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Wells Fargo 20th Annual Healthcare Conference 2025

Sep 3, 2025

Derek Archila
Senior Biotech Analyst, Wells Fargo

All right, everyone, I think we'll get started with our next session. My name is Derek Archila. I'm one of the Senior Biotech Analysts here at Wells Fargo. Very excited to have Apellis Pharmaceuticals with us here for our next fireside discussion. We've got Tim Sullivan, the Chief Financial Officer, as well as David Acheson, the Chief Commercial Officer. Gentlemen, thank you so much for coming and looking forward to the discussion here.

Tim Sullivan
Chief Financial Officer, Apellis Pharmaceuticals

Thanks, Derek.

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Thank you, Derek.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Awesome. Maybe let's just kick things off in terms of state of affairs. Maybe Tim or David, do you guys want to give us a sense of where the business is today? Then we can start delving into some of the key questions here.

Tim Sullivan
Chief Financial Officer, Apellis Pharmaceuticals

Perfect. For those who are not familiar with Apellis Pharmaceuticals, it's a commercial biopharmaceutical company that's focused on complement biology. For those of you who are not familiar, complement is part of your innate immune system. It's sort of your original old immune system. When it's dysregulated, it can cause a number of serious diseases. Apellis Pharmaceuticals was founded with the idea of targeting diseases within the complement system, but doing so at the center of the complement system, a target called C3. This is the central point of the complement system in the way our drugs work. We have two commercial drugs. One is called SYFOVRE, which targets geographic atrophy in the eye, or treats geographic atrophy in the eye, and then also EMPAVELI, which is for a number of rare diseases we'll discuss in a minute.

Both of those drugs have the same active ingredient, which is pegcetacoplan, which targets the central component of the complement system. As a result, it sort of acts as the, you know, either the Swiss Army knife or the Nvidia chip or whatever.

Derek Archila
Senior Biotech Analyst, Wells Fargo

The columnist.

Tim Sullivan
Chief Financial Officer, Apellis Pharmaceuticals

Yeah, exactly. The complement system is really, as far as we know, almost a litmus test for whether a disease is a complement-related disease. There are tons of drugs being developed in the complement system. We believe pegcetacoplan is sort of the most effective in the sense that it can treat pretty much all diseases within complement because it's centrally located right below all three of the activation pathways. That's what makes pegcetacoplan different and what makes our two products unique. As I mentioned, we have these two commercial products. In terms of where we stand today, we have three approvals, three approvals in the last few years, which is a great accomplishment. For, again, for SYFOVRE, this is a product that was approved for geographic atrophy. It was the first product approved for geographic atrophy, and it's also the market leader in the geographic atrophy market.

Then we have EMPAVELI, which was approved for PNH in May 2021, and has subsequently, as of this year, been approved for two other diseases of the kidney, two diseases of the kidney, excuse me. The first one is C3G, or C3 glomerulopathy, and then IC-MPGN, which is a related disease, which expands our market by approximately 5,000 patients. These are rare diseases. It's a rare disease drug. Both PNH, as well as C3G and IC-MPGN, are rare disease opportunities. In our pipeline, we're developing a drug called APL-3007, which is an siRNA, which is being used to also improve the characteristics of SYFOVRE in combination. We have a kind of a combination geographic atrophy targeting approach, which we're hoping to kind of increase the durability and effectiveness of SYFOVRE. We have some other earlier stage programs, which we'll talk about at some point in the future.

Very recently, we just did a financing deal. It was a royalty deal with our partner, Sobi. They have the ex-US rights to EMPAVELI. We did a royalty deal, a royalty monetization with them, where we added $275 million to our balance sheet. We come into this conversation with a very strong balance sheet, with two commercial products that are strong and growing. We feel very good about where we are. I don't know if you have anything else.

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

No, that's great.

Derek Archila
Senior Biotech Analyst, Wells Fargo

All right. Thanks, Tim. Maybe let's discuss the recent launch of EMPAVELI in C3G and IC-MPGN. Obviously, as you stated, it's a rare disease market. Maybe, if you guys want to start just in terms of what are we seeing in the early innings since approval? How do you guys think that launch will ramp over the next couple of quarters?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah, yeah, it sounds good. Thank you, Derek. First of all, we're super excited about what we've seen for a label from the FDA for both C3G and IC-MPGN. It's a very broad label, and it gives us both pediatric as well as adults in both disease stages, which is really, really good. Also, there's post-transplant data in the label for patients that have gone through transplant that could also, C3G patients in particular, could be utilized for those patients as well. It's a very broad label, which is positive. We're a little over four weeks into the launch of those two indications, and I can tell you, I think things are going extremely well, at least from a connection to the right customers, the KOLs, and there's a lot of interest because of the broad label and what we've seen in our data.

I think it's important to remember that we hit what we call the trifecta. Proteinuria reduction of 70%, which is the lead endpoint in the studies. We also showed that 71% of the patients after the studies actually had zero C3 staining left in the kidney. That's a very big number and hugely impressive. We were able to show stabilization of eGFR. If you talk to a nephrologist, proteinuria is key, but it's not the only factor they're looking for. We cover off on the other two, which is staining as well as eGFR stabilization. It's exciting.

I can tell you that as soon as we got the label, we were in the process of trying to move our expanded access program patients over, which is going to take till the end of the year, and we're in the process now of getting folks REM certified and putting our opportunities for patients to get start forms in and get on the product. It's still very, very early, as you can imagine, but the news is exciting in the space, and we're excited to be able to help patients in the two disease stages.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Can you just remind us, in terms of the number of EAP patients? I guess, again, you said by the end of the year, what are some of the factors that would dictate that being faster or?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah, that's great. In our expanded access program, we have 50 patients that are in the process of moving over. The program obviously was one of those things that was available for patients because there was a high unmet need. Now what we're working on is making sure that those patients move over to the commercial product. It takes a little bit of time to do that. In general, once a patient's identified and they go through vaccinations and the process for start forms and all of the prior auth work and those types of things that happen in rare disease, it takes about four to six weeks to move most patients over. We anticipate it'll take to get those 50 patients moved over, it'll take some time in Q4. At the same time, we've got new patients that are coming into the process as well.

Excited to see it, and we'll see how things continue to move.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Can you discuss the REM certification and how long is that, you know, mostly just kind of like paperwork or what's that actually entail for some of these centers?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah, REM certification for a physician is actually, it's by doctor, and it's actually a fairly simple attestation. They need to read through what needs to happen from a labeling perspective, in particular the vaccinations for these patients, which there's three, and then they need to attest to the fact that the patient is indicated, in the process of the label and that they're being vaccinated or have been vaccinated, and then they sign off on it. It takes 15 to 20 minutes. It's actually not that hard. The work really comes into start forms and working with the payer and the specialty pharmacy and all of the logistics with the patient. That's really where the work is.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Gotcha. Can you just remind us in terms of that vaccine requirement? Obviously, this is something that EMPAVELI had prior with PNH as well, but typically, what's the length in terms of getting those vaccines? I think a lot of people are trying to understand, is there going to be a bolus of patients that just come on beyond the expanded access program? Is there really kind of a slight waiting period because of the vaccination requirement here?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah, no worries. Let me address the vaccination process first. There are three vaccinations that every patient needs to have, one of which is meningitis that has two vaccinations that have to occur. We, in our label, as long as you've been vaccinated for influenza B, strep pneumo, and the first meningitis injection or vaccination, two weeks post those injections or those vaccinations, a patient can start product. They can also go on prophylactic oral antibiotics if that's what's needed, especially for the distance between the first and second injection for meningitis. That, in a nutshell, is kind of how that works, right? That is super important because obviously, it's a safety event that we want to keep from happening. That process takes a little bit of time.

We can help facilitate that through our specialty pharmacy and through processes that we have in place so we can speed that process up if a patient's having a hard time finding a way to get vaccinated. We can work with their offices and their physicians to do that. Once that's done, then the start forms go in, all the information is sent into the specialty pharmacy, and then it just takes time with the payers and what processes and logistics to make sure we get the patients to the product within a window of time that is and is paid for by a payer. Okay. From a bolus perspective or what we see for patient uptake, I definitely think, Derek, one of the things that you're going to find in any space like this that hasn't had a lot of options until recently, they had no options, right?

These patients are being, they're waiting. We've got some patient advocates that we work with to help educate our internal teams as well as some work that they do externally for us. They've been anxiously waiting for a product like this to come through that will keep them from actually going to dialysis or potentially post transplant. It is super important to them. I do anticipate, like any disease like that, that you're going to have some bolus and patients that come through in the front end of the launch. We anticipate that it's going to be a typical ultra-rare disease launch after that, where you're going to see nice, good, steady growth. There's about 5,000 patients that we see in the U.S., and all of them, for the most part, should qualify for treatment.

It depends on where they are in their treatment, kind of processes in the disease state. At some point, they should all be educated at least on the product and the availability of it.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Would you see any centers of excellence or KOLs start to vaccinate and prepare these patients prior to approval, just assuming that it would get approved because of the data?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Sure. Yeah. We actually had a number of REMS enrollments that came through from physicians before we actually had approval. All of our EAP patients, for the most part, were vaccinated because they were on product. We had some patients that were already starting to go through the process to be able to get connected to the physician and to education on the product before the label was actually available. All of that started to happen early on, and it continues as now that we're launching as well.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Gotcha. Do you think, you know, of the two opportunities, different, like pre-post-transplant, obviously, like adult, you know, children, like pediatric? How do you think about kind of all these opportunities and ultimately like where physicians see kind of the greatest unmet need?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah. It's interesting you ask that because if you go through our label, you can see pediatric patients for C3G. You can see adult patients for C3G, IC-MPGN. You can see post-transplant patients for C3G for adults. You've got the whole gamut, right? Probably the two areas of the highest unmet need that we're interested in and have concerted efforts around is to make sure that the pediatric patient nephrologists are well educated and know about this product early because that's a super high unmet need with kids, right, that are potentially in a situation where over their lifetime, they'll end up having to have a transplant or certainly go to dialysis if this thing is not taken care of. That's one.

Then post-transplant patients, because one of the things that we know is that in a post-transplant patient, 90% of these patients within 30 days see recurrence of staining of C3 in the new kidney, which, because it's a genetic disease, it's always going to reoccur, right? Those patients should be high unmet need patients, and they are high unmet need patients with some urgency, to make sure that that group of physicians is also well trained on the product so they know how to identify when and how to put patients through the process to get to product and treatment. The rest of it is C3G patients that are adults, which are super important, but a little better understood on how to manage because they've been adults with the disease for quite some time, and now they have options to be treated that they didn't have before.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Now that you're in the market, what are you seeing from Novartis in terms of IPTO, COPAN, any sort of counter detailing? Obviously, you have a more extensive label. Maybe you could just give us some of the dynamics as you see a couple of weeks in the launch.

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah. I think one of the biggest advantages we have is our label, because of the broad spectrum number of patients and the age range that we can treat, right, for both indications, which really separates us. What we get told by the physicians and by the KOLs is what will separate us further is the efficacy that goes along with those broad indications, which we can see in those three areas I talked about, which is proteinuria, C3 staining reduction, and eGFR stabilization. They're super tuned in to the fact that we can bring them all three. That's not necessarily the case in other situations, right? I think the other thing that's super important is most people would say, you've got an oral versus what would be a sub-Q injectable product. We have a very easy-to-use wearable device that goes on when the patients use it.

They get dosed twice a week. They put the device on, they hit the button, they never see a needle, and the device delivers the product over a window of time of about 30 minutes, right? Oral has to be taken twice a day, and so there's areas there where the physicians are saying two things. Efficacy will always outweigh the path that the patients will take the product. The second thing is, they're very intrigued with the fact that we've got something that's so simple to use for these patients with the product that can treat these diseases.

Derek Archila
Senior Biotech Analyst, Wells Fargo

How important is compliance? I mean, you just kind of maybe outline that a little bit, but maybe just in terms of both of those, like if you miss a dose of pegcetacoplan, you know, is that a major issue relative to, you know, for this type of disease?

Look, I think in these disease states, these patients have been living with these situations in the nephrology space for their lives, right? At least since they've been diagnosed. Obviously, the best thing to do is to stay on treatment and make sure that they can continuously be treated. If they happen to miss a dose, actually, in these disease states, they can go right back to treatment. For us, though, the good thing is that we've got, we know that it's a lot fewer times that they have to take this product. Twice a week is significantly different than what you'll see, patient compliance when you have to take it twice a day.

Gotcha. Maybe just a higher-level question in terms of the market opportunity here. I think there's debate whether, you know, is this very niche and small, a couple hundred million to, you know, could this be a billion plus? I guess how do you guys think about this market opportunity and what peak penetration could really look like, you know, across C3G and IC-MPGN?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

I think if you take, I'm actually going to add PNH into it too, right? You add in PNH, you have C3G and IC-MPGN. We absolutely believe we have a blockbuster product. If you take a look at the 5,000 patients that are out there, they're diagnosed through, all of them are diagnosed through a biopsy. We know that those patients exist, especially on C3G because the ICD-10 code exists for us to be able to track. IC-MPGN is actually an epi model to get to that. We've got some learning to do, right, on the number of patients that exist. Now that there's a treatment out there like this that can actually modify the disease versus treat the symptoms, we actually think there's probably more patients that could surface as diagnosed moving forward, which is typical in most rare disease situations where there haven't been options before.

We're very confident in the 5,000 patients. We think there's probably more out there that could come to the table at some point in time, as a result of a product that can treat what their needs are that wasn't there until recently.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Gotcha. How do you think about EMPAVELI, just, you know, broader across, you know, the three indications in terms of IP, lifecycle, you know, management? Is this something that's kind of on the radar? I think the patents are up in like, you know, I don't know if it's late 2020s or early 2030s, but, you know, finite nonetheless. How do you kind of think about that given that you want to continue to expand in some other renal locations, you know, based on the data you've seen?

Tim Sullivan
Chief Financial Officer, Apellis Pharmaceuticals

Sure. From a patent perspective, the composition of matter is, depending on the geography, it's 2032, 2033, but with customary extensions, 2034, 2035. We feel very good about the patent life. We also, when we started our next two clinical studies, our pivotal studies for EMPAVELI, we looked at patent life and said, okay, we want to have these things be ready for or ready for approval by 2030. That was sort of the impetus for us when we were looking at FSGS and DGF. We thought that was the appropriate timeframe and we're continuing to look at lifecycle management opportunities.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Gotcha. Okay. Maybe just shift gears to SYFOVRE for a bit. You know, just, you know, where are we in terms of like, you know, kind of stabilization? You know, last quarter, it seems like, you know, again, some modest growth there, and maybe the business is stabilizing. Do you feel like, you know, we've finally kind of done that, or do we still need to kind of look out a couple more quarters before we feel like that business is kind of starting to grow off that base modestly?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah. So look, I'm excited about SYFOVRE. I think one of the things that we've been through is a bit of a roller coaster ride with the brand, right, in the last couple of years. I think we're through a big part of that. We came out of ASRS and I felt really positive that the storyline there was about efficacy. It's about real-world data. It's about the things that patients and physicians are seeing with the product. Because we've got patients today now, Derek, they've been on product for four years, right? The amount of tissue that can actually be saved in that four-year period of time, after they started patients early and they've kept them on for a long period of time, is very substantial. That's a great story for us. I'm excited about what the brand can do for patients.

You know, I think at the end of the day, it's still important for us to know that this is a space that we're still continuing to build out. There's lots of room for growth. That takes a lot of disease state education still. There's a lot of work that needs to happen in offices where patients currently exist, not to mention the referral processes. I think, you know, what we've put out for guidance is that low to mid-single-digit growth is something that can get us where we want to go and can hopefully be consistent on the injection side, is what we're looking for.

I will tell you, I think, coming out of the last couple of meetings, in particular ASRS, I'm excited about the brand and I'm excited about what we're starting to see for some really positive real-world data that I think can help us long term. I would echo that.

Tim Sullivan
Chief Financial Officer, Apellis Pharmaceuticals

I would say that, you know, the franchise has finally hit sort of that stabilization after that tumultuous period. We feel very good about that stabilization. In terms of the things that are going to kind of push growth to that next level, those are kind of in the next kind of the 12-month timeframe.

You know, those things include like the prefilled syringe and some of the technologies that we use to help, we're going to use to help patients, physicians, and caregivers understand the value of treating the disease, which I think is a missing piece of that dialogue right now because, as you know, it isn't one of those things where you see the effect right away, right? It's one of those things you have to have a little bit of faith in. It's much easier when you can apply certain technologies to help people see the benefit over time and what it means to treat or not treat. Once people can really understand that, the decision is very easy to treat.

Derek Archila
Senior Biotech Analyst, Wells Fargo

What have you seen tangibly from, you know, maybe ASRS or some of these meetings where you guys are highlighting some additional data with some physicians or groups of physicians? Has it really started to change their prescribing or increase it? I guess, again, give us some anecdotes in terms of what you're seeing on the ground that's a result of some of this more recent data.

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah, no, I think it's great. Let me just make sure everyone remembers where we sit in the marketplace. We are definitely the market leader, and in every market or every metric that we look at, in all the markers, we lead the market in market share, new new brand, revenue, all of those, all of the things that you can look at as far as metrics we lead. I think it's important to know that, you know, when we left Q2, we were running in the 55% range on new to brand prescriptions, and we own over 60% in regards to TRXs. As a market leader, I think we should feel good about that. I think what you're starting to hear now is when you, and I'll reflect back on ASRS, it is clear when you talk to physicians, they know that we have a high efficacious product.

In comparison to the competitor, they know that our product has robust data that can help them make decisions for their patients and do the right things. That four years, that 48 months of data from the GALE study helps them understand what can happen after someone's been on a product for a period of time in regards to tissue preservation. Nobody else can talk about that data, and our competition will never have that. I think it's important for us to really highlight those things. I think the general sentiment from the physicians is we see lots of opportunity to help patients, but they also tell us, look, it takes time and education and work. That's why we've settled in on the kind of growth factors that we put out, you know, for guidance, at least at this point in time.

Derek Archila
Senior Biotech Analyst, Wells Fargo

I guess based on your trajectory as well as what we're seeing from, you know, a sell-off with yesterday, how has that informed or changed your view of the overall opportunity for GA? I mean, do you still, I mean, I think the idea was that this could be multi-billion. There's a million, you know, plus patients. Is that, has that changed at all?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Look, I think the market's there, right? There's a million and a half patients that we know of that exist in the space. I can give you a quick example. One of our largest accounts has several thousand patients that sit inside their current retina offices, and maybe 10% of them are treated, right? In a group that maybe has 40,000 total patients that are GA identified, you're talking five to ten thousand that have been treated, right? The opportunity alone in some of these accounts without referrals coming in is quite big. The opportunity for patients that are identified that are not in the treating offices, the process to move them over is super important because then they can see someone who can actually do an injection for them.

The market itself, I think, is large, and now it's an effort to make sure education and getting to the patients and then getting the patients into the right places to be treated is what we're focused on.

Derek Archila
Senior Biotech Analyst, Wells Fargo

What's the most important thing that physicians do to activate those patients that are just apparently sitting there? Is it just the fact that they don't like injections, or they don't see value in maybe slowly slowing the progression versus reversing some of the vision loss? What are the things that the physicians really need to do?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah. I think that the patients that sit inside a retina specialty office already, those physicians are working to activate that patient because they already know that they have the disease. They've already had these conversations, right? It's a little easier than someone that might be coming in from the outside that's newly diagnosed. It takes a little less time for the office staff. They don't have to go through all the new workup, that kind of thing. There's the education that takes some time, right? It's a dedication by the patient to make sure they spend the time talking with the physician about potential treatments. At the end of the day, I think we help supply the tools and the information for them to have that dialogue. In many cases, you'll see them talking disease state education with things that we provided.

We're also doing everything that we can to help educate patients so they'll go in and ask for it. That information is available in multiple different areas for them to find online and through other channels in DTC and that kind of thing.

Yeah, I was going to ask on DTC, like, do you feel like, you know, I guess have you been ramping that over the last 6 to 12 months? You know, how does that evolve, you know, as you kind of try to break into some of these additional patients, you know, whether it be at those types of practices or new practices?

Yeah. We have, so DTC, remember, is not just about television. Everyone remembers the television, but there's a lot of.

Derek Archila
Senior Biotech Analyst, Wells Fargo

I hear it more on the radio, by the way.

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yes.

Derek Archila
Senior Biotech Analyst, Wells Fargo

I still listen on the radio.

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yes, on the radio too, but there's a lot that happens to make sure patients have access to really understand what their challenge or their disease state is and how to go ask their physician what the next steps might be. We're continuing to use our work in DTC. We know it has impact. We know that we're connected to somebody with Henry Winkler that helps us kind of capture the attention of this patient base, and then they listen and get educated on the things that they need to ask questions about. We're dedicated to it, and it works. We see lots of good feedback that comes out of it.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Maybe just another question in terms of how this market evolves and the competitive landscape. Obviously, we've got some other drugs in the clinic, probably get some data next year. I guess, how do you guys kind of feel about those programs? If they were to show some sort of benefit on vision or BCVA, how does that kind of change the way people might view SYFOVRE?

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Yeah, I think there's a couple of things to put in perspective here. I don't think everyone may understand that it's quite a while before anything else comes, right? Several years, probably. If you take a look at where we started, it took us 20 years to get across the line with the FDA with the first approved GA treatment globally, right? It takes a lot of work. What we're more focused on, quite honestly, is what's that next step for us to be able to treat patients that have GA, which is what Tim talked about, which is our siRNA and our APL-3007 molecule. You can actually treat the level of C3 systemically and bring that level down and then treat patients with an IVT and do kind of a double whammy to make sure that you're helping that patient manage the complement.

That is where we're really focused, on what's next for us, because we think we've got a really good opportunity in that space to continue to be the leaders in GA treatment as well as somebody that can bring new products to this space for patients.

Tim Sullivan
Chief Financial Officer, Apellis Pharmaceuticals

I would also say that it would be a fundamental misinterpretation of understanding GA to think that in a year or a two-year study, you can materially impact BCVA. The idea of how to structure a trial to do that effectively is really hard for us to understand. You know, we have the best slowing of GA lesions of any drug tested so far, and yet we still can't get a BCVA signal that is significant in a two-year timeframe. That would be a high expectation for a GA study. If a GA study is positive in the one to two-year timeframe with a sample size under, I don't know, five or ten thousand for BCVA, then it's probably luck, right? That's what we saw for our competitor products.

For Eiservay, they had a signal in the 15-letter loss for BCVA over the first year of a study, and then in the second year, it disappeared entirely. It was random walk, right? That's because to understand GA, you have to understand that the disease is a slow-growing disease, right? It typically affects the peripheral vision more than it does the central vision, at least initially, and eventually comes into the central vision. Until it crosses into that zone, and you have to get a ton of patients who are in that exact spot to then have a drug that slows 30% or 40% to change that BCVA outcome materially. Also, we as humans have the ability, at least temporarily, to adjust a little bit when something encroaches in the fovea, when the disease encroaches in the fovea.

It's way too noisy to expect that to be a significant thing to understand in a two-year study. From our perspective, lesion slowing is the biomarker you need to look at. What you really should be looking at is that functional vision by using microperimetry, which is what we do, right? That's the measure we use to figure out for each kind of, for each photoreceptor, what it looks like, how well it sees, and so forth, and be able to extrapolate that using AI to understand the functional impact of GA over time in shorter periods of time. You really can't do that with BCVA. I don't think we're worried specifically about BCVA being a material measure of disease for any of these drugs. Also, again, as David said, these are pretty far away.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Gotcha. Maybe just, you know, as we kind of get more experience with these drugs, there's more data, you know, longer-term data. Does that at all, you know, put the EU back on the table at all? Is that or is that completely, you know, just good put for now?

Tim Sullivan
Chief Financial Officer, Apellis Pharmaceuticals

We'll see. We keep generating data. You know, again, BCVA is something everybody anchors to because it's something they understand, right? It's easy to understand, but it is not the correct measure for GA. We're adamant about that. I think we know more, honestly, I think we know more about GA than really anybody because of our studies and particularly our understanding of microperimetry. Four years of data, we have four years of data with people who have microperimetry to understand what happens to their vision over time. Microperimetry, for those of you who don't have the background, is a way of shining light on these points in the retina and understanding what they can see. Over time, as the retina dies off, you can see what happens to each one of these little pixels and understand how well each one of them sees.

Over time, we have hundreds of thousands of these data points where we can now really tell you what's happening on a functional basis in the retina from GA. Just using a central ability to read a chart is way too noisy over that period of time. We're the ones who have those data. I think we can bring that to bear for a general understanding that really isn't there, even among regulatory authorities. We'll see what happens.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Gotcha. Maybe the last couple of minutes, just kind of how we think about Apellis Pharmaceuticals evolving as a company with, obviously, EMPAVELI as a base, SYFOVRE as a base, and then also, kind of future pipeline programs. I mean, you hinted at, obviously, APL-3007. Where do you think the investment goes after getting through some of these launches, in terms of the pipeline? Where do you want to be in, like, five years?

Tim Sullivan
Chief Financial Officer, Apellis Pharmaceuticals

Yeah, I mean, we have dreams of building a great company, right? We're already there in terms of the basics. I think what fundamentally, I don't want to say it's a misunderstanding, but there's, you know, we have history, right, over the past six, seven years as a public company. If you look at us today, you know, we have these two approved products, three approvals, both with exceptional growth opportunity. We have a strong balance sheet, and we are an innovative company. As an integrated biopharmaceutical company that is independent, I think that may be slightly underappreciated that we're in a pretty good spot. There are another two other pieces of this. One is that we are highly differentiated in both of our markets, right? Our C3 glomerulopathy (C3G) and IC-MPGN markets, we have highly, highly differentiated data. I know David has talked about the trifecta.

We have the, you know, double the proteinuria of the competitive product pretty much. We have this C3 staining, which nobody's seen before in any product. Then we have the stabilization in eGFR. That's highly differentiated. It's hard to underscore that enough. In terms of SYFOVRE, we have what we believe is obviously the best lesion slowing. We think we have a highly, highly differentiated product there as well. We are the market leader, and from that perspective, we feel really good about the existing products. What's also probably not super well understood is that targeting C3 the way we do really is the most effective way we believe to target complement in general. While there are hundreds of programs out there, I mean, literally hundreds being developed in complement, we have yet to see one of them outperform the effect of pegcetacoplan in targeting a complement-related disease.

We really have that fundamental technology. I guess the last thing I would say is that we're committed to innovation over time, right? We have SYFOVRE where this is a perfect example, right? We have SYFOVRE where we keep generating data beyond two years, three years, four years. We keep presenting more and more data. We're developing these algorithms to try to understand functional vision. Over time, we're putting, you know, we're developing this siRNA in combination with SYFOVRE. We're not leaving these GA patients behind, right? We're fundamentally committed to these GA patients and to the retina. Similarly, in the kidney, you know, we're just sort of launching there. We have that same innovative mindset as well. We're obviously, as you know, developing EMPAVELI in DGF and FSGS. We're committed to that innovation over time in the key markets where we're focused right now.

Obviously, as we talked about, APL-3007. We have other pipeline products that are emerging over time, which we'll talk about more.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Yeah. Cool. Gentlemen, we'll leave it there. Tim, David, great to see you. Thanks for having us.

David Acheson
Chief Commercial Officer, Apellis Pharmaceuticals

Thank you. Thanks, Derek. Appreciate it.

Derek Archila
Senior Biotech Analyst, Wells Fargo

Awesome, guys. It's good to see you.

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