argenx SE

Good morning. My name is Rob and I will be your conference operator today. At this time, I'd 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 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 the star one. Thank you. I'd like to introduce Beth DelGiaco, Vice President of Corporate Communications and Investor Relations. You may now begin your conference.

Thank you, Operator. A press release was issued earlier today with our full year 2022 financial results and the recent 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 development, regulatory timeline, 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, Carl Gubitz, Chief Financial Officer, and Keith Woods, Chief Operating Officer. I will now turn the call over to Tim.

Thank you, Beth, and welcome, everyone. Next slide. Reflecting on 2022, it was a year of many achievements for Argenix as we evolved from an R&D organization into a fully integrated R&D and commercial organization. We launched our first-in-class FCRN blocker, RISCARD, in the U.S., Japan, and Germany and exceeded our own expectations, generating $401 billion in global net product revenue. I'm very proud of the team for the strategy they built and executed, solidifying our reputation with our key stakeholders. With this significant momentum, we start 2023 in a position of strength. We also have our marching orders in hand for multidimensional expansion to reach more patients with DivGuard through anticipated regulatory approvals and launches in new regions and by driving usage earlier in the GMG treatment paradigm. Next slide. This year, we also generated a significant amount of data on our first-in-class SCNN blocker through clinical trial readout, translation research, and peer-reviewed publications. We reported phase III data from our sub-Q bridging study in GMG, which demonstrated non-inferiority to the IV, based on IgG lowering, as well as consistency across secondary efficacy endpoints and safety. We have now filed for approval with the FDA, EMA, and PMDA, and have a PDUFA target action date in the US of June 2023. we received a communication from the FDA in January notifying us of a three-month clock extension. Since that time, the FDA's review continues, and we are engaging regularly with the agency, being responsive to inquiries as we advance through some anticipated approval. We reported phase III ITT data from our first registration trial last May, and presented these data during a plenary session at ASH in December, which triggered significant interest from the hematology community. We are preparing to submit an MAA in Japan in the middle of this year for approval based on the first ADVANCE trial and will await the data readout of ADVANCE SubQ in the second half of this year to support filing in the US and Europe. Across all of our FGATIGAMA studies, we also generated significant data in 22 to broaden the scope of our safety database, which now includes more than 3,000 commercial patients globally, more than 1,300 clinical subjects, up to 19 cycles of intermittent cyclic dosing, and more than two years of weekly chronic dosing, all with a cumulative exposure of more than 1,000 patient years. Lastly, before we look ahead to 2023, I want to call out a strong sign that continues to serve as the cornerstone of Argenix, solidifying our leadership in SCRN as both the first in class and likely best in class therapy. Our teams are committed to building a deep repertoire of preclinical and translational data, which can serve as an important basis for indication selection or to underscore the clinical or commercial data we are generating. We had several key publications in 22, including on, one, the long-term remissions we observed in pemphigus and the underlying reduction of autoreactive B cells. Two, the differentiation of our FC fragment and its intercellular fate, keeping FCN in its recycling path without interfering with albumin homeostasis. Three, review of the humoral immune response to vaccines during Avogadro treatment. And four, multiple publications and presentations on the health economic outcomes front, highlighting the value that DivGuard can bring to GMG patients from a quality of life perspective.