Zevra at TD Cowen Conference: Strategic Shifts in Rare Disease Focus

On Wednesday, March 5, 2025, Zevra Therapeutics (NASDAQ: ZVRA) presented at the TD Cowen 45th Annual Healthcare Conference, outlining its strategic advancements and challenges. CEO Neil McFarland highlighted the company’s focus on rare diseases, emphasizing significant transformations over the past year. While the company showcased promising developments, it also faces hurdles in its commercial and clinical endeavors.

Key Takeaways

• Zevra has transformed significantly, focusing on rare diseases with two commercial-stage products.
• Recent FDA approval of MyPlifa for Niemann-Pick disease type C marks a major milestone.
• The company plans to sell a pediatric priority review voucher for $150 million.
• Zevra’s commercial infrastructure supports further program expansion.
• Strategic alternatives are being explored for KP ten seventy-seven.

Financial Results

• Zevra has entered a definitive agreement to sell a pediatric priority review voucher for $150 million.
• The company reported approximately $95.5 million in cash at the end of Q3.
• Compassionate use and expanded access programs in Europe generate about $2 million per quarter from pre-commercial sales.

Operational Updates

• MyPlifa has seen 90 prescription enrollment forms in the first five weeks post-launch, with 30% approved for reimbursement.
• The launch of MyPlifa has exceeded expectations, with 69 forms from the expanded access program and 21 new patients.
• Zevra is re-establishing its MyPlifa application in Europe after a previous withdrawal.
• Olprova is being targeted towards active adult patients for urea cycle disorders, with a focus on a single-dose envelope for ammonia control.

Future Outlook

• The Phase 3 DISCOVER trial for Saliprolol in vascular Ehlers-Danlos syndrome is re-enrolling patients.
• Zevra is seeking strategic alternatives for KP ten seventy-seven, with potential expansion into narcolepsy.
• The company is optimistic about supporting additional programs with its existing commercial infrastructure.

Q&A Highlights

• Zevra’s commercial infrastructure, consisting of approximately a dozen sales and commercial staff, is poised for expansion.
• Mygliostat is widely used among patients, with 80% having been on the treatment.
• CEO Neil McFarland expressed satisfaction with the launch progress and the company’s financial position.

In conclusion, Zevra’s presentation at the TD Cowen Conference highlighted both its achievements and ongoing efforts in the rare disease sector. For further details, readers are encouraged to refer to the full transcript below.

Full transcript - TD Cowen 45th Annual Healthcare Conference:

Stacy Ku, Biotech Analyst, Cowen: Okay. Well, good morning. Thanks so much for everyone to be joining our forty fifth annual healthcare conference. I’m Stacy Ku, one of the biotech analysts. I’m joined by my colleague, Vish Shah.

So it’s our pleasure to introduce Neil McFarland, CEO of Zevra, who’s gonna be giving us a presentation today. So looking forward to it. Thank you.

Neil McFarland, CEO, Zevra: Thank you, Stacy. Thank you, Cowen, for having us. Look forward to spend a little bit of time running through a deck with you today. And I’ll start by stating that we’ll be making some forward looking statements. So please take a look at our filings, our most recent filings on our website for most up to date information.

Zevra’s had quite a transformational, twelve months. And then if we think about what we are all about, it’s about the opportunity to impact people living with rare diseases. We’ve got two late stage sorry. Two commercial, stage rare disease products. We’ve got an advanced clinical pipeline with a phase three program as well as an a phase two program that, has read out data.

I’ll talk a little bit more about that in a minute. We’re building an infrastructure to support our future growth at this point. And we’ve got a fairly strong financial position with some updated information that I could talk about later on as well. So we’re really building the company to impact patients living with rare diseases. If I focus on what our mission is as we’re bringing these chain life changing therapeutics to the community, there are three really important things that we focus on.

One is execution, and we’ve got to focus on a few things, and we got to continue to innovate. The resources that we are actually, building today are around four pillars. We talked about this on our q three call around our strategic plan. And these four pillars are commercial excellence, pipeline and innovation, talent and culture, and corporate foundation. And and I’ll work through these as we walk through today’s presentation, but on commercial excellence is about our ability to synergize our products, supporting the patients that we’ve got, and building, that muscle that allows us to be able to do it again and again after we earn that right.

It’s about building and innovating on a pipeline that leverages the infrastructure that we have on our commercial, business. Building a team, that’s not just rare disease expertise, but people that are built around a culture that really want to make an impact in the lives of patients, and demonstrating that financial discipline with our corporate foundation that allows us to continue to build a sustainable business moving forward. We do that with having the right people around the table. What you see here in, in the team that’s that is the c suite here, but in addition to all of our teammates, they come with rare disease experience, rare disease product launches, and understand how to be able to make that impact for patients. I want to talk a little bit about the portfolio because this is really how we’re going to deliver that value.

Most recently, we’ve got an approval of MyPlifa, which has been developed for Niemann Pick disease types type c. We got an approval in September of last year, and we put the medicine in channel at the November. And, with with solid intellectual property as well via orphan drug exclusivity. Oprova, which is a product that we acquired out of our our ACER transaction about a year a little over a year ago, and that is for certain urea cycle disorders. That product’s been launched, and we’ve have great synergies between both of these commercial assets that allow us to be able to leverage an infrastructure that is quite lean, on the commercial side.

We’ve got a partner product in Astarus, which was developed, by the by the company and, we collect royalties and milestones from, the sale of Astarus through our partner. And then on our pipeline, I think it’s importantly enough, one of our catalysts coming in 2025 is around how do we expand our MyPlifa, Niemann Pick disease type c program ex US. And we’re exploring regulatory pathways in Europe and other countries, and and understanding our MAA filing and how we move that forward will be our next big catalyst in 2025. We’ll we’ll come back to you in, and let you know more about that as we uncover, the appropriate approaches to take there. And then with saliparol, that’s a phase three program being developed for vascular Ehlers Danlos syndrome.

That’s an ongoing trial, and we’re enrolling those patients as we mentioned in our q three call. We restarted enrollment, and we’ll be talking more next week on our earnings call about the updates in q four. The phase two program I mentioned earlier on was KP ten seventy seven. It was being developed for idiopathic hypersomnia as well as narcolepsy. We actually delivered our end of phase two, or our phase two data, which was positive.

And then we went to an end of phase two meeting with the agency. We have a pathway forward for a single pivotal trial, and, and understand the safety data that we need to collect as well in the ten seventy seven program. However, we’re looking for strategic alternatives for that program versus doing it ourselves because the end goal of being able to provide a commercial infrastructure that would be able to, adequately get KP ten seventy seven of those patients in the sleep space is a much larger infrastructure and not really provides this doesn’t really provide the synergies that we’re looking to try and continue to develop and leverage with our commercial infrastructure for MyPlifa and Opruva. So we’re in in the process of of, doing that right now. Let me, switch briefly and focus on what what, is near, and and we’re executing on, which is around our commercial infrastructure.

We’ve got a very efficient team that’s, providing access to our medicines that are commercially available today. It starts with our rare disease specialists. We’ve got the marketing support. We’ve got patient reimbursement services, account management, and and from a national payer perspective as well as from a regional payer perspective. And we surround that with very solid medical affairs and patient advocacy in this space of of rare disease, which is critical to impacting, small disease populations.

We’ve got a program called Amplify Assist that makes our, it’s a comprehensive support system for getting patients on an individualized basis through this, challenge of getting medicines to patients through the payer coverage, through education and disease state education, as well as some personalized insurance coverage. So we work through that process through our Amplify Assist program for both of our products, which I think is an important perspective as well. We do that for My Plifa and for Olprova and get some synergies out of that as well. I’m gonna spend a few minutes talking about My Plifa, our most recent launch, and, you can take a look at the for treatment of patients two years and older with Niemann Pick disease type c. The reason you see these different colored boxes is because it’s a weight based therapy.

It’s between, it’s it’s given orally, or via feeding tubes, three times a day in various dosages that you see here. Quickly about Niemann Pick disease type c, it’s a neurodegenerative lysosomal storage disorder, builds up of cholesterol in the cell. And this progressive buildup of lipids in the cell leads to cell death. And ultimately, based on, what when you present earlier, you may have more visceral, type, implications of the spleen, liver, and and the brain, but then actually the older presentations you may see more, issues with the brain. It it it results in loss of cognition, speech, the ability to swallow, fine motor skills, ambulation, which is also part of the tool that is utilized to be able to show disease progression over time.

And and, and these these once you lose these skills, you don’t get them back. So moving forward, about eighteen hundred patients from a prevalence perspective between The US and Europe, about nine hundred of those patients from a prevalence perspective in The United States, and approximately three hundred, three hundred and fifty patients are diagnosed or treated through some work that was done previously through ICD nine and ICD 10 codes in The United States. I mentioned that you can have onset at any age and the differences between earlier onset and later onset. There are there’s more to that, but, for the sake of time, I wanna, move on to some of the data. The average age, is about 13 years old in terms of mean age of death.

About eighty percent of the patients that we’ve seen in all clinical trials, whether it be ours or others, and the experience we’ve seen in our, expanded access program are on Myglastat. And then it’s a primary treatment and approved for use in Europe, but not in The United States. So as, as we move to the next slide, I mentioned, and I’ll mention this in more detail around the efficacy data that we saw around twelve months of treatment. The NPCCSS, which is the only validated score that allows for, the progression to show the progression of the disease. We saw halting of the progression of the disease with myofin combination with myglyastat, and I’ll talk about this in a moment, versus a two point progression of disease, which is significant.

A one point progression of the disease can actually mean the difference between somebody ambulating or being in a wheelchair or the ability of them having to feed themselves or needing to be fed by others. It’s it is a, devastating, disease. And from a safety perspective, very well tolerated, adverse effects were mild and moderate, and you can see this in the prescribing information. And we’ve actually had a long, time frame of patients who have been in the clinical trials to the open label extension to the expanded access program and then all the way through now, now converting to commercial drug, but over two seventy Niemann Piksi patients between our expanded access program in Europe as well as our expanded access program in The U. S.

Had been treated. So we have quite a history of from a safety perspective. I mentioned the dosing three times a day and, I also mentioned some of the differentiation and efficacy data. Let me get into that in a little bit more detail here. And and I mentioned to you, if you look at this chart on the bottom left, what you see in the rising bar over twelve, rising, line over twelve months is progression of the disease with a patient who is, an eighty percent of these patients actually, all of these patients were on miglostat only.

You add myofen, what you see is no progression of the disease or even halting of the disease through twelve months when you add myglistat and myplyfa together. I mentioned that, this is the revised four domain NPCCSS, and one point of progression is meaningful. Two points of progression is life changing for patients, and and we’re happy to be able to provide, my Plifen in combination with Myblestat to these patients. On the right hand side, you see the safety profile tolerated, well tolerated safety profile. And, and as I spoke about earlier on, this is information through October 31 that we announced on our Q3 call.

Of those three hundred to three fifty patients, we had 90 prescription enrollment forms. So physicians that, enrolled patients, for MyPlifa in the first five weeks post launch. Sixty nine of those were came from our expanded access program, and twenty one of those patients, importantly, were new patients de novo to MyPlifa and were outside of our EAP. So and and then the the statistic on the bottom, all of these, are are, dated because they’re through October 31. We’ll provide more information next week in our earnings call.

But at the time, October 31, approximately five weeks post launch, we had about 30% of the prescription forms at that point that had been approved for reimbursement. Now you can imagine we have more prescription forms, we have more enrollments, and more coverage determination as well that we’ll, we’ll talk a little bit more about next week. I also mentioned that, the next major catalyst for us is also understanding the regulatory pathway for an MAA in Europe. We are we’ve been working through the FDA documentation to be able to understand how the best approach is to reestablish our application in Europe. And, we’re we’re not ready to to let folks know what’s transpiring there yet because we’re still working through that internally.

But very soon, we’ll be able to, announce a pathway. In Europe, importantly, we have about seventy to eighty patients that are in compassionate use and expanded access programs today that are being treated with, aramoclonal And, and through the French, through a French program that allows for pre commercial sales, we net about $2,000,000 per quarter, out of that program. And and again, plan to continue to, have, aramoclonal and and hopefully MyPlifa in Europe shortly for patients across the continent. I wanna skip quickly to, the program that we launched earlier about a year ago, actually this time last year, that we launched after our acquisition of Acer Therapeutics. It’s Ultruva.

The prescribing information is available online. And this is been developed, for certain UCD certain UCDs. There are about six enzymes and a couple of transporters that, process ammonia and nitrogen out of, the body. When you have defects in some of these enzymes or transporters, you get buildup of ammonia, and, and nitrogen and, you actually get neurotoxic and cognitive damage that actually is irreversible. So there are nitrogen scavengers that are out there or a number of them are that are out there.

Olpruva, has been developed as one of those nitrogen scavengers to that’s got a clinically differentiated profile that we’re offering for for patients. The marketplace in The US, about eleven hundred patients diagnosed, approximately eighty percent of them are in the the enzymes, that we are approved for, which gets to about eight hundred patients receiving therapy in The US. Important part is of those eight hundred patients that are receiving therapy, still twenty five percent of them or so have hyperememic crises, and a lot of that is based on adherence. Olpruva was and I’m gonna skip to the next slide here. Olpruva was developed, through a five zero five b two filing pathways, but to be able to actually provide some of the differentiation to some of the products that are out there today, which I’ll talk about in a minute, and had a safety profile that’s consistent with the other sodium phenylbutyrates that have been on the market for decades.

The dosing is one of those areas that is actually a differentiation. It’s a convenient single dose envelope, and we call it ammonia control on the go. It was developed to be able to have palatability and and and, an adherence type of of differentiators. One being that you can actually put this, dissolve it in water and, was designed to be, taste masked for up to five minutes And, and provide that palatability that patients don’t have with some of the current therapies that are out there today. As I mentioned, poor adherence, recurrent hospital visits, and and patients that are not optimally treated, or have mild diagnosis that, don’t take medicines or they may have diet or other areas that they do to control their nitrogen buildup.

But these are some of the real reasons why OPRAV was developed. And as I mentioned, we’re driving this adoption of OPRAVA in multiple, avenues, but it’s we we pivoted our strategy in q three to those more active adult patients that are looking for that ammonia control on on the go. They were missing their midday doses because they, had to pull out, you know, an oil and take it in the middle of their day. We wanted to be able to provide a more discreet therapy, for these patients and a more private opportunity for them to take their and manage their ammonia. And we’re working through that strategy right now.

We’ll have more in terms of, the adoption of that strategy as we get through the next couple of quarters, and, we’ll we’ll talk more about that on our call next week as well. I wanna quickly, in the last few minutes that I’ve got, talk about our pipeline. One is, Soliprolol. Soliprolol is a program that’s being developed for the treatment of vascular Ehlers Danlos syndrome, which is a connective tissue disorder, and it’s the most severe of the the Eilers Danlos syndrome subtypes. It’s, it’s a caused by COL3A1 gene mutation.

It leads to defect in the vessel walls, of arteries as well as hollow organs. And, you can have aneurysms, you can have dissections, or you can have ruptures of hollow organs, with this connective tissue disorder. There’s a significant unmet need for VEDS. In The United States, there are no approved actually, there are no approved treatments in The US or in Europe, but I’ll talk a little bit about that in a moment. About seventy five hundred patients in The United States, have a, live with vEDS and about ninety five percent of them are genetically confirmed COL three a one diagnosis.

So, it’s about twenty five percent of the patients experience an event before the age of 20. Ninety percent of these patients will experience event by the time they’re 40. Their median age of survival is 51 years old. And quite frankly, the only treatment, today is to surgical intervention if you know that you have it and you’re monitored, to see if you can fix the the, ruptures and or, and catch them ahead of time. The one thing I mentioned a moment ago, it’s there’s no approved treatment for vEDS in The US or Europe.

However, soprolol was approved decades ago and found to be, effective in vEDS patients in Europe, and it is the standard of care in several European countries today, but is not available in The United States. Talking a little bit about, the potential treatment of saloprolol for VEDS patients, it’s designed to reduce the mechanical stress of that constant, pressure that comes by, on the five on the, arterial walls. It’s a selective adrenergic modulator and and the mechanism of action is thought to be through vascular dilution and smooth muscle relaxation. So you don’t have that pressure build up on on the walls and that leads to then a rupture. There is quite a bit of data, that’s been developed on silyprolop throughout the years.

There was the trial called the B BEST trial, that showed, improvements with silyprolop versus non treated patients. There’s long term French data that, that was put forth also showing benefits. There’s an observational Swedish study that showed benefits. And then our DISCOVER trial, which is ongoing right now. So there’s a lot of data out there showing that saloprolol, has the ability to, impact, VEDS patients.

And we’re running this trial. It’s a 50 patients, randomized decentralized trial. And we have started reenrolling patients after a hiatus from the previous sponsor at the end of q three. And we’ll talk a little bit more about our our path forward as well as, impact on what we’ve done over the last quarter, on our call next week. Switching gears, very quickly to KP ten seventy seven.

This is the program that, certix methylphenidate we were developing for idiopathic hypersomnia. Idiopathic hypersomnia is a devastating, sleep disorder that, it’s debilitating by nature in regards to just being chronically tired. It’s characterized by excessive long sleepy times. You still actually never feel refreshed even after long naps. And, there is a, it’s usually measured by the IHSS, which is idiopathic hypersomnia severity scale or the traditional Epworth sleepiness scale.

And I’m gonna quickly run through this because I’m running out of time. There are approximately thirty seven thousand patients in The United States with IH. The about sixty six percent of these patients still have, report symptoms of not being treated well enough, and have excessive daytime sleepiness, brain fog, so on and so forth. About eighty five percent of them need more than one therapy, and, and the current treatments just don’t seem to address the needs. So let me tell you a little bit about our our program.

Certix methylphenidate, is a proprietary prodrug of d methylphenidate. We looked at two different dosing regimens once a day at bedtime at very high doses, that then allowed for patients to be able to get their doses elevated throughout the evening and then get a high dose of stimulant on the board approximately ten hours later when you’re trying to get patients up, which is what they have, a lot of times with with the sleep inertia that they get. We looked at both the safety and tolerability of existing treatments and, we saw some greater tolerability, some lower cardiovascular effects, and there’s no drug drug or there’s there’s limited drug drug interactions with other unlike other sleep, areas. So there’s some real differentiators, that we bring with certax methylphenidate. It’s got orphan drug exclusivity.

We we the important part here is you see some of the data on the right hand side, which was very encouraging for us to be able to go to an end of phase two meeting with the FDA at the end of q three and allowed us, to be able to work with them on on a path forward for a single pivotal trial, with collecting the appropriate safety data that will allow us to will allow us to be able to move forward into IH and the potential to expand into narcolepsy. After looking at both the clinical, the the clinical development pathway as well as the commercialization needs that we have, it just doesn’t have a fit that we have with what we’re currently doing in the ultra rare 40 centers of excellence, the high synergies that we have versus the sleep, space, which is a slightly diff it’s a different call point than what we have. We decided that we would look for some strategic alternatives, and we’re we’re exploring that with outside parties today. I am going to stop in one minute, and let you know that, this data that I’m showing you right here was as of Q3.

We talked about, having approximately $95,500,000 on the balance sheet at the end of Q3. Obviously, the state is a bit stale. What I can tell you though is is that, last week, we announced, definitive agreement to, to sell the pediatric or the priority review the pediatric priority review voucher that, we received with the approval of MyPlifa for a hundred and $50,000,000 to an undisclosed party, and we’re working towards closing that in the next, thirty to forty five days. So with that, we feel like, we’re in a even stronger position, from a a corporate foundation perspective. And I’ll leave you with, we, we believe that, Zevra is a unique opportunity, for not only patients that we serve, but also for investors that are looking in, investing in an organization that’s got two commercial rare disease programs.

We’ve got a pipeline that, is both late stage, which which we believe is derisked from a clinical perspective, and we’ll we’ll do the trials accordingly. But in addition to that, also discipline that, we understand if we start something in a phase three program, we gotta be able to commercialize it and are looking to be able to find the appropriate partners for $10.77 for phase three ready sleep program. And, we’ve got an infrastructure here that’s supportive of future growth. We have two programs that we commercialize with a very lean infrastructure, and we’re and and our goal is to be able to execute, what we have today and earn the right to potentially leverage our commercial infrastructure in the future. As I mentioned, between commercial revenue, royalties and milestones and, the monetization of a PRV, we feel like we are in a strong, position to be able to execute our mission.

Thank you, Stacy. Wonderful.

Stacy Ku, Biotech Analyst, Cowen: So I wanna make sure you all are given the opportunity to to ask some questions. If if not, we certainly have a few.

Neil McFarland, CEO, Zevra: So the question was, how many programs do we think we can support with the commercial infrastructure and the infrastructure we’re building in the rare disease space. I’ll I’ll say to you that when we think about where we are today in the synergies with with about a dozen, people on the ground between sales and commercial, and then we also have, reimbursement specialists that we we ramp up and down through a contracting organization that we work with that that if there are 40 centers of excellence that we call on today that are the vast majority of these lysosomal storage disease, we could add additional programs into our infrastructure today to commercialize it with our back office, our specialty pharmacy, our hub solutions, all of the other things pretty easily. But what what we have to do right now is execute on what we’ve got in front of us and earn that right. How many MDC patients in The US are Mygliostat? The question was how many patients in The United States on Mygliostat and the typical duration of treatment?

Did I get that right? Okay. What we know, in terms of megalostat utilization is based on our clinical data, our EAP data, other clinical data that from other, competitors that have been out there and and other programs have been developed. Approximately eighty percent of all patients have been on are on Mygliostat or have been on Mygliostat throughout their journey. What we understand from physicians and and, patient advocacies groups as well as patients is is that, the the, safety profile of myelostat and and it’s sometimes GI effects that it has, patients will dose up and down, throughout, but they’ll try to stay on myelostat, as best they can.

So of the three hundred to three fifty patients, our best guess is that eighty percent of those patients, are on megalostat and remain on megalostat based on the safety profile.

Stacy Ku, Biotech Analyst, Cowen: And it might be helpful for you to maybe comment, on the patients that are that you’re kind of seeing get on the FLIPA. It sounds like you do have some patients that are de novo and obviously a little outdated information, but maybe comment on what we’re seeing as we think about maybe those that are diagnosed and treated and maybe those that are undiagnosed and could really enter the funnel.

Neil McFarland, CEO, Zevra: So, great question. And, we are pulling out all the stops from our, educational perspectives, our genetic testing perspective, all the things that we’re doing to be able to make sure that we’re educating people that my PIPA is available as a cornerstone therapy in combination with miglostat. So when we looked at our Q3 data that again, first five weeks, we saw that there were both newly diagnosed patients, patients who were converting, from our EAP program and those who had not never been on the program before. We’ll talk a little bit more about this next week, but we you know, what I can say is is that we continue to see enrollments coming in in those buckets as well. Continuing to have conversion from our EAP patients, continuing to have de novo patients, and continuing to have newly diagnosed patients as well, which I think is that unlocking of the 300 to three fifty to the 900 patients that are that from a prevalence perspective.

So it is early in the launch. You know, I’m I’m incredibly grateful and pleased with our team and and the and and the support from the patient advocacy organizations and all of the people. It takes a village, to unlock and provide these therapies to patients. But I have to tell you, the launch to date is, is exceeding my expectations for sure.

Stacy Ku, Biotech Analyst, Cowen: Wonderful. Well, to exceeding your expectations, that comment, maybe for some context provide your original expectations for maybe when your EAP, would have been kind of run through, so to speak, and maybe, latest public, expectations around when that would close?

Neil McFarland, CEO, Zevra: Okay. So great great question. Two things. One is we had 83 patients that were in the EAP when we when we got approval and and the program was no longer enrolling patients. We actually had about a dozen, and I’m gonna say about a dozen because I can’t tell you the exact number because I don’t know what the and I can’t remember what the exact number is.

Patients that after our advisory committee and the strength of the vote of the advisory committee, there was a bit of a rush to patients wanting to get into our EAP program. So we went from approximately 70 patients in our EAP program up to 83 once we got approval. That was also telling, but in a lot of other rare disease drug launches that I’ve done and others in our organization, usually take about twelve months to get your EAP patients converted. The expectation going in was that, you know, we would see something similar to that. What we mentioned what I mentioned on our q three call is is that actually we accelerated that and we brought that in.

We think that we’re gonna be able to, at that time, in q three, which we announced and we’ll we’ll we’ll tell you the update, next week, that we felt like we’d bring that into the end of q two, which was less than a year. So the expectations of trying to transition all those patients within a year, we now feel well, we had we announced in q three that, we felt we could do that by the end of q two. And we’ll have, an update for, for the public, next week. Okay.

Stacy Ku, Biotech Analyst, Cowen: And and you kind of started by saying you are really focused on the ex US opportunity. So maybe, add a few more details there. Where where where are you in the boots on the ground kind of approach?

Neil McFarland, CEO, Zevra: So, if I bring, the the the audience back, the product was originally filed in Europe First from the previous sponsor and then taken back at, you know, withdrawn the application at about the same time the FDA provided The U. S. With the CRL. So we knew what the issues were and we knew what The U. S.

Issues were. They were very similar and we worked over a couple of years to get the new data that would allow us to be able to get a successful regulatory approval in The US. Now we’re taking that data and actually understand how we can position that for an MAA. And that’s been the work since, you know, all of Q4 and today. The team is doing extraordinary effort in trying to bring the most efficient package we can and understand how we can get this product to patients in Europe.

So about 1,000 patients in Europe, 11 Hundred patients in Europe, as I mentioned in the presentation. Miglostat is approved in Europe and I think much more of a mature market. There are more patients that are treated and diagnosed because they’ve got an approved product. We’re hopeful to be able to bring my play for those patients and then have a cornerstone of therapy and halt the progression of the disease just like we did with, the FDA label.

Stacy Ku, Biotech Analyst, Cowen: Wonderful. Any final questions? Okay. With that, I think we’re on at time or like we’ll touch over. So thank you so much.

Neil McFarland, CEO, Zevra: Appreciate the extra time.

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