argenx SE

Good morning. My name is Rocco, and I will be your conference operator today. At this time, I would like to welcome everyone to the conference call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during that time, simply press star followed by the number one on your telephone keypad. If you would like to withdraw your question, please press the star key followed by the number two. We do ask that you please limit yourself to one question and a single follow-up. You may re-queue if you have further questions. Please also note today's event is being recorded. Thank you. I would now like to introduce Beth DelGiaco, Vice President of Investor Relations. You may begin your conference.

Thank you, Rocco. A press release was issued earlier today with our third quarter 2020 financial results and business updates. This can be found on our website along with the presentation for today's webcast. Before we begin, I'd like to remind you on slide two that forward-looking statements may be presented during this call. These may include statements about our future expectations, clinical development, regulatory timelines, the potential success of our product candidates, financial projections, and upcoming milestones. Actual results may differ materially from those indicated by these statements. Argenix is not under any obligation to update statements regarding the future or to conform those statements in relation to actual results unless required by law. I'm joined on the call today by Tim Van Hauwenmeren, Chief Executive Officer, Keith Woods, Chief Operating Officer, and Erica Staldy, Chief Financial Officer. I will now turn the call over to Tim.

Thank you, Beth, and welcome, everyone. We appreciate you joining the call today. This has been an exceptional year for Argenix. We have achieved several significant milestones, most notably the positive readout of our face-to-death trial and also in an unprecedented environment. While we are still managing the impact of COVID-19 on our lives and business, I'm continually impressed by the team we have built and their ability to definitely maneuver around the challenges which we've been presented with. We are in a very strong position as we close out 2020 and look forward to our first commercial launch next year. We know that FCNN antagonists as a new class of medicines have tremendous potential to transform the treatment of serious autoimmune diseases and we are closer than ever to reaching people living with our first indication, generalized myasthenia gravis. We are excited to discuss the continued progress we have made in advancing our first and potentially best-in-class FC fragment. As we focus on maximizing our leadership position with FGATIGIMOD, we remain equally committed to investing in R&D and enriching our deep pipeline of differentiated early and late-stage assets to generate long-term value. You'll find today's agenda on slide number three. I will share details on the progress we've made with Avocatigemot, specifically the new data we presented from ADAPT, updates on our ITP, PV, and CIDP trials, and our fifth indication. I will then move to the rest of our pipeline, including Cusatuzumab and Regenix 117. It will provide more context on our commercial readiness activities, followed by a financial update from Eric, before we take your questions. Let me begin with Abkhatijamot. In May, we reported that a global phase three ADAPT trial met its primary endpoint, which was a responder analysis in acetylcholine receptor antibody positive patients during the first treatment cycle. 67.7%. of abgadigemot-treated patients compared to 29.7% of placebo patients achieved a greater than two-point improvement on the MG-ADL score for at least four consecutive weeks. Similar results were seen on the QMG score. 84.1% of responders had an onset of response in the first two weeks. Slide five shows that 40% of all patients or about two-thirds of responders achieved an MJADL of zero or one, classified as minimal symptom expression. And a substantial proportion of responders saw impressive durability, with almost 60% of patients experiencing a benefit of eight weeks or longer, and one-third of patients experiencing a benefit of 12 weeks or longer. Additional data from ADAPT were presented earlier this month, at the MGFA Scientific Session by our principal investigator, Dr. James Howard. These data confirmed the rapid and clinically meaningful responses to F-gartigimab that we saw from the top line, but also showed additional analyses on the magnitude and repeatability of response. On slide six, to assess the magnitude of response, We looked at the MG, ADL, and QMG score reductions at week four or one week after the last infusion. As you can look at increasing thresholds of improvement, the treatment arm continues to show benefit while the placebo arm goes to zero. We were particularly pleased to see that 50% or more of patients achieved at least a five-point improvement on the MG-ADL and at least a six-point improvement on the QMG. A third of patients achieved a QMG improvement of at least nine points. These responses and the proportion of patients achieving minimum symptom expression are unprecedented and capture the most important component of this trial. These are not just scores. These are patients who are feeling better. and a substantial number are virtually symptom-free. With regards to repeatability, if you look across cycles one and two, almost 80% of abgartigem of patients were considered MgADL responders. This included 36.8% of patients who were not MgADL responders in cycle one, but were in cycle two. We saw that the MgADL score reduction was consistent in both cycles with a mean change from baseline of 4.6 in cycle one and 5.1 in cycle two. We also showed some additional analyses on the acetylcholine receptor antibody-negative patients. You'll recall we saw a high placebo response in these patients based on the MGA-ADL score by including the more objective measure QMG in the analysis. we started to see more separation from treated and placebo patients. Put simply, the data show that if patients responded to Avgarditumab, they did so quickly and with a significant depth and duration of improvement. This is supported by a safety profile comparable to placebo with no reduction in serum albumin. These data further demonstrate how Avgarditumab is uniquely differentiated and has the potential to be the best-in-class FCRM antagonist. On slide nine, you can see on our pipeline that next steps for Afghan teacher model in GMG are on track and we are pleased to confirm that we plan to file the BLA by the end of this year. In addition, our interaction with the FDA regarding the subcutaneous bridging strategy in MG is set to occur before year end, and we will communicate on the path forward once we have regulatory feedback. We have heard from patients, physicians, and payers that having both an IV and a subcutaneous formulation for Afgafigimab will be a significant competitive advantage in NG and other indications. Our exclusive access to the HeloSign technology for the FCRN targets enables our subcutaneous administration to be a single, fast injection, which we also believe is optimal for patient comfort and convenience. As we talk through our other indications and the specifics of those trials, you can see that we are actively prioritizing sub-Q development to accommodate patient preferences and to adjust to this new normal where patients may not always have easy access to all types of care. So this brings me to the evolving strategy of our ITP program on slide number 10. We discussed the strategy on our last update call to address the enrollment delays we've been facing due to COVID-19. We consolidated the original three registration studies into two. One is the ongoing IV only advanced trial, and the other is the subcutaneous only advanced sub-Q trial, which is on track to begin by the end of this year. This program realignment shows our ability to adapt and be nimble as an organization, and I'm pleased with the direction in which we are headed. Ultimately, we hope this change will expedite the path for FGAT-TGMO in ITP. Moving on to CITP on slide 11. The Phase II adhered trial of subcutaneous FGAT-TGMO is enrolling well, and the go-no-go decision to expand the trial up to 130 patients will occur after the first 30 patients are treated in part A. As stated in today's specialties, we now expect that decision to occur in the first half of 2021 with a specific timing dependent on the impact of COVID-19 delays. Based on initial interactions with the FDA, we believe the expanded trial could be sufficient for registration in CIDB. We will also be evaluating sub-Q F-graftigimab in our Phase III ADDRESS trial in Pemphigus, which is on track to start soon. I'd like to take a moment to describe this trial in more detail. As we did with developing our Phase III trial in MG, we are working closely with patients and physicians to design the ADDRESS trial as well. By paneling with key stakeholders, we can more fully understand the remaining unmet needs for pemphigus patients and identify how FGAT-TGEMOT might best offer a solution to fit into the existing treatment paradigm. Pemphigus is a skin barrier disease, so speed of onset is a crucial aspect of potential therapy. Patients want to see healing of lesions and achievement of disease control and clinical remission as quickly as possible. We also learned that patients prioritized the ability to taper off steroids to manage the side effects. Slide 12, the Phase III Address Trial will be a randomized, double-blinded, placebo-controlled study where the objective is to assess efficacy, safety, and tolerability in up to 150 newly diagnosed or relapsing patients with moderate to severe pathologies. The trial will include both pentacus vulgaris, or PV, and pentacus volatilis, or PF, though the PF population will be kept in a similar manner to the acetylcholine receptor negative population in V and G study. Patients will be randomized to receive either sub-Q F-gatigemot or placebo for 30 weeks. Patients will start on concomitant steroids based on what we determine to be the optimized dosing regimen from the phase two study. The primary endpoint will assess the proportion of patients who achieve complete remission on a minimal steroid dose at 30 weeks. Before I move to the rest of our pipeline, I'd like to briefly mention the fifth indication for abratitumab, which will be discussed in more detail during an investor event in the first half of 2021. So this program is modulate. Everything is on track and we're moving forward. We're just not sharing yet what this indication exactly is at this time. Given the evolving competitive landscape in the FCRM class, we have decided that it would be most prudent to provide full context around this new indication closer to the phase two trial initiation mid next year. Our team is on track that all phase two preparation work and they're working hard to design a possible trial as we've done with all indications to date. While we wait to share details until next year, we can say that we are very excited by the opportunity this indication presents to rare disease patients. It meets the criteria we use for our indication selection strategy. There is a clear biology rationale around the role of autoantibodies and the defined clinical and regulatory path forward. It also fits greatly into our emerging franchise structure with an attractive commercial opportunity across more than one of our potential therapeutic franchises. In this way, it aligns with our broader strategy to leverage our growing commercial capabilities across multiple indications. We continue to have the broadest development program among FCLN antagonists and are committed to roll out new indications at the pace of at least one per year. Going forward, we will take an approach to talk about each indication just before the phase two initiation, so not to share information before it's necessary. Now I want to choose a tissue map, Endergenics 117. On slide 13, We will be sharing top-line data from the Phase II Culminate trial in early 2021 as promised. Based on an early look from Culminate, we have selected 20 mg per kg as the go-forward dose and will be prioritizing a combination approach of Cusapusumab with the emerging standard of care Venetoclax. The Phase I B-Elevate trial, evaluating double and triple combinations of Cusa, Aza, and Ven, continues to enroll following a pause due to COVID-19. Slide 14. We started the Phase I Healthy Volunteer Study of ARGENIX 117, targeting C2. Data from this study are expected mid-2021. The Phase I trial will assess pre-KPD, pre-C2 levels, and bioavailability of both IV and sub-Q formulations. We will also use the phase one study to identify a phase two dose. That's with our approach to agratizumab. We plan to launch multiple phase two proof of concept trials on the use of strong phase one data. We're looking at severe autoimmune diseases and have already selected multifocal motor neuropathy, or MMM, as an initial indication. We also continue to look at potential kidney indications. Finally, as part of our Argenix 2021 vision, we aim to be a global integrated immunology company, prioritizing both our commercial efforts as well as our R&D engine through our immunology innovation program. Argenix 118 is the lead optimization stages as we determine how best to address the groundbreaking biology on the role of Charcoal-Leyden crystals in severe airway inflammation out of the lab of Bartlett and Ray. We are also on track to finalize ARGENIX 119 this year and will likely be communicating more on this pipeline candidate early next year. As we've said many times about our IIP, this is a program about co-creation which is at the heart of everything we do at ARGENIX. We recognize that partnering with leading disease biologists will allow us to unlock in our key commercial franchises and bring forward the most innovative therapies to patients. Continued investment in our antibody engineering capabilities is a key component of our role in IIP relationships. We recently announced two new technology partnerships with Shugai and the Clayton Foundation, underscoring our commitment to the IIP by enhancing our capabilities to build the best antibodies possible and broaden the range of targets that we may address. We also announced that we will be expanding the scope of our collaboration with Herozyme for access to the enhanced drug delivery technology. Under the expansion, we gained three additional exclusive targets upon nomination, which brings the total to six potential targets. We have already exercised access for two targets, FCRM and C2. We believe that having access to the enhanced technology for current and future product candidates will allow us to reach more patients with our therapeutic antibodies. With that, I'd like to turn the call over to Keith to discuss our commercial preparations in more detail. Keith?