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spk04: Good morning. My name is Rob and I will be your conference operator today. I would like to welcome everyone to the call. At this time, all lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you'd like to ask a question during this time, simply press star followed by the number one on your telephone keypad. If you would like to withdraw your question, again, press star one. Thank you. I'd like to introduce Beth DelGiaco, Vice President of Corporate Communications and Investor Relations. You may begin your conference.
spk02: Thank you, operator. A press release was issued earlier today with our first quarter 2023 financial results and a recent 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 on slide two that forward-looking statements may be presented during this call. These may include statements about our future expectations, clinical development, regulatory timelines, the potential success of our product candidates, financial projections, and upcoming milestones. Actual results may differ materially from those indicated by these statements. Argenix is not under any obligation to update statements regarding the future or to conform those statements in relation to actual results unless required by law. I'm joined on the call today by Tim Van Haramaren, Chief Executive Officer, Carl Gubitz, Chief Financial Officer, and Karen Massey, Chief Operating Officer. I'll now turn the call over to Tim.
spk24: Thank you, Beth, and welcome, everyone. Flight three. I'm pleased to be here today to share another quarter of strong execution with our global safeguard launch. We continue to deliver results, both in our ability to reach patients and drive revenue, but also with the progress we are making on the regulatory side. We recently announced regulatory approvals in Israel and the U.K., marking our fourth and fifth approvals globally, and we expect more to follow later this year. This is a remarkable achievement from our global submission teams. We are preparing for additional regulatory milestones with the potential approval of SubQ FGATIGAMOP in the United States. Our PDUFA date of June 20 is fast approaching, after which we hope to have the broadest GMG offering for patients, both with an IV infusion and a simple subcutaneous injection. We know that gaining traction in early-line patients will be important for the continued trajectory of our launch, and the approval of a sub-Q product may help us achieve this. I was able to attend the AAN meeting last week where we had a significant presence, demonstrating our long-standing commitment to the neurology community. We had six aspects accepted for presentation, including new data from the ADAPT over-label extension studies, highlighting the consistent efficacy and safety profile of both IV and sub-QL More importantly, we were able to connect with leading neurologists, many who are using DivGuard in their practices.
spk26: Slide four.
spk24: When we first started development in GMG, we made a commitment to the patient community. We had listened to the challenges they faced with long diagnosis journey and significant disease burden and wanted to create an entirely new standard of treatment elevating the expectations of physicians and patients on what well-controlled means. It was clear from our interactions at AAN that we are achieving this with VisGuard. We heard stories of patients where VisGuard had a transformational impact and it felt like we had come full circle in honoring that initial commitment. Beyond the upcoming subcutaneous approval decision, We also have a busy upcoming quarter and remainder of the year with our pipeline. We continue to expect five data readouts across FGAR-Tigamot and Dargenix 117, all of which will showcase the depth of potential within our immunology programs.
spk26: Slide 5. First with FGAR-Tigamot.
spk24: Since our last call, we crossed the 88 event mark in the ADHIEV trial and are making progress towards the readout of top-line results in CIDP. Reaching 88 events does not mark the end of the trial, and the team still has to gather final data points from patients and complete some mechanics associated with rollover to the open label extension study. This is an important readout for us and for the CIDP community. We want to put ourselves in the best position to bring in new innovation to CIDP patients as quickly as possible. As part of this, we will give our team sufficient time to close the trial and review the data in a high-quality way, which is why we now expect top-line data in July. We also have exciting updates from the advanced sub-Q and ADDRESS trials, both of which have completed enrollment. This puts us firmly on track to have top-line results in immune thrombocytopenia and pemphigus in the fourth quarter of this year. All of this sets us up to have notable potential approvals over the next two years, advancing us towards achieving our 2025 vision of reaching autoimmune patients globally across our franchises. With ITP, this process will start in Japan in the middle of this year, when we expect to file the dossier for approval based on the IV data. The rest of our Rev. Gartingelot programs remain on track, and we expect to start several trials in new indications and report initial results in post-COVID thoughts Boruspensigoid, myositis, jergens, and membranous nephropathy all before the end of 2024. Slide 6. We also expect the first clinical efficacy data on ARGENIX117 in the middle of this year from the phase 2 trial in multifocal motor neuropathy. We have a couple of objectives with the first cohort of nine patients. We want to confirm the safety profile we observed in the Phase I Healthy Volatility Study, but now in MMM patients. We also want to understand the extent of complement blockade we can achieve with the initial cohort one dose scheme. Based on what we learned from that and the corresponding efficacy results, we will start to build out a key KPD model that will ultimately be used to identify our Phase III dose level. And lastly, we want to understand whether we can maintain or even improve efficacy scores when switching from IVIG to ARGENIX 117. So the trial, while a small initial sample, will provide us with a lot of information.
spk26: Slide 7.
spk24: And last, within our wholly owned pipeline, ARGENIX 119. We started the phase one trial earlier this year and will be evaluating single and multiple sending doses. In the higher dose levels, we will also evaluate responses in a congenital myasthenia syndrome patient cohort. Before I turn the call to Carol, I want to take a moment to thank our teams. The feedback we hear from physicians at AAM is just a small window into the achievements over the last year particularly from our field teams who engage from-line with physicians. We are over one year into our GMG launch and are preparing for the second launch with SubQ. This is still just the beginning of what we hope to achieve on our mission to transform autoimmunity. With that, I will turn the call over to Karl to talk about our financial results.
spk25: Thank you, Tim. Our first quarter 2023 financial results are detailed in your press release from this morning. I will only highlight the key points here on slides 8 and 9. We generated $230 million in total revenues for the first quarter, which includes $280 million in global net product revenues, $1 million in collaboration revenues, and $11 million in ever-operating income. Cost of sales for the quarter were $18 million. In looking at the breakdown of our global product sales, you can see that $196 million was from the US, $10 million from Japan, and $12 million from Europe and our distributor markets. With Europe, the large majority of revenues comes from Germany, and this will be the case for the rest of the year. even with a price adjustment which will take place through the Amnok process. Recall that we will land on our negotiated price in September, but will start to recognize revenue at that expected new price six months ahead of that, so effectively March 2023. Our R&D expenses for the first quarter were approximately $166 million, and can mainly be attributed to the external research and development expenses and personnel expenses incurred from clinical development activities. Our SG&A expenses for the first quarter were $149 million and was mainly driven by professional and marketing fees linked to the commercialization of VivCard in the US, Japan, and the EU. We ended the quarter with $2 billion in cash, cash equivalents, and current financial assets. Based on our current operating plans and the projected 2023 cash burn of approximately $500 million, we expect our existing cash, cash equivalents, and current financial assets together with anticipated future product revenue to fund the company to profitability. You can find additional details behind these numbers in the press release we issued this morning. I'll now hand the call to Karen for a commercial update.
spk03: Thank you, Carl. Slide 10. It's been an exciting first six weeks for me at Argenix, and I'm delighted to be working with an exceptional group of colleagues who are creating value quarter over quarter, not just commercially, but truly across the business. When I first made the decision to join Argenix, it was the culture that initially drew me in and the innovative science and patient-driven mission that made me realize the uniqueness of the company and of the opportunity. I spent most of the time in the last six weeks meeting the team, listening to the conversations among them, and learning. I've witnessed a team of people who are talented, passionate, and engaged. They are at the core of our successful launch and have executed on a strong plan. I believe that the strategies that were developed at the beginning of the launch are the right ones, and now we have the opportunity to expand further on this. We need to optimize how we engage with our key stakeholders to continue to bring value to patients and society. I look forward to building on these achievements together with the team as we expand the VivGuard opportunity into new geographies and new patient populations. Turning now to a review of our recent commercial progress with our global launch. As Carl mentioned, we generated $218 million in global net product revenues this quarter, which shows the continued demand we are getting from physicians and patients. The unmet need in GMG is significant, and with VivGuard, our first-in-class FDRN blocker, we have been able to reframe expectations of what a therapy should offer the patient. This has been a driving force of the consistent growth we see. I also had the opportunity to attend AAN last week, and one of my key observations is that the value proposition of BizGuard is clear. The data we generated from ADAPT are playing out in the real world in a very consistent way, especially in the ADAPT-like patient population. The commercial and medical teams last year did an amazing job establishing the product as a new treatment option for GMG. The opportunity in there is to entrench it as a go-to therapy among neurologists. There is nothing more valuable than physician experience to achieve this. A physician having direct positive experience is much more powerful than the impact of reading published data. One story I heard from a physician was so rewarding. Someone who had had early experience with VivGuard but hadn't moved up the adoption curve yet. only took one patient with a transformational response to change their perspective, pushing the doctor to now use ZivGuard in the earlier adapt-like population, after ISTs or even Mestinon. The patient said they are experiencing a new normal that they didn't realize was possible. This is what we want to achieve when we talk about changing expectations. We also want to build on the momentum we established amongst patients. finding more and different ways to reach patients, especially those earlier in line of therapy. We see more of our patients coming to VivGuard directly from ISTs and orals, and continuing this shift to early-aligned patients will be instrumental for our growth trajectory. Of course, this takes time. It means further challenging physician inertia and the disconnect between patients and physicians on what well-controlled could mean. We hope that the sub-Q approval will be a potential momentum driver for this shift. We are now just six weeks away from our sub-Q producer date, and we are busy preparing for our second potential product launch. We believe the most powerful way to create value is through innovation, and the most powerful offering to the GMG community is to provide more choice. For us, this means further individualizing GMG treatment, not only with the dosing schedule, but also by offering both an IV and sub-Q option to capture the broadest number of patients. A sub-Q option may also serve us well from a payer perspective. Our engagement with payers in the U.S. was a success driver for the Big Guard launch, and we will focus again on early conversations, centered on the value we can create for patients and society. Even with our proactive strategy, we expect to navigate potential hurdles in securing early reimbursement, With any launch, we'll face new market blocks. We are planning to launch our SubQ product in the U.S., but we are also filed in both Europe and Japan. By early 2024, we hope to have both IV and SubQ BizGuard products approved across all three priority regions, and we'll be working in the background on a next-generation presentation with a pre-filled syringe. I want to leave plenty of time for your questions, so before we close the call, I'll quickly touch on our global expansion because we continue to expect new approvals through the end of this year. Slide 12. We received notification last week from the Ministry of Health in Israel that together with our partner, Medicine Pharma, we've received approval of Dugard for GMG. This marks the fifth approval in less than 18 months, an achievement that is truly remarkable for a first-time launch company. When I talk about being impressed by the commitment to patience and the team's ability to execute and deliver on its goal, this is a perfect example of that and makes me proud to be part of such a dedicated team. We also received approval in the UK earlier in the quarter and are already in price negotiations as we aim for a late 23, early 24 launch. Across Europe and the UK, we are now in pricing and reimbursement discussions in more than 10 countries. So from a revenue perspective, Germany will be the primary driver this year, even with the price adjustments that Carl mentioned. We are also on track in both China and Canada to receive approval decisions by the end of the year, which would mark the sixth and seventh approvals for VivGuard. I'm going to close by reiterating how excited I am to be part of the Argenix team. We have a lot to accomplish in the months ahead, but it is clear that we are just scratching the surface with the vastness of our opportunity, both with VivGuard as well as our future programs. Successful biotech companies are built on a foundation of innovative science, great talent, and a strong culture. And at Argenix, we have all three. I'm so inspired by what I've already experienced, and I'm ready to make the most of the opportunity before us, positioning Argenix for future growth. And with that, I'll now turn the call back to Tim for closing remarks.
spk24: Thanks, Karen. Slide 13. Sitting here in May, we still have many milestones ahead of us this year. which for us means that we have the opportunity to reach more patients, advance our clinical programs, invest in our strong science, and innovate across every corner of the company to create more value for our stakeholders. And we're not stopping here. Our new term data readouts are the first of many important stepping stones to unlock the full potential of our differentiated immunology pipeline programs and we look forward to communicating on those in the coming months. The value we are creating today is laying a strong foundation for the long term as we build a global, integrated, multi-asset immunology company focused on transforming the future for autoimmune patients. Thank you for your time today. I would now like to open the call to your questions.
spk04: at this time i would like to remind everyone in order to ask a question press star then the number one on your telephone keypad we ask you please limit yourself to one question only your first question comes from the line of derek akila from wells fargo your line is open hi everyone and uh congrats on the the quarter and the great execution appreciate you taking me taking my questions um so
spk13: Just recognizing that we're five quarters into the VivGuard launch in MG and things are going very well, I guess, what are some of the factors that are keeping you from putting out official sales guidance?
spk24: Derek, it's great to have you with us today, and thank you for your question. This is one for my colleague, Carl. Would you mind taking this question?
spk25: Thank you, Tim. Thank you, Derek. Yeah, we will not be providing guidance this year There are still too many variables. We're only one year into the launch. We have benchmark also, and we don't see any reason to do it this year. We will, of course, stay close to our analysts and make sure that our launch is well understood. Thank you, Derek.
spk13: Thanks. Maybe just pushing a little bit, I guess you said the key variables. That's kind of the question that I'm asking is, like, what are those variables that are most important to putting out sales guidance?
spk25: Those variables include, of course, the subcube launch. It includes pricing and reimbursement discussions, which are ongoing in the various European markets. We don't know when they will conclude. Those variables include the German price. We don't have any price in Europe yet. We're still negotiating the German price, and that will be our first price. the China launch, which of course can be a big variable. There's some uncertainty around that, Canada, and then also the speed of which we will continue to move into the earlier lines in the U.S., which will be a key factor in the trajectory of the launch going forward.
spk13: All right, great. Thanks. Thank you. Congrats on the progress.
spk04: Your next question comes from the line of Tazin Ahmad from Bank of America. Your line is open.
spk20: Hi, good morning. Thank you for taking my question. Maybe just a little bit more color, Tim. I know you had given us an explanation on the prepped remarks about the push to July for the top-line readout. But since you have had those 88 events already occur and it's still early May, can you give us a little bit more color on why you think you will need until July to get us that top-level data? Thank you.
spk24: Thank you, Celina. Looking forward to being at the conference next week. So the good news is we have 88 events, and I think the uncertainty about events is out of the system. So from now on, it's just a linear trajectory, right, to data readout. It's not because we have 88 events that the study is finished. So we're still dealing with the mechanics of unwinding the study. So are we enrolling all the patients, or are we basically getting the patients then out of the study? So there are a number of steps to be taken to database lock. And then after database log, we can go pretty fast to a top line data readout. So we're working very hard. The team is working very hard. But of course, this is such an important study that we also want to allow the team to do quality work. So we're not going to put any unnecessary time pressure on them. Thanks for the question.
spk26: Thank you.
spk04: Your next question comes from the line of Rahan Sharma from Goldman Sachs. Your line is open.
spk07: hi thanks for the question it's uh rajan sharma from goldman tax um maybe one uh for karen um just thinking about the dynamics between um iv and subcutaneous um that's got tijamot in my senior gravis um be good to get your thoughts on that and how you think it may play out in the long term yeah thanks for the question um happy to to talk about it um we're excited um to be on track
spk03: for the subcutaneous launch with a different date of June 20th. You heard from Carl and also earlier, the big focus and the opportunity for us is with early aligned patients with VivGuard. And we believe that there's an opportunity with the subcutaneous to really help us open up more early aligned patients. And the reason for that is because it aligned with our strategy of offering choice to prescribers and to patients. So we can see that it might be for prescribers, different reasons, potentially in the community that they might prefer. Maybe there's infusion capacity issues. They might prefer the subcutaneous option. And for patients, depending on their lifestyle, depending on their payer coverage. So the fact that we're offering options aligns with our strategy, similar to the individualized dosing. We're not providing guidance or a forecast of how the business mix will evolve between IV and subcutaneous, but we will be focusing on keeping them both on the market and providing that choice to patients and prescribers.
spk04: Your next question comes from the line of Yatin Senia from Guggenheim. Your line is open.
spk21: Hi. Thank you for taking my question. question on the CIDP study. Are you willing to share the relative mix of the, basically the baseline of the CIDP patient, how that might look relative to the first 40 patient baseline that you had shared? If you can answer it, I wanted to ask about Europe, especially as it relates to the GMG knowledge, like what are some of the major hurdles for you to really accelerate the uptake in Europe, similar to what you're seeing in the US. Thank you so much.
spk24: Thank you, Yatin, for your quick question. So on the first one, the answer is simple. I think we'll need to wait for the top line data release because we don't have the information at this point in time. And just to show that in the typical style of the house, we will release top line data such that there will be sufficient clarity about the context and the meaning of the data. with regards to hurdles to a European launch. Maybe, Carl, this is a question which you feel comfortable to take.
spk25: Yeah, thank you, Tim. I would say that the main hurdles, of course, is the pricing and reimbursement processes. We have only launched in Germany, and that is through the Amnok process, where we are still, throughout the 12 months, we are negotiating the price. That leads to some positions in Germany have a natural conservatism as we go through this process. But that said, if we look at Germany, the unmet need is definitely there. The feedback we get back from physicians and patients are really positive. In terms of the rest of Europe, we say what we are in pricing and reimbursement discussions in more than 10 countries, but basically also European countries. We are in various stages of that. We hope to conclude some of those processes before the end of the year. But really, from a planning perspective, we see it more as 2024 launches. And that is basically the main hurdle. I just want to end back with the unmet need is there, and the initial feedback from the payers and their physicians and the patients where we have it is really positive. And we look forward to successful launches, but it will take time to get there.
spk04: Your next question comes from a line of Akash Tiwari from Jefferies. Your line is open.
spk10: Hey, thanks so much. So just, number one, any color on exactly when you hit the 88 events for CIDP? And on the placebo arm, you know, what signals are you seeing internally that gives you confidence that placebo is behaving kind of like the PAT study did and not a kind of bearish scenario where relapse rates are trending more in the 40% to 50% range? Thank you.
spk24: Thank you, Akash. Thanks for being with us today. So we're not getting any more granularity on exactly when these ADA events happen. I also don't think that's important. I think the most important fact is that we now have them, and from now we're actually in control of the final steps in the process. We're not aware of any data, so we will need to wait for placebo data until we see the data, and we're then ready, of course, to present them to all of you. So stay tuned. We will be talking about placebo during the top-line release. Thank you.
spk04: Your next question comes from a line of Thomas Smith from SVP Security. Your line is open.
spk14: Hey, guys. Good morning. Thanks for taking the questions. You mentioned work going on in the background on a next-gen subcutaneous presentation of FCART. I was just wondering if you could provide a little bit more details. Is this still using the enhanced enabled technology? And maybe if you could provide some an update on the Electrify collaboration. That would be super helpful.
spk24: Thanks. Thank you for the question. I think we've doubled it on the fact that we're going to launch with a kind of first-generation subcube, which is a pull-up product from the vial, like so many other product executions look. But then we are, of course, diligently working on a pre-filled syringe. So that is the next generation that you're alluding to. you know that this company likes to plan for the long haul. So we're working on multiple product presentations, including, of course, a few product presentations. And that's where you have to contextualize the Electrify collaboration. More on that, you know, when we're more advanced in the process. So multiple generations in the works, not just for VidGuard, but also applicable to other pipeline assets. Thank you.
spk04: Your next question comes from the line of Danielle Brill from Raymond James. Your line is open.
spk16: Hi, guys. Good morning. Thank you for the question. So it sounds like you're launching the subqueue before we have top line CIDP data. So I'm just curious how this impacts your thinking on pricing strategy for the subqueue.
spk24: Hey, Danielle, thanks for being with us today, and thank you for the pricing question. Typically, we don't give any color on pricing, you know, until the date of launch. So I would encourage you to have a few months' patience to see, you know, how we're launching, and the commitment of the companies, of course, to give you transparency on positioning of the product and pricing at the day of launch. Okay? So bear with us.
spk04: Your next question comes from a line of Joel Beattie from Baird. Your line is open.
spk19: Great. Thanks. For the CIDB trial, what's most important to achieve for the trial to be considered a success?
spk24: Hey, Joel. Thanks for being with us. The way we have been calibrating expectations is by taking our audience back to the historical IVIG registrational trials. What we have been talking about is the type of data coming out of the ADE study, which would put us in a position to effectively compete with IVIG. So we would be triangulating towards kind of 50% response in stage A, and I think an effect size of 20 to 30% difference between active and placebo in stage B. I think that would put us in a position to effectively compete with IVIG. That's a comparable efficacy, roughly speaking. You know, of course, that, you know, the product will also be judged by its risks. It's not just the benefit, also the risks. I think we're offering a very safe and tolerable product. And then, of course, convenience, which will be a game changer, comparing, you know, a 30 to 120-second cell injection compared to a life which is basically organized around the infusion chair. So I hope this answers your question.
spk00: Thank you.
spk04: Your next question comes from the line of Alex Thompson from Stiefel. Your line is open.
spk09: Hey, great. Thanks for taking my question. I was hoping maybe you could provide a little bit more details around sort of the current patient mix in the U.S. on VivGuard. Can you talk a little bit about the proportion of patients that are, you know, on drugs that are IVIG naive? And then maybe if you could comment on whether you're seeing any meaningful off-label use either in sort of non-acetylcholine receptor positive antibody patients or in other indications. Thanks.
spk03: Yeah, this is Karen. I can provide some of those answers. What we've seen since launch is that we've been progressively moving up to earlier line patients. So more momentum after orals and ISTs. In terms of the IVIG, around 50 to 55% of our patients currently are coming from IVIG. As I mentioned earlier, our strategy is to continue to move earlier in the treatment paradigm. In terms of off-label, we don't track that data. We don't support off-label use of the product through PSP, but we don't, from what we understand, we don't see significant off-label use.
spk14: Great, thanks.
spk04: Your next question comes from the line of Allison Bratzel from Piper Sandler. Your line is open.
spk01: Hi, good morning, and thank you for taking my questions. So just a commercial question on DivGuard. Could you comment on any, you know, first quarter seasonal effects you saw, you know, payer resets or re-verification of benefits? Was that a meaningful factor in the quarter, at least in the U.S.? Any color there would be helpful. Thanks.
spk03: Yes. Thanks for the question. So as with other products, we did see recertifications in January. As you all well know, we have very strong payer access for VivGuard. And so what we saw was some slowdown in January, but really those patients were back on therapy for February or March. So it was really more of a push by a month or two rather than anything more.
spk04: And your next question comes from the line of Yaron Werber from Cowan. Your line is open.
spk22: Great. I have a question on CIDP. It's sort of a two-part question that's interrelated. The first one, can you give us a little bit of a sense, I mean, that you only really stopped recruiting back in February. So it sounds to us like you probably ended up recruiting close to 180 patients at the upper end of the study. Is that correct? And then secondly, when you're talking about just, you know, before you're able to lock the database and moving patients to the open label extension, how does that work? It's not, it doesn't happen automatically at week 48, or you're referring to really now taking all the placebo patients and moving them over whatever remaining patients there are. I'm just trying to understand, you know, why that's going to take a little bit longer than normal. Thank you.
spk24: Yeah, thank you, Jeroen. Thank you for the question. So, you correctly call out that we continue to enroll patients to the trial, even if we thought that we had sufficient patients in stage B to ultimately hit the 88 events. So you will see the final details of the patients we effectively enrolled to, you know, the run-in stage A and stage B when we do the top-line data readout. The rollover mechanics are relatively complicated in that sense that a patient has an option to either go to the off-label extension, or not go over to the overlap extension, and then you need to do some closeout visits, you need to collect some final information, et cetera. So you have the decision fork in the road, and it just involves confirmatory visits, data collection, and basically that study is in the shape that you can say, okay, we have all the data, interlock the database, and go with quality data to a top-line data readout. Okay, I hope that makes sense. Thank you, Jeroen.
spk04: Your next question comes from a line of Samantha Simon cow from city. Your line is open.
spk17: Thank you very much for taking your question. Um, I, so you have, you're not talking about the 180 patients that you're enrolling in it here. You aren't going to give any additional details on that at this time. Um, maybe could you just talk about, um, uh, sorry, excuse me.
spk26: I have to,
spk24: off on here this may be an opportunity for me to um finish up on on yarun's question once there is an event happening of course as a patient you can make the decision to roll over or basically exit the study so you don't have to be 48 weeks in the study it is an event-driven trial not a time-driven trial and i hope that That answers also the question which we have from Citi. Let's move on. Thank you.
spk04: Your next question comes from a line of Miles Minter from William Blair. Your line is open.
spk06: Thanks for taking the question. It's actually on the GenMab collaboration. Just curious as to how dedicated you are stepping into oncology in the joint venture. I know previously you sort of outlicensed an asset to AbbVie, but Just wondering whether that's a pillar outside of immunology or tangential to that that Argenix is really dedicated to, or is GenMAP really the lead on that oncology asset, and you're focusing on the immunology part of that deal? Thanks very much.
spk24: Hey, Miles. Hey, Miles, thanks for that question. That's a great question. So the way you need to think about our GenMAP collaboration is in the context of our IIP program. We are externally focused. We like to partner with expert partners externally to go after novel type of biology. And this is just an interesting immunology pathway where we see utility in both autoimmunity and oncology. We think there's a real exciting increase in probability of success by joining forces. I mean, two antibody powerhouses with pretty complementary technology platforms and expertise. So we're having a go at this pathway. We will basically go in there 50-50. We will share any investments and costs 50-50, and we will share any upside 50-50. So whenever there is upside in oncology, we can share. Whenever there is upside in autoimmunity, we can share. So that's the gist of the collaboration more than anything else. Okay? Thank you for your question.
spk04: And your next question comes from a line of Neil Alexander from Deutsche Bank. Your line is open.
spk08: Hi guys, it's Neil Alexander from Deutsche Bank. It'd be good to get your views of how sale trends will evolve as we get through the year in the US and in the European regions. Thanks.
spk03: Yeah, this is Karen. I can comment on that. As Carl mentioned earlier, we won't be providing guidance, but in terms of the trend, we've seen consistent growth and momentum in the US market, and we expect that to continue as we move to early-aligned patients through the year. Carl already shared some thoughts on the European market. We're pursuing pricing and reimbursement, and so we think that will be a slower growth trajectory. It's also very important to note that we do have competition coming later in the year, and so that will be a further unknown factor. But we're confident in our efficacy, safety, and convenience profile that we can establish as those competitors come into the market.
spk04: And your next question comes from a line of Susandi from Kempen and Company. Your line is open.
spk12: Hi, team. This is Suzanne. Thanks for taking my questions. I was wondering beyond F-cortigamide for Argenix 117 and the upcoming MID23 data release, can you give some more granularity? This is the first cohort readout. How many patients are there? How many are on drug and placebo? The doses that you're testing? And what other cohorts are there in this study next? And then I have a follow-up after that.
spk24: That's great, Susan. It's an excellent question. Of course, Argenix 117 is a molecule we're very excited about. The way you have to think about the phase 2 trial is the following. Nobody has any idea what level of C2 inhibition one needs to have a clinical effect in MNM patients. So what we really tried to build is a PKPD model based on the phase 2 data, which can reliably predict the phase 3 dose in MNM. assuming, of course, the molecule is going to work in MMM. So think of cohort one, the data we will be talking about middle of the year, as a first dose and dosing regimen cohort where we're aiming for a certain percentage of C2 inhibition. So we will be talking about nine patients in total where we will basically have an idea, you know, for that type of C2 inhibition, what clinical efficacy you can expect. Based on those data, we have an option to either up the dose or lower the dose then conclude the phase two trial remember all mmn patients which we are realistically able to attract to the trial are basically on ivig so this is again going to be kind of randomized a trial design where we basically see a stable cadence on ivig for these patients and then they get randomized after a running period either on argenix 117 or on placebo and we hope to see that we can keep these IVIG patients stable on ARGENIX117, whilst, of course, you would expect the placebo patients to need the treatment after a while with IVIG. So that's the type of data we're going to talk about. You had another question?
spk12: Yes, yes. Thanks a lot. That's very helpful. I just have one clarification question on the ADAPT Plus data presentation at AM. There seem to have been more adverse events of grade 3 or higher than before. Were those related to the COVID-19 infections or is there any other color you can give on this?
spk24: Thanks. There's of course a caveat here, Suzanne. The open-label extension is much longer, of course, than the randomized controlled trial. So the absolute number of events is going up because you're just measuring over a longer period of time. I invite you to look at the column where we normalize per time units. you will actually see that the safety profile of VidGuard continues to be very clean and in line with the ADAPT study itself. So I think the use of this post is outstanding. We have now multiple years of experience for VidGuard, by the way, not just in my senior grades, but across many indications, and the safety profile is consistent and encouraging. I think this is setting a whole new standard in terms of benefit-risk profile in the severe autoimmune space. Thank you.
spk12: Got it. Thank you.
spk04: And your next question comes from a line of Trevor Allred from Oppenheimer. Your line is open.
spk15: Hey, thanks for taking the question. I just wanted to ask, so Tepeza had a miss earlier just a few days ago. I wanted to see if you had any updated thoughts on the opportunity there and where Ethicard Ticket Mod fits in. Thanks.
spk24: Trevor? We don't feel sufficiently informed to talk about sales dynamics behind the PEDLA, so I think this would be a question that's asked to Horizon. We repeat our conviction in the indication, and the reason we selected that indication for this graph is based on solid biology understanding, and then, of course, the feasibility of running clinical trials and the remaining unmet medical needs. Thank you for the question.
spk04: Your next question comes from a line of Douglas South from HCW. Your line is open.
spk05: Hi, good morning. Thanks for taking the questions and congrats on the progress, Tim. So maybe just for 117 as well as 119, I'm just curious because you've identified, sort of named three indications for 117. You sort of noted a couple potential ones for 119. How quickly do After those initial studies, do you think you might ramp into other indications? And I say that just because with F-Card Digimod, we very quickly went from MG to a full pipeline of over double-digit number of ongoing trials. And so do you see the same potential for those assets? And do we think that F-Card Digimod represents a roadmap for how quickly we might see the development programs for those assets expand? Thank you. Thank you.
spk24: I appreciate the question. So you're right. When we select pipeline assets, we love to go for novel biology. We like to do that with what we think will ultimately be best-in-class antibody molecules. And we like optionality. So we love molecules which can play in multiple indications. What ultimately the number of indications is going to be for ARGENIX 117 and 119, we do not know yet. We think there is sufficient opportunity in front of us. But you know that we like to march based on conviction on the biology. So rest assured that Argenix is doing its homework from a biology point of view on opportunity above and beyond the indications we mentioned. And we will inform you when we see sufficient ground to expand beyond the initial indications. Thanks for the question.
spk04: Your next question comes from a line of June Lee from Truist Securities. Your line is open.
spk11: Hey, congrats on the quarter, and thanks for taking our questions. For the planned TEP trial, it's sort of interesting to consider the possibility that targeting SCRN could address not just the ocular manifestations, but also the systemic manifestations of hypothyroidism. Along that line, would you consider an endpoint other than proptosis for possibly a broader label? And as a follow-up, is there any data out there pointing to how much IgG reduction you would need to have a clinical impact on proptosis? Thank you.
spk24: Yeah, thank you for the question. I'd love to answer it, but the commitment we made during the JP Morgan conference was that clinical trial design details will be disclosed when the study will go online on clinicaltrials.gov. So I encourage us to be a little bit patient, and then we can talk about primary, secondary, endpoints, inclusion, exclusion criteria. So we will be transparent. It's just too early to talk about it. In terms of IgG reduction required, to have a clinical and meaningful effect in dead patients, you know that we typically like to triangulate by a plasma exchange or immunosorption data. We also believe that some of the competitive data in the SDREN class constitute an interesting data point to triangulate at. So, you know that with DivGuard, we achieve a comparable IgG level reduction as with plasma exchange, and we think that will be sufficient to have a marked impact in TAD patients.
spk04: And your next question comes from the line of Charles Pittman from Barclays. Your line is open.
spk18: Hi, thanks very much for taking my question. I've got a question on just MG and whether or not you could give us any details on the number of new patients that were accrued in 1Q versus retreatments, and whether you can give us any insight on the discontinuation rate of these patients now, and then maybe just a quick follow-up if I'm not sure you'll give us much detail on this, but on the German pricing negotiation you about to start, how much is that likely to Are there any benchmarks that we should be looking at when we're considering that? Thank you.
spk24: Charles, I'll give the first question to Carl on patient numbers and what is the formal line there. Then on this continuation, I will give the floor to Karen. And then maybe, Carl, you also have to take the question on any benchmarks for negotiated prices under the MNOC process in Germany, right?
spk25: Okay, thank you, Tim. On patient numbers, thank you for the question, but we are not going to give patient numbers now. We do say that the launch is consistent, and we believe that the revenue number will be there to guide you, so we're not going to get into more details now. In terms of the German negotiations, we are, of course, at a very sensitive stage of a negotiation, so we're not going to provide any comments, so please bear with us. We will, of course, start accruing for the difference between the old price and the new price effective March, i.e. you'll see an impact on total revenues in Europe, i.e. Germany going down. But we're not going to provide any details due to the sensitivity, and it will all be disclosed in September when the negotiations conclude. Thank you for the question.
spk03: Thanks, Karl. And on the discontinuation rates, I would say it's too early for us to share any numbers. The data is still maturing, but it's about what you would expect or about what we would expect at this point in launch from what we've seen. One important factor to remember is that early on in the launch, we were getting more refractory patients. We've moved and are gaining momentum in the earlier line patients, and the discontinuation rate will reflect that as we move forward.
spk04: And your final question comes from the line of Simon Baker from Redburn. Your line is open.
spk23: Thank you for taking my question. Another one we've got in MCHIP. As I understand it, the only barrier to early line usage is physician awareness. So I wonder if you could give us an idea of the rate at which you think physician awareness will drive early line usage at a greater extent the active efforts you're doing to advance that more quickly, and the importance of the subcutaneous formulation in driving earlier line usage. Thanks so much.
spk03: Yeah, thanks for the question. It's a really good one. So I would say that during, of course, the first year of launch, a lot of the momentum that we gained as you said, was through physicians gaining awareness and early experience with VivGuard. And what we've heard, the feedback, I was at AAN last week and I heard this consistently, is that the experience and the early experience with VivGuard reflects what they expected from the ADAPT study. And so actually what we see is the more experience as the physicians move from awareness to experience with VivGuard, That's what's really driving the earlier line use and what's helping us with momentum in terms of moving earlier lines. So we expect that to continue as we continue through executing on the launch as experience grows. In terms of the subcutaneous, that will help us, as I mentioned earlier, With early-aligned use, there are some patients that might not want to move from an oral to an infusion. There are some prescribers who might prefer a subcutaneous option for logistics reasons, for patient preference, whatever it may be. So we believe it opens up a whole new patient opportunity for us in the early line to have the opportunity to have either IV or subcutaneous.
spk23: Great. Thanks very much.
spk04: This concludes today's conference call. We thank you for your participation today. You may now disconnect.
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