argenx SE

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.

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.

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.