Apellis Pharmaceuticals Inc at Citi Global Healthcare Conference Summary

The following is excerpted from the question-and-answer section of the transcript.

Question: Yigal Nochomovitz - Citi - Analyst : <_ALACRA_META_ABSTRACT>Yeah. Well, yeah, that was a good meeting with Pascal and the old guard, I guess. Okay. So let's start with the new indication, which -- well, it's not so new for you, but for -- maybe for investors that need to understand it better in kidney disease, C3G, IC-MPGN, you had some very nice data, Phase 3 data recently a few months ago. Tell us about it, tell us about how you believe it could impact patients, and I'm sure we'll have plenty of detailed questions soon? Cedric Francois - Apellis Pharmaceuticals Inc - President, Chief Executive Officer, Co-Founder, Director Okay. Thank you so much, Yigal. Well, first of all, thank you for inviting us to this conference. It's wonderful to be here. So yeah, the data in C3G and IC-MPGN in the so-called VALIANT study was a study run in 124 subjects, where we -- I'd say like it's a little bit of a tradition of ours to go very broad in these populations and afterwards find out where the real defects lie. And so we enrolled in this study adolescent adults, C3G patients, IC-MPGN patients, pretransplant, post-transplant, C3 depleted patients, C3 competent patients. And across the board in all of these phenotypes, saw the same kind of remarkable efficacy profile that Empaveli and the ingredient pegcetacoplan has shown time and again across so many indications now. Results far outweighed what we had even expected from this trial. And to make a long story short, I mean, across the three parameters, which are proteinuria, eGFR stabilization, and then also, and very importantly, the deposition of C3C, which you can find on REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 6:00PM, APLS.OQ - Apellis Pharmaceuticals Inc at Citi Global Healthcare Conference histopathology in these kidneys, seeing that disappear completely in about 70% of patients after six months when these kidneys in these patients basically look normal, that was really incredibly gratifying to see. That means that if you're an adolescent or a young adult with this disease and normally having to face a kind of an unstoppable progression towards the end-stage renal disease, hemodialysis, and maybe transplantation, the efficacy profile of this drug seems to indicate that you may never need those interventions in the future. Another element that is worth pointing out is that the need for concomitant medications may be drastically reduced to something that we see in VALIANT, and we will be talking about more. That is, of course, very important. These are strong immunosuppressants, including steroids, that have side effects for these patients. We're going to find out if that is even still needed. But for now, we are incredibly happy with the data that we found.

Question: Yigal Nochomovitz - Citi - Analyst : So since you've covered all year basis in terms of both of the phenotypes and then the pre- and the post-transplant and in terms of pediatrics and adults. So, you have covered everything. And the competitive molecule is oral. So there's some discussion around oral versus subcu, but there's not a parallel there in terms of what's been explored in the clinical setting?

Question: Yigal Nochomovitz - Citi - Analyst : And so just really quickly, just remind everybody when you're filing that's, I guess, an sNDA or how would you characterize it?

Question: Yigal Nochomovitz - Citi - Analyst : Okay. And you have teams in place that you're working on building out that commercial effort?

Question: Yigal Nochomovitz - Citi - Analyst : I mean, we've talked to a number of KOLs, as you know. And they obviously highlighted the very strong efficacy. And I think some of your experts obviously have noted that having the efficacy should be the determining factor over maybe slightly better conveniences with oral. I guess I've kind of summarized it, but anything else you want to add to that statement?

Question: Yigal Nochomovitz - Citi - Analyst : And like in some other areas in development with your pipeline, you've done long-term extension studies. Do you have such a plan for C3G, IC-MPGN to continue to monitor or like to see longitudinal benefits on eGFR beyond the primary endpoint?

Question: Yigal Nochomovitz - Citi - Analyst : Okay. And then what are the plans outside of the United States to develop this indication?

Question: Yigal Nochomovitz - Citi - Analyst : So they have the rights to the glomerulopathy, okay.

Question: Yigal Nochomovitz - Citi - Analyst : Okay. And what about other territories, they have everything outside the United States?

Question: Yigal Nochomovitz - Citi - Analyst : So Asia as well?

Question: Yigal Nochomovitz - Citi - Analyst : Okay. Great. And as far as other conference presentations, I mean, you've given a lot already, but what's the -- is there another data point that we should be looking for outside of the typical regulatory?

Question: Yigal Nochomovitz - Citi - Analyst : Yes, just in terms of other clinical -- I mean, I know, for example, I mean in ophthalmology, you have many studies that beyond the -- go beyond the primary data?

Question: Yigal Nochomovitz - Citi - Analyst : I'm curious, what other settings would you be worth pursuing? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 6:00PM, APLS.OQ - Apellis Pharmaceuticals Inc at Citi Global Healthcare Conference

Question: Yigal Nochomovitz - Citi - Analyst : Okay. Well, those -- we follow a lot of those companies as well in IgA and all cards of interesting possible scenarios for combining because those, for example, like Travere, the different, I mean (inaudible) is a steroid, but Travere, that's maybe like an orthogonal MOA, so it could be interesting. Okay. Very good. So maybe we should spend a little bit of time on ophthalmology. So just give us the latest updates as far as how you're seeing -- how you're seeing the launch progress. We're -- you're now almost -- well, I guess, 7.5, 7 quarters in something like that?

Question: Yigal Nochomovitz - Citi - Analyst : Tell us how you've got that -- is it almost five years now? How long -- how much time do you have on that? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 6:00PM, APLS.OQ - Apellis Pharmaceuticals Inc at Citi Global Healthcare Conference

Question: Yigal Nochomovitz - Citi - Analyst : Beyond four, okay. That's almost five.

Question: Yigal Nochomovitz - Citi - Analyst : Tell -- I mean, we want everyone to succeed, but still -- I mean, I remember when Adam Townsend made the point about getting the two-year data on the label from the outset and that whole situation back then, which we won't discuss too much. But basically, that clearly was the right decision. And then -- and now, of course, Izervay doesn't have that claim, at least not for a little bit. So how is that -- how are the retina experts thinking about that because that's clearly an important point?

Question: Yigal Nochomovitz - Citi - Analyst : And they can't improvise, right? They can't take, I mean there's no data to support that? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 6:00PM, APLS.OQ - Apellis Pharmaceuticals Inc at Citi Global Healthcare Conference

Question: Yigal Nochomovitz - Citi - Analyst : I know, obviously, it's been a tougher road across the pond. Is there anything to say there? Is there another strategy? Is there -- could that come back into more focus with more data, real-world data, more KOLs? I mean both you and Iveric, Astellas have had similar challenges over there?

Question: Yigal Nochomovitz - Citi - Analyst : But it's -- like you've explained to me and others many times, I mean this BCVA, this is an old tool, right? I mean, you put it on the wall, and you read letters. It somehow may not be the most appropriate assay, so to speak, right, for GA because of the nature of GA and the way that the lesions creep around the fovea and so things of that nature?

Question: Yigal Nochomovitz - Citi - Analyst : You're not going to move your head around trying to see every part of the big letter?

Question: Yigal Nochomovitz - Citi - Analyst : Understood. Let's spend a little time on PNH, if we could. It's doing well, but it's sort of trending to -- I don't know what you want to say, but it's decelerating. So talk about where you see that growth if it's really more of a mature product now. Or how should we think about that for going forward?

Question: Yigal Nochomovitz - Citi - Analyst : Okay. Let's talk about pipeline. I mean you've done some enterprising trials, some sort of high risk, high reward, i.e., ALS, which you characterize as a high-risk study. But there's some other programs that are in the earlier pipeline, if you could elaborate. And then you have a program to help with gene therapy, correct?

Question: Yigal Nochomovitz - Citi - Analyst : Those are disclosed programs? Or what -- can you say more there?

Question: Yigal Nochomovitz - Citi - Analyst : So you'll do teaser for the spring, okay.

Question: Yigal Nochomovitz - Citi - Analyst : By the way, are you going to have a different trade name for C3G? Are you going to call it Empaveli or what's the plan there? I'm just curious. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 6:00PM, APLS.OQ - Apellis Pharmaceuticals Inc at Citi Global Healthcare Conference

Question: Yigal Nochomovitz - Citi - Analyst : Okay. I do get a lot of questions around the path to profitability and the financing and the cash. So can you just maybe Tim just walk us through how you're thinking about that? I think you've made the point that you can make it to profitability with the growth of the existing business and the current expectations around the OpEx?

Question: Yigal Nochomovitz - Citi - Analyst : And what -- just to remind everyone what was that vehicle that allowed you to limit that risk on the --?

Question: Yigal Nochomovitz - Citi - Analyst : And then the balance sheet. So just to remind everyone, you -- is there -- have you extinguished all the debt? Or is there a piece there that's still existing? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 6:00PM, APLS.OQ - Apellis Pharmaceuticals Inc at Citi Global Healthcare Conference

Question: Yigal Nochomovitz - Citi - Analyst : That's a straight debt, not convertible?

Question: Yigal Nochomovitz - Citi - Analyst : When does that mature?

Question: Yigal Nochomovitz - Citi - Analyst : So what's the plan for the $93 million? You're just going to let it convert or you're going to early --

Question: Yigal Nochomovitz - Citi - Analyst : Okay. It's only $93 million. All right. Now any other -- what about external activity in BD? Anything -- are you looking at external assets? To what extent is that a relevant part of the business plan? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 6:00PM, APLS.OQ - Apellis Pharmaceuticals Inc at Citi Global Healthcare Conference

Question: Yigal Nochomovitz - Citi - Analyst : Okay. Well, we have some more time. So a few other kind of grab baggy questions. So everyone's asking about a few things, AI. Does that feature in any way in your organization, either for efficiency purposes or actually for real drug development or not?

Question: Yigal Nochomovitz - Citi - Analyst : Okay. And then as far as the marketing efforts with the social media and of course, the Henry Winkler and all these other -- so you -- how is that going? And do you expect to leverage that sort of effort for -- I mean, for C3G, maybe it's not really necessary, right? But I just --

Question: Yigal Nochomovitz - Citi - Analyst : Invite him to the next fireside? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 05, 2024 / 6:00PM, APLS.OQ - Apellis Pharmaceuticals Inc at Citi Global Healthcare Conference

Question: Yigal Nochomovitz - Citi - Analyst : Okay. Another new question, which only emerged after November 5 or 6 or whenever the election was. So I mean, on the manufacturing, just remind everybody, where do you source -- how much is sourced internally in the United States versus anything offshore as regards to the potential, more protectionist trade policy that could happen?

Question: Yigal Nochomovitz - Citi - Analyst : Okay. And you don't have to answer this one, but you can if you want. So any thoughts on the potential changes in leadership at the following HHS, CMS and the FDA?

Question: Yigal Nochomovitz - Citi - Analyst : More concrete question. I mean, given the change in leadership specifically at the your division, does that impact you in any meaningful way or, I mean, you're obviously approved. So it's kind of in the past, but I'm just wondering if that has any relevance now for you.

Question: Yigal Nochomovitz - Citi - Analyst : Yeah.

Question: Yigal Nochomovitz - Citi - Analyst : Perfect. Okay. Well, thanks again. We'll have Henry Winkler here next year.