argenx SE

Good morning, everyone. My name is Jamie, and I will be your conference operator today. At this time, I would like to welcome everyone to the conference. All lines have been placed on mute to prevent 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 keypads. If you would like to withdraw your question, you may press star and then two. Thank you. At this time, I'd like to introduce Beth DelGiacco, Vice President of Corporate Communications and Investor Relations. Ma'am, you may begin.

Thank you, Operator. A press release was issued earlier today with our first half 2021 financial results and second quarter business update. This can be found on our website along with the presentation for today's webcast. I also encourage you all to visit our R&D Day microsite, also on the investor page of our website, to watch a replay of the presentations and engage with the additional resources we've provided as part of the event. 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 Harenmeren, Chief Executive Officer, Carl Gubitz, 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. to discuss our half-year results and second quarter business update. We're going to keep the prepared remarks brief, because we provided a substantial update during our R&D day last week. The first half of 2021 has been marked by several achievements across our immunology pipeline, notably the progress we've made in advancing our first-in-class abstinence antagonists, abgartigamot, in six indications. We are on track to reach generalized myasthenia gravis patients later this year and have been busy with hiring activities and key stakeholder engagements in anticipation of our December 17th PDUFA date. Slide three. We have registrational trials ongoing in four severe autoimmune indications, MG, ITP, CIDP, and panfugus, and we announced two new F-gardigimod indications last week, myositis and bullous panthigoid. We are preparing to launch trials this year in both of the new indications, pending FDA interactions. With regard to our ongoing registration trials, we have said that this year would be one of execution, and we would provide clarity on enrollment as able. We are delivering on that commitment today. We expect to complete enrollment this year in the ongoing ADAPT-SubQ trial in GNG and the advanced IV trial in ITP. This sets us up to have top-line data for both trials in the first half of 2022. Recall that with ADAPT-SubQ, the primary endpoint is based on PD effect and is taken at day 29. The trial continues out to 12 weeks before patients roll over into an open-label extension study. The advanced IV trial follows patients out to 24 weeks before they can roll over into an open-label extension. The other registration trials at Karthikemat, ADVANCE-Q, ADHERE, and ADDRESS, are all making progress. We additionally expect our partner Xilab Limited to start enrolling patients into these global trials by the end of this year. We continue to believe that CARTICOMOD is well positioned to be not only first in class, but also the leader in the FCRM space, with its unique structure as an FC fragment, with optionality, with both intravenous and subcutaneous formulations in development, with clear clinical proof-of-concept established in four out of four indications, and perhaps most importantly, a growing safety database which supports a favorable benefit-to-risk ratio. On site four, you can see that specifically over 600 patients and healthy volunteers have been dosed with F-card ticumab. This includes 125 patients who have been on abgartigamot for over 12 months and 100 patients for over 18 months. Across all studies, we have seen no evidence of dose-limiting toxicities and no reduction in human serum albumin, a topic on which we provided new perspectives during the R&D day. We believe the broad therapeutic window that we have observed allows us to dose F-Carticumab to the maximum benefit of patients and enables our indication selection strategy, which will continue to be our guide as we expand the F-Carticumab program further. Slide five. The list of IgG-mediated diseases which F-Carticumab can potentially address is vast. And as we expand our development program, we will stick to our strategy, which has been successful to date. starting first with a clear biology rationale. Our fifth indication, myositis, and our sixth indication, bullous panthigoids, both aligned to this strategy. With myositis on slide six, we are building an innovative trial design, exploring three subsets of myositis in which the unifying feature is muscle weakness. In the best characterized subset, immune-mediated necrotizing myopathy, we clearly understand the role of the autoantibody in driving the muscle weakness. In antisensitive syndrome and dermatomyositis, patients present with a similar muscle weakness, but the role of the autoantibody is not as well characterized in driving it. The proposed myositis trial will be the first time the company is pursuing a basket trial design based on a unifying biology. In this way, we aim to maximize the potential opportunity to reach a broader population of myositis patients while also minimizing the potential risk of interrogating biology while doing so. With bullous panthrigoid on slide 7, the role of the autoantibody is very clear in the disease pathogenesis and based on the robust efficacy and favorable safety we have observed in the PENFIGUS Phase II trial, we believe we can advance directly to a registrational trial within Bullis-PENFIGOID. Having announced our next FGAR ticker mod indications, we can start to look ahead at how we ramp up to more indications as quickly as possible, whether through our own registration programs, proof-of-concept trials in the hands of a partner such as XyLab or through externally sponsored research. Slide eight. During our R&D day last week, we introduced our Argenix 2025 vision, outlining specific goals as we transition to a global integrated immunology company. First, we aspire to make Advocatiq a model available for patients globally. and to expand the commercial and development footprint of abcarticumab into 15 indications across our expanding therapeutic franchises in neuromuscular diseases, hematology, and dermatology. Second, we seek to advance our second immunology pipeline candidate, Argenix 117, into multiple late-stage trials. The Phase 1 data we showed last week indicates The Dargenix 117 has the potential to be an efficient C2 blocker that can durably knock down free C2 levels by 99%. The safety data, while still blinded, show primarily grade 1 events and no increased risk of infection. The Phase II trial of ARGENIX117 in multifocal motor neuropathy is on track to start this year in which we evaluate the potential for an attractive, infrequent dosing based on the observed PKPD profile. Third, as we shift to be a commercial organization, With a deep development pipeline, we are not taking our foot off the gas when it comes to our investment in discovery and earlier stage programs. Slide nine. This is exemplified by the breadth of assets which have emerged from our immunology innovation program. We talked for the first time last week about Argenix 119, a simple antibody that aims to boost the neuromuscular junction across a range of indications. This is an older pipeline and a product focused on oral indications that fit squarely into our therapeutic franchise. With Edgar Tigamot, Argenix 117, and now with GenX 119, we have a company within a company emerging from our neuromuscular franchise. This is why we like the franchise model so much. The strategic investments we are making now with our current pipeline will continue to benefit us in the long term as we recognize economies of scale and synergies across multiple assets and multiple indications. I would now like to turn the call over to Carl Gubitz, who is joining us for the first time as our newly appointed Chief Financial Officer.