Okay, great. Well, thank you for joining the healthcare conference here at Oppenheimer. My name is Frank Brisebois. I'm one of the biotech analysts at the firm. The next presenting company is called Zevra Therapeutics. From the company, we'll have CEO, new CEO, Neil McFarlane, to present. Then what we're gonna do here in terms of format is we'll have the company present for 20 minutes-25 minutes, however long it takes. The whole period is 30 minutes, and then we'll follow up with some Q&A. If you have any questions, feel free to put them in the Q&A tab. If not, my email is francois.brisebois@opco.com. So with that, thank you very much, Neil, for joining, and I'll let you take it away.
Frank, thank you so much for having us. I appreciate the opportunity to share Zevra's story with you and let you know where we're off to. I will be making some forward-looking statements today, so I'd like to make sure that everybody goes to the most recent filings in order to get the most up-to-date information. It's an exciting time for us at Zevra. We're on a journey to become a leading rare disease company, with a mission to be able to bring life-changing therapies to patients with rare diseases. We've got a great team that's come together. We have a team focused on making sure that we can commercialize and have access to patients with rare diseases, the therapeutics that we're developing. And we've got a growing pipeline that'll allow us to be able to continue the company growth.
Talking about the team, I've most recently joined along with a number of other team members who have deep expertise in the rare disease experience. You see some of the names here that are the flagship rare disease organizations that the team has come from and launch experience as we become this commercial rare disease company. It's important, as we continue our journey to becoming a leading rare disease company, that we establish and have very robust relationships with advocacy partners. You see some of the partners that we have here today, in the rare disease space. It is imperative that you work with the advocacy partners to be able to drive the awareness, but also the movement through the development into the commercialization.
Let's talk a little bit about our diversified portfolio. We're a commercial stage company, today. We brought on board OLPRUVA, which is for urea cycle disorders. And in that first launch took place a few weeks ago. We have arimoclomol, which is for Niemann-Pick disease type C. And most recently, we refiled with the agency and now have a PDUFA date for June 21st, 2024. And I think it's imperative that I talk a little bit here around arimoclomol and bring back to the patient advocacy organizations. We're actually working directly with the advocacy organizations, and they're in full support of our program moving forward for Niemann-Pick disease type C. We also have celiprolol, which is for vascular Ehlers-Danlos syndrome, and that phase III is ongoing. We have a program that was developed internally for KP1077 for idiopathic hypersomnia.
That phase II top line data is expected in the first half of 2024. And we also completed a phase I trial in narcolepsy with the same agent. Those that package together will inform us moving forward. AZSTARYS is a partnered product that we receive royalties and milestones from, and that is being commercialized by Corium. So with this diversified portfolio, you see that we're a commercial stage company today that's building a portfolio of rare disease assets that'll allow us to leverage the synergies of the infrastructure that we've built.
Let me take a moment to walk us through a little bit of what we're doing on the commercial side in order to be able to ensure access to for patients to our therapies. It really starts with having a great overlap in centers of excellence between our OLPRUVA commercial infrastructure that we're building today, and then with the approval of arimoclomol moving forward.
We have rare disease specialists that are on the ground calling on prescribers and physicians. We have a patient services organization along with the resources to be able to help patients and their families navigate through this treatment journey. Marketing organization, medical affairs, and patient advocacy, and I can't harp enough on the patient advocacy components on what we do to be able to advance the education and awareness and diagnosis of patients, for physicians. But in addition to that, an account management structure that allows us to ensure that we've got the appropriate coverage that we need to allow patients to go through the, what we call the, specialty pharmacy, pathways to get, products for patients. Talking about our commercial product, we have a product, OLPRUVA. It is a nitrogen scavenger. It removes excess ammonia.
As I mentioned before, urea cycle disorders, and certain urea cycle disorders, have an enzyme deficiency that doesn't allow patients to be able to cycle through the urea cycle, nitrogen. Then you get buildup of ammonia, can lead to neurotoxicity, cognition, neurocognitive challenges, potential coma, and even death in certain cases. It is a orphan disease in urea cycle disorders. And again, this poor adherence can lead to neurocognitive damage. There are about 1,000 patients diagnosed in the U.S. About 800 of them are treated today. They're treated with phenylbutyrates. And across the board, there's an unmet need in the palatability, the odor, the route of administration, that allows for patients to really have some challenges in the adherence.
What we have done with OLPRUVA is provided a unique formulation with a single dose envelope, with a polymer-coated product that allows for rapid dissolution, but also allows you to have this taste masking for up to five minutes. It's a convenient single dose envelope that allows for patients to have better palatability, less odor, ease of administration. And we hope that this will allow for better compliance and then better outcome for patients. Let me quickly move towards the pipeline. As we're moving forward, our lead program that we have right now in our pipeline that has right now been submitted to the agency for a PDUFA June 21st is first for Niemann-Pick disease type C. It's a progressive lysosomal storage disorder where you get buildup in cholesterol leads to cell death.
Arimoclomol is a program that may enhance the cholesterol metabolism through lysosomal function and allows patients to halt the disease progression. I'm gonna go into a little bit of the prevalence here. There are about 1,800 patients diagnosed worldwide, about 900 patients estimated in the United States, of which 300-350 patients are currently being treated. This is a devastating disease. The mean age of death in these patients is 13 years old. It is irreversible, and a fatal disease. This neurocognitive decline that I talked about, it adversely affects these patients and their ability to swallow, to have fine motor movement, to then they have all of the cognitive areas that go along, cognitive deficiencies that go along with this t errible, terrible disease and an unmet need t here are no approved treatments in the United States today.
We're hopeful that arimoclomol will be positioned to be the first line treatment for NPC patients. We have put together a package with evidence that indicates that it acts in multiple areas to reduce the lipid buildup and provide for better cholesterol metabolism in the lysosome. It's the first in class treatment. It's an oral capsule that'll be given three times a day. Extensive clinical experience in this program with over 600 patients being treated in a number of different indications with a safety profile that we think is very tolerable for patients.
The data suggests that, with our ongoing expanded access program, the natural history disease, and with over 150 patients in our expanded access program over both the U.S., which has about 70 patients, and the rest of them around the world, that patients are willing to go through the hoops to really get there. And we've now gotten data that's been in our package that we've resubmitted to help us tell the story of arimoclomol and its ability to impact the lives of patients with Niemann-Pick C. It is an orphan-designated product that has fast track breakthrough, and we will be eligible, if approved, for a rare disease pediatric voucher.
The next program we have that is currently in phase III is for vascular Ehlers-Danlos syndrome. This disease impairs connective tissue and then leads to vascular rupture. Celiprolol is actually designed to be able to reduce the mechanical stress on that collagen within the arterial walls, and allow for patients to have less events. It is a autosomal dominant disease, and it's a mutation in COL3A1 gene. A lot of these events happen before the patients turn 20 years old, and about 90% of events occur by the age of 40.
So this is also a disease area that is prevalent in the United States. And it's an orphan disease, it's prevalent in the United States for about 7,500 patients diagnosed in the U.S., t here are no approved options in the U.S., and the current treatments are really focused on surgical intervention of these arterial ruptures that take place in large vessels.
Celiprolol is approved in Europe, and it's become the primary treatment for vEDS in several European countries, but there is no current approved therapy in the United States. Where celiprolol comes in is that it is a selective adrenergic modulator, and it's thought to be able to increase vascular dilation and smooth muscle relaxation, which then allows for the reduced mechanical stress on the collagen fibers and the arterial walls. There was some clinical experience done in Europe that allowed for proof of concept for us to move forward in a study in France. There's some long-term data there. Now we're moving forward in our phase III program in a decentralized, virtual program that is under a SPA with the agency. We will have a new chemical entity regulatory status, and there's IP out to 2038.
Let me pivot here to our phase two program in KP1077. Right now it is for idiopathic hypersomnia. This is a sleep disorder, a rare sleep disorder that causes excessive daytime sleepiness, and sleep inertia, which is that real challenge of being able to wake up, and then brain fog. It is also characterized by excessive long sleep times, and even with long naps, they're unrefreshing. So this sleep inertia and brain fog continues to be an unmet need in the idiopathic hypersomnia area. The prevalence is about 3,700 patients in the United States, and the total population we think could be much larger than that. Today, patients are treated with an approved medication, and then also the stimulants are also utilized throughout the day.
There are comorbidities and complications, like cardiovascular issues and patient demographics that challenge the current medication profile with the use of stimulants and contraindications with antidepressants and contraindications with contraceptives as well as antidepressants and antihistamines. We think that the unique PK profile of KP1077, which is a proprietary prodrug of methylphenidate, allows you to be given this product in the evening when you go to sleep, and the release profile allows you to be able to have a high level of stimulant when you awake in the morning, when you need to get up and have that push to be able to wake and get you through sleep inertia. So the hypothesis for us is that this unique PK profile will overcome some key symptoms, allow for better tolerability, and having less drug-drug interactions with the contraceptives and antidepressants.
We believe that the orphan drug designation and having solid IP will allow us to be able to move this program forward in IH, and the program will read out at the end, in the first half of this year, the phase II data, which will then allow us to move forward into a phase III program. On this last slide here, I wanna reiterate our mission. The patients you see on this slide are real patients in the areas that we're focused on, working with the advocacy partners. We want to be able to bring life-changing therapies to people living with rare diseases. We have a rare disease team that's got strong experience in the commercial launches and how to be able to provide white glove service to this community.
We have a growing pipeline that we believe will allow us to be able to have synergies that we've currently built to be able to commercialize in this rare disease space. Between our arimoclomol PDUFA date of June 21st, our celiprolol program that is ongoing, and our KP1077 top-line results that will be in the first half of 2024, we believe we have a very strong story to be able to build a leading rare disease company. With that, I would like to thank you and open the door to François for a few questions.
Great. Well, thank you, Neil. It's very, very good background on the company. A lot going on. I was just wondering, maybe just a little more high-level because you haven't been at the company for a very long time, when you did your due diligence, can you just help us understand what kind of triggered you to want to join the company?
Yeah. Yeah. Thanks, Frank. So, you know, I've been in the rare disease community, as you know, in multiple different iterations and with various companies. Zevra really spoke to me. It's not often you find a commercial stage rare disease company with a pipeline that is under the radar, quite frankly, of a lot of investors and operators like myself, that has the ability to really impact the lives of patients in the way that Zevra does. So for me in the diligence, there were a number of these exciting pieces that led me to believe that we can set up a management team and a team of employees that can really drive in the rare disease space and deliver value to patients and shareholders.
Do you think, like, why, why do you think that is, that it's been a little bit under the radar? What, what's the history of Zevra? Where does it come from? How did it get to market again?
Yeah. The parent company of Zevra made a pivot a few years ago to becoming a rare disease company from being a prodrug company. With that pivot, they made some in-licenses and some acquisitions of rare disease assets that they could really put together the synergies that were necessary. You know, a single product company becomes very challenging in any disease area, but the ability to have a let's call it a foundation, as a commercial stage company with a product that was approved, it lowers the bar for regulatory risk.
But then having the talents within the organization to be able to invest in a product that had a CRL in arimoclomol, but know that the history of the organization and the talents to overcome CRLs and provide a very clear story from non-clinical to clinical to clinical trial data, working with patient advocacy organizations to get products approved, I think, provided me and I think a lot of other people that have joined the organization with a lot of faith that that pivot to rare disease is something we can execute on.
Okay. That's great. And you just touched on something interesting. So it's always, you know, it's always good to know the history a little bit. So, arimoclomol had a CRL. And can you just help us understand the history there, for what you can share about interactions with the FDA and how we finally got to a PDUFA date of June here this summer?
Yes. So the company that was previously developing arimoclomol is being developed in a number of indications, not only for Niemann-Pick disease type C, but also for Gaucher disease and other areas. The complete response letter that came from the agency was not a request of, "Hey, you have to go out and do another phase III trial." It was really focused in three areas, one in regards to the scale, another in regards to the statistics and how to impute missing data, but also the confirmatory evidence that's necessary in these rare diseases where you have such small patient populations that you're working with and how to be able to tell that story. The company, Zevra and the talents that were here, had overcome a number of CRLs in the past.
So they had a history in the end, and I think the scar tissue built up in order to be able to know that they felt like they couldn't answer these questions, with multiple interactions with the FDA, and then providing some substance of new ways to impute the scale, ways to be able to move forward with the missing data, that we've been able to do. And then also how to be able to add additional non-clinical studies to the package that tell the story of non-clinical to clinical data with natural history, with our open label extension data that now goes out after four years, as well as the clinical data.
And it tells a very nice story that the agency, you know, with the acceptance of our package has, you know, basically said that you have answered the questions and now you have your PDUFA date. So it's a review issue at this point in the game, but we do believe that we put our best foot forward with the talents within the organization. And with the advocacy organization, I might add, you know, there are a collection of a number of advocacy organizations that came together to put in an informal petition to the agency in support of arimoclomol. And we think that that's a very strong component that adds value to the need for this program to be approved by the agency and flexibility be given, as they often say.
That's great. And, you know, brutal disease, super rare, ultra rare disease. You mentioned that there's nothing approved for it now. What do patients do? What is the, you know, there's nothing approved, but they're still probably taking care of the patients somehow. What is the standard of care to make sure, you know, investors can feel comfortable that this should be first line?
So today there is off-label utilization of a number of symptomatic, disease control areas and also a product called miglustat that is approved in Europe and not approved in the U.S. for Niemann-Pick disease type C, but it's approved for another indication that's being utilized. I think the quality of our package, and there were a number of patients within our study that were on miglustat as well as arimoclomol. I think what we've shown to the agency is those patients who have miglustat and arimoclomol or arimoclomol alone; we've given that data. We believe that the strength of the package that we've put forth will allow for arimoclomol to be a foundational therapy for Niemann-Pick disease type C.
Oh, yeah. It must have been, yeah, so rare. It must have been difficult to enroll the trial, especially if there's a treatment or there's off-label usage or you can't get these patients, you know, to wash out necessarily. How comfortable do you feel that, you know, PDUFA is coming up in terms of the commercial side to it? You know, how, you know, how aware that just can you talk maybe about the market education? Is that easy because there is another product that's been used? Like, how quickly could we hit the ground running once approval happens here, hopefully?
Yeah. Thank you for the question. I think this gets back to the second slide in our deck. Our engagement with the advocacy partners who are very much aligned in this area has delivered. We have. I hope our exceptional partners, but we've delivered as much awareness and education, and they have in turn supported where we're moving forward with. But of the 350 patients in the United States that are identified and are on, and some of them are on various treatments. I mentioned our early access program. We have approximately 70 patients in the United States that are currently receiving arimoclomol through our expanded access program.
And we're hopeful that the awareness of both the informal petition that went through that had over, we understand that had almost 1,000 comments, I'm sorry, 1,000 participants who, you know, signed into that letter. That really shows you the level of awareness for arimoclomol. And this was in support of the approval of arimoclomol to the agency. But in addition to that, we've had, as I mentioned, 70 patients that of the 350 that are already in our EAP program, that we'd like to make sure we continue to serve.
Okay. Okay. Great. And then, maybe if we turn to there's a phase II coming up here, the readout in IH. You see a lot of companies. The sleep space has has definitely gained a lot of interest. I mean, there's been kind of a one player for a long time in terms of biotech or pharma. It's been around and and recently there's more players in the space. Rarely do you see companies go after IH before narcolepsy? Or I was just wondering, is it because the narcolepsy space is a little more crowded? Any thoughts there on how you strategize, you know, kind of prioritizing IH over narcolepsy, at least to start?
Yeah. Well, I think a lot of this starts with what is the unmet need? And today, as you mentioned, the sleep space has a lot of players in there. One that has predominantly driven, I think, a lot of the innovation and treated a lot of patients in the sleep space. But when it comes to our KP1077 program, which is our serdexmethylphenidate program, and the ability to be able to capture the unmet need that still is in the idiopathic hypersomnia space, which is this sleep inertia. Even though there is an approved product in this rare area where there's 37,000 patients, we believe today, there is still an unmet need, and that is in the sleep inertia.
The current therapy today, you take at night and then you have to wake yourself up in the middle of the night to take it again so you don't have this excessive daytime sleepiness. We believe that, you know, and again, our hypothesis, the data has to share to show this, but our hypothesis is such that, being able to give a stimulant at night, at the levels at which we can, which are higher than that you can take in a normal day and then have this release profile that then gets you when you need to wake up in the morning, that stimulant already on board.
We believe that fills the unmet need and can, and again, the hypothesis is that we'll wait for the phase II data, but we believe that will allow patients to be able to get up and get that sleep inertia, hopefully be able to impact the brain fog and the cognition challenges that come with that, and then really be able to get that excessive daytime sleepiness under control during the day as well. So we a lot of players, I think, have done wonderful things for the patient community and the sleep community, but there's still an unmet need that we believe we can hit with IH. And then we'll see where we can go from there, if narcolepsy or other areas fit the profile that we're bringing to the table.
Yeah. No, that makes a lot of sense. It's just striking to me, you know, because when you speak to docs in the space, it does seem like as much as these patients sleep an extended period of time and they're not rested still, the true IH patients really sleep for a long time. But what's interesting is it's not that they need to wake up. I mean, sometimes life just forces that. But what's interesting is the docs will tell you that they need that sleep to get some sort of rest. So to wake them up in the middle of the night for a second dose does sound kind of counterintuitive to the disease. But, you know, sales are still going well. So there is an unmet need in IH. And so that's interesting.
And that 37,000, I mean, this, it's a rare disease, but it's not ultra rare, right? It's, and it, you know. It seems like the diagnosis is not always simple, but as the awareness grows, the population, this might be a pretty big rare unmet need sort of thing. So, okay, that's very helpful. And then maybe if you can help us understand just in terms of the business a little bit and how to strategize, do you guys share your, you know, what's the latest cash position that you've shared publicly on probably the third quarter call here? And where do you think that gets you?
It is. Yes. No, we shared that we had approximately $83.5 million in cash at the end of Q3, and we'll be going through our year-end numbers moving forward. At that time, we announced that we had enough cash to get us into 2026. I think the important part for us is that anticipated having a commercial launch of arimoclomol and the expenses that were needed to be able to do that. We've brought those expenses forward by six months by launching OLPRUVA in January and hired commercial field organization from sales, market access, inside sales, medical affairs, and patient services. That expense was anticipated in what we had in our numbers.
And once we get to our Q4 numbers, I think we'll be able to shed a little bit more light now that they're actuals, in Q4 as we, you know, developed and then launched. And you know, I want to pat the organization on the back a little bit here. We closed a deal at the end of November, and within six weeks, we hired the field-facing organization and our ability to really lean in and raise that awareness of OLPRUVA in the UCD space, and launched a program in six weeks.
I think that goes to tell you a little bit of the team that's been put forth here and their ability to, you know, not just recruit and hire people who have got 20 and 30-year experience of being able to launch programs in ultra rare space, but they know these centers of excellence and they have the depth of relationships that are necessary for us to increase the awareness of OLPRUVA and, with an approval of arimoclomol, leverage that infrastructure to do it, and convert those EAP programs and try to get arimoclomol to as many patients that can be helpful.
That's great. Well, that's great on my end. Hopefully the conference can help get some more eyes on the story. I do think it's a little, you know, it's definitely not as popular as it probably should be here. So, hopefully this helped. Anything else, you know, and congrats on joining as a CEO recently. Anything else you wanted to mention maybe before we end the call here?
I want to thank you, Frank. I appreciate the opportunity and spending some time with us this morning. We're looking forward to spending time with the slate of investors that have lined up for us at this meeting. So thank you so much. We look forward to keeping in touch and enjoy the conference.