Apellis Pharmaceuticals, Inc.

Good morning, ladies and gentlemen. Thank you for standing by, and welcome to the Appellus Pharmaceuticals' fourth quarter 2023 earnings conference call. At this time, all participants are on a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you'll need to press star-1-1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star-1-1 again. Please be advised today's conference is being recorded. I will now hand the conference over to your speaker host, Meredith Kyer, Senior Vice President, Institute of Relations and Strategic Finance. Please go ahead.

Good morning, and thank you for joining us to discuss Appellus' fourth quarter and full year 2023 financial results. With me on the call are co-founder and chief executive officer, Dr. Cedric Francois, chief operating officer, Adam Townsend, chief medical officer, Dr. Caroline Baumel, and chief financial officer, Tim Sullivan. Before we begin, let me point out that we will be making forward-looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and actual results may differ materially. I encourage you to consult the risk factors discussed in our SEC filings for additional detail. Now I'll turn the call over to Cedric.

Thank you, Meredith, and thank you all for joining us this morning. As I reflect on this past year, I am extremely proud of our team and all of our achievements in 2023. Our two commercial products, CyphoRe and Mpavirin, are making meaningful differences for patients. The year ended with SIFO-3 in a very strong position, and we remain encouraged by the continued uptake and high compliance rates for EmpaDeli and PNH. We also had multiple positive data readouts, including up to three years of efficacy and safety data from both of our approved therapies. While 2023 certainly had its challenges, our accomplishments and our resilience position as well for continued execution in 2024 and to deliver on our mission for patients now and in the future. Let me share some details on this past year and our priorities for 2024. Beginning with Cyphovre. We recently celebrated Cyphovre's one-year approval anniversary, and what a year it has been. The launch had exceeded our expectations, even with the unexpected challenges we faced last summer. More than 200,000 cytophery doses have now been distributed to physician practices since launch through mid-February, and we estimate that approximately 215,000 cytophery injections have been administered to patients through mid-February, including our Phase III clinical trials. These numbers underscore the retina community's confidence in the benefits of cytophery and the central role it plays in improving patients' lives. And importantly, based on these numbers, the risk of developing retinal vasculitis remains rare at an estimated rate of approximately 0.01% per injection. In the fourth quarter, we reported $114 million in SIFO-RE US net product revenue. We achieved $275 million in SIFO-RE revenues for the full year. While we have experienced modest seasonality in the first quarter so far, Cyphovre continues to show meaningful growth, and we look forward to bringing it to more and more patients throughout the year. In 2024, our priorities with Cyphovre are to maximize access to patients in need in the U.S. and to bring Cyphovre to patients with GA worldwide. Key to these are our commercial and medical activities. including building on the relationships we've already established in the retina community. Given our global expansion goal with Cyphovri, let me comment on the European Union. As you know, we received a negative opinion by the Committee of Medicinal Products for Human Use, or CHMP, for the marketing application of Cyphovri in the EU. While this is disappointing, we remain steadfast in our efforts given the more than two and a half million GA patients in need of treatment across Europe. The feedback and support we have received for stag forgery from the physician and patient communities in Europe has been extraordinary. We have been informed of several letters sent to EMEA from multiple European retinal organizations, highlighting the need for new treatments and how important stag forgery would be for patients. As such, we are initiating a reexamination of our application, which includes revising our dossier to ensure that key questions are addressed. We expect a final opinion in connection with this reexamination to be issued at an upcoming CHMP meeting in the second quarter. If positive, a decision by the European Commission is anticipated in the third quarter. To be clear, and as we've said many times before, this is not going to be easy. It will be an uphill battle, and we do not know the likelihood of success at this stage. But we strongly believe in our data, including analyses that many experts agree demonstrate a functional benefit with cycle brain treatment, and we will continue to work closely with the CHMP and the retina community throughout the process. Turning to M5A, we generated $24 million in fourth quarter U.S. net product revenue and $91 million for the full The Epavedi injector, an innovative and first-of-its-kind high-tech volume injector, was approved and launched last fall. We've been very pleased with the feedback and adoption so far. And in 2024, we will remain focused on maximizing our market position in P&H and also look forward to the Phase 3 data readout for systemic Mexita core plan in C3G and ICMPGN. two rare and debilitating kidney diseases. Our phase three value study is fully enrolled, and we expect top-line data from this study mid-year. Over the past couple of years, we've been very focused on our commercial products and new launches, but we have other exciting earlier stage programs advancing. Let me shift to our fourth priority for 2024, which is to progress our early RNB pipeline. with an eye to the long-term. In addition to the Abavelli initiatives in C3G and ICMPGN, we are focused on our siRNA program, APL3007, which is now in a Phase I dose escalation study, with data expected later this year. Additionally, we are advancing the gene editing program with BEAM, on which we also expect to share more this year. We are entering 2024 from a position of strength with an unwavering commitment to address unmet needs for patients and to create even greater value for our shareholders. And with that, I will now turn it over to Adam to discuss our commercial and medical affairs activity.