Good afternoon, and welcome to the Ultragenyx Pharmaceutical conference call to discuss the U.S. Food and Drug Administration approval of GENGLYCOS, known as DTX401, for the treatment of glycogen storage disease type Ia, or GSDIa. 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. Thank you, Joshua. Please go ahead. Thank you, and good afternoon, everyone. We appreciate you all for making time to join us on such short notice to discuss this important day for Ultragenyx. The press release we issued announcing the approval of GENGLYCOS is available on our website at ultragenyx.com. Joining me on today's call are Emil Kakkis, Chief Executive Officer and President, Eric Crombez, Chief Medical Officer, Erik Harris, Chief Commercial Officer, and Howard Horn, Chief Financial Officer. Before we begin, I'd like to remind everyone that during today's call, we will be making forward-looking statements. These statements are subject to certain risks and uncertainties, and our actual results may differ materially. Please refer to the risk factors discussed in our SEC filings. With that, I'll turn the call over to Emil. Thank you, Josh, and good afternoon, everyone. Today is an important day for Ultragenyx and for the GSDIa community. Earlier today, the FDA approved GENGLYCOS, marking Ultragenyx's first gene therapy approval and the fifth approved medicine in our company's history. It is also the first ever FDA-approved treatment designed to address the underlying cause of glycogen storage disease type Ia, representing exactly the type of breakthrough therapy Ultragenyx was built to deliver. This is a day that thousands of people living with GSDIa in the United States and elsewhere, and the families and clinicians who care for them, have hoped and advocated and fought for over the years. Until today, the only thing standing between a person living with GSDIa and a life-threatening episode of hypoglycemia was cornstarch taken as a slurry every three to four hours around the clock, day and night. Now there's an additional treatment with the potential to reduce their dependence on cornstarch and ease the constant burden of care that defines their lives. We're grateful to everyone across the GSDIa community who helped make this today possible, and we want to especially thank the patients and families for participating in our clinical trials. As a reminder, our randomized placebo-controlled phase III study met its primary endpoint with a statistically significant reduction in daily cornstarch intake and was also positive in multiple secondary endpoints. Late during the review, the FDA decided to consider cornstarch reduction as a surrogate endpoint and so approved GENGLYCOS by accelerated approval pathway. The FDA requests additional evidence to confirm this reduction associated with clinical benefit and improved fasting tolerance over time. We agreed to generate that data through enhancements to our existing disease monitoring program, or DMP, and Eric will share some additional details of our post-marketing program when he walks through our clinical data. Ultragenyx was created to lead the future of rare disease medicine, and that vision goes beyond the moment of approval and into our conduct as a commercial company. We are committed to helping patients access the treatments they need and reduce financial barriers that may stand in their way. GENGLYCOS extends that leadership into gene therapy, and it does so as a medicine we manufacture end-to-end entirely in-house at our gene therapy manufacturing facility in Bedford, Massachusetts. With that, I will turn the call over to Eric Crombez and Erik Harris to walk you through our data and launch plans, and finally Howard Horn to comment on the PRV and our expectations for launch. Thank you, Emil. The approval of GENGLYCOS fulfills our commitment to provide the first therapy that directly targets the root cause of GSDIa. The reduced reliance on cornstarch demonstrates this liver-directed gene therapy's capability to deliver the necessary transgene to enable patients' livers to break down glycogen and to produce glucose during fasting or metabolic stress. This ability to regulate glucose levels has alleviated the disease burden and mitigated the risk of severe or life-threatening hypoglycemia for these patients. GENGLYCOS is indicated for the treatment of adult and pediatric patients eight years of age and older with glycogen storage disease type Ia who do not have antibodies to AAV8. The BLA was based on data from our phase III randomized, double-blind, placebo-controlled study, which enrolled 46 patients aged eight years and older. Results from the phase III study show that patients treated with GENGLYCOS reduced daily cornstarch requirements compared to placebo while maintaining glycemic control. GENGLYCOS was well-tolerated with an acceptable safety profile. The most common treatment-related events were transient elevations in liver enzymes that were generally non-serious and managed with a prophylactic corticosteroid regimen. The results across the entirety of the clinical development program, which encompasses data on 52 treated patients over eight years of follow-up, speaks to both the magnitude and to the durability of effect of this gene therapy. In regard to the post-marketing requirements that Emil referenced, we will collect two years of data from 50 commercially treated patients through enhancements to our existing disease monitoring program, evaluating daily cornstarch intake and time to hypoglycemia in a controlled fasting challenge. We will also follow 20 untreated patients who have AAV8 antibodies over the same time period as a control group. Unlike the phase III study, this DMP evaluation will be performed in the open label setting with patients and the physicians receiving real-time glucose measurements. We believe that this will allow for greater corn starch reduction and improved metabolic control compared to what can be achieved in a blinded study without real-time glucose information available to patients. As a reminder, the use of a DMP is the strategy that we have used for four prior approvals as the sole and comprehensive evaluation of all of our treatments in the post-marketing setting. The GSDIa DMP will collect 10 years of treatment data across clinical study participants and new commercially treated patients. This DMP is already active and enrolling clinical trial patients as they cross over from the phase III study to long-term follow-up. With that, I'll hand it to Erik to discuss how we are bringing GENGLYCOS to patients. Thank you, Eric. As we have approached each launch in our portfolio, our guiding principle has been to start with understanding the needs of the patients and caregivers. The repeated success of this approach has informed our preparation. Supporting patients is at the center of our work. Our UltraCare program has an established track record of helping patients and families successfully navigate access to our approved therapy. Treatment with gene therapy is a multi-step process. So we have adapted our offering with that reality in mind. We have established a new dedicated role called UltraCare Gene Therapy Guides, who will help patients navigate insurance coverage, assist in obtaining treatment support, and answer questions about the treatment process. In coordination with providers, treatment will be delivered through a national network of qualified treatment centers, institutions with specialized expertise and training to safely administer gene therapy. We selected our network of centers based on clinical experience with GSDIa and gene therapy administration, as well as for a geographic footprint that is right-sized for anticipated demand while minimizing travel burden for patients and their families. Our teams are being deployed immediately upon this approval to train teams at QTCs on the final label and will continue to onboard and train centers to ensure medical readiness on an enrolling basis as contracts are finalized. A list of QTCs will be available on ultragenyx.com once that website is live in the coming days. As we've mentioned previously, there is roughly 75% overlap in GSDIa providers with the treating community we know well from MEPSEVII and DOJOLVI. Our commercial organization is already in the field and able to leverage established trusted relationships with key providers. Shifting to payers. Our work to lay the foundation for quality coverage has been extensive, with numerous interactions across state Medicaids and large national payers. Consistently, payers appreciate the severity of GSDIa and associated unmet needs. We are also encouraged that they recognize a reduction in cornstarch dependence as a clinically meaningful endpoint that represents the potential to deliver substantial impact for patients, caregivers, and their families. We have set the U.S. per-patient wholesale acquisition cost of GENGLYCOS at $2.7 million, reflecting its potential to reduce patients' reliance on cornstarch and enable better metabolic control of glucose. As Emil emphasized at the outset, we have a strong commitment and track record of supporting patients and families in helping to access our approved medicines, and that approach will extend to gene therapy. Consistent with what's been observed for other newly approved gene therapies, we expect access will flow through single-case agreements in the initial phase of launch. We are familiar with and experienced in this pathway. We stand ready to help patients and providers in navigating the process. GENGLYCOS is manufactured entirely in-house at our gene therapy manufacturing facility in Bedford, Massachusetts. We have existing commercial inventory to meet anticipated demand and will continue to scale production as the launch continues. I'll close by reminding you this is our fifth commercial launch in rare disease. We are leaders in navigating, or in some cases building, complex paths from product to patient. Our team brings the experience, agility, and most importantly, the commitment for patients as we deliver our first gene therapy in the commercial setting. With that, I'll turn to Howard to touch on launch expectations. Thank you, Erik. Consistent with our practice, we plan to provide revenue guidance once we have sufficient visibility into market dynamics, which historically has been approximately six quarters after launch. In the interim, we look forward to updating you on metrics such as how our QTC network is growing and ultimately the number of patients that have been treated. With clear urgency to treat supporting patient demand and our highly leveraged commercial model, we expect GENGLYCOS will make an important contribution to our profitability. Additionally, I can confirm that Ultragenyx received a priority review voucher for GENGLYCOS' approval. We plan to monetize the PRV to bolster our balance sheet and support our path to profitability. With that, I'll turn it back to Emil to close. Thank you, Howard. Developing first-ever treatments for rare and ultra-rare diseases is why we exist as a company, and because we believe we have a responsibility to put the best available science to work for patients and families who are too often left behind. Our teams are prepared to bring a new treatment option for GSDIa to patients that need it. The key characteristic of successful gene therapy launches is related to the urgency of the disease, and GSDIa is an urgent disease with round-the-clock demands on patients every day and night, without holiday or break. The gene therapy design to deliver the missing enzyme is the ideal way to address this severe ultra-rare genetic disease. We look forward to updating you on our progress. In closing, I want to pause again to reflect the enormity of this milestone. It's a first for us, for our gene therapy manufacturing facility, and most importantly, for the GSDIa community. After 50 years of cornstarch as the only available option for patients or families, it's extraordinarily meaningful to be able to take advantage of the science that exists to deliver an option designed to directly treat the underlying cause of their disease. This approval reflects the work of a remarkable team spanning many years and organizations. We're deeply grateful to the original team from Dimension Therapeutics who worked on the program, and to Dennis Chi, who did some of the early research at NIH. We're also thankful to Dr. David Weinstein, whose scientific leadership laid critical groundwork and whose support was involved in the early clinical trial. We must also thank the patients, the families, and PIs, investigators who made our clinical studies possible, and the myriad of Ultragenyx employees throughout the company who supported this program through development and across the finish line. Their collective commitment and perseverance have transformed a scientific vision into a new therapeutic option for patients. Operator, please provide the Q&A instructions. Thank you. We will now be conducting a question and answer session. We ask that you please limit yourself to one question and one follow-up. Thank you. If you would like to ask a question, please press star one on your telephone keypad. A confirmation tone will indicate that your line is in the question queue. You may press star two if you would like to remove a question. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. One moment please while we poll for questions. The first question comes from the line of Anupam Rama with JP Morgan. Please proceed with your question. Hey, guys. Thanks so much for taking the question and congrats on the approval. Really cool to see. Can you walk us through the segmenting of this GSDIa market? What portion of patients are treated at sort of medical genetics centers of excellence, and how are you defining the various call points here? Thanks so much. Yeah, no, it's an interesting question. The majority of the patients are treated by medical genetics doctors. That's where they usually get diagnosed and usually where they're managed, although there are some endocrinologists who do manage the patients. As I think Erik mentioned, we have about 75% overlap with the doctors we're already seeing. So we leverage our commercial footprint pretty well right now, and we're not really concerned about it. We also have our patient find program, which will go out and find patients who may not be at the centers where we're operating in to discover where they are and help bring them into the fold or add new QTC centers or call points. But right now, we think the majority will be within our catchment area, and we'll always be looking to find those other patients as well. I do think we can leverage our investment in the field that we've been building over the last few years. Thanks so much for taking the question. Congrats again. The next question comes from the line of Yaron Werber with TD Cowen. Please proceed with your question. Yeah, terrific. Let me add that this is really a terrific milestone. Congrats. I know this is years and years of work. I have maybe just a couple of questions. The first one, Emil, you mentioned inventory and the confirmatory work as part of the DMP. Is that going to be under commercial settings or is that going to be as a clinical study protocol? Then finally, just remind us, because this is a big milestone also for your risk for the MPS IIIA factor, what's the overlap? Thank you. Yeah. Regarding the inventory, we have inventory to cover the launch and the confirmed 50 patients are commercial patients treated commercially. As we are treating commercial patients, if we find some patients who have antibodies to AAV8 or cannot qualify for treatment, we will include some of the 20 of those to be in the control group. We will conduct that through our DMP program, but they are commercial patients, revenue-generating patients. That allows us to do a larger program and allows us to get more data, and that was, we think, a better way to go. It is a high-quality, fully sponsored trial, though. All of our DMP programs, we do not do registries. We do DMP for all post-marketing. It is our way of solving the question of how to get high-quality post-marketing data and put the money where it means something, where you can do something with it. In regard at what it means for MPS IIIA, the GSDIa is fill-finished and produced at the same Bedford plant where UX111 is also fill-finished. So there is overlap in the facilities. We cannot speak yet to comments about the UX111 review, which is ongoing, but the plant is approved now to do fill-finish for GSDIa, so obviously that has some read-through on the processes and conditions there required to get approval for fill-finish that overlap with the UX111 program. Thank you, Yaron. Thank you. The next question comes from the line of Kristen Kluska with Cantor Fitzgerald. Please proceed with your question. Hi, guys. It is Ross on for Kristen. Thank you for taking our question and congrats on the approval. We were curious if you guys could provide any color as it relates to the treatment center capacity. Also, how many treatment centers do you expect to have online by the year-end and in 2027? Thank you. Good. I'll put a few high-level comments if Erik wants to give a little bit more color on the number. Our goal with the treatment centers is to set up enough centers to be able to accommodate all the patients we need, but we also expect that we're going to have to continue adding. We've done a lot of work in building up a network, and we haven't put out the exact numbers, but Erik, you can provide maybe a little more of where we are on building QTCs. Yeah. I won't give out the exact number. We feel confident we have a sufficient number of QTCs under contract now and expect that to expand very quickly now following approval, and with the potential to double by the end of the year. So we'll be able to meet the initial demand. As I also stated, we'll continue to add treatment centers as demand grows. Thank you. Thank you. The next question comes from the line of Yigal Nochomovitz with Citigroup. Please proceed with your question. Hi, Emil and team. Thank you, and congratulations as well on the approval. I just wanted to ask what triggers the full approval. Do you have to wait for the two years for the safety and efficacy data from the 50 patients and 20 control, or does the FDA also want to see some additional duration? You mentioned that you are going to be following these patients for a total of 10 years. Also, if you could please comment just what percent of the population do you expect would be positive for AAV8 antibodies? Thanks. The commitment is to complete two years of data for the conversion approval and to study endpoints we have studied, the number of doses of cornstarch, as well as the time to hypoglycemia between 70 and 54. Those are endpoints we have evaluated, so we are comfortable with that evaluation. We are going to do it now in open label treatment format, which will allow the treatment to be implemented, let's say, in more real-world setting. It is two years of that. We will follow patients for 10 years as part of our commitment we do in all of our programs. It is not part of the confirmatory program. It is part of our general commitment. Gene therapies in general have a longer lead time in monitoring for safety in any case. We as a company commit to investing in these longer-term programs as part of our commitment to the rare disease community. Regarding the percent of the population, it is varied. It is maybe a fourth of the patients, but it is around that range. It varies in different areas and regions, so I do not want to give you a precise number, but it is around that range. We expect it to be similar now. So that is where we are at the current moment. Great. Thank you very much. Thank you. The next question comes from the line of Ellie Merle with Barclays. Please proceed with your question. Hi, this is AJ Khan for Ellie. Thanks for taking our question. Could you just remind us about the plans and timelines for ex-U.S.? Then with about three-quarters of that population outside the U.S., how do you see that dollar opportunity shaping up relative to the U.S., given all the different pricing and reimbursement dynamics there? Thanks. AJ Khan, could you give me the first part of your question? I missed something. It just broke up a little bit. First part of your question again. Sorry, just on the latest timelines and expectations for ex-U.S. approval and the relative size of the opportunity. Okay. Yeah. So we do expect to commercialize elsewhere, and we think that we're within the worldwide price band that has been successful for other programs. I think it depends on the level of urgency of the disease, in fact, whether we're successful elsewhere. We haven't put out the exact timing. We are planning and will be filing and have filed in other territories. The commercial value of U.S. relative to the rest of the world, we usually consider the rest of the world as being a larger fraction of the total market. But in gene therapy, that may not be true. The pricing may have more pressures, and we're going to have to work through that, but we actually think there's already been some price standard set that are above even where we are with this particular ultra-rare disease, and we think we have excellent randomized control data to support it. So we believe we're going to find a place to be able to commercialize and make substantial revenue ex-U.S. as well as U.S. So we're not looking at this as a pure U.S. play. We think we're going to look at it globally, and we certainly have operations in Europe, Latin America, Japan, as well as supportive distributions in other regions like Middle East, et cetera. So we've gotten inquiries from around the world for this disease and getting treated. So I expect there will be demand and important supply, and contribution to our future revenue. Thank you. The next question comes from the line of Salveen Richter with Goldman Sachs. Please proceed with your question. Hi, this is Lydia on for Salveen. Congrats on the approval, and thanks so much for taking our questions. Could you just speak to when you expect GENGLYCOS to be commercially available and your expectation for the shape of the early launch curve here? And just a timeline for when patients initially engage with their healthcare provider to actually getting the treatment. Thanks so much. Yeah. We would expect it to be available within 30-60 days from that timeframe. There is a process where FDA also releases, and that process is ongoing. We're putting the last pieces together, but it should be relatively soon. We haven't put forward launch curve or the revenue expectations. We don't like to do that because we think there is urgent need and a number of patients that want to get treated. Rather than project out, we're going to let the market work and then provide some updates on that. The last item was about the doctors. Is that right? Just a timeline from the initial interaction to actually when the treatment would occur. Oh, I see. We don't have you in how long it takes to have a start form and turn that into a treatment event. I'd expect in the beginning it's all going to be operating off of exceptions, right? Of case-by-case exceptions. I don't know, Erik, is that something you'd want to touch on a little bit here about that process between start form to getting treatment implemented? Yeah. Emil, I think they also want to know how quickly can you work up a patient to get treated, but let me take the enrollment. Once we receive the enrollment form, we will start working with the patient to navigate the reimbursement process. As I stated in the opening remarks, they'll take some time to work through their policies for treatment. But we expect, based on our discussions that we've had with them, and they recognize the unmet need and the value that this product brings, that we'll be able to get patients approved on a case-by-case basis. Yeah. The workup part is the main thing they have to get is an anti-ADA antibody titer. They have to have the direct diagnosis, but our patients generally have had it already. ADA antibody should not be a particular issue. Then, when they get the gene therapy, they don't start any immune modulation or steroids till afterwards, so there's no other preoperative effect going on. It's really, I guess, anti-ADA antibody and start form and reimbursement process. Thank you for the question. Thank you. The next question comes from the line of Maxwell Skor with Morgan Stanley. Please proceed with your question. Great. Thank you very much, and congratulations on the approval. I see the label asks for steroids in every patient and six months of liver monitoring. Is that a hurdle for any of the QTCs? Can you just walk us through how that looks and any color around that, the monitoring process would be very helpful. Thank you. Sure. I'll let Eric add some color, but it's not a big deal. We've been doing this now in the trial, so we have a comfortable plan where we start the steroids on a time basis at two weeks, and then they're monitored at intervals. I don't know, maybe Eric can provide a little more color. But they're pretty used to this. This is pretty common and standard. I don't think there's any big deal here. We didn't really have a really major problem with the transaminases. These are relatively lower dose gene therapies. It's not like situations with the very high dose gene therapies where there's more safety risk. But I know, Eric, you want to provide any color on the steroid regimen and the burden for QTCs. Yeah, no. I think absolutely for systemically administered gene therapy, I think this really is the standard use of steroids that everyone has gotten very comfortable with. Again, important to stress this isn't a significant safety concern for patients. We just really want to use the steroids to control any type of immune response to preserve as much transgene in these hepatocytes as possible. But I think we are very comfortable that these treating physicians will be very comfortable with the use of steroids in this context. Great. Thank you. The next question comes from the line of Raghuram Selvaraju with H.C. Wainwright. Please proceed with your question. Hi, this is Raghuram. Thank you for taking our question, and congrats on the approval. I was just wondering, the label contraindicates treatment in severe hepatic fibrosis patients and advises against using it in patients with preexisting hepatic impairment. What criteria will centers be using to define hepatic impairment? And could you give some color on what proportion of otherwise eligible patients do you expect to lose because of liver findings, if any? Thank you. Well, thank you for the question. I will let Eric in a moment add a little to that. I do not think we have had that situation. I think it was something that we involved in the trials, and that is why it is being reflected there. But we did not really have anyone I am aware of being excluded. I do not think there is any specific criterion. The idea is if they have very limited liver function or they have really bad liver injury, the question is, do you want to put a gene therapy on top of that because of the transient effects of it? But we have not really seen significant issues, so I am not really concerned about this. But Eric, any thoughts on this liver fibrosis question? Yeah. Again, this is something generally listed for inclusion criteria for liver-directed gene therapy. It is exactly as Emil said. You want as healthy of a liver as possible when you are dosing with gene therapy. So we did not encounter any patients who needed to be excluded during the course of the entirety of the development program. I think there is always possible that a patient can have a concomitant disease that could lead to this type of damage, but I think if anything, it will be a very small number of patients. Thank you. The next question comes from the line of Joe Schwartz with Leerink Partners. Please proceed with your question. Hi there, it's Ashley on for Joe. Congrats on the approval today. Just one question from us. It seems like patients are mostly severe, so is there any segmentation as to who you guys would consider early adopters? Any color there would be really helpful. Thank you. Yeah, Ashley. When you look at the genotypes, something like 81% of the genotypes are severe or very low efficiency. There are some that have maybe a little bit of enzyme, but the truth is, all the patients that are getting diagnosed tend to be severe patients with real disease. It's rarely very limited, so I don't think that segmentation would matter as much. I don't know, Eric, if there's anything you'd want to add to that segmentation piece. I actually think- Yeah, no, absolutely. Quite well. Yeah, go ahead. Yeah, absolutely. I think, with a lot of these inborn errors of metabolism, we do see quite a range of severity in disease. That is not the case with GSDIa. It really is a very consistent phenotype with patients. I think when we are talking about patients needing cornstarch every two to four hours around the clock, that is consistent with what we see here. I think those are patients who are obviously ideal candidates for this. As Emil mentioned, we really do not see mild patients with GSDIa who do not have that type of cornstarch requirement. Great. Thank you. Thank you. The next question comes from the line of Ben Burnett with Wells Fargo. Please proceed with your question. Hey, thank you. I will add my congratulations to you as well. I wanted to ask, what is your expectation for the adoption curve? Just based on everything you are seeing from a patient-finding perspective, just curious if you could speak to your expectations for the rate that you may find patients and the rate that this could grow from a revenue perspective. Should we expect 2026 to be a meaningful year in terms of revenue? Yeah, very good. I will put it this way. We ran the trial. It enrolled quickly. We had demand. It enrolled quickly in different countries. There was urgent demand. The reason is that there are so many patients who are on this treadmill running and running every day doing this. They want to get off it, right? You have to think of the urgency of the disease. We found it very urgent. We think there will be significant demand. We have a number of patients found. I think there will be a reasonable degree of urgency. That is why we think it will be a successful launch. We have not put forth any numbers because, in the beginning, we have to work through reimbursement and policies and exception processes, et cetera, but we would expect some revenue in 2026. But I do not think that is going to necessarily reflect how it will do. I do think that this is a gene therapy that has no other good solution or treatment, no other specific treatment, and for which the patients are on a treadmill that is a crazy seven-day a week, 365 day a year, multiple around the clock, right? You have to imagine yourself in that setting. Would you want to get off that thing? Or yeah, you probably would. Especially when you imagine that any time at night you miss something, you could die if you mess up, right? It is a terrible way to live, and we had a sense of a lot of urgency among patients to do something better. We think it will do well. We are not yet able to project out how we think what the numbers will be, but we have a sense from the trials alone that enrollment was not an issue. In fact, when we closed enrollment, we had people upset they could not get in the trial. I can feel the demand is there, and we are excited about the potential of treating a number of patients out there as we go commercial. Great. Thank you for the color. Thank you. Our next question comes from the line of Maury Raycroft with Jefferies. Please proceed with your question. Hi, this is James on for Maury. Congrats on approval, and thanks for taking our questions. Can you confirm whether the 96-week durability data is reflected in the label. Separately, beyond the 200+ payer engagements completed as of 2Q, what's the current state of payer coverage policy. Then separately, just a third quick one. How are you thinking about gross and net expectations. Do you expect mid- 20% in line with some of the other gene therapies. Thanks. Wow, you popped them all in there, huh. I'll let Erik handle the last one on the percent, the gross to net. I think that's what that question was. We'll talk about payer. First one was I'm sorry, what was your first question. 96-week data. Oh, yeah, 96 data. There is some 96-week data in the label related to the 61% reduction in the crossover. That is in the label under the clinical trials section. So that is there. Most labels, I will point out to you, have very limited clipped bits of data, so it is pretty common, rare that you do not see all the data in the label. We do have a publication that has been accepted at group that will be coming out soon. That paper will have the more complete data and be available. With regard to the payers, we have done a lot. There are no policies yet. We have been talking, and I have personally been involved with a number of the pipe presentations. But there are some groups that will not really engage until the approval occurs, but others we have had meetings and so forth. I think they are aware of the urgency and part of my role to help to understand the urgency and severity of the disease. I think we will be operating purely under the exception mode beginning, and then we will get into getting policies. But because we have been doing this legwork all year long, I think we have prepped a lot of the biggest plans with what this is about, why it is important, and I think that has laid good pipe for having a productive discussion about ultimate policy coverage. Erik, did you want to talk about the, I think you were asking about sort of the gross to net kind of expectations and discount. Is that right? Yes. I will just make one comment and then turn it over to Howard to talk about the gross to net. Okay. As you stated in your question, we do expect it to be very similar to what you have seen with other gene therapies, where they will be on a case-by-case basis as they work toward policies. But we fully expect these patients to get access in a reasonable time as they did with other gene therapies. Howard, do you want to take this? I will build on that. Consistent with our past comments about overall average net pricing, I think we have talked in the past about a range of $1 million-$2 million. Given the lack of 2.7, I think we should anticipate it to be at the top end of that range or near the top end. Thanks for the context. Congratulations again. Yep. Thank you, and this now concludes our question and answer session. I would like to turn the floor back over to Joshua Higa for any closing comments. Great. Well, thank you all for joining us on such short notice. If you have any follow-up questions, please reach out via email at ir@ultragenyx.com. Thanks again for joining us. Thank you, ladies and gentlemen. That does conclude today's teleconference. We thank you for your participation. You may disconnect your lines at this time.