This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.
spk14: We continue to deliver strong results from our global ZipGuard launch, making progress towards our goal of bringing a paradigm-shifting treatment to people living with generalized myasthenia gravis. We set out at the beginning of the year to prove that we could launch a drug that, with our experienced team and core strategies, we could transition into a global, integrated immunology company. Now, three full quarters into the launch, we have generated almost $230 million in net product revenue, demonstrating that our capabilities as an organization extend beyond antibody engineering, immunology research, and clinical development, and into bringing in new therapy and new modality to patients. Slide four. We have identified three key drivers of our launch success, which we hope to replicate as we plan for the anticipated approval and launch of our sub-Q products in the first half of next year. First, the unmet need and severity of the disease was even more significant than we realized, and the demand for a new treatment option has been high. During the third quarter, we crossed the 2,000 mark of patients on drugs globally. We hear every day how debilitating the disease is And in fact, our market research shows that severe Mg is second only to ALS as the worst disease neurologists treat. Many patients give up their jobs due to the severity of symptoms or side effects from current medication. They require walking support or feeding tubes because they have difficulty swallowing. Many GMG caregivers are also out of work to provide full-time care to their loved ones. To quantify this burden, we have invested a lot of time in gathering real-world evidence, which has corroborated the anecdotes we heard on significant loss of productivity and impact on quality of life. Second, the data with healthcare providers is setting a new standard of treatment based on the efficacy and safety profile observed in the data and ADEPT+, and these data are translating in clinical practice. We hear positive feedback from physicians on the speed of onset and depth of response, including patients achieving minimum symptom expression. In fact, we observed 40% of all treated patients achieving MSP in ADEPT after just one treatment cycle. This is quickly becoming an essential outcome measure for patients and physicians. And third, the core strategies we put in place appear to be the right ones, and we have strong engagement with our patients, physicians, and payers. We still provide updates on these strategies, but I'm very proud of our cross-functional team, which has worked closely in collaboration to achieve these results. Slide five, as you know, We believe GMG is just the beginning for this card, and next year will be a busy year with label expansion opportunities. Topline data are expected from our ongoing registration trials in CIDP, ITP, and PV, and each of these indications represents a sizable opportunity given the treatment gaps patients still face. We implemented innovative trial designs across the board that will provide us with important data for physicians in how they treat their patients. This year, we continue to anticipate top-line results in CRDP in the first quarter of 2023. Slide six, we'll enhance our learnings from precedent trials to design a year in an innovative way. Patients need to have confirmed, active CRDP in order to roll into stage A. where they must demonstrate a response to Epgraftigemot before advancing to stage B. In stage B, patients are randomized to stay on Epgraftigemot or switch to placebo, and the study stops once you have 88 events or relapses. Remember, this is an event-driven trial, which means you monitor the rate of dropouts to assess timing to data. We will, of course, continue to update you on this timing as necessary. We're also expecting top line results in the second half of 2023 from our second IPP trial, Advanced SubQ. The two advanced studies will support registration in the effort. Slide seven highlights results from the first advanced IV trial. Shortly after we reported data from the advanced IV study in May, we held an advisory board to gain feedback from the hematology community. The physicians were very excited by the fast onset of action and increased separation between the placid counts of treated patients and placebo throughout the study. They were also encouraged by the safety profile, which was consistent with what we observed in ADAPT. Consensus was that these data support positioning of VIFGAC as a third-line treatment option after a first TPO. They agreed that depending on additional data, there could be potential in early life patients as well. Slide eight. Many of the physicians on our advisory board were part of the ITP International Working Group that was formed to build assessment criteria of clinical trials more aligned to clinical practice. The IWG agreed that the goal of an ITP therapy should be to get patients to a safe place at camp to prevent clinically significant bleeding and to do so with little toxicity. In advance, 51% of treated patients were responded based on the IWG criteria, defined as achieving platelet counts over 30,000, a two-fold increase from baseline, and the absence of any bleeding. This IWG response rate resonated particularly well with our hematologists and further support our expected disregard positioning With the Drascopenticus, we expect data in the second half of 2023 as well. By 9, this is an indication where we launched translational studies to uncover more about the carcinoma based on the durable remissions we observed in phase 2. In these patients, autoantibody levels did not return to baseline in the same way as total IgG levels. Looking deeper, is correlated with a reduction in the autoreactive B cells, which are responsible for producing the autoantibodies. We are now looking more broadly across the indications to see if we observe a similar disease-modifying outcome. This type of translational work is part of who we are as a company. Through our commitment to both patients and the science, we want to advance our leadership in FCRN biology with new data and publications on the differentiation of our FC fragments. You'll also see our commitment to immunology innovation with our earlier stage pipeline and our partner programs, both of which emerged from our immunology innovation program. Our partner programs can take two forms, whether a licensing relationship or a spin-off company based around an Argenix asset. ARGENIX117 is our first-in-class sweeping antibody targeting C2 in a complement cascade. Last year, we shared Phase I data indicating that with repeat dosing, we can reduce C2 levels by over 95% for a sustained period of time. This would indicate the potential for an effective dosing profile. We are currently evaluating ARGENIX117 in a Phase II trial for the treatment of multifocal motor neuropathy, another very serious neuromuscular autoimmune disease, and the second largest IVIG indication within neuro. We believe there's a lot of opportunity with our C2 inhibitors across several of our therapeutic franchises, and we will aim to show the first set of clinical data next year. Rounding out the efforts in our neuromuscular franchise on slide 11, We are on track to file a CTA by the end of the year for our musk agonist, Argenix 119. We will start a phase 1 trial in the first quarter of 2023, in which we will assess patient cohorts at the higher doses, including congenital myasthenic syndrome and musk MG. Like 12, we are also seeing exciting recent progress with two of our partner molecules. with ASRI advancing ARGENIX 115 to the next stage of development and LEO exercising its option on ARGENIX 112. If you look back at our ARGENIX pipeline of antibody candidates, we have demonstrated human proof of concept in eight candidates out of our immunology innovation program, whether in our own hands or with a partner. This is exactly the reason we continue to invest in our innovation engine because it is efficient and productive with a strong track record to date. Before I turn the call over to Carol for a financial update, I want to spend a few minutes talking about my co-founder Hans de Haag, who will retire at the end of this year. Hans leaves us with an incredible legacy of scientific innovation. We founded the company based on his breakthrough in antibody engineering, which has been the backbone to most of our pipeline candidates. But it was his humility and drive to always learn about the scientific breakthroughs of others that led us to assets like Afgat-Tijemot and Argenis-117. We are very grateful that he will continue as an advisor to our Immunology Innovation Program, pushing us to find novel disease targets and promising new pathways, and as a strategic advisor to the R&D committee of our board. He will still gain from Hans' sound scientific guidance while he moves to the next stage of his career. We're also fortunate that we have such strong successes to step into the important role of chief scientific officer. Peter Ulrich has been with the company since 2010 was foundational to the creation of F-cathetumab and has been committed to the development of F-cathetumab since its creation. He watched the first subject ever to be dosed with the drug and has been leading F-cathetumab clinical science since that time. He served as the scientific leader of our neuromuscular franchise, strategizing on F-cathetumab, Argenix 117 and Argenix 119, and most recently took over as head of all theoretical science. This will be a very natural transition for the company, and Hums and Peter will be working side by side for the next couple of months, as they have been now for more than a decade. And with that, I will turn the call to Carl.
spk15: Thank you, Tim. Our third quarter 2022 results are detailed in your press release from this morning. so I will only highlight the key points here. On slide 13, you will find global net product revenues from the VivCard launch for the first three quarters of the year. In the third quarter, we generated $131.3 million in global net product revenues, which was comprised of $124.1 million from the U.S., $6 million from Japan, and 1.2 million from Europe and our distributor markets. Together with the 21 million from the first quarter and the 75 million from the second quarter, this puts our year-to-date global product revenues at 227.3 million. As with previous quarters, inventory in the channel at quarter end was well-managed, and reflects less than two weeks' worth of vials. Slide 14. Total revenues for the quarter were 146.5 million, which also includes 6.7 million in collaboration revenue driven by a 5 million euro milestone from Lille Pharma following the option exercise for Organics 112 and $8.5 million in other operating income. Cost of sales for the quarter were $10.3 million. Our total R&D and SG&A expenses for the third quarter were approximately $236.7 million and $108.2 million, respectively, and can mainly be attributed to FCAR Digimod and other pipeline research expenses as well as marketing and headcount expenses related to our global launch. The increase in research and development expense was mainly driven by the recognition of a priority review voucher submitted with a DLA filing for sub-Q at Cartigamot. On the cash balance, we ended the third quarter with almost $2.4 billion in cash, cash equivalents, and current financial assets. We continue to expect to utilize up to $1 billion of available cash in 2022, which will support our ambitious growth plans, specifically the rollout of our global launch, clinical development of Epcot Igimot in 10 indications and Organics 117 in 2 indications, investment in the global supply chain, and the pipeline expansion through our immunology innovation programs. You can find additional details behind these numbers in the press release we issued this morning. I'll now hand the call to Keith for a commercial update.
spk05: Thank you, Carl. Slide 15. I'd like to start by saying that I'm really proud of our global team for their execution and accomplishments over the last year. We are now 10 months into our U.S. launch, five months into our Japan launch, and almost two months into our Germany launch. Every day, I see dedication to deliver on our mission to serve GMG patients who are suffering from this devastating disease. We have now filed in Israel, Canada, and China, so 2023 will bring additional approvals that will expand our patient reach even further. As Tim mentioned, our core strategies are working to engage our key stakeholders, patients, physicians, and payers. Slide 16. On the patient side, we saw more than 50% growth of GMG patients globally on therapy, which indicates we had another quarter of significant demand for VivGuard. We believe we are setting a new standard in how GMG patients can manage their disease based on the efficacy and safety data we have shown in ADAPT. Most importantly, the data we saw in ADAPT are translating into the real-world setting. Approximately 50% of our patients are still coming from IVIG, meaning that IVIG is the most advanced therapy that they have experienced. We will be watching closely over the next several quarters to see if we continue to see the shift into earlier treatment segments. We want to expand to reach those patients who have only experienced mestinone, steroids, or broad immunosuppressants. This will be an indicator of our long-term trajectory on our path to reach 17,000 addressable patients. Our sales team has done a great job engaging a broad group of neurologists focused primarily on their top target physicians in the first quarters of launch. We still face the effects of the pandemic, So the decision to hire an experienced sales force has been crucial to our early launch success. By the end of the third quarter, we see continued breadth of prescribers, though most have still only written one or two scripts. The opportunity ahead of us is to drive more experience with current prescribers and reach new potential customers that our field team have not yet engaged with. Moving on to payers. Engaging with the payers prior to approval was a key driver on early uptake of VivGuard, and as a result, patients have been able to successfully gain access. At the start of the third quarter, we received a dedicated J-code, which helped to shorten the time between script and infusion and improve the rate of patients going on therapy. We will be taking a similar approach of the early engagement with payers ahead of the expected subcutaneous decision. Following our European approval in August, we are also spending significant time with payers in this region. We launched in Germany and are able to sell through the Amnog process while we negotiate price. The value dossiers have also been submitted in key countries like France, Italy, and the UK. We would like to point out that the reimbursement process can take several years, so the growth trajectory in Europe is expected to be consistent but much more gradual than what we've seen in the U.S. and Japan. Slide 17, patients and physicians have embraced the individualized treatment approach with VivGuard, feeling that it aligns with the GMG experience. It's still too early to understand the distribution of treatment cycles on an annualized basis, but it seems to be aligning with what we saw in the ADAPT and ADAPT Plus data based so far. We believe the value of individualized treatment approach goes beyond dosing schedule and extends to delivery as well. We are looking forward to the FDA decision on our subcutaneous BLA filing in the first half of next year because it gives the opportunity to bring the patient more ways in which they can individualize their treatment. It also gives us a second opportunity to show our commitment to the patient community by seeking approval in adult GMG patients regardless of antibody status. Slide 18. Before I turn the call back to Tim, I want to once again commend our global team. They have exemplified teamwork and dedication, always putting the patients first as we advance on our mission. We believe we have a truly transformative therapy with VidGuard, and we're motivated every day by the stories we hear from patients. We know that the connections we are building now within our neuromuscular franchise will serve us well as we look ahead to our future launches. There are still many unknowns on the long-term variables of our launch, but we are delivering where we can to generate demand and convert that demand into patients on therapy. Tim?
spk14: Thank you, slide 19. In closing, we are very happy to report another strong quarter of results. We are energized by the early response to VivGuard and the engagement from our stakeholders. We are ready to drive forward in 2023 with more global launch expansion and approval decision on our subcutaneous product in GMG and several key data readouts with EvGuard Digimop and Digenics 117. Autoimmunity is ready for a revolution patients across all of our indications need more options especially options that may come with a fast onset of action a robust depth of response and few side effects similar to what happened in the field of oncology we are shifting towards precision interventions in autoimmunity, and algenics is part of this shift. We believe that if we stick to our strategy of focusing on both the science and the patients, bringing first-in-class immunology breakthroughs to the patients who need them, that we have a significant future of value creation ahead. I would now like to open the call to your questions. Operator?
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 that you please limit yourself to one question and then rejoin the queue for further questions. Your first question comes from the line of Yaron Werber from Cowan. Your line is open.
spk18: Great. Thanks so much for taking the question and congrats on a terrific quarter team. Maybe just a quick sort of integrated question. Carl, can you give us a little bit of a sense on what the gross to net impact is? I know you typically give it every six months in your financials, but is there any change from what we saw sort of in the first half? And then secondly, are you expecting typical seasonality sort of in Q1? I know it's obviously an IV product. It's a buy and build, but still, is there going to be an impact? Thanks so much.
spk13: Thank you, Jeroen, and thank you for joining us today. I'm going to hand the first question, indeed, to Carl, and then, Keith, I will ask you the question on possible seasonality rights. So, Carl?
spk15: Jeroen, thank you for your question. Indeed, we don't provide detailed financials in Q3. If you go back to the Q2 financials, which is on our website, you'll see the gross to net ease there. It's in note 12. It's 88%.
spk05: and we don't have an update on that and also no reason for that to move materially so thank you i think on the second question excuse me on the second question about seasonality um i'll tell you one thing that we're learning during this launch is that uh gmg is very promotionally sensitive uh which means when there's times when our team is not in the sales out in the field or physicians and customers are not in their office, we could potentially see a lag. We also know that here in quarter four around Thanksgiving and towards the end of the year, there are fewer days that infusion centers are open, and you may have fewer patients making trips to their doctors. So we're expecting some seasonality. Thanks.
spk04: Your next question comes from the line of Tazin Ahmad from Bank of America Securities. Your line is open.
spk16: Hi, guys. Good afternoon to you. Thanks for taking my question. A real quick one here for your sub-Q for VivCard. Can you just remind us if there's any differences in how it's dosed relative to the IV formulation, specifically the regimen, the cycle interval? And to the extent that you can, how are you thinking about pricing for that formulation versus the current formulation of IV? Thank you.
spk13: Thank you, Tazin. Thank you for joining us today. I will quickly handle these two questions. So think of the sub-Q product presentation as being dosed in exactly the same way as we do for IV. Remember, it's the explicit wish of patients to go to individualized dosing, and therefore the sub-Q product has been tested and will be marketed following the same individualized dosing as we did for IV. From a pricing point of view, the homework is still in full process. So Stay tuned. We will communicate about the price in a similar way as we did for IV when we get closer to the market.
spk14: Thanks for the question.
spk19: Thanks, Tim.
spk04: Your next question comes from the line of Yatin Sineya from Guggenheim Partners. Your line is open.
spk03: Thank you very much. A couple questions for me, mostly on the CIDP front. Could you just comment on how you are handling the use in the study? That's the first part. Can you also talk about the historical relapse rate that we should be thinking about? I think when we look at the literature, it's somewhere around 45, 50%, and you're looking at 88 events. What does that translate to? And what would you like to show once the data are out? What sort of efficacy profile you are looking for? Thank you.
spk13: Thank you, Yatin. The way the CIDP trial has been designed is such that after we confirm or validate your CIDP diagnosis, we will basically take away your current medication and see you worsen to a certain degree on any of three scales. And then we will try to restore what you lost in terms of function with a catheter monotherapy. So that's how you have to think about how we deal with steroids. They will basically be taken away from the patients. Remember that we enroll naive patients, so newly diagnosed patients on steroids or patients on IVIG. So we do it with both steroid and IVIG patients. Newly diagnosed patients, of course, will not have to go through that first step. Then on expectations around response rates, the IVIG study, which we typically compare and reference to, would be the ICE trial. Were you roughly saw a 50% response rate on IVIG? Nothing to say on relapse rate. It's too early to comment from that point of view. Thanks for the question. We can move to the next.
spk04: Your next question comes from the line of Manos Mastarakis from Deutsche Bank. Your line is open.
spk02: Hi, thank you for taking the question. So I just wanted to ask if you could give us an update on the duration of treatment and the average number of cycles that you are seeing so far since launch in the real world, as well as how should we think about Germany and Japan ramp versus the US ramp uptake, as well as finally, if you could comment on the impact of the J-code launch in July and how should we think about its impact, basically. Thank you.
spk13: Thanks for the question. And maybe, Keith, you want to talk about how we see the distribution of the cycle use emerge in the real world compared to the depth study, and then maybe also briefly comment on how the Germany and Japan grant is going. On J-code, we can be very brief. This is mainly facilitating the turnaround time between enrollment and drug administration. But, Keith, go ahead. On the first two questions, please.
spk05: Sure, happy to do so, Tim. So first of all, on the duration of treatment in the real world, what we're seeing is very consistent with what we've seen in the ADAPT study. I can tell you that we're getting to a point where more patients and physicians are getting very comfortable with the individualized dosing because I think one of the key things about the individualized dosing is that once each individual patient has established that interval between cycle one and cycle two and then between cycle two and cycle three it stabilizes and that becomes that becomes their own individual cadence it makes it very easy to project when they'll need their next cycle and very easy to schedule their time in the infusion chair right now it's still too early to give annual projections But I would say that we're on target for what we've seen in the ADAPT study and the ADAPT Plus study on the typical patient requiring, you know, roughly five cycles per year. As far as the Japan launch and the Germany launch, in Japan, you know, we have the broadest label on the globe where we include seronegatives. I'm really pleased to share that we're adding not only seronegative patients, but also acetylcholine receptor positive patients. We have consistent results. growth in that marketplace through our first two quarters. And so we believe that the Japan pace of adding patients will continue to remain consistent. Germany, we're barely two months into the launch. We're off to a nice start, but as you know, we're negotiating reimbursement. What I can say, having spent a few weeks in Germany last month, is that the unmet medical need for GMG patients in Germany is no different than we see other places on the globe. They haven't had new therapies available to them in decades, and they're really looking for a new option in their treatment.
spk06: Your next question comes from the line of Derek Aquila from Wells Fargo.
spk04: Your line is open.
spk11: Hey, good morning. Thanks for taking the question, and congrats on the progress during the quarter. So just two quick ones from us. I guess know one of your future competitors has kind of commented on fcrm's potential indications like ra uh that might not solely be auto body uh and auto antibody driven so just kind of curious if you've done any pre-clinical work there and just your thoughts on pursuing those types of indications uh and then also is it fair to think that a good benchmark for the pace of enrollment for both the ballot trial and the uh alkydia trial uh would it be similar to cidp thanks
spk13: Thanks, Derek. Thanks for being with us today. On enrollment speeds, it's very difficult to triangulate from current or past trials. I would say that each trial has its own dynamics, and enrolling a global trial in a rare disease is always hard work, as we know. So stay tuned, Derek. We're going to update you on a regular basis on how enrollment will be going. On the topic of rheumatoid arthritis RA, we're not excluding that subsets of patients actually would see an active role of autoantibodies. It's just that the biology is less clear, it's less linear than what we like to see for the indications we prioritize. So we always start from a solid biology rationale and then we take it from there. So this is not a priority indication so far for us. Thank you.
spk04: Your next question comes from a line of Alex Thompson from Stiefel. Your line is open.
spk08: Hey, thanks for taking my question. On sub-QF Gratigma, I was wondering if you could comment on your confidence in obtaining a broad label given that your new evidence here is from the IV formulation and sort of how the FDA might view that. And then assuming you do get approved in a broad label, how much larger do you view the addressable population? Thanks.
spk13: Thanks, Alex. Thank you for this question. So you do know that we have a longstanding commitment to the MG patient population. We do have evidence that abgapticamot may work in the seronegative patients. We saw that not only in the open label extension study ADAPT+, but we also saw that in the sub-Q study. So these data have been submitted, and we are indeed going for a broad label. The outcome of that interaction with the regulator, of course, will have to be seen. So let's leave that as a review issue, but it doesn't diminish our long-term commitment to the MG patient population.
spk06: Thank you.
spk04: Your next question comes from the line of Matthew Harrison from Morgan Stanley. Your line is open.
spk07: Great afternoon. Thanks for taking the question. I guess two things I wanted to ask about. So the first is just a follow-up to the prior question. around some of these larger indications where the biology is less clear. Just how do you think strategically about that? Obviously, the strength of the business is improving and sort of your position in terms of ability to see sort of the runway of where revenue could be is improving. How do you think about potentially taking some more risk in terms of some of these potentially larger indications? And then secondly, just a question on on phasing. I know people have asked about Japan and Germany. Just maybe more broadly, as you think about some of the other countries that could come online next year, anything we should be thinking about specifically about those countries in terms of phasing? Thank you.
spk13: Thank you, Matthew. And I will hand over the question on global expansion to Keith in a minute. On the larger indications, Matthew, of course, we'll be looking at some of them. I think we're pretty explicit about what our next priorities will be in terms of the 10 indications we're targeting. Remember that we stated the aspiration to be in 15 indications by 2025. I think we're well on track to do that. But for the moment, we're not going to compromise on the biology rationale for these indications. So we will always start from biology, then overlay that with clinical feasibility, and then finally look also at commercial opportunity We like commercial spaces where the unmet medical need is very high and where you actually have an ability to influence the treatment paradigm, similar to what we have seen for some of the indications where we already play. And maybe, Keith, you want to address the second question on global expansion, what to expect next year?
spk05: Sure. Happy to do so, Tim. So, Matthew, as you know, we have the European regulatory approval, but we actually only promote in one country in Europe right now, and that's Germany. In the other countries, we have submitted dossiers so that we can get reimbursement and then begin to promote. So, you know, I call out particular to you, France, Italy, the UK, where we'll continue to make progress. But that's just the beginning. So when you think about phasing, think about our desire to be able to serve patients across the European Union. Secondly, we mentioned Israel. Canada and China, where we've already filed. So I think you can expect to see us to begin to commercialize in those countries in 2023. And I think next year we'll give further clarity on some of our expansion plans to other parts of the globe so we can maximize our ability to serve patients.
spk04: Your next question comes from the line of Danielle Brill from Raymond James. Your line is open.
spk20: Hi, guys. Good morning. Congrats on the quarter, and thanks for the question. I'm curious how we should be thinking about the cadence of new patient ads moving forward and then looking ahead to the sub-Q availability, what impact that might have on broadening use. Are there any early indications that docs are waiting for the sub-Q to prescribe more broadly?
spk13: Thank you. Thank you, Danielle, and thank you for joining us on the call today. Keep with your minds taking these two questions, please.
spk05: yeah happy to do so so danielle as far as new patient ads you know i think we gave some some clarity here that we passed the 2000 patient mark during quarter three and we've seen over 50 percent growth uh in quarter two for over quarter two in our patient ads but i think the real question is what's the trajectory going to continue with the launch um you know all cons all of our demand indicators continue to show consistent growth but you have to remember that right now So many of our patients, almost 50%, are coming from their most recent therapy being IVIG. If we don't change that as our main pond that we fish in, that pond will begin to dry up. So our long-term trajectory is gonna depend on our ability to shift into earlier lines of treatment of patients that have only experienced oral therapies and be the first infusible therapy that is utilized. As far as subcutaneous and pent-up demand, In the market research that we've done with physicians and with patients, this is providing optionality. So it's providing options to patients. It's providing it to the healthcare professionals, but also to payers. I think that we will see our sub-Q uptake, a major part of it will be reimbursement and the fact that sub-Q will go into Part D versus our IV that goes in Part B and what's associated with out-of-pocket expense. The bottom line, as a company, we are agnostic as to which formulation a patient or a healthcare provider or a payer selects. We want to make sure that we can serve as many patients as possible, and that's why it's going to be beneficial to add this to our bag of tools. Thank you.
spk04: Your next question comes from a line of Akash Tiwari from Jefferies. Your line is open.
spk10: Hey, guys. So a couple. How comfortable are you with the perception that BFGAR could have IVIG-like efficacy in CDP? We've seen UCB fail in this indication with a different trial design, and then even your team kind of baked in a go or no-go interim. You know, so are you comfortable with that base case expectation, and is it possible that BFGAR could actually differentiate from IVIG from a response rate perspective? Additionally, on the GMG launch, it looks like new patients did start to slow down to kind of 600. Is that kind of a fair assumption on a go-forward new patient ad in the US for next year? Or could that start to pick up once this opportunity comes in line? And what have you seen in terms of your persistence rate so far? So basically, patients who started on drugs in Q1, what percent of those patients are still on drug today? Thank you.
spk13: Thank you, Akash. I will give you question two and three to keep talking about, you know, how we look at patient ads going forward and discontinuation. On CIDP, Akash, we have always been very transparent about why we think this is an IgG-mediated disease. We derive confidence and conviction from the immune absorption data, the plasma exchange data, and to a certain extent, the IVIG data. I think we also went at great lengths to explain what typical risks or pitfalls are associated with the running of clinical trials in CIDP. It's very tricky, and I think we have a rational approach to de-risking the study. This being said, it's still a clinical experiment. I mean, if we would be 100 percent sure, we would not need to do the experiment. I think it's reasonably de-risked from a trial design point of view, this conviction on biology. But let's do the experiment first now and then talk about outcomes. Keith, do you want to take the two remaining questions, please?
spk05: Sure, happy to do so. So when it comes to new patient ads, you know, all I can say is that our indicators for demand continue to show consistent growth. So we continue to get new patients that are interested in being on VivGuard and new scripts coming in. We're not going to give projections on the trajectory as we're just three quarters into this. But overall, as I mentioned before, we are going to need to expand into the earlier treatment lines. I can say directionally, we have been headed that way with more patients, VivGuard being the first infusible therapy that they worked. but still the majority of our patients are coming from IVIG. Second, in regard to discontinuations, we have seen some discontinuations. That's really not a surprise. As you know, between cycle one and cycle two, VivGuard works in roughly 80% of patients that are exposed to the product. So we would expect to see a discontinuation rate. We're not at that level at this point. That's not really that big of a surprise either, considering that the majority of patients that are on VivGuard started on therapy in quarter two and quarter three um so overall um no exact projections on discontinuation rate um but you know we expect that we will continue to see some over time your next question comes from the line of joel beattie from baird your line is open hi thanks for taking the question but i i understand that
spk21: 50% of patients are coming from off of IVIG, but looking at it from a different way. So what percent of patients on IVIG are kind of starting on afterectizumab? And how does that compare with what percent of earlier line patients are starting on VivGuard?
spk14: Thank you, Joel, for this question.
spk13: And if you want to address this question on what we think the percentage of patients on IVIG is that is effectively starting on Vivgard?
spk05: Yeah, I mean, Joel, the breakdown that we've given is what we've already shared, which is almost 50%. The last or the most extensive therapy that they were on is IVIG. That doesn't mean that they switched directly from it. It means that was as far as they've gone in their overall treatment paradigm before they switched over to Vivgard. I don't have the exact data on who was actually being infused IBIG and switched directly to VivGuard versus, you know, had they used IBIG three months ago and switched over to VivGuard.
spk04: Your next question comes from the line of Douglas Sale from HC Wainwright. Your line is open.
spk22: Good morning. Thanks for taking the questions. Just maybe when we think about the launch in sub-Q next year, just curious, you know, Have you already started to engage with payers? And obviously with the IV, you know, the pace of getting coverage was really impressive. Do you expect to see a similar percentage of patients covered at the time of launch? And, you know, what dynamics should we think about? I think you touched on it. You know, this is going to be Part D versus Part B that we should consider in terms of the pace of the launch. Thank you.
spk13: Thank you, Douglas. I think we can be brief on this question. It's too early for us to comment. You have seen our modus operandi for IV. We like to reach out proactively to pay it and be transparent about the value we bring to the table and how we think about pricing and calibrate the pricing points. So expect this company to continue to execute along these lines, but we will comment on it when we get closer to launch, okay?
spk14: Thanks for the question. Okay.
spk04: Your next question comes from a line of Allison Bradsell from Piper Sandler. Your line is open.
spk12: Hi. Good morning. Thanks for taking the question. Just another on the VivGuard launch. I know you said the U.S. patient mix by severity has stayed relatively constant since launch, about half of patients from IVIG. But just thinking about the other 50% of patients, I guess – Wondering if you could talk to whether you've seen any impact or slowdown in patients switching from Celiris to VivGuard now that we're a few months into the Ultimiris launch. Or is there any discernible impact to demand that you would call out?
spk13: Thanks. Alison, thanks for being with us today. Thanks for joining, and thank you for your question. Keith, do you want to comment on the other 50% which are non-IVIG experienced and the dynamics there, please?
spk05: Yeah, happy to do so. So Allison, the other 50% is going to come from anywhere across the treatment paradigm. So they can come from patients that have only experienced mestinon, some that have had mestinon and steroids, others that have had, you know, broad immunosuppressants. Also in that other 50%, we do see patients whose most advanced therapy happened to have been rituximab, off-label rituximab. And we do have some patients from C that have been on, previously been on C5s. Quite frankly, we don't really focus on C5 refractory patients or rituximab refractory patients because we think our real competition in this space is steroids and broad immunosuppressant therapies. That's how we get to that 17,000 total addressable market. And that's really where our failed team focuses on.
spk06: But we do get some of those biologic refractory patients in our starts each quarter.
spk04: Your next question comes from the line of June Lee from Truist Securities. Your line is open.
spk17: Hey, thanks for taking our questions. I think one of your C5 competitor in GMG has a bit of a more modest expectation for the addressable GMG patient market, maybe less than half of what you think you can address with Zipcar. Can you elaborate on the differences a bit and how you hope to get there? Thank you.
spk13: Thanks for the question and thanks for being with us. It's not up to us, I think, to comment on the homework of one of the competitors or colleagues who is going to help us to build out this space into what it could be. I think we have been very transparent into how we derive the total addressable market population. I think our analysis is reasonable. It starts from a total patient population as it was published in the first publication in 2001, which we think is one of the most credible sources. And then we look down into how many of those are GMG, how many of those do we think are not well managed with currently used therapeutics, and then we apply reasonable penetration of that patient's group. So I think we can talk about how we do our analysis to come to the 17,000, and we feel comfortable with the assumptions we have been making so far. Thank you.
spk04: Your next question comes from the line of Nick Hellatt from Goldman Sachs. Your line is open.
spk01: Hi there, it's Nick here on Fair Care. Just a couple of questions coming back to the subcut. Has your experience of the IV launch in MG changed your expectations around the long-term IV subcut mix there? And then is there anything we should be thinking about indication specific that you see driving a meaningful variation across the different indications you're going into. Thank you.
spk13: Thank you, Nick. Thanks for joining us. In general, we always said, Nick, that for each indication, we have the ambition to be both on the market serving patients with the IV and the sub-Q product presentation. Maybe, Keith, do you want to share any high-level comments we have concerning the long-term mix between IV and sub-Q?
spk05: Yeah, Nick, I think, honestly, the mix between IV and sub-Q, you'll probably see more dramatic from a geographical point of view than you will see from an indication point of view. I think when it comes to potentially Europe and Japan, you can see sub-Q step in and begin to take over the marketplace as far as Eftartigamod, but still offering both formulations across the globe. In the U.S., I think there's going to be a lot of considerations that will not only be patient-driven and healthcare professional-driven, but I also think there will be a payer-driven aspect, in particular when it comes to the Medicare population. So that could very much affect the mix, regardless of indication within the U.S.
spk06: geography.
spk04: Your next question comes from a line of Thomas Smith from SVB Securities. Your line is open.
spk09: Hey, guys. Good afternoon. Thanks for taking our questions, and let me have my congrats on the strong launch. Just on the DisGuard prescribing patterns, can you talk a little bit about the split between DisGuard use and uptake in the community versus the academic centers? Are you seeing any noticeable differences in prescribing patterns between these two settings, and how has that evolved over the last few months?
spk14: Thank you, Thomas. Thank you for this question. Do you want to comment on this split between community versus academic?
spk05: Yeah, happy to. So, Thomas, we actually haven't given the breakdown of community versus academic. We have made it clear that we have patients coming from both of these settings. I think what's really key to remember here is that, you know, of the 70 percent of prescribers that have utilized VivGuard still have only written one or two scripts. So regardless if they're in the community or academic, that's the overall population. That is our opportunity for growth. The other aspect is, you know, the additional reach of physicians that we need to get to. So, you know, the pandemic still plays into this. Sometimes academic centers are much more difficult to get into. So we still need to increase our reach to potential prescribers.
spk06: Got it. Thanks, guys.
spk04: Your next question comes from the line of Emily Field from Barclays. Your line is open.
spk19: Hi, thanks for taking my question. Just a couple of quick ones. I was just wondering, in CIDP, is the subcutaneous dose of F-carcinoma the same as it is in myasthenia gravis? I was just kind of asking that, thinking about how the number of injections on an annual basis could impact annual pricing per patient in the U.S. And then secondly, just I wanted to ask about your confidence in sort of comparing IVIG response rates and CIDP. It's part of what's underlying, you know, thinking that the ICE study is a good comparator that, you know, as part of their trial design for ICE, they also did require patients to have deterioration going into the study, whereas the other FCRN competitor in their trial didn't show efficacy, noted that perhaps that result was driven by of skewing of stable CIDP patients in the study. Thank you.
spk13: Thank you, Emily. I will leave the question on dosing frequency and potential impact on pricing with Keith for CIDP, but it's going to be a high-level answer only. I mean, we need to wait for the data. Concerning the IVIG response versus an FGAP signal response in CIDP, well, you'll get as good as mine. I think it's a fair calibration point, the 50% or the ICE trial response rate. It also helps us to calibrate what we think we need in order to effectively compete. We have not seen the detailed data from UCB in CIDP, but my mind was initially going first to the fact they did not do an independent verification of the diagnosis. And we know, and we actually made posters on that, We noted about 50% of CIDP patients turned out not to be true CIDP patients. So without such an independent adjudication committee, you just wonder, you know, what patients have been tested in this small phase 2 trial. Let's leave it there for today. And maybe, Keith, is there any high-level comments you would like to give concerning pricing in an indication like CIDP?
spk05: Yeah, Emily, I mean, first of all, the sub-Q is a flat dose. It's 1,000 milligram flat dose. We did that as non-inferior to 10 milligram per kilogram IV. So it is going to be the same 1,000 milligram flat dose for MG as it is in CIDP. As you know from the CIDP study design, which was shown in the slides, we do dose that continuous and much more frequent in the primary part of that study. Let us get the final results from that, as well as look at the data from the open label extension, and then I think we'll be able to provide more clarity around dosing and where that can lead for pricing implications. Thanks.
spk04: And your next question comes from the line of Trevor Allred from Oppenheimer. Your line is open.
spk18: Hey, good afternoon. Thanks for taking the question. Can you give us an idea of how concentrated the U.S. regional contributions have been? Is it mostly on the coast, or are we seeing a totally dispersed uptake?
spk06: Thanks.
spk14: Thank you, Trevor. Thanks for joining us. Keith, is this a question you would like to take, please?
spk05: Yeah, I'm happy to because I'm really proud of the team. The delivery and serving patients is coming across the U.S. Our eight regions and our 71 territory business managers are all on the board with the majority of them, as you can imagine, from this successful launch in the first three quarters ahead of plan. So really pleased. We went out and hired neurology-experienced people, and it definitely paid off for us.
spk06: Thanks. This concludes today's conference call.
spk04: Thank you for your participation. You may now disconnect.
Disclaimer