argenx SE

Good morning. My name is Andrew and I will be your conference operator today. Welcome to the Argenix full year and fourth quarter 2020 financial results conference call. At this time, I would like to welcome everyone to the call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you'd like to ask a question during this time, simply press star followed by the number one on your telephone keypad. If you'd like to withdraw your question, press star then two. If you require operator assistance, please press star then zero. Please note this conference is being recorded. Thank you. I'd like to introduce Beth DelGiaco, Vice President of Corporate Communications and Investor Relations. You may begin your conference.

Thank you. A press release was issued earlier today with our full year 2020 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. Argenix is not under any obligation to update statements regarding the future or to conform those statements in relation to actual results unless required by law. I'm joined on the call today by Tim Van Hauermeren, Chief Executive Officer, Eric Castaldi, Chief Financial Officer, and Keith Woods, Chief Operating Officer. I will now turn the call over to Tim.

Thank you, Beth, and good morning to everyone on the call. We appreciate your joining. I'm proud of the exceptional progress we made in 2020, despite many unexpected global challenges. We believe we are closer than ever to reaching patients and strongly positioned to create long-term, sustainable value for our shareholders. I'd like to briefly highlight some recent milestones and then provide updates on our deep and differentiated pipeline. First, as we announced earlier this week, we are pleased that our BLA was accepted for review by the FDA. This decision is an achievement for us as a company, but more importantly, we are thinking about what this means for our patients. We see the very real unmet needs of people living with Mg. We hear about it from patients directly and from their supporters, and we are hopeful that FGT may provide a new treatment option for this community. The acceptance of our BLA serves as an obligating event on our path to our first commercial launch, and we remain sharply focused on execution at every level of the organization. Between now and the potential approval of FGF Digimod, we will be working in collaboration with the FDA to answer their questions and coordinate site inspections. I can tell you that we are planning to be ready well ahead of our December PDUFA date because we know that patients are waiting. Still, these are exceptional times with much uncertainty ahead. We are proactively planning for a launch in various scenarios, fully virtual for one or, now that we have more time for the COVID vaccine program to be implemented, only partially virtual. We continue to make progress in engaging with a large proportion of our target stakeholders in a digital environment, which Keith will discuss later in the call. With that said, we would welcome the opportunity for our field force to hold meetings in person. Regardless of scenario, we plan to use every day we have until launch to further implement these important campaigns. The US remains our priority for our first launch and our largest market, but we are also well underway with preparations in Japan, where we expect to file our marketing application in the first half of 2021. This sets us up for a potential additional approval shortly after the US. In the European Union, we are seeking scientific advice in the first half of this year, with plans to file our marketing authorization in the second half. Our European General Manager is very experienced in market access and is working on a strategic reimbursement plan in the highest priority countries. Secondly, we recently announced a goal decision to continue enrollment in the Registrational Adhere Trial, evaluating sub-Q FGATIGIMOPS in CIDP. With this update, Edgar Tijemot now has four out of four successful proof-of-concept trials under his belt. This further emphasizes the breadth and potential before us with Edgar Tijemot and validates our careful indication selection process and innovative trial design. we will continue to leverage the same comprehensive strategy with future indications, which we believe will strengthen our leadership in the FCRN class overall. Third, Argenix has a history of raising capital on the back of strong data, and the recent financing was no different. The $1.1 billion in gross proceeds strongly position us for the launch, and will facilitate the expansion of our diversified pipeline and the growth of our team. On that note, I would like to take this opportunity to thank and recognize the outstanding Argenix team. We have grown to over 450 colleagues over the past year and plan to increase to approximately 750 in 2021 across our four offices. In particular, I would like to thank our Chief Financial Officer, Eric Castaldi, who has been with Argenix since 2014. As stated in the press release from this morning, we have started a planned transition process to recruit a U.S.-based successor for Eric. This is part of our evolution to a commercial-stage company and will all happen in close coordination with Eric, our executive leadership team, and the Argenix board. We're very grateful for Eric's long tenure with the company and for its invaluable contributions. Moving on to the pipeline updates, starting with FGAT Digimod. We continue to believe that we have a first-in-class and potentially best-in-class FCRN antagonist. The structure and mechanism of action of FGAT Digimod are depicted on slide number four, and you can see that FGAT Digimod is unique due to our close collaboration with Dr. Sally Ward. Levgatigemot is the only IgG1 smart fragment which binds to FCRN in a way that mimics the natural ligands and preserves the pH-dependent binding of endogenous IgG to FCRN. In our studies, we have not observed an effect on other serum proteins like IgM, IgA, or a reduction of human serum albumin. To date, over 350 subjects have been dosed with Argatigemab, and we continue to observe a favorable tolerability profile, which has been observed to be consistent across indications. On slide number six, we also see a consistent PD effect across trials that has translated into promising clinical benefits. The phase three ADAPT trial demonstrated a response rate of almost 80% across two treatment cycles, a fast onset of action, depth of response, and the potential for individualized dosing. The consistent PD effect also enabled our ongoing sub-Q bridging strategy for MG as seen on slide number seven. The study is a registrational non-inferiority trial which compares the pharmacodynamic effect of 1,000 mg SubQ-Afgatidumab with 10 mg per kilogram IV-Afgatidumab and is expected to enroll approximately 50 patients. The primary endpoint will be taken at day 29, and thereafter we will continue to build a safety database to support the filing of the SubQ-BLA. Slide 8. CIDP is the second Edgar Teacher model indication within our neuromuscular franchise and the fourth established proof-of-concept indication. I covered the goal decision earlier in the call, so I will just mention a few key points. We believe we designed a very innovative trial with ADHIEV using screening criteria to identify patients with an active, confirmed diagnosis of CIDP. We also included a planned interim analysis. Before committing valuable time and resources to running a registration program, we wanted to be confident of the role of the autoantibody in the disease pathophysiology. We set the bar high using precedent trials to define our thresholds, and we matched the criteria. We can now move forward with confidence as we open new trial sites for enrollment in this registration trial. CIDP is an indication that there has been little innovation, and we are advancing the program as quickly as possible. Next, we are enrolling patients in our ITP trials, as well as our PV trial, which can be seen on slides 9 and 10. For ITP, We have brought forward our SUBQ strategy based on feedback from patients and physicians, and are running two concurrent advanced trials, one with IV and one with SUBQ. We expect these trials will support registration of both formulations. In PEMFIGUS, we are evaluating SUBQ-F-Gatigemot in the addressed registrational trial, and as part of this are specifically evaluating the ability to taper patients of steroids we listened to patients describe the ongoing burden of current treatments and incorporated this evaluation into our trial design as you can see we take a forward-thinking approach when designing our trials based on feedback from physicians and patients we will continue to infuse their feedback into future trial designs so that we're optimally positioning F-Gatigamot within current treatment paradigms. This is true for our fifth and sixth indications, which we will disclose closer to the start of clinical trials. Turning to slide 11. We believe that there is a broad landscape of IgG-mediated severe autoimmune diseases for which F-Gatigamot may provide clinical benefit to patients. We will continue to roll out indications ourselves and will also expand and accelerate the pace of F-Cartigemot development globally through our strategic agreement with Xylabs. Our MG launch in the US is just the beginning. Beyond F-Cartigemot, we look forward to sharing the first set of clinical data from Argenix 117, our second pipeline candidate with broad potential in severe autoimmunity. We remain on track to provide data mid-year from a phase one healthy volunteer study from both IV and sub-Q formulations of Argenix 117. We expect to provide information on safety and tolerability, PK and PD effects, bioavailability, and the selected phase two dose and dosing regimen based on three C2 levels. Slide 12. The first indication we have highlighted for ARGENIX 117 is Multifocal Motor Neuropathy, or MMN, which fits squarely into our neuromuscular franchise. You can see our neuromuscular focus taking shape with NG, CIDP, our Fisk-Abgaard-Dijamab indication, ARGENIX 117, and ARGENIX 119. We know that the investments we are making now in both neuromuscular and other franchises will benefit us over time as the franchises grow. Slide 13. Quickly on Cusatuzumab, for which we have a strategic collaboration with Janssen. We have shifted our priority for this program to the Elevate trial, evaluating a triple combination of Cusatuzumab, venetoclax, and azacitidine. This was based on one In trim phase two data from the colony trial and two, the shifting treatment landscape for newly diagnosed elderly AML patients where venetoclax has emerged as standard of care. In evaluating elevate data, we will look at overall response rate, durability of response, and safety and tolerability to consider where quesotuzumab could address unmet needs in this indication. With Elevate and the future of the program overall, we will continue to make data-based decisions on the path forward for cures of Tuzumab, as we do with all our clinical trials. Slide 14. We will not spend time on our broader pipeline today, but we are committed to providing updates as necessary. Investing in our Immunology Innovation Program continues to be a core part of our pipeline strategy. As we look towards commercialization, we seek to create optionality within our pipeline, whether through wholly owned candidates, founded programs, or asset-centric spin-off companies. We believe this is an important part of our growth story as we transition into an integrated immunology organization. I will now turn the call over to Eric for our financial results.