argenx SE

Good morning. My name is Rob, and I will be your conference operator today. I would like to welcome everyone to the call. At this time, 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 this time, simply press star followed by the number one on your telephone keypad. If you would like to withdraw your question, again press star one. Thank you. I'd like to introduce Beth DelGiaco, Vice President, Global Head of Corporate Communications and Investor Relations. You may now begin your conference.

Thank you, Operator. A press release was issued earlier today with our third quarter 2023 financial results and 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 these statements in relation to actual results unless required by law. I'm joined on the call today by Tim van Harmeren, Chief Executive Officer, Carl Gubitz, Chief Financial Officer, and Karen Massey, Chief Operating Officer. I will now turn the call over to Tim.

Thank you, Beth, and welcome, everyone. Slide three. It is incredible to see what the Argenix team has accomplished this year, meticulously delivering on the ambitious plan they laid out in January. We are reaching more and more GMG patients around the world and brought a subcutaneous product to the market only 18 months after the IV launch. We reported stellar CIDP data and now have a path forward to approval next year. We advanced our pipeline with an important goal decision in MMN with Ambassi Brevard and are also keeping our promise to invest in the next generation of exciting preclinical assets which we will talk about next year. Stepping back, we are working on 13 indications for BibGuard alone with more slated to begin before 2025. We have accumulated extensive clinical trial and real world experience with our first in class F7 inhibitor and continue to publish and present on its differentiated efficacy and safety profile. All of this is furthering our leadership in the space and broadening our understanding of the potential of safeguard to change how we view autoimmunity. I would first like to share key highlights from the quarter and then move on to milestones ahead for the remainder of the year. The strength of our launch continues with double digit growth quarter over quarter. Karen will share additional details later in the call. but at a high level, I'm really pleased with where we are today. We continue to hear countless patient stories and testimonials about VivGuard, and this is motivating our teams to drive further growth. We focus our VivGuard expansion goals across three key areas, expanding within the GMG treatment paradigm, expanding geographically, and expanding into new indications. First on expanding within GMG, we are already seeing traction with new patients and new prescribers three months into the Hytrulo launch. We are the only GMG treatment that is available as an IV and a simple sub-Q injection, both offering a consistently strong clinical benefit. This choice in how and where patients want to be treated will help us move into earlier lines. Looking to our geographic expansion, we received VivGuard approval in Canada, have our first patients in Italy and China, and moved another step closer to bringing Hytrulo to patients in Europe with a positive CHMP opinion, including self-administration on the label. In addition to our commercial success, we achieved a key win for the CIDP community over the quarter and are planning ahead for indication expansion. We had a positive meeting with the FDA and can confirm we are on track to file our SBLA before the end of the year. As you will recall, the top-line results from our adhered trial were nothing short of game-changing. This was the largest global clinical trial for CIDP to date and we not only confirmed CRDP as an IgG-mediated disease, but also set a new standard for CRDP trials in the future. Efficacy was apparent across patient subtypes, and we saw a phenomenal 99% rollover into the open-label extension study. Given the high unmet need for a safe and effective treatment alternative, we feel a strong sense of urgency to bring our therapy to CIDP patients as quickly as possible. Therefore, I'm also pleased to tell you that we have notified the FDA of our intention to use our PRV with the CIDP submission. Slide four. Today, I'm speaking to you from ANEM in Phoenix, Arizona, where the Argenix team is presenting a significant amount of data on VidGuard. we have now dosed more than 1,400 patients, generating more than 1,000 patient years of data across all clinical trials. And in MG, we now have almost two years of real-world experience in approximately 6,000 patients. This has provided us confidence in the consistency of the data and a deep understanding of the clinical profile of our FC fragments. And why? the way in which it binds to FCRN, can lead to differentiated results. At the conference, we are presenting aggregated data from ADAPT, ADAPT SubQ, and the associated open-label extension studies, which extend out beyond three years and 19 treatment cycles. We see that responses are repeatable cycle over cycle, and that clinical benefit improvements are of a consistent magnitude. We also see consistent results on minimal symptom expression. In every data set, we are able to achieve approximately 40% MSC, which is an important part of our value proposition to patients. We also show in our data that patients who are able to achieve MSC have quality of life measurements comparable to healthy populations. which is why we believe this metric should be the goal that physicians seek to attain with their energy patients. 50 continues to be a key difference shift, and we show that across all indications and all dosing schedules, treatment emergent adverse events are mild to moderate and do not increase with longer exposure. And we do not see reduction in albumin or increase in cholesterol. The unique clinical profile that we confirm with this data can be linked to the unique design of F-cortisumab, which of course was born out of Dr. Sally Schwartz's groundbreaking immunology research on FCRN biology. Since that time, we have generated a new understanding of the role of FCRN beyond a regulator of IgG levels in circulation. We know that FCRN is important in the trafficking of antibodies into tissues, and that by binding FCRN, our fragment can reach the tissues and site of disease very fast. It is also involved in the autoantigen presentation process, which may explain the data we saw in Tempfigus in reducing autoreactive B cells. Lastly, given the natural way in which we bind FCRN, we can uniquely modulate the target, blocking IgGs from binding, but not derailing FCRN itself into the lysosome for degradation. This has allowed us to select doses and dosing regimens that optimize the clinical benefit of Elgatigemot without having to manage for dose-related adverse events. Our leadership in FCRN presents itself through our business. From the efforts of our commercial and medical affairs teams, to the ongoing translational work of our scientists, who are rewriting the textbooks of immunology. Slide five. Looking ahead, we are excited to advance our pipeline with two upcoming phase three readouts. The first readout will be the top line results from our ITP advanced sub-Q study, followed by our tantricus address study around year end. Let's begin with a high level overview of Hythrulo in ITP. Our goal for the sub-2 study is to replicate the phase 3 IV results, which were recently published in the Lancet. The study has the same design and endpoints, although we increased enrollment to give ourselves more room for success with a highly refractory patient population. The primary endpoint is challenging, so in terms of the threshold for success, we will be focused on the delta between treated and placebo. This endpoint is designed to meet the regulatory requirements, but we also will be looking at the fast onset of action, the IWG score, and safety. The IWG score is what we view as the most clinically meaningful endpoint, as it is based on how clinicians make treatment decisions in the real world. ITP patients are often very fit, but suffer from fatigue and anxiety due to the risk associated with an unexpected bleed. They typically cycle through multiple treatments in the trial and error approach, including through multiple TPO's. What we have learned in our conversations with the ITP medical community is that there is a real need for a new modality to treat ITP, particularly new modalities that come with a favorable safety profile, which is potentially where VivGuard could step in. Slide six. Next, we can expect to see data from the address study in Penficus around year end, meaning the data readout will fall right before or after the year end as we navigate the data analysis and communication around the holiday period. The phase three trial for Penficus was built on the adaptive phase two results where we saw a fast onset of action with 90% of patients achieving disease control after just one to two infusions, and a quick time to CR on a low dose of steroids. In the 50 study, we implemented an official steroid-taking protocol, and this is integrated in our primary endpoint. Similar to ITP, the primary endpoint is a challenging one. defined as the proportion of patients achieving complete remission on a minimum dose of steroids within 30 weeks. It combines reaching complete clinical remission, taping 2, and maintaining a low dose of steroids and sustaining this for 8 weeks. The current standard of care, including steroids and rituximab, leaves ample of room for a fast, new, durable treatment with few side effects. This will be especially important in a post-COVID setting, now that we have a better understanding of the detriments of long-term immune suppression. Slide 7. In 2024, we have multiple catalysts from our pipeline to look forward to. I'm particularly excited for the MMN top-line results to be shared next year. This is the first indication for our second pipeline in a product, EMPA, and is a very serious disease which fits perfectly within the infrastructure we are building for GMG and CIDP. We also have two upcoming go-no-go decisions, first in bolus panfugoides around the end of this year, and also in myositis, which is expected in the second half of 2024. Phase II POTS results are expected in the first quarter of 2024 and has the potential to be a sizable opportunity. This is a study where we will learn a lot about the role of IgGs in this growing indication. And Sjogren's results are expected to be shared in the first half of 2024. This is an exciting opportunity within rheumatology where we believe strongly in the role of IgGs as a disease driver. Lastly, we are focused on long-term sustainable innovation and know to achieve this, we need to invest in the growth of our early stage pipeline. We have several exciting programs through our IIP, which we will communicate as we get closer to INDs, but we are working from a strong track record of success. Every program in our pipeline, including those that we are developing ourselves, and those that are in the hands of others have all been co-created with a top-notch academic collaborator and are grounded in a breakthrough immunology innovation. The new programs will also have this characteristic feature of assets from our IIT.