Apellis at Cantor Global Healthcare: Strategic Focus on Growth

Published 04/09/2025, 15:26
Apellis at Cantor Global Healthcare: Strategic Focus on Growth

On Thursday, 04 September 2025, Apellis Pharmaceuticals Inc. (NASDAQ:APLS) presented at the Cantor Global Healthcare Conference 2025. The company outlined its strategic initiatives, emphasizing the launch of Empavelli and Cyfovri, while addressing competitive dynamics and financial stability. Despite challenges, Apellis remains optimistic about its market leadership and future growth prospects.

Key Takeaways

  • Apellis is focusing on expanding the indications for Empavelli, particularly in kidney diseases like C3G and ICMPGN.
  • Cyfovri is experiencing stable growth, maintaining market leadership in key metrics.
  • The company is confident in its financial stability following a royalty deal with Sobe, ending the last quarter with $370 million.
  • Ongoing Phase 3 trials in FSGS and delayed graft function are set to enroll later this year.
  • Apellis is leveraging AI and functional OCT to enhance patient care.

Financial Results

  • The royalty deal with Sobe has bolstered Apellis’s balance sheet, providing a path to profitability.
  • The company ended the previous quarter with $370 million in cash reserves.
  • A $13 million impact from unpaid vials was noted, but the royalty agreement is expected to capture upside potential.

Operational Updates

Empavelli

  • The launch is progressing as anticipated, with a focus on patient vaccinations.
  • Significant reduction in C3 deposition in kidneys by 70% has been observed.
  • Compliance rates are high at 97%, with between 2,500 and 3,000 patient years of dosage recorded.

Cyfovri

  • Stable growth is observed, with low to mid-single-digit injection growth expected in upcoming quarters.
  • Apellis leads in new injections, total injections, market share, revenue, payer preference, and academic presence.
  • The GALE study is ongoing, with additional data expected to support Cyfovri’s functional benefits.

Future Outlook

  • Apellis plans to leverage the GALE study to guide Cyfovri sales next year.
  • The company aims to build on the Empavelli franchise with forthcoming Phase 3 trials.
  • Innovative approaches, such as functional OCT, are being developed to improve patient outcomes.

Q&A Highlights

  • Apellis emphasized the breadth of data supporting Empavelli’s impact on kidney function.
  • Long-term safety profiles address concerns about chronic use and infection risk.
  • The company anticipates regulatory openness to new technologies beyond traditional eye chart assessments.

For more detailed insights, please refer to the full transcript below.

Full transcript - Cantor Global Healthcare Conference 2025:

Stephen: I would love to, obviously, there’s a lot, going on at APELIS. It’s an exciting time. You have a new drug launch. So I would just ask an open ended question. Could you update us on current state of affairs at Appellis and the outlook for the rest of the year?

And we’ll dive into the Q and A thereafter.

Unidentified speaker: Thank you, Stephen. Great to be here after Labor Day and for the first time at Cantor. Congratulations. Thank you. So this is this is a very exciting time for us at Apellis.

We are going into the later part of this year as the company that leads the field in complement in general with two proof products on the market between Ciforvi and Empavelli and an exciting pipeline to look forward to. The key behind the science that we have is that complement factor C3 sits central in that complement cascade and allows the best possible control of all of the downstream effects of complement regardless of what the source of activation is. Of course, we have been in the launch with Empivedi for PNH for a couple of years now, but now with this new approval of C3GN, ICMPGN, a lot of exciting things to look forward to and an incredible opportunity to make a big difference in the lives of the patients that suffer from this disease. With CyFovri, we are now in a period of stable growth. We have given guidance in the form of low to mid single digit growth for several quarters to come.

But what’s important there is that only a small number of patients have been treated so far and the opportunity to really, I’d say educate the retina community around the disease state, what it means for patients, what the drug can do for patients, and importantly the functional benefits that are associated with the lesion size reductions that you see with Cyphovri on which more and more data matures is also a really unique opportunity for us. Then as it relates to the pipeline, we have one exciting Phase two clinical trial that is currently ongoing where we combine a subcutaneous injection with Cyphovri and that allows if the trial is successful and the program is successful, would allow us to reduce the injections from every two months to every three months and of course also looking for an outsized benefit in terms of both lesion size reduction and again that functional benefit that we are looking for. We also have two Phase three clinical trials in the kidney coming up in FSGS and delayed graft function. Those are going to start enrollment is planned for, again, the later part of this year. And then preclinical programs that are maturing as well, which we will be talking about more next year.

All of this on the background of a stable financial situation. You may have seen what we did in terms of our royalty deal with Sobe that provide us with provides us with a solid balance sheet and kind of a great growth platform for us as we go into next year.

Stephen: It’s a great overview, and hopefully, we’ll cover most if not all of that in thirty minutes we have. I do, of course, wanna start with the c three g launch though and drill down on that. How is that going? So it is it

Unidentified speaker: is going as predicted. What is what really stands out there is the unmet need that exists in c three g and c and p g and. When we meet with investors, we often get you know, it’s a new disease. Right? So the the demographics are difficult to gather.

I mean, you have to find out where are these patients, who are they, how many of them are there. But the one thing that I want people to remember is that doing a kidney biopsy, which is really kind of the criterion that you need to identify a patient that’s having a disease, is a serious intervention. Right? I mean, a kidney biopsy is painful, can be dangerous, and is not done lightly by nephrologists. So when we say the number five thousand patients that we have identified that we believe are out there with t three gs and iCMPGN, that is based on highly robust data.

It is also worth noting that if that biopsy is done, it’s because the patient is highly symptomatic. So the unmet need there and the symptomatic implications of having C3G and ICMPGN are not to be underestimated. So really unique opportunity to make a difference. Obviously in Valiant, we had that broad readouts where we did not just see these unique and first in class reductions in proteinuria, but had the trifecta of readouts also on deposition of C3 in the kidneys, proteinuria reduction and EGFR stabilization across iCMPGN, C3G, pre transplant, post transplant, pediatric, and adults. So really kind of the everything has been covered there and that really stands out with patients.

Now we are building on other things that we think will further contribute. And one little element that I think is worth noting there is that the ability that, you know, that we have seen that we will continue to explore to potentially reduce concomitant medications, including steroids is another really important opportunity for these patients. So the launch is going as expected. It will be a gradual uptake, course. Patients need to get vaccinated, etcetera, but really happy where we stand.

Stephen: We’ve been trying we’ve been speaking to nephrologists obviously and, you know, asking have you prescribed Empivalli, but also trying to draw some parallels from the Fab Halta launch, which, you know, has a few more months in C3G, but of course is only indicated in certain population of C3G, not in ICMPGN, not post transplant. And yet the uptake seems strong. I mean, we can find doctors that have already prescribed it to twenty five percent of their eligible C3G patients. Is there any reason why demand in ICMPGN or in adolescents or in post transplant, the areas where FevPolt is not even indicated, would be any different, stronger or weaker than the adult c three g population that seems to

Unidentified speaker: be pretty, you know, eager for treatment here? Yeah. So it’s a little bit early to kind of make those nuanced interpretations, but I’d say what stands out for us competitively is not just the breadth, again, of the populations as you mentioned that we studied, but most importantly the impact that it has on the kidney, right? I mean, so the the trifecta of data like we call it, and most importantly how that c three deposition in the kidney goes away completely in as many as seventy percent of patients after six months of treatments and the continued benefits that you see after fifty two weeks treatment, that is really something that we believe we can can build on.

Stephen: What expectations would you set for maybe fourth quarter, think third quarter? I guess the expectation would be don’t expect much. It’s very early. Patients have to get vaccinated, etcetera. And then what metrics do you plan on disclosing?

Can you just remind us of how we’re going to measure success of this launch in the back half of the year?

Unidentified speaker: Yes. Thank you, Steve. So in the first earnings report that we will have, we will just talk about patient start forms and payer uptake. And then after some subsequent quarters, will expand from that, of course. Okay.

Stephen: The fifty two week data that was presented at ERA, how can you leverage that in the market? I mean, just we’ve heard sort of competing views from some of the physicians we’ve been talking to, one being, I mean, look, there’s actually experience with this, but also FabHolt. I mean, we know complement in other diseases, PNH, for instance, you have a lot of chronic safety data. But then we’ve heard from some other physicians like, look, we only have fifty two weeks of data, I don’t know, maybe there’s theoretically an infection risk long term with chronic use. I found that very odd, and you also have a long term extension study, right, which will gather years of data.

So, I mean, do you think that’s do you think chronic use and taking people off therapy is something that’s going to happen or there’s just no basis for expecting anything like that? How can you leverage the long term extension data that you’re gonna get to assuage any of those concerns?

Unidentified speaker: Yeah. I think once patients are on treatments, we’ve seen that in PNH, I mean, see very good compliance rates. PNH is of course a different disease, but ninety seven percent compliant. It’s hard to find in any disease state. And I think what really stands out from the experience that we’ve had with Empavity where we are now somewhere between twenty five hundred and three thousand patient years of dosing is not just the outstanding efficacy that we have observed, but also the remarkable safety profile of Empavalli.

I think, you know, if we go back even five years only and had said that the meningococcal infection rates with Empavelli you know would be I mean so far no infections that would encapsulate the meningococcus. After that many patients years where you would expect it to see as is the case with c five inhibitors, everyone in two hundred to three hundred patient years thinking that c three could be potentially safer than c five, that is something that is, you know, really something that we can continue to work on and and that stands out here. And all of that that has everything to do with the mechanism by which Pexeta coplan controls c three, which is a buffered way of, you know, putting that that complex interacting system of enzymes on ice rather than trying to remove one element out of it and trying to control the whole thing that way. So that is a it’s a mechanism that has given us those remarkable efficacy profiles in all these trials that we’ve run now, but also this remarkable safety profile that we’ve observed.

Stephen: Can you talk about just reimbursement in c three g, just anything from a co pay assistance or out of pocket sort of assistance effort that Appellis is doing and maybe how that compares to FebHalta because we’ve heard different things about just, you know, Novartis making ways to ease access to to that. And is there anything noteworthy here or is this gonna be pretty straightforward getting everyone covered?

Tim, Appellis: No. I think it’s pretty straightforward. You know, it’s the same. We’ve we’ve been, you know, obviously, Empivalli has been on the market for a while, so we’re pretty used to it, and it’s a pretty similar it’s a very similar, you know, prescriber base.

Stephen: So Okay. What about the receptivity of c three g patients, I guess, based on the historical standard of care, the polypharmacology that they’ve sort of experienced historically to an injection versus obviously the oral option? I mean, would patients sort of defer to the oral option and have to be swayed by the efficacy data of Pexatacaplan or do you think they’re in your in your the early experience that you have or just through the clinical trials, has there been any issue or resistance to the subcutaneous device?

Unidentified speaker: Yeah. No. It’s it is it’s actually quite quite easy to self administer this product. You know, everyone who’s who has tried this, you know, it’s it’s very easy to learn and and you get used to it rapidly. I think what’s interesting there is that, again, the most important element is efficacy differentiation.

But then the fact that in a disease like this, which are very serious conditions, of course, being able to control your complement system without having kind of the anxiety around compliance as well is something that we’ve also heard from nephrologists. In other words, forgetting a pill as we hold on can be very easy to do. With Empaviri, should you forget to self administer or, you know, there’s a delay because you’re traveling, whatever it is, you can catch up for that for a couple of days and kind of go back on the schedule where you were without affecting the efficacy profile. So again, I think it all comes down to what is the unmet need, how serious is this disease and it’s very serious for the majority of patients. And how well do you want to stabilize the trajectory for yourself, right?

And those are the things that we can really detail on and plan to do so.

Stephen: The other thing that we’ve heard from nephrologists which I think is very fascinating is there’s maybe a hesitancy in this disease to actually transplant patients because they’re just gonna be refractory and there’s actually nothing to do. And so you’re putting them through a transplant maybe essentially for not a lot of good reason. That’s changed now obviously, the epithelium can be used post transplant and so there would be actually more of an incentive to transplant patient who could benefit from it. I mean, what’s your thought on that? Are you hearing that as well?

And do you think that just globally sort of beyond amphibelli and isolation, but just the treatment paradigm for these patients, you can sort of improve and improve awareness of and leverage that to build a brand in c three g?

Unidentified speaker: It’s hard to speculate on that, of course, but I think what really stands out here and you you you pointed it out is the the excitement around what Empathyroid can do in transplanted patients, periods. I mean, that’s the it’s an area where most development programs do not even try to go in the transplant of population because it’s complex and and and, you know, you know, more difficult to do operationally and also commercially. But in this particular case, the data really stands out that we’ve generated. And also hand in hand with that, as we’ve talked about before, the impact that Empavedi has had on the xenotransplantation field, is emerging, of course, and wherein all of the the last transplants that were done, the xenotransplantation field, c five inhibitors were supplanted with Empavalli, which were, you know, organ saving interventions at that moment in time. So these are all transplantation nephrologists and transplantation surgeons that of course are diving into the differentiation between c three and c five and seeing the impact that Empavity can have.

Stephen: Maybe let’s pivot to GA. At this point, we can probably come back and I certainly wanna ask about some of the other opportunities in kidney later on if we have time, the phase three programs. But on GA, I guess first question is when do you expect you’ll start providing Cyphovri annual sales guidance? Would that come next year?

Tim, Appellis: Yeah. It’s a good question. We, you know, we did give some guidance in terms of injection growth. We said low to mid single digits, which tracks what we saw in the first and the second quarter. You know, this is sort of the first steady, I would say, steady year.

We had a, you know, two year kind of launch period that had some competitive dynamics and dynamics

Unidentified speaker: There’s a lot going on.

Tim, Appellis: Yeah. That were unique to Apellis and and Cyfovri. So I think it was pretty it was impossible for us to consider giving guidance at that time. Now we’re starting to see Cyphovri in a steady place, which is really encouraging for us. Whether or not we can give guidance next year is a is a good question because it might be odd for us to give guidance on Cyphovri, but not to do it with Empibelli and, you know, people like total So, revenue you know, it’s a timing concept, but, but certainly we’re in much better place to consider that going forward.

And Okay. You know, we’ll try to give any metrics we can to help understand what we think is gonna happen with Cyphovir.

Stephen: Okay. And and, yeah, just to clarify, so the the low to mid single digit growth that you’ve spoken about, I mean, that’s volume. Right?

Tim, Appellis: That’s volume. That’s injections. Yeah. So that is injections into the, you know, into patients. Yeah.

Stephen: I guess how has the market share been tracking since the last update? Because it seems there’s always been a bit of a disparity between you and the competitor Astellas and what’s been reported in terms of aligning on time, reporting new patient share versus total share, and it seems to maybe be ebbing and flowing among new patients. So what’s the current status? If you could update us on any share that you’d be willing to comment on.

Unidentified speaker: Yeah. Well, you don’t have to do mental gymnastics to understand the leadership position of Cyphovir. Right? It’s the market leader on new injections, on total number of injections, on total market share, on revenue, on payer preference, on academic presence. I mean

Stephen: That’s currently or that’s just speaking sort of historically to the last six months or the last year?

Unidentified speaker: Is currently the case. The only metric on which we lost Terrain to our compete competing drug was in the summer of last year, summer and the fall of last year, where on new injections, we fell behind them. And we ended up in a situation back then as to going as low as sixty percent of new injections to the competing product versus 40% for us. That flipped around completely since then and is now at about fifty five percent of new treatments in favor of CyFluvirin. And just in terms of setting expectations for people, I mean, would there be reasons, I don’t know, getting into new clinics or just the way that you’ve seen the sort of competing marketing efforts playing out, why that would continue to ebb and flow?

Do you think it’s stable now or it’s just going to

Stephen: be 55, 45 in perpetuity? Or are there ways that either you or the competitor can actually sort of sustainably change that trend?

Unidentified speaker: Yeah. I think there’s two important dynamics there. One is the the generation of additional data. I mean, have this, you know, amazing Gale extension study, right, which has generated the largest datasets by a mile in geographic atrophy ever generated. And that is something that allows us to have all of these presentation.

I mean, every major retina conference, we have multiple podium presentations. At the SRS, we had, you know, more than an hour completely dedicated to Cyphovir. There were zero for our competing products. And that is of course because the data is not there for that competing product. That’s something that will continue to stand out.

The second aspect, and I think this is really important, is that we’ve not just learned about the efficacy of CyFovri in patients and continue to learn more, we learn a lot more about the impact that this disease has on patients. Most importantly on the functional impact that it has on patients and the functional benefit that CyFovri can provide to these patients. And that is something where again the competitive differentiation is very, very clear. So I think competitively we feel incredibly good with where we are and where we are going. The important second piece to this though is growing the overall market.

And that goes hand in hand with, you know, sharing those data, teaching physicians that these patients with geographic atrophy rapidly decline, know. And it’s not just best corrected visual acuity which is a poor measure. A little side note, I mean, we’ve spoken about this ad nauseam. Mean, patients with geographic atrophy can have stable best corrected visual acuity for years while their overall visual function declines dramatically and they lose more and more activities of daily living. That is something that we have an opportunity to use to to let physicians understand what they can do for their patients.

Stephen: I have more market questions, but actually on this point, just wanted to pause because you mentioned the phase two program with the siRNA and then extending the dosing interval of Cyfovri as a result of that. I mean, that’s sort of a line extension strategy for Cyfovri. But it also feels like an opportunity where you’re in clinical studies with Cyphovri here and you can generate more data and you can address maybe some of the gaps that you think would exist in people’s understanding of the profile. So I guess how can you leverage that development program to your advantage for that combo product but also say Folri and compound is I’m asking a lot of questions at once here, just the development path in GA, has that changed at all since you ran the Phase three Derby Oaks program? Do you have an understanding of how

Unidentified speaker: So it hasn’t really changed, but we believe that it will change. Okay. Because, you know, right now with artificial intelligence, with new methodologies methodologies in which, you know, because of our data, are able to be the leader as well. We are now working on something called functional OCT where with a simple OCT measurements, you can actually determine what the world looks like for that patient. Not just today, but if you have a patient with historical OCTs, can go back and say like what the world looked like five years ago.

This is how it has evolved over these years, and it’s dramatic. Right? I mean it’s not just can this patient read letters on a chart, but you know what does the picture look like? How does that picture evolve over time? And it provides most importantly an understanding not just for the physician but also for the family members of that patient to understand what they’re going through, and then secondarily, of course, to understand what the impact of treatment will be.

And I personally am convinced that in the very near future, in phase three clinical trials when you use those functional endpoints, right, there’s no more question on, okay, what does lesion size growth reduction mean? What is that? How is that correlated, etcetera? That I’m convinced will go away.

Stephen: You regulators would be amenable to some of these new technologies that move past the eye chart? I mean, they already move past the eye chart obviously in GA.

Unidentified speaker: Correct. This would be a functional I am I am personally convinced that they will. I mean, already with the easy zone analysis, which Yeah. To be clear, is not a functional analysis. It’s kind of a an interpretation of a anatomical endpoints.

Right? But that’s, in my opinion, clearly indicates that the FDA and outside of the FDA for sure, the regulatory agencies are interested in exploring how to measure function in GA. Okay.

Stephen: And are you you’re using this functional OCT prospectively in the phase two program for this or this

Unidentified speaker: is something We to are. Okay.

Stephen: And given what you said though about the fact that you can go back to old OCT reading, I mean, you go back and read adjudicate Derby and Oaks and sort of see what the functional OCT data look like in those phase threes?

Unidentified speaker: We can indeed. And it is it’s it’s remarkable. Much more to follow on that starting at angiogenesis in February. We’ll have more more to present on that.

Stephen: Terrific. Okay. Back to ciforoviri in the market. Do you have a good handle on seasonality of this market at this point? Just thinking ahead to quarterly expectations.

I mean, set the volume growth, but there’s some other dynamics, the paid versus unpaid vials, and that’s been affected by the patient, know, co pay assistant charities and the disruption there. So I mean, just in terms of third quarter, fourth quarter for Cyphovri specifically, anything to note that you’ve learned?

Tim, Appellis: Yeah. We’re we’re learning a lot this year. Again, this is the first year that we’ve really had what I would call a steady state in the market. So, you know, even with the co pay assistance organizations being underfunded that, you know, that throws another wrench into it. But we can get a sense, I think, based on how things go this year and, you know, get better guidance next year on what we think seasonality looks like.

Stephen: Just on the the charity, I mean, there any potential for refunding of the co pay assistance charity or a new charity, specifically the one that would be dedicated to GA, so patient support?

Tim, Appellis: Look. At this point, our understanding is that the, the charity is closed to new patients, and we don’t expect that to be a meaningful part of our, you know, business plan going forward in terms of expectations of patients on on commercial drug.

Stephen: What would be, the expectation you’d wanna set for just unpaid vials and by number or by proportion to the total vials going forward?

Tim, Appellis: Yeah. That number was 13,000,000 in the last quarter in terms of dollars, that impact that incremental impact of unpaid files from this. We did say that we didn’t expect that to change. I think it was it was a little bit less than that in the first quarter, and it may even be more this quarter. I mean, it’s it’s it’s something we can’t really tell.

It’s it’s we’ll out what it is, but

Stephen: Maybe a little more this quarter. Said slightly knows.

Tim, Appellis: Right? It could go up. It could go down a little bit. It’s it’s it’s gonna stay in that zone. Right?

It give or take a couple million. So

Stephen: Okay. So on the pipeline, I mean, there’s there’s so much focus obviously on the commercial programs that it’s I’ve done it myself here, left five minutes to discuss your other phase three programs, and we talked about the phase two, but would love for you to set the stage just the expected timelines on plate graph function and FSGS as well. Just where we could see data and where do think the opportunity is?

Unidentified speaker: Yeah. So these are two exciting phase three clinical trials that we kind of of course built on the data that we generate in Valiant and C3G where we saw kind of that exquisite target engagement that happened in the glomerulus. These two Phase three clinical trials are on track to initiate in the second half of this year. And you know, we’re not guiding yet on when they will be completed or the designs, etcetera. But I think as as opportunities to build on the franchise of Empavedi, really

Right? I mean, with FSGS, probably as many as thirteen thousand patients in The US alone that could be candidates for treatment. And then with delayed graft function, you know, in in deceased kidney donors in transplantation, you are probably talking a little north of twenty thousand patients per year. Mhmm. That would be treated for approximately three months in case that they have DGF.

So again, meaningful opportunity and and again, the the biology behind this is firmly supporting what we’re trying to do.

Stephen: Yep. I mean, in delayed graph function, is there any precedence that you draw from in terms of conviction that complement inhibition, C3 inhibition specifically is going be affected? You have all this experience in transplant. Can it be applied to GGF and why?

Unidentified speaker: It’s yes and no, right? I mean of course we learn a lot from transplantation that directly will translate into that experience as it relates to safety, etcetera, of course, as well. But, you know, the the role of complement in delayed graft function is was already known when I was doing transplantation work twenty years ago. Right? So it’s it’s not you know, I feel old.

You know, really exciting fields, one that we know very well and where we think we can make a big difference.

Stephen: And I think in FSGS, I mean, we know some and we’ll learn more about just the regulatory path and expectations there. But in in DGF, guess it’s maybe not what as well understood or or appreciated by folks, there is a guidance document I think that exists prevention sort of trial designs. But are you comfortable with your understanding of what it takes to get to get through the regulatory checks

Unidentified speaker: there? Yes. So again, kind of, you know, we as as competitors learn from us, we learn from our competitors and that regulatory path is available and and and one that we think

Stephen: we can explore. Okay. So you mentioned upfront the decision to sell the remaining Ampovelli European economics to Soviet. Would love just Tim to get your sort of postmortem on that. Why’d you do that?

I mean, the current state of the balance sheet in Appellis financially, how are feeling?

Tim, Appellis: I mean, honestly, that seems like a lifetime ago. It was July 1 and it was seems like forever ago because we’ve got our c three g approval and everything in the meantime. But at the time, obviously, we were we were getting a lot of questions about our balance sheet even though we felt we were in a very position. We ended the quarter with 370,000,000 in the bank, and we thought that was great. I mean, we were and that that would take us to profitability.

But we still were getting questions, and we also had people kind of, you know, not being able to get their arms around the market opportunity. So doing this deal really shined a light on on the opportunity and the value of even just that royalty ex US. And just to make one point, we did get we did cap that royalty. So on an NPV basis, we have a very nice NPV for that royalty, and by capping it, we’re able to capture the upside beyond, you know, the the expectation of when that, you know, that will run out, and we’ve paid that essentially 300,000,000 or or whatever it is back.

Stephen: Nice deal.

Tim, Appellis: We feel very good about it. Yeah. We feel great.

Stephen: Was it competitive or was it always Sobe knows what they have here and

Unidentified speaker: let’s just go to

Tim, Appellis: It was actually extremely competitive. It was a lot there were a lot of third parties, but Sobe stepped up and they obviously know the value of this program, better than anyone. So What

Stephen: would you like you mentioned some preclinical programs that maybe will come to light next year or over time. Anything you can say about just areas of interest, mechanisms, what you’ve been working on behind the scenes?

Unidentified speaker: Yeah. So there there are two important programs that we’re working on. One is that I highlight one of them that we’re very excited about, and that is the neonatal Fc gamma receptor gene editing program. This is a program where in our collaboration with Beam, what we’ve done is taken that receptor and with the single base edits be able to completely eliminate the recycling of immunoglobulins from the circulation, which as we all know, of course, leads to a reduction of approximately 50% of IgGs in circulation without affecting albumin. So in other words, you know, with a single injection, you can have that treatments and have the impact and the benefit of that for the rest of your life.

Now, of course, in gene editing and gene therapies, there, you know, it has been a there has been a lot of turmoil recently. What stands out here is that we use LNPs, not AAVs to deliver the editing cassette. So that is important. And the second piece is that we are, with this program, not going after momogenic diseases, which, you know, has been the focal point of most gene therapies recently and where, you know, the risk arguably is greater. So we’re gonna we believe we have a path forward and we’ll share more about that next year to do this in a highly receptive population where the safety the risk benefit profile will be very clear compared to what may have happened before.

Stephen: Looking forward to that. And thanks so much for for broad conversation. Thanks everyone in the room for listening. Thanks everyone on the webcast. Hope everyone enjoys the rest of the day at the conference and join me in thanking Appellis for conversation today.

Thank you, Steve.

Tim, Appellis: Thanks, Steve.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Latest comments

Risk Disclosure: Trading in financial instruments and/or cryptocurrencies involves high risks including the risk of losing some, or all, of your investment amount, and may not be suitable for all investors. Prices of cryptocurrencies are extremely volatile and may be affected by external factors such as financial, regulatory or political events. Trading on margin increases the financial risks.
Before deciding to trade in financial instrument or cryptocurrencies you should be fully informed of the risks and costs associated with trading the financial markets, carefully consider your investment objectives, level of experience, and risk appetite, and seek professional advice where needed.
Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. The data and prices on the website are not necessarily provided by any market or exchange, but may be provided by market makers, and so prices may not be accurate and may differ from the actual price at any given market, meaning prices are indicative and not appropriate for trading purposes. Fusion Media and any provider of the data contained in this website will not accept liability for any loss or damage as a result of your trading, or your reliance on the information contained within this website.
It is prohibited to use, store, reproduce, display, modify, transmit or distribute the data contained in this website without the explicit prior written permission of Fusion Media and/or the data provider. All intellectual property rights are reserved by the providers and/or the exchange providing the data contained in this website.
Fusion Media may be compensated by the advertisers that appear on the website, based on your interaction with the advertisements or advertisers
© 2007-2025 - Fusion Media Limited. All Rights Reserved.