Good afternoon. My name is Valerie, and I will be your conference operator today. At this time, I would like to welcome everyone to Apellis Pharmaceuticals Fourth Quarter and Full Year 2020 Fiscal Results Conference Call. Today's call is being recorded and a replay will available at apellis.com.

I would now like to turn the call over to Tracy Vineis, Vice President of Communications at Apellis..

Thank you, Valerie. Good afternoon, and thank you for joining us today to discuss Apellis' fourth quarter and full year 2020 financial results. With me on the call are Co-Founder and Chief Executive Officer, Dr. Cedric Francois; Chief Medical Officer, Dr.

Federico Grossi; Chief Commercial Officer, Adam Townsend; and Chief Financial Officer, Timothy Sullivan. Before we begin, I would 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'm pleased to turn the call over to Cedric..

Thank you, Tracy, and good afternoon to everyone, joining us today for our first quarterly conference call. 2020 was a defining year for Apellis, marked by the positive Phase III PEGASUS data in paroxysmal nocturnal hemoglobinuria or PNH.

The PEGASUS results highlighted the potential of pegcetacoplan to elevate the standard of care in PNH and the broad platform potential of targeting C3 for complements driven diseases.

In 2021, we look forward to a transformational year for Apellis as we further build on our global leadership in complement across a broad range of the diseases with high unmet need. As you can see on this slide, our corporate strategy is based on 3 strategic objectives, and we expect to make significant progress against each of these this year.

Our first objective is to establish systemic pegcetacoplan, a targeted C3 therapy as a disruptive treatment across rare complements-driven diseases. We have a PDUFA date of May 14 for the potential U.S.

approval of pegcetacoplan in PNH; and our Chief Commercial Officer, Adam Townsend, will discuss our work to prepare for a successful commercial launch. We also expect to advance 4 additional registrational programs of pegcetacoplan with our partner, Swedish Orphan Biovitrum, or Sobi, as we work to maximize the broad potential of targeting C3.

The second objective for our company is to be #1 in treating retinal diseases. In what will be a seminal event for Apellis, we expect top line results from our Phase III clinical studies of pegcetacoplan in geographic atrophy, or GA, in the third quarter of this year.

GA is a relentless and disabling disease that affects approximately 5 million people around the world, and we have a unique and exciting opportunity to advance what could become the first drug for people living with GA. Our third objective is to develop new technologies to control complement.

Our research team has been very active on a number of fronts, and we look forward to advancing 3 new product candidates into clinical development by the end of next year. As you can see, we have an ambitious strategy and several key milestones in 2021 to advance our global leadership in the treatment of complements-driven diseases.

Now let's dive deeper into our first objective, establishing systemic pegcetacoplan as a disruptive therapy for rare diseases. I will now turn the call over to our Chief Medical Officer, Dr. Federico Grossi, to review our recent data on pegcetacoplan in PNH.

Fede?.

Thank you, Cedric. I'd like to begin by reminding everyone why we believe that pegcetacoplan has the potential to elevate the standard of care for people living with PNH. PNH is characterized by the destruction of pulp season carrying red blood cells through both extravascular and intravascular hemolysis caused by uncontrolled complement activation.

Current treatments inherent the complement cascade downstream at C5, controlling intravascular but not extravascular hemolysis. As a result, while C5 inhibitors offer improvement patient survival, they do not add as many debilitating symptoms from which people living with PNH continue to suffer.

Early pegcetacoplan target complement centrally at C3, controlling both intra and extravascular hemolysis.

As a result, pegcetacoplan met the primary endpoint in the Phase III PEGASUS study and became the first and only investigational therapy to demonstrate superiority compared to Soliris or eculizumab, with an improvement in adjusted means of 3.8 grams per deciliter of hemoglobin at week 60, as well as sustained improvements in other key clinical measures.

The safety profile of pegcetacoplan was comparable to Soliris. Based on these results, we received priority review from the FDA for pegcetacoplan in PNH with a PDUFA date of May 14. Additionally, in December, we announced top line data at week 48 from the PEGASUS study.

These long-term results show that pegcetacoplan has the potential to help PNH patients gain and maintain more complete control of the disease. At week 48, hemoglobin increases were sustained with pegcetacoplan-treated patients with a mean improvement from baseline equal to the increase in existing in pegcetacoplan-treated patients.

Importantly, as you can see in the graph, Soliris-treated patients who switched to pegcetacoplan during the open-label period also experienced sustained improvement in hemoglobin and other key clinical measures, similar to patients treated with pegcetacoplan monotherapy during the randomized controlled period.

Sustained improvements in transfusion avoidance, reticulocyte count, lactate dehydrogenase or LDH levels and functional assessment of chronic illness therapy or FACIT-fatigue scores were also observed in patients treated with pegcetacoplan.

At week 48, the safety profile of pegcetacoplan was consistent with previously reported data and non-new safety signals were identified. Also, in December, at ASH, we presented a matching adjusted indirect comparison, or MAIC, analysis across the pivotal studies called pegcetacoplan and Ultomiris or ravulizumab, a longer C5 inhibitor.

In the absence of clinical head-to-head study, MAIC is a valid and accepted method for comparative effectiveness research used by health technology assessment bodies across the world. The MAIC showed that pegcetacoplan, 76% more patients achieved hemoglobin stabilization compared to patients on Ultomiris.

Also, 64 more patients in pegcetacoplan achieved LDH normalization. This is remarkable because LDH is a biomarker of intravascular hemolysis, the top hemolysis that C5 inhibitor like Ultomiris control well..

Thank you, Fede. The positive Phase III PEGASUS results showed the potential of pegcetacoplan to elevate the standard of care in PNH. And we are working hard to prepare for its successful U.S. launch in anticipation of our May PDUFA date.

As you can see on this slide, people with PNH continue to suffer from significant unmet need despite their current treatment with C5 inhibitors like Soliris and Ultomiris. Clinical data and our own market research have shown that about 1/3 of patients on C5 inhibitors continue to require transfusions to address their falling hemoglobin levels.

Another 1/3 of these patients continue to be severely anemic and experience other symptoms like severe fatigue. This has a huge impact on these patients. The final 1/3 of patients have closer to normal hemoglobin levels but only achieve that at the expense of maximum output of red blood cells from their bone marrow.

As these data show, there is an urgent need for new treatments within PNH. Over the last 2 years, we have built a robust commercial organization in preparation for our first launch of pegcetacoplan within PNH.

Our integrated team is focused on ensuring that we are ready to effectively address the needs of patients at launch, and our progress is highlighted on this slide. Our value and access team is fully staffed and engaging with high priority payers representing more than 80% of all U.S. PNH patients.

Our discussions with those payers have yielded positive feedback on the clinical profile of pegcetacoplan. Apellis is also in the late stages of finalizing our distribution model and patient support resources and programs.

We will aim to provide patients with a consistent positive experience, both at the time of treatment initiation with pegcetacoplan as well as long-term assistance as and when needed.

As an example, we have established Apellis Assist, a patient-focused program designed to ensure a high-quality patient treatment experience, including the recruitment of our care educator team, which will interact directly with patients through product and drug administration education.

In parallel with our commercial activities, our medical team has also been preparing for launch. As shown on this slide, our medical affairs colleagues have been actively engaging with the top treating physicians via our virtual presence at medical meetings and in-person engagements when appropriate.

They have also initiated an early access program for pegcetacoplan in the U.S. and already established multiple sites to treat PNH patients who are experiencing ongoing disease activity despite treatment with Soliris or Ultomiris.

On marketing efforts, early activities with health care professionals, or HCPs, have been really strong and positive, showing high engagement above industry benchmarks. Almost 90% of targeted U.S. PNH HCPs have accessed our content focused on the unmet need in PNH, examples of which can be seen on the left-hand side of this slide..

Thank you, Adam. The MP&A to advancing 4 registrational programs of systemic pegcetacoplan in rare diseases with high unmet need with our partner, Sobi.

As seen on this slide, in the second half of the year, Apellis expects to initiate a Phase III study to further our registrational program in immune complex membranoproliferative glomerulonephritis or IC-MPGN and C3 glomerulopathy or C3G.

And Sobi launches start registration of problems in cold agglutinin disease, CAD, and hematopoietic stem cell transplantation-associated thrombotic microangiopathy or HSCT-TMA.

Also, in the second half of this year, Apellis expects to complete enrollment for our potentially registrational Phase II MERIDIAN study in amyotrophic lateral sclerosis or ALS.

In parallel to our work in systemic pegcetacoplan, we continue to execute our Phase III studies of intravital pegcetacoplan in GA, with top line results expected in the third quarter.

We believe that our GA program represents a unique opportunity to make a difference in the life of 5 million people at risk for blindless with no treatment options available and few opportunities in the horizon.

Results from the largest retrospective study in GA secondary age-related macular degeneration, or AMD, were presented as a late-breaker at the American Academy of Ophthalmology meeting. The study, which was conducted in partnership with Verana Health, highlighted that the saving impact this disease has on quality of life..

Thank you, Fede. Since we issued a press release earlier today with the full financial results, I will just focus on the highlights for the full year 2020. As of December 31, 2020, Apellis had $877.6 million in cash, cash equivalents and short-term marketable securities compared to $352 million in cash and cash equivalents as of December 31, 2019.

This increase primarily reflects the addition of cash from our follow-on offering for gross proceeds of $404 million in January 2020, our convertible offering for gross proceeds of $329 million in May of 2020 and also the receipt of $250 million in the upfront proceeds for the Sobi transaction in October 2020, less our cash used in operations.

Research and development expenses were $325 million for the full year ending 2020 compared to $221 million for the same period in 2019.

The increase in R&D expense for the full year 2020 was primarily attributable to an increase in manufacturing expenses for our Phase III clinical trials and potential commercial launch, costs associated with ongoing and planned clinical trials, compensation and related personnel costs primarily due to the hiring of additional personnel in 2020, among others.

General and administrative expenses were $139.4 million for the full year ending 2020 compared to $67 million for the same period in 2019.

The increase in general and administrative expenses for the full year 2020 was primarily attributable to an increase in professional and consulting fees, employee-related costs due to the hiring of additional personnel and directors stock compensation expense, among others.

For the full year ending December 31, 2020, Apellis reported a net loss of $344.8 million compared to a net loss of $304.7 million for the same period in 2019. We remain well capitalized to execute on the potential launch of pegcetacoplan in PNH and to continue to advance our robust clinical development plan.

Our cash runway is expected to fund operations into the second half of 2022. I will now turn the call back over to Cedric for closing remarks..

Thank you, Tim. As you heard today, we have a transformational year ahead and the key commercial, clinical and regulatory milestones are shown on this slide. As you can see, we have a busy 2021 as we work to deliver on the full potential of targeting C3 across a broad range of complement-driven diseases.

Before we move to Q&A, I would like to thank the patients, investigators and caregivers who have participated in our clinical trials, and our employees and investors who have helped advance Apellis through this difficult year. We look forward to keeping you updated on our upcoming milestones. And now, operator, please open the call for questions..

Our first question comes from Anupam Rama of JPMorgan..

One of the most common questions we've gotten recently is in DERBY and OAKS, how do you think about the potential impact of sort of missed injections? And how that impacts a study, the stats plan, how we should be thinking about this? Is the FILLY every other month on maybe a reasonable proxy for how we should be thinking about the potential effect of APL-2 with missed doses?.

Thank you so much, Anupam. And great hearing you, and thank you to everyone for joining this call. The brief answer to this question is that the study, DERBY and OAKS continue to be well powered to show what we intend to show in these studies in spite of the missed injections that we have seen so far.

Obviously, we did have missed injections, and the study was impacted by COVID. But we have a way of looking back, as you alluded to, to the FILLY trial and compare the frequency of missed injections, how these missed injections occurred and based on that, make an assessment of the quality of the Phase III clinical trials.

And it is those assessments that make us highly confident that we are in a good place to measure the primary endpoint with good quality..

Our next question comes from Umer Raffat of Evercore..

Cedric, what percentage of the patients had wet AMD at baseline in the fellow eye? I'd be very curious.

And also sort of separately, for the primary endpoint, can you remind us how the autofluorescence was done in Phase II and how it's being done in Phase III? But, I guess, what I'm really asking is, are you using CSLO, the scanning laser, or using the standard fundus camera?.

Thank you so much, Umer, for that question. So I'm going to start with the second part of your question, which is that we measure autofluorescence in exactly the same way in the Phase III as we did in the Phase II clinical trial. And then I have to ask you to repeat the first part because the line was breaking up a little bit, please..

No problem. What percentage of these 600 patients in each trial had wet AMD in the fellow eye? Because I recall, one of the discussions with FDA was whether they should or should not be included and FDA encouraged you guys to put them in. But presumably, that will impact the conversion rates.

So I'm curious what percentage you have at that baseline?.

Yes. No, thank you. So in the FILLY trial, as you may recall, at baseline, we had 38% of patients who had geographic atrophy in the study eye and wet AMD in the contralateral eye. That compares to a normal natural frequency of that demographic of approximately 25%.

So we had many more patients that came into our Phase II clinical trial, having that phenotype presence. The reason for that is that back then, there were 2 large Phase III clinical trials that were enrolling with lampalizumab, which excluded those patients from their studies.

In the Phase III clinical trials, we are not commenting, of course, yet on the baseline characteristics that will come in due time. But everyone should expect, I think, a lower present -- a naturally lower occurrence of patients coming into the study with that particular phenotype.

The reason simply being that we do not compete anymore with these other studies and that I think we will be closer to that 25% normal demographic spread..

Our next question comes from Steve Seedhouse of Raymond James..

Maybe I'll ask about the COVID data that's coming up. I'm just curious if you have hurdle that you set that determine next steps of that asset in that indication? I think Alexion has talked about a 20% mortality delta or having statistical confidence in a 20% reduction in mortality at their interim analysis, which ultimately they didn't meet.

I'm wondering if you have something simpler established that will depend if you advance that Phase III or not..

Yes. Thank you so much, Steve, for that question as well. So as we have previously commonly or publicly reported, I should say, in December -- at the end of December, towards Christmas, we completed the enrollment in this study. So we are now getting close to the point in time where the data safety monitoring board will make an assessment.

We will -- that should be expected in the near future. And then the top line results will be after the full data analysis has been completed..

Our next question comes from Derek Archila of Stifel..

Maybe one on PNH. I mean, Cedric, can you just provide some color how you think about the competitive landscape shaping up in that indication, particularly with the orals that are in development? And then maybe I'll just throw in one more in terms of some of the new programs you might be launching from now to 2022.

Is there an oral complement inhibitor in the mix there?.

Yes. Thank you so much, Derek, for that question. So as we have commented many times, we are excited about the development of new complement inhibitors by others and ourselves. I think we're really just at the beginning of what complement control can do in a wide range of indications.

Of course, the launch in PNH being the first one now where, very importantly, for patients, I believe, and we all believe, that the control of extravascular hemolysis will elevate the standard of care in these patients.

In a couple of years, particularly in PNH, we may be looking forward to the introduction of oral products that can control the alternative pathway of complement in these patients, and thereby address extravascular hemolysis.

And while on the surface, it may seem much more appealing to have an oral product compared to a twice-a-week subcutaneous product, it's important to bear in mind that in a disease like PNH, there is no room for error.

What I mean by that is that when you are reliant on a pill that you need to take twice per day, for example, forgetting to take a pill could have important consequences. That is a liability that many patients are not very comfortable with, neither our physicians.

In line with that also is the fact that we still need to find out if the majority of patients respond well to these products and whether the control of PNH is durable in the long run. So we look forward to the Phase III readouts. But beyond that, I want to end this on a positive note.

I'm personally very excited about the development of oral products, in PNH, not so much, but I think in other indications where kind of the exquisite control of complement is less important, it will provide a lot of opportunities.

As it relates to our own internal programs, we are not yet ready to comment on that, but we look forward and are excited about sharing more in the months to come..

Our next question comes from Justin Kim of Oppenheimer & Co..

Just maybe on PRINCE.

As we prepare for that readout, could you walk us through maybe perhaps what unique insights we may see from this naïve to complement treatment population, and particularly maybe where the geographies where APL-2 is being used here may differ from like a standard of care perspective?.

Thank you so much for the question, Justin. I'm going to hand that one over to our Chief Medical Officer, Dr. Grossi.

Fede?.

Thank you, Cedric and thank you, Justin, for that. We're really looking forward to show the outcome of that study and sharing with you the results. So the study, as you point out, is looking at a PNH population that is naïve to complement inhibitors and the primary endpoint is hemoglobin stabilization, which is a typical endpoint for PNH studies.

And, in addition to that, we're looking at quality of life and transfusion dependency. So that -- the population, this population does not differ from the treatment-naïve population that you see on regions where eculizumab is available.

We had to do the studies in regions where eculizumab is not available in order to do it, but the population does not defer..

Okay. Got it. And maybe just a follow-up to a previous question and maybe ask maybe slightly differently.

When you think about pan-AMD sort of therapies, do you -- is it a fair assumption that we may see sort of modalities outside of intravitreal injection, if we're looking at populations who maybe have less severe disease?.

So can you repeat the question on AMD? Or.

Just wondering, as we think about sort of new agents in a pan-AMD sort of therapeutic, does that suggest a non-injection based therapy, potentially?.

Well, when you look at -- this is outside of AMD. For diseases into the eye, the level of systemic exposure that you will get from treatments that are not injected into the eye maybe too high.

So there will have to be a therapy that is systemically, you can achieve good levels in the high exposure to patients to market the drug and having safety concerns from a systemic perspective..

Our next question comes from Alethia Young of Cantor Fitzgerald..

One, just for me is on -- I mean, obviously, Alexion kind of -- is going to be acquired. And sometimes there's disruption which can occur.

I just wanted to get your perspective on whether, from a human resource standpoint, or even a commercial standpoint, that you're sensing any sort of opportunity that you might be able to take advantage of, even though you do is already quite robust? And then the second question I had is just on the 4 programs that are kind of moving towards registrational, is -- analyst is always interesting and a little bit challenging.

I mean, do you perceive that as being a higher risk, higher reward program? Or should we think about them all kind of a little bit more in equivalence?.

Yes. No, thank you. Well, starting with the first question. Our primary goal is to elevate the standard of care in PNH. The competitive landscape has changed. But, at the end of the day, we believe that the important unmet need that exists in PNH is going to be the main driver of sales, and we look forward to addressing that.

Then, as it relates to the new -- or the other indications, which are currently in registrational developments, of which there are 4, so C3 glomerulopathy with IC-MPGN, ALS, cold agglutinin disease and HSCT associated thrombotic microangiopathy. The ALS trial has a special place.

It has a special place as the first neurological indication that we are targeting. It has a special place as, of course, an indication with an incredible unmet need that we hope to address. And also, as a place where we believe that C3 offers advantages over other places in the continent cascade where this disease can be controlled.

So, for us, again, the primary motivation for us was to bring the science together with the molecular entity that we have and our belief that we can address the unmet need. Is this a high-risk program? Of course, it is.

Any trial in ALS is with a high-reward associated, but one that we do truly believe fits very well into kind of the plethora of indications that we are pursuing with systemic pegcetacoplan..

Our next question comes from of Cowen..

So my question is actually about the allergic reaction to PEG that have been sporadically seen in people who have received the Pfizer and Moderna vaccines. I believe, the CDC recommends that anyone who experiences an immediate allergic reaction to PEG should not receive the booster.

So I was just wondering what gives you confidence that patients are being sensitized to PEG? And I assume that is your belief, but if it's not, why that's also the case?.

Yes. Thank you, Laura, for that question. So as you correctly mentioned, there are a couple of isolated cases of anaphylaxis that have been associated with PEG sensitization. This is something that is well-known, that can sometimes occur.

It's important, therefore, that when you introduce products like ours, especially in the beginning to make sure that there is a good follow-up.

What is important to note here, however, as well, is that with the introduction of the vaccines with the PEG element in them, there does not seem to be an extra sensitization because the booster vaccines do not seem to be associated with any type of additional immune reactions. So we feel very comfortable with where we stand right now.

Will we, in the future, in these rare diseases, right, I mean, once in a while, see an anaphylactic reaction, that is possible. Thus far, that has not been the case..

Our next question comes from Matthew Luchini of BMO Capital..

So first, on PNH, commercially, I guess I'd love to get a little sense as to what your internal market research is telling you about the initial launch.

And what I'm really looking for is a little bit more of the color around things like are patients -- are your doctors telling you that the patients are asking for the drug? Are they planning to call patients in versus waiting for the next visit? Any kind of color there that could help sort of set our expectations around what the initial launch is going to look like? And then secondly, just kind of a housekeeping question.

It looks like R&D saw a pretty decent sequential step down in 4Q.

And I'm just wondering if that's something that we should think about as sort of the new baseline going forward? Or how we should think about that as we think forward into this year and beyond?.

Thank you, Matthew. I will hand the first question over to our Chief Commercial Officer, Adam Townsend, and then Tim will take your second question..

Thank you, Cedric, and thank you, Matthew, for the question. So we've spent a lot of time with the PNH community, patients, caregivers and everything that surrounds the patient. And we do believe that we expect some patients to have a conversation with their physician about the potential to elevate the standard of care with pegcetacoplan.

We've got various patient-focused marketing activities out there, and we're getting a great response from them as they interact with our content. They truly understand the unmet need that exists within the market.

So whilst this is very much an efficacy driven story for us, we think that at launch, that physicians will have identified the patients with the highest unmet need. And you've seen from our presentation and our previous discussions, we look at the market of C5 treated in.

So we expect to transition with -- from the patients that have the highest unmet need to the broader patient and unmet need population as we work through. A core piece of that will be when patients go in to have a conversation with their physicians.

And we also expect some physicians to already identified the patients and actually potentially call them in. Still a rare disease. It will be a very thoughtful approach to launch. And obviously, this is an important conversation for patients and physicians that have around the potential of pegcetacoplan..

Sure. And thanks, Matthew, for the question. I'll take the R&D question. So what you're seeing in terms of the 4Q step down in R&D is actually more of an accounting and Sobi-related concept. So Sobi, as you know, from the structure of the deal, we'll reimburse Apellis for $80 million worth of R&D expenses over the course of the next 4 years.

The way those are accounted for and when those begin started in the fourth quarter, primarily of 2020. And so that is actually counted as a counter account. And so from a GAAP perspective, those amounts were deducted from R&D and that just brings the number down.

Ultimately, the reimbursement for that $80 million will come over time over the next 4 years. So I would look at the true R&D expenses much closer to the third quarter, flat to a little bit up, and probably it will steadily, although not dramatically rise over the next year..

Our next question comes from Yigal Nochomovitz of Citigroup..

I had one on DERBY and OAKS. So as you know, Cedric, in FILLY, the difference in the absolute lesion growth area between the sham and the monthly pegcetacoplan was 0.66 meters square, and that was obviously deeply stat sig.

Now presumably, the bar is lower in DERBY and OAKS given a higher power so could you comment at all on the hurdle that you need to hit for the difference in the GA lesion growth area for DERBY and OAKS to be successful?.

Thank you, Yigal. So the Phase III clinical trials are more than 95% powered to show the same effect that we saw in the Phase II clinical trial for the monthly dosed individuals. And somewhere between 80% and 90% for every other month dose individuals. This is, of course, on the presumption of similar variability and accounts for a p-value of 0.05.

So again, for us to kind of protect the p-value that we got in the Phase II clinical trial, we made sure to have as few changes as possible. We are studying the same exact patient population. We analyze and reap the findings in the same way. All of that was maintained between Phase II and Phase III..

Our next question from Laura Chico of Wedbush..

I just wanted to circle back on one with respect to wet AMD. Cedric, I think you indicated the normal frequency is around 25%.

I'm wondering if you could just comment then around maybe what is an acceptable rate of new onset exitation that we should be thinking about in DERBY and OAKS, a, from a regulatory perspective; and b, from a commercial perspective? Just kind of curious if patients do have a lower baseline frequency there, how might that change the expectation? And then a quick follow-up.

Just with respect to PNH, could you just remind us or talk a little bit about your expectation of whether, Cedric, APL-2 labeling would also extend to include Ultomiris-treated patients. I think you had a slide there comparing that with Ultomiris.

So what type of data or guidance might you be able to provide for patients thinking about a transition?.

Yes. Thank you so much.

So starting with the first part of your question, the wet AMD occurrences that we had in the Phase II clinical trial are important to contextualize, right? These were small expedites, which did not lead to significant vision loss and that is why in the Phase III clinical trial, we are studying the exact same patient population as we did in the Phase II.

So your question is, what is acceptable from a physician perspective or from a regulatory perspective in terms of exudation, it is the exudation rate that we saw in the Phase II clinical trial.

And that exudation rate was arguably artificially higher than what you should expect in the Phase III because we had so many patients in the study that had already went AMD in the contralateral eye when they came into the study.

And also because in the Phase II with the benefit of hindsight, we have some investigator bias that may have contributed to an increased frequency of treatment with anti-VEGF as well. All of that will be corrected in the Phase III clinical trial.

But I think the most important take-home message here is that our Phase III clinical trial, if it hadn't been for COVID, if it hadn't been for the kind of the INPDs that we had to wait for in Europe would have been rolled probably in 9 months.

I mean, that gives you a sense of the unmet need that exists and the willingness and desire of physicians to treat these patients. So we are -- we start with what I believe is a very strong baseline, and we will see what we see in the Phase III clinical trial.

But importantly, exudations in patients with geographic atrophy are a normal phenomenon, and I would encourage listeners that haven't done that to look at our presentation at the American Academy of Ophthalmology. It was a late-breaking abstract where we looked retrospectively at 69,000 patients with geographic atrophy.

It's the largest study of its kind ever done, and the purpose was to find out how frequent it is for patients with geographic atrophy to develop wet AMD.

And on a base case, the number to bear in mind is that patients with pure geographic atrophy, meaning no exudation yet anywhere, developed wet AMD based on claims based data and 8% of cases over 2 years.

If you start off with wet AMD in one eye and GA in the contralateral eye, then that GA eye has a 22% of developing wet AMD over the course of 2 years. So not at all unusual. And again, it comes down to what is the nature of these exudation that are observed. Then, to get back to your second question as it relates to the label.

So we are not yet commenting on labeling. We believe that we will have a broad label when we get our approval. A broad label would mean, including treatment-naïve patients. And in that particular case, would, of course, be applicable and usable in patients that are on baseline Ultomiris as well..

I'm showing no further questions at this time. I'd like to turn the call back over to Cedric Francois for any closing remarks..

Thank you so much, and thank you, everyone, for joining us on our inaugural financial results conference call. We are excited about the transformational year ahead for us. I would like to close by reiterating our corporate strategy for leadership in complement.

We aim to establish systemic pegcetacoplan as a disruptive therapy across rare complements-driven diseases. We plan to become #1 in the retina with the first treatment for geographic atrophy, and we continue to advance innovative technologies to control complements with a focus on complement factor C3. Thank you again for joining us today..

Thank you. Ladies and gentlemen, this does conclude today's conference. Thank you all for participating. You may all disconnect. Have a great day..