Zevra Therapeutics, Inc. (ZVRA) Earnings Call Transcript & Summary

Good morning, everyone. My name is Susmita Roy. I'm an associate on the JPMorgan Healthcare Investment Banking team. On behalf of JPMorgan, thank you all for attending the Healthcare Conference. And I'm thrilled and honored to introduce Zevra Therapeutics for their company presentation today. A little bit about Zevra before we get started. Zevra Therapeutics is a commercial-stage company with a unique opportunity to impact people living with rare diseases. The company is in growth mode, with operating runway to execute on its commercial and development priorities. Zevra is commercializing its lead product, MIPLYFFA, in the U.S. for Niemann-Pick disease type C. In addition, the company has an MAA under review by the EMA, is broadening access through geographic expansion opportunities and has a pipeline of rare disease programs. Zevra has multiple revenue sources with net revenue generation of $72.3 million in the first 9 months of last year and exited Q3 with a strong cash position of $230.4 million. We are delighted to welcome their President and CEO, Neil McFarlane, to the podium to tell us more. Thank you, Neil. And I'll turn it over to him. Please note that Q&A will be afterwards. And if you have a question in the audience, please wait for the mic. Thank you so much.

Thank you, Susmita, and thank you, JPMorgan, for allowing us the opportunity to present this week. I realize it's been a long week. And for all of you in the room, thank you for coming here on the last day of the conference. At Zevra, we're taking a rare approach to taking care of patients. As you see on the screen here today, this is an adult patient with Niemann-Pick disease type C. And I'm looking forward to spending some time with you today talking to you about Niemann-Pick and what we're doing here at Zevra to help patients. I'm going to be making some forward-looking statements today. So please take a look at our most recent SEC filings to get the most up-to-date information. Moving forward, we're a purpose-driven company. I think it's important that I spend a little time on this slide. We have a mission to redefine what is possible in bringing life-changing therapies to people living with rare diseases. And we've got a vision to become a leading rare disease therapeutics company. Our values, as you see on this slide, start with patient centricity. It's what we do when we make decisions. It's what we do when think about how to service the rare disease community. We're accountable to each other, we're accountable to the community and we're accountable to our shareholders as we move forward. Integrity. Innovation. Courage. All of these values drive how we operate as an organization. We also have a very unique opportunity to impact people living with rare diseases. We're a commercial-stage company with a late-stage pipeline. We've got geographic expansion underway with an MAA, as well as an existing infrastructure that we can leverage to actually launch the products that we have and earn the right to go and bring new products in and be a partner of choice. As Susmita mentioned, we have multiple revenue sources. And through the first 9 months of 2025, $72.3 million. We exited Q3 2025 with a strong cash position of $230.4 million. And we are in growth mode. Spending a little bit of time on the portfolio, we have a diversified portfolio. As I mentioned previously, MIPLYFFA with Niemann-Pick disease type C. Importantly, we have that product that was FDA-approved in September of 2024. At that time, and today, we actually guide that we have orphan drug exclusivity, the regulatory pathway to keep exclusivity through 2031. I'll talk a little bit more about some recent evolution of our patent term extension application and what we are hopeful there's the potential to extend to 2031 and beyond. The other product we have is OLPRUVA, and it's for urea cycle disorders, approved in 2022 with intellectual property through 2036. AZSTARYS is a product that we developed internally and licensed and have a partnership where we receive royalties and milestones through 2037. And as I mentioned, arimoclomol, which is the name of MIPLYFFA, for Niemann-Pick C is under review by the MAA. Celiprolol is our Phase III program that is ongoing right now, with IP through 2038. And we've done some work in Phase II for sleep disorders with KP1077 in idiopathic hypersomnia and narcolepsy that allows for a Phase III-ready trial with very durable IP. So this diversified portfolio is fulfilling our mission, and it's just getting started to bring life-changing therapies to people with rare diseases. We're doing this through intense focus on the things that we can drive and on the impact that we can make as an organization. The team has been executing very well. And we're innovating not only in bringing new therapeutics to market, but we're actually also innovating in how we bring therapeutics to patients ultimately. We're prioritizing our resources and been incredibly disciplined with how we've moved forward through 4 pillars. Our marketed therapies that are in growth mode for MIPLYFFA, as I mentioned, as our lead product. A pipeline that's got synergistic growth opportunities with us to be able to leverage our infrastructure, but also develop and get approved products through the regulatory and clinical development periods. And then we've got an experienced team. We call it talent and culture, and I think this is one of the most important parts of any company. And our experienced team that's continuing to grow and lean in to deliver for patients, I'm very proud of. And we're doing all that with a corporate foundation that's got a very strong balance sheet and the fiscal discipline to make investments that are paying off for us through focus, and not trying to, I like to say, we're not trying to boil the ocean, we're not trying to invest in everything. We're investing in things that can drive real value and sustainable growth for the company. Let me talk a little bit about 2025, because it's really paved the way. It's a transformational year, no doubt, for us in 2025. But it's paved the way for some multiple growth drivers as we look into 2026. Our team has really established MIPLYFFA as a foundational therapy in NPC. In the first 12 months post launch, we've achieved approximately 40% of the diagnosed patient population. I've been in the rare disease space for a long time, and I've been incredibly impressed with the launch here. I've never seen 40% of the diagnosed patient population in 12 months. So we're really making an impact, and I think it's a testament to the team. In our pipeline, we've got some significant growth opportunities. As I mentioned, we have our MAA that is under review by the European authorities. We announced that 120-day response would be coming at the end of 2025. We received that response. While I can't talk about all of what's in the response, what I can say is that we've seen no new questions or concerns that have come up from the response that we've got. And our team is incredibly well prepared for all of what we received. Very similar to the pathway that we've taken with the FDA to get the product approved. And I'll talk a little bit more about what we've done and the data and the sustainability of the data that we've put forth for the application. Importantly, we actually have been able to have 92 expanded access patients in Europe and the U.K. in a very small number of markets. And that number is growing on a quarter-over-quarter basis. Very proud of the opportunity to be able to serve patients that expand access to MIPLYFFA in Europe. But this is also a testament to our ability to be able to expand the markets and get the product to patients. The other perspective here is that we have our pivotal Phase III trial undergoing for celiprolol under the growth opportunities. I'll talk a little bit more about that when I get into the pipeline and innovation section. And we're demonstrating leadership through advocacy, through community engagement, through patient and caregiver support services as we develop ourselves as a partner of choice in the rare disease products. And recently, over the last 6 to 9 months, we've actually established a presence in Boston, one of the leading biotech rare disease hubs, and it's allowing us to be able to tap into that ecosystem and then drive additional value for the company. And as I mentioned, a very strong balance sheet. In 2025, we were able to monetize our pediatric Priority Review Voucher for $150 million that added nondilutive capital to our balance sheet. And as I mentioned, $230 million at the end of Q3, where we have not burned capital for a few quarters. So we're very proud of what we're doing in terms of setting the foundation for the future. Let me spend a minute on our marketed therapies here, and I'm going to primarily focus on Niemann-Pick disease type C, which is an ultra-rare genetic disorder that leads to premature mortality. NPC, as I mentioned, devastating, fatal lysosomal storage disease. And what really transpires here is you get cholesterol buildup in the lysosome in cells that lead to cell death. NPC is a gene that the mutations produce abnormal protein and it doesn't allow for the cholesterol to be -- go in and out of the lysosome. And this progressive buildup then leads to cell death. That cell death is in organs. It causes organ dysfunctions. It causes spleen, liver and brain buildup and then cell death. The signs and symptoms of NPC are very heterogeneous. The age of onset is -- well, you have it forever. It's genetic. However, the symptoms can show up either as a child or as an adult. What we've seen so far in our experience, both in our expanded access program and in the 137 patient enrollments that we've had, is about a 50-50 split between children and adults, which a lot of times people see this as just a childhood disease. And as we are moving forward in our launch and engaging with patients with NPC, we're realizing that we're diagnosing patients who are adults for the first time or adults who have been misdiagnosed as well. So the rate of progression is obviously different if you have these symptoms of cognition, speech, ambulation, swallowing, fine motor skills and the visceral, primarily hepatosplenomegaly. But these presentation of symptoms are challenging, like in every rare disease. It presents a diagnostic odyssey for a lot of physicians to be able to try and find out what is transpiring. And for us, we've demonstrated through multiple trials the impact that MIPLYFFA has on NPC. And it's proven effectiveness now both as a short term in terms of improvement -- and what you see in this slide here is MIPLYFFA that's utilized in combination with miglustat. On the bottom, you see the Niemann-Pick C clinical severity scale of MIPLYFFA and miglustat, where you see no progression through 12 months and improvement as early as 12 weeks. I think that's an important perspective. You get it early and then you have the durability. On the right-hand side, you see the same revised 4-domain Niemann-Pick C clinical severity scale, over a period of 48 months through our open-label extension study. And you see that limited progression and the halting of this devastating disease, durable through 5 years. This is, for us, a real game changer, when you can bring the -- with, by the way, I should say, a very acceptable safety profile, importantly. This allows us, though, to be able to establish MIPLYFFA as foundational therapy for NPC. The mechanism of action and up-regulation of the CLEAR gene network allows for the impact that we see on the halting of the progression of the disease, the strength of our clinical data, the magnitude of our safety database. And we're continuing to expand the body of evidence as we're moving forward now through both our expanded access program that we had in the United States, our pediatric investigational study and substudy, which is complete, as well as our open-label extension going through 5 years. The magnitude of this data, it's -- and what we've submitted actually to the MAA is really the largest collection of data for NPC patients ever. And we're proud of the ability to be able to get that into the committee. We're also driving patient access. Very proud of the organization right now. In payer coverage, about 66% of covered lives as of the end of Q3, which is very on target for us in terms of the first 12 months of launch. Patients are gaining coverage even when not on formulary through the medical exception pathway. And through the end of Q3, we're still having a very high adherence through our enrollments, and very proud of that. We'll get our first glimpse at the end of Q1, into Q2 on persistency, patients that have been on product for 12 months. And I'm looking forward to being able to continue to drive that. This next section is an important section for us. We have been increasing the identification of patients. As I mentioned, approximately 40% of the diagnosed patient population, which equals 137 patient enrollments. We had 8 patient enrollments in Q3. Importantly though, some of the work that we've been doing around the identification of disease state awareness, offering genetic testing and some of the collaborations have started to unlock newly-diagnosed patients. We saw some of this in Q2, very early in our launch. In Q3, we talked about it also on our earnings call that the identification of newly-diagnosed patients is really key for us to be able to establish ourselves, not with the diagnosed patients we have today, but the TAM being between that 300 to 350 patients that we know are diagnosed and being treated today and the 900 prevalent patient population. In Q3, we saw more. And in Q4, we've seen even more newly-diagnosed patients, which is giving me a lot of confidence that the TAM is somewhere between this 300 to 350 and the 900, which the team has been working through bringing these patients on to product. The other part is we have a bespoke AI predictive modeling that our commercial team has been working through to find potentially new patients that have either been misdiagnosed or a suspicion index that allows for patients to walk through the process to get potentially diagnosed with NPC earlier. These tactics are starting to pay off and I think is giving me a lot of confidence that this market and the TAM is somewhere between that 300 to 900 patients. We're early, but I'm confident that we're making headway there. The team [ fielded some ] market research at the end of the year that suggested that MIPLYFFA is a preferred NPC therapy trusted by most clinicians and showed improvement in balance, swallowing, cognition, speech and reducing falling. I think these are some of the feedback that we're getting. In the Q&A., maybe we can spend a little bit more time. We'll get Josh up, our Chief Commercial Officer, to work through that. What's next for us is capturing this next wave of growth for MIPLYFFA. As I mentioned, the U.S. prevalence of approximately 900 patients in the U.S. living with NPC, and this 300 to 350 that are diagnosed and treated. This ability for us to continue to drive penetration of the undiagnosed patient population is what I think has given us a lot of confidence that the market is larger than what we originally see, the patients that are diagnosed. In addition to that, the European prevalence is about 1,100 individuals living with NPC, but the diagnosis rates are much higher than that of the U.S. because they've had miglustat approved for well over a decade and have been able to educate and drive patients to therapy. We see both the European prevalence and the high number of patients who have been treated and the last 3 quarters of continued growth of newly-diagnosed patients putting us in a place where we feel like we can unlock the opportunity in the United States. And then our expanded access programs with the geographic expansion that we've most recently discussed with -- if we look at the umbrella of the geographic expansion, it's both through our marketing authorization application in Europe and potentially the U.K. It's through the fact that we've got our EAP, Expanded Access Program, that is driving patients today and physicians are asking for the product through name patient basis. And in addition to that, outside of Europe and the U.S., we just recently signed a distribution agreement with a partner in Europe that -- with a partner in Europe to support markets outside of Europe, outside of the U.K. And we're actually now shipping product to those patients under named patient basis. So broadening access to MIPLYFFA is the next wave of growth, the EU prevalence and getting into EU with an MAA, and continued driving of our U.S. launch is capturing that wave of growth for us. I'm going to quickly run through some of our pipeline. I'm going to focus here just on vascular Ehlers-Danlos Syndrome, which as I mentioned is a severe autosomal dominant genetic connective tissue disorder. And it's the most severe form of Ehlers-Danlos Syndrome as a subtype. As I mentioned, it's a connective tissue disorder caused by COL3A1 gene mutations. It leads to a defect in type 3 collagen in both the vessel walls as well as hollow organs. And it's usually characterized by arterial aneurysms or hollow organ ruptures. In the United States, about 7,500 individuals diagnosed with VEDS and that are genetic -- about 95% of the diagnoses are usually confirmed by the COL3A1 genetic testing. As I mentioned, devastating disease. Most -- or 25% of patients will experience an event, either the aneurysms or hollow organ ruptures by the age of 20. And about 90% of patients will experience an event by the age of 40. Unfortunately, arterial rupture is the most common cause of sudden death in these patients, with a median survival age of 51 years. Celiprolol is a product that we're developing as a potential treatment for COL3A1 VEDS. And it's addressing VEDS by reducing the mechanical stress on collagen fibers as a selective adrenergic modulator. We actually have quite a bit of data that has been developed with celiprolol's use outside of the U.S. through a randomized and real-world data across the BBEST study in a few European cohorts. And what we saw is that the event rates, an arterial and/or hollow organ event, is decreased versus the untreated patients, and improved survival. Now we're running a DiSCOVER trial, which is a Phase III trial. It's a decentralized pivotal trial being run under a special protocol assessment with the FDA, of which, as of the end of Q3, we've enrolled 44 of the 150 patients. As I mentioned before, it is an event-driven trial. And the interim analysis is that 28 events, of which if we hit the positive marker there, we will then move towards a -- sorry, then we don't have to move towards the final event rates, which is 46 events. The one important part here around celiprolol is it is a -- although it has never been approved for VEDS anywhere in the world, it is the primary treatment for VEDS in Europe where the product was approved many decades ago. Let me -- I know I'm running a little short on time here, let me quickly talk about the fact and how we're demonstrating financial discipline. As I mentioned, through the end of Q3, we have had net revenues of $26.1 million in Q3. And the large chunk of this is through our efforts in the U.S. with MIPLYFFA. We do get pre-commercial revenues through our EAP program in France right now. That will be expanded with the execution of our distribution agreement that we announced over the holidays, that will bring us new revenue that are pre-commercial revenues in select territories. We get the license royalties from the AZSTARYS agreement and OLPRUVA. Importantly though, our balance sheet is incredibly strong with $230.4 million, with just $61 million of debt. In closing here, our growth drivers in 2026 and beyond are meaningful. The additional -- the continued execution of our U.S. launch unlocks the opportunity for us. As I mentioned, our conviction internally around the opportunity size for MIPLYFFA through our genetic testing collaborations and through the early undiagnosed patients that are being identified and put on therapy today has given us a lot of confidence in that. We're driving the additional clinical and scientific evidence that will allow for us to be able to further develop the foundation of therapy for us. We have this possible patent term extension, which we believe is an unlocking event and a re-rating event for the stock. Every additional potential year that the IP would be extended beyond our current LOE with the orphan drug exclusivity is meaningful. And we believe that that should come in 2026, the final determination from the U.S. Patent Office. And then we submitted our MAA and as part of our geographic expansion and continue to drive EAP patients in Europe and physicians with the experience in taking care of patients through named patient basis, but also broadening that outside of Europe and the U.S. I've mentioned this a few times on our earnings calls, our go-to-market strategy in Europe is still under evaluation. And we believe that as we continue to drive expanded access patients and utilization by key opinion leaders in Europe, we'll be primed to be able to make a decision whether we go it alone or we partner it. But today, we'll continue to drive the expansion through EAP where we can service patients. And with that, I'm going to stop and see if we can open it up for some Q&A. Thank you for your time. I'm going to also ask Josh Schafer to join us on stage, who's our Chief Commercial Officer.

Amazing. Thank you so much for the presentation. And first of all, congratulations on the success of MIPLYFFA. To kind of kick things off for the Q&A, and if there's any questions, obviously, we can continue to take that from the audience, but what does the team attribute the success for MIPLYFFA today? And what do you all think will be the specific drivers for that continued success moving into 2026 and beyond?

What I'll do is I'll maybe start with the broad company, and I attribute the success that we've had so far to the focus that we put in to driving the things that we can control and execution that we've had and the way that we're innovating to be able to get product to patients. But maybe I'll ask Josh to talk a little bit about all the wonderful things we're doing on the commercial side.

Yes. Neil presented a slide which I think really speaks to what's going on with MIPLYFFA and how we've been able to drive that performance. Most notably is the clinical differentiation that we've been able to establish through publications, through building the awareness of that data. The commercial and medical teams have done a great job around building awareness of the disease and the need to treat. And our collaboration with genetic testing organizations has really driven the identification of formerly undiagnosed patients, which all led to really outstanding performance in '25.

Awesome. Now just to kind of continue on that and double-click on it, throughout the presentation, you mentioned that the awareness of the disease, diagnosis, the under and misdiagnosis of the disease that we've seen, outside of -- maybe through genetic testing, just double-clicking on it, could you just speak a bit more about how improvements could be made in diagnosis and how MIPLYFFA can get to those who obviously need it? And your thoughts on how -- and who you're working with. That would be great to hear a bit more detail about.

Yes. Well, first of all, this is a really small patient population, which prior to our approval in 2024, there were no approved treatments for Niemann-Pick. So many clinicians outside of those experts are just really unaware of it. Many of them read -- there was one line in med school that they read about it. So we have really taken on the charge of helping to build the awareness, helping to identify these patients through a symptomatic presentation. And then if a patient presents with a certain array of symptoms, which were listed in terms of swallowing, speech, ambulation issues, these patients might likely benefit from a confirmatory genetic test. And we've partnered with a couple of companies who provide that test, and that's really helping to drive the awareness and identification of those patients.

Josh, maybe you can spend a little bit of time educating me and everybody else on the bespoke kind of AI work that we're doing with EMRs and claims data to be able to try and help people who otherwise may have not been diagnosed or been misdiagnosed previously, that now have the opportunity to get diagnosed.

Yes. This is a really, really cool work that I take no credit for, the team is doing its way above my complete understanding. But what we've done is we've gone through the claims data and we've identified patients who have been confirmed or who have been diagnosed with NPC. And we've looked at the array of symptoms that they have. We've looked at their treatment odyssey and misdiagnoses and clinicians that they've seen and a number of other parameters. And we've then looked at the larger claims database of patients who have those same symptoms, same travel through the health care system that were never diagnosed with NPC. And there is a surprisingly large number of patients who look like an NPC patient. And that allows us as a sales team, as a commercial team to interact with those clinicians to say, "Hey, if you have a patient who looks like this, you might think about NPC as a disease that your patient might have. And we have a treatment option and we offer a way for you to confirm that diagnosis." So it's really -- and it's an evolving, of course, with all AI things, it's an evolving model. But as we get new information, it helps to inform how we go forward.

That's incredible. Kind of along those lines, as you've been talking to physicians and trying to educate them and also interacting with patients and payers, et cetera, what's been the feedback on MIPLYFFA? And how has that been? How -- what have you guys kind of been doing with that feedback? And would love to just hear a bit more details about that.

Yes. The research that was fielded at the end of last year is providing us with a lot of very actionable, timely feedback that allows us to be able to pivot. But Josh, maybe you can talk a little bit of some of the specifics because it's pretty good data. And it's important, it is anonymized. The work that we have done is not Zevra going out doing research. This is work that's been done in the community without us knowing that it's Zevra.

Yes. We went out to payers, patients and clinicians and did some independent market research and went through a list of questions around their view of the disease state and MIPLYFFA specifically. And the feedback was overwhelmingly positive, in which clinicians stated that they really view MIPLYFFA as the foundational treatment and the best option for patients in terms of halting the progression of the disease. And caregivers went so far as to say, when their children or loved ones were on MIPLYFFA, it gave them confidence and they saw improvements in balance and ambulation, and really resounding feedback. But it also gave us the opportunity to understand our messages, what's working, what's not. And we're refining that according to that feedback.

No, that's amazing. And of course, always helpful to get that feedback. I think one of the things that stood out to me in the presentation was with -- given the success of MIPLYFFA, there's also so much more that the company is looking ahead to this year and beyond. Could you just maybe double-click on the MAA and EMA processes that are going on? And would love to hear a bit more about, I know you mentioned some time line, but any further details that could be helpful for everyone to know would be awesome.

Yes. So I'll take that one. So as I mentioned previously, we submitted our MAA at the end of July and through a standard MAA approach. But as a new application, I think that's an important perspective. We didn't actually refer back to our previously submitted application in our conversations with the regulators. It was really important that because of the magnitude of new data that we were going to be providing in the application around our pediatric substudy -- and for those of you that are listening, usually, you have a pediatric investigational plan that you do after your filing. We fortunately have the ability to have finalized that pediatric investigational plan as well as the study. So that data has been submitted. Our longer-term OLE, the open-label extension data going out 5 years, we were able to submit as well. Along with we collect the data for a number of years in the United States through our expanded access program, about -- and then as we got approval, we were able to shut that program down and then convert those patients to commercial product. But we shut the database down at the end of March 2025. And as we locked that database, we're able to bring all that data into the filing as well. So the substance behind our application is much more robust than even what we had for the FDA. Now moving forward, the European approaches is that you file and then they come to you with a 120-day list of questions, and they're clarifying questions. I can't talk about the questions or the process, but we're in the clock stop period today. And what I can say is that our team was prepared for every question that's come back, and there have been no surprises, which gives me a lot of confidence that now that we can actually provide the responses and further inform the dossier for our next step, which will be at the 150-day mark after this clock stop is over and we respond. So I can't tell you what a specific timing is at this point. What I can tell you though is that we are in a position today of providing the most robust set of data ever collected at NPC to help drive and get expanded access to patients in Europe.

Awesome. It's very exciting to hear. And I mean, I think given that you talked a lot about how this will help you all expand into the EU a bit more. But beyond the U.S. and the EU, are there any thoughts that you might have on like overall global strategy and getting access to NPC patients worldwide?

Yes, our goal -- thank you for that question. Our goal is to maximize the impact we make for patients with NPC everywhere in the world. We are in a very interesting time when I think about the geopolitical space, the opportunities that we have to be able to expand access across the world. We are taking a very disciplined approach where we will provide compassionate use where we can, we will provide named patient basis access that may or may not have a pre-commercial revenue with it. But we're also being very disciplined to protect our optionality to expand into Europe and not impact our pricing and not impact Most Favored Nation discussions that are ongoing, which I'm not sure anybody knows what it is today. But for us, we're taking a disciplined approach. How do we expand the product to as many patients as we can and provide access? But also being very disciplined not to shoot ourselves in the foot. So today, through this distribution agreement that we signed over the holidays, it allows us to be able to service select territories. And for the first territory that we're going into under this agreement, we're actually able to have name patient basis reimbursement at U.S. WAC pricing. And this territory is outside of Europe, outside of the U.K., outside of the U.S. So that discipline of the identified patients that we've been working on with these distributors -- or this distributor for a number of months allows us now to service those patients but not impact our opportunity in Europe. Or the rest of the world for that matter.

Yes. No. And given the circumstances, it's always -- this is a tough path for companies to navigate, and the distribution agreement seems like it's a great step in the direction of figuring it out. I think for the next part of kind of talking more about the vision and next steps in the future, could you just, kind of going back a bit, could you just double-click a bit more on MIPLYFFA? You mentioned the potential patent protection, extending that. Are there any key updates that we should know about or the team is looking forward to present soon? And what are your thoughts about that?

This is a very big opportunity for us, and I mentioned it just in passing that it's a re-rating opportunity for this company. MIPLYFFA is clearly the key asset that we have in our organization today, with the largest amount of growth that we see, not just today in 2026, but in the next, today, 7 years that we have the orphan drug exclusivity. As I mentioned, orphan drug exclusivity is through the regulatory pathways of the FDA. They provide you this through the orphan drug legislation. On top of that though, there's patent term extension. And today, we have an Orange Book listed patent that expires in 2029. So our orphan drug exclusivity is actually longer than that of our patent. As part of our approval process, we can go back to the Patent Office and actually ask for an extension of our patent based on the amount of time it took for the product to get approved and through that process. We've worked with the FDA to identify the number of days that we're eligible for. We've now -- the FDA has now gone back to the Patent Office and said, "This we agree with the company," the sponsor in this case of those days. And the Patent Office opened up an open public commentary for about 60 days. That 60 days expired at the end of November, and without any comments. So we expect the Patent Office to now rule as to how long we will get from our 2029 patent. The maximum is 5 years. We don't know what that will look like in terms of what the ultimate outcome is. But every month, every quarter, every year on top of what we have today of 2031, could go out to 2034, is a very meaningful impact to the company. So that's why we think, in 2026, this is a re-rating event for the company, and not yet being recognized in our stock.

No, that's definitely, yes, the impact of that will definitely be something we'll all be keeping our eye out for. Other than the milestones that were mentioned briefly in the presentation, are there any upcoming catalysts that investors or the audience should be aware of before we wrap up in a few minutes?

Yes. The keys to our sustainable growth over the next 5 years plus is execution on our U.S. opportunity. And as I mentioned, we have a lot more confidence that we are getting to the undiagnosed patient population this early in our launch, giving us a lot of confidence that this market opportunity is somewhere between 300 and 900. We just don't -- it's too early in our launch to know exactly what that looks like, but this has given us a lot of confidence. The other one is in regards to our geographic expansion. We know that there are a large number of patients in Europe, as I mentioned, 1,100 patients from a prevalence perspective, and the majority of them have been on miglustat and where our product is used in combination with miglustat. So we see that geographic expansion as an important next step, while we also tap into the ex Europe, ex U.K., ex U.S. named patient basis opportunities to expand access for patients there as well. And lastly, it's really about the Patent Office too. What we see there and what they come back with will be a great opportunity for us in terms of immediate re-rating. And it gives us time to go out and make sure we're impacting more patients that we can. Any month or a year that we can continue to invest in getting patients diagnosed and treated with a product like MIPLYFFA, it's going to be great for us all.

Awesome. Just quick last question. To address those goals and visions that you've just outlined, what would you consider the team's strongest tools to kind of impact that? You have a strong balance sheet, we've been seeing that. But anything else at the -- that you could consider an asset or tool that the company is leveraging to make sure those visions can come true?

Yes. We've spent a lot of time over the last 12 months as part of our strategic planning process refining the first strategic plan we had in 2024. And part of that has been around how are we going to show up as an organization. It started with the mission and vision and values that I discussed. And we're trying to redefine how to impact rare disease and how to get product to patients. I think that that focus that we have throughout our entire organization and the people that we've brought together, and we'll continue to build the culture of running through walls for patients and executing, is what's going to make us one of the most successful rare disease companies in the future.

Amazing. Those are all my questions. Any closing remarks from the team or anything else?

Thank you for being here. What a crowd. Appreciate you on a Thursday.