Ultragenyx Pharmaceutical Inc. (RARE) September 2026 Conference Transcript

Good afternoon, and welcome to the Ultragenics Pharmaceutical Conference Call to discuss the U.S. Food and Drug Administration approval of the FAYUV for the treatment of the MPS IIIA, also known as Sanfilippo Syndrome Type A. At this time, all participants are in a listen-only mode. Following the prepared remarks, there will be an opportunity to ask questions. It is now my pleasure to turn the call over to Joshua Higa, Chief of Staff and Vice President of Investor Relations. Please go ahead.

We're in the U.S. FDA approval of Fayuvie, the first ever treatment for San Filippo syndrome type A. We appreciate all of you making time to join us this afternoon to discuss this important milestone. A press release with details on this announcement is available on our website, along with an updated corporate presentation. Joining me today are Emil Kakas, Chief Executive Officer and President, Eric Krombez, Chief Medical Officer, and Eric Harris, Chief Commercial Officer. Howard Horne, Chief Financial Officer, is also on the line for Q&A. Before we begin, I'd like to remind everyone that today's discussion will include forward-looking statements. These statements involve risks and uncertainties, and actual results may differ materially. Please refer to the risk factors described in our SEC filings. And with that, I'll turn the call to Emil.

Thank you, Josh, and good afternoon, everyone. Earlier today, the FDA approved FIUV, the first-ever treatment for San Filippo syndrome type A, and our second gene therapy approval in less than a month. This milestone represents our sixth FDA approval overall and further establishes Ultranetix as a leader in both rare disease medicine and gene therapy. Before we discuss in detail the significance of this achievement for our company and the MPS community, I want to briefly acknowledge the recent outcome from our Phase III ESPAR study for GTX-102 for Angelman Syndrome. This was truly disappointing for the company and for the Angelman community. We expect to provide a more comprehensive update by our next quarterly financial results We will address your questions on the Angelman results and our expense management plans in greater detail at that time. But for today, we're going to focus on the FIUBI approval and what it means for children in families with Sanfilippo syndrome type A. Sanfilippo syndrome type A is one of the most heartbreaking diseases I have encountered in my career. These children often appear healthy early in life. Then by ages two to six-year-old, as the disease progresses, they gradually lose their ability to learn, to communicate, and to engage with the world around them. And after a period of hyperactivity and altered behavior, the children slow down as family watch skills disappear one by one, leading to a bedridden state. The children eventually die from this devastating disease, usually as teenagers. Until now, families and clinicians have had to face this diagnosis without any approved treatment with the potential to stop or slow this inevitable decline. We're here today because parents, advocates, clinicians, researchers, and organizations that across the broader MPS community refused to accept that reality, they raised awareness, funded research, participated in clinical studies, and never stopped fighting for their children. Now, thanks to their perseverance, families receiving a diagnosis of Sanfilippo syndrome type A can have hope that their children's future may look different. Ultranex was found to pursue diseases that others overlooked and deliver treatments where none existed before. In less than one month, we've now achieved our consecutive gene therapy approvals for two severe rare diseases, demonstrating not only the strength of our science, but our ability to translate innovation into approved medicines for patients. Even though Ultranex was not originally working on Sanfilippo syndrome, we received a call from Abiona's CEO, Vish Sashadri. When funding constraints arose, despite positive clinical data, Abiona made the pivotal decision to outlysis the asset to ultragenics, ensuring this vital treatment reached the finish line for patients. When we picked up the program, our team found a way to make it work, relentlessly and courageously taking on the task, making sure the PROC on hand was able to treat kids and getting the program to a BLA filing. It has not been an easy road for us either, but we were gratified that all of this work has enabled us to bring forth a first approved treatment for Sanfilippo type A. Developing new therapies is only one part of our mission. We've also believed that approval only matters if patients can actually obtain treatment, and we are committed to helping eligible families in the U.S. access FIUV as quickly as possible. We also know and have gotten urgent calls for treatment from families elsewhere in the world, also in great need, and we will work on finding a way to treat some patients globally while we seek approval in other regions. I'll now turn it to Eric Krombes to review the clinical data, and Eric Harris to discuss our commercial launch preparations.

Thank you, Emil. FAYUV is indicated for the treatment of neurologic manifestations of muco polysaccharidosis type 3A or San Filippo syndrome type A in pediatric patients with preserved neurodevelopmental function. As Amel noted, this is a rare and fatal lysosomal storage disease that primarily affects the brain and is marked by neurodegeneration beginning in early childhood. The disease is caused by an enzymatic deficiency, which results in the accumulation of heparin sulfate and progressive damage to the central nervous system. Feiyude is a single-dose AAV9 gene therapy designed to directly address this underlying enzymatic deficiency. Conceptually, this gene therapy provides the ability to produce fully functioning enzymes in some cells in the brain to break down heparin sulfate. These cells will then secrete the enzyme that can cross-correct other brain cells, resulting in a decrease in heparin sulfate substrate levels throughout the brain. The BLA was based on data from the Phase I, II, III Transfer A study and long-term follow-up, demonstrating improvements in key developmental domains compared with natural history, along with substantial and durable reductions in CSF heparin sulfate. Clinical data now extends to nearly eight years of follow-up. FAUV was generally well-tolerated and maintained a favorable safety profile. The most frequently reported treatment emerged in adverse events for elevation and liver enzymes, which were mostly mild or moderate. While we originally submitted failure date for accelerated approval, the agency recognized the robustness of our clinical data package during the review and granted standard full approval rather than accelerated approval. Importantly, this approval covers the entire pediatric age range and reflects the deep understanding by the FDA of the unmet need for children and families affected by this disease. Over time, we expect that children will be diagnosed earlier through newborn screening, enabling the option for early treatment prior to the accumulation of heparin sulfate and irreversible damage to the central nervous system. As with all of our therapies, we will monitor clinical trial patients and an additional cohort of commercially treated patients in our disease monitoring program through 10 years of follow-up. The totality of evidence generated throughout development gives us confidence in the potential of a UVA to meaningfully alter the course of this devastating disease. With that, I'll turn the call over to Eric.

Thanks, Eric. First, I'd like to acknowledge what a rare privilege it is to have the opportunity to launch two drugs in a month, impacting the lives of patients with significant unmet needs. The approach you heard me reference a few weeks ago, understanding the need of patients and caregivers first, remains our guiding principle for this second launch. Families with children that live with Sanfilippo syndrome type A understand that this disease is progressive and relentless. Every day matters to preserve precious neurologic function, and each day without treatment risks the potential loss of critical skills and abilities that understanding has shaped our preparation we are fortunate that both gene glycos and fay uv fall within our inborn areas of metabolism franchise so there are significant synergies between our preparations for both gene therapy launches based on extensive pre-approval engagement with both medicaid programs and leading commercial plans, it is clear that payers understand the devastating nature of Sanfilippo syndrome type A and the importance of treating as soon as possible. Beyond the clinical impact, payers recognize that the lifetime cost of care for a child with Sanfilippo syndrome type A who can spend years in a bedridden state can exceed eight million dollars and that this burden grows as the disease advances. We set the U.S. per patient wholesale acquisition cost of Fayubi at 3.95 million dollars as the first and only one-time gene therapy treatment for this ultra-rare progressive and fatal neurodegenerative disease with the potential to slow or stop its irreversible decline. Consistent with our expectations for genglycose, we expect that access in the initial phase of launch for UV will flow through single-case agreements. Our UltraCare program has a proven history of helping patients and families navigate access to therapies and overcome potential barriers to care. As I shared at the approval of gene glycos, we have expanded that offering to include dedicated gene therapy guides who will help families navigate insurance coverage, assist in obtaining treatment support, and answer questions about treatment process. Treatment will be provided through a national network of qualified treatment centers selected for clinical expertise and experience in gene therapy administration. The network is highly synergistic with our footprint for genglycose, with many centers planning to offer both treatments. A list of QTCs will be available on fayuv.com once that website is live in the coming days. As with gene glycos, we have established relationships with treating community, with the treating community. Like families, providers understand that the time is of the essence for these patients and approach treatment decisions with a strong sense of urgency. So we are expecting a fairly rapid request for treatment. Bay UV is manufactured entirely within the U.S. at our gene therapy manufacturing facility in Bedford, Massachusetts, and at Andalin Biosciences in Columbus, Ohio. We believe we have adequate commercial supply to meet initial demand and will continue our efforts to scale manufacturing over time. We expect that the commercial product will be ready to ship to QTCs in approximately 30 to 60 days, consistent with the expectations we shared for genglycose. This is now our sixth commercial approval and second gene therapy launch. The infrastructure, capabilities, and experience we have built in commercializing rare disease therapies are highly applicable across our entire portfolio of approved medicines, with purposeful alignment built into our approach for FayUV. We have been preparing for these two launches for months, and we are excited to begin delivering this treatment to patients. With that, I'll turn it over to Emil.

Thank you, Eric. This approval carries special personal meaning for me, having worked on treatments for MPS diseases for more than 30 years. For the first time, families affected by Sanfilippo syndrome type A have an approved treatment option. After years of advocacy, scientific innovation, unwavering determination from a remarkable community, we have reached a milestone that many once believed might never come. Now the work begins to treat as many patients as we can, as promptly as we can. For alternative today's approval, further values the vision we've pursued since our founding of delivering first-ever medicines to patients with serious, rare, and ultra-rare diseases, regardless of scientific or commercial complexity. The approvals of both FaiUV and GenGlycos demonstrate strength to our platform, ability to execute, and our commitment to leading the future of rare disease medicine. The company received a priority review voucher upon FIUV approval, just as we did for Gen Glycos. The final delivery of this therapy did not come without tremendous difficulties during its development, and we hope this approval will revitalize investment in other ultra-rare gene therapies. The therapy was developed by Haiyan Fu and Doug McCarty during their tenure at Ohio State University Nationwide Children's Hospital and was licensed to Abiona. We thank Dr. Hayen Fu, her laboratory, the development and leadership team at Abiona, and so many patients, families, and investigators that worked tirelessly through so many obstacles over the many years to lead us to this moment of shared success. We're honored to stand alongside them, and we're committed to ensuring this approval is only the beginning. With that, we can open the line for questions. Operator, please provide the Q&A instructions.

Thank you. We will now be conducting a question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star 2 if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. We ask that you please limit yourself to one question and one follow-up. Thank you. One moment while we pull for questions. Our first question comes from Yigol Nochomovitz with Citi. Please proceed with your question.

Hi, great. Thank you. And congratulations, Emil and team. Very, very nice to see this. I guess I just wanted to probe a little bit more, if I may, on the label language with respect to preserve neurodevelopmental function. You know, can you just drill into that a little bit more? What does that mean? How is that exactly defined in terms of, you know, a Bayley score or specific retained skills or just a physician-caregiver judgment?

Yeah, I think a good question, Igal. And one of the really important pieces of this story is to let doctors practice medicine. I think the FDA were very sensitive to the fact there's a range of patients who might want to get treated, and they offered something more general about this. It doesn't say neurocognition, so it's not talking Bayley score. It's neurodevelopmental, and what they're saying is they have some perverted function, meaning in our interpretation, if someone is bedridden and unresponsive, that's someone who doesn't have preserved function. So it's leaving open the definition of what a doctor and parent wants to do with regard to level of function, some preserved function, meaning they're able to respond or have some ability to participate in life, I think it's really important that it not be Bailey and that Bailey is not part of it nor part of the discussion. So I think they offered an open-ended view of it, but I think we'd all agree that someone who's at end-stage disease is not a good candidate for gene therapy, and the goal is treat as young as possible. So this issue is more of an issue at the start because as time goes on, we should be newborn screening and treating kids before they're age two, even before age one. And we have some kids treat at six months have done really well, and I think that's when we've got to get to. So right now they've offered something broad in terms of preserved neurodevelopmental function and did not set any testing standards on what that means, and I think it was very thoughtful of the FDA, and I appreciate their thinking through of letting doctors and patients decide for themselves what's the right decision on treatment.

Okay, great. And if I could just do one follow-up for Howard or for you, Emil. Just could you comment on the timelines to monetize the two PRVs which are important for your cash position?

Well, I'll let Howard answer that one.

Yeah, Miguel, thanks. Same answer as the last time. We will monetize both of them. we'll monetize the first one expeditiously and the second one, you know, when we think the time is right.

All right. Well, congratulations again.

I think it's a good market for them, so we expect to be an important addition to our cash position at the company. You know, puts it in a very good position. Next question.

Thank you. Our next question comes from Anupam Rama with J.P. Morgan. Please proceed with your question.

Hey, guys. Thanks so much for taking the question, and congrats on the approval here. The press release talked about shipping to qualified centers in the next 30 to 60 days. Can you talk a little bit about how many centers of excellence we're talking about here that are equipped for infusions today and how that could grow over time? Thanks so much.

Sure, and I think Eric Harris can provide a little bit on that. Are you able to do that, Eric, on the numbers? But there's a few dozen of them already, but it's growing. Eric, Harris, did you want to answer?

Yeah, sure. Overall, there are about 120 or so centers that focus on these areas. But with regards to qualified treatment centers, we have over 25 under contract now, and they are growing daily. I get updates almost every day, it seems like now, with us adding another treatment center. And as consistent with what I said during the Gen Glycos call, we expected to get to 40 or so by the end of qualified treatment centers by the end of the year. more than enough to meet the initial demand that we're expecting for both products. And most of the sites are dual sites for both Gen Glycos and Fayuvie.

Congrats again on the approval, guys.

Thank you.

Your next question comes from Yaron Werber with TD Cohen. Please proceed with your question.

Good afternoon, management. Thank you so much for taking the question and congrats on the approval. It's an important day for patients. This is Stephen Ayanoff on For Your Own. Among the 3,000 to 5,000 patients that you've identified as prevalent within the Sanfilippo type A community, what, based on the neurodevelopmental stage, would you consider the pool that would be most accessible or most, I suppose, the first logical treatment candidate for FIUV? and how many of those patients are identified and present at current qualified treatment centers. Thank you very much.

Yeah, so right now we're aware of, in the United States, around 300 to 400 patients that are potentially within the range. With regard to the development function, obviously people don't post that routinely about their patients, But generally, when patients reach the 10, 11 years old, they start to be at the end of their period. They become bedridden often as teenagers. So we're talking about patients generally are going to be younger than that, probably less than 10 or 11, more likely. The average age will be more like 5. But we'd expect there might be patients up to age 10 that might be still in function. We haven't precisely put it down the list yet, but there's a lot of patients to treat already around, and our hope is to treat as many of the ones that are prevalent now and hopefully to enhance the diagnosis of the young ones where the most good can be done before age two or really before age one. But we don't have a precise number, but that gives you at least enough idea for the U.S. right now.

And just to confirm that 300 to 400 within the range, that range you're referring to is uh the range of ages that you were just speaking of right yeah it's about that yes thank you and congrats again thank you our next question comes from maury raycroft with jeffries please proceed with your question hi congrats uh on the update thanks for taking my question um for eric's comment around the expectation for rapid requests for treatment is there more color on that for providing what could initial demand look like and there more perspective on what that could be for what patient

demand for 2026 maybe through 2027 to look like well I'll let Eric answer in a second but we generally not going to put out forecasts or guidance on what's going to happen in the first six quarters that's been our habit so we don't want to overstate and create an issue we are going to work promptly to to get as many as we can. We have enough treatment centers up, and they continue to add them to service patients. So we do think that the drug is going to do well. That is, we think that there's a higher C-treat, and we have product available, and we expect to do well. But right now, Moria, I don't think we're going to give any precise predictions on the ramp yet. But I realize that's a very important issue for gene therapy because there have been, you know, some ones have done extremely well and some they've done very poorly. I don't know, Eric, Harris, is there any other color you would provide on that?

Nothing else to add at this particular time. But other than that, there's very strong interest from patients and the patient community. Got it.

That's helpful. And maybe just one follow-up for the price. Do you expect that some payers are going to require an outcomes-based reimbursement arrangement? And what could that look like? Or is this something that you're offering proactively to payers?

I think we expect in May. I don't know, Eric, do you want to comment on OBAs?

We'll be prepared to engage payers regarding OBAs if that is something that we'll get them more comfortable with providing reimbursement. But we're not expecting to have to do that with the majority of the payers. Got it. Understood.

Congrats again. Thank you. Thank you.

Thank you. Your next question comes from Kristen Kluska with Cantor. Please proceed with your question.

Hi, all. Congrats on this approval and appreciate you taking this program forward so that these patients now have an option. So I wanted to ask on patient identification efforts. I remember a couple years ago at World, one of the themes was looking at different facial recognition features for different MPS patients to help identify it. And that was before this AI wave. So I'm curious if there are ways you can be creative about trying to find more patients.

Yes, I think there are. I think the facial recognition for San Filippo is probably the weakest feature because they have the least effect on the facial features, where for other MPS, there's much more bone effect and therefore more facial effect. The big thing really is the early screening for developmental delay, any developmental delay or anything to help identify these cases before they've gone too long. And I think the challenge with that, of course, is that once some developmental delay, that means they're already having some brain effect going on. The truth is that the best thing is to work on newborn screening and to get it advanced into newborn screening. There is already MPS1 and 2 newborn screening or on the RUSP. We need to get this added. We have been supporting work to have the same type of enzyme assay that's used for those others to be used for screening, and we think that needs to get implemented. I think the RUSP process needs to change. The committee has come apart right now, but Secretary Kennedy has been nominating, putting things on the list on his own without an advisory committee. We need to get this on that, and we need to get it implemented across the United States so these kids are getting treated when they're like six months old, which I think will give us the best effect. So there are some ways to screen symptomatically, but ultimately it's got to be newer screening, and we've got to press for it, and we're actively doing that. Okay, next question.

Your next question comes from Salveen Richer with Goldman Sachs. Please proceed with your question.

Hi, this is Lydia on for Salveen. Thanks so much for taking your question, and congrats on the approval. Could you just speak to the overlap with the prescriber base with your existing commercial franchise and also the sales force that has been deployed for GenVycos? Thanks so much.

Certainly. There is a big overlap. the majority are medical geneticists that make the diagnosis, and so there is a very large overlap. And I can let, in a moment, Eric wants to add a little more about that overlap. So medical geneticists, which is what I'm trained as, make the diagnosis commonly. There are some cases, areas where a pediatric neurologist might make the diagnosis for San Filippo, where there might not be a medical geneticist. So those are a couple of the specialties. I don't know if you have anything else to add, Eric Harris, on that.

Yeah, and you asked a question about our current field team. As stated with the GenGlycos launch, this falls right into our Inborn Errors of Metabolism franchise. So the same team that has been calling on these centers for years now for both Mepsevi and De Jovi will also continue to call on these same institutions. And we estimate probably 75% or more overlap. So this will be a highly leveraged franchise that has established relationships with many of these doctors already.

Thanks so much. Your next question comes from Ellie Merle with Barclays. Please proceed with your question.

Hi, this is Tejas on for Ellie. Thanks for taking our question. As you begin to launch both of these two therapies in the U.S., can you remind us what key metrics you'll provide for the launch, like start forms or something similar? And then in terms of the treatment timeline, how should we think about the timing between start form and patients eventually receiving drugs?

So with regard to, we'll be talking about how many QTC centers we have up and running and ultimately the number of patients that have been treated. So we could do start forms, but I guess it's really treated is probably more an accurate And the second question?

Time to treatment, identify to treatment.

The time to treatment will obviously be evolving over time. I mean, in the beginning, they're all exceptions and we're waiting for their policies. Eric, do you have anything to say, I think, about time to treatment? Well, obviously it matters a lot for Sanfilippo, so we don't want to be – it can't take a long time. We need to drive this along. We've told all the payers, by the way, that it needs to be treated like Zolgensma for SMA. Eric, any thought on time to treatment?

Yeah, so we have several patients in process right now as they work and consider treatment. And, in fact, we've already received some initial start forms for genglycose. And as we stated, we'll work with the patient and the caregivers through the reimbursement process and expect these patients to, we'll expect probably to be available within 30 to 60 days, and that's on track. So I think somewhere in that time frame, we should have our initial treatment of patients for both therapies.

So something like 30 to 60 days to time to treatment. I think with ViUV, we're going to want to, as time gets going, we'll want to make it as tight as possible. And we've actually set up our systems and the testing that needs to happen and other things to make sure we can move this along as promptly as possible. Thank you for the question. Let's go on to the next question.

Your next question comes from Joe Schwartz with Lyrinc Partners. Please proceed with your question.

Thanks and congrats on the approval. Given the release frame's global access as a longer-term goal, I was just wondering what's the timing for other territories in particular? Where does the MAA and the EU's stand. And then can you remind us of the Aviona economics in terms of things like the approval milestone, if there is one, royalty rate, and is there any sharing of any proceeds from a PRV?

Thanks.

Yeah, we can comment. I'll let Howard comment on some of that in a moment. With regard to global access, our plans are we are going to file in a number of countries shortly or have, and we'll probably put out a little more detail a little bit later, but we expect to work toward Europe particularly soon. But we're also seeing many named patient inquiries from the Middle East and other places for So we'd also expect to support named patient treatment outside the U.S. in the meantime, while we are also pursuing formal filings and approvals.

Yeah, I'll take the Abiona economics, and there's more on this in our corporate deck and on our other filings. But the short answer is no sharing of PRV, and the commercial milestones, I think, range up to about $30 million or so, and there's a mid to high single-digit royalty. Thank you.

Your next question comes from Ben Burnett with Wells Fargo. Please proceed with your question.

This is Tien Chi calling for Ben. Congratulations on approval. Understanding that, you know, the target prescribing physician is largely kind of same, but can you please just quantify how much of, you know, maybe expenses that will be associated with this launch? Thank you.

Sure. Well, we're actually leveraging almost everything that's been gone, and the few things we added for GenGlycos will be used here as well. So it's actually a relatively modest incremental spend for us because the field team was already selling DelJolvi and Mepsevi, and that team's already out there, and maybe there's a few people. But I would say the expense of being relatively modest. We do have a gene therapy treatment team, like a physician, to help manage the ongoing treatment, but we already set that up for GenGlycose, and they're managing both. So the true inclemental expense would be modest, and it's going to really leverage a lot of what we've done before. So we think this will help it become accretive to us more rapidly with that base cost already being covered.

Thanks, and congrats again on approval.

Thank you.

Your next question comes from Maxwell Score with Morgan Stanley. Please proceed with your question.

Great. Thank you very much for taking my question, and congratulations. I was just wondering if you can provide any insights into gross-to-net, particularly in the early stages of the launch. Thanks.

We can sometimes. There are obviously a number of government discounts. Howard or Eric, do you want to comment on that?

Yeah, so what Eric had said earlier is that our WAC price is going to be $3.95, and what we've shared in the past is that our net range is $2 million to $4 million, which, of course, may not be all that insightful because it includes the WAC price. We can help sharpen it maybe later, but that, at the moment, is what we've shared.

Great. Thank you.

Your next question comes from Jack Allen with Baird. Please proceed with your question.

Hi, thanks, everyone. Good afternoon. Congrats on the approval. This is Chris on for Jack. Kind of just piggybacking off that last question, you know, commercial opportunity, I know it's early days, but, you know, just back of the envelope, Matt, it seems like, you know, your projections, you know, could be a billion-dollar drug. Just your thoughts on that. And then just one on CMC. I know you, you know, you made some alterations to that in response to the CRL, to 111, and just anything from those changes that you think might be able to help in glycos and type A. Thanks.

Yeah, well, on the second one, we do the cell finish in the same place, so the changes we made helped work with each other in terms of CMC. We didn't change the process at this point. The process being run is a relatively smaller scale process. We opted not to try to change process in the middle here, but we will probably expect to scale the process further as time goes on, as we move ahead. We haven't talked about the commercial opportunity. I would say we are not thinking of it as a billion-dollar opportunity. I think that would be a reach. But we do think it's a substantial opportunity. And as we can say very clearly from history, whatever you're in gene therapy, that the size of the opportunity is not necessarily just the patients, the number of patients exactly, but it's the urgency and how many get treated. I think in San Filippo the urgency will be high, though the population is relatively smaller, we will actually see a lot of these patients want to get treated and get treated quickly. They're not going to wait and see. So I do think it's going to be a very reasonable opportunity, probably larger than what people think, but I don't think that it's a billion-dollar opportunity. I wouldn't want people to believe that.

And then, Jack, let me pull a few things together that have been answered in different parts of today. So 3,000 to 5,000 patients in commercially addressable markets, and we've talked about maybe a quarter of those in the U.S. WAC price, we've talked about a net price. We've talked about WAC of 3.95 and net somewhere between two and four. We've talked about the fact that we have a pre-existing field force that doesn't really need to be augmented much to get this out there. And we've talked about a PRV coming along with it. So those are building blocks of the model that we've shared with folks.

Got it. Thank you and congrats again.

Thank you.

Your next question comes from Laura Chico with Wedbush Securities. Please proceed with your question.

Thank you very much. Congratulations, Emil and team on the approval. I guess just one, and I don't know if I should direct this towards Eric a little bit, but I'm trying to better understand what is the capacity of these centers to treat? And I believe I heard about 40 centers, but I guess I'm trying to understand. Obviously, there is a high urgency to get folks in the door here, but there's also a pretty complicated cases. So just wondering if you could expand on what happens to get centers up and running and what that capacity looks like. Thank you very much.

Yeah, well, I think Eric had told you we had 25. We're adding them regularly. In fact, some of the first gen glycos patients were at new centers. So we actually have a whole team set up to set up and train centers and train them wherever they are. We're not going to be stiff about requiring only certain centers. We made them to be quick as possible, but based on payer issues, region, state issues, we have to make sure to be adaptive, and we are planning to be. I don't think capacity of the centers to treat patients is going to be a limiter in our ability to commercialize at all. I think we've got plenty of QTCs, and we will add them as needed, wherever they're needed, to make sure we are treating everyone that needs to get treated. So right now, I don't think that the QTC capacity is going to be an issue. I don't know if you had anything else to say, Eric Harris, on it.

That's right. I mentioned we've grown more than, added more than 25 now, expect to get up to 40 or so by the end of the year. We'll keep adding as we move forward with expanded demand. So I don't want you to think we're going to cap the number of treatment centers at 40 will continue to expand as needed.

Thank you very much. Congratulations. Expansion, maybe. Yeah, good. Okay. Thank you, Laura.

The last question comes from Ram Selvaradjou with H.C. Wainwright. Please proceed with your question.

Hi. This is Jadon for Ram. Thank you so much for taking your question and congrats on the two gene therapy launches Could you just discuss quickly what real-world evidence data is likely to be collected following the launch in your patients?

Well, real-world evidence is talked about a lot. We actually believe in another model we call the Disease Monitoring Program model, which is a fully sponsored model where we collect accurate, high-quality data and support the doctors and the patients and getting that data, which will allow us to do it with great precision and quality, with limiting missing data. The FDA has accepted the DMPs now for all six of our programs that have been approved as a single postmarketing commitment in which we do all the clinical work. It's a different model than real-world data, but we will collect data in our programs for up to 10 years, but all the patients in the program are on commercial drug, so they don't get drugged as part of this program, they get monitored or they get testing done by The difference then with real-world data, instead of using electronic records and other sources to pull up information, we're able to quantitatively, more like a study, measure individual data. Now there's no doubt that claims data or other real-world data will be a supplement to it, but it's not our main focus. We definitely think there's an opportunity to use real-world data, it just won't be the the main thrust of our approach to monitoring patients long-term.

Great, thanks for explaining that. And just quickly, could you discuss the mild label restrictions on liver function, particularly as it pertains to AAV therapies?

Sure, I'll let Eric do that. I think the general view is that all AAVs go to the liver, and so you don't want to have a liver that's highly injured so that you don't hurt them. But Eric, do you want to say anything about them? Eric, Krombus?

Yeah, right. Thank you. Yeah, so certainly no restrictions in the indication. You know, we always, as Amal said, wanted to dose patients with relatively healthy livers, but that's not an issue here. That wasn't like a screening issue or something that we really encountered while we were enrolling these trials, so we don't expect it to be an issue at all in the commercial setting either.

Yeah. The sample patients don't have liver injury. There are some disease where there is, but they usually don't have significant liver Thank you for the question.

Thank you so much.

I think we have the last question coming from Sammy Corwin at William Blair. Sammy, go ahead.

This is Josh on for Sammy. Congrats on the approval. We were wondering what launch metrics the company plans on sharing, and how soon should we expect those updates?

So, we're… Do you want to put it? And we'll tell you about how many QT centers and how many patients have been treated for both diseases. That will be it. We'll probably be doing that on our quarterly calls is our expectation. And at points where we're providing revenue numbers, we'll provide revenue numbers. But those will be the metrics right now. I think those are the ones that are important. It should give you a feel for how things are going. By the, you know, the first call that's coming here, I'm not sure there's a lot to be said yet, but we do expect then every quarter to provide qualified treatment centers, number of patients treated, revenue numbers for each program.

Okay.

Very good.

This now concludes our question and answer session. I would like to turn the floor back over to Joshua Higa for closing comments.

Thank you. This concludes today's call. please reach out to us at IR at ultragenyx.com if you have any additional questions. Thanks.

Ladies and gentlemen, thank you for your participation. This does conclude today's teleconference. Please disconnect your lines and have a wonderful day.