Okay, great. Thank you very much for joining here on day two of the Oppenheimer Healthcare Conference. My name is Frank Brisebois. I'm one of the biotech analysts at Oppenheimer. Our next presenting company here is Zevra Therapeutics from the company. We have CFO LaDuane Clifton to present. Thank you very much for taking the time here. And what we'll do here in terms of format is we'll have LaDuane present the company for about 15 minutes, 20 minutes, and then we'll have some Q&A. So feel free to send some questions in the Q&A tab or to my email, and I'll try my best to get to them. But with that, thank you very much again for joining, and take it away.
Thank you, Frank, and good afternoon, everyone. We appreciate you joining this introduction to Zevra Therapeutics. We'll just jump right in and go quickly, but as Frank mentioned, we would welcome your questions during the presentation. Zevra is a rare disease therapeutics company, and this slide really presents for us in a snapshot why we think this is a great opportunity both to bring important products to people living with rare diseases and also a great place to invest in our future. We have two commercial-stage rare disease products, an advanced clinical development pipeline, and existing infrastructure supportive of our future growth and a strong financial position with the balance sheet and cash and resources that take us into 2027. Our mission, as I mentioned at the start, is to bring life-changing therapeutics to the rare disease community.
We intend to do that by executing on our strategic plan and on our commercial launches specifically to focus where we can bring and have the most impact for patients, and then continue to innovate in how we deliver services to those important patients. Over the last couple of years, we've spent a lot of time building both our commercial infrastructure, and we believe we have a best-in-class team designed to bring excellence to the task. We have a pipeline, and we pride ourselves with a history and a legacy of innovation in developing and bringing products forward to approval. We've built a fantastic team with a significant rare disease experience, and we expect that talent and culture ultimately be what we build our future on.
And then, of course, as I mentioned, with a strong balance sheet, we have a corporate foundation to reach forward to our goals, and we intend to do that through prudent investments and maintaining financial discipline. This gives you a quick snapshot of our team, and you can see quickly that we have a significant amount of rare disease experience, both in terms of development, getting through the regulatory process successfully, and then ultimately being able to launch a host of products that have been very meaningful in the lives of people living with rare disease. This slide gives a real nice snapshot of our diversified portfolio, and we'll just start with the first two, and then we'll dig deeper as we get deeper into the deck.
MIPLYFFA, which is our commercially now launched product for Niemann-Pick disease type C, just approved on September 20th, and then officially product in channel in two patients beginning on November 21st. We have been pleased to see the trajectory of that launch as it has begun, and we'll give you more updates as we go through. A second product we have that's in commercial stage is OLPRUVA, and our team is actively working to bring this treatment for urea cycle disorders to patients. This actually is a product that we picked up through our acquisition of Acer Therapeutics back in late 2023. Our team officially launched it at the end of January last year, and we continue to drive forward in making that available to patients.
The AZSTARYS program is actually an asset which we brought forward and was brought to approval originally from our own discovery efforts, went through an entire development cycle, and then ultimately led to approval back in March of 2021. This is a treatment design that is indicated for the treatment of attention deficit hyperactivity disorder, or ADHD, and this one is an asset that is partnered and currently being commercialized by Corium. We receive royalties and milestones on net sales, and we continue to see its impact to us. And while we don't control the commercialization, we follow it closely and look forward to continuing to receive those royalties and milestones as they continue to market the product in that market. Our development pipeline includes arimoclomol. Note that that's the same molecule as MIPLYFFA.
Here, we refer to the fact that now that with FDA approval in our pocket and our ability to launch in the U.S., we now have turned our attention to exploring the regulatory pathway in Europe and potentially other countries outside of the United States. So that's part of our regulatory development pipeline. Celiprolol is an asset which is in an active Phase III trial. It's a decentralized event-driven trial and was originally part of, again, that Acer acquisition I mentioned a moment ago. This is for Vascular Ehlers-Danlos Syndrome, another ultra-rare disease associated with cardiovascular issues and specifically ruptures or the potential for ruptures based on the cell walls, excuse me, the vascular walls. That's an ongoing Phase III trial, and we'll give you more details on that as well.
Finally, we have our asset KP1077, which is intended for the treatment of idiopathic hypersomnia and may also have applications in narcolepsy. This is a product candidate that went through a Phase II, which we completed successfully in mid-2024, and we reported out the results, and we had a follow-up with the FDA as we began the process of designing a Phase III. This product, being in rare sleep disorders, is one that, as we looked through our strategic planning process during 2024, realized that this is a program that has significant value in the right hands, and we think that we're currently looking for a strategic alternative or potentially partners that can take that forward through a Phase III and potentially ultimately be able to commercialize that with a sales force team that is designed to focus in on sleep disorders.
And so you can see we have very much a diversified portfolio with continuing not only two products that we can commercially launch, but also multiple shots on goal in terms of our continuing development to drive value for our shareholders. If we think about the commercial team that we've built, we really have put together a best-in-class style team, which includes rare disease specialists, rare disease-specific marketing teams, patient reimbursement services intended to guide and assist patients and providers as they seek to get commercial approval from payers, account management contracting, and ultimately a solid medical affairs and patient advocacy team that really seeks to support the patient community.
In addition to that, we've introduced our AmplifyAssist program as tools and resources to assist patients through navigating to get to a place where there is support for all of the tasks required to ultimately get approval by payers and see the prescriptions coming through and being able to begin treatment. This gives you a quick snapshot of MIPLYFFA, which, as I said, was just approved in September 2020, is actively being launched. If you don't know a lot about NPC, I'd tell you a quick summary on this slide. NPC is a neurodegenerative lysosomal storage disorder that, if you have this disorder, there's a buildup of cholesterol that leads to cell death. This can happen in many cases, but in other disease states, but in NPC specifically, it tends to focus in on CNS-type areas.
This buildup of cells leading to cell death can ultimately lead to organ dysfunction in the spleen, the liver, and the brain, and as I mentioned, in NPC, it tends to be significant in terms of CNS symptoms. There's about 1,800 people in the U.S. and Europe combined that are diagnosed and that live with NPC. We believe around 900 of those are in the U.S., of which only 300 currently are diagnosed or treated. The onset can actually occur at any age, but it is a genetic disorder, and so often you do see that it begins to take an effect in children quite often. It is heterogeneous in the sense that the way its symptoms appear sometimes is not always the same, and therefore it can be very difficult to diagnose.
Patients often go through a very long period of time trying to different routes to understand what they're seeing in their children, only to find that ultimately it's been driven by NPC. Being an ultra-rare disease, sometimes that's the last diagnosis to be made. The average age of death is 13 years, and so this is a devastating disease. And of note, miglustat, while not approved for NPC in the U.S., is often used off-label to treat it. And in our trials, our studies, around 80% of patients took miglustat, and MIPLYFFA can be taken alongside with miglustat to great effect. So looking at the differences and the differentiation of MIPLYFFA, and based on our label that was approved by the FDA, we have data that indicated that it was proven to be effective using a 5-domain NPC Clinical Severity Scale.
And based on the results of a 12-month trial when used in combination with miglustat, it was actually shown to halt disease progression. And generally, this is a well-tolerated therapy. Adverse events were very mild. And there was a lot of patient experience over the course not only of the original 12-month trial, but then there was an additional four-year extension trial. In addition to that, we had an expanded access program that's been in place for a very significant amount of time, both in the U.S. and in multiple countries in Europe. And so there's been a lot of safety data generated through the various sources there. And we can see for sure that through these different elements, this is a very safe and well-tolerated therapy. Dosing is oral and it's weight-based dosing, typically 3x a day.
It is a capsule, so it has the benefit of ease of administration depending on the state or progression of the disease in the various patients. To be clear, MIPLYFFA is the first approved product for the treatment of the NPC in the U.S., and it's the only product approved in combination with miglustat that shows that it is able to halt disease progression through 12 months. This chart on the left side kind of indicates MIPLYFFA with miglustat and placebo with miglustat. As I pointed out, a majority of patients in our trials were on miglustat about 80%. And you can see here, this is a picture worth a thousand words. And you can see very much the blue line when MIPLYFFA is taken in combination with miglustat, you can see that there's very limited progression in the disease.
We think this really positions MIPLYFFA to be the cornerstone of treatment for patients living with this devastating disease. On the right side, just some data again from our studies and from our label indicating the well-tolerated safety profile, as I mentioned before. If we take a look at our early launch metrics, these are as of October 31st. So when we did our Q3 earnings call back in November, we were able to give this brief snapshot to kind of give a sense of where we were so far. And I will tell you that the enthusiasm for this launch has been fantastic. The patient community had been watching. They were very supportive as we went through the regulatory process with the FDA. And that is certainly. That enthusiasm has come through in the early days of the launch.
By October 31st, again, remember, we were approved on September 20th. We had already seen 90 prescription enrollment forms. 69 were from our expanded access programs in the U.S. And there was an additional 21 patients that were naive to MIPLYFFA that were not actually participants in the EAP. And then as of October 31st, around 30% of those initial forms had already been approved for reimbursement by payers. Of course, you can expect that as we've come into the end of the year and into 2025, these numbers have grown and increased. And we look forward to being able to give a more fulsome update through the end of Q4 at our upcoming earnings call to be announced very soon. As I mentioned a moment ago as well, we have now also turned our regulatory team's attention towards exploring the regulatory path to gain approval in Europe.
We expect to give additional guidance and updates on the potential timeline for filings and the things that will be required to take that forward again in our upcoming earnings call. Of note, one of the EAP programs that we have in Europe is the French EAP. They actually have a program which provides reimbursements in advance of approval. That has been netting us around $2.1 million per quarter in net reimbursements, which again has been a key part of funding and taking forward the development efforts for MIPLYFFA. Now we'll turn our attention to OLPRUVA, which is intended for urea cycle disorders. Urea cycle disorders, or UCDs, is a rare inherited metabolic disorder characterized by hyperammonemia. Whenever ammonia levels are elevated, they can be neurotoxic, leading to neurocognitive damage and even death if left untreated.
Often patients that get into this state will have to go to the emergency room and deal with it in a very careful way. And so OLPRUVA, when taken as prescribed and taken consistently, it does a very good job of managing the ammonia levels for folks. Similar to MIPLYFFA, or excuse me, for NPC, this is also considered to be an ultra-rare disease. There's about 1,100 individuals diagnosed in the U.S. And actually it's less than 800 of those 1,100 are actually seeking treatment. And as I was pointing out a moment ago, more than 25% of hyperammonemic crises stem from poor treatment adherence. And why is that important? There's a number of reasons why we believe OLPRUVA is really a fantastic treatment differentiated for patients. First of all, its efficacy has been clearly demonstrated through a 505(b)(2) filing.
Sodium phenylbutyrate, which is the active there, has demonstrated efficacy for sure. In addition to that, there's a long history of safety with this and other nitrogen scavengers. The key features here of OLPRUVA is that it includes convenient pre-measured single-dose envelopes for ease of use and ammonia control on the go. So for example, other products like RAVICTI, which are in the marketplace, are this oily substance that has to be sort of shot down the back of the throat. It's very difficult to swallow. It doesn't taste very good. It's frankly the reason for their poor adherence. Similarly, other products on the market have almost no taste masking and can be very difficult to take. OLPRUVA is unique because it can be poured into a glass of water, stirred up, and taken very easily, and actually is able to mask the taste for up to five minutes.
And that's because of its novel dual-coated formulation, allowing that delay and therefore making it palatable and easy to take on the go. And so when we think about then, what are we trying to do with bringing this product to patients? We're hoping to improve adherence. Again, we want to avoid the hyperammonemic crises. And that's best done by consistently taking the medicine on time and allowing those levels to never rise to that level. Of course, that would avoid the hospital visits. And then also some folks who choose not to be on a treatment, this would actually potentially be palatable enough to bring them on to treatment. We're doing a lot of work to drive adoption of OLPRUVA through patient experience.
We're very much focused on adult patients who have been diagnosed and that can really see the benefits of having the portability of the product, folks that have a busy lifestyle. In addition to that, we're looking to find ways for people that have already diagnosed, focusing on those where adherence has been an issue, and make sure that they understand the potential benefits of using OLPRUVA in order to address that and ultimately lead to better outcomes. Next, we'll spend a moment on celiprolol, which is the potential treatment for Vascular Ehlers-Danlos Syndrome. As we said a few minutes ago, this is another inherited connective tissue disorder caused by a certain gene mutation. Essentially, it causes sort of a breakdown in the elasticity of collagen in the vascular walls.
At some point, and sometimes that's hard to predict when, that will lead to a significant aneurysm or a potential rupture and often can lead to severe issues or even death. There's around 7,500 individuals in the U.S. that have been diagnosed with VEDS. About 25% of those typically experience an event before the age of 20, which is pretty remarkable. 90% of patients actually experience an event by age of 40. You can see how this can be very much a devastating disorder and sometimes not even realize that you have the disorder. Surgical intervention is the current treatment paradigm. To be honest, celiprolol, there is no approved treatment in the U.S., but c eliprolol has a history as being the primary treatment for VEDS in several European countries. We do believe that U.S. patients would benefit from having this treatment available.
I'm going to kind of continue here. Celiprolol basically is designed to reduce the stress on the collagen fibers within the arterial wall, and thereby then you avoid the potential, at least reduce the potential for the vascular rupture. To be honest, I'm the CFO. I'm not the CMO, but we would be glad to take more questions as we go through this. I would give you a sense that we have the ongoing Phase III trial, this DiSCOVER trial , which is currently ongoing, and it is subject to a Special Protocol Assessment or an SPA with the FDA, meaning that if we get to a certain level of enrollment and we see a certain number of events at the midpoint, that we could potentially stop the study early and then be able to submit if we've met the criteria of that SPA.
Finally, we'll end up quickly on KP1077, our potential treatment for idiopathic hypersomnia. IH is a rare sleep disorder that can be debilitating. It causes brain fog, so really an inability to think clearly. And I don't mean just that you didn't sleep enough the night before, but rather you really cannot focus your thoughts on any specific task. Excessive daytime sleepiness. And then the inability to wake up, sleep inertia without significant help potentially from other caregivers. And so, being able, there's significant unmet needs in this area. And I would say existing treatments do not adequately address several of these symptoms. There's around 37,000 individuals that have been diagnosed in the U.S. with IH. But some recent studies have suggested the population could be larger.
As I've said a moment ago, the symptoms that have been noticed as high impact include the things we described a moment ago. You can sleep and never feel refreshed, chronic fatigue, excessive daytime sleepiness, and brain fog, which really becomes debilitating. Current treatments do not address these needs. Patients have said that medication effectiveness of current treatments is very poor. This is the results from the Phase II trial that I mentioned was completed in the first part of 2024. You can see here that we saw meaningful differences in the Epworth Sleepiness Scale as well as in the Idiopathic Hypersomnia Severity Score. We believe that this is very promising and was very informative into the design of a Phase III trial. With that, I'll come to the last part, which is just give you a quick snapshot at our financial stream.
And really with our just looking focus on our balance sheet, we closed Q3 with $95.5 million in cash equivalents and investments on the balance sheet, which extends our cash runway into 2027. And one thing I didn't mention a moment ago is when MIPLYFFA was approved by the FDA, we did receive a Priority Review Voucher, which you may know is transferable. And so our cash runway does not include the potential proceeds from a sale of the PRV. And if you follow the PRV market, you may know that recent sales of PRVs, there's been three or four recently that were at the $150 million mark approximately. And so this is an asset that we have expressed publicly that we would intend to sell. And we're watching that market carefully for an opportunity to do so.
That would give us an interesting source of non-dilutive capital to come into our balance sheet. In addition, we have a debt facility of $60 million. We currently have $20 million out. We have $60 million outstanding with $20 million tranche available if we wanted to take it down, and then you can see here our common and fully diluted shares outstanding. To summarize quickly, to bring you back, we are pleased that we have an opportunity to impact people living with rare diseases through the two commercial products we mentioned, MIPLYFFA and OLPRUVA, and we are very focused on executing those launches during 2025 to make sure those treatments get to patients and we're able to begin having an impact.
We also continue to be focused on advancing our clinical development pipeline by focusing in on the regulatory submission in Europe for arimoclomol, as well as continuing to work and accelerate the enrollment in the Phase III program for the celiprolol program. Ultimately, as we build out our infrastructure, we want to make sure we do it with an eye toward the future. And we aspire to become a partner of choice for rare disease products, which would allow us to bring incremental value into the organization and leverage the commercial capabilities that we've built. And all of this, as I mentioned before, we'll do through disciplined investment, continue to work through with a strong balance sheet the opportunities that we have in front of us. So with that, Frank, we can open it up to Q&A.
Okay, great. Thank you very much for a good breakdown. There's a lot going on at Zevra, and congrats on the approval and the launch. Those metrics from October, knowing that you got, you know, you launched in September, look really interesting. Is there, you know, without, obviously, you've got an earnings that you said you're coming up on. But in terms of the metrics there, is this sort of market where the penetration should be quick? Or, you know, how should we look into 30% were reimbursed? Is that like, what's the next challenge for people to have a better feel for how the launch should go here?
Yeah, it's really good to frame that up a bit. So I'll start on the payer question. The 30% metric we gave there simply was a function of time. So the enrollment forms were coming in, and 30% of those were able to be processed successfully.
That doesn't mean we were having trouble with the others. It was just literally a process of time. It could take 30 days- 60 days to get through the process with payers depending on each process. So we've continued to see those come through and our patient services team supporting everywhere they can to make that easier for patients. With regard to how you would expect the penetration, I think clearly with 69 patients coming through from our expanded access program in the U.S., I would point out that at the date of approval, we had about 83 patients in the EAP. So that represented a meaningful number of those had already converted. And we certainly have a goal of converting all of those. And so we look forward to being able to provide updates on that.
But then in addition, with 21 additional patients that had come in as of October, again, folks that were watching or maybe had never even been or heard about MIPLYFFA, but certainly not part of the Early Access Program, you know, those are really where we target next. Once you've converted the EAP, then you start thinking about how you can expand knowledge of the product that it's available and make sure they understand how it can benefit patients. So I think there will be sort of this early adoption by the EAP participants and folks that were following the story. And then we go into the next phase, which is driving beyond those to find the next set of patients to take us up to that 300 mark of the diagnosed patients. And that's a part of the process that the team is actively executing on.
Okay. In terms of the pricing here, what has been shared on the pricing side?
Based on the average weight of the patients that were in our expanded access program, I think, and even those that have been prescribed so far, it comes to around $1 million per patient per year for MIPLYFFA.
Okay. There's probably no pushback here. It's a small market, right? So I'm sure that reimbursement hasn't been an issue. Okay.
Yeah, we've seen success with payers so far. And as you pointed out, very limited therapies. We were the first one approved in the space. So payer relationships have been going well. There's always a discussion, always a time where we need to make sure they understand the benefits of the product. And our team is fully engaged in that.
Remind me just quickly, you talked about EU. How's the process going? Is the prevalence similar in the EU? And how's the process going there?
So the EU is a bit more of a more mature market. We mentioned briefly the miglustat product. That's actually been approved in Europe for the treatment of NPC. I believe it's around 10 years or so. So their ability to diagnose because there were treatments available is a bit more advanced. So when you think about Europe, then you have a higher degree of diagnoses there. I think, let's see, with around 900, sorry, with around 900 or so patients in Europe itself, there's a much higher level of diagnosis there. And so we think that when it comes to our opportunity in Europe, particularly with the data that demonstrates that MIPLYFFA plus miglustat has this ability to potentially halt progression, we think there will be an interesting differentiation there.
At the same time, the process to gain approval in Europe, while we did the resubmission to the FDA, they had also looked at the original application and they have their own set of questions, which we are now evaluating and assessing. A lot of the data and the elements of the application for the U.S. will certainly be applicable to those questions. But it's a unique process. And so you have to tailor the submission to their process and their specific questions. So our team is spending a lot of time on the strategy for completing that submission, the required timelines to make sure that we have a comprehensive answer to those questions they had asked when they originally reviewed the application.
Okay. Well, super helpful. Congrats on all the progress. And thanks again for the time today.
Thank you. And thank you, everyone, for joining.