Apellis Pharmaceuticals, Inc.

Hello, and thank you for standing by. Welcome to the Q3 2022 Appellus Pharmaceuticals Earnings Conference Call. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, please press star 11 on your telephone, and you will hear an automated message advising your hand is raised. Please keep in mind that today's conference is being recorded. I would now like to hand the conference over to your speaker, Meredith Kaya, Senior Vice President of Investor Relations and Strategic Finance. Please go ahead.

Good afternoon, and thank you for joining us to discuss the Palace's third quarter 2022 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 details. Now, I'll turn the call over to Cedric.

Thank you, Meredith, and thank you all for joining us today. Let me start this call with what is on all of our minds right now, which is our decision to submit the 24-month efficacy data from the Phase III Derby and Oaks studies to the FDA. This submission will be considered a major amendment to the NDA, extending our review timeline by three months, with a new PDUFA expected in February 2023. Let me just state up front, since we have received so many questions about this, that this decision was ours. This was not requested by the FDA. The reason why we decided to do this is because including these efficacy data gives us the opportunity to have the best product profile at launch with minimal impact to our launch timing. Recall that they already had the 24-month safety data as part of the 120-day update. The inclusion of these additional data in the label would allow us to better educate physicians and patients with a clear message around the robust treatment effect of dexetacolpan over the full two-year period. Remember, at 24 months, both every other month and monthly treatment with dexetacolpan resulted in meaningful slowing of GA progression with increasing effects over time. Between months 18 and 24, 24% and 30% respectively, with every other month and monthly treatment. Importantly, at month 24, the data were remarkably consistent between Derby and Oaks, meaning that we can spend more time discussing the efficacy of the two studies rather than the differences between them. And we continued to see a favorable safety profile in over 1,200 patients with nearly 12,000 injections. Now let me talk about why we did this now, which we recognize appears unusual given how close we were to our original BDUFA date. At the time of our 24-month top-line readout in August, given how strong the data were, we had the discussion internally as to whether we would file a major amendment. We decided against it because we believed that it would delay the timing of our launch and the timing of our permanent J-code by three months. We also did not expect the 24-month data package to be completed before the B2FA without compromising the European filing deadlines. However, as we continued to prepare for the launch, some of these factors changed. The completion of our 24-month data package happened faster than we expected, putting us in a position to submit the data prior to our B2FA without delaying our European filing. Also, given the December holidays, changes to medical coverage at the start of the year, and finalizing the supply logistics, we had already made the decision to launch in January, which also meant that we would receive our J-code in October. The combination of these factors meant that the impact of the extended review period would only be a six weeks delay to commercial launch, with no impact on J-code timing and the best possible label. The FDA is aligned with our decision to submit these data. We understand that this came as a surprise, and we did not take this decision lightly. There is a significant unmet need in this disease, and patients are waiting for a treatment. But we also strongly believe that this is the right decision. It is the fastest way to get our 24-month data into the label and to deliver the best product profile to physicians and patients. The U.S. approval is the first step in our overall strategy to bring Pepsitacotan to GA patients around the world. We remain on track with our EU marketing authorization application and plan to submit our MAA by the end of this year. Turning now to the rest of the business, the Empaveli launch continues to progress well in its second year on the market with U.S. product sales of $17.7 million in the third quarter. Globally, our partner, Sobe, is also making progress in bringing Aspavedi to people with PNH. Beyond PNH, we continue to advance Ampavedi as a transformative therapy for other rare, complement-driven diseases, AAV-based gene therapy products, and neurodegenerative conditions. And our early-stage pipeline including both our collaboration with BEAM and our internal programs, continue to advance. And with that, I will turn it over to Adam.