Apellis Pharmaceuticals, Inc. Q1 FY2021 Earnings Call

Good afternoon. My name is Sarah, and I will be your conference operator today. At this time, I would like to welcome everyone to the Apellis Pharmaceuticals First Quarter 2021 Financial Results Conference Call. Today's call is being recorded and a replay will be available at apellis.com. I would now like to turn the call over to Tracy Vineis, Vice President of Communications at Apellis.

Good afternoon and thank you for joining us today to discuss Apellis' first quarter 2021 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 conference call. It is an exciting time at Apellis with a potential U.S. approval of pegcetacoplan and paroxysmal nocturnal hemoglobinuria or PNH just a couple of weeks away. We are fully prepared for our PDUFA of May 14 and ready to launch pegcetacoplan shortly after approval. Our Chief Commercial Officer Adam Townsend will provide the latest updates on our efforts to ensure a successful launch in PNH. As this slide shows, we are focused on advancing our global leadership in complementary therapies and our potential approval in PNH is just the start of what will be a transformational year for Apellis. With PNH as our lead indication, we aim to establish systemic pegcetacoplan as our targeted C3 therapy and a disruptive treatment across rare, complement-driven diseases. We plan to become number one in the retina with the first treatment for geographic atrophy, and we continue to advance innovative technologies to control complement with a focus on complement factor C3. Last month we were thrilled to see results from our programs in PNH and geographic atrophy, or GA published in three leading medical journals. In PNH, results from our phase three PEGASUS study were published in the prestigious New England Journal of Medicine. The recognition by any major journal underscores the importance of the PEGASUS data to the PNH community and the high caliber of science that we are advancing at Apellis. Additionally, two separate analyses of our affiliate study in GA were published in the American Journal of Ophthalmology and in ophthalmology. Our Chief Medical Officer Dr. Federico Grossi will provide additional details later. But together these publications showcase the broad platform potential of our targeted C3 therapy for complement-driven diseases. The GA publications also reinforce our confidence in our upcoming phase three readouts which we continue to expect in the third quarter of this year. Topline results from our DERBY and OAKS studies will be an important event for Apellis and for the more than 5 million people worldwide living with this relentless and disabling disease.

Thank you, Cedric. I will first begin with PNH, the program that serves as the foundation of a rare disease. We're all looking forward to our PDUFA day and remain on track for May 14. The positive phase three PEGASUS results demonstrated the potential effects of pegcetacoplan to elevate the standard of care in PNH, and the recent publication by the New England Journal of Medicine reinforced the significance of these results for the medical community and patients with PNH. We hope to build on the strong clinical profile of pegcetacoplan leading to the upcoming results from the PRINCE study. As shown on the slide, our phase 3 PRINCE study in treatment-naïve PNH patients is designed to evaluate two primary endpoints at week 26: hemoglobin stabilization in the absence of transfusions and reductions in LDH levels. These co-primary endpoints were intentionally aligned with those used in previous PNH studies of C5 inhibitors and therefore focus on intravascular hemolysis analysis. However, since PNH is characterized by both intra and extra vascular hemolysis, we believe that certain secondary endpoints including hemoglobin levels, transfusions, and FACIT-fatigue scores are critical measures of the overall impact this disease has on patients. I would also like to remind you that we set a high bar for pegcetacoplan on the primary endpoint for hemoglobin stabilization compared to previous studies of C5 inhibitors. In PRINCE, patients are considered to have unstable hemoglobin levels if those levels dropped by only one gram per deciliter, whereas studies of C5 inhibitors historically allowed hemoglobin levels to drop by two grams per deciliter before being considered unstable.

Thank you, Federico. Our commercial organization is excited and ready for our May PDUFA date, and we are laser-focused on ensuring a successful U.S. launch of pegcetacoplan in PNH. Our market research continues to reinforce the urgent need for new treatments for PNH. As you can see on this slide, people with PNH continue to suffer from significant unmet needs despite their current treatment with C5 inhibitors like Soliris and Ultomiris. Retrospective studies show that one-third of patients on C5 inhibitors continue to require transfusions to address falling hemoglobin levels. Another third of these patients remain anemic and experience other symptoms like severe fatigue. The final third of patients on C5 inhibitors have closer to normal hemoglobin levels, but many only achieve that at the expense of maximum output of red blood cells from their bone marrow. Thus, we believe there is a significant need for pegcetacoplan to elevate the standard of care in PNH, and our commercial team is ready to address the needs of people living with PNH at launch. Our latest launch preparedness activities are outlined on this slide. Our value and access team is engaging with high-priority payers representing more than 80% of all U.S. PNH patients. This includes completing more than 50 unique payer interactions during which we received positive feedback on the clinical efficacy and safety profile of pegcetacoplan. On average, we anticipate that reimbursement decisions to be made approximately three to six months post-launch. We will work hard to ensure that any patient who chooses pegcetacoplan will have access to it from day one of our launch. We've established our distribution model as well as our patient support resources and programs. This includes the Apellis Assist program designed to provide a comprehensive support system for patients throughout their treatment journey, including dedicated Apellis care educators who will help train patients on self-administering pegcetacoplan. Apellis Assist will help ensure a high-quality treatment experience and provide infusion training, continuing education on the treatment of PNH, and ongoing support with copay assistance where eligibility criteria are met. As we continue to navigate through COVID-19, our teams, both commercial and medical affairs, are closely monitoring regional COVID-19 restrictions. In areas where we can have in-person engagements, we have field teams meeting healthcare professionals, or HCPs, in their offices, and we will remain diligent in following all appropriate guidelines.

Thank you, Adam. Beyond PNH, we are advancing our registrational programs of systemic pegcetacoplan, with programs focused on complement-driven diseases where patients have few or no payment options and where we believe targeting C3 has the potential to offer a differentiated product profile. The upcoming milestones are outlined on this slide, including several study initiations in the second half of this year. In rare diseases, we continue to execute on our phase three studies of intravitreal pegcetacoplan in GA, with top-line results expected in the third quarter. We're excited about the potential effects of pegcetacoplan to become the first treatment for people with GA, and recent publications in two leading journals reinforced the clinical profile of our targeted C3 therapy. Last month, publications in the American Journal of Ophthalmology and ophthalmology highlighted post-hoc analysis of a positive phase 2 study of pegcetacoplan in GA. The AJO publication shows that pegcetacoplan reduced lesion growth similarly across patient subgroups, and the results remain significant even when accounting for risk factors associated with more rapid progression. In the ophthalmology publication, the authors characterized the explanations reported in the filing and found that investigator-determined accommodations were responsive to standard anti-VEGF therapy and did not have a clinically meaningful impact on vision. The analysis also found that certain adverse events were more common in patients with a history of wet AMD in the eye. This observation is consistent with real-world clinical data mined from the AO Iris registry in our ongoing collaboration with Verona Health.

Thank you, Federico. Since we issued a press release earlier today with our full financial results, I will just focus on the highlights for the first quarter of 2021. As of March 31, 2021, Apellis had $723.7 million in cash, cash equivalents, and short-term marketable securities. Research and Development expenses were $84 million for the first quarter of 2021 compared to $69.3 million for the same period in 2020. The increase in R&D expense was primarily attributable to costs associated with ongoing and planned clinical trials, compensation related personnel costs primarily due to the hiring of additional personnel, among others.

Thank you, Tim. We've made strong progress this quarter and have several important commercial, clinical, and regulatory milestones anticipated over the next several months, as shown on this slide. With PNH just over two weeks away, we would like to take this opportunity to recognize and thank the patients, investigators, caregivers, partners, employees, and investors, who have helped us reach these pivotal moments. We look forward to keeping you updated on our progress. And now operator, please open the call for questions.

Thank you. First question comes from the line of Anupam Rama with JPMorgan. Your line is now open.

I was wondering if I could ask a question that I've been getting a lot recently which is related to the phase three geographic atrophy studies and how you're thinking about presenting some of the top-line data as it relates to exudates, whether it's the rate of oxidation in the different arms or the portion of patients getting a VEGF treatment or the rate of CNV and how we should be thinking about this and what are the most important metrics on this topic related from your market research from physicians?

Thank you so much, Anupam, and great hearing your voice. So that is indeed a question that we get quite often. I think the first element to mention is that we will report these exudates as physician-reported exudates. So in that sense, very similar to how it was done in the phase 2 clinical trial, with the caveat that, as we have discussed many times before, the phase 3 clinical trial has better controls to compensate for potential investigator bias. And with the knowledge that in the phase 3 clinical trial, the number of patients that came into the DERBY and OAKS studies demographically were much less represented by patients who started off the study with wet-AMD in one eye already at baseline. This is important because it is well known that patients with wet-AMD in one eye have quite a high rate of exudation development based on a large study that we conducted, with an extent of approximately 21% over the course of two years. I will also briefly hand it to Adam to give brief feedback on what we have heard from physicians in terms of adoption.

Thanks, Cedric, and thanks Anupam. So in our market research with GA physicians, be they injecting ophthalmologists or retina specialists, we find that some of the important impacts that resonate very well with them start with the ability to slow lesion growth. That's the number one piece that resonates very strongly. Based on our market research from the FILLY data, we found that a clinically meaningful average based on lesion size showed a reduction of about 20% to 30%, which we get consistently from KOLs, retina specialists, and ophthalmologists. So lesion size, lesion growth, and the ability to replicate some of the FILLY data resonate strongly with our prescriber base. They also are very thoughtful around safety, dosing, route of administration, and mechanism. One thing that is consistent is there's an emotional burden for these physicians who currently can't help these patients as much as they would like. So we're excited to see the data towards the end of the year, and we hope to have a big impact on GA patients moving forward.

And then, if I could just squeeze one more quick question in? From a high level, could you give us some perspective on your regulatory interactions here going into the PNH approval in terms of any concerns you have right now about the PDUFA being pushed out?

Yes. Thank you so much, Anupam. We get a lot of questions on that as well. We are within two weeks of our PDUFA date, so we are not commenting on regulatory questions at this point in time. It is public knowledge that we went through the whole process without any major findings, and we are hopeful that in the middle of this month we will have something good to announce.

Thanks so much for taking questions, guys.

Thank you.

Thank you. Our next question comes from the line of Jonathan Miller with Evercore ISI. Your line is now open.

Hi guys. Thanks so much for taking the question. I have two; one on PNH launch and then a follow-up on GA. How are reimbursement discussions going for the PNH launch at this point? You mentioned three to six months for commercial payers getting through the reimbursement process. Can you talk us through how you expect that payer ramp up to happen and what percentage of those patients that you're talking about are commercial versus government and what's the launch up for government payers as well?

All right. I'm going to hand that one over to Mr. Townsend. Adam?

Yes. Thank you. Thank you, Jonathan, for the question. So firstly, our value and access team is fully staffed and has been engaging with high-priority payers for the last couple of months, representing more than 80% of all U.S. PNH lives. One thing that stands out from our payer interactions is that they've been overwhelmingly positive. We expect broad coverage and we don't see any major issues with reimbursement. It does take, on average, between three and six months post-launch for us to work with those payers and get through their internal processes. As for the second part of your question, 50% of the patients that we expect will have commercial private insurance, and the other 50% are governed under Medicare and Medicaid with some variations. We expect that for the Medicare plans, they typically take up to about 180 days, and the Medicaid plans take up to about six months. As I said before, the commercial plans usually take three to six months. Our team is really impressive in how they've been interacting with the payers. The value story of pegcetacoplan is very strong, so we believe that we'll be able to smoothly move through those discussions with U.S.-based payers, and we're prioritizing access so any patient that wants to come onto our drug will be given access, even as we work through that payer landscape as I've described.

Great. That makes sense. I guess then a follow-up on GA. I noticed that you highlighted some efficacy updates from the phase 1 safety study but it looks to me like the efficacy delta we're seeing there has plateaued a little bit versus the last update. Obviously still a very impressive headline number, but should we expect this to be the general observation with pegcetacoplan and GA? Should we expect there to be this plateau at around that 50% level or could that delta continue to expand as doses increase?

Yes, thank you so much, Jonathan. Again, that is a great question. Of course, by the mechanism in which pegcetacoplan works in geographic atrophy, we look forward to the readout from one year to two years. What we disclosed on that phase 1b study is a very small sample set of patients. It becomes very interesting when you start looking on an individual patient level even but we are going to need results from the larger studies to make a determination in that regard.

All right. Very nice, thanks a lot.

Thank you, John.

Thank you. Our next question comes from the line of Madhu Kumar with Goldman Sachs. Your line is now open.

Hi guys, thanks for taking our questions. So I'll start with the PRINCE trial. How should we think about the PRINCE trial readout and what kind of impact it has on your PNH strategy and kind of how contingent is that on what happens with the PDUFA date?

Thank you so much for that question, Madhu. So the PRINCE trial, as we've mentioned many times before, is a phase three clinical trial in treatment-naive patients with PNH that was designed to serve as an insurance policy. When we did the PEGASUS study, which was the phase three clinical trial on which our approval is currently based, that study aimed to do three things in one. First, it aimed to show that systemic pegcetacoplan was a treatment for PNH. Second, to show that it would be superior to Soliris, and third, to show that you could safely switch between the two drugs in either direction. Because that was a lot to handle for one trial, we didn't know the outcome in advance. We wanted PRINCE to be there in case things did not go according to plan. As it turns out, the PEGASUS study gave us everything we had hoped for and then some. PRINCE is now here to give us a snapshot into patients with PNH from a much more uniform background, rather than patients with PNH on treatment with Soliris that have what you could call sub-optimal responses. What we expect to see in PRINCE is, as we've seen in all of our studies so far, good control of PNH, measured in this case by the control of lactate dehydrogenase and stabilization of hemoglobin levels.

Okay. Great. So I'll follow up with a question you're probably not going to be able to answer, but I've got to try it. It's two questions. One, make the case for approval of pegcetacoplan outside of post-Soliris treatment in PNH based on the data you have so far and then, to what extent do you think there is a case for approval broadly in PNH given the design of PEGASUS?

So as you correctly assumed, we cannot comment on label discussions at this point in time, of course, since we are only two weeks away from the PDUFA. We have commented in the past that we believe that in the PEGASUS study we showed good control of PNH in a long period of treatment-naive patients, I should say, with monotherapy using pegcetacoplan.

Okay. I'll have one go at a GA question. So how do you think about dose frequency for GA and kind of the similarities and differences to the treatment of geographic atrophy compared to the treatment of wet AMD?

Thank you so much for that question, Madhu. So that is a nuanced question, as the two are very different manifestations of likely a similar underlying disease process. We've done a lot of work as it relates to understanding physician and patient receptivity to these two regimens. I will hand the word to Adam in a minute to elaborate on that, but the bottom line is that currently there is no treatment for geographic atrophy, which is a debilitating and blinding disease affecting 5 million patients worldwide. It is a disease that, when treated, will not have immediate feedback for the physician, unlike an anti-VEGF treatment where the exudates in the retina go away. This is a treatment that will rely more on the physician and the patient sticking to the prescribed regimen as we establish it, hopefully in our phase three clinical trials. We are also working on an artificial intelligence background, and you will hear much more about that at the conference as well to predict patients with GA and what the natural rate of photoreceptor cell loss is in these patients, and what a drug could do to slow that down. Adam, do you want to add something regarding the monthly versus every other month dosing?

Yes. Thanks, Cedric. Thanks, Madhu. So yes, obviously the one thing that drives excitement is that we have the chance of being the first approvable product in GA. We get some great responses from retina specialists when we speak to them regarding dosing, and they share similar themes. We find that physicians believe that monthly dosing is acceptable. They are motivated by their wet-AMD patients and can envision GA patients being equally driven. Additionally, we receive comments regarding infrastructure, with some saying they have the capability to inject patients every month. So I think the excitement about having a treatment for GA gives us great options as we progress, and I look forward to seeing the data towards the end of this year.

All right. Great. Thanks for taking our questions. I guess we'll hear from you all in a couple of weeks.

Yes. Thank you, Madhu.

Thank you. Our next question comes from the line of Yigal Nochomovitz with Citigroup. Your line is now open.

Hi Cedric and team, thank you very much for taking the questions. I had two on GA. Regarding DERBY and OAKS, if you get very good news in the third quarter and both the monthly and every other month end up being effective – what will be the commercial plan? Will you just move forward with the every other month since that's a lower treatment burden, or is there any reason why you would also want to market the monthly regimen? My second question is if you could just explain the reasons why you're confident that pegcetacoplan isn't causing the exudation?

Thank you so much, Yigal. Again, two very good questions. On geographic atrophy, I will hand it to Adam but it is worth mentioning that based on everything we have seen, I think it's reasonable to expect a dose response if you want to call it that. In other words, the efficacy for a monthly dose is unlikely to be identical to every other month. So from that starting premise, that efficacy advantage would be something to consider as it relates to commercialization. Adam, do you want to add something to that?

No, I think you said it very well. I think both will be important for us moving forward.

Thank you, Adam. And then as it relates to the pegylation, I think it is very important to point out that the few publications that exist which have looked at polyethylene glycol as an agent that can promote exudations in certain models are highly artificial models. These are not animal models of macular degeneration. One example involves laser-induced ablation. Essentially, you shine a laser in the retina and induce a type of wound that is driven by digestion, where polyethylene glycol may affect wound healing. This is very different from what we see in macular degeneration, and we do not believe that the polyethylene glycol that is part of pegcetacoplan affects the exudates in our patients.

Got it. Thank you, Cedric.

Thank you, Yigal.

Thank you. Our next question comes from the line of Alethia Young with Cantor Fitzgerald. Your line is now open.

Okay. Thanks for taking my questions too. Can you talk about the pegcetacoplan market and how you envision segmenting it, practically speaking, just in light of the study that you're running? And could you also characterize any notable differences in conversations with the EMA versus the U.S. on pegcetacoplan?

Thank you so much, Alethia. I will give the first part of that question to Adam.

Thanks, Alethia. So yes, segmenting the GA market obviously reflects sizable patient demand. One thing we found that's consistent is we expect the market to be segmented a little bit by GA size and location. Clinical features that resonate when you speak to physicians about treatment are how central or non-central the lesion is and how big the lesion is—small, medium, or large. And you’ll find that when you apply some patient parameters to that segmentation, it seems to be consistent as well. They also consider whether the patients have bilateral GA, etc., and whether they have a history of wet AMD. So lesion size, location, and patient demographics is the way we envisage segmentation occurring within the GA market.

Thank you, Adam. As it relates to your second question, Alethia, regarding European commercialization and regulatory pathways on systemic pegcetacoplan, and PNH and other indications, I would ask you to speak directly with Sobi. What I can tell you from our perspective is that this has been a fantastic partnership with no change in guidance as we set out a couple of months ago.

Yes, I figured it was worth a try. Thanks.

Thank you.

Thank you. Our next question comes from the line of Philip Nadeau with Cowen & Company. Your line is now open.

On DERBY and OAKS, in the past you've been very good about giving updates on missed visits as COVID has waxed and waned. With the trials drawing toward close, I would be curious if you have any update on missed visits today. Are they still within the tolerances that you set out when you designed the trial and any update on your discussions with FDA over how missed visits and mis-measures are going to be handled statistically in the study?

Yes. Thank you so much, Phil. As you correctly mentioned, of course, this has been top of mind for us since the spring of last year. Our guidance has not changed. Ever since the spring of last year, when I’d say the trial came under control, even with the subsequent waves, etc., the patient visits and the injections which we track on a weekly basis are where we wanted them to be. We have had regulatory interactions with the FDA and we know, without providing any specific comments, we feel that both trials are well powered to meet our primary endpoint in the third quarter.

I believe in your analyst meeting you discussed the change to the statistics or how they're going to be handled with missed visits. Am I mischaracterizing that or are the statistics the same as initially designed? Has there been some sort of adjustment made for missed visits?

Yes, we have not changed the statistical analysis plan. Based on the control of the trial, we feel very good about the readout based on the existing sets.

Perfect. Thank you. And then second question on PNH. I guess, what are your most recent thoughts on pricing? What type of price would open up access the best or the most and when will you disclose the price? Do you think you'll be in a position to disclose the exact price at the time of approval or would you wait until the launch actually begins in earnest?

Thank you, Phil. I will hand that one over to Adam.

Thanks for the question, Phil. Yes, we're committed to ensuring that every patient who wants pegcetacoplan can access it, regardless of their ability to pay. We've made sure that patient access is at the core of our pricing strategy. Our pricing strategy involves benchmarking the clinical value that we believe we can deliver to PNH patients and we compared ourselves to the standard of care in PNH at the moment, namely Soliris and Ultomiris, as well as some other similar rare disease drugs. As we get closer to our potential approval, we'll finalize our pricing terms, and then post-approval, we'll start to talk about the value we believe we can have for PNH patients. So more to come.

Perfect. Thanks for taking my questions.

Thank you so much, Phil.

Thank you. Our next question comes from the line of Steve Seedhouse with Raymond James. Your line is now open.

Hi, this is a question for Steve Seedhouse. So thank you for taking my question. We have a question about PEGASUS. Do you think PEGASUS alone provides enough data to prove that you have a robust effect on intravascular hemolysis since the primary endpoint was a change in hemoglobin? And then relatedly, I am not sure if you can answer this, but has the FDA asked you to quantify the contribution to intra versus extravascular hemolysis pegcetacoplan brings?

Thank you so much for those questions. We are not commenting on the regulatory process at this point in time since we are only two weeks away from our PDUFA. However, PEGASUS as a study was and is the only study included in this filing, which went through a proper pre-FDA meeting and proper evaluations. So the data contained in the PEGASUS study is deemed sufficient to evaluate the efficacy of pegcetacoplan in PNH.

Okay. Thank you very much.

Yes. Thank you.

Thank you. Our next question comes from the line of Justin Kim with Oppenheimer. Your line is open.

Hi, good afternoon and thanks for taking the question. Maybe touched on this a little bit, but as we approach near-term, could you discuss how these tools and findings may influence your thinking on potential commercial use of pegcetacoplan and maybe even help enhance development approaches for next-generation compounds in AMD?

Thank you, Justin. That is an excellent question. This is work we're really very excited about. We have a lot of data, as you know, but specifically the work around artificial intelligence is very important. Ultimately, in the context of long-range planning, being able to predict the natural rate of progression of GA in patients and what a patient and physician could reasonably expect the drug to do, obviously sets up an interesting framework for future pricing strategies. Adam, would you like to add to that?

Yes, you articulated that nicely, Cedric.

Thank you, Adam. Another piece to bear in mind is that if you tell a patient with geographic atrophy that we are going to give you, for example, a monthly injection with pegcetacoplan, I suspect there will be complaints in the first year. But over time, if patients forget their injections or are on holiday, they won't see a difference. Thus, retaining these patients in the long run while providing them with good treatment will require something like this.

Got it. Great. And maybe just one question on the phase three MPGN C3 program. Can you just discuss what steps remain, if any, before initiating the study? Are any interactions with regulatory agencies required?

Yes. Thank you for that question as well. This trial requires close interaction with our partner Sobi, and the guidance on the start of the phase three trial has not changed. It is set to begin in the second half of this year, and we are excited to explore systemic pegcetacoplan in those conditions.

Great. Thanks very much.

Thank you, Justin.

Thank you. Our next question comes from the line of Matthew Luchini with BMO Capital Market. Your line is open.

Thank you. Good afternoon. One question for Adam in the context of geographic atrophy patient segmentation: Can you talk about physician enthusiasm for treating earlier stage patients with the disease? I'm thinking in light of the EU retina data from last year?

Thank you so much, Matthew. Adam, would you like to address that?

Yes. Thank you. Based on the segmentation discussed, we actually find that there is strong motivation driven often by the patient and supported by the retina specialists for earlier use than we perhaps anticipated. While looking at our segmentation, if you examine the lesion size being small and non-central versus central and small, you'll find that physicians are motivated based on what we've seen in FILLY, and I believe they will be even more motivated based on the new data that we're publishing. There is a good groundswell for wanting to use the treatment earlier to meet some unmet needs in GA.

Great, thanks. One more question: Assuming geographic atrophy is successful, pegcetacoplan could be a fairly large drug. What’s your approach towards pricing and your views on evolving pricing dynamics coming out of Washington since Part B in particular?

Adam, would you like to take that?

Sure. You're correct in noting that we expect about 95% of GA patients to be on Medicare. We have conducted initial pricing work and value discussions primarily informed by the FILLY data. It's super early for us, but we have noticed that in discussions with payers, physicians, and experts outside the U.S., they tend to gravitate toward standard treatment pricing as their initial point of reference. Our rationale remains centered on prioritizing patient access. If our drug proves as effective as hoped, we want to ensure those patients can access it, and we will communicate the drug's value to GA patients. So additional updates will be provided, but your assessment of the access market is spot on.

All right, thank you very much.

Thank you, Matthew.

Thank you. Our next question comes from the line of Joseph Stringer with Needham & Company. Your line is now open.

Thanks for taking our questions. I'll try another one on a potential PNH label. Could you help us understand what a most favorable scenario would be and maybe a least favorable type of label? If you can't speak to specifics, could you provide some context on how those two scenarios compare in terms of potential patient numbers?

Yes. Thank you so much, Joe. Again, we're not commenting on the label at this point in time since we are only two weeks away from our PDUFA.

Okay.

We will be very happy to speak about that.

Thank you. Our final question comes from the line of another unidentified analyst. Your line is now open.

Hi, thanks so much for taking the question this afternoon. Starting with PNH, Adam, could you comment on how early education is going for the subcutaneous administration? What feedback are you getting on ease of use and how much education do you think patients will need following approval?

Yes. Thank you for your question. We're preparing to launch with a robust patient services model and have been interacting with PNH patients for the last couple of months to understand what support they need when it comes to administration. Our aim is to ensure that we can educate people about the unmet need in PNH treatment and provide any support they might need through our Apellis care educators. We've found that patients have responded positively to the opportunity for home treatment. The COVID scenario has amplified this, and we believe we can support patients well with both face-to-face and virtual interactions.

Great. Thanks. And then on DERBY and OAKS just procedurally: Will all of those patients remain randomized and blinded out to 24 months or will the study be unblinded at the 12-month readout? I was just wondering how the recent phase 1b data continues to strengthen over up to 24 months and wondering if you'd be able to see that play out in the phase 3 data.

Thank you for that question. Patients will stay randomized. Federico, do you want to add something to that?

Yes. That is the simple answer. The study will continue to be double-masked, and patients will remain on the randomized schedule all the way to 24 months.

Great. Thanks.

Thank you. Our next question comes from the line of Laura Chico with Wedbush Securities. Your line is now open.

Thanks very much for taking the question. Adam, I thought the commentary around launch prep was helpful. What is the size of the expanded access program at present and what proportion of those patients are within the U.S.?

Thanks, Laura. I'll be handing the expanded access program over to Dr. Federico Grossi.

We opened our expanded access program in the U.S. to help patients with higher medical need receive pegcetacoplan while the application is being reviewed by the FDA. We don't expect the FDA program to affect our commercial metrics or revenue, and as we are close to PDUFA, we're not commenting further on our early access program.

Okay, that's helpful. Maybe one other question: The latest 10-Q continues to point towards cash runway into the second half of 22. Could you elaborate on the levers that could either contract or extend that runway, especially against the backdrop of DERBY and OAKS, with the primary endpoint reading out in the third quarter of 21?

Thank you, Laura. Tim, do you want to take that?

Sure. Thanks, Laura. That's a great question because the calculation around cash runway contemplates a geographic atrophy success scenario. We’ve built up our global launch capabilities and moved forward in other studies related to pegcetacoplan. The calculation will be very different if GA doesn't succeed, and our cash runway would extend beyond that. Beyond that, typical factors relate to how we perform in our commercial launch in PNH.

Thanks, guys.

Thank you so much, Laura. Well, thank you all for joining us on our first quarter conference call. We look forward to our May 14th PDUFA date in PNH and we are excited about the transformational year ahead for Apellis as we continue to build our global leadership in complement-driven therapies. Thank you again for joining us today.

Ladies and gentlemen, this concludes today's conference call. Thank you for your participation. You may now disconnect.