Zevra Therapeutics at Citizens JMP: Strategic Focus on MyPlayfa and Soliprolol

On Wednesday, 07 May 2025, Zevra Therapeutics (NASDAQ:ZVRA) presented at The Citizens JMP Life Sciences Conference 2025, outlining its strategic initiatives. The company showcased the successful launch of MyPlayfa for Niemann-Pick Type C disease and emphasized its strong financial position following the monetization of a Priority Review Voucher. While the MyPlayfa launch exceeded initial expectations, challenges remain with the relaunch of Opruva.

Key Takeaways

• Zevra’s MyPlayfa launch for NPC disease has received positive feedback from physicians.
• The company holds a cash balance of $217 million, bolstered by a PRV sale.
• Opruva’s relaunch strategy now targets a specific patient segment after initial setbacks.
• Soliprolol is in a Phase 3 trial for VEDS under a Special Protocol Assessment with the FDA.
• Zevra plans to file for MyPlayfa’s MAA in Europe later this year.

Financial Results

• Zevra reported a cash position of $68.7 million before the PRV sale.
• The PRV sale netted $148 million, raising the cash balance to $217 million as of April 1st.
• The funds will support MyPlayfa’s launch, soliprolol development, and potential acquisitions.

Operational Updates

• MyPlayfa Launch:

- Received 109 prescription enrollment forms by the end of Q4, covering about one-third of diagnosed patients.

- Focused on increasing disease awareness and diagnosis rates through media and campaigns.

- Transition from the Expanded Access Program to commercial drug is ongoing.

• Opruva Relaunch:

- Initial relaunch efforts were unsuccessful, prompting a strategic pivot.

- New focus on OTC and female carriers, highlighting portability and personalized dosing.

• Soliprolol (VEDS):

- Phase 3 trial is underway with FDA’s Special Protocol Assessment.

- Patient re-enrollment focuses on those with COL3A1 diagnoses.

Future Outlook

• Strategic Plan:

- Emphasizes commercial execution, pipeline development, and corporate foundation.

- Short-term goals (2025-2026) prioritize current program execution to enable future opportunities.

- Exploring strategic acquisitions to leverage infrastructure and development pipeline.

• European Market:

- MAA filing for MyPlayfa is planned for the second half of the year.

- Expanding the EAP in Europe to provide patient access ahead of potential approval.

Q&A Highlights

• MyPlayfa and Miglustat:

- Physicians’ experience with Miglustat benefits MyPlayfa’s launch, despite its off-label status.

• European Market Opportunity:

- The total addressable market in Europe is similar to the US, with faster penetration expected due to higher diagnosis rates and prior Miglustat use.

For further details, please refer to the full transcript provided below.

Full transcript - The Citizens JMP Life Sciences Conference 2025:

Unidentified speaker, Conference Host: We’re gonna go ahead and get started. Thanks again everybody for joining us this morning at the Citizens Life Sciences Conference. Really pleased to be joined next by Zevra Therapeutics, CEO Neil McFarlane, CFO LeDuane Clifton. Zevra is a company focused on, developing and commercializing therapies in in the rare disease space. The company has a portfolio now of approved products.

The the the one we’re focused on most near term is is MyPlayfa for Niemann Pick type c, which was launched, towards the end of last year and and and, you know, look, our our view is we’ve been really impressed by the launch so far. So so welcome Neil and LeDuane, and and maybe I could just ask you to give a a quick thirty second, intro.

Neil McFarlane, CEO, Zevra Therapeutics: Yeah. Thank you. Ledwain, quick, quick intro.

LeDuane Clifton, CFO, Zevra Therapeutics: Hello. I’m Ledwain Clifton. Been with the company for about ten years, and I think this is a very exciting time for the company as we’ve got two commercial products, product of a lot of strategic planning over time and seeing it come together along with a very solid corporate foundation has been a great journey.

Neil McFarlane, CEO, Zevra Therapeutics: Shall we go on? Sure. Let’s make it happen.

Unidentified speaker, Conference Host: So mentioned that MyPlay for launch is going really well. Can you maybe just give us the background to the product, a little bit of an overview of the clinical profile, and how you approached the launch last year?

Neil McFarlane, CEO, Zevra Therapeutics: Yeah, thank you. So we’re really pleased with, the work that happened prior to launch, leading up to launch, then, the launch for a rare disease, as you mentioned, Niemann Pick disease type C, which is a lysosomal storage disorder, and you get buildup of cholesterol in the cells, which leads to either visceral, presentations in the early diagnostic phase or more, psychiatric, presentations in the later phases of the disease. MyPlypa, as you mentioned, was approved in combination with Miglostat in September of last year, and a great breakthrough for patients having the first product ever approved in The United States for Neiman Pick disease type C. And with data that’s quite strong. In combination with Miglostat, we see that you have approximately a two point difference between miglustat alone and the progression of the disease through the Niemann Pick C Severity Scale, and then actually halting of the disease.

And when you think about neurodegenerative diseases that continue to progress, the holy grail of what patients, families, and physicians want to see is how can you halt the progression. That’s what our data says through twelve months based on the NPCCSS with a very acceptable safety profile.

Unidentified speaker, Conference Host: Right, so a true disease modifying profile. Absolutely right. Can you just give us a little bit of background on the disease? You said it’s a lysosomal storage disorder, but how many patients are there? Where are those patients?

And what are they, treated with today? Understanding that you were the first FDA approved therapy.

Neil McFarlane, CEO, Zevra Therapeutics: Right, so in The United States there are approximately nine hundred patients from a prevalence perspective and of those nine hundred, three hundred to three fifty of them have been diagnosed. Importantly, you know, the data that I mentioned that’s in the FDA approved label doesn’t also mention the fact that we’ve had an ongoing expanded access program for patients up to five to seven years, both on efficacy and on safety. And this data and the strength of the data and over the long duration of therapy, think has been part of the success that we’ve had from launch to today.

Unidentified speaker, Conference Host: Great. You mentioned the EAP program and you’ve and rapidly transitioned patients over now to commercial drug. Reimbursement is never easy, right? So can you speak to just how that process has been and how you’ve been, what you’ve done to help patients get onto therapy and get onto reimbursed therapy as quickly as you have?

Neil McFarlane, CEO, Zevra Therapeutics: So let me start with the EAP program because I think this is an important component. As I mentioned, the longevity of the data really differentiates MyPlifer from anything else that’s out there and that’s been developed to date. We traditionally had about 70 to 80 patients in our EAP. And after we had our advisory committee last year and the strength of the data came out and the support from the advisory committee and then leading to approval in about a short period of time, about a month thereabouts, we had a rush of patients who we like to say these are the really in tune patients or patients who are in the patient advocacy orbit. They are patients who are wanting and and willing to get into clinical programs like an EAP.

So we had this, you know, half a dozen patients that came in at the very tail end that were trying to get into our EAP prior to approval. And then once we had those patients, we started to be able to then work with the sites. We had about 13 to 14 EAP sites and patients had to go to the site to be able to get their previously quarters worth of medicine. Then we were able to get that down to about a month worth of medicine. Patients were being seen on a regular basis.

That really allowed us to catapult our transition of patients from the clinical program into prescription enrollment form that would then actually lead through reimbursement and then patients on commercial product, while supporting them through patient services, while supporting them with field reimbursement, and then obviously while supporting the education of the payers when it comes to the strength of our data and why it’s so important for patients to be on a disease modifying therapy.

Unidentified speaker, Conference Host: Can you speak to what you’ve heard from physicians early in the launch? That’s obviously there’s physicians that have been, have used the drug before, had patients in the AP, but you’ve also had patients that are new to therapy since the launch. So what are you hearing from physicians about what attributes of the drug they like, what they’re seeing in patients in the early experience?

Neil McFarlane, CEO, Zevra Therapeutics: So I think that having launched a number of rare disease programs and been in the pharma industry for a while, you always get the same things once you launch a program. Physicians say, I need more data before I prescribe it, or I need more safety data, or I need more efficacy data. And and, you know, for us, you know, it was a challenging time to get, my PlyFit to the marketplace. But it also allowed for us to continue to invest in this long term dataset. So when you come to market and you have data that’s not just twelve months in a disease modifying label, but you have five years worth of of longitudinal data and patients and physicians who’ve had experience, it lends itself to those physicians who know that in the rare disease space that have heterogeneous presentations, you need multi therapeutic, avenues in order to be able to treat these patients.

And then you’ve got those physicians now, and we’ve heard from those physicians that, they’re now able to be able to drive both with Miglestat and MyPlifa, which is one on our label, and look at the modality of therapies that they have more than one to work with. The opportunity for those physicians who are seeing patients and getting them for the first time on a disease modifying label comes with very different types of feedback, which is, a lot of times, education that medical science liaisons and our field representatives are doing on the data itself. They have never utilized a disease modifying therapy. So it’s a bit of the folks who’ve been there who’ve had a long duration of therapy on MyPlifa and Minglostatin, and then those who are new to therapy that we’re having to educate both the physicians, the office staff on how to move forward with, especially pharmacy, and then pulling patients through through the bottom. So, our efforts are paying off.

As as you mentioned, through the end of q four, we had a hundred and nine prescription forms and prescription enrollment forms that came in. That’s approximately a third of the diagnosed patient And next week we’ll be announcing earnings for our Q1 number, which we’ll be pleased to share

Unidentified speaker, Conference Host: next week. So I won’t ask you that number now. You mentioned basically a third of the diagnosed patient population now has a patient enrollment form. How should we, and a lot of that, a good portion of that was in the EAP, so how should we think about the cadence moving forward of new patient adds? Mean, of those remaining 200, two 50 patients, how well identified do you think they are?

How accessible do you think they are to go and get on drug?

Neil McFarlane, CEO, Zevra Therapeutics: Well this is part of our learning and this is part of what a launch is all about. As we have now started to expand beyond our EAP, which again are these very engaged patients’ families, you know, run through walls to try and get, anything they can for their families. There are also families who have been treated for just symptoms. You may have had epilepsy as your symptomatology, you may have had some visceral symptoms, and only treated with symptoms. That’s why it’s important that we continue to educate, do our disease state education, you know, get our reps out there and making it happen, and driving.

Our media campaigns that have been moving forward have been incredibly touching. Our earned media, profiles that that have been going out have been picked up nationally on the national stage. We launched a new disease state awareness campaign that’s got really good insights, and and next week, Josh will talk more about some of the benefits that we’re seeing there. But it’s not just about the 300 to three fifty patients. We’re also seeing newly diagnosed patients now as well, which is a very different learning for us.

So it’s the three fifty to the 900 patients that we’re seeing as part of this global, you know, our US Campaign Of Disease state awareness and making sure people know there’s actually a product available for these families now. That opens up new doors and we’re seeing newly diagnosed patients as well. So different learnings for different segments if you will.

Unidentified speaker, Conference Host: So clearly part of the answer to this question is that there hasn’t been an approved therapy before, but why is the diagnosis rate as low as it is and what does that tell you about how you can get that diagnosis rate up? I mean, I’m not sure there’s an answer to this part but where can you get to 600 diagnosed patients? Can you get to 900 diagnosed patients? What are the pulls and pushes about finding as many patients as possible for the therapy?

Neil McFarlane, CEO, Zevra Therapeutics: Well, there’s a marker that we utilize and something that actually has already been done as a comp that we like to be able to share. In Europe, there’s about 1,100 patients from a prevalence perspective. And we know that, based on our work and based on some of the publications that there are, the size of the diagnosed patient population in Europe is much higher than that in The US, and a lot of that is attributed to the fact that they’ve had an approved product in Myglustat there for a decade. So we’ve seen this gap between the diagnosed patients and those from a prevalence perspective through disease state education and through awareness campaigns really drive towards the prevalence number versus towards the diagnostic number we see in The US. So that comp for us gives us a lot of confidence that our ability to be able to now have a product approved, now get the disease state education out there, and really drive towards all of these varying things we announced on our q four call, you know, genetic testing availability, obviously all the media work that’s happening.

And the media work is really important. I can’t tell you that as a rare disease company with a rare disease business model where we have a very focused footprint, about a dozen people between medical affairs, sales, patient services that that handle our centers of excellence for for MyPlifa, we need the ability to have surround sound and and people who pick it up and do it for us as well. Some of these media, earned media that’s come up has come into not dozens of markets, but over a hundred different markets nationally across The United States. That is how we are gonna amplify our voice, and that’s how we’re gonna continue to make sure we execute on this rare disease business model.

Unidentified speaker, Conference Host: Can we talk a little bit about Miglustat? So your label specifically speaks to the combination of the two products. How has that impacted the launch helped or in any way pressured the launch and do you expect to see more patients go on to Miglustat now because MyPlifer is approved?

Neil McFarlane, CEO, Zevra Therapeutics: I can’t answer the second part of that question if more patients will go on to What I can say is that the experience we’ve seen through our specialty pharmacy and the enrollment form for MyPlifa, again, we are commercializing MyPlifa. The label says, you know, our prescription enrollment form says, MyPlifa, what dose do you need? How many times a day? And are you on or have you been on Megalostat? Check that box.

We don’t promote it per se because it is off label, but I do think that our early experience has shown that because there’s a large percentage of patients who are on Miglostat, you know, we see between sixty and eighty percent in all the data or have been on or on it, that prescribers to get an unapproved off label product that that is not cheap through a specialty pharmacy system, they have experience. So that experience helps us when we now bring a product that shows you a label that says, Miglostat alone, here’s the progression of the disease, Miglostat and MyPlite, but together you you you, halt the progression of the disease. That education for us and the ability for them to then go out and do another specialty pharmacy order for a product that has a potential to impact the disease course, we believe it’s very helpful.

Unidentified speaker, Conference Host: Let me ask a question a different way. Looking forward, how much monotherapy use of MyPlayFit do you expect to see?

Neil McFarlane, CEO, Zevra Therapeutics: I can’t answer that on what we expect because this is really early in the launch. But what I can tell you is that we have had and seen patients who have been on monotherapy or have been on miglostat before, unable to tolerate it, and physicians have been willing to actually, put their patients onto MyPlifa. Yeah. I mean in our own clinical trials, in our phase three clinical trials, eighty percent of the patients approximately were on Myglostat. So we had, although it was a small number of patients and hard to read into what that data is, patients that were not on Myglostat, it happens.

Unidentified speaker, Conference Host: Still early in the launch but you said on the 4Q update that you are seeing patients getting refills. When you think about the patients that are new to drug, are you seeing refills in that population? And I guess how long, when can you start talking about what the persistence rate is on the drug?

Neil McFarlane, CEO, Zevra Therapeutics: Really too early. You know, five weeks is all we had medicine and channel at the end of Q4 and obviously now we have a full quarter. What I can tell you though is that our expanded access program has shown us that patients when they get onto MyPlayFa stay on MyPlayFa. Again, up to five years, in the data. You saw this in the, advisory committee meeting and and the patients who’ve talked about the impact that MyPlifer has made on their lives and on their child’s lives.

So we see a huge level of consistency in that seventy to eighty patients that we had in The US. Actually, we ended at eighty three patients. But in addition to that, we’ve had a global EAP program running in Europe. And in our European global we call it our global EAP versus a US EAP. We’ve had 70 to 80 patients consistently now also for a long time.

So when we think about persistency and compliance, the best, comp we have is actually our EAP patients who, by the way, they have to run through hurdles, to go to the site every quarter, to get, you know, data generated and to do all those types of things. You know, it’s a real blessing that these patients do that, but they continue to do it for a reason. So we think the compliance rate, that’s our best comp that we’ve gotten. We expect with the safety profile that we have for the product that we’ll see that in the real world experience play out. How do you think about the European opportunity for the drug given that Miglestat is approved there?

What are your regulatory submission plans and commercial strategy in Europe? So to lay it out, about 1,100 patients in Europe from a prevalence perspective, a lot more diagnosed patients, I can’t give you an exact, but it’s a lot more than The US, and Miglustat which has kind of been there in the marketplace for some time. So as we think about our data out of our phase three and that you had Megalstat versus Megalstat and MyPlifa and the split as I mentioned, we’re now talking about the opportunity to bring something that can help help patients in Europe as well. So we took the FDA package, we then went to two different outside consultants to help us to address some of the grounds for refusal that came through previously, and now with all this new data, we announced on our Q4 call that we’ll be filing our MAA in the second half of this year. And in addition to that, we’re gonna continue to expand on our EAP program in Europe to make sure that patients now with an FDA label and the data that’s out there, that we’ll be able to continue to drive patients into our compassionate use programs.

When I think about the market opportunity in The US if you just go with the 300 to three 50 patients that are there today and a much larger number of patients who are being diagnosed and treated in The US, we see the TAM of about the same. Even if you calculate the fact that in Europe we would expect to see a lower price, You see a faster penetration because there’s more amiglestat that’s been utilized in larger patient numbers. So as as we’re ramping The US market, and again, exceeding all expectations, nobody in this company, maybe I think maybe you might admit this too, but I’m not sure if you can. We’re exceeding expectations by a long shot in terms of how fast we’ve transitioned to EAP and brought on de novo patients. We expect that if, you know, with a European label, we can actually do that even faster because there

Unidentified speaker, Conference Host: are more patients that have been diagnosed. We have written multiple times that you’re exceeding expectations and, that’s on management too, this great execution. We’ve written that too. No, it’s been a great launch. So another thing that I think is really important right now is balance sheet cash runway.

Part of the MyPlay for approval came with a PRV. You very successfully monetized that at higher value than I think anybody was expecting prior to the approval. Can you just speak to cash position, how you think about how that enables you to continue to execute on the MyPlay for launch?

LeDuane Clifton, CFO, Zevra Therapeutics: Well we’re in a very good position as you noted. We reported out that we were sitting it with the PRV sales that came in on April 1. We already reported our cash balance. We were at 68.7. We got proceeds of net proceeds of 148 so it put us at $217,000,000 of cash on the balance sheet.

So that puts us in a great position to continue supporting the launch and making sure we execute and focus on getting the MyPlifle launch, taking care of all the activities that Neil just mentioned, continuing to support all approval launch and also working to accelerate the rate of enrollment in our soliprolol program, which is our phase three for VEDS. So we have all we need to do that and so now we look forward to just seeing how we continue to execute, look how these revenue trends change, and then we’ll see where it goes from there.

Unidentified speaker, Conference Host: So with that backdrop of a really strong cash position, Neil, over the last couple of years you’ve really focused the company on this portfolio of rare disease assets. You’ve prioritized within both the commercial focus and the pipeline. How should we think about looking forward? How you’re going to continue to to generate or build shareholder value? Are there more assets that you could bring into the platform?

You know, clearly the focus today is on MyPlayFo is likely the focus tomorrow as well, but just how should we think about the the broader strategic vision of the company?

Neil McFarlane, CEO, Zevra Therapeutics: Yeah. So you know, over the last, eighteen plus months, the company’s had undergone a transformational, change. Right? We’ve made a lot, as you mentioned, we’ve made a lot of of very focused decisions that have allowed us to then execute, with the infrastructure we’ve had and and to have this success, quite frankly, we’ve had. Two things that are important.

We we’re not doing this just because we had one or two We do we’re doing this because we delivered to the board a strategic plan. And that strategic plan, had a had a four pillars that we started to outline at the end of q three last year, around, commercial execution or commercial excellence, we call it, you know, pipeline and innovation, our teams. We have to be able to get our talent and culture right, and then the and then the corporate foundation as as LaDuane mentioned. So throughout that, we have short, medium, and long term goals.

The short term, this twenty five to twenty six time frame is about executing what we have in front of us to earn the right to do something else. Yep. Now, we also had in there a lot of different scenarios and strategies. So a lot of, you know, the $217,000,000 that LeDuane talked about gives us 217,000,000 reasons to be able to have more scenarios that we can execute on to see how we might be able to leverage our infrastructure, how we can leverage our development pipeline to see where we can go from there. But it starts, everything starts in that short term window of executing what is here today.

It’s our commercial launches and it’s our late stage development pipeline. Then we’ll earn the right to do something else.

Unidentified speaker, Conference Host: Got it. So maybe we could just talk about a couple of those other assets for minute. Opruva is your other commercial stage asset that you’re commercializing yourself. There’s been some headwinds there with that launch. Can you talk a little bit about what you’re doing today and what you’re doing differently to push that launch forward?

Neil McFarlane, CEO, Zevra Therapeutics: Sure. So it started, as you mentioned, we relaunched the program in February. We then, came out of the gates, with really great engagement with physicians and awareness of the product that had very limited awareness, but the pull through was challenging. And we went back to the drawing board and looked at all the feedback we’ve received, and then we executed on some work that allowed us to pivot We needed to be able to go after where the best clinical differentiation of the product supported the patients.

That’s an OTC, more female carriers, patients who could actually benefit from the portability of the product, the palatability of the product, as well as the personal personalized dosing where you could actually, differentiate from the competitors that were out there. We kicked that off in in q three. We’ve been executing on it now in q four early. It’s gonna take some time to move forward, but we will update next week on our earnings call kinda how that strategy is coming together. I I quite frankly think that it’s providing us with some learnings that will allow us to continue to to to deliver on the strategy, but it’s about making sure that we put the product in the right place to differentiate against the competition and then see if we can get a toehold.

Unidentified speaker, Conference Host: LeDuane mentioned, soliprolol in that phase three program. You you went through a review of the program and decided to keep investing in the phase three program. Can you tell us why you did that and how you’re, how you think about the value opportunity for saliprolol?

Neil McFarlane, CEO, Zevra Therapeutics: Sure. So so as you mentioned, prior, as part of the the Acer acquisition that, we made about a year and a half ago, we did an analysis of not only the of the Acer portfolio that we, acquired, but also our internal portfolio of of a lot of work that has been done over the decade plus, that the company had invested in. And one of those areas was the VEDS program. It’s the Vascular Ehlers Danlos Syndrome program. There are about seven thousand five hundred patients in The US.

And, this was a program that, had actually tried to do a paper filing, didn’t work out so well, but had then the ability to work with the FDA to get a single phase three under a SPA decentralized trial, 150 patients, two to one randomization that allowed us to be able to continue to invest in that program for a truly unmet need and nothing else in development for those patients. And what we believe is a clinically de risked program because, saliprolol is available in Europe and is utilized off label as a standard of care in certain European countries and has been for some time. Gives us a lot of, faith that we can invest in this program, which in a very well designed trial under a SPA, and then try to be able to get this product to patients in The US. It made a lot of sense for us to do it. We then kind of went through the screening patients who had been on sidelines for some time that wanted to get into trial, but under the previous sponsor wasn’t able to actually enroll because they ran out of funds.

Then we’ve gone through that now, we’ve started re enrollment, we’ve got patients on board, got the program opened up. Now we’ve been able to work centers that have call three A1 diagnosed patients and make sure that they know that there’s a trial available to them to be able to enroll in now. So we’re starting to see some progression there, but we’ve got to be able to enroll this trial quickly because it’s an event driven trial and as part of our ability to execute and get in the product to market means that we’ve got to get the trial enrolled as fast as possible. Great. Well, Neil, LeDuane, really appreciate you being

Unidentified speaker, Conference Host: with us this morning. Exciting times and looking forward to the earnings update next week.

Neil McFarlane, CEO, Zevra Therapeutics: Thank you. Thank you.

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