Good morning. Thank you for joining us at the Bank of America Healthcare Conference. I'm Tazeen Ahmad. I'm one of the senior SMID biotech analysts here at the bank. It's my pleasure to have with me our next presenting company, argenx. Sitting next to me is Chief Executive Officer Tim Van Hauwermeiren. Tim, good morning. Thank you for making the long trip from Europe over to Las Vegas. We really appreciate it.
It's my pleasure, Tazeen. Thanks for having us today. It's a great conference. Thank you.
Maybe let's just go straight into the company. Within biotech, of course, you're one of the most well-known companies, but for those who don't have an appreciation as much of the details, can you give us a quick overview of the company, and then we can go into specific questions?
Definitely. So we play in the field of severe autoimmunity. We like to go after novel biology, novel targets, because we think that in severe autoimmunity, what we badly need is a better understanding of disease biology. We drug these novel targets with antibody molecules, which we engineer in-house. And then we typically like to go after molecules which can play in multiple disease indications. So pipelines in products. The lead molecule, efgartigimod, is in active development in 13 indications today and will be in 15 indications by end of next year. And then empasiprubart, ARGX-119, and four IND candidates are following in the pipeline.
Okay. So there's a lot going on at the company, but, for the time being, folks are focused on the continued launch of VYVGART in GMG, as well as the potential upcoming launch in CIDP. So maybe let's just start with GMG and the ongoing launch. You've been able to beat expectations virtually every quarter since you started the launch, which is pretty rare in the space. So I don't think people are questioning your commercial capabilities. I think more questions, though, nowadays are coming in about the, I guess, the steepness of the launch for GMG. So, you know, on the first quarter results, you did beat expectations, but the growth level, people are wondering whether that's starting to flatten out. And we're getting questions about whether competition is impacting VYVGART.
Can you just talk about the 1Q result and the dynamics around it?
Yeah, definitely. I'm happy to do so. I think MG is a big market. And just to give you a feeling for, you know, where we stand in developing that market, if you would sum up the market share of all branded biologics in MG, we're not even hitting the 10%. So there's a ton of work in front of us, a ton of opportunity, and potential to grow into. The rate at which, actually, that growth will happen remains to be seen. But what we did say in the Q1 call is that, you know, our underlying business fundamentals are very strong. You know that we're building the market by going upstream in the treatment paradigm.
Indeed, more than 50% of the new patient adds in Q1 were coming straight from the orals, so straight from Mestinon, steroids with or without an IST, which I think is boding very well. So we're building, we're growing this market. And within that growing market, we have further grown our market share. So we're pretty bullish on the market opportunity. We think the fundamentals of the business are strong. Yes, of course, you know, we were not immune to some of the weaknesses of Q1, which are the typical recertifications. We had some serious weather, etc. But that's okay. I think the long trajectory of the launch is intact.
Yeah. And I think the important thing that some folks might have missed is that, you're not only maintaining market share, but you're growing the market and you're growing share within that market. Is that right? So, you know, as you think about where efgartigimod can go from here, do you based on what you said, I don't want to put words in your mouth, do you think that the growth rate is flatlining, or do you think that it's going to continue to accelerate?
When we look at, we're on a continuous trajectory, so we don't see any slowing down there. Ultimately, of course, the curve will start to flatten a little bit. But that's why I mentioned the number of the 10% market share of branded biologics. There's a ton of work to do. We have a number of ex-MS people in the company. They, of course, like to draw these parallels with the multiple sclerosis market. It took them 10 years to bring innovation really frontline in these MS patients instead of reserving innovation for the last-line patients. So I think that's where we also need to take the MG market. And as we have consistently said in this launch, that will all be about education.
Yeah. And on that point, people are also, I think, talking about the different ways in which VYVGART can be dosed. The traditional original IV, VYVGART Hytrulo, you're going to have presumably a prefilled syringe next, and then in the future, a potential autoinjector. So, you know, what are the dynamics, or what have they been for VYVGART Hytrulo in the U.S. since it got approved? Are you actively trying to switch patients from IV to VYVGART Hytrulo, or are you trying to just provide another option?
Yeah, that's an excellent question. So, as a leader in the space, we want to lead from an efficacy point of view, from a safety and tolerability point of view, but we also want to offer the broadest, product presentation, offering. That means we have a very clean IV product. That's the product we used when we launched. Then quickly thereafter, we launched VYVGART Hytrulo. That's a first-generation subcutaneous product, which is an execution which is still administered by an HCP here in the United States. In Japan, in Europe, it's self-administered. And now that prefilled syringe is quickly coming online, we were public in the Q1 call that the prefilled syringe file will be submitted with the FDA, before the end of June. Now, the dynamics of Hytrulo are very encouraging. Remember what I just explained? We need to move upstream in the treatment paradigm.
That means we need to convince people who are typically only on oral drugs to come on a biological. And the step, it looks like the step from an oral to a sub-Q injection, because that's what it is. It's a 30 second injection, is a smaller step than to an IV infusion. If you take an IV infusion, it feels like you're a worse patient. Since the J-code has been in place on the 1st of January, we really see an acceleration of VYVGART Hytrulo. It's really gaining traction. And very interestingly, we mainly see VYVGART-naïve patients coming on drug. So this is not a switch strategy. This is not a dynamic where IV patients mainly will switch over to sub-Q. We see some of it. But the lion's share of these patients are new patients.
That's going very well, of course, for that long-term market-building ambition which we have.
Okay. So in terms of how easy it is for a patient, you know, IV, obviously, you have to go to a facility and be in a chair. What is the inherent advantage going from Hytrulo to a prefilled syringe?
I think the prefilled syringe is the gating event, I think, to self-administration in the United States. As a patient-centric company, we believe that ultimately, you want to maximally enable your patient, and that means self-administration at home, and not HCP administration.
Yep.
Think of the prefilled syringe, you know, as an execution which you have in your home fridge. The only thing you would need to do is take the package out of the fridge, allow it to come to room temperature, and then it's a matter of, you know, seconds for self-administration, which I think is very compelling.
Okay. Now, as we think about the maturing of the launch of gMG, oftentimes investors ask us, "Well, when do you think argenx will start to provide sales guidance?" What's your view on that?
So, you know, Tazeen, this is a topic which we very actively discuss also with you, you know, with our best analysts. It is a good question, and we're paying a lot of attention. Today, with still so many moving parts, we think it's still premature to give sales guidance. It will come. Maybe we need to work our way through the CIDP launch and be again on a steady trajectory there before we can really start to consider that seriously. But we're paying attention, and we try to stay as close as possible to our analysts, such that you know, their guidance is you know, a reasonable alternative to guidance from the company.
Okay. Maybe a couple of questions on the competitive landscape. So you're obviously the market leader, have a big runway of being first to market in gMG, for sure, and about to start a CIDP launch later this year. But I think there's more questions coming in as more companies have seen the success, frankly, that argenx has had and are seemingly trying to pursue the same indications that your company is trying to say that there could be certain advantages. So, without going into specifics about other people's data, can we just talk about? I think we did talk about the route of administration advantage, but what about the efficacy advantage that you feel that VYVGART provides?
Yeah, I think the best way to deal with competition is to delight your customer. And if you look at the real-world data for VYVGART, and again, we have been showing more data at AAN, they're very consistent with a clinical study profile. That means about one in two MG patients on VYVGART can achieve minimum symptom expression. That means you live your life without symptoms, without being reminded of your disease. So you're fully functional, back to the state you had before you were diagnosed with myasthenia. That's a big deal. 50% MSE, I think, is a big deal. And it is consistent between the IV execution and the sub-Q execution. From a safety and tolerability point of view, the safety database is growing now very fast.
We have more tdhan 4,000 patient years' worth of safety data, not just in MG, but also across indications and across product presentations. No black box warning, no vaccination, no REMS, no labs. It's a pretty clean drug so far. That's also what the real-world experience is telling back to us. And then, of course, you want to delight your customer, you know, with the most complete product presentation offering. IV will always be important here in the States, will always represent a significant chunk of the business. But the speed by which we're rolling out, the sub-Q product variants, I think, is high. It's fast. And I think it's being received very well by the customers. So I think we've put the bar very high.
Is there a difference, actually, going back to Hytrulo, on how Europe is receiving it versus the U.S.?
That's an excellent question. Outside of the United States, you do see a switch dynamic. I think the way the healthcare systems are designed are such that, you know, sub-Q is favored over IV, and you see a very rapid switch from IV to sub-Q. That is a very fast dynamic. In Europe, and also in Japan, where we launched VYVDURA, that launch is going very well.
Yeah. Now, I guess staying on Europe for a second, Germany had an, you know, a price change because your volumes were, frankly, very high. And so, you're in process, I think, of, of finalizing what the new price is. But in the meantime, I think people are wondering, should you expect to see the same impact across other countries in Europe that you've seen, just given the popularity that you've had with VYVGART in Germany?
So in Europe, each country is different from an access point of view. It is true in Germany, when you hit EUR 30 million sales, that you lose your orphan status and that you need to go back to the negotiation table. So we are negotiating whilst we speak, and we will be able to give you the final verdict, you know, in at the beginning of next year. In the meantime, we're accruing for an anticipated price reduction. But, I mean, what we showed in the Q1 results is that despite that anticipated price cut, the business has still been offsetting that with volume growth. So I think, again, back to what I said at the beginning of the call, the underlying dynamics of the business are very healthy and continue on a steady growth trajectory.
Yeah. So to clarify, not being considered orphan anymore in Germany has not interrupted treatment for those patients?
No, it did not. Actually, to your question, each other country in Europe has its own different dynamics. So what happens in Germany is not necessarily true for what's going to happen in other European countries.
Okay. Good. So maybe let's move on to CIDP. You do have a PDUFA upcoming 21st June in the U.S. And, maybe we could just talk in general about how interactions with the FDA have been going as you kind of reach the final few weeks.
Yeah, happy to do so. So from where we stand, Tazeen, we think that we're on track with the review of the file. Remember, the whole CMC section, which is, you know, 50% of the review, has already been cleared by the approval of VYVGART Hytrulo. It's the same formulation, the same process. So CMC, I think, was immediately a big tick in the box. And then I think you have the normal site inspections, the review of the clinical data, etc. From everything we can tell, we're getting ready to enter into label discussions.
Okay. And if you do get an on-time approval on 21st June are you ready the next day to launch?
So technically, we will be ready to launch. I mean, we have the product. We have the J-code. We have the field force, in action. Of course, we have the 4th of July, in between. But technically speaking, we're ready to start. Yeah.
I guess because of the experience you've had, fortunately, with neurologists already for VYVGART, is it safe to say that a lot of the physicians that would be recommending the drug for CIDP are physicians that argenx already knows?
I think that's a good point. There are real economies of scale between MG and CIDP. So it's the same relationship, either with the same physician or with the same office, where, you know, it's a neighboring physician in the same corridor, you know, who is treating CIDP patients. So the way that we scaled the field force is by shrinking the territories and adding more territories and more people, but allowing them to sell within their territory both MG and CIDP because the overlap between the practices is almost complete. And of course, that is a bonus we have. That's a tailwind. These physicians will know about VYVGART. They will also know about its excellent safety and tolerability profile. So at least that hurdle will have been taken already. Yeah.
So I guess based on how you're thinking about the launch, you know, managing the launch, is there any concern that, you know, salespeople that have gotten good traction and continue to grow, the GMG penetration will be needing to also focus on CIDP, and that could impact GMG at all?
That's why we're shrinking territories and adding territories. So we're still in full MG launch, as we just discussed. So the task here will be for the field force to continue an excellent MG launch and launch CIDP. And I think there could be synergies between being able to tell both an MG and a CIDP story to the same neuromuscular or neurology office. So I think we will have a bonus there. The launch dynamics between CIDP and MG will be distinctly different. CIDP patients are on medication, typically IVIG. So this will be a switch dynamic. We will need to convince patients to switch.
Right. So maybe let's spend a minute on that. So unlike GMG, where you're not competing against IVIG as a labeled indication, you will be doing so for CIDP. Why is that important?
That is important.
Even though you do have inherent advantages based on data?
Yep. As always, we will try to market from the data. It basically means that the plasma fractionators can countermessage on label, which they could not do in MG because, of course, it was not an approved indication. And there is a big loyalty between a patient and his or her IVIG product. Remember, it's the only product which allows them to lead a reasonable life. So IVIG is an ally. Of course, you know, there are shortcomings to the product, and we will be selling based on our own strengths. But it's a big mental step for the patient, I think, to leave a tried-and-trusted medication and come on an innovation.
Understood on that point. But, you know, Hytrulo itself, if you were to compare how a patient's going to receive that versus how they have to currently receive IVIG, wouldn't that in and of itself be attractive to a patient?
Definitely. I think from a treatment burden point of view, this is going to be day and night. I mean, you just cannot compare, spending 1-2 days in an infusion chair to get your IVIG or getting a 30 second sub-Q injection. So I think that by itself should be compelling. Same with safety and tolerability. Side effects of IVIG are infrequent, but can be catastrophic. But I think we need to talk about efficacy. And I think the real message here is what we started to deliver at AAN on the podium is that actually patients or a significant amount of patients regain function compared to their baseline when they enter the study, when they're being put on VYVGART.
So here, I think the promise is that by putting a patient on a drug which is hitting the drug, the disease biology in its heart, the pathogenic antibodies, you have the right to see a distinctly different and better outcome.
Okay. And we've all been doing checks with physicians, as we all do, heading into every launch. And those checks are, you know, quite positive and indicate that there is high unmet need, actually, in the CIDP space. But I think some of the feedback that we've been getting, and I think some of the feedback that you've been getting, is that argenx sounds conservative about the CIDP launch. Why, why is that true or not true?
Well, you remember when we launched MG. I spent a significant amount of my time in the field. I can tell you that the average neurologist out there is not the neurologist which we get on the phone when we do our market research. I mean, these are early adopters. That is a biased sample. Your average neurologist in the field is a pretty conservative person. I think you will see the same as we saw for MG. They will start to try the drug on one patient, maybe two patients. They will be their worst patients. And only when they, in their own hands, see efficacy and success, they will start to move, you know, up the treatment ladder. So it's going to take time. It's a conservative audience. There's a ton of work to be done.
I think all of us were pleasantly surprised, though, with GMG. I think you, you know, you messaged similar dynamics right before VYVGART launched there. Just based on all of the, the data points that we're receiving, it, it does seem that potentially the launch is going to be stronger than you're indicating. And, and if so, that, that's great for patients and for the company. But I think people are also wondering how we should think about the first couple of quarters of the launch, per se, even if we think ultimately it's going to be a strong launch. Is it going to be strong right out of the gate?
I think that's a great question. We will not escape the payer dynamics. I mean, it typically takes two quarters to install your payer policies. In absence of a payer policy, many neurologists will just hold off prescribing the drug. That's also what we have seen with the launch in MG. So that will not be different. So I think you need to give us at least 2 quarters to install the payer policies. You know that the philosophy of the company, by the way, is to create broad access for patients. So we will try to create favorable policies for the lion's share of the CIDP patients. And that takes time. So we're very bullish on the total opportunity of CIDP, but we need to be realistic about, you know, the ramp up to peak sales. Yeah.
Can you just remind us what the current market for IVIG sales into CIDP are?
Next to primary immunodeficiency, which is the biggest indication for IVIG, I think, CIDP is the second biggest indication. It's also an indication which is growing double-digit per year. So it's a vibrant market. It's a large market for these players. It is on label. So we also expect a fair amount of pushback from these people, unlike, you know, what could happen in MG, where IVIG was not on label.
Do you think there could be some pricing discounts that the IVIG manufacturers will try to offer to keep share? And how does that impact? I mean, ultimately, your, your VYVGART price is already set. It's, it's based on the number of, of vials that a patient's using. But is that a dynamic that, that still needs to be fleshed out?
The work which we have to do is actually, enter with our payers into a value-based agreement, like we did for Myasthenia. Because you're right, we priced VYVGART. The milligram price is known. We priced in MG already, keeping the other indications in mind. But you can calculate, you know, mathematically what the cost would be of a CIDP patient based on vial consumption. What we need to do is to overlay that with an attractive value-based agreement with the payers, where we come to a win-win relationship to create that broad access. That's a different game than what plasma fractionators are doing, that typically discount into that channel. That's also how they compete. Because I think the perception out there is that IVIG products are pretty much replaceable amongst each other.
And I think it's a little bit of flavor of the month, depending on the discount which is being given into the channel. So that creates loyalty by itself. But it's a different dynamic than the dynamic which we need to follow, which is install value-based agreements with the payers.
Have you already started those conversations?
Yeah. We're in the midst of these discussions. That's why we're also careful not to signal too much about the price, the ultimate price in CIDP. Let us land these conversations. And our commitment, Tazeen, is that at the day of launch, we will give you transparency on what the price per patient will be so that our investors, you know, have no difficulty understanding the impact in their models.
Okay. In the early days of the CIDP launch, should we look for metrics that were similar to what you provided in the early days of GMG to give people a sense of how the launch was going?
Yeah. I think that's a fair assumption. I think we want to be transparent to our investors. So we will, in the beginning of the launch not forever, of course, but in the beginning, use some indicators of early demand and early pickup, similar to what we have been doing for MG. That's correct.
Okay, so we can probably keep talking about this for the rest of the time, but there are other many other things going on at argenx. One of those I wanted to talk about is the upcoming data release for the phase II/III myositis study that's scheduled for the second half of this year, correct?
That is correct. I mean, this year we have six phase II data points. We already talked about Sjögren's, and we will show the data at the clinical conference. Then before mid of the year is PC-POTS. And then importantly, indeed, is the three myositis subsets, second half of the year.
Right. And so, what level of data should we expect to see at that press release?
The press release will be more in line with what we did for CIDP. This is a seamless phase II, phase III study, where basically we have a gating event after 30 patients for each subset, where we can make a go/no-go decision point. So the decision will be, do we continue based on what we see in these subsets of myositis, yes or no? And we also have an ability to adjust sample size. What will give us conviction to make the go decision? That will be seeing an efficacy signal, which can be reasonably expected to be better than placebo.
Is there a certain point difference that you're looking for there, based on the scale that you're using?
We're not public on it. But, you know, we use the TIS scale, the total improvement, a scale. That's a scale which we agreed on with the FDA. That's also the scale which was used for the IVIG proderm study in dermatomyositis successfully. So that study, I think, is giving you a good feeling of what a clinically minimum improvement would be. That's a delta of 20 points on the TIS scale. Yeah.
I guess in general, as we talk about pipeline, efgartigimod was always pitched as a pipeline within a product. Now, argenx has the benefit of mostly, you know, beating expectations on all fronts with investors. And so when there was the inevitable study or two that didn't work, I think people were, frankly, shocked and surprised by it. But since then, you know, you've had a go decision, like you said, for Sjögren's, which was, I would say, also de-risked by J&J's own study. You do have a COVID, you know, PC-POTS study that's going to have a go/no-go. And then we talked about myositis. It does seem to me that there's a little bit of a disconnect maybe right now, based on valuation, of everything that's going on with the pipeline for argenx and whether or not investors are willing to give credit for that.
So as the person who's, you know, the front person for the company, what do you think is that disconnect? And what do you think argenx can do to address that?
Look, we're a database company. The data will need to speak. I think indeed we come from a period, you know, where people thought that each and every clinical trial we did would be successful. And we hope the same, and we design for the same. But that is, of course, not clinical reality. I think today we're a little bit in the other extreme camp, which is zero credit for all the ongoing work. Let us look at the data first. That is a fine baseline. So let's turn these data cards. Let's build up again the trust and the confidence in these indications. There are many indications coming. Yes, we will continue to have some attrition in the pipeline. That is unavoidable.
But we try to do our best explaining what we think the risks are of these studies, how we mitigate these risks in the clinical trial designs. We also performed a thorough portfolio review after the Pemphigus unexpected result. And there we announced during Q1 that of all the indications we're pursuing for both VYVGART and empasiprubart, all these indications are in good shape, except for AAV, where we actually could not see a way around a high dose of IV steroids. And therefore we decided to be disciplined and stop AAV and replace it by the next indication on the list. Despite the fact that actually the drug has the right to work in AAV, we just saw the publication of an impressive case report where VYVGART moved the needle big time in an AAV patient.
I guess keeping on pipeline, you've announced that there's going to be an R&D day this summer. Why is now the right time to remind investors about what's going on? And should we expect to see new data when you have that R&D day?
Yeah. So there's a big ask from our investor base, you know, to have an R&D day. The formula of the past is a formula which people appreciated. That includes disease education on some of the new indications coming. For example, Sjögren's, MMN, are diseases which are not very well known and appreciated by our audience. We are also thinking about, you know, presenting substantial data on MMN. So stay tuned. That agenda will be made public. But the save the date is just out. That will be July 16th, in New York.
Okay. With that, we're just about out of time. So I will say thank you for sitting with me for the last half hour and answering questions. It's always good to catch up. Thanks, everybody, for joining, and hope everybody has a good rest of the session.
Thanks, Tazeen. Thank you. Thank you all.