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 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 RFP 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 incontinence and our potential approval in PNH is just the start to what will be a transformational year for Apellis.

With PNH as our lead indication we aim to establish systemic pegcetacoplan on our targeted C3 therapy as 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 GM 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 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 complements-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 result 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 since the date with the upcoming results from the PRINCR study. As shown on the slide or phase 3 PRINCR study in treatment-naïve PNH patients is assigned to evaluate two primary endpoints at weeks 26 hemoglobin stabilization in the absence of transmissions 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 analysis.

However, since PNH is characterized by both intra and extra vascular analysis, we believe that certain secondary endpoints including hemoglobin levels transfusions and FACIT-fatigue score 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 sterilization 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 where 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 1/3 of patients on C5 inhibitors continue to require transfusions to address that 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 make sure 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 a Apellis Assist, program design 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 team both commercial and medical affairs is 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 obviously remain diligent in following all appropriate guidelines..

Thank you Adam. Beyond PNH, we are advancing for registrational programs of systemic pegcetacoplan with a programs are focused on component 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 intravital 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 meeting of journals reinforced the clinical profile of a targeted C3 therapy.

Last month publications in the American Medical Journal of Ophthalmology or AJO and ophthalmology highlighted post hoc analysis of a positive phase 2 study of pegcetacoplan in GA.

The AJO publication show 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 author's characterize the explanations reported in feeling and found that investigator determined accommodations were responsive to standard anti-VDF therapy and did not have a clinical meaningful impact on vision. The analysis also found that 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.

for PNH just over two weeks away we would like to take this opportunity to recognize and thanks to patients, investigators, caregivers, partners, employees, and investors, who have helped us, get to 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..

Question.

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 there is that we will report these exudations as physician-reported exudations.

So in that sense very similar to how it was done in the phase 2 clinical trial with the caveat as we have discussed many times before that the phase 3 clinical trial is better controls to compensate for potential investigator bias, etc.

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 and contra that rely because when we did the city study, the phase two study we were competing with another large phase three program that was excluding that particular type of patients and that matters because it is well known that patients with wet-AMD in one eye have quite a high rate of exudation development with wet-AMD development in the GA based on a large study that we did to the 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 and retina specialists we find that some of the important impacts that resonate very well with them starts with the ability to slow lesion growth.

That's the number one piece that resonates very strongly and then if you use the parameters that we saw within our FILLY study we found that a clinically meaningful average based on our market research of lesion, less lesion growth is about 20% to 30% and we get that consistently through KOLs retina specialist and ophthalmologists.

So lesion size, lesion growth and the ability to replicate some of the FILLY data very strongly with our prescriber base. They also are very thoughtful around safety, dosing, roof administration and mechanism. One thing that is consistent is there's a emotional burden for these physicians who currently can't help these patients as much as they can.

So we're excited to see the data towards the end of the year and we hope to really-really 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 maybe you could give us some perspective on your regulatory interactions here going into the PNH approval in terms of I guess there's a concern right now on PDUFA is getting pushed out given kind of the -.

Yes. Thank you so much and of course 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 the middle of this month 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. Two; one on PNH launch and then a follow-up on GA I guess. How are reimbursement discussions going for 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 the high priority payers for the last couple of months and those payers are representing of more than 80% of all U.S. PNH lives. One thing that stands out from our peer interactions is they've been hugely 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 and a quick side step to 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 slight error bars around the edges.

We expect that on the Medicare plans they typically take up to about 180 days and the Medicaid plans up to about six months and as I said before the commercial plan is three to six months. Our team is really-really impressive in how they've been in interacting with the payers. The value story of pegcetacoplan is very-very strong.

So we believe that we'll be able to smoothly move through those discussions with the 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 of that efficacy update you gave 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 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 plateaued at around that 50% level or could that delta continue to expand as dose increases?.

Yes, thank you so much, Tom. Again that is a great question. Of course by the way or by the mechanism I should say 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 the 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 very much designed to be there as an insurance policy because when we did the PEGASUS study which was the phase three clinical trial on which our approval is currently based that was a study that aimed to do three things in one.

First of all to show that systemic pegcetacoplan was a treatment for PNH. Second to show that it would be superior to Soliris and number three to show that you could safely switch between the two drugs in either direction and because that was a lot to handle for one trial we, of course, didn't know the outcome in advance.

We wanted PRINCE to be there in case things would not go according to plan. Well, as it turns out the PEGASUS study gave us everything we had hoped for and then some. PRINCE there is now to really give us a snapshot into patients with PNH that are from a much more uniform background.

So not patients with PNH on treatment with Soliris that have what you could call sub-optimal responses but more representative of the broad population and what we expect to see in PRINCE is as we've seen in all of our studies so far good control of PNH as measured in this case by the measurement of lactate dehydrogenase control and stabilization of the hemoglobin levels..

Okay. Great. So I'll follow up with a question you're probably not going to be able to answer but I got to try but it's like 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 kind of design of PEGASUS and kind of all together?.

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 I should say monotherapy with 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, the two are very different manifestations of probably a similar underlying disease process.

We've done a lot of work as it relates to understanding I'd say physician and patient receptivity to these two regimens and I will hand the word to Adam in a minute as it relates to 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 in the world and it is a disease that when it will be treated is will not have I'll call it the kind of immediate feedback to the physician as you have with an anti-VEGF treatment the exudates in the retina go away.

So this is a treatment that will much more rely on the physician and the patients sticking to the prescribed regimen as we will establish it hopefully in our phase three clinical trials and where we are working on an artificial intelligence, background you will see much more about that at the conference as well to be able to predict in patients with GA what is going to be the natural rate of photoreceptor cell loss in these particular patients and what could a drug do in order to slow that down.

So I don't know Adam if you want to add something as it relates to monthly versus every other month..

Yes. Thanks Cedric. Thanks Madhu. So yes so obviously the one thing that drives excitement is that we have the chance of being the first approvable product in GA and apparently no treatment.

So we get some great responses from retina specialists when we speak to them around dosing and they're along these type of themes, so it's we get the I think monthly dosing is fine.

My wet-AMD patients are motivated and I can see GA patients being equally driven and we also get infrastructure comments like we have the infrastructure to inject patients every month.

So I think the excitement about a current treatment gives us great options as we progress and I'm just looking forward to seeing the data come out towards the end of this year..

All right. Great. Thanks for taking our questions and I guess we'll hear from you all in a couple 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 -- 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? And then my second question was if you could just explain the reasons why you're confident that the pegulation isn't causing the exudation? Thank you..

Thank you so much Yigal. Again two very good questions. On geographic atrophy I will again 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 that. In other words the efficacy for a monthly is unlikely to be identical to every other month.

So from that starting premise that efficacy advantage would be something to take into account as it relates to commercialization.

Adam I don't know if you want to add something to that?.

No, I think you said it very well. I think they're both important for us moving forward. .

Thank you Adam and then as it relates to the pegulation. So I think it is very important to point out that kind of the few publications that are out there that have looked at polyethylene glycol as an agent that can promote I'll call it exudations in certain models are highly artificial models. So these are not animal models of macular degeneration.

I'll give one example where they do a laser-induced ablation. So essentially you shine a laser in the retina and you induce a type of wounds that is going to be very much driven by digest and where polyethylene glycol may have an effect on how that wound healing occurs.

That is of course very different from what we see in macular degeneration and we do not believe that the polyethylene glycol that is part of the pegcetacoplan has an effect on 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 just talk obviously big market, but can you just talk a little bit about pegcetacoplan and how they think about segmenting it practically speaking just in light of the study that you're running and then just as it relates to the U.S.

and Europe as it relates to pegcetacoplan can you characterize like if there have been any kind of notable differences in like the EMA conversation versus the U.S.? Thanks..

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

Thanks Alethia. So yes segmenting the GA market obviously a very sizable patient demand there. So one thing we found that's been consistent is we expect the market to be segmented a little bit by GA size and location.

So some of the clinical features that naturally resonate when you speak to physicians about treatment is the lesion central or nonsensual and how big is the lesion small, medium, or large and then you find that you apply some patient parameters to that segmentation and that seems to be consistent.

They also take into account bilateral GA, etc and patients with wet-AMD. So lesion size and location and patient demographic is the way that we can see the segmentation occurring within the GA market..

Thank you, Adam and then as it relates to your second question Alethia.

So for questions related to the European commercialization and the regulatory path on systemic pegcetacoplan and PNH and the other indications, I would ask you to speak with Sobi, directly what I can tell you from our perspective is that this has been a fantastic partnership with no change in guidance as we set it 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 DEBY and OAKS in the past you've been very good about giving updates on missed visits as COVID as waxed and waned. With the trials drawing toward close I'd be curious if you have any update on unmissed visits today.

Are they still within the tolerances that you set out when you design the trial and any update on your discussions with FDA over how mis-visits, mis-measures are going to be handled statistically in the study..

Yes. Thank you so much Phil. So as you correctly mentioned of course this is something that has been top of mind for us since the spring of last year of course and our guidance has not changed. So ever since the spring of last year when I'd say that 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 make us meet our primary end point in this third quarter. .

I believe in your analyst meeting you discussed the change to the statistics or how they're going to be handled with mis-visits.

Am I mischaracterizing that or are the statistics the same as initially designed, has there been some sort of adjustment made for mis-visits?.

Yes we have not changed the statistical analysis plan and again 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? Thanks..

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

Thanks for the question, Phil. So yes we're committed as I said to ensuring that every patient who wants pegcetacoplan can have access regardless of their ability to pay. So we've made sure that that patient access was the core of our pricing strategy and all of our work.

So our pricing strategy involved benchmarking the clinical value that we believe that we can deliver to PNH patients and we compared ourselves to the standard of care in PNH at the moment. So 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 in terms of our label and then post approval we'll start to talk about the value that we believe that 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. .

Yes. Hi this is for Steve Seedhouse. So thank you for taking your question and so 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 and not sure if you can answer this but has the FDA asked you to quantify the contribution to intro versus extravascular hemolysis pegcetacoplan brings?.

Thank you so much for those questions and nice to hear you. So we are not commenting on the regulatory process at this point in time again since we are only two weeks away from PDUFA but PEGASUS as a study has been and is the only study included in this filing which went through a proper pre-FDA meeting and the proper evaluations.

So the data contained in the PEGASUS study is deemed sufficient to evaluate the efficacy of pegcetacoplan and 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 with approaching near-term can you just discuss a little bit about how these tools and findings may influence your thinking on potential commercial use of pegcetacoplan and maybe even help enhance the development approach for next generation compounds in AMD..

Thank you Justin. That is an excellent question. So this is work that we're really very excited about.

I mean, we have a lot of as you know but specifically the work around the artificial intelligence is very important and I think ultimately in the long range planning context being able to predict in the patients what the natural rate of progression of GA is going to be and what a patient and a physician could reasonably expect the drug to do obviously sets up an interesting framework for future pricing strategies.

Adam I'm going to hand it over to you if you want to comment beyond that..

Yes you said it very nicely Cedric..

Thank you Adam and I think yes this is really something that we're very excited about.

I think another piece to bear in mind here is that if you tell a patient with geographic atrophy we are going to give you for example a monthly injection with pegcetacoplan traditionally I'm sure that in the first year there will be good complaints but at some point when you forget to have an injection or you're on holiday you won't see a difference.

So to be able to retain these patients in the long run and being able to hopefully give them a good treatment for the long run is going to require something like this..

Got it. Great.

And maybe just one question on the phase three MPGN C3 program can you just discuss with us what steps remain if any before initiating the study whether there's any sort of interaction with regulatory agencies that are required?.

Yes. Thank you for that question as well. So as you know this is of course a trial that requires close interaction and collaboration with our partner Sobi but the guidance on the start of the phase three has not changed. It will start 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 now open. .

Thank you. Good afternoon.

So I think one for me would be just in the context, this is probably for Adam in the context of geographic atrophy patient segmentation I'd just be curious if you could talk a little bit about physician level of enthusiasm for treating earlier stage patients those with disease, I'm thinking in the context of the EU retina data from last year?.

Thank you so much Matthew.

Adam do you want to take that?.

Yes. Thank you.

Based on the segmentation that we've discussed we actually find that and again we used of in total transparency our data used for FILLY data when we did our market research and we're obviously updating that we found that actually there was a strong motivation driven often by the patient and supported by the retina specialists for earlier use than we perhaps even anticipated.

So again if you look at our segmentation and you look at the lesion size being small and non-central and you look at the central and small lesion size you find that the physicians are motivated based on what we've seen in FILLY and I think they'll be even more motivated based on what we see in everything else that we're publishing at the moment that there was a good groundswell for wanting to use the treatment earlier.

So again we're excited to see the data towards the end of the year. So a good opportunity for us to help meet some unmet needs in GA..

Great thanks.

And then the second one a little bit perhaps hard to answer at this point but it's assuming that geographic atrophy is successful pegcetacoplan would be a presumably a fairly large part being drug and would just be curious to think to get your thinking around sort of your approach to price your just your views on the evolving pricing dynamics coming out of Washington since part b in particular and VEGF drugs tend to be particularly sensitive from a political perspective? Thank you..

Adam you want to take that?.

Absolutely. So yes, so you're right we expect about 95% OGA to be Medicare.

We've done our initial pricing work and our value discussions obviously based again on what we're seeing coming out of FILLY and I mean it's super-super early for us so one thing I will say that we have seen is that people naturally gravitate when you speak to payers, physicians and experts in outside of the U.S.

they naturally gravitate towards the centers in as their first initial gut when it comes to pricing benchmarks for us to anchor on. Again we have the same rationale as we have within PNH.

We want to prioritize patient access if our drug is as good as we hope it can be then we want to make sure that patients have access to that and we'll make sure that we'll talk about the value of treating GA patients. So definitely more to come but you're spot on with your analysis of the access market. .

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 maybe help us understand what a most favorable scenario would be and maybe a least favorable type of label would be and I guess if you can't sort of speak on that on the details of that maybe help us understand or put it into context those two scenarios in terms of potential number of patients or the difference? How big the gap is between those two types of scenarios? Thank you..

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 PDUFA. .

Okay..

We are very happy to speak about that. .

Okay. Fair enough. Thanks for taking our question. .

Thank you. .

Thank you. Our next question comes from a line of. Your line is now open. .

Hi, thanks so much for taking the question this afternoon.

Starting with PNH Adam could you comment on how the early education is going for the sub-q administration? Kind of any feedback you're getting on ease of use and how much education you think patients will need following approval?.

Yes. Thank you Colin. Great question. So we're obviously going to make sure that we launch with a very robust patient services model and we've been interacting with PNH patients for the last couple of months to make sure that we understand their needs when it comes to that support and patient service approach.

So as I said we will have a good footprint to make sure that we can educate people about the unmet need about PNH treatment and also any support that they might need with administration through our Apellis care educators as we call them that will be there to help the PNH patients.

One thing we found is that actually patients have responded very well to the ability to have treatment at home and the COVID scenario has amplified that and we found some great methods where we believe that again through face-to-face interactions we're allowed but also virtual interactions that we can support patients incredibly well with their administration and we're very much looking forward to doing so.

.

Great. Thanks. And then on DERBY, 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? Just thinking about how the recent phase 1b data continued to strengthen up to 24 months and just wonder if you'd be able to see that play out in the phase 3 data. .

Thank you Colin.

Patients who will stay randomized, Federico, I don't know if you want to add something to them?.

Yes. That is I think the simple answer. So the study will continue to be double mask and the patient will continue and then randomize 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 actually pretty helpful and sorry if I missed it but I'm just curious what is the size of the expanded access program at present and what proportion of those patients are within the U.S.?.

Thanks Laura for the question. I'll actually hand the expanded access program over to Dr. Federico Grossi..

Well we open our expanded access program in the U.S. for now to help the patients with higher medical need to receive pegcetacoplan while the drug is or the application is being reviewed by the FDA.

We don't expect the FDA program to affect our commercial metrics or revenue and this is close to PDUFA we're not commenting any further in our early access program. .

Okay that's helpful. Maybe one other question then I think the latest 10-Q continues to point towards cash runway into the second half of 22.

I'm just wondering if you could elaborate a little bit more on the levers there that could either contract or extend that runway a bit and just against the backdrop of DERBY and OAKS the primary endpoint reading out the third quarter 21 the studies do run for two years or so.

So in the 3Q of 22 so just trying to understand how the spend and the studies might change after that primary readout or how we should be thinking about that. Thanks. .

Thank you, Laura.

Tim do you want to take that?.

Sure. Thanks Laura. Yes that's a great question because actually that calculation around cash runway contemplates a GA success scenario wherein we built up our global launch capabilities and also moved forward in other studies related to pegcetacoplan intravitreally.

So ultimately the calculation becomes very different if GA were not to be successful and we were not to move forward and our cash runway would get extended quite a bit beyond that. Aside from that the typical things relate to how we perform in terms of our commercial launch in PNH pretty much. So hope that answers your question. .

Thanks guys..

Thank you so much Laura..

Thank you..

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 and complement. Thank you again so much for joining us today. .

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