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spk07: Good morning. My name is Rob, and I will be your conference operator today. At this time, I would like to welcome everyone to the call. 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 would like to ask a question during this time, simply press star, followed by the number 1 on your telephone keypad. If you would like to withdraw your question, again press star 1. Thank you. I'd like to introduce Beth Diljaco, Vice President of Corporate Communications and Investor Relations. You may now begin your conference.
spk09: Thank you, Operator. A press release was issued earlier today with our first half 2022 financial results and second quarter 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 developments, regulatory timelines, the potential success of our product candidates, financial projections, and upcoming milestones. Actual results may differ materially from those indicated by these statements. 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 Hauermeren, Chief Executive Officer. Carl Gubitz, Chief Financial Officer, and Keith Woods, Chief Operating Officer. I'll now turn the call over to Tim.
spk21: Thank you, Beth, and welcome, everyone. We are just halfway through 2022, and it has already been a year of incredible execution by our team outlined on slide number three. We are delivering on two commercial launches, one in the United States and one in Japan, and are now looking ahead to broaden the global reach of VidGuard. We had two positive phase three data readouts in the first half and are positioned for the series of trial starts and additional data readouts over the next 18 months. And we were able to finance our ambitious business plan in a difficult market environment. We live by our reputation for execution, so I want to start by saying thank you to the Argenix colleagues for their thorough preparation and steadfast determination to achieve our goals. and to our stakeholders, our shareholders, our physician partners, and of course, our patients. To begin, the second quarter of our safeguard launch showed significant growth from the first quarter with global net product sales of $75 million. It will expand on our progress across our patient, physician, and payer segments later in the call, but at a high level, I'm really happy with the team's hard work. They were well prepared going into approval with carefully crafted strategies and continued to execute on them. A few highlights to point out on slide number four. Our market access team delivered what they promised and secured broad coverage in the first two quarters of launch. This was crucial in converting physician prescriptions to patients on therapy. We are getting regular feedback from physicians and patients on their experience with VivGuard, which, while anecdotal, aligns with data from the ADAPT trial, including on the fast onset of action and minimum symptom expression. MSC was an exploratory endpoint in our trial, but a metric that is growing in importance amongst neurologists because of its practical meaning for patients. This feedback is also in line with what we hear from GMG patients and their advocates on the significant unmet need that still exists. We have learned firsthand, and through our outcomes research, what a devastating and debilitating disease GMG can be. With its novel mechanism of action, DriftCard has the potential to transform the treatment landscape and significantly improve patients' lives. Hearing stories from patients who are already experiencing this benefit has been the most rewarding part of the last six months. I am equally very proud of our ongoing commitment to be leaders in FCRN, not only from the commercial side, but also the scientific. Our approach starts with solid science and is backed by strong data, and we continue to draw on this to further elucidate FCRN biology and how best to modulate it. We believe our science-based approach will remain a powerful tool as we engage with our key stakeholders. Beyond the goodness of our launch, we have had other accomplishments this year across our F-Gratigma program. Our ambition is to reach many more patients suffering from autoimmune diseases through our sub-Q product launch and label expansions. Slide five, in March, we delivered positive phase three data from our ADAPT sub-Q trial. Recall that we had to show non-inferiority of sub-Q to IV based on IgG reduction and meet safety database requirements. We have accomplished both, putting us firmly on track to file the BLA by end of the year. Slide six, in May, we announced positive phase three data from our advanced IV trial. we met a very difficult primary endpoint in a highly refractory ITP patient population. In ITP, Avgatigamot showed a consistent response profile, as we've seen in other indications, a fast onset of action and patients with sustained responses who were able to switch to every-other-week dosing. We have gotten feedback from physicians on the data, and continue to hear about the importance of the IWG score in how they treat patients. We saw a 51% response rate on the IWG scale, where patients had to show a sustained platelet count over 30,000, and in the absence of any bleeding events, for two separate consecutive weekly visits. Day-to-day profile of VivCard in this chronic dosing setting was also consistent with previous clinical trials. Slide seven, we promised that we would look at key learnings from advance and apply them to our ongoing advanced sub-Q study. We did this and have made a database decision to adjust our powering assumptions and expand enrollment in the SC trial. This pushes the timing of top-line data to the second half of 2023, which we believe is in the best interest of ITP patients. We are set up for a very busy 2023. We expect top-line CIDP data in the first quarter and PV and ITP data in the second half. Before I turn the call over to Carl for our financial results, I want to briefly talk about OncoVerity on slide eight. We announced this morning in our press release that we decided to take the company creation approach to advance development of fusotuzumab in the AML patients. Here is why we think this is the right decision for us, for the drug, and for patients. We have the unique and exciting opportunity to take the novel translational biology insights from Dr. Clayton Smith at the University of Colorado and combine them with our first-in-class asset, Cusatuzumab. Dr. Smith and team, have uncovered important insights on the role of the CD70-CD27 pathway in AML, and more specifically in venetoclax non-responsiveness or resistance, which we think will complement the encouraging data we have already demonstrated in newly diagnosed AML patients unfit for chemotherapy. We believe the data we have shown to date warrant further development of Cusartuzumab, and through OncoVerity, This work will happen in an organization that has the expertise and the bandwidth to devote the right time and resources to this opportunity. This is the best decision for EML patients. OncoVarity is also the fourth company to emerge of our discovery engine, where we have built a company around the promising assets while taking a stake in the development. This strategy requires minimal financial commitment and allows us to maintain significant value creation potential for our shareholders. Between our commercial launch, our progress in R&D, and our commitment to immunology innovation, we are off to a strong start for 2022. Our focus going forward will be about continued execution and sustained growth and expansion. With that, I will turn the call over to Carl. Thank you, Tim. Our first half and second quarter 2022 results are detailed in our press release from this morning, so I will only highlight the key points here on slide nine. For the second quarter, our global net product revenues from the WebCard launch were $74.8 million, which includes $1.5 million from Japan and some named patient sales from Israel. Together with the $21 million from the first quarter, this puts our year-to-date global product revenues at $96 million. Inventory in the channel at quarter end was well managed and reflects less than two weeks' worth of hours. Total revenues for the quarter were $85.2 million, which also includes $10 million in collaboration revenue and other operating income. Cost of sales for the quarter were approximately $5 million. Total R&D and SG&A expenses for the second quarter were approximately $126.9 million and $127.8 million, respectively. These can mainly be attributed to FCAR TIGIMOD and other pipeline research expenses, as well as marketing and headcount expenses related to our global launch. On our cash, we ended the second quarter with $2.6 billion in cash, cash equivalents, and current financial assets. This includes net proceeds of $761 million from our March financing. We continue to believe that our net cash burn this year will be up to $1 billion, which will specifically support the rollout of our global launch clinical development of F-card tigimod in 10 indications and organics 117 in 2 indications, investment in the global supply chain, and pipeline expansion through our immunology innovation program. Our business plan is an ambitious one, so we were very happy to be able to bring in the additional capital earlier this year to sustain our ability to drive considerable value for our shareholders. You can find additional details behind these numbers in the press release we issued this month. I'll now hand the call to Keith for a commercial update.
spk15: Thanks, Carl. Slide 10. We are very happy with the continued momentum this quarter with our VivGuard launch, driven by consistent growth in both patient and physician demand. The first six months of a launch are crucial in building the right foundation for the product, so I am incredibly proud of the team for their demonstrated commitment to getting us off to a strong start. While we are still in the early days, we are seeing positive signals across each of our stakeholder groups, all of which were key drivers in the $75 million in revenue we generated this quarter. Let's start with patience. Slide 11. We currently have approximately 1400 patients on therapy globally, which translated to over 1000 new patient ads in the second quarter. This number includes initial contribution from the first six weeks of the Japan launch. I was grateful to spend time in Japan with our commercial team and engage with some of our key customers and overall we are very pleased with the strong start. We had patients on therapy within the first week, including seronegative patients who benefit from the broader label in Japan. Looking at the types of patients who are going on VivGuard, it remains similar to last quarter. Of the 1,400 patients, about 50% have experience with IVIG, while the other 50% have experience across the treatment paradigm from as early as just mestinone or steroids or other biologics. This was a tremendous result from both our field teams engagement with physicians and patients asking about VivGuard by name. It was a true multi-channel approach and also the outcome we had hoped for by empowering patients. The second quarter was one of extraordinary growth in terms of patient ads, but we expect to see more of a balance between repeat and new patients going forward. The reason being that most patients on VivGuard to date have IVIG or other biologic experience. We are closely watching whether we can gain broad adoption earlier in the treatment paradigm because this will be an important driver of our overall launch trajectory. Before I move on to physicians, I want to share some of the health economic outcomes research we gathered. This reinforces the potential value proposition of VivGuard to GMG patients. First, the majority of patients, when entering into the ADAPT study, reported issues with mobility, self-care, and completing their usual daily tasks. Often, these challenges were accompanied by pain, discomfort, anxiety, and depression. From the early data we gathered, GMG patients in ADEPT experienced rapid and substantial improvement in health-related quality of life following VibGard treatment. At peak response, patients approached the quality of life of the general population. Additionally, we know that hospitalizations can pose a significant burden on GMG patients. In ADAPT, VivGuard-treated patients experienced a 67% lower risk of MG-related hospitalizations than placebo. Overall, it's very rewarding to hear this initial research showcasing another aspect of the value proposition of VivGuard to GMG patients. Moving on to physicians on slide 12. We see that our messages are resonating with physicians. The strength of the ADAPT data in driving robust and deep responses combined with our favorable safety profile. With our launch strategy, we made a commitment to market from the science and stay data driven. This is paying off and physicians are showing increased awareness on the mechanism and action of FCRN and the mechanism of disease of GMG as being antibody driven. We are seeing breadth in physician prescribers, but also depth from some early adopters. One of the drivers of the breadth we are seeing is that our sales team is focused on and allocated resources to our top priority targets. Our goal is for these early prescribers to share their experience with colleagues and peers. In looking ahead to upcoming quarters, the big unknown with our physicians is going to be driving that adoption curve. At this stage, 78% of our prescribing physicians have written one or two scripts. So shifting these prescribers from initial use to broad adoption is going to be a key indicator in our launch growth trajectory. Finally, I'd like to cover the excellent work that's being done by our market access team on slide 13. We continue to have constructive conversations with payers And as we committed last quarter, we have achieved broad coverage. VivGuard specific policies have been published in Medicare and commercial plans covering almost 85% of covered lives. The majority of these policies are favorable and aligned to the VivGuard label. Patients need to fail only methadone or steroids to gain access and approvals are typically for six to 12 months at a time. Our J-code went into effect on July 1st as expected, which we believe will facilitate a more seamless conversion from enrollment to infusion and give confidence to some prescribers that the process should be straightforward. Moving on to the progress of our global launch on slide 14, the team in Europe is ready to go after receiving a positive CHMP opinion last month. We expect a decision next month and will first prioritize Germany, where we can book revenues while we go through the AMNOG process and work to obtain reimbursement. We have broad commercial infrastructure in place across Europe, including France, Benelux, Italy, Spain, and also the UK. We are building infrastructure in Canada to support a filing and potential approval with Health Canada. Our partner Xilab filed for approval in China, and the filing was accepted earlier this month. And finally, we continue to look at distributor arrangements in other geographies beyond those we have in place. In fact, we already saw some main patient sales this quarter in Israel through our partner in medicine. To conclude, on slide 15, I'm really pleased with where we stand after six months of launch. We see strong signals across all of our initial launch priorities. to empower patients, provide best-in-class patient support, drive rapid HCP adoption, and enable appropriate access. It is still early days, though, and the same unknowns we outlined from last quarter are still at play. First, we are in a unique situation where a competitive product launched just five months after us, and we still do not yet know the impact of this. And we still have patients primarily on their first and second cycles, so it's too soon to understand dropout rates or how individualized dosing will play out over longer periods. I can say with certainty that our team has built a strong foundation for success, and we are determined to build upon this. We are not satisfied with the 1,400 patients, but thinking of the total number of GMG patients globally that would benefit from a new therapy. This is what is driving each of us each day to be out there engaging with physicians and working hard on behalf of patients. I'll now turn the call back to Tim to conclude.
spk21: Thanks, Keith. Before we open up the call for your questions, I would like to conclude on slide 16 with the following. In the first half of 2022, we have delivered on a number of commercial, regulatory, and clinical achievements As we look ahead for the next 12 to 18 months, we are focused on achieving the multitude of goals we have set. Continue to reach GMG patients with VidGuard. This is how we come to work every day to be able to improve the lives of patients and deliver solutions to those who need them. Bring more optionality to the GMG community with our project SubQ product launch. Advance our best-in-class FCLN pipeline with 50 data readouts of FGATIGIMOD in three indications next year. Expand our next pipeline in a product opportunity with our first-in-class C2 inhibitors, Argenix 117, and further develop our earlier assets that have emerged from our unique discovery engine, including Argenix 119. We have an outstanding team that continues to execute on these plans, and a strong balance sheet to support our ambitious goals. It is our hope that by leading with science and building our genetics into the next great immunology company, we can continue to reach patients in need and create long-term value for all stakeholders. With that, we can begin the Q&A. Operator?
spk07: 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 that you please limit yourself to one question and then rejoin the queue for further questions. We'll pause for just a moment to compile the Q&A roster. And your first question comes from the line of Danielle Brill from Ray James. Your line is open.
spk16: Hi guys, good morning. Congrats on the strong quarter and thanks so much for the question. Keith, you mentioned that you expect more more of a balance with 3P in new patients moving forward. Can you elaborate a bit on that? I'm just trying to, I know you said it's too soon to understand dropout rates, but qualitatively, like, can you provide some color on whether you're seeing the patients that began treatment in 1P returning, and any additional insights on a potential bolus effect here? Thank you.
spk21: Hi, Danielle. This is Tim speaking. Thank you for joining us on the call. Keith, I think this is a question for you in your camp, right?
spk15: Yeah, Danielle, thanks for the question. What I can tell you is that we do have the majority of patients that have had completed one round of therapy. And this is to be expected considering that, you know, 1,000 of the 1,400 patients globally were added in second quarter. We do have quite a few patients who have gone on to complete a second round of therapy. Most of these are patients that did start, in fact, in the first quarter. And there's a small handful of patients, Danielle, where physicians have taken a more aggressive approach of four weeks on, four weeks off. So we have a handful of patients that have actually gone through three cycles already. So we are seeing a return of patients, and that's what I meant by the statement of a mix of repeat patients and new ads moving forward.
spk06: Got it. Thank you so much. Your next question comes from the line of Yerever from Cowan. Your line is open.
spk22: Great. Thanks for taking my questions and congrats on a very strong launch. Maybe just as a quick follow-on to Danielle's question, are you expecting that most patients are going to do an individual treatment approach or sort of the cycle on off? And then secondly, do you expect, I saw you only did one and a half million in Japan, so most of the number obviously is in the U.S., Are you expecting some kind of an upfront channel fill as you normally see in Japan? Thank you.
spk21: Hey Jeroen, thanks for joining us this morning and for your great question. Before I hand over the work to Keith, I want to remind everyone of the fact that myasthenia gravis is what we call a snowflake disease. Each and every patient is experiencing his or her disease in their own unique fashion. So there is no one size fits all. And maybe Keith, this is a good point for you to continue to answer, right?
spk15: Yeah, yeah. Thank you for the question. So first of all, we expect that you're going to see a variety. Number one, we see patients that are going on cycle therapy, individualized treatment. And as Tim mentioned, every single MG patient is different. So we have some patients who have received one cycle and are still maintaining a great response. We have others that I mentioned are being treated a little bit more aggressive. The bottom line is, in the real world, it's not about a two-point drop in MGADL like it is in a clinical trial. It's getting patients to minimal symptom expression to where they can walk around and live a normal life like the rest of the general population. So when we say individualized dosing, it is truly going to be that moving forward. I think you can see some of the comments that patients have made that are enjoying this because they don't want to go back in for treatment when they're feeling perfectly fine. We expect this trend to be continuing, and as physicians get more comfortable with the drug, I think you'll see a variety of approaches around the cycling of individualized and some that might go to a little bit more chronic.
spk21: Thank you, Keith. I would like to hand over to Carl for the Japan-related question. So thank you for the question. The question was relating to inventory in Japan. So our supply chain in Japan, we recognize revenue when the wholesalers ship to hospitals. The wholesaler doesn't carry any inventory, so there is no wholesaler stocking in Japan. In the US, of course, the specialty pharmacies do carry inventory, and that is where we comment it's less than two weeks. It's well managed at court end.
spk06: Thank you.
spk07: Your next question comes from the line of Yatin Sinea from Guggenheim Partners. Your line is open.
spk20: Hey, guys. Congrats on great performance. A couple questions for me, a little quick ones. Can you maybe just help us understand how we should think about the cadence of patients coming on to the drug? Obviously, you added 1,000 patients in Q2. Just trying to get a sense of what's sustainable or what sort of cadence should we expect And then just overall in terms of the U.S. market when the drug was launched, I think you had articulated 17,000 eligible patients. Any revision to that number? Just trying to get a sense of what level of penetration is achievable in the U.S. now that you have more commercial experience. I just wanted to get some updated thoughts on the U.S. opportunity. Thank you.
spk21: Thank you, Yatin. And I think Keith, you're well-positioned to talk about our thoughts for the future cadence of patient enrollment, right? Why don't you go ahead with that question first?
spk15: Yeah, thanks for the question, Yassin. First of all, I would like to say that quarter two, 1,000 patient ads was an extraordinary quarter. It really was. This is where physicians were beginning to hear from other physicians about VivGuard. Patients were going in and asking for it by name, and the team was really clicking on all cylinders. So I don't think that I would set up a model that says 1,000 patients every quarter is going to be the norm. But, you know, we will have to wait and see. As far as your 17,000, that is still the plan. That is still the target. That is still the number of patients that we believe that is appropriate for VivGuard therapy here just in the U.S. and patients that could truly benefit from VivGuard. So no revision to that as our target number.
spk06: Your next question comes from a line of Matthew Harrison from Morgan Stanley. Your line is open. Matthew Harrison from Morgan Stanley. Your line is open. Your next question comes from a line of Derek Aquila from Wells Fargo. Your line is open.
spk12: Hey, good morning, and thanks for taking the questions. Congrats on the quarter. So just two quick ones for us. I think they're for Keith. But just following up on your commentary, Keith, about the treatment algorithm. So what do you think is really needed to get VivGuard utilization earlier in the treatment algorithm? So just not the IVIG experience and, like, C5 refractory. So that's just question number one. And then just a follow-up clarification. So, Keith, you said 78% of the prescribers are writing one to two scripts I guess what percent of these are for generally like six to 12 months of treatment, or is it just for one treatment cycle?
spk06: Thanks. Thank you, Derek. These are two good questions. Keith, can I ask you please to address those?
spk15: Yeah, I'm happy to do so. Derek, thank you for the question. So the treatment algorithm and how do we get to earlier lines of therapy? I first want to call out to you that, you know, while 50% experienced IVIG, the other 50% were aligned with the ADAPT trial. So we do have patients that have started VivGuard after only being on Mestinon. or only being on Mestinon and a steroid. We also have those that have started at VidGuard after Mestinon steroid on their first IST. I think the key to moving earlier into this treatment algorithm is going to be the experience curve. So it actually ties into the second part of your question, which is if 78% of our prescribers have only prescribed for one or two patients, it's getting them to see that patient experience. One of the things that I've heard from clinicians is how rapid the onset of response is, how their patient is doing so much better after one or two doses. And they think about how long some of these oral therapies, particularly ISTs, take to kick in. That combined with favorable payer policy is our plan on how we go earlier into the treatment paradigm. As far as the 78% that have only written one or two scripts and where do they fall into those policy buckets of a six-month or a 12-month, I don't have that data broken down and in front of me at this time. So I can't report to you on that.
spk06: Your next question comes from the line of Allison Bradzell from Piper Sandler.
spk07: Your line is open.
spk14: Hi. Good morning. Thanks for taking the question and congrats on the quarter. So a question on redosing. I know it's early in launch, but could you comment just on whether redosing has been more patient or physician driven and any trends you're seeing there in terms of what is driving the initiation of second or third dosing cycles? And maybe talk to the role of nurse case managers from MyVivGuardPath in helping drive that. And then just on a related note, are you seeing any telehealth prescribing That is, are docs comfortable prescribing ZipGuard over telehealth, particularly for those second or third dosing cycles? Thanks.
spk21: Thanks, Alison. And these are, again, two questions for Keith, one with regards to triggering redosing and then the telehealth question, right, Keith?
spk15: Yeah, thanks for the question, Alison. So on redosing, we're really seeing two schools of thought. We're seeing a reactive approach, which that would be patient driven. So the patient gets a cycle of DivGuard. They respond and they're feeling better. And they're given direction that when you begin to lose response, when you begin to feel symptoms, please call and we'll get you in for your next cycle. So that's the patient driven example. The other one that we're seeing is a physician driven. That is where a physician gives a cycle of IVGART and decides when they're going to bring that patient back for cycle two. We're seeing some physicians that say we're going to go four on four off. And if you have maintained response the whole time before your second cycle, then we'll try to stretch that interval for past four weeks and into five, six, seven, eight weeks. You have others that are going to start with the cycle and bring them back, similar to what is stated in the package insert, will bring them back roughly 50 days later for that second cycle. So those are where they're physician driven. Nurse case managers, as far as driving the need for the second cycle, it's really not the role of the nurse case manager. These are not the treaters of the patients. Nurse case managers are there to assist patients, answer questions, and support them overall on their VidGuard journey. So it is really more of a physician and patient decision on their next treatment cycle. Finally, telehealth. It's too early to say on telehealth whether you're going to see somebody prescribe a naive patient with telehealth. As far as patients coming back in for second cycle, You know that many of the majority of our policies, once a patient is prescribed DivGuard, they're approved for six to 12 months. So these patients are coming back in and getting their second cycle.
spk07: Your next question comes from a line of Akash Tiwari from Jeffries. Your line is open.
spk08: Hey, thanks so much. So first of all, congrats on the quarter. You've shown really robust GMG numbers so far, 1,000 patients in that quarter. That said, we have... consensus gone up, and I think we're estimating if you account for the drop-ups, you need about kind of 500 and 600 starts each quarter in the U.S. to kind of hit numbers in the back half of next year. You know, this question was asked before, but I just kind of want to double down on it. What's your current view on a reasonable go forward for new patients going into next year? And then also for the starts that you had, just any more color on how many were IBIG or switches, delirious refractory patients,
spk06: Thanks so much.
spk21: Thanks, Akash. And I would like to give the floor to Carl to talk about, you know, projections for next year. But before I do so, maybe Keith, you want to comment on that second question, right?
spk15: Yeah. Akash, IVIG experience patients, it was almost 50%. The percent was almost spot on to what we saw in the first quarter of treatment. As far as the rest of the patient population, they came from across the board. So it is some that are moving into earlier treatment. But we do continue to get Solaris refractory patients that are added in quarter two. This is not a surprise. And, you know, I'm happy to share with you that some of our Solaris refractory patients in quarter one are indeed VivGuard responders.
spk21: Thank you, Keith. Back to Carl. Akash, thank you for the question. We are not going to give any guidance on patient numbers or revenues at this time. It is just too early in the launch. We want the data to mature, and then we can start thinking about giving guidance on revenues and patients. Thank you.
spk07: Your next question comes from the line of Matthew Harrison from Morgan Stanley. Your line is open.
spk04: Can you hear me this time? We can, Matthew. Welcome. Thanks. I guess I got too excited last time. So just a question for Keith again. Can you just talk a little bit about J-code and if you expect any inflection off of that, or even is there a certain mix of customers that you're not seeing participate very much at this point that you would expect to see participate once you have the J-code?
spk06: Yeah, so Matthew, thanks for the question.
spk15: Go ahead, Keith. Thank you, Tim. Matthew, thanks for the question. And we have our J code, our J code live. It's been running live since July 1st. Really what the J code is doing is it is making it easier for physicians and offices to get patients through the process. It's making the conversion process go quicker from time of prescription to time for actual infusion. So as far as it opening up additional audiences, you know, really the unmet medical need and the success that patients and physicians are seeing with VivGuard is why we've had pretty substantial demand here in the first couple of quarters. So I don't know that you're going to see an uptick because you have a J code as much as you're going to see the ease of getting the patient through the process faster.
spk07: Your next question comes from a line of William Olds from Evercore. Your line is open.
spk03: Hey, guys. Thanks so much. And just a really spectacular quarter. I had a couple questions. So the first one is, you know, you guys are at 85% patient lives covered right now. What's the goal you guys think is realistic by year end? And then my second question is, what are you guys thinking in terms of COGS long-term for extractive gymnastics?
spk21: Hey William, two great questions. Let me start by giving the floor to Keith to talk about how we last covered. And then I would like to hand over to Carl to give a brief comment on Cox. So Keith, why don't you go first?
spk15: Yeah, William, thanks for the question. In regard to coverage, we're really pleased with the progress that we've made through the second quarter. In particular, we're really pleased with a number of the policies. The majority of them are very favorable. They align with the ADAPT inclusion criteria, which means we can have patients as early as second line treatment. As far as the remainder of the population, look, our team is not going to rest. We are going to continue in that minority of policies that are put in place that aren't as favorable as the ADAPT inclusion criteria. We're going to work with those payers to see that we can get them to a favorable status. And the remaining 15% will continue to work on. But we haven't been public with setting the next number forward.
spk21: Thanks, Keith. Carl? Thank you, Tim. So in terms of cost of sales, clearly we're not going to give detailed guidance here, but safe to say as we move to larger production facilities, currently the vials is coming out of Slough, a 1K reactor, then we have Singapore, a 2K reactor, which is also FTA approved, and later on Portsmouth, an even bigger facility will come online that will drive efficiencies. Also important for you to remember is that once you get sub-Q, there is that mid-single-digit royalty for Valazan and so on on the sub-Q. Thanks, Carla.
spk07: Your next question comes from the line of Tazin Ahmad from Bank of America Securities. Your line is open.
spk13: Okay. Thank you for taking my question. I apologize that this has already been asked. I did join a little bit late. But can you talk about the reliability of tracking Scripps intra-quarter because it did seem like Symphony in particular was tracking relatively close to the number you reported this quarter. Is that just by chance or do you think that there is a reliable component to looking at those trends? And then second part of the question is should we expect to see any seasonality in the second half of the year, particularly in 4Q? You know, is it a trend? based on what you know about the GMT space, that patients tend to be less compliant during the holidays, or should we not assume any kind of seasonality? Thank you.
spk21: Hi, Tazine. Thank you for joining us in the call today. I would like to give the first question to my colleague here, Carl. How do we think about the reliability of tracking scripts, please? Hi, Tazine. Thank you for the question. I think if we look at symphony data and IQ, it is... It's difficult for us to comment on it. We don't know how the algorithms work. I think it takes time for those algorithms to get more accurate. At the moment, I'll be very careful in using it. And also, if you look forward, the algorithms don't know the discontinue factor, which needs to be built in. The cycling, if we start patients moving from one cycle to the next cycle, none of those will be built into the algorithms. I think we really need to be careful in using these data points. Thank you, Carl. Keith, is there anything you want to say with regards to potential seasonality in the treatment of GMG patients?
spk15: Yeah, Tazim, it's a good question. As you know, we haven't been through the holiday season yet with VivGuard commercially available. But what I will tell you is that when you're treating a patient that that has been through other therapies and not had a positive experience, and now they're able to have a quality of life that's similar to someone that doesn't even have the disease, they're going to want to stay on therapy. I have one of our patients that happened to walk up to me and said, I actually didn't realize how bad off I was or how different my life was until I was being treated and realized what I had been missing out on. So I'd actually If I put my mind in that of a patient, I would say, hey, I want to be treated so I can enjoy my holidays even more. So let's wait and see on the seasonality, but I'm not expecting it.
spk13: Okay.
spk07: Thanks, Keith. Your next question comes from the line of Thomas Smith from SVP Securities.
spk06: Your line is open.
spk10: Hey, guys. Thanks for taking our questions, and let me add my congrats on the really strong launch here. Just wanted to follow up on the new patient start. Can you give us any more granularity on kind of the monthly pace here through the quarter and into July? Maybe just help us better understand the shape of the curve here as we exit the quarter. And then it's obviously off-label in the U.S., and I know you aren't promoting to it, but can you comment on whether you're seeing any DIVGARD uptake in seronegative patients in the U.S.? I appreciate the comments on Japan. I'm just curious what you're seeing in the U.S., whether you're seeing prescribing for these patients, whether payers are facilitating access here. Thanks.
spk21: Thank you, Thomas. Thanks for being with us on the call today. I'd like to give the first question to Karl about new patient starts and monthly visibility. And then, Keith, I'd like to hand over to you for any comments we can give on the question regarding off-label. But, Karl, could you start, please? Thank you. And, Thomas, thank you for the question. What we see is a gradual, consistent month-over-month growth. Thank you.
spk06: Keith?
spk15: Yeah, thanks for the question, Thomas. So as you pointed out, in Japan where the label is broader, we are seeing utilization in musk patients, in seronegative patients, also in acetylcholine receptor positive patients. So a real nice across the label utilization in the first quarter of launch. As far as in the US, we remain consistent with the label. We will not promote off-label. That also means that if it is a patient that is off-label, they're not able to participate in our MyVivGuardPath program. So if there is, we have far less insight into these off-label patients. Thank you.
spk07: Your next question comes from a line of Jason Butler from JMP Securities. Your line is open.
spk02: Hi, thanks for taking the question, and let me add my congrats on the quarter as well. Just had a question about OncoVerity. Have you already established a development strategy for KUZA, either the overall ML population, subpopulations? How are you thinking about combinations with standard of care? And then is there a specific funding commitment from Argenix to the new entity? Thanks.
spk21: Thanks, Jason. This is a deliberate choice to spin off Fusituzumab in an asset-centric company. It means we can continue to focus and double down on our autoimmune franchise. Now, the co-creation in play here is a unique one because the translational biology insights from Dr. Clayton Smith on the role of the CD70-CD27 pathway in either venetoclax non-responsiveness or refractory patients is a unique complement to what we already have in our hands with our first-in-class assets and pretty strong data in first-line AML patients unfit for transplant. So expect the development path to be a precision shot on goal, fine-tuned with the know-how of Colorado University in terms of inclusion-exclusion criteria for our AML patients. This is an asset we believe in. This is a company we will shoulder, and therefore, yes, there is a financial commitment, both from Argenix and from Colorado. It's a two-step commitment, where first we need to finalize a specific piece of homework, and then actually there's a second, bigger branch of commitment from our company. So we're very pleased with the path forward. I think it's a data-based path forward, and we're equipping QSF to use a map with the highest probability of success. So thank you for the question.
spk07: Your next question comes from the line of Alex Thompson from Stiefel. Your line is open.
spk01: Hey, thanks for taking my question. Congrats on the quarter. I wonder if you could go back to GMG, if you could comment a little bit on net price per patient in the U.S., whether that's tracking with your initial expectations so far, and also if you'd comment on in that price per patient in Japan. Thanks.
spk21: Alex, thank you for joining us on the call today, and this is a question, I think, for Carl, right? Yeah, thank you, Tim. Alex, thank you for the question. The typical price for a patient, 225, that number which we communicated at the beginning of the year, still stands. We don't see any reason for that changing. The gross to net, everything adds up to that number. In terms of Japan... The price for a VAL in Japan is 421,000 Japanese yen. If you do a conversion, you'll see it's between 50% and 60% of the U.S. price. Gross to net in Japan is a typical gross to net. It's a very small number there. Thanks, Carl. Thank you.
spk07: Your next question comes from the line of June Lee from Truist Securities. Your line is open.
spk11: Hi, thanks for taking our questions. For the advanced SC, what specific learnings from the IV trial have you incorporated? And other than just the size, I mean, more specific to the increase in the size and powering assumptions, can you comment on the number of patients you additional, the additional number of patients you're enrolling? Thank you.
spk21: Thanks, Jun, for the question, and thanks for being with us today. So as we said, the benefits of having the two trials running in a slightly staggered fashion is that we can deploy learnings from the first study and see how they impact the second sister study. And remember, for the first study, we had a very convincing sign of efficacy in the IWG score. Primary endpoints was a clear win, but with a smaller power than we had anticipated. So we have been revisiting the initial powering assumptions. We took a blinded look at the patient population for the second study. It looks like, broadly speaking, a similar patient population as for the first study, and therefore we can now be more accurate and precise in our powering assumptions. That means that in order to achieve the power we feel comfortable with, we need to up the number, and that's why actually we are reopening the study to add a number of patients. I would like to hold off until we're public on clinicaltrials.gov to show you that number, but it's not a dramatic number. It's, I think, a reasonable number. to give us a significant increase in power. So stay tuned. It will pop up on clinicaltrans.gov soon. Thanks for the question.
spk07: Your next question comes from a line of Colin White from UBS. Your line is open.
spk18: Hi, Colin White here on for Laura Sutcliffe at UBS. My question is about UCB's termination of ROSA and ITP. And if that gives you any pause for thought in terms of the commercial opportunity in the therapy area. Thank you.
spk21: Hi, Colin. Thank you for your question. Yes, so we also took note of the UCB news. I think it's good news for us. It means there is less competition out there. I think we're formulating our plans and our strategies based on our own data. So I think our conviction level in the potential of FGT and modern ITP is significant. I think the data we saw in such a refractory patient population are actually quite impressive with a more than 50% IWG response rate. I think the safety profile came out very consistently and very clean. And we have documented very carefully the unmet medical need in ITP. I can also tell you that the initial feedback from the field from treating physicians has been very positive and very consistent. So we're steaming forward with our ITP opportunity. Thanks for the question.
spk07: Your next question comes from a line of Douglas Sayle from HC Wainwright. Your line is open.
spk05: Hi, good morning. Thanks for taking the questions. I think in the slides you indicated that most scriptwriters... Hello, can you hear me?
spk21: We can hear you fine, Doug.
spk05: Okay, yeah, sorry. Just... You indicated most writers have written one to two scripts. I'm just curious, do you have any perspective in terms of the types of patients they're selecting for those initial scripts? Is there a certain profile? Are they waiting for the patients who are perhaps not doing as well on IVIG or their existing therapy? Or is it just sort of happenstance just to be the first patient that walks in the door since the approval?
spk21: Thank you, Doug. That's an excellent question. And Keith, would you mind taking this one?
spk15: Yeah, happy to do so. So, Doug, we don't actually see an enormous difference in the 78% that have written one or two compared to the remainder that have more patients on therapy. Again, with almost 50% of the patients experiencing IVIG prior to getting VivGuard, it appears that in the early going, we have a higher percent of refractory patients than we expect to have in the future. As I mentioned, we'll continue to try to get earlier in the treatment paradigm, so certainly placed before IVIG and certainly placed before other infusible therapies. But right now, as a new option on the block, we are getting a high number of refractory patients. The good news is I think that those physicians see that experience and the experience that their patient is having. That's where we have the opportunity, the real opportunity to advance earlier into the treatment paradigm, but also take that 78% and take them from being with one or two patients on therapy to now even more than that.
spk07: Your next question comes from a line of Joel Beattie from Baird. Your line is open.
spk19: All right, thanks for taking the question and congrats on that quarter. Going back to an earlier comment about how 1,000 patient ads was extraordinary and it seems not to be expected to be repeated, is that based on feedback that there may have been kind of a pent-up demand effect here?
spk21: This is a great question and an opportunity, Keith, to reiterate the views of the company and the position we're in. Keith, why don't you go ahead?
spk15: Yeah, happy to. Actually, Joel, the point that we're making is that it was a very powerful quarter in patient ads. I mean, 1,000 GMG patients and a quarter in a rare disease is quite a number for any therapy. And so the fact that we were able to deliver that is quite impressive. All I was saying is that if I was... Building out my model, I don't think I would be placing that as all of a sudden the new standard that you can expect to occur quarter over quarter.
spk07: Your next question comes from the line of Manos Masterakis from Deutsche Bank. Your line is open.
spk17: Yes, hello. My question was asked just earlier in terms of the ITP opportunity, but maybe I could just add if you could say anything else. more quantitatively perhaps about the market opportunity.
spk06: Thank you.
spk21: So for ITP, what we learned with the data in hand from the field and from the physician audience we have been interacting with is that there is substantial medical need in primary ITP patients. What basically happens today is that when patients are moved from a steroid to a TPO, and they fail a first TPO, they would be typically cycled on a second TPO. And now that there's a third TPO out there on the market, even to a third TPO. So it doesn't make a great deal of sense to switch patients between medication using a similar mode of action. It doesn't make sense to move them after a first TPO failure onto a totally different mode of action, which actually has proven to be a very powerful mode of action, which is the reduction of pathogenic IgGs. which are responsible for clearing plate, but also attacking the mega carrier sites. So we think there is a clear positioning here for third line therapy in the ITP market space. What we quantitatively hear from physicians is in line with what some of our analysts have been writing. For example, I have been reading about a 10% market share based on a third line positioning. The company, based on its data and its market research, would actually feel comfortable with that.
spk07: And there are no further questions at this time. This concludes today's conference call. Thank you for your participation. You may now disconnect.
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