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argenx SE
Earnings Call Analysis
Q3-2024 Analysis
argenx SE
In the third quarter of 2024, argenx reported a remarkable total operating income of $589 million, a significant increase of 20% quarter-over-quarter and 74% year-over-year. Product net sales reached $573 million, with the U.S. contributing $492 million. The strong sales growth reflects the successful launch of VYVGART Hytrulo, particularly in the CIDP segment.
Total operating expenses for the quarter were $575 million, up $40 million from Q2 2024, largely due to increased Selling, General, and Administrative (SG&A) expenses associated with the CIDP launch. The gross margin remained high at 90%, and despite a modest operating profit of $14 million for the quarter, the company faced a year-to-date operating loss of $125 million, highlighting the ongoing investment in growth.
The launch of VYVGART Hytrulo for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) has been successful. Over 300 patients have been treated in the U.S., primarily from the existing IVIg therapy market. The company aims to capture this switch market by establishing favorable payer policies, which now cover approximately 54% of U.S. commercial lives. Initial feedback from prescribers indicates excitement and a broadening of the prescriber base, which could bode well for future growth.
The patient-centric strategy implemented by argenx has led to significant improvements in the quality of life for patients switching to VYVGART. Positive patient outcomes have been reported, including regained mobility and enhanced daily activities. This success underscores the company's commitment to providing transformative therapies for patients with unmet medical needs.
Argenx remains focused on innovation and expanding its pipeline. The management is prioritizing indications that yield transformative impacts over those with marginal benefits. The company is preparing to advance several programs, including a go-no-go decision on three pipeline candidates before the year’s end. The ambition to reach 50,000 treated patients by 2030 is contingent on ongoing clinical developments across its therapeutic areas.
Looking ahead, management provided no specific revenue guidance for 2025 but expects payers to continue aligning with VYVGART’s value proposition. The company has maintained its combined SG&A and R&D expense guidance of $2 billion for the year. The CIDP opportunity remains promising, with a defined addressable market of approximately 12,000 patients exhibiting unmet needs. However, ongoing market dynamics and competition will influence the trajectory of growth.
The competitive landscape for CIDP is evolving as other therapies enter the market. Argenx acknowledges that while it has captured a substantial portion of the CIDP population early in the launch, it needs to remain vigilant regarding market dynamics and competitive responses. The company's leadership is optimistic yet cautious, emphasizing the need to monitor response rates and treatment adherence as part of its ongoing evaluation of the CIDP launch.
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Good morning. My name is Rob, and I will be your conference operator today. I would like to welcome everyone to the call. [Operator Instructions]. I'd now like to introduce Beth DelGiacco, Vice President, Corporate Communications and Investor Relations. You may begin your conference.
Thank you. A press release was issued earlier today with our third quarter 2024 financial results and business update. This can be found on our website along with the presentation for today's webcast. Before we begin, 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 time lines, 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 confirm these statements in relation to actual results unless required by law. I'm joined on the call today by Tim Van Hauwermeiren n Here, our Chief Executive Officer; Karl Gubitz, Chief Financial Officer; and Karen Massey, Chief Operating Officer. I will now turn the call over to Tim.
Thank you, Beth, and welcome, everyone. I'll begin on Slide 3. As we approach the end of 2024, it is incredible to reflect on all that we have accomplished, having achieved the goals set out in our 2025 vision ahead of plan. We moved the gold cost again in July raising our ambition with Vision 2030. Building on the momentum from R&D Day, we are now executing across the business to advance this vision beginning with our commercial expansion. This is our 11th consecutive quarter of growth, and I continue to reinvest with the team's efforts to reach more GMG patients and prescribers and build our model data in support of [indiscernible] as the leading branded biologic in MG. The CIDP launch has started strong as we leverage the powerful data from ET and our established relationships with BASE and prescribers to support the quarter that surpassed our expectations.
Overall, we are thrilled with our initial progress, bringing innovation to CIDP patients. Karen will provide metrics that highlight the positive reception of the [indiscernible] across key stakeholders, but overall, we are very happy with our strong start. We are navigating the unique market dynamics of CIDP well and were able to deliver our innovation to more than 300 CIDP patients last quarter, which correlates to revenue that is approaching what we saw in the first quarter of our MG launch.
Slide 4, we now have the benefit of seeing the adhere data drive real-world outcomes with some patients already experiencing retailer function early into that treatment journey. My personal favorite story comes from the first CIDP patient dosed with VYVGART. The patient's wife shared at prior treatment per husbands struggle with mobility. We recently checked in and the patient is now more active. Key and his wife have enjoyed traveling again and were able to attend a friend's wedding. He has now been able to take care of himself more independently and is eager to return to activities he loves like golfing and going to the gym. The life of the patient expressed for gratitude for the renewed lease on life. This is the type of story that makes this industry solely warning.
Slide 5. Earlier this month, we had a strong presence at ANN where we were able to present new clinical trial and were data demonstrating our long-term commitment to the neurology community and driving transformational outcomes for patients. We continue to set a high bar with VYVGART driving rapid deep and sustained responses. And now we have multiyear data demonstrating durable efficacy supporting maintenance use. Over 50% of GMG patients on this card achieved MSE which physicians agreed is an important goal of treatment for their patients, and they hear the speed of onset is a clear advantage.
We now have more than 8,000 patient years of safety data which demonstrates consistent favorable safety, no black box warning, no impact on albumin levels, no REMS program, no monitoring requirements and no need to vaccinate Consistent with our goal to bring VYVGART into earlier lung treatment settings, we also presented new real data that patients can meaningfully reduce steroid use within 6 months of treatment. This effect was maintained and improved through 12 months with 1 out of 4 patients taking to 0 milligrams of steroids and 42% of patients taping to 5 milligrams or less. A&M was notably the first congress where we showcased preclinical programs from our pipeline, including empasiprubart and ARGX-119.
Through these programs, we continue to solidify our leadership in neuromuscular medicine, advancing clinical development across multiple indications, including MG, CIDP, MMN, CMS and ALS and generating early excitement as we address unmet needs with our precision medicines. Slide 6. Before turning the call over to Karl, I would like to highlight some of the opportunities ahead to advance innovation as we approach the end. Remember, we like novel targets with pipeline in the product potential and seek out indications where we believe we can drive the most impact for patients.
Indications continue to mature across our 3 pipeline programs in the clinic, [indiscernible] ARGX-119. Next purpose or my lies go-no-go decision before year end. As you may recall, we thoughtfully designed ALKIVIA, as our first basket study that would allow us to simultaneously evaluate efgartigimod in see success of myositis, necrotizing antisynthetase and dermatomyositis. The primary endpoint of the study is the same across all subsets and is based on the total improvement score. With the SMEs Phase III design, we started enrolling in the Phase III as soon as we completed enrollment in the Phase II, which means that the decision we will make in the coming weeks is whether we will continue recruitment in one or more subsets based on the signal we see in the first 90 patients from the Phase II.
Lastly, as you saw in today's release, we made a decision to discontinue the development of efgartigimod in M&A. As a part of our strategic alliance with [indiscernible], we are leveraging Zai's clinical development in [indiscernible], two kidney indications prevalent in China. We did not see a sufficient efficacy signal from the interim data to warn further investment in MM. Importantly, no safety signals were detected and now we look forward to the Phase II data in [indiscernible]. It is part of our mission to prioritize programs that have the potential to drive transformational outcomes in patients, and we do not see this opportunity with efgartigimod in MM.
To close, we are well positioned to continue investing in our innovation, advancing high-value novel treatments that can improve outcomes for patients and maximize value for shareholders. I will now turn the call over to Karl.
Thank you, Tim. Slide 7. The third quarter 2024 financial results are detailed in the press release of this morning. Total operating income in the third quarter totaled $589 million. This reflects $573 million in product net sales and $16 million in other operating income. The product net sales of $573 million represent 20% quarter-over-quarter growth and 74% growth compared with a corresponding prior year quarter. The product revenue breaks down by region to $492 million in the U.S., $24 million in Japan 46 million in the rest of the world and $11 million for product supply to Zai Lab in China, but net sales in the U.S. is inclusive of both CIDP and MG revenue. Karen will provide additional color in her section on the initial launch performance. Slide 8.
Total operating expense in Q3 are $575 million an increase of $40 million compared to Q2 2024. The increase is due to a $22 million increase in SG&A, reflecting incremental expenses on the CIDP launch in the U.S., an $11 million increase in R&D and an increase of $7 million in cost of sales. Cost of sales is $59 million in Q3. This reflects a gross margin of 90% and which is in line with previous quarters. In Q3, SG&A expenses are $278 million, and R&D expenses are $236 million. This results in operating profit for Q3 of $14 million. On a year-to-date basis, operating loss is $125 million. The quarterly net financial income is $41 million, and it also benefits from unrealized exchange gains of $34 million on our euro-denominated cash balances. After tax, the profit for the quarter is $91 million, and the year-to-date profit after tax is $59 million.
Our cash balance represented by cash, cash equivalents and current financial assets is $3.4 billion at quarter end. The balance increased by EUR 272 million in the quarter and our cash guidance for 2024 no longer applies. The financial guidance on the combined SG&A and R&D spend of $2 billion that remains unchanged. I will now turn the call over to Karen, who will provide details on the commercial front.
Thank you, Karl. Slide 9. It's an exciting time for argenx. I want to first give a huge thank you to the entire team who continue to execute on all fronts to make this possible. Patients are at the center of our innovation mission, and I'm pleased to our clear patient-centric strategy and disciplined approach has delivered continuous growth in GMG while at the same time contributing to a strong first quarter of launching CIDP in the U.S. and ITP in Japan. I'll begin with the details of our CAP launch before zooming out the overall performance for the quarter, highlighting the dynamics driving our GMG performance and sharing our outlook for sustained growth.
Slide 10. The team has successfully advanced each of the core strategies that we outlined at the CIDP approval to reach our key stakeholders, patients, physicians and payers. Let's begin with payers. We're enabling broad patient access has been an area of core focus. Since CIDP approval, it has been our priority to secure access as quickly as possible leveraging the credibility and the strong partnerships we established with pay us through our MG experience. Today, we are pleased to have broadly favorable policies in place that cover approximately 54% of U.S. commercial lives, most but not all policies require some prior IG utilization. This is not surprising to us and supports our expected growth trajectory because most CIDP patients have prior IVIg experience and is commonly part of diagnosis.
Important to note is that for those policies that require prior IVIg use is usually exposure of 3 months or less at any time with the patient's history which is favorable for VYVGART Hytrulo. It typically takes 2 quarters to get most policies in place after any launch, and we're on track with this time line with the remaining policies expected to come into place over the next few months. Overall, payers recognize the value that Hytrulo can bring to patients. Moving on to prescribers. The strength of our data and the clear value proposition of VYVGART Hytrulo to patients is also driving initial uptake with physicians.
Neurologists are excited to gain experience with the first CIDP innovation in 30 years of safe and effective treatment that has the power to drive functional benefit in patients with a 30- to 92nd simple injection. At the time of launch, we shared that we have an estimated 10,000 total neurologists spanning CIDP and MG, with approximately 72% overlap in physicians who treat both. We expected that physicians with prior got experience would most likely be the first CADP prescribers, but our goal was also to broaden the VYVGART prescriber base.
I'm pleased to share that 25% of CIDP prescribers to date of first time did got prescribed and some who started with CIDP have now also prescribed the GMG. We are seeing initial bread food out prescriber base as well as depth. Many prescribers have already started multiple patients on treatment.
Lastly and most importantly, we are thrilled to see innovation deliver tangible impact as VYVGART Hytrulo reaches CIDP patients in need. Expanding on Tim's earlier story, were inspired by the feedback we've received so far. Many patients are able to enjoy more moments with the ones and regain the independence to do the activities that you and I often take for granted. We are pleased to share that as of the end of the quarter, there are over 300 patients on VYVGART Hytrulo, delivering a strong quarter of CAP revenue, which approached that of our first MG quarter. Of these patients, 85% to 90% are coming from IVIG which supports our position that this would largely be a switch market.
Overall, the team has done an incredible job getting patients on treatment smoothly, and we'll continue to monitor how the launch trajectory progresses. We will specifically be looking at response rates, patients getting on and staying on treatment and how utilization trends over time. In particular, in light of the OLE data that we will share next year with biweekly or every 3-week dosing. Now let's turn our attention to MG, Slide 11. MG drove the majority of growth over the quarter with continued momentum and steady growth across VYVGART and VYVGART Hytrulo.
In the U.S., Hytrulo continues to attract both new patients and new prescribers, this is important for a couple of reasons. First, over 60% of new VYVGART and Hytrulo patients are coming from orals, which is consistent with our goal to reach earlier line patients. Second, we continue to expand our prescriber base for MG with Hytrulo and 12% of prescribers were brand new to VYVGART. Our goal is for patients not to have the constant reminder of their disease and we hope to achieve this with our efficacy results delivering MSC in over half of our patients and our demonstrated safety.
We're generating real-world evidence that shows that more consistent cycles leads to sustained efficacy for patients and that over half of patients can meaningfully taper off steroids after they've got initiation. We also aim to enhance the patient experience by offering both IV and a simple subcutaneous option. VYVGART Hytrulo was an important step forward with this strategy and advancing our prefilled syringe is next. A significant amount of innovation went into this regulatory submission, and we're looking forward to the PFS PDUFA date in April 2025. We Slide 12. We also had strong momentum in MG sales globally and are proud that VYVGART is now available to over 80% of the gMG population in the EU.
The ITP launch in Japan is showing early success and the data from advance are playing out in the real world with 50% of patients responding. And last, we are working to bring our innovation to CIDP patients worldwide. Regulatory reviews are ongoing in China, Japan and Europe with approval decisions on track for 2025.
Slide 13. We have a lot to look forward to. There is significant opportunity in the pipeline as we expand into new diseases that all have high unmet need for innovation. And the commercial organization is laser-focused on executing our plan to maintain momentum and growth in MG, CIDP and ITP, all to fuel our differentiated pipeline and broaden our patient impact globally.
I will now turn the call back to Tim.
I'd like to thank the argenx team for their ongoing commitment to changing the lives of patients through our signs. As we approach the end of the year, we are part of all that we have accomplished gaining invaluable insights along the way to solidify our deep understanding of immunology. We are energized by the opportunity ahead to amplify our impact recognizing we're still early into the CIDP launch and have the potential to reach new patients across multiple indications in the clinic. With Grand Insights, our best-in-class innovation shows no sign of slowing down as we continue to build out long-term value with VYVGART and move swiftly to advance our promising clinical candidates. Thank you for your time today. I would now like to open the call to your questions.
[Operator Instructions]. Your first question today comes from the line of Derek Archila from Wells Fargo.
Congrats on the strong performance here. Maybe just first off, I was hoping you could talk about the trajectory of the CIDP launch. Since the two patients Karen talked about the R&D Day to now be over 300 at the end of the third quarter, has that been fairly linear? Or was there a recent bolus? And then just a follow-up to that. I guess how do you think about some of the variables in the CIDP launch? And how does that figure into your thinking on potentially issuing guidance in 2025?
Thank you, Derek. Thanks for being with us today, and thank you for the compliments on a very strong quarter. I think this is a question for you, right, Karen.
Yes. Thanks for the question. And first of all, thanks for recognizing the strong quarter, I think we're really proud of the team and the disciplined execution, the focus that they have delivered a strong quarter. In terms of your first question, we have not seen a bolus in the CIDP launch. And to you your phrase, I would say the patients start have been relatively linear through the quarter. As with any launch, it takes time from when the prescription is written to when the patient gets on therapy as payer policies have come on board through the quarter.
So the majority of our patients start were in September. But there has not been a bolus that we've seen. On your second question related to what we're seeing in terms of the CIDP launch dynamics, I would say we're exactly where we thought we would be, and we're really proud of the strong start that we have. In terms of how we laid out the strategy, the payer policies, we said they would take 2 quarters to come in to get the payer policies in place, and we're right on track with that. We shared that we have about 54% of commercial lives covered, and we expect that the payer policies will continue to come in over the next quarter. We've certainly seen strong uptake amongst physicians. We shared that 75% of the CIDP prescribers have had prior they've got used, but we've also seen a broadening in the prescriber base. So of prescribers with CIDP are new to the VYVGART franchise. And in fact, some of those that wrote for CADP first have also now started a patient on MG.
So that's also a really good signal. The reflection from the neurologists is that the regain of function data is resonating very well with them and with their patients. So I would say, we're off to a strong start. The dynamics are exactly as we expected. Moving forward, what we plan to provide in terms of launch metrics is not guidance on revenue or number of patients, but rather we'll continue to provide those metrics or those KPIs that are most relevant to how the launch is progressing. Thanks for the question.
Our next question comes from the line of Tazeen Ahmad from Bank of America.
Maybe this is also for Karen. I think at the beginning of the launch, you talked about the time it might take to get on to formulary and the time it might take for a patient who received treatment for CIDP from the time of [ script ] is written. Can you give us an update on both of those metrics, maybe a little bit more focused on the latter because I think people are still wondering if your initial cautious guidance at that time it would take to receive treatment is still true? Or is that trending above expectations?
Yes. Thanks for the question. Happy to address this. So with any launch, actually with any patient start, there is a delay between when the prescription is written and the patient actually received the treatment. And at launch, this is often longer than once you're in steady state because payer policies are coming online during that time. What we've seen for CIDP is in line with what we saw for MG and in line with what our expectations are. So patients are moving relatively smoothly from prescription to patients to getting on therapy when there are payer policies in place. And I really do want to recognize the team for their efforts, not just to getting payer policies in place, but also to the team that does support getting patients through that process and getting them onto treatment.
Our next question comes from the line of James Gordon from JPMorgan.
James Gordon, JPMorgan. The question was just if we try and break out how much of sales came from CIDP or MG during the quarter, would it be fair that most of the ads were still MG, and it was something like a 20 million add in terms of CIDP? And if that is right, is that pretty much all in the last month based on when the patient came on to therapy. So is that then a figure that we could annualize -- or would we need to be a bit careful sort of extrapolating what you did in the final month of the quarter and what that would equate to on a yearly basis?
Thank you, James, for the question. These are actually two questions in one. I think, Karl, why don't you comment on question #1 and then we can handle question number two, I can.
Yes, thank you, James. I mean, I think directionally, your numbers are correct in terms of -- we have 300 -- more than 300 patients on treatment or CIDP at the end of the quarter. That compares to 380, which we had for MG and during the first quarter for MG, we had $21 million of sales. So your extrapolation to CDP makes sense because most of those patients came on board or started initiated retreatment, I should say, at the end of that quarter in September. So I think that makes perfect sense.
And then on your second question, James, a quick talk from Karen, right?
Yes. In terms of extrapolating moving forward, look, I'd say it's still early days. I wouldn't try to get ahead of ourselves just yet. We still have a few dynamics that we need to see play out in the CIDP launch. Just a few of those, really quick. Don't forget that with the ADHERE data, there was a 67% response rate. So we need to see how that plays out in the real world. We also have open-label extension data that we'll be sharing next year on biweekly and every 3-week dosing. So we need to see what real utilization looks like. And of course, there is competition in this market as well that we'll have to be watching. So I'd say it's still early days.
Your next question comes from the line of Allison Bratzel from Piper Sandler.
As the quarter I think you indicated that around 85% or 90% of CIDP patients have switched from IVIg. Could you just talk to those who aren't IVIg switchers Were any of those newly diagnosed patients? Or were they coming from a different therapy? Any color there would be helpful.
Thanks, Allison, for being with us today. So it is absolutely right that about 85% of patients are actually on IVIg when they come on to Hytrulo. Remember that's perfectly in line with expectations, right? The lion's share of the CIDP patients are on IVIg that's also used as a diagnostic tool in the diagnostic journey. So no surprise the other 15% or so of patients think of them as mainly coming from steroids and other immunoship [ residents ]. Thank you for the question.
Your next question comes from the line of Myles Minter from William Blair.
Can you guys give us a little bit more color on exactly what the prior step-through therapy on immunoglobulin looks like in that payer formulary coverage for CIDP. Like is it you have to respond and then go into SCIG maintenance and then relapse and vacation that would be considered successful in a prior setting? Or is it really up to the clinician to see how their patients are responding to IVIg before they can kind of tick that box and put them on to a bit [indiscernible].
Thanks for the question. So we're really pleased with where we've landed with our payer policies. I'd say they're generally favorable. And as you said, they generally do have some IVIg or prior experience in general. And what that means is that it's not -- that the -- there has to be some documentation of prior IVIg or subcutaneous IG use. But as you say, there's not a documentation of, for example, a response rate or IROs or any of those measures that need to be used. So it is favorable. It's also generally prior IVIg or subcutaneous in a lengthy period of time sometimes through the patient's entire treatment cycle. So the policies are favorable, and we're pleased with where they're at.
Our next question comes from the line of Alex Thompson from Stifel.
Congrats on the quarter as well. I guess as a follow-up to the payer question. I guess, can you talk a little bit about your discussions around this IG experience? And just given the broader population of it here, do you expect to sort of be able to push past that in the future? Or is this sort of how you expect to see payer policies moving forward?
Yes. Alex, I guess this is a variation of the same theme. So typically pay policies position us behind IG, whether that's IV or subcu Ig, which is not surprising given the price point, the data, which really resonates very well with BASE is a region of function data. That's where they see value above and beyond the current therapies out there. And when we say that the payer policies are typically favorable it basically means that there needs to be some sort of documentation at some point in the past where the patient had an insufficient response to IG or and/or a tolerability issue. That's how the language reads some of these policies are actually visible publicly. But that's what it is. And we think that's not really going to be a practical handicap to the launch of the product.
Your next question comes from the line of Danielle Brill from Raymond James.
Congrats on the strong start of the launch. So on a percent of TAM basis, it looks like you captured more of the CIDP population right out of the gate than you even did with MG. Has this early strength impacted your thinking on the overall size of the opportunity? I mean to ask you a little more directly, you said you're committed to treating 50,000 patients by 2030. Could you potentially get there with just energy and CIDP? Thank you.
Thank you, Danielle. This is an excellent question. And maybe, Karen, you would like to take it.
Yes, certainly, I'll talk to this. So in terms of the CIDP, we are off to a strong start, exactly as you say, and with that early signs being strong. The total patient population that we shared for CIDP or the addressable market is 12,000 patients, and we're not changing that. We believe that that's still the patient population that the addressable market, and that's those that are uncontrolled on IVIg. We believe that between MG, CIDP as well as the expansion indications that we have coming over the coming years, we're excited to look forward to the 50,000 patients in 2030, but that goes beyond MG and CIDP.
Your next question comes from the line of Akash Tewari from Jefferies
This is Amy Li on for Akash. Congrats on the quarter. Is there a potential for you to combine your C2 with VYVGART and CIDP? And when can we start seeing -- when can we start these studies? And what could they show? And then also, when can we get an update on your remaining Zai Lab trial in lupus nephritis? And what's your internal confidence in this?
Yes. Amy, thank you for the questions. that is probably always opportunity to start combining certain therapies in severe community just like people are doing in cancer. For the moment, we're really focused on launching [indiscernible], which I think has shown unparalleled data in CIDP, just launching that product into the time which we just have been discussing. We do know that complement is in place probably as a result of all what is driving that. So we think mpa deserves its own individual shut on goal in IDP. So combo is a theoretical possibility. Let's not take it off the table, but we have plenty of work set out for us for both VYVGART and MPA.
With Zai Lab, remember they were doing 2 proof of concept trials to help us with the big volume of work we need to lift 2 kidney indications, memories property and [indiscernible]. Both studies have been executed very well by Zai or being executed very well by Zai. So stay tuned when the data become public, we will definitely talk about it. So far, the trial is enrolling very well. and we are very pleased with the quality, which Zai has shown in its clinical trial execution across both indications. Thank you for the question.
Our next question comes from the line of Yaron Weber from TD Cowen.
Congrats on a really terrific quarter. Maybe a quick question, the myositis GO/NO GO decision is coming up in 3 indications. We all know that the Imam passive transfer data was the sort of the best objective support moving into Phase III, but there's a lot of biological support for the other 2. Maybe just how do you feel about all 3 indications?
Yes. Thank you, Yaron thank you for the question. You're right to call out myositis because this is the last data card, which we would like to turn before we leave a very productive and successful 2024 and equally strong biology conviction across the three with a slight preference indeed, as you call out for M&M, I think that is convincing biology in place for all three of them. I think the fields in myositis is evolving. More and more are people classifying the subsets of myositis not just based on clinical symptoms, but also serology, i.e., the presence of these pathogenic IgG autoantibodies in the serum of the patients.
So I'm positive about all 3 based on what we know about the biology. There's always risk associated with the clinical experiment. And that's, I think, why we made a responsible decision to do that go on or go decision point once we're enrolling at risk, of course, the Phase III trial. So in practice, what you will see us do is just make stop decisions in case they are warranted but the Phase II is enrolling in all 3 subsets.
Your next question comes from the line of Vikram Purohit from Morgan Stanley.
Sorry if this was discussed already and we missed it, but I was curious to hear your thoughts on what you're seeing in terms of dosing frequency. for CIDP patients that have started therapy, especially for those that started in July and August?
Yes. Thanks for the question. So it's a little too early for us to tell. The majority of patients are just getting started on therapy for -- so -- and what we know is, as I mentioned earlier, that we have the open-label extension study coming with the biweekly and the every 3-week dosing. So we really need to see how it plays out with real-world utilization as we get more patients on therapy.
Thank you, Karen. And Vikram, I think it's fair to assume in an early launch that typically patients will be dosed according to label.
Our next question comes from the line of Charles Pitman from Barclays.
Charles Ptman for Barclays. And I'll also add my congratulations on the very strong quarter. Just following up on the dosing frequency. I was wondering if you could just reiterate to us your expected or you're currently booked price for Hytrulo in CIDP, originally, the 450,000, I believe was an expected balance between weekly and biweekly obviously, given the early launches made in line with the label that suggests more weekly, so potentially a higher level of cost booked. So if you could just kind of outline what your assumptions behind that are and how that's progressing, that would be great.
Thank you, Charles. It's Karl here. Yes, the 450 is the number we came out at launch. And just as a reminder, how we got to that 450. It's, of course, it depends on the number of true vials. So we utilization -- it needs to be taken into consideration. So at the moment, we all know the study was done weekly. But in the real world, we will have to see how that play out. We have no new information to talk about. And then, of course, you also have to talk about gross to net, but which is part of that $450,000. So I think it's too early to talk about to give more information now.
Our next question comes from the line of Samantha Semenkow from Citi.
Let me just add my congratulations on the strong quarter. Just turning back to the pipeline for members nephropathy. I guess maybe could you give a little bit of background on what drove the initial interest in this indication? And a little bit more color on your thoughts on the lack of a sufficient signal in this disease. Is there any read-through to other indications in development?
Thank you for the question, Samantha. So first of all, that was conviction. And then based on the convincing biology or that this is indeed a pathogenic antibody, which is mediating the disease biology we know the identity of the antibody. We know that the tighter correlates with disease severity and so on. So typically, the facts which we like to see in order to build confidence we were alerted by the DSMB throughout the study that basically there is no real signal here, and they [indiscernible] consider continuation of the study. That's where we decided to take a look at the data and make this decision.
It comes a bit of a surprise, that's why you do Phase II trials. I mean you want to further validate the biology before you make a Phase III investment. It's actually perfectly serving the purpose. The only thing we can think of at the moment is that and that is specific for MN, these patients lose a ton of protein to the kidney. So these kidneys are extremely leaky. And we're going to double-click on the data set when it comes to exposure to the drug but I would certainly not look at NAV to other indications, but this is actually not the case. So stay tuned, we will be communicating more about it later.
Your next question comes from the line of Gavin Clarke Gartner from Evercore ISI.
Congrats on all the progress. I was just wondering for the over 300 CIDP patients that were on treatment at the end of the quarter, are all these reimbursed patients? Or are any of these patients on ridge or free drug supply, I'm still awaiting reimbursement approval?
Gavin, thank you for your question and by all reimbursed.
Your next question comes from the line of [indiscernible] versus from Kempen.
It relates to pipeline news flow, I understand you guide on time lines when appropriate, but there's still a lot of impact, so to say. So can you frame a bit what we should expect for the coming 12 to 18 months. For example, for VYVGART would be help to understand the sequence. We should expect the registrational trials to oil out data. And on the other hand, for MBA and 119, a number of mid-stage trials have been ongoing for a bit. So. Yes. Can we expect the data rich ahead year ahead for these pipeline assets?
Yes. Thank you, Suzanne, and thank you for being with us today. typically the start of the new year, we would announce the clinical calendar for the year. So expect us to show up at JPMorgan with a more granular view on what 2025 will bring in terms of clinical milestones. You're right to call out, there's going to be a lot of activity. If I do the math right, I think we will have between 5 and 10 Phase III trials only and then, of course, a whole slew of Phase II work and then a number of IND candidates getting into the clinic. So for the moment, focused on ending a strong years, focusing on executing the last elements of the plan, including hopefully a strong Q4 and positive IMM data. And then on the podium in January, we will be talking to you about a detailed clinical calendar for 2025. Okay? So stay tuned. Thank you.
Your next question comes from the line of Joel Beatty from Baird.
Congrats on the quarter. For the 300 patients who started in CIDP where those patients that generally had formal payer policies in place? Or were some of those kind of one-offs without formal policies in place yet.
Yes, thanks for the question. It's a mix of patients that had policies in place and did not. Generally, for policies in place, the patients are able to get on therapy more quickly. But we have -- at this point in launch, we have a mix.
Your next question comes from the line of Joon Lee from Truist Securities.
Congrats on the strong quarter be curious to know your latest thoughts on the opportunity for ARGS 1:1 in IgA nephropathy given several competitor presentations at ASN last week, how do you think ARG 124, the sweeping antibody can differentiate versus, say, those targeting CD28 or bath and April?
Yes. Thank you, Joon, for the question. I think what we see is very exciting. I mean, IG nephropathy is a disease which is waiting to be unlocked by innovation, and you basically start to see the first generations of innovation coming into the space. But I think this is going to be a sizable opportunity is going to be an opportunity, which will require multiple generations of innovation to really get there. I think what we have seen for the moment is encouraging data, but clearly room for improvement.
And that is, I think, the promise, which is held by argenx 121. I haven't seen any program, which is so selective and precise in eliminating the bad guys and doing that with the speed and the depth of response, which is just unparalleled by any of the data we have seen. So I think the design of the molecule is strong. Now we need to wait for the clinical experiment and really see with the [indiscernible] strength of design translates into superior clinical efficacy. So I'm very excited about this molecule. Remember, it can play in more than one indication. argenx property is one which speaks to the imagination, but there are, of course, more indications on our table because we typically like to pursue pipelines in the products. Thanks for the question.
Your next question comes from the line of Victor Flock from BNP Paribas.
I do have a question on the retail syringe opportunity. I mean, we've seen true driving earlier line penetration in MG. So on paper, PFS looks like a game changer for patients. But in practice, I was wondering if you are expecting it to be also a significant driver to the [indiscernible]. And if I can squeeze just another one. I was wondering if you can give us a bit of visibility on when you will be able to launch the PSL x U.S.
Thank you, Victor, for the question. I think this is a question for Karen.
Yes, absolutely. Look, we're really excited for the April PDUFA date for PFS. And I think what it will do is continue the momentum that we've seen in and continue to sustain the launch in CAP. So it really aligns with the strategy of providing not just the best efficacy and safety but also convenience for patients, particularly with our goal of self-administration. So that we can really compete very well for patients in both of these markets. And it aligns with our strategy of early aligned use in MG as well as broadening the prescriber base. So we're really looking forward to the April PDUFA date.
And then do you want to just touch on when we expect to launch PFS x U.S. second part
Yes. So we have filings ongoing for the PFS ex U.S., and they will follow over the coming months and years.
Your next question comes from the line of Xian Deng from UBS.
So I have a question on CIDP, please. I understand that 85%, 90% of patients are actually coming from IVIG. But just wondering what is the typical sort of patient poultries look like? So just wondering, are these patients who really have very poor disease control with IVIG and the switching or do you see actually patients with actually very good disease control but the switching for convenience? Thank you very much.
I think the typical phenotype of the patients you would see so early in the launch are actually patients which have a real difficult time tolerating the drug or who are really weakening in between IVIG cycles or actually don't have full function. So I think these would be the 2 main drivers for these early patients coming on drug and the convenience of dosing does not go on in August. I mean you need to imagine that a lot of CIDP patients who would be on IVIg, which spend 1 to 2 days a month in an IV infusion shares, typically spending the bulk of the day in that share.
And then, of course, the 30 to 902nd subcu injection is very compelling. But I would say, right, Karen, it's fair to say that it's mainly driven by insufficient response to IG and/or tolerability issues.
Absolutely. And it's exactly what we would expect at this moment in launch.
Your next question comes from the line of Leland Gershell from Oppenheimer.
Congrats on the quarter as well. Just teeing off the earlier comments on the decision to discontinue MN, -- is it fair to say that, that was based primarily on biomarkers versus having to need to see lack of clinical response, and maybe more broadly, as we think about focusing R&D into the future, is it fair to say that argenx comply a fairly high bar with respect to Go-no-go decisions given the breadth of opportunity and as you look to achieve consistent profitability.
No, I like that question. I think your second point is a very important one. I mean we're looking for indications where we can have a truly transformative effect, not a marginal effect or an incremental effect. It needs to be a game-changing effect. And that's where we will basically put the mission of the company to work here, I think it's very well known and then that there are a couple of protein biomarkers in the urine, which would basically be broadly accepted as proxies for clinical efficacy. So
I was not close to the data. That was an EDF in the company, chaired by our Chief Medical Officer, who was close to the data, but I believe it was based on an EGFR or protein urea market. Thanks for the question.
Your next question comes from the line of Andy Chen from Wolfe Research.
On your pipeline, for both 213 and the IgA drug, can you remind us if the 2 drugs have been optimized into subcu formulation because the data so far looks like it's IV? And then also, if you can quickly provide an update for the auto-injector status as you can.
And thank you for the question. Obviously, the drugs we design are getting the secure possibilities in them. So yes, we do have the subcutaneous product presentation in the plant for both molecules, and we strongly believe we can get there. From an auto-injector point of view, it's a bit premature for us to be public on time lines. We are fully focused on PFS. But I think you're right to call out that we are working in the background already very hard on that auto injectors, where we said already a while ago that we have passed the prototyping phase. We're into the industrialization phase. So that project is going forward at full speed. But we will be communicating about time lines when we get closer to a final product. Thanks for the questions.
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Your next question comes from the line of Rajan Sharma from Goldman Sachs.
Just on the CIDP launch, obviously, you kind of talked to number of patients on therapy after the first quarter relative to myasthenia gravis. Just thinking about kind of trajectory from here and run rate, would you kind of cautionary gain utilizing that same run rate that we saw that they've got in myasthenia CDP.
Yes. Thanks for the question. And we're asking the same question. I think it's a good one. Since we're pleased with where we're at. But as I said earlier, I think it's important that we don't get ahead of ourselves. The market dynamics in CIDP are quite different than MG. So you'll recall -- it's a switch market, whereas MG was an add-on market, the competitive set is different. The patients are different and also our data is different. So look, in particular, what I think is important to take into account is that 67% response rate in it here. Let's see how that plays out in the real world. And it's still early days in terms of the competition and seeing VYVGART out only for 1 quarter. Let's also see what the response is there.
Your next question comes from the line of Thomas Smith from Leerink Partners.
Let me have my congrats on the strong quarter. You saw you had a very strong presence at the recent AANEM meeting and there were a ton of also quite a number of competitor data sets there in MG. So wondering if you could just comment on how you're thinking about some of these emerging targets like CD19 and then separately, I wanted to ask about thyroid eye disease. And it sounds like the Phase III is progressing as planned, but I was wondering if you could comment on enrollment. Just remind us what your expectations are when a Phase III top line readout.
Thomas, thank you for the question. I think you're right to call out our leadership at ANM. I'm very proud of the continuous stream of data, which we continue to produce as a market leader in the first-in-class agents we continue to see in the real world that we have put a very high bar. If we talk about more than 50% MSC, if we talk about more than 80,000 patients worth of safety data and then the absence of REMS programs, fascination requirements, labs, no albumin issue.
And then, of course, from a convenience point of view, we're the only ones showing such a spectrum of dosing possibilities which really allows you to tailor to the individual lead of the patients. So we've put a bar high, we see competitors coming into the space that I haven't seen any particular data which is actually coming close to the high bar, which we have set -- we also continue to evolve and lead the space by moving upstream in the treatment paradigm. I think we have shown impressive steroid tapering data. I'm very proud of the fact that after 1 year, 25% of patients are of steroid. It's a big deal and more than 40% of that 5-milligram or lower a day that again, is a paradigm shift in the treatment of myasthenia. So our conclusion of all the new data coming to the spaces we've put the bar very high, and we continue to shift that bar. Thank you for the question.
Your next question comes from the line of Yatin Suneja from Guggenheim.
This is [indiscernible]. Congrats for the quarter. for the sugar program, can you please clarify if you have reached alignment with the FDA on the pivotal program? And is there any color you can share on trial design and statistical plan.
Thank you for the question. I think we're on track to start the study before the end of the year. I think that's the key cash for our investment audience. We did have the appropriate FDA interactions. So if you were align them, we have calibrated study design and endpoints. So we're actually in operationalization motors and we're on track to start the study before the end of the year. By the way, on TED, there's a question I forgot to answer a minute ago. You're right, everything is on plan and on track. But too early, of course, to talk about completion dates for both TED and sugars. Thank you for the questions.
Your next question comes from the line of Simon Baker from Redburn Landec. Simon, your line is open. And our next question comes from the line of Douglas Tsao from HC Wainwright.
I'm just curious on the commentary regarding the CIDP launch leading to new prescribers in MG I'm just curious in terms of the profile for some of those prescribers who are now starting. Were they do not have big practices in MG, Were they just not familiar with the product? And just -- do you have any color in terms of why just given the such early success you had in MG, why this was the catalyst for them? Congrats on the results.
Yes. Thank you for the question. Look, I would say you will recall that when we were characterizing the CIDP opportunity, there's a lot most CIDP patients that are out with community neurologists than there are MG patients. However, there are MG patients also with community urologists. So we've broadened our target list in line with the CAP launch and that is reflected by the field force expansion that we also invested in, in preparation for the CIDP launch. So with that field expansion and the target expansion, we're reaching neurologists. Some of these are new to VYVGART and some of them, as you heard, have started with CIDP and then expanded their prescribing to M&G.
Our next question comes from the line of David Seynnaeve from Petercam.
Regarding the CIDP trial with AMP, but related to 1 of the earlier questions asked. Can you say a few words on your decision to go immediately into a registrational study, what your conviction is based on? And maybe what exactly you expect from the outcome of study keeping in mind the data you've generated with FFO already?
Thank you, David, for the question. Look, there remains work to be done in CIDP. No one has shown a higher response rate than we did with War in the trial. If you're fair, that's actually a 70% response rate in Stage A. The question is what happened to the 30% patients who did not respond in that first 12 weeks. We have also seen the data from Solovis program underlining the rolloff complement, clearly complement being recruited by the disease causing antibodies. And based on the in-house [ Xevo ] data, the data from the field, we think it is warranted to launch [ Mpala ] into Phase III, especially given the regain of function data we have seen in MM think of MMN as the little sister indication of CIDP.
So if you look at the totality of data, we did not want to lose any time in terms of Phase II work we think it's warranted to go forward into Phase III. So we're very excited about the opportunity. And remember, there's a ton of work to be done still in CIDP.
And your next question comes from the line of Emmanuel Papadakis from Deutsche Bank.
Maybe just a follow-on on the CIDP launch. Given you're starting to get a bit of feel for the market in the U.S., perhaps you could give us your best current estimate of the absolute number of CIDP patients in the U.S. that are either refractory or unsatisfied from a convenience perspective with their current IG treatment? And maybe just in a quick addition, reimbursement time lines in Europe CODB, could that start to contribute next year? Or is that more of a 2026 then.
Yes, thanks for the two questions. So we have sized the addressable market for CIDP as 12,000 patients that we believe that are on therapy but have residual symptoms or are unsatisfied with their current therapy. So that's the addressable market that we see. In terms of ex U.S., the CIDP filings are ongoing, obviously, pricing and reimbursement takes a little bit longer. But as soon as we have those approvals in hand, we'll be working diligently and quickly to get access for patients across the globe.
And your final question today comes from the line of Simon Baker from Redburn Atlantic.
A question on VYVGART. And I wonder if you could give us any qualitative commentary on rebating and formal negotiation trends into 2025. And how's the addition of CIDP as an indication affected those negotiations meaningfully at all at this stage?
Simon, thank you. It's Karl. Thank you for the question. What I can say is where we are today, gross to net continues to be around 12%, that's public, that's in our financials. We don't see a deterioration there. And I think that's what you can expect for the rest of the year. For 2025 and with preferring all of those variables, I think it's a little bit too early to talk about that, and that can be part of a later discussion. Thank you for the question.
And this concludes our question-and-answer session and does conclude today's conference call. We thank you for your participation today. You may now disconnect.