Conference Transcript - RARE 2026-08-19

Operator

Good afternoon, and welcome to the Ultragenix Pharmaceutical Conference Call to discuss the U.S. Food and Drug Administration approval of Gen Glycose, known as DTX401, for the treatment of glycogen storage disease type 1A or GSD1A. At this time, all participants are in a listen-only mode. Following the prepared remarks, there will be an opportunity to ask questions. And 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.

Joshua Higa, Head of Investor Relations

Joshua Higa 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 Ultragenix. The press release we issued announcing the approval of Gen Glycos is available on our website at ultragenix.com. Joining me on today's call are Emil Kakas, Chief Executive Officer and President, Eric Krombez, Chief Medical Officer, Eric 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. And with that, I'll turn the call over to Emil.

Emil Kakkis, CEO

Thank you, Josh, and good afternoon, everyone. Today is an important day for Ultragenix and for the GSD1A community. Earlier today, the FDA approved GenGlycose, marking Ultragenix'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-storeyase type 1A, representing exactly the type of breakthrough therapy Ultragenix was built to deliver. This is a day that thousands of people living with GST1A 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 GST1A and a life-threatening episode of hypoglycemia was cornstarch taken as a flurry 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 GST1 community who helped make this today possible, and we want to especially thank the patients and families who have participated in our clinical trials. As a reminder, our randomized placebo-controlled Phase III study met its primary endpoint with a significant reduction in daily cornstarch intake and was also positive in multiple secondary endpoints. Late during the review, the AHC decided to consider cornstarch reduction as a surrogate endpoint and so approved glenshin glycosis 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 DNP. And Eric will share some additional details of our post-marketing program when he walks through our clinical data. Now, L-Chinx 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're committed to helping patients access the treatments they need and reduce financial barriers that may stand in their way. Genglaxis extends that leadership into gene therapy, and it does so as a medicine that we manufacture end-to-end entirely in-house at our gene therapy manufacturing facility in Bedford, Massachusetts. With that, I'll turn the call over to Eric Krombes and Eric Harris to walk you through our data and launch plans, and finally, Howard Horn to comment on the PRV and our expectations for launch.

Eric Crombez, CTO

Thank you, Emil. The approval of GenGlycose fulfills our commitment to provide the first therapy that directly targets the root cause of GST1A. The reduced reliance on corn starch 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 hyperglycemia for these patients. Genglycose is indicated for the treatment of adult and pediatric patients eight years of age and older with glycogen storage disease type 1A 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 genglycose reduced daily cornstarch requirements compared to placebo while maintaining glycemic control. Genglycose 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 effects of this gene therapy. In regard to the post-marketing requirements that AMO 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 3 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 GSC1A 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 three study to long-term follow-up. With that, I'll hand it to Eric to discuss how we are bringing GenGlycos to patients.

Erik Harris

Thanks, 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 ultra care 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 ultra care 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 GSD 1a 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 in 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 a rolling basis as contracts are finalized. A list of QTCs will be available on genglycos.com once that website is live in the coming days. As we've mentioned previously, there is roughly 75% overlap in GSD 1A providers with the treating community we know well from FSEVI and DeJolvi. 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 Medicaid and large national payers. Consistently, payers appreciate the severity of GSD 1A 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 gene glycos at 2.7 million dollars reflecting its potential to reduce patients reliance on cornstarch and enable better metabolic control of glucose as they will emphasize at the outset we have a strong commitment and track record of supporting patients and families and 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 an experience in this pathway so So, we stand ready to help patients and providers in navigating the process. GenGlycose 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.

Howard Horn, CFO

Thank you, Eric. Consistent with our practice, we plan to provide revenue guidance once we have sufficient visibility in the market dynamics, which historically has been approximately six quarters after launch. In the interim, we look forward to updating your metrics, such as our TTC 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 GenGlycose will make an important contribution to our profitability. Additionally, I can confirm that Ultragenix received a priority review voucher with GenGlycose's approval. We plan to monetize the PRV to bolster our balance sheet and support our path of profitability. With that, I'll turn it back to Emil to close.

Emil Kakkis, CEO

Thank you, Howard. Developing first-ever treatment for rare and ultra-rare disease 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 GSD1A to patients that need it. The key characteristic of successful gene therapy launches is related to the urgency of the disease. And GST1A is an urgent disease with round-the-clock demands on patients every day and night without holiday or break. A gene therapy designed 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 GST1A 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 are deeply grateful to the original team from Dimension Therapeutics, who worked on the program, and to Dennis Chu, 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 ultra-ex employees throughout the company who supported this program through development and across the finish line.

Operator

Their collective commitment per series have transformed a scientific vision into a new therapy 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 1 on your telephone keypad a confirmation tone will indicate that your line is in the question queue you may press star 2 if you'd like to remove a question for participants using speaker equipment it may be necessary to pick up your hands up before pressing the star keys. One moment, please, while we poll for questions. And the first question comes from the line of Anupam Rama with J.P. Morgan. Please proceed with your question.

Speaker 15

Hey, guys. Thanks so much for taking the question, and congrats on the approval. Really cool to see.

Speaker 8

Can you walk us through the segmenting of this GSD1A 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.

Emil Kakkis, CEO

Yeah, no, it's an interesting question. The majority of the patients are treated by medical genetics patients, doctors. That's where they usually get diagnosed and usually where they're managed, although there are some endocrinologists that do manage the patients. But as I think Eric mentioned, we have about 75% overlap overlap with the doctors we're already seeing. So it's a pretty, we leverage our commercial footprint pretty well right now. 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. But I do think we can leverage our investment in the field that we've been building over the last few years.

Operator

Thanks so much for taking the question. Congrats again.

Operator

And the next question comes from the line of Yaron Werber with TD Cowan. Please proceed with your question.

Yaron Werber, Analyst — TD Cowan

Yeah, terrific. Let me add that this is really a terrific milestone, and congrats. I know this is years and years of work. I have maybe just a couple of questions. The first one, Emil, you mentioned you inventory, and the confirmatory work as part of the DMP, is that going to be under commercial settings, or is that going to be under a clinical study protocol? And then finally, just remind us, because this is a big milestone, or a de-risker for the MPS3A program, and what's the overlap?

Emil Kakkis, CEO

Yeah. So, with regard to the inventory, we asked Intra to cover the launch. and what the confirmatory 50 patients are commercial patients treated commercially. And as we're treating commercial patients, if we find some patients who have antibodies to AB8 or can't qualify for treatment, we'll include some of those, 20 of those, to be in the control group. So we'll 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's a high-quality, fully-sponsored trial, though. All of our DNP programs, we don't do registries. We all do DNP for all post-marketing, and it's 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 to what it means for MPS3A, the GSD 1A is fill-finished and produced at the same Bedford plant where 111 is also fill-finished. So, there's overlap in the facilities. We can't speak yet to comments about the 111 review, which is ongoing, but the plant is approved now to do fill finish for GST 1A, so obviously that has some read-through on the processes and conditions they're required to get approval for fill finish that overlap with the 111 program.

Operator

Thank you, Ron.

Operator

Thank you. And the next question comes from the line of Kristen Kluska with Cantor Fitzgerald. Please proceed with your question.

Speaker 12

Hi, guys. It's Ross on for Kristen, and 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 kind of the treatment center capacity, and also how many treatment centers you expect to have online by the year end and in 2027.

Emil Kakkis, CEO

I'll put a few high-level comments if Eric wants to give a little bit more color on the number. Our goal with the treatment center is to set up enough centers to be able to accommodate all the patients we need, but we also kind of 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 number. But, Eric, you can provide maybe a little more of where we are in building QTCs.

Erik Harris

Yeah, well, 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. We'll be able to meet the initial demand. And as I also stated, we'll continue to add treatment centers as demand grows.

Operator

Thank you.

Operator

Thank you. And the next question comes from the line of Yagal Nushomovits with Citigroup. Please proceed with your question.

Speaker 15

Hi, I'm Bill and team. 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? Because you mentioned that you're going to be following these patients for a total of 10 years. And then also, if you could please comment, just what percent of the population do you expect would be positive for AV8 antibodies. Thanks.

Emil Kakkis, CEO

So 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 hike up hypoglyceme between 70 and 54. So those are endpoints we've evaluated, so we're comfortable with that evaluation. But we're going to do it now in open label treatment format, which will allow the treatment to be implemented, let's say, in a more real-world setting. So it's two years of that. We will follow patients for 10 years as part of our commitment we do in all of our programs. It's not part of the confirmatory program, all right? It's part of our general commitment. And gene therapies in general have a longer lead time in monitoring for safety in any case. But 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's varied. It's maybe a fourth of the patients, but it's around that range. It varies in different areas and regions, so I don't want to give you a precise number, but it's around that range, and we expect it to be similar now. So that's where we are at the current moment.

Operator

Thank you very much.

Operator

Thank you. And the next question comes to the line of Ellie Merle with Barclays. please proceed with your question.

Speaker 2

Hi, this is for Ellie. Thanks for taking your question. Could you just remind us about the plans and timelines for ex-US and then with, you know, about three quarters of that population outside the US, how do you see that dollar opportunity shaping up relative to the US given all the different pricing and reimbursement dynamics there thanks uh david 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 um the latest timelines and expectations for x us approval and then the relative size of the opportunity okay yeah so we do expect to commercialize elsewhere and and we think that we're within the worldwide price

Emil Kakkis, CEO

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, you know, 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. it, that pricing maybe have more pressures, and we're going to have to work through that. But we actually think there's already been some price standards set there above even where we are with this particular ulcer rare disease. And we think we have excellent randomized control data to support it. And so we believe we're going to find a place to be able to commercialize and make substantial revenue ex-US as well as US. So we're not looking at this as a pure US play. We think we're going to look at it globally. And we certainly have operations, you know, in Europe, Latin America, Japan, as well as supportive distributorships 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.

Operator

Thank you.

Operator

The next question comes from the line of Salvine Richter with Goldman Sachs. Please proceed with your question.

Speaker 11

Hi, this is Lydia on for Selvin. Congrats on the approval, and thanks so much for taking our questions. Could you just speak to when you expect genglycose to be commercially available and your expectation for the shape of the early launch curve here, and just the timeline for when patients initially engage with their healthcare provider to actually getting the treatment? Thanks so much.

Emil Kakkis, CEO

Yeah, so we would expect it to be available within 30 to 60 days from that timeframe. There is a process where FDA also releases, and that process is ongoing, but 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. There are a number of patients that want to get treated, but rather than project out, we're going to let the market work and then provide some updates on that. And the last item was about the doctors. Is that right?

Speaker 11

Just a timeline from the initial interaction to actually when the treatment would occur.

Emil Kakkis, CEO

Oh, I see. Well, we don't know how long it takes to have a start form and turn that into a treatment event. You know, I'd expect, you know, in the beginning, it's all going to be operating off of exceptions, right, of case-by-case exceptions. I don't know, Eric, if that's something you'd want to touch on a little bit here about that process between start form to getting treatment implemented.

Erik Harris

Yeah, and Emil, I think they also want to know how quick how quickly can you work up a patient to get treated but let me let me take the uh the enrollment once we receive the enrollment form um we will start working with the patient uh to navigate the reimbursement process and as i've stated in the opening remarks um they'll they'll take some time to to work through uh 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.

Emil Kakkis, CEO

Yeah, so the workup part is the main thing they have to get is an anti-AV8 antibody They have to have the direct diagnosis, but our patients generally have had it already. AV8 antibodies should not be a particular issue. And then when they get the gene therapy, they don't start any immune modulation or steroids till afterwards. So there's no other preparative effect going on. It's really, I guess, anti-AV antibody and start form and reimbursement process. Thank you for the question.

Operator

Thank you. And the next question comes from the line of Maxwell Score with Morgan Stanley. Please proceed with your question.

Maxwell Score, Analyst — Morgan Stanley

Great. Thank you very much and congratulations on the approval. So 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, yeah, any color around that, the monitoring process would be very helpful.

Emil Kakkis, CEO

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 if 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. And 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.

Eric Crombez, CTO

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, you know, important to stress this, you know, 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, you know, I think we are very comfortable that these treating physicians will be very comfortable with the use of steroids in this context.

Erik Harris

Great. Thank you.

Operator

And the next question comes from the line of Raghuram Silvaraju with H.C. Wainwright. Please proceed with your question.

Speaker 14

Hi, this is Amit Anwaram. Thank you for taking our question and congrats on the approval. I was just wondering on the label contraindicates treatment in severe hepatic fibrosis patients and advises against using it in patients with pre-existing 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.

Emil Kakkis, CEO

Well, thank you for the question. I'll let Eric in a moment add a little to that. I don't think we've had that situation. I think it was something that we involved in the trials, and that's why it's being reflected there. But we didn't really have anyone I'm aware of being excluded. I don't think there's any specific criteria. And the idea is that 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'm not really concerned about this. But Eric, any thoughts on this liver fibrosis question?

Eric Crombez, CTO

Yeah, and again, you know, this is something, you know, generally listed for inclusion criteria for liver-directed gene therapy, and that's exactly as Amal said. You want, you know, as healthy of a liver as possible when you're dosing with gene therapy. So, we didn't encounter any patients who needed to be excluded during the course of the entirety of the development program. I think, you know, there's always possible that, you know, 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.

Operator

Thank you.

Operator

And the next question comes from the line of Joe Schwartz with Leering Partners. Please proceed with your question.

Speaker 10

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.

Emil Kakkis, CEO

Yeah, Ashley. So when you look at the genotypes, something like 81% of the genotypes are severe or very low efficiency, and there are some that are 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 really very limited. So I don't think that segmentation matters much. I don't know, Eric, if there's anything you'd want to add to that segmentation piece. I actually think the mildness is quite well. Yeah, go ahead.

Eric Crombez, CTO

Yeah, absolutely. I think, you know, 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 GSD1A. It really is a very consistent phenotype with patients. And, you know, I think when we're talking about patients needing cornstarch every two to four hours around the clock, you know, that is consistent in what we see here. And I think, you know, those are patients who are obviously ideal candidates for this. But as Emil mentioned, we really don't see mild patients with GSD-1A who don't have that type of cornstarch requirement.

Speaker 10

Great. Thank you.

Operator

Thank you. And the next question comes from the line of Ben Burnett with Wells Fargo. Please proceed with your question.

Yaron Werber, Analyst — TD Cowan

Hey, thank you.

Operator

And I'll add my congratulations here as well.

Eric Crombez, CTO

I wanted to ask, what is your expectation for the adoption curve? Just based on everything you're seeing from a sort of patient finding perspective, Just curious if you could speak to your expectations for sort of the rate that you may find patients and sort of the rate that this could grow from a revenue perspective. And should we expect 2026 to be a meaningful year in terms of revenue?

Emil Kakkis, CEO

Yeah, very good. Well, look, we – I'll put it this way. When we ran the trial, it enrolled quickly. Like, we had demand. It enrolled quickly in different countries. There was urgent demand. And the reason is that there's so many patients who are on this treadmill running and running every day doing this. They want to get off it, right? So 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. We think it will be a successful launch. We haven't put forth any numbers because in the beginning, you know, we have to work through reimbursement and policies and exception processes, We would expect some revenue 26, but I don't want to – I don't think that's 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, you know, 365-day-a-year, multiple round-the-clock, right? You have to imagine yourself in that setting. would you want to get off that thing? Or yeah, you probably would. And especially when you imagine that at any time at night you miss something, you could die if you mess up, right? It's a terrible way to live. And we have a sense of a lot of urgency among patients to do something better. So we think it will do well. We're not yet able to project out how we think that 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 couldn't get in the trial. So I feel that the – I can feel the demand there. We're excited about the potential of treating a number of patients out there as we go commercial.

Operator

Great.

Joshua Higa, Head of Investor Relations

Thank you for the color.

Operator

Thank you. And our next question comes from the line of Maury Raycroft with Jeffries. Please proceed with your question.

Speaker 8

Hi, this is James on for Maury. Congrats on the approval and thanks for taking our questions. Can you confirm whether the 96 Weak durability data is reflected in the label, and separately beyond the 200-plus payer engagements completed as a 2Q, what's the current state of payer coverage policies? And then separately, just a third quick one, how are you thinking about growth to net expectations? Do you expect mid-20% in line with some of the other gene therapies?

Emil Kakkis, CEO

Wow, you popped them all in there, huh? I'll let Eric handle the last one on the percent, the growth to net. I think that's what that question was. and we'll talk about pairs. First one was, I'm sorry, what was your first question? I missed it.

Eric Crombez, CTO

96-week data.

Emil Kakkis, CEO

Oh, yeah, 96 data. Yeah, there is some 96-week data in the label related to the 61% reduction in the crossover. That's in the label under the clinical trial section. And so that's there. Most labels I will point out to you have very limited clipped bits of data. So it's pretty common and rare that you don't see all the data in the label. We do have a publication that's been accepted at Proof that will be coming out soon. And that paper will have the more complete data and be available. With regard to the payers, we've done a lot. There are no policies yet. We've been talking, and I have personally been involved in a number of the PI presentations. But there's some groups that won't really engage until the approval occurs, but others we haven't had meetings and so forth. So I think they are aware of, I think they're 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'll get into getting policies. But because we've been doing this legwork all year long, I think we've prepped a lot of the biggest plans with what this is about, why it's important. And I think that's laid good pipe for having a productive discussion about ultimate policy coverage. Eric, did you want to talk about the, I think you're asking about sort of the gross to net kind of expectations and discounts. Is that right?

Erik Harris

I'll just make, yes, I'll just make one comment and then turn it over to Howard to talk about the gross to net. And 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'll 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 Horn, CFO

And I'll build on that, you know, consistent with our past comments about overall average net pricing. I think we've talked in the past about a range of 1 to 2 million. Given the WAC of 2.7, I think we should anticipate it to be at the top end of that range or near the top end.

Operator

Thanks for the context. Congratulations again.

Operator

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.

Joshua Higa, Head of Investor Relations

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.ultragenics.com. Thanks again for joining us.

Operator

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.