Apellis Pharmaceuticals, Inc.

Good day, and thank you for standing by. Welcome to Appella's fourth quarter and full year 2021 financial results conference call. At this time, all participants are on a listen-only mode. After the speaker's presentation, there'll be a question and answer session. To ask a question during the session, you'll need to press star one on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press star zero. I would now like to hand the conference over to Meredith Kaya, Senior Vice President of Investor Relations and Strategic Finance. Please go ahead.

Good afternoon, and thank you for joining us to discuss Appellus' fourth quarter and year-end 2021 financial results. With me on the call are Co-Founder and Chief Executive Officer, Dr. Cedric Francois, Chief Commercial Officer, Adam Townsend, Chief Medical Officer, Dr. Federico Grossi, and Chief Financial Officer, Tim Sullivan. Before we begin, I'd like to 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 our 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 all for joining us today. 2021 was a remarkable year for Appelis. We received our first MDA approval for MPAveli delivered an exceptional initial launch, reported top-line results from our Derby and Oaks studies positioning us to potentially receive our second FDA approval, and advanced our broader pipeline. Or simply said, we strengthened our position as a global leader in complement. Let me start with Empaveli. In May of last year, Empaveli was approved by the FDA for the treatment of adults with paroxysmal nocturnal hemoglobinuria, or PNH. This approval marked the transition from us being an R&D-focused company with a passion to develop transformative therapies to now also being a commercial stage company delivering the first ever targeted C3 therapy to patients. And Paveri is well-positioned to elevate the standard of care, and I am excited by the progress we are making so far with the launch. Physician feedback is strong, patients are reporting significant improvements, compliance is high, and we are seeing continued positive recognition by payers. Globally, we and our partner Sobi were also thrilled to see additional approvals over these past few months, including in the European Union. In ophthalmology, the phase three results from Derby and Oaks in geographic atrophy, or GA, were a critical step in our efforts to bring the first ever treatment for GA to patients. Since reporting top line results last September, the team has been engaging closely with the retinal community through medical meetings, one-on-one discussions, and many other forums. We received positive FDA feedback last fall and recently completed the pre-NDA meeting, the final step in the run-up to our submission. More than ever, we believe Pexita CoPlan represents a potential breakthrough for the 5 million patients globally who are living with GA, a relentless disease that is a leading cause of blindness worldwide. We also made progress in advancing our broader pipeline, including our late-stage programs with systemic Pexita CoPlan and our three preclinical programs. Let me now spend a moment on our key priorities for 2022. First, as I mentioned, we are focused on bringing texetacoplan to the market as the first-ever therapy for patients with GA. GA can best be described as a forest fire raging through your retina, which is a continuous process with irreversible retinal cell death. Once these cells are gone, you start going blind, and there are currently no approved treatments. We believe that with Bexia Taculplan, we have an opportunity to slow this process and preserve patients' vision for longer. We are actively preparing our NDA and are on track to submit it to the FDA in the second quarter. We will include 18-month safety and efficacy data from Derby and Oaks in our NDA and and plan to share these data publicly in March. In parallel, we are beginning the pre-submission discussions with European regulators. Our initial market research has been quite encouraging, and as we approach a potential approval, we will continue our efforts to educate the physician and patient communities. Secondly, we aim to elevate the standard of care in PNH and further establish Empaveli as a first-line treatment. We want to ensure that all patients with PNH, regardless of their baseline hemoglobin levels, have the potential to benefit from Empaveli. Beyond PNH, we are seeking to advance Empaveli as a transformative therapy for rare, complement-driven diseases. Together with SOBI, we plan to have four late-stage programs underway this year. Collectively, these opportunities could address the needs of as many as 35,000 patients per year, significantly expanding the opportunity for mPaVirine. Third, we want to advance systemic pexetacoplan as a novel approach to enabling adeno-associated viruses, or AAVs, for gene therapies by controlling the many issues that are associated with these therapies. By targeting C3, We believe that we may be able to see benefits such as increasing safety and tolerability, decreasing the dose needed, and allowing for dosing in patients with preexisting antibodies. In collaboration with our research partners, we look forward to sharing preclinical data in the first half of this year. And finally, we are continuing to advance our pipeline and plan to expand our clinical portfolio with the submission of an IND for APL 1030 our first-in-class brain-active C3 inhibitor in the second half of this year. We also are continuing to progress additional programs, including APL2006 and our siRNA plus mPavetti program towards the clinic over the next 18 months. We look forward to reporting on our progress across these four strategic priorities over the course of 2022. By the end of this year, we could have two commercial products, a robust pipeline encompassing multiple late-stage rare disease programs, and additional preclinical programs heading into the clinic, further cementing our position as a global leader in complement. I am amazed by the extraordinary science that has been pioneered by the team here at Apelis. 2021 was an incredible year, and we look forward to building on our momentum in 2022. And let me now turn the call over to Adam for a commercial update. Adam?