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spk13: presentation, there will be an opportunity to ask questions. Please note this event is being recorded. I'd now like to turn the conference over to Beth DelGiaco. Please go ahead.
spk18: Thank you. A press release was issued earlier today with our first quarter 2021 financial results and a 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. Our GenX 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 Hauer-Miren, Chief Executive Officer, Eric Costaldi, Chief Financial Officer, and Keith Woods, Chief Operating Officer. I will now turn the call over to Tim.
spk11: Thank you, Beth, and good morning, everyone. We appreciate you joining us today. Starting with slide number three. During our R&D day in 2019, we shared our plan for how Argenix could become a fully integrated immunology company that reaches patients globally who are suffering from autoimmune diseases. We called it our 2021 vision, and it outlines the key drivers that will continue to build value year over year, even beyond 2021. Based on where we are today, we have executed well against our 2021 ambitions. First, with reaching patients. This year, we are on track with our transformation into a commercial organization with the potential U.S. approval of F-Gatigamot in generalized myasthenia gravis, followed shortly by a potential launch in Japan. We are also moving forward in Europe and in China with Xylax. At the same time, we want to be a company known for clinical execution and good business decisions when it comes to pipeline prioritization. We are focused on assets with a lot of breadth, like FGATIGAMOT and Nargenics 117, and have demonstrated strong capabilities in advancing these programs. Notably, we have shown proof of concept in all four of our initial FGATIGAMOT indications. We are hoping for a similar track record with Igenix 117. And finally, we want to be a company that continues to capitalize on early innovation so that we operate at all stages of the value chain. This is the core purpose of our immunology innovation program. We will continue to grow our pipeline with differentiated candidates that emerge from an immunology breakthrough. We will update you on our recent achievements related to each of these key drivers, our path to reaching patients, our clinical execution, and our early stage programs. These are all critical elements of our strategy to become a global, sustainable immunology company. First, our path to reaching patients. We announced this morning that we have filed the marketing authorization application to the dispositions as well for an early cadence of expected launches in MG, first in the U.S. around our PDUFA date of December 17, and second in Japan. We are on track to file our marketing application in Europe in the second half of 2021, and XI anticipates discussions with the regulators in China this year about a potential accelerated pathway. With close to 200,000 NG patients, the market opportunity in China is one of the largest in the world. We are incredibly excited that we've made important progress towards our global launch in just one year since we presented data from the Phase III ADAPT trial. There is one core motivator across all of our hardworking employees, and that is the patients. we've been able to spend considerable time with the MG community and have heard firsthand about the challenges they face. We hear that people living with MG have had to accept a new reality, either due to disease symptoms or side effects from current treatments. This is a truly debilitating disease, characterized by fatigue, depression, and an inability to perform simple daily activities. In some cases, it may result in a life-threatening crisis. Not only does this hinder patients' ability to live their own personal and professional lives to the fullest, but it takes a considerable toll on their friends and families as well. It is clear that the battle we are fighting with MG is far from over. Based on the positive phase III data we showed from the ADEPT trial, shown on slide four, we believe we can offer a new treatment option to patients with a promising value proposition. We showed an unparalleled response rate of 78% across the first two treatment cycles and a fast onset of action in 84% of responders. With a depth of response where 60% of responders achieved an MGA-DL of 0 or 1, patients could think about minimal manifestations of their disease. Furthermore, the trial demonstrated the potential for individualized dosing based on the durability of responses we observed, which may provide enhanced optionality to patients. And importantly, the safety profile in ADAPT was comparable to placebo, which is a crucial element to our key stakeholders. As of today, a significant majority of patients who completed ADAPT and rolled over to ADAPT Plus still remain in study. In addition to advancing IV at Cartigamot, we have also made progress in advancing our SubQ products forward with the goal of reaching NG patients. Slide 5, the ADAPT SubQ trial is underway with a target enrollment of 50 patients. In this non-inferiority trial, we will compare IgG reductions between the IV and sub-Q products at day 29 as the primary endpoint. In addition, we have a safety database requirement for filing, and we'll work to achieve this by switching eligible and intercept patients from ADAPT plus to ADAPT sub-Q. The formulation we are evaluating in this trial which is being used in all ongoing trials of sub-QF-Gratigemot, is equipped with the Halozyme enhanced technology. With this product candidate, we hope to offer patients a self-administered single subcutaneous injection that only takes a few minutes to deliver. We believe that by advancing both an IV and a self-administered sub-QF-Gratigemot, that we are capturing patient preferences and can reach a larger population of people suffering from autoimmune diseases. Before moving to the rest of our pipeline, I'd like to close on MG by sharing my pride and gratitude to our team who strongly executed despite the global pandemic. Between our experienced global launch team, our strong ADEPT data, and our dual development of both IV and sub-Q formulations, we hope to support a new treatment option for people living with generalized myasthenia gravis. This positive readout from ADAPT was not only a significant milestone for the company, but it further validated the role of adjaticumab may have in addressing a range of IgG-mediated autoimmune diseases. This brings me to the second key driver, clinical development within our differentiated antibody pipeline, slide six. As I mentioned earlier, we have demonstrated proof of concepts with F-cardigomab in all four of our initial indications and currently have registrational trials ongoing across each. Today, we have dosed over 400 subjects with F-cardigomab some of whom have been treated with F-Guard for well over two years. With each trial and through our ongoing translational work, we continue to learn more about our FC fragment and how the unique engineering of F-Guard may contribute to the unique efficacy and safety profile we have seen to date. Our most recent achievement within the FGATIGMA program occurred during the first quarter of the announcement that we surpassed a predefined goal threshold in the adhered trial in chronic inflammatory demyelinating polyneuropathy. We had built a planned efficacy assessment into the trial because the role of the autoantibody in CIDP disease progression is less defined than it is for NG. Following this GO decision, we can confidently expand enrollment up to approximately 130 CIDP patients into the randomized portion of the trial, which is depicted on slide number seven. This decision also validated our indication selection strategy as we move into additional adjacent indications within our therapeutic franchises. We're also actively enrolling patients into the ADVANCE and ADVANCE-SUBQ trials for ITP and the ADDRESS trial for PUNFIGUS, which are shown on slides 8 and 9. Given the still unpredictable situation with COVID-19, it is too early to provide guidance on these trials. We will look to provide updates where possible on our upcoming quarterly earnings calls. We're also well underway with our fifth and sixth indications, and we'll be initiating trials this year. We look forward to sharing more about these during our R&D day in July, but have already confirmed that the fifth is within our neuromuscular franchise. Slide 10. As a first-in-class and potentially best-in-class FJRM antagonist, we recognize the vast potential that FGF could have in autoimmunity. We want to roll out new indications as quickly as we can. This is why we were particularly excited to select Zai as our partner in China, because their strong development capabilities will be an asset to enhance the long-term value of FGAT Igamot. Slide 11. By contributing patients to ongoing global trials, we hope Zai will help accelerate the path to approval for each respective indication. And with XyLearning Phase 2 proof-of-concept trials, in future F-Graftigamot indications, we hope to expand the scope of our overall pipeline. We aspire to take F-Graftigamot into 10 indications over time. Slide 12. We also recognize that we may have an other pipeline in the product opportunity with ARGENIX117. We look forward to showing the first clinical dataset mid-year from both an IV formulation and the subcube formulation equipped with Halozymes-enhanced technology. With our Phase I data, we will be showing safety and tolerability, TKPD properties, and we look to identify dosing regimens based on complement biomarkers to take forward into future Phase II trials. Similar to the engineering enhancements we made to our Gratigumab, we also optimized Argenix 117 to have sweeping capabilities. We expect these modifications will lead to differentiation in terms of the dosing levels and schedule we can achieve with our C2 antibody. Like 13, we have identified our first indication for IGENX117 to be multifocal motor neuropathy, or MMN, which will sit within our neuromuscular franchise. We used our proven indication selection strategy for MMN and we'll do the same for additional Phase II trials that we will start. First, we rely on biology. MMM is an IgM-mediated disease where IgM autoantibodies activate complement via the classical pathway. P2 sits at the intersection of the classical and lectin pathway, making it an ideal target for an indication like MMM. We continue to invest in translational work in the disease pathways of MMM and will share more of this data in the future. Beyond the solid biology rationale, there are also known clinical and regulatory endpoints in MMM from precedent trials and a strong commercial case. This is a patient population where a significant unmet need still exists. As you can see, We are very excited to advance ARGENIX 117 forward as we hope to reach even more patients suffering from autoimmune disease. Before we move to the last key driver, our early innovation, I'd like to reiterate that our development program of Cusartuzumab in collaboration with Janssen remains ongoing as seen on slide 14. We announced earlier this year that we are prioritizing the triple combination of CUSA, azacitidine, and venetoclax in the ELEVATE trial. We will make decisions on next steps for the collaboration once we review data from ELEVATE, specifically around response rate, durability, safety, and tolerability, and whether there may be trends to identify from AML subsets in the trial. Slide 15. Now on to our Immunology Innovation Program, or IIP, a centerpiece to our long-term value creation strategy. Through our IIP, we have been able to add value year over year by turning an immunology breakthrough of our academic collaborators into an agenics pipeline candidate. We have done this with each candidate to date. whether it's our wholly-owned assets like Avgot or Argenix 117, or our partner programs with AbbVie, Leah, or Janssen, or asset-centric companies like Agamap or Staten, who are working with Argenix-created molecules. With our wholly-owned candidates, we prioritize those that make sense within our therapeutic franchises in order to leverage core capabilities across the value chain. Slide 16. In order to boost our IIP toolkit, we are continually looking to enhance our technology capabilities. We have our proprietary V-region and FC engineering technologies, Simple, Enhance, Fortelligence, and Advec. We also have our license agreements with Shugai and Clayton to amplify our FC engineering capabilities. With long-term lifecycle management of Edgar Teacher Modern Minds, we are planning for a broad product delivery platform. We have our collaboration with Helozyme, for which we still have four target nominations available. And today, we also announced our recent collaboration with Electrify, a Boston-based company with capabilities to highly concentrate biologics into smaller volumes. While still very early in development, we believe the technology could provide us the opportunity to those at Garte-Jamot and other future products with next-generation delivery systems. Similar to our collaboration with Aerozyme, we have target exclusivity for FCRN and one additional target. These types of technology agreements will continue to be part of our early discovery strategy. We don't intend to communicate on each one, but we want to share our commitment to evolving our overall capabilities as we grow and as the next-generation technologies emerge. We hope that this continued investment will help us build the most differentiated pipeline possible. Slide 17. With the acceptance of both our applications for IVF Graf Tegermatt in the U.S. and Japan, we are solidly positioned for a steady cadence of launches. We're hopeful that the stellar results from the ADAPT trial will position us for success in MG. This is a space in which there has been little innovation and patients are still in need of more options. We are later focused on execution as we, one, grow our team, two, expand into new indications for AdGuard, Three, develop our second pipeline in a product opportunity, Argenix 117. And four, identify new high-potential assets through our IIP. Finally, as we mentioned in the press release this morning, we look forward to providing updates on our deep and differentiated pipeline of assets at our upcoming R&D day in July. With that, I will turn the call over to Eric for a financial update.
spk14: Thanks, Tim. Slide 18 covers our first quarter 2021 operating results, which are detailed in today's press release and regulatory findings. As we stated in this morning's press release, as of January 1, 2021, we changed our functional and presentation currency from euro to U.S. dollars. You will see our financial highlights reported in U.S. dollars going forward. Total operating income increased by $141.6 million for the first quarter 2021 to $167.4 million, compared to $25.8 million for the same period in 2020. The increase was primarily due to the closing of our strategic collaboration for Edgar T. DeMode with XyLab. resulting in the recognition of $151.9 million in collaboration revenue. R&D expenses increased by $17.7 million for the quarter to $122.3 million, compared to $104.7 million for the same period last year. This increase resulted primarily from higher external R&D expenses, mainly related to our evaluation of Elgar-Tigimod in multiple indications and other clinical and preclinical programs. The higher expenses were also due to a planned increase in headcount and the increased cost of the share-based payment compensation plans related to the grant of stock options. SG&E expenses totaled $56.3 million for the first quarter, compared to $27.6 million for the same period in 2020. The increase resulted primarily from higher personal expenses, including the cost of the share-based payment compensation plans related to the grant of stock options, and also consulting fees linked to the preparation of a possible future commercialization of Elgar-Sigismund. We saw an increase in fair value on non-current financial assets of $11.2 million for the first quarter. This is the result of Agomab Therapeutics' closing of Series B financing round. As you are aware, we maintain a profit share in Agomab for granting the license of Arginix 114. Exchange losses total $28.8 million for the three months ended March 31, 2021, compared to an exchange gain of $23 million for the same period in the prior year. Because of the change in our currency, the exchange losses for the first quarter reflect the unfavorable change in the EURUSD exchange rate. Finally, we ended the quarter with cash, cash equivalents and current financial assets totaling $2.9 billion compared to $2 billion on December 31, 2020. This increase resulted primarily from the closing of our global offering in February 2021, resulting in $1.1 billion in net proceeds, and the net receipt of our development cost-sharing payment received from Xilab. These were partially offset by our payments to buyers for a priority review voucher and the other net cash flows used in operating activities. I will now turn the call over to Keith for an update on our commercial activities. Keith.
spk12: Thank you, Eric. To echo Tim's sentiments, it's amazing to consider that the pivotal ADAPT readout of EFGAR-Tigamata EMG was just one year ago. This was a significant gating event, which led us to meaningfully expand and accelerate the development of our commercial organization. The driving force which continues to push us forward as we approach a potential launch is the magnitude of what this treatment could mean for patients. There is a significant unmet need for innovative, fast-acting, safe treatments for people living with MG. We hear this every day from key stakeholders, including the patients themselves, their caregivers, advocacy partners, and the physicians who treat them. As part of our ongoing commitment to the MG community, we recently launched our pre-approval access program. We are preparing for an end-of-year approval in the U.S., but in the meantime, we want to ensure that we can offer F-Gar-Tigamod to MG patients who meet the pre-approval access criteria. Our PAA is currently open in the U.S., Canada, and seven countries in Europe. We also continue to provide IV efcar-tigamod to patients who remain on the ADAPT Plus study and sub-Q efcar-tigamod to patients enrolling in our ADAPT sub-Q trial, whether it's from rollover from IV or as new participants. As Tim already mentioned, we are still the only company actively evaluating both IV and sub-Q formulations of an FCRN antagonist. This is important for us to reach as many patients as possible. We believe this optionality will be a significant competitive advantage from both a reimbursement standpoint and from a patient and physician preference perspective. On slide 19, we remain sharply focused on our engagement efforts with all key stakeholders, including patients, healthcare providers, and payers. For patients, we have awareness and advocacy initiatives well underway, as depicted on slide 20. Our MG United platform has ongoing engagement from over 25,000 unique visitors. Our real-world evidence study continues to enroll and now has close to 2,000 participants. Data from this study, in addition to our health economics outcomes work, will be instrumental in helping us better understand the disease burden associated with MG. All of these initiatives are guiding our engagement efforts with the broader MG community as we prepare for our potential launch. We have also engaged most of the leading GMG treating neurologists through our disease state awareness campaign. Our medical affairs team has been actively engaging with the neurologists as well, most recently at the AAN meeting a few weeks ago. Our MRLs and TRLs are hard at work in their education efforts as we know how crucial this will be at launch given the new mechanism of action. From a payer perspective, We continue to engage with national and regional payers on the potential value that F. vertigimod could provide to MG patients. Our team is also engaging with specialty pharmacies, specialty distributors, and infusion networks to ensure broad access for patients at launch. Another key part of this will be our patient support program, which will be managed closely by our team of nurse case managers who will work towards building a critical infrastructure to help support access for GMG patients prescribed efgar-tigamod following its approval. Slide 21. From a regulatory perspective, as you saw in the press release this morning, we are thrilled to have the JMAA accepted for review by PMDA in Japan with a targeted launch in 2022. we continue to make strategic hires and expand our global organization and support a series of launches in the coming years. We now have over 500 employees globally, including growing commercial organizations within the U.S., Europe, and Japan. While our key functional heads have been in place for more than a year, we're now in the process of hiring our field sales forces. We recently brought on our regional business directors and are starting the interview process for our territory business managers i've been very impressed with the high caliber of candidates we are engaging with as we expand the breadth of experience and core cultural alignment they bring to our organization will help us to get the differentiated medicines to patients in need slide 22. Additionally, we know that the strategic investments we are making now in top-tier candidates will benefit us as we expand our neuromuscular franchise into CIDP and future indications with FGAR-Tigamod and Argenix 117. We plan to execute a similar smart growth strategy for our other evolving franchises as we base our future hires around key data events. Finally, We have strong manufacturing and logistic partnerships in place with world-class companies like Lonza for drug substance, Better for fill and finish, and Cardinal Health for third-party logistics. We're actively collaborating with global regulators to ensure that in-person or virtual inspections can occur as needed. To conclude, we see signs of hope that the effects of the global pandemic will be somewhat attenuated by our December 17th Paducah date, Even so, we are preparing for the likelihood that we will launch in a partially virtual environment and that growth will be gradual and steady as we are engaging with customers regarding this new mechanism of action. I will now turn the call back to Tim for some concluding remarks. Tim?
spk11: Thanks, Keith. Before we begin the Q&A, I would like to conclude with slide 23. We are working hard every day to build the next great integrated global immunology organization that is strongly positioned for long-term sustainable growth. We are well capitalized, and our strong balance sheet will provide a foundation to expand our team and reach new indications and geographies. We have made meaningful progress with our lead assets at Gatigamat and are focused on execution as we advance forward six indications. We look forward to expanding the breadth of this pipeline with the help of our strategic partner, Xilab. We are also approaching the first clinical data readout from Argenix 117, which we hope will launch our next broad pipeline opportunity into meaningful autoimmune indications. And finally, we remain firmly rooted in groundbreaking immunology research. as we grow through our IIT and collaborative efforts in order to help improve the lives of patients around the world. With that, I will turn the call back to the operator to open the call to your questions.
spk13: We will now begin the question and answer session. To ask a question, you may press star, then 1 on your touch-tone phone. If you're using a speakerphone, please pick up your handset before pressing the keys. To withdraw your question, please press star, then 2. In the interest of answering as many questions as possible, each person in the queue is limited to one question only. At this time, we'll pause momentarily to assemble our roster. Our first question comes from Derek Archewa from Stiefel. Please go ahead.
spk05: Hey, good morning, and congrats on the progress, guys. One question. All right. So maybe this is for Keith. You talked about the pre-approval access program for F-card, Ticamod, and MG patients. So I just want to kind of get a sense of what the patient criteria are for getting access. And I guess how are you communicating that program, if you can at all? And is there a cap on the number of patients you can actually enroll into that program? Thanks.
spk12: Good morning, Derek, and thanks for the question. So, you know, we were really pleased to offer the pre-approval access program because this program demonstrates our commitment to the patients who are living with MG. At the time, at this time right now, the commitment is to only for patients that are with MG that cannot participate in a clinical trial. Because remember, if a patient can participate in the clinical trial, we are actively enrolling our sub-Q FGAR-Tigamod MG trial. If they cannot participate in the clinical trial, they can go into the pre-approval access program, but basically it has the same strict inclusion exclusion criteria that we used in our phase three clinical trial. Again, this is just another way to honor our commitment to patients. You know that all of our patients that participated in ADAPT were eligible to roll over into our OLE. and more than 75% of the patients that rolled over are still on. After they complete a year in that OLA, they will have the option to remain on therapy. We're committed to them. Or, as Tim stated in the prepared remarks, they can roll over into our sub-Q bridging study.
spk05: Got it. Okay. And there's no cap on the number of patients you can get into that access program?
spk12: So we have an internal cap right now just because of the amount of supply that we've shipped to our partner, Clinogen. But, you know, we can always change that as needed. Cool.
spk05: All right. Thanks, guys, and congrats again on the progress.
spk13: Thank you. The next question comes from Akash Tiwari from Wolf Research. Please go ahead.
spk02: Hi, this is Amy . Thanks so much for taking our question. On your electricity partnership, is this more of a backup option, or are you seeing any sort of benefit with their microparticulate suspension technology versus halos in areas including AE, or PK? And then if we could just sneak in one more on NG. Given you and both ran 26-week NG trials, but you have an earlier eight-week primary endpoint, has the FDA specifically said anything on what they expect for efficacy between the weeks 8 and 26. And that's it. Thank you so much.
spk11: Thank you for the question. Let me take the Electrify question first, and then I will hand over to Keith for the FDA-related question. So the way you have to think about our collaboration with Electrify is that, you know, we're always thinking five steps ahead. This is a relatively early stage technology, and it's a promising technology if you want to break through the barriers of the typical physical limits of the concentration you can achieve with a biological like an antibody or an antibody fragment. You may remember that at Cartigamot, we already reached a 200 milligram per milliliter concentration, which is phenomenal. But if you want to break through that, you need different type of technology, and that's what we're seeking to access through the Electrify collaboration. The backbone of our subcutaneous product presentation approach continues, of course, to be the helozyme technology. That sub-Q execution is now in play across all our indications. Maybe Keith wants to address the FDA question.
spk12: Happy to do so, Tim. So, Amy, thanks. Yeah, our primary endpoint was, in fact, at week eight. But, you know, we continue to retreat these patients throughout the entire study. In fact, we've shared that, you know, at that primary endpoint at week eight, we had a 67.7 percent response rate. But after a second cycle, almost 80% of patients that were exposed to F-Gar-Tigamod had a response. So we're sharing the continued positive clinical efficacy data with the FDA as we go through a review.
spk02: Okay, great. Thank you so much.
spk13: The next question comes from Kazin Ahmad from Bank of America. Please go ahead.
spk01: Hi, good morning. Thanks so much for taking my question. Just wanted to get a little bit of color regarding the specifics of your collaboration with XyLab. Can you just remind us what are the total milestones potentially expected and when could the next milestone be in the collaboration? Thanks.
spk11: Thank you, Tazine. Thank you for being with us today and thank you for your question on XyLab. It's a partnership we're very excited about. Remember, we spoke about a total of $175 million up front, partially in cash upon signing, partially in equity, and a smaller fraction associated with a milestone, a regulated milestone which will happen downstream. And then all remaining parts of the economics actually situate themselves in a royalty as ILAC would pay to us on net sales in their territory. Thank you.
spk13: The next question comes from June Lee from Truist Securities. Please go ahead.
spk07: Hi. Thanks for taking our questions and for the updates. Can you tell us a little bit about your ongoing dialogue with payers ahead of the approval and how you're thinking about pricing per vial given your individualized dosing regimen, how much of an interpatient or even intrapatient variation are there in frequency, and how quickly can you also get a J-code established post-approval? Thank you.
spk12: Yeah, so June, a few things. First of all, we have a fully staffed U.S. market access team. So we not only have our teams that cover the national payers, but we also throughout the U.S. have a team place that's covering all of the regional payers. So we continue to have regular dialogue with the payers. I want to remind you that, you know, they're pleased with an approach such as individualized dosing because they don't want to have to pay for a therapy that or medication when it's not needed. What does leave the question for them, okay, what can I expect? And so, you know, what we are sharing over time is what occurred in the ADAPT trial and in the ADAPT Plus trial so that we can have an idea of what is the average number of cycles that are going to be required in a year. And you're going to have some, we have some patients that get a really long benefit from one cycle of efgartigamide. And those patients are going to be, you know, the less expensive patients. We have others that are going to require more chronic treatment. But what we will do is look at the average and see how that distribution curve shapes out. And then we are pricing for the annual value to manage a patient, to put a patient in the minimal symptom expression and then be able to maintain them there. And we will price for the average on that. And that backs us right into our vial price. Great. Thank you.
spk13: The next question comes from Danielle Brill from Raymond James. Please go ahead.
spk19: Hi, guys. Good morning. Thanks so much for the question. I was just wondering if you could maybe comment a little bit more on the PAA program enrollment. I'm curious how it's tracking compared to your internal expectations. And, I mean, if you could share how many patients you've enrolled, that would be great. Thank you.
spk12: Yeah, Danielle, we have not made that information public yet. I can tell you that the PAA, there's been demand coming from the U.S., from Canada, and from Europe already. It's a process that we go through to actually enroll the patients and get them started. So the demand, it meets our expectations. And at this point, we just haven't disclosed the total number of patients that are in the PAA. Thanks.
spk13: The next question comes from Jan Werber from Talon. Please go ahead.
spk15: Great. Thanks for taking my question. I actually have a question about 117 for MNN. Maybe can you, I don't know if you can, share with us how are you thinking about the time to evaluate in the primary endpoint, and are all patients going to have to be anti-GM1, IgM antibody positive to be enrolled? And I assume they need to be second line onwards. Thank you.
spk11: Thank you for being with us today. What concerns MMN? Of course, so far we have been talking mainly about our conviction around the biology of this disease, you know, clearly driven by pathogenic IgM antibodies, which do recruit the classical pathway. We haven't disclosed details yet on the clinical trial design, but we will do so when we're progressing through the R&D day. The trial design is something you can get inspiration for when you look at the IVIG trials, which we can all study in the MMM space. These are the trials which have basically established the clinical and regulatory endpoints. To answer your second question, no. We will probably not use anti-GM1 antibodies presence as an inclusion criterion. We will disclose, of course, in more detail inclusion-exclusion criteria, but we believe there is recent evidence amongst others from our key collaborator at the Utrecht University suggesting that all NMN patients actually have these autoantibodies, and actually the type of these autoantibodies directly correlates with disease severity. Thanks for the question.
spk13: The next question comes from Jason Butler from JMP. Please go ahead.
spk10: Hi, thanks. I had another one on 117. Actually, thanks for taking the question. Just if you're thinking about the potential for a longer duration or durability effect versus IVIG and the fact that this is a slower progressing disease, just how are you thinking about the control arm and and how to optimize around that. Thanks.
spk11: Jason, thank you for being with us today. Thank you for this question. We haven't disclosed trial design yet, and we will do so. You know that we do that I would suggest that you take a look at, for example, the CIDP trial, which we unveiled the trial design to give you a feeling of, you know, how you could work in this type of diseases. We consider NMN to be very similar to CIDP when it comes to thinking through the pitfalls of clinical trial and execution, design and execution. Thank you.
spk13: Great. Thanks. The next question comes from James Gordon from J.P. Morgan. Please go ahead. Hello, James Gordon, JP Morgan.
spk00: Thanks for taking the question. I had a question on a new mechanism. So I saw that Alexia had taken their all-factor D inhibitor into phase two for MG. The question is, how promising do you see that sort of approach for treatment of a disease like MG? And could an all-factor D work even more broadly than MG, like any of the other indications that you're planning on targeting with an FCR inhibitor, or the reason not to be too optimistic there? and also just a clarification just on the electricity formulation. When is the earliest that that might potentially be able to come to market, please?
spk11: Thank you, James. Concerning your question on the involvement of complements in MG, whether you have a C5 blocker or a factor D blocker, What we know about the disease biology is that actually the autoantibody is at the heart of the disease biology. It's exerting multiple pathogenic modes of action at the neuromuscular junction. Complement recruitment is just one of them. It's also a satirical immunoreceptor blockade, cross-linking, and internalization, which are in play. And therefore, we think that an IgG-removing agent like F-cartitumab should play upstream of any complement inhibitor, regardless whether that would be a C5 blocker or a factor D blocker. Concerning your question on Electrify, it's too early to give you a timeline for if and when a product could hit the market. This is a novel technology which we, to a certain extent, are going to pioneer in close collaboration with our partner, Electrify.
spk13: Thank you. The next question comes from Douglas Tarso from HC Wainwright. Please go ahead.
spk08: Hi, good morning. Thanks for taking the questions. Just wanted to revisit the question on pricing. You sort of indicated, you know, sort of price to the average duration. I'm just curious, have you engaged with payers in sort of more of a value-based model, meaning just sort of setting an annual price, regardless of how many treatments they need, but just, you know, sort of controlling somebody's disease, the MG or any other disease in development? Thanks.
spk12: Yeah, so, Doug, we have engaged with payers on exactly that. We've also done a great deal of market research after showing them the data and asking them what they believe the annual value is. Now, I've stated this before. You have a product that's being used in MG right now that's approved. with Solaris that has a retail at up to $700,000 per year. And it's being pretty well covered by payers for relapsed refractory MG. I think that we are going to be able to offer a great value to the payers and to the patients because of the clinical impact that we can have on these patients. And, again, you know, we're looking at FBRT in the big picture. It's bigger than just the launch of MG. You know, we're already in four indications. We'll be in six before the end of the year with aspirations to go up to ten indications. So we need to think about that as we go to price as well. Thanks. Okay.
spk13: The next question comes from Greg Suvaneve from Goldman Sachs. Please go ahead.
spk09: Good morning, good afternoon. Thank you for taking my question. I had a question just on your expected announcement of fifth indication and a sixth indication for F-car TG mod. Could you just remind us if those indications will evaluate both an IV and a sub-Q, or if you're leading with a sub-Q formulation? And then in terms of the indications in themselves, if one or both of them will be overlapping with your existing disease areas of interest or if they will establish a new beachhead. And then secondly, maybe just a quick one on your upcoming R&D day, how much of that R&D day will you spend talking about perhaps your non-Escar-Tigamod, non-117, and non-Cosatuzumab pipeline, whether they be proprietary or partner. Thank you.
spk11: Thank you, Greg. Thank you for being with us today. Concerning your first question, it is our vision to have both the IV and sub-Q products available in each and all of our indications. You will see from the pipeline updates that we have been prioritizing for the more recent indications the sub-Q product execution, simply because it gives us more degrees of freedom during this COVID pandemic. We already disclosed publicly that the fifth indication will fit squarely in Keats' neuromuscular franchise, and we will be talking more about, you know, the sixth indication, of course, soon. The R&D data will mainly focus, I think, on adgratidumox and fusotuzumab, but the agenda will be made public soon, and then maybe we can interact for further questions on the agenda then. Thank you.
spk13: The next question comes from Rosie Turner from Barclays. Please go ahead.
spk16: Good morning, good afternoon. Thank you very much for taking my question. Just one on manufacturing, if I may. So I know Lonza is your key partner there and they've been having some issues manufacturing the Moderna vaccine in terms of delays. And I was just wondering how your discussions with them are progressing and if there's any indication it could be some delays in the beginning of 2022 when you're ready to start launching. Thank you.
spk11: Thank you, Rosie. Maybe Keith, you would like to take this question?
spk12: Sure, happy to. So, Rosie, first of all, I want you to rest assured that we already have commercial supply that is manufactured and ready to go for launch. Additionally, we know that the global pandemic has put additional pressure on the supply chain. Fortunately, we had already ramped up production prior to the pandemic. because you can see how much we're putting into our pipeline and then going into China where there's a huge population. So we wanted to make sure that we had ramped up our supply well in advance, and we did that. And finally, regarding communication with Lonza, We have a very good relationship with Lonza, and I can tell you that we speak to them pretty much on a weekly basis going through all of the logistics. So I feel quite confident in where we are now, and we've taken all the precautions that we can. So unless something unforeseen, we should be in good shape.
spk17: So just one follow-up. So is that both sub-Q and IV supply? Okay.
spk12: Yes. I mean, right now we're focused on the IV supply from a commercial point of view, and the sub-Q supply we're focused on for clinical trial drugs, two separate.
spk17: Okay. Perfect. Thank you very much.
spk13: Thank you. The next question comes from Yatin Sunajay from Guggenheim. Please go ahead.
spk06: Hey, guys, this is Eddie on free out. And thanks for taking the question. So I just wanted to ask how you're thinking about the filing for ITP. Are the two advanced trials each sufficient on their own? Or will you need two separate SPLAs? Or do you need to see success in both and file together? And then are there any safety requirements or gaining factors that differ between those two formulations? Thanks.
spk11: Thank you, Eddie. So the idea is that both the IV and the sub-Q trial in ITP would jointly satisfy the requirements of the FDA for the BLA filing, so the data packages of both trials would feed into one and the same BLA, and that then indeed would meet their requirements in terms of two independent studies, but also the requirement in terms of size of the safety database.
spk13: Thank you. The next question comes from Lanny Van Steen. Hi, sir, from KBC. Please go ahead.
spk04: Good afternoon. Lanny here. Thanks for taking the question. Quick one on 117. We see that the plan is to involve XyLABS actively in aphrotegamote clinical development to expand the breadth of the program. I was wondering if this is a strategy that you would consider as well earlier on in 1.17 development as we enter that phase 2 development at the end of the year, or would you prefer to take a more stepwise approach from seeing first initial proof of concept before then expanding to additional geographies for development? Thank you.
spk11: Thank you, Lenny. Thanks for being with us. So, you're right that the Scobabread partnership is limited to at Cartegemoth. It does not reach into any other pipeline asset of the company. And look, there's a lot of work on the plate of Xylab if they want to help us on up to 10 indications. And you can see how the partnership evolves. This is, to a certain extent, new territory for us. It's an important strategic component to design. And let's see how that goes before we make any further pipeline decisions. Thank you. All right. Thanks.
spk13: The next question comes from Yana Nzou from Wells Fargo. Please go ahead.
spk03: Hi. Thanks for taking my question. Just wondering about the upcoming data readout for the healthy volunteer study of Organics 117. Could you comment on What level of PD biomarkers could translate into clinical meaningfulness in patients in terms of the C2 level reduction, free C2, total C2, as well as the CH50 titers? Just what kind of expectation do you have for the data from the Healthy Volunteer Study? Thanks.
spk11: Thank you for the question about the healthy volunteer studies. So, you know, this is a pretty robust study. We do single-sending dose and multiple-sending dose work for both the IV and the sub-Q product. The sub-Q product is equipped with helozymes-enhanced technology. and you're spot on above and beyond your classical Phase I readouts, which would be safety tolerability and a safe dose for Phase II, there would be meaningful information deducted from biomarkers. Complement is a very interesting system to study from a biomarker point of view, and you're right, we are studying total C2 levels, free C2 levels, and complement activity. And we will be showing you some of these data when we release phase one data around the middle of the year, and how then these data will have informed us about how to dose in phase two, first indication being MMM. So stay tuned, but you can expect data similar to the synemologist-minted data, which we unveiled during the R&D day in New York in 2019. Thank you.
spk07: Got it.
spk11: Thank you.
spk13: The next question comes from Colleen QC from Baird. Please go ahead.
spk20: Hi, good morning. Thanks for taking our questions. So for MG, in your market research, do you have a sense for how many patients might prefer sub-Q versus IV formulation?
spk12: So, Colleen, I can only give you, this is not going to be true market research. It's only going to be in speaking with roughly a cohort of about 50 physicians throughout the U.S. I think that you will see that there will be a larger demand to be on sub-Q, and there's up to 30% of patients that do not want to stick themselves with a sub-Q injection and would want to stay on IV. I've actually also seen a very similar figure to that when some of the analysts have done calls with physicians. So haven't done any broad based official market research, but that's roughly the numbers that we're thinking.
spk13: There are no more questions in the queue. This concludes our question and answer session. I'd like to turn the conference back over to Tim Van Hauwermeyer for any closing remarks.
spk11: Thank you, operator. This company is executing strongly on the business plan. I think we're moving forward on all fronts, as you could see in the Q1 earnings update. We would like to conclude here, and thank you for your participation to the call and the questionnaire today. Thank you.
spk13: The conference is now concluded.
spk01: Goodbye.
spk13: Thank you for attending today's presentation. You may now disconnect.
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