-line biologic that's used. One of the key pieces to that strategy is around the prefilled syringe, and so maybe a quick update on the prefilled syringe that we shared that we would be filing for our prefilled syringe by -- before the end of June. We're on track with that commitment. And what that means is potential for depending on the approval time -- the review time, potential for approval before the end of the year or Q1 of next year. That prefilled syringe and potential for self-administration is a key part of our expansion strategy and moving into early online treatments because you can imagine a patient going from an oral directly to a prefilled syringe is a much easier step than going to an infusion. And I think once you put into context of the package of the real-world efficacy that we're seeing with VYVGART in MG. I mean in the real world, we see over 50% of patients that are consistently in MSE, minimal symptom expression, which means they're living their lives like a normal patient, like any one of you or I, that efficacy continues to stand up, the real-world safety that we continue to see, whether it's in our open-label extension studies, in our ongoing trials, or in the real world, the safety profile is differentiated for VYVGART and then obviously the treatment burden or the convenience of 1-hour infusion subcutaneous and then PFS. I think once you put all of that together, you can start to see why VYVGART would be first line and would drive significant growth in the MG market. And maybe just one quick clarification that we -- when we file for the PFS that will be in both MG and CIDP.

Great point.

Just on the PFS point and just on -- in terms of kind of formulation and the strategy you'll make sense on the PFS, but you've also been clear that you think you need multiple formulations in MG. Why is that the case?

Yes. I mean I think our strategy, whether in MG or CIDP, or across the board is that we want to -- we are patient-centric, and we want to meet patients where they are. And what we find -- and so our strategy around our formulations is not a life cycle management strategy. But rather, our strategy is to make sure that patients have the -- and neurologists have the option of whatever formulation meets their needs that they have that option available with VYVGART.

Okay. Just maybe going back to MG and kind of competitive dynamics. You obviously have another FcRn in the market now, I think, 9 or 10 months, we talked about it this morning. What are you seeing from that perspective in terms of impact on VYVGART?

The way we think about competition and the way we've talked about it is that especially in a market like MG, we think innovation is good. It's great to have more biologics coming into the market and expanding, raising awareness amongst neurologists, around MG, expanding treatment and expanding the patient population. And that's exactly what we're seeing is expansion in the MG market. And then within that expansion, we see that VYVGART is really well positioned as the leader as the first line given the clinical profile. So I think we welcome innovation into that market. And frankly, across autoimmune, there's a huge unmet need and more innovation means better outcomes for patients.

Okay. And maybe just going back to the kind of formulation piece and you've got -- you may potentially have 3. And there's MG, there's CIDP, but there's a whole raft of other indications that you're investigating for VYVGART. Are there any indications for any reason that may have a preference for a subcu or an IV or a prefilled syringe?

I mean our ambition is to have both IV and subcu available in all of our indications because that's really what sets us up best to capture the most physician preferences, the most patient preferences, reaching most broadly across patient populations. And also to navigate payer dynamics, which I think is often forgotten. And so that is our goal. Right now for most future indications, we're leading with a subcu strategy, whether it's Hytrulo or prefilled syringe, but it is our goal to have both -- all formulations available on each indication.

Okay. And on that piece on reimbursement and pricing negotiations, can you just remind us where you are in Europe from myasthenia gravis?

Yes, I can give a quick update on that. So obviously, launched in Germany and have reimbursement. The launch continues to go -- to be very strong. You saw the Q1 results, so you will see in the Q1 results, where we continue to have growth in Germany. You'll recall that in Germany, we hit the limit for the -- for the orphan drug budget. And so we do have to renegotiate price in Germany. That will happen in January or will hit in January of next year, but we're already accruing for -- based on some assumptions for Germany. So we will renegotiate but the launch is going incredibly well in Germany, we are seeing similar dynamics in Germany, by the way, as we are in the U.S. where -- and actually in Japan as well, where it's not just the centers, the academic centers that are prescribing VYVGART, it's actually more -- even more beyond out into the community. So German launch is going well. That's the dynamic there. We recently got Italy and Spain reimbursement, those launches are progressing very well. And what we're seeing is we have good clinical advocacy, and we're right on track with where we should be. Ongoing conversations with France and the U.K. So that rounds out sort of the big 5 within Europe. And what we're seeing is in the sort of let's call the midsized markets, Belgium, which is important for us as a Belgian company. For example, we've got reimbursement. The uptake is really strong. We have some other markets as well. So in Europe, overall, I would say I'm really proud of the team. They've done an incredible job of getting access while maintaining value for the health care system as well as value for our argenx . And we're seeing the clinical advocacy, we're seeing the uptick. And I think we'll continue to see growth in Europe, similar to what we saw in Q1, which is great to see some diversification outside of U.S. and Japan.

Okay. Before we move away from MG, just wanted to get your thoughts on a comment made by AstraZeneca at their Capital Markets Day, and I think it kind of caused a bit of an investor conversation. Just they said that they were now gaining more share from FcRn as opposed to losing it for their complement products. Could you just kind of put that into context as to how you see it?

Yes, absolutely. I mean I think that's -- we see that exactly how we see the market unfolding and the way that our strategy -- reflects our strategy in MG. And what I mean by that is, I think, in our view, patients should start with mestinon or steroids, immediately go when they -- if they're uncontrolled, they should be on VYVGART as the first line. Patients should stay on VYVGART as a maintenance therapy. And we're seeing -- I mean, we've seen that in our open-label extension. We have patients up to 9 cycles continuing to stay in MSC. So we know that patients should stay on long term. However, there are some patients that won't respond to VYVGART. I mean in the clinical trial, we saw 20% of patients, let's say, that are nonresponders. Those are the patients that, yes, maybe you want to move on to the more refractory patients, the most that might move on to the complement. And I think we're seeing that play out in the market. We're getting 50% of our new patients are coming off orals. So we're clearly positioned there. And then if there's a need to move on from VYVGART, for some patients, there will be, then they can go to the complement.

Okay. Perfect. In terms of kind of additional indications for VYVGART from here, of course, kind of end of last year, there were -- first time that there were some not positive trial readouts for VYVGART. So you subsequently kind of went through a review process. Could you just kind of provide us with some commentary as to your confidence in those indications that you are now following and the learnings from that review?

Yes, absolutely. I can comment on this and maybe, Beth, if you want to add anything. Maybe I'll just add at the highest level. What we -- after those -- the clinical trial readouts, we went through and did a robust review of the entire portfolio of clinical trials for VYVGART as well as empasiprubart, by the way, not -- and to look at well -- so the way that we select indications at argenx is, first, we look at the biology rationale, is there a clear and strong biology rationale. That didn't change based on the PV and ITP readout. The second filter that we use is a clinical development program is that can we execute a clinical development program? Can we design a clinical trial appropriately? Are there regulatory endpoints? Can we really -- what's the likelihood of development? That's where we did a double-click based on PV and ITP to make sure do we have the right endpoint? Do we have the right clinical trial designs? Are we confident in our execution, and then the third filter by the way, is that -- is what's the unmet need and the commercial assessment, and we didn't need to look at that, when we did the full portfolio review, just to make sure and double-click on to make sure we were controlling placebo and background medications and clinical trial execution all of that, what it resulted in is more confidence in the indications that we've selected. With the exception of ANCA vasculitis that was the one that we've announced that we decided. Actually, we saw that, that was a difficult path forward, and we decided to end that indication.

And actually, we switched in sort of ANCA, we've announced systemic vasculitis.

That's correct. The other outcome of it is, remember that the biology of pemphigus is very similar to bullous pemphigoid and what we learned from pemphigus is actually that a low dose of steroids, 1/3 of the treatment guidelines could put patients into complete remission and actually you could then taper down those steroids, and this is because steroids turn out to have an effect on the autoantibodies, reducing them about 50%. So we wanted to take those learnings from pemphigus and apply them to our understanding of bullous pemphigoid, which does have a similar biology rationale behind it and -- so we decided to change the bullous pemphigoid Phase II/III into a Phase II. We stopped enrollment, and we want to allow those data to mature, the patients that are currently enrolled in the Phase II. And what we owe you still by the end of the year is a decision on bullous pemphigoid. And I think there's 3 pathways that we could consider. One would be that the data show enough of a signal that we feel confident to move forward into Phase III, and we have the information to design that Phase III. The second would be that we would run a new Phase II rather than ending up in kind of the same outcome as pemphigus, maybe redesign and ask a different question with our clinical trial design. And then third would be that we wouldn't move forward with BP. So we still do all that by the end of the year.

Okay. The other thing you also -- or say is full data for empa. So could you just kind of provide us with a sense of what we should be looking for there to kind of provide confidence in that data set?

Yes. So we showed -- I guess, middle of last year, we made the decision to -- we looked at Cohort 1, and we made the decision to advance to Cohort 2. And then during the -- earlier in January, we gave highlights at top level look at that Cohort 1 data, and they're very exciting data, and we saw a 91% reduction in the need for IVIg rescue with empa treated patients compared to placebo-treated patients. . We also saw, if you remember how the trial is designed, first patients had to demonstrate IVIg dependency then we established a cadence of IVIg dosing. And at that moment of kind of peak response in IVIg, we then switch them over to empa. And from that moment, we learned that 94% of patients feel improved on empa than they do compared to IVIg. So how a patient feels that was a really important outcome of those Phase I data or those Cohort 1 data. We will show more of the Cohort 1 data in upcoming medical meeting. And our commitment now is to show the full data set, including the outcome of Cohort 2 at a future date at an upcoming date. And really, what we want to see is consistency of response. And we also want to establish a dose response because we're looking for information on what dose to take forward into Phase III.

Okay. And -- there's obviously been a lot of focus on VYVGART, but empa is kind of something that I know you are excited about and Tim talks about the playbook that you have. So could you just, from a high level, and I'm sure we'll get more details when we have the data, but how do you think about empa? Is it another pipeline and a product type asset?

So I mean I think it's unfair to have VYVGART be the bar for all future molecules. What we're really thinking about is empa is a first-in-class molecule. I mean we really like first-in-class molecules. We're not -- we don't want to make a pipeline of me-too's. The second is that it there's a really differentiated profile with empa. I mean, from an efficacy perspective, safety, and I think you'll learn more about the dosing frequency. And there's several opportunities from an indication perspective from which we're going to take forward with empa. We already have 3 underway. So that's MMN, which I was just talking about, and then delayed graft function in the kidney transplant setting and dermatomyositis, and we're still working on additional indications that we'll bring forward.

Okay. Just into kind of last 90 seconds or a minute or so, and I know there's a lot of cash left, but could you just kind of, in that time, just kind of outline the key things that investors should be focused on for the remainder of the year?

Yes, definitely. So we still owe 2 Phase II data points on whether we're going to move forward into Phase III with those. One is POTS, which will come this month and the other is those 3 subsets of myositis, the necrotizing myositis, antisynthetase and dermatomyositis. So all of that will come by the end of the year. And what I mentioned there is the outcome that you were really focused on there is will we move forward into Phase III. Do we see enough of a signal to justify that the investment into a Phase III, and do we have the right information to go and design a winning Phase III. So that's what we're looking for with those indications. I already mentioned the key data point and the full Phase II from MMN. And beyond that, it's just going to be commercial execution and some additional expansion there.

I think that's right. And you mentioned it earlier, but just for holistic, that PFS was the other thing that we said on track, for PFS filing before the end of June. And then not to forget -- I know it's not a catalyst event, but it's important, is the R&D Day that we have coming up that will be important as well.

Yes, then we're going to be able to share what we see as the argenx future, our vision over the next 5 to 6 years of growth.

Okay. We'll very much look forward to that. Thank you so much.

Thanks for having us.