Good morning ladies and gentlemen. Thank you for standing by and welcome to the Apellis Pharmaceuticals EMPAVELI FDA Approval Call for C3G and IC-MPGN. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question- and- answer session. To ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. Please be advised that today's call is being recorded. I will now turn the call over to Tracy Vineis, Vice President of Communications. Please go ahead.
Hello everyone and thank you for joining us to discuss the FDA approval of EMPAVELI, also known as systemic pegcetacoplan for C3G and Primary IC-MPGN. With me on the call are Co-Founder and Chief Executive Officer Dr. Cedric Francois, Chief Medical Officer Dr. Caroline Baumal, and Executive Vice President, Commercial David Acheson. Chief Financial Officer Tim Sullivan is on the line for Q&A. Before we begin, let me 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 actual results may differ materially. I encourage you to consult the risk factors discussed in our SEC filings for additional details. Now I'll turn the call over to Cedric.
Thank you Tracy and thank you all for joining the call. We are excited to announce that for the first time a C3- targeting therapy is approved for patients with C3 Glomerulopathy or C3G and Primary Immune Complex Membranoproliferative Glomerulonephritis or IC-MPGN. Today we achieved a significant milestone with the FDA approval of EMPAVELI for the treatment of patients 12 years and older with C3G or Primary IC-MPGN. We are thrilled with this approval, which establishes EMPAVELI as the only product that has demonstrated the "trifecta" of outcomes across all three key markers of these diseases: proteinuria reduction, eGFR stabilization, and substantial clearance of C3 deposits. EMPAVELI's broad label is a breakthrough for patients. It addresses several populations for which there were previously no approved treatments, including pediatric patients with C3G, Primary IC-MPGN patients aged 12 years and older, and patients with post-transplant C3G disease recurrence.
Importantly, EMPAVELI's safety profile is well- established and this label remains in line with the existing label for PNH. Until recently, many people living with C3G and Primary IC-MPGN have been underserved by the available treatment options. As a disease-modifying therapy, EMPAVELI has the potential to transform the care for these patient communities. With a broad label secured, our focus now shifts to getting this treatment to patients. Our field team has been preparing for this launch and working hard to educate the physician community on the importance of addressing the three key markers of C3G and IC-MPGN. Again, those are proteinuria reduction, eGFR stabilization, and clearance of C3 deposits. We have also been working with Payers to help secure access to EMPAVELI for all patients. David will share more on our commercial launch plans shortly.
I'm very proud to share that this label expansion marks the second FDA approval for EMPAVELI and Apellis' third FDA approval in just four years. This milestone reflects our ability to execute at the clinical and regulatory level and the dedication of our team. I want to thank everyone at Apellis for your unwavering commitment to improving the lives of people with serious diseases. As the only approved C3- targeting therapy, this achievement further validates the power of targeting C3, the central protein of the complement cascade. Building on our commercial success in PNH and EMPAVELI's demonstrated efficacy in C3G and Primary IC-MPGN, we are advancing Registrational Programs in two additional complement-driven kidney diseases, Focal Segmental Glomerulosclerosis (FSGS) and Delayed G raft Function (DGF). Both are serious conditions with no approved treatments, and we remain on track to initiate Pivotal Trials in these indications before year- end.
I would like to thank the many people who supported our efforts and made this approval possible. We are especially grateful to the patients who participated in our clinical trials, their families and caregivers, and the healthcare professionals who contributed to the studies. I will now turn the call over to our Chief Medical Officer, Dr. Caroline Baumal, to review the data that supported this approval and the label granted by the FDA.
Caroline
Thank you Cedric. I'm incredibly excited that we can now provide this important treatment to people living with C3G and Primary IC-MPGN. Both of these diseases are characterized by Complement overactivation and C3 deposition, leading to inflammation and damaged kidney tissue. Diagnosis of these diseases typically occurs when patients are in their late teens to mid-20s, a time when most are just beginning to establish independence, pursue higher education, or start their careers. Disease progression can be rapid, with 50% of patients progressing to kidney failure within five- to- 10 years of diagnosis. Historically, these diseases were insufficiently managed with chronic immunosuppressants. Even kidney transplantation is not curative, as approximately 90% of patients who receive transplants experience disease recurrence. Today's FDA approval is based on positive results from the phase III VALIANT study.
VALIANT enrolled 124 patients, making it the largest single trial conducted in these populations and the only Pivotal Study to enroll adult and pediatric patients with native and transplanted kidneys. Results clearly demonstrated EMPAVELI's ability to address the underlying cause of these diseases by targeting C3. EMPAVELI hit the "trifecta" of outcomes across the three key markers of these diseases. I'd like to walk you through the importance of each. First, proteinuria is the presence of protein in the urine and a hallmark of kidney damage. VALIANT showed a 68% reduction in proteinuria as compared to placebo at Week 26. Importantly, more than half of patients treated with EMPAVELI achieved less than one gram of proteinuria, which is an indication of disease remission. Next, the study also evaluated the effect of EMPAVELI on eGFR, or estimated glomerular filtration rate, which is a key measure of kidney function.
Treatment with EMPAVELI resulted in a stabilization of the eGFR relative to placebo over 26 weeks. This is important because if left untreated, patients with C3G or Primary IC-MPGN continue to lose kidney function and typically progress to End-S tage Kidney Disease or dialysis. Finally, C3 staining is used to detect the deposition of C3 in kidney tissue and is a key measurement used in the diagnosis of these diseases. After 26 weeks of EMPAVELI treatment, more than 70% of patients treated showed complete clearance of C3 deposits as demonstrated by zero C3 staining intensity. The VALIANT study is the only study to date to demonstrate these outstanding effects on the key biomarkers of these diseases. Consistent with the extensive real- world experience we've seen in PNH and Clinical Studies, EMPAVELI treatment was well- tolerated in the VALIANT study.
At the ERA Congress last month, long- term results were presented showing that robust proteinuria reduction and stable kidney function were maintained after 52 weeks of treatment. In line with the outstanding compliance we've seen for EMPAVELI in PNH, more than 90% of patients remained adherent during the Open-L abel Extension Period of the study. Now turning to the broad Prescribing Label. As we shared, EMPAVELI has been approved for the treatment of adult and pediatric patients aged 12 years and older with C3G or Primary IC-MPGN to reduce proteinuria. This broad label includes post- transplant C3G patients. EMPAVELI is also the only approved treatment to demonstrate efficacy in C3G patients who have undergone a kidney transplant. The VALIANT study results are unparalleled in these diseases and we are now able to offer this innovative treatment to patients.
Since we announced the data, excitement has continued to build among nephrologists and patients. With this broad label in hand, we look forward to bringing this important treatment to the community. I will now turn the call over to David Acheson, our Executive Vice President of Commercial, for an overview of our launch plans and the commercial opportunity.
Thank you, Caroline. We are thrilled to make EMPAVELI available to people living with (C3G) and Primary IC-MPGN. Our commercial organization has been preparing for this approval, and we are ready to start delivering this transformative treatment to patients. I'll start with the commercial opportunity, then discuss our launch plans. In the U.S., we estimate there are approximately 5,000 C3G and Primary IC-MPGN patients. EMPAVELI's broad label makes it the first treatment approved for pediatric patients with C3G, Primary IC-MPGN patients aged 12 years and older, and patients with post-transplant C3G disease recurrence. We expect EMPAVELI to become the preferred treatment across all C3G and Primary IC-MPGN patients given its meaningfully differentiated clinical profile and broad label. With the opportunity in front of us, we believe EMPAVELI is on path to "blockbuster" status.
Keep in mind this is a Rare Disease Launch in indications where there have previously been few or no approved treatments. As a result, we expect gradual uptake in the early stages of launch, with momentum building as disease Awareness and physician familiarity grows. From the time a patient is identified, we expect it to take four to six weeks for treatment to begin. Therefore, we expect meaningful revenue generation to start in the fourth quarter of 2025. Current annual WAC price is approximately $505,000 per patient. We expect Gross- to-N et to be in the mid-teens for 2025. Shifting now to our launch plans, following the VALIANT study results, we expanded our EMPAVELI Field Force. Our commercial team's experience within the Nephrology Community will be a significant advantage moving forward.
With EMPAVELI's established role in Hematology, we already have a strong presence in the Healthcare Organizations where many of the nephrology patients are treated. We estimate that approximately 60% of C3G and Primary IC-MPGN patients are managed in these organizations. Beyond these target organizations, we have identified a large portion of treating nephrologists, and they are confident that we can reach all healthcare providers currently managing these patients. Our Launch Strategy has three components. First, raise awareness about the availability of EMPAVELI as a disease-modifying therapy that targets the underlying cause of the disease. We've been actively engaging nephrologists for months to communicate the importance of treating the key markers of disease. We continue to have a strong presence in physician offices and Medical Conferences to drive awareness of the benefits of EMPAVELI. Second, establish EMPAVELI as the treatment of choice among nephrologists.
We are focused on leveraging the strength of VALIANT data and EMPAVELI's unique mechanism of targeting C3. Our goal is for physicians and patients to equate early use of EMPAVELI with preserving kidney function and achieving long- term disease control. Nephrologists consistently emphasize that efficacy, not route of administration, will drive treatment decisions, and finally, secure broad access as soon as possible. We care deeply for patients and are committed to ensuring that every patient who may benefit from EMPAVELI has access to treatment. EMPAVELI already has robust coverage for PNH, and we have been actively engaging with Payers to highlight its differentiated value for C3G and Primary IC-MPGN. We are confident about ensuring broad coverage. In summary, we are well- positioned to maximize EMPAVELI's potential as we expand into C3G and Primary IC-MPGN.
We have a highly- effective treatment with a well-established safety profile and an exceptional team driving our Launch in Rare Nephrology. I'll hand it back to Cedric for Closing Remarks. Thank you, David.
As the first and only targeted C3 therapy available for these diseases, EMPAVELI has the potential to redefine treatments for the C3G and Primary IC-MPGN community. Today marks another impressive milestone for Apellis Pharmaceuticals and we believe we have only begun to unlock the full potential of targeting C3. Operator, please open the call to Q&A.
Thank you. As a reminder to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one. Again, please limit yourself to one question. One moment for questions. Our first question comes from Jonathan Miller with Evercore ISI. You may proceed.
Hi guys.
Thanks so much for taking my question.
Congrats on the label.
It looks very strong. I would love to start with commercial expectations. Both you and Novartis are suggesting penetration is going to be slow here. I guess how soon will you
be able to tell how your market
share is evolving versus the competitor here. How soon will you be able to have enough commercial data to be very confident in how big the full market opportunity is? I guess if the penetration is going
to be slow, how will you plan
on communicating how it's evolving to the market over the next few quarters?
Thank you, John. Great to hear you. I'm going to hand it over to David to answer the question.
Hey John, good morning. Thanks for the question. Appreciate it. Great question. What we do expect as we start out with the Launch is that there's likely some pent- up demand in the market waiting for this product to come, which is typically true in most Rare Disease type of launches that have had virtually nothing else to be available for patients, at least up to this point in time. What we do expect is that we're going to see our transition of EAP and Compassionate Use atients move over in the next couple of months, which is a good start for patients to go on commercial product. We'll find out more as the Launch kind of goes on here. I do think one of the questions or part of the question you had was around the metrics.
Early on what we're going to look at is really the Start Forms coming in along with the REMS enrollments that we have from physicians. Everything else after that we'll pull together over time and report out at some point in time patients on product. Early on in the Launch that's where we're going to spend most of our time. We do think we'll produce meaningful revenues in Q4. We're launching as of today and excited to see things come together. We'll tell you more as we get into the Launch in Q4.
Thank you. Our next question comes from Anupam Rama with JPMorgan. You may proceed.
Hi guys, this is Priyanka on Anupam. Congratulations on the approval. What are your marketing messages going to be competitively in C3G relative to, and have you observed any counter detailing heading into yesterday's PDUFA and this Launch in Medical Education Forums?
That will also be a question for David Acheson.
Yeah, great, thank you for the question. Right now what we're focused on is getting into the segments of the discussion where we can actually separate the product out significantly. You gotta remember this is the first time that the broad label that we just received by the FDA, some of these patients will have access to any product that can actually treat their disease state. Just as a reminder, part of that, the segments I'm talking about are the pediatric patients over 12 years of age with C3G, Primary IC-MPGN patients 12 years and older, and patients with post- transplant C3G disease recurrence. We do anticipate that, you know, obviously there's patients out there that have a High Unmet Need right now. We anticipate the Severe Patients will probably be the Early Starts as long as we can figure out where they all are located.
That's what we've been working on the last several months. The post- transplant patients obviously are also the target for us that is unique to our label. We'll be in these accounts talking about all of these broad babel Disease States across all of the Segments starting today.
Thank you. Our next question comes from Tazeen Ahmad with Bank of America. You may proceed.
Hey guys, good morning.
This is Daniel on for Tazeen. Thank you for taking our question. I was wondering, some of our "doctor
checks" have indicated that doctors see C3G and
IC-MPGN and a sort of spectrum of diseases. I was wondering from a decision and maybe also a Payer perspective, how well
understood i s the difference between the two, and how is that going to impact
your prescribing of the different agents that are available?
Thank you so much. I will hand that over to Caroline, but I think the answer to that question is correct.
Right.
So C3G and IC-MPGN are diseases with overlapping phenotypes. The key differentiator is actually noted on histopathology when you take a biopsy from the kidney and look at the presence of antibodies in these deposits. Caroline, maybe you want to elaborate a little bit.
Thank you so much. You're absolutely right. We're learning a lot about these diseases right now, and we are involved with leaders in the field about following natural history studies and defining this disease better. What I think we've consistently heard from KOLs and physicians is that they're excited that our study included a broad range of patients, including both patients with C3G and IC-MPGN. The results that we saw in our clinical trial were effective across both Disease States, as well as pediatric patients age 12 and older with both Disease States, and post- transplant patients with C3G. Physicians have told us that what's going to drive their decisions and treatment of patients is the results and efficacy from clinical trials. Thank you. Thanks.
Thank you. Our next question comes from Yigal Nochomovitz with Citigroup. You may proceed.
Hi Cedric and team. Congratulations on the approval.
I was just curious, you mentioned the pricing. Just to clarify, is this pricing the
same or different from PNH pricing? I was wondering, was there
ever a consideration given to a different trade name for the drug?
I know it's the same molecular entity, but I was just wondering if that was consideration at all to differentiate in terms of the marketing pitch. Thank you.
Hey, this is David. I'll answer that question for you real quick. The pricing is consistent across all indications and that was always the plan, as the dosing is also the same across all indications as well. That was considered. I think we're in a good spot with the pricing side of it. As far as a different trade name, we always consider all of those things, but because it's an extension to the label as a Supplemental NDA, certainly it was advantageous for us to have that pathway versus something under a new trade name. Again, the dosing and the extension to the label is consistent with everything else that we've done in PNH.
Okay, thank you.
Thank you. Our next question comes from Salveen Richter with Goldman Sachs Group. You may proceed.
Hi, [audio distortion] for Salveen. Congratulations on the approval. Quick question on the Payer work. Have you been receiving any feedback from the Payer work that you've done to date, and when do you expect to see the inclusion of EMPAVELI
for
hese diseases in the major form ularies?
I'm sorry, we have a hard time hearing you. Would you mind repeating your question?
Sure. Is it better now?
This is a little bit better. Yes.
Yeah. Congratulations on the approval. Any feedback that you've received from your Payer work to date, and when do you expect to see the use of EMPAVELI for these diseases in the major formulary group?
Yeah, I still had a hard time hearing that. Would you mind trying one more time?
Sure. Okay. Just a question on the Payer work that you've done. Any feedback you've received there, and when do you expect to see the inclusion of EMPAVELI for C3G and IC-MPGN in major formularies? Sorry for the disturbance on my end.
Okay, thank you so much. Now that was clear. David.
Just to make sure I'm clear, it's about the work at the Payer level, right, Cedric?
Yes, how the Payer conversations have come.
Great. Thank you, [Salveen] . Sorry we can't hear you better. That work has been done extensively. We've met with all the Key Payers and the downstream accounts, including the big accounts that are attached to the PBMs. We feel very good that they understand the story and the clinical data. They are pleased with what we bring to them with an opportunity to treat these patients, and I think it's been clearly stated by them that the path forward here is going to be a lot like what you see with any other Rare Disease Product. There will be work back and forth early in the Launch to make sure that the right patients are getting to product and then after that it should become fairly standard in regards to approval.
There's always a prior auth attached to every one of these prescriptions because of the product and the disease states. We're prepared to handle that with the plans and the payers and work
it through our specialty pharmacy.
So very successful, I think, in getting prepared for that. The second part of the question, I just want to make sure I'm clear. Can you repeat the second part of the question?
When do you expect to see EMPAVELI for these indications on the major formularies?
I think we're there in many cases already, and I think that the work that's been done has us in a Policy inside a lot of these PBMs and Payers. I am convinced and very confident that as of today we're launching the product. If a Prescription went in today, those prescriptions would be dealt with in a typical fashion for Rare Disease. They'll work with our Specialty Pharmacy to get the Patients Covered to Product as soon as we can.
Thank you.
Thank you. Our next question comes from Steve Seedhouse with Cantor. You may proceed. Good morning.
Thank you and congratulations. Wanted to ask specifically, maybe multi-part question about the Addressable Market and really just your conviction in how many C3G, IC-MPGN Patients Eligible for Therapy there are. I guess the question is, can you just expand on the methodology or if there's more than one methodology, what you're using to assess how many patients there are and if you're making any assumptions about Diagnosis Rates and whether those would be changing over time. Would love to just hear your xxpanded thoughts on how you're assessing the market size. Thank you.
Thank you so much, Steve. Great hearing you. That is an important and a very good question. The methodology that we used was based on Payer Codes, so we actually went, or Diagnostic Codes, I should say. We went back eight years.
The requirements to be included in what we consider to be 5,000 patients in the U.S. alone is that there had to be two Diagnostic Codes in the last eight years, with one of those two codes having occurred at least in the last three years. These are quite stringent criteria. I think it's important to note here that for a Diagnostic Code, typically, you need a Confirmed Biopsy. Patients who receive a biopsy obviously are typically Severely Symptomatic. These are numbers that we feel very good about as being conservative, and in the field as well, when we go to the Site of Care, that certainly seems to be in line with what our expectations should be.
Thank you.
Thank you.
Thank you. Our next question comes from Colleen Kusy with Baird. You may proceed.
Great. Good morning. Thanks for taking our questions and congratulations on the approval. Maybe a quick follow- up on that, Cedric, will patients need a fresh biopsy prior to going on therapy? If you could just discuss the REMS Program for EMPAVELI versus.
How you've seen that play out for FABHALTA.
Yeah.
Thank you so much, Colleen. I will hand that over to David as well.
Yeah. Hi, Colleen. By the way, thanks for the question. On the question around the REMS, the REMS is consistent between the Complement Inhibitors. Our ability to have people do a REMS does not take very long for doctors to sign up, and it's consistent between brands and products. It's pretty easy for a doctor to be REMS Certified, and then we can start patients. That physician can then start patients to process to become treated with the product.
Just on the fresh tumor
biopsy.
There's no new biopsy needed before treatment.
Great.
Thank you.
Thank you, Colleen.
Thank you. Our next question comes from Phil Nadeau with TD Cowen. You may proceed.
Good morning.
Let us add our congratulations on the approval. Two from us. First, in terms of the 5,000 patients in the U.S., do you have an estimate of how many are in categories that are unique to the EMPAVELI Label? Second, can you remind us, did you seek a Label in Post-T ransplant IC-MPGN, and if so, what happened there?
Thanks.
Sorry, my line got scrambled a little bit. Would you mind repeating that, please?
Sure.
The first question is, of the 5,000 patients in the U.S., how many are in categories that are unique to the EMPAVELI Label? In terms of Post-T ransplant IC-MPGN, c an you remind us if you sought an indication there and if so, what happened?
Thanks.
Thank you so much. I'll start with the second part of the question. This was, of course, something that we were hoping to get into the Label. I think the small number of patients for IC-MPGN made it difficult to get that as part of the Label. Again, I want to point out that it's viewed as a Spectrum Disease and something that certainly we can pursue afterwards. With respect to the first question, not entirely clear what you mean with the connection between the Biopsies and the Prescriptions.
No.
I'm asking, there's certain parts of your label that are not shared by FABHALTA. Do you have an estimate of how many patients those parts of your label refer to?
Yeah, thank you so much again, that's a very good question. The numbers that we have are approximately 5,000 patients in the U.S., which includes everything that is in the label here. Of course, the separation there is about half of these patients, 2,500, we believe, have IC-MPGN, 2,500 have C3G. If we kind of go down to the place where we will be competing with FABHALTA, that takes us from 5,000 down to 2,500. About 20% of these patients are transplanted patients. That takes you down to 2,000. Within that, pediatric population is probably also approximately 20%, 15%- 20%. The place where we will really be competing with FABHALTA is somewhere between 1,500 and 2,000 patients.
Of course, we hit the trifecta of efficacy. Hitting on all these elements and having that included, all of these efficacy elements included in the label is, of course, hugely important for us.
That's very helpful.
Congratulations again .
Thank you.
Thank you.
Our next question comes from Ellie Merle with UBS. You may proceed.
You may proceed.
Hi, this is [Chase] on for Ellie. Congrats on the approval. Just some question on how do you
expect uptake to be in the pre-
versus post-transplant setting initially and especially
in the post-transplant setting,
who are those prescribers?
Are they more in that 60% that you mentioned earlier or less?
David.
Yeah, thank you for the question. I appreciate it. I think when you take a step back and break this down a little bit, post- transplant patients that have C3G are a critical patient for these physicians to treat because this is a recurrence opportunity for the disease. Right. They'll want to prevent that so they can protect the second kidney. I think we should view that as a very important patient type and a very big opportunity for us with the product that we have in EMPAVELI and with the broad label that we've been provided. That's the first thing. As far as the breakdown of the physicians, it kind of depends by account, but you'll see what Cedric talked about a few minutes ago.
There's about a fifth, or 1,000, one out of five patients that we have that are actually transplant patients that we can see in our data. We're going to learn more as we move forward, but that's where we're going to start now and that's certainly an important segment for us as we launch.
Thank you. Our next question comes from Lachlan Hanbury- Brown with William Blair. You may proceed.
Hey guys, I'll add Mike. Congrats and thanks for the question, I guess. David, you mentioned converting the EAP and Compassionate Use patients over the next few months. I was wondering if you can give any color on how many patients you actually have on various pre-approval access, whether it's the Open-Label Extension, early access, Compassionate Use, or some other program. Sure, yeah.
What we. Oh, go ahead. I'm sorry, Sarah, go ahead.
No, go ahead, David.
Yeah, what we've focused on here and have talked about and communicated is that we've had a very high participation in our EAP and Compassionate Use Programs, which is exciting for patients, and the majority of those patients will be moved over within the next couple of months to commercial products. That's really the focus that we've got right now, to move them as quickly as possible.
Thank you. Our next question comes from Annabel Samimy with Stifel. You may proceed.
Hi everyone. Thanks for taking the question. Congratulations. Just quick question on how realistically, how early you expect physicians to start treating these patients, and do you expect any level of restriction by Payers, like patients need to have certain level of proteinuria or eGFR before treatment, or is diagnosis sufficient for treatment?
Yeah, thank you, Annabel, great question. I think, at least the feedback that we get from the field, from the physicians, is that because of the efficacy profile that we saw with EMPAVELI and VALIANT , physicians will be considering to treat very early, earlier even in terms, or I'd say more benign, even in terms of proteinuria and eGFRs than what was studied within the VALIANT study. I think the best way to think about it is when you're a teenager or a young adult and you're looking at a disease where over the course of 10 years, 50% of these patients are at risk of losing their kidneys, you're going to want to do whatever you can to stabilize the disease as much as possible.
I think the enthusiasm is there, I think the appreciation of the data is there, and we're very excited about bringing this therapy to patients.
Thank you.
Thank you. Our next question comes from Joseph Stringer with Needham & Company. You may proceed.
Hi, good morning. Thanks for taking our question. Just curious what your expectations are on compliance rates for EMPAVELI in these patient populations compared to, say, what they have been for EMPAVELI and PNH.
Yeah, thank you so much. Caroline, would you like to elaborate?
Thank you so much. What we have seen in the clinical trial was excellent compliance rates. With continuation in the Open-L abel Extension, almost all of the patients continued on to that. We've also had excellent compliance when patients who are taking EMPAVELI for PNH. We are really excited for having this in the real world. With this combined with our amazing team that does the EMPAVELI Injector and offers patient assistance for this, we are expecting excellent compliance in the real world. Do you have anything else to add to that, David?
No. Look, I will tell you that we've been very pleased with what we saw in what we've seen in PNH. We've got 97% compliance with PNH patients, which is excellent. It's actually an unheard of number in most disease states. In the VALIANT study and the post-marketing study or the post-phase III study, we're at greater than 90% with patients that are being treated for C3G. Very excited about that and I think it'll play out in the real world based off of what we've seen on the PNH side of things.
Great, thank you.
Thank you. Our next question comes from Biren Amin with Piper Sandler. You may proceed.
Yeah. Hi guys. Thanks for taking my questions and congrats on the approval. Maybe another question on market size. Novartis in the past has talked about 10,000 patients in the U.S. with C3G, which is clearly higher than your estimate. It seems like they're calculating their patient estimate based on biopsy- diagnosed prevalent patients. Can you maybe talk about what the differences are to their number? Second question, can you quantify for us the number of compassionate use patients on EMPAVELI currently with C3G?
Yeah.
Thank you, Biren. We don't know exactly their methodology, but I think, again, as we outlined before, the number 5,000 for us is a conservative estimate. It's certainly not difficult to imagine that there's a lot of patients out there who are, for example, symptomatic, who haven't received a biopsy because no treatment was available. Quantifying that is very difficult. We'll see, as the drug gets on the market, you know where exactly that sits.
Thank you. Our next question comes from Ryan Deschner with Raymond James. You may proceed.
Good morning and congrats on the broad label from FDA. Can you talk a little bit more about your strategy for identifying and targeting C3G and IC-MPGN patients, including how big your field team is currently and what post marketing requirements for EMPAVELI and C3G and IC-MPGN? What do they look like? Thanks.
Thank you so much, David.
Yeah. I'll answer part of the question first up front on the size of the organization that's out ready for the launch. We have about 100 people across the various cross-functional teams that are 100% focused on the launch for C3G and IC-MPGN, which we are very excited about. As far as the breakdown, I think that was the next question. I think Cedric Francois went through the details in regards to how we look at the market and how we've identified the patients. Obviously, we're going to learn more as the market starts to take in a broad label that we've got awarded to us as of yesterday. I think it's important to remember that Cedric mentioned a couple times the trifecta. We're the only product that has achieved a reduction in proteinuria. We've had a stabilization in eGFR and C3 deposit clearance has been very significant.
There's obviously an opportunity for us to educate there and I think it'll span across both C3G and IC-MPGN patients as we work forward.
Thank you. Our next question comes from Douglas Tsao with H.C. Wainwright. You may proceed.
Hi, good morning. Thanks for taking the questions, Cedric. I appreciate sort of the conservative element in terms of your work in terms of coming up with the number 5,000 patients, which obviously is somewhat different than what Novartis has done. I'm just curious, I think you had said you'd offered a couple metrics in terms of when patients had had biopsies. I'm just curious what was significant about those time points and what led you to think about that being the criteria for thinking about a patient or identifying a patient from patient records and getting to the point where could we potentially see upside or the type of patient who might have been missed by that analysis but could very well be a good candidate for therapy. Thank you.
Yeah, thank you so much. Our Business Intelligence Group looks at multiple parameters and then, as mentioned already, comes up with a conservative estimate using a methodology that they feel comfortable with. I think the main unknown that could drive potentially many more patients going on therapy is going to be with those patients that are more mildly symptomatic, where again the disease phenotype is unclear and where the biopsy has not happened. It is not hard to imagine a nephrologist who a couple of years ago may have said, I suspect C3G but I don't have strong proteinuria, I'm below 1 g, let's just follow this because there's no therapy for C3G anyway or for IC-MPGN, that is now going to change. I think that's kind of the X factor that we'll have to look out for.
Again, the number 5,000 is a conservative estimate that we feel very good about.
Cedric, a follow- up if I may. Do you have from talking to clinicians, do you get a sense that with a mildly symptomatic patient with the availability of EMPAVELI now and given the strength of the data that they would want to initiate treatment earlier in order to preserve long term kidney function? Thank you and congrats.
Thank you so much. Yes, that is indeed the case. There was a meeting with KOLs in Iceland about two weeks ago where people were talking about hundreds of grams of proteinuria as already being sufficient to think about a proper diagnosis and treatment. I think it's going to create a new world, quite frankly, for these patients where hopefully we'll be able to make a big difference.
Thank you. Our next question comes from Simone Nasroodin with Wells Fargo . You may proceed.
Hi, this is Simone on Frederick. Congrats on the label. I just have two questions. One, can you remind us again on EMPAVELI's IP runway and more color on the lifecycle management programs that are underway.
Thank you so much.
We have
a patent life until 2035 with customary extensions. In terms of life cycle management, we are looking into products that will improve the convenience of the product while taking advantage of the efficacy and the safety that we have seen for EMPAVELI so far. More to follow about that.
Thank you.
Thank you. Our next question comes from Judah Frommer with Morgan Stanley. You may proceed.
Yeah, hi.
Congrats on the approval, and thanks for taking the question.
We were just curious about marketing and education efforts that are being made specifically for younger patients.
Are those patients treated by the same
Docs that the adult patients are treated and specifically for the pediatric and the 12+ year old patients that were not being included in the FABHALTA label. Are there any special efforts to go after them early? Thanks.
Yeah, absolutely. I will hand that over to David. I think.
David,
I'll take that.
Thank you, Caroline.
Some of our clinical study extended to include many pediatric nephrologists, many of these KOLs. I think that we have a good connection with this community. One of the things that they are most impressed with is the "trifecta" of our results that shows this reduced proteinuria, stabilization of the eGFR, and the resolution of deposits in the kidney. This is most imperative for these adolescent patients, ages 12 and up, where the end result of disease can be loss of even kidney transplantation. We have a good connection with these doctors. Yes, they are different than the adult nephrologists that treat this disease.
Great.
Thank you.
Thank you.
Our next question comes from Greg Harrison with Scotiabank. You may proceed.
Hey, good morning. Thanks for taking the question and congrats on the approval. Curious how you would foresee the competitive landscape playing out across disease severity.
Is it likely that maybe you would be
having higher uptake among more severe patients, whereas FABHALTA may be, you know, preferred among more mild patients? I don't know if that's the right way to think about it. Do you have any strategies for EMPAVELI to also capture patients on the milder end of the spectrum?
Thank you so much for that question. I think what stands out here is the quality of the data that we have with EMPAVELI. Also, the large safety profile that has been established already, of course, is the prior authorization on PNH and the real world experience there. I think the fact that we have the pediatric population there as well included is very important. Typically, that would be the population where the milder form of the disease is located, but where our competing product has not been tested.
I think what is important here is to understand the severity of the disease. Imagine being a young adult with a 50% chance of end stage renal disease over the course of 10 years. You're going to want to take the product that gives you the best chance of stabilizing the disease as much as possible. We have the data to support that.
Thank you. Our next question comes from Greg Suvannevejh with Mizuho Securities. You may proceed.
Good morning. Thanks for taking my question and congrats on the approval. I just wanted to ask a question on kind of how we should think about uptake. I think I heard earlier that perhaps there is a bolus of patients or there is some pent up demand for the product, which sounds great for uptake, but could you also comment on awareness of the product amongst prescribers and if there's any element of education that is needed to ensure optimal uptake.
Thanks.
Thank you, David.
Yeah, thanks for the question. Appreciate it. Like I mentioned a few minutes ago, we do think that there is some pent up demand just like you'll see in any ultra- rare disease where treatments have not been accessible or available for patients that are in a severe situation such as kidney disease. We definitely feel like that's going to be the case. What I do think for us to set the expectation on is this is ultra- rare disease. After we get through that pent- up demand, I think you're going to continue to see over time growth that we expect to be relatively steady and gradual.
We're going to find more out as we launch as we start today with the actual label in our hand, which is, as you know, very broad and covers a significant number of patient categories that are unique to us and to the brand. As far as the education in the offices, we've had the team that I talked about a little bit ago in the field since about April. Our medical team has been in the field talking about this at a significant clinical level since last year about this time. We are in a position and poised to continue to educate. My own experience, having been in the field recently with a number of nephrologists, is they're excited about the program product. They are not differentiating between, you know, where they would start it versus not start it.
They're spending most of their time thinking about patients that they could bring in, whether they're mild, moderate, or severe, because they know they're all going to progress to end stage disease state or potentially transplant. I think we're well on our way and we'll learn more as the launch continues starting today.
Thank you. Wish you well in that launch.
Thank you.
Thank you.
Thank you. Our next question comes from Lisa Walter with RBC. You may proceed.
Oh great. Thanks so much for taking my question and congratulations on the approval. You know, the indication section of the label reads pretty broad, but I'm just wondering if you can clarify for us whether post transplant IC-MPGN patients will be eligible for EMPAVELI. Any color here will be helpful. Thank you.
Thank you so much for that question. It is not included in the label, but of course, we had a couple of patients where it was tested, and the data seems to be consistent. We will do more work moving forward to find out, you know, whether we can get that ultimately in label included as well.
Thank you. As a reminder, to ask a question, please press star one one on your telephone. Our next question comes from Jonathan Miller with Evercore ISI. You may proceed.
Hey guys, thanks so much for taking my follow-up. I just had one more on the pediatric population.
You've said a lot of patients getting diagnosed that early.
You mentioned 50% to 20% of the prevalent pool is in that peds indication.
What percentage of patients are diagnosed
during the years that are covered in your label and not in Novartis? I'm thinking not just in terms of the initial launch, but in terms of how many patients are going to start on therapy where you are the only option available to them.
Thanks.
Thank you. That is an excellent question. We don't have exact numbers on that, but it's of course much higher than that. I think, I don't know, Caroline, if you have an exact number around that, but you know, it's definitely higher than the prevalent number.
Thank you. I would now like to turn the call back over to Cedric Francois for any closing remarks.
Thank you so much, and thank you all for joining us today. We look forward to updating you on our progress, and we are excited for the future as we continue to build our leadership in complement. Please reach out with any follow-up questions, and have a great rest of your day. Thank you.
Thank you. This concludes the conference. Thank you for your participation.
You may now disconnect.