Earnings call transcript: Zevra Therapeutics Q2 2025 misses EPS, revenue shines

Zevra Therapeutics reported its Q2 2025 earnings, revealing a mixed financial performance. The company posted an earnings per share (EPS) of -$0.06, significantly missing the forecasted $2.19. However, revenue came in at $25.9 million, surpassing expectations by 15.16%. The strong revenue performance aligns with InvestingPro data showing impressive 46.47% revenue growth over the last twelve months. Despite the EPS miss, the stock rose 2.04% in after-hours trading, suggesting investors may be focusing on the revenue beat and strategic progress.

Key Takeaways

• Zevra’s revenue exceeded forecasts by 15.16%, driven by MyPlifa’s strong performance.
• The company reported a substantial EPS miss, with a surprise of -102.74%.
• Stock price increased by 2.04% in after-hours trading, despite the EPS miss.
• Significant cash position improvement following a $150 million asset sale.
• Strategic focus on rare disease markets with ongoing product developments.

Company Performance

Zevra Therapeutics demonstrated robust revenue growth in Q2 2025, largely driven by MyPlifa, which saw a 26% quarter-over-quarter increase. However, the company’s EPS fell short of expectations, highlighting potential profitability challenges. The increase in cash reserves from a strategic asset sale positions Zevra well for future investments in its pipeline.

Financial Highlights

• Revenue: $25.9 million, up from the forecast of $22.49 million.
• EPS: -$0.06, significantly below the forecasted $2.19.
• Net Income: $74.7 million, or $1.24 per basic share.
• Adjusted Net Loss: $3.2 million.
• Cash and Equivalents: $217.7 million, a notable increase from the previous quarter.

Earnings vs. Forecast

Zevra Therapeutics’ Q2 2025 EPS of -$0.06 fell short of the forecasted $2.19 by a wide margin, marking a 102.74% surprise. In contrast, the company reported a revenue beat, with actual figures exceeding projections by 15.16%, highlighting a strong sales performance.

Market Reaction

Despite the significant EPS miss, Zevra’s stock rose by 2.04% in after-hours trading, reaching $11.939. This positive movement, against the backdrop of an EPS miss, may reflect investor confidence in the company’s revenue growth and strategic initiatives. The stock remains near the higher end of its 52-week range.

Outlook & Guidance

Zevra continues to focus on expanding MyPlifa’s commercial reach and pursuing European approval. The company is also advancing its pipeline with the Phase 3 DISCOVER trial for ciliprolol and strengthening its position in the rare disease market.

Executive Commentary

Neil McFarlane, CEO, emphasized the company’s commitment to serving patients with rare diseases, stating, "Our vision is to build a leading life sciences company whose core mission is to serve patients by bringing life-changing therapeutics to people living with rare diseases." He also highlighted the importance of their disease awareness campaigns in driving early diagnosis.

Risks and Challenges

• The large EPS miss suggests ongoing profitability challenges.
• Limited uptake of Olpruva raises concerns about product acceptance.
• The adjusted net loss indicates potential operational inefficiencies.
• Market saturation and competition in the rare disease sector could impact growth.
• Macroeconomic pressures may affect future financial performance.

Q&A

During the earnings call, analysts focused on patient enrollment trends and reimbursement processes. Zevra’s efforts to expand genetic testing initiatives were positively received, but questions about the limited uptake of Olpruva highlighted potential market challenges.

Full transcript - Zevra Therapeutics Inc (ZVRA) Q2 2025:

Conference Operator: Good afternoon and thank you for joining Zebra’s Second Quarter twenty twenty five Financial Results and Corporate Update Conference Call. Today’s call is being recorded and will be available via the Investor Relations section of the company’s website later today. The host for today’s call is Nicole Osher, Zebra’s Vice President of Investor Relations and Corporate Communications. Please go ahead.

Nicole Osher, VP of Investor Relations and Corporate Communications, Zevra Therapeutics: Thank you, and welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments followed by a review of our second quarter financial results. I encourage you to read our financial news release, which was distributed this afternoon and is available in the Investor Relations section of our website. Before we begin the call, please note that certain information shared today will include forward looking statements. Actual results may differ materially from those stated or implied by any forward looking statements due to risks and uncertainties associated with Severe’s business.

Forward looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other important factors that may lead actual results to differ materially from the projections made and should be evaluated together with the Risk Factors section in our most recently filed quarterly report on Form 10 Q, annual report on Form 10 ks and other filings with the SEC. In addition, we will disclose certain non GAAP information on today’s call, specifically adjusted net loss and adjusted net loss per share. Reconciling information to GAAP can be found in our financial results news release. I’m pleased to welcome Zevra’s management team members participating in today’s call. Neil McFarlane, Zevra’s President and Chief Executive Officer Lizwain Clifton, our Chief Financial Officer and Josh Schafer, our Chief Commercial Officer.

Our Chief Medical Officer, Adrian Cortell, will be available for today’s question and answer session. Now it’s my pleasure to hand the call over to Neil.

Neil McFarlane, President and CEO, Zevra Therapeutics: Thank you, Nicole, and thank you to everyone who’s joining our update call this afternoon. At Zebra, our vision is to build a leading life sciences company whose core mission is to serve patients by bringing life changing therapeutics to people living with rare diseases. We are demonstrating our ability to execute in a complex regulatory, clinical development and commercial environment to advance promising therapies with an approach that balances science to patient need. As a reminder, this vision is driven by the four pillars of our corporate strategy: commercial excellence, pipeline and innovation, talent and culture and corporate foundation. The execution of our strategy delivered remarkable performance in the second quarter and sets a strong foundation for continued momentum in Q3.

On today’s call, we’ll highlight several key achievements. Q2 net revenue reached $25,900,000 reflecting robust demand and effective operational execution. We completed the sale of our PRV for $150,000,000 and strengthened our balance sheet. We also submitted our marketing authorization application or MAA for Aramako Mall in Europe, marking an important milestone in our geographic expansion efforts. Let’s dive in with an update on The U.

S. Launch of MycLypha, the first approved treatment for people diagnosed with Niemann Pick disease type C or NPC, and the only treatment that has been shown to halt disease progression by addressing its underlying pathology. As a quick reminder, NPC is an ultra rare, relentlessly progressive neurodegenerative disease caused by inherited lysosomal storage disorder and leads to premature mortality. In The U. S, we estimate that roughly three hundred to three fifty patients have been diagnosed with NPC out of the estimated nine hundred people living with the disease.

The approval of MyPlaza marked a fundamental shift in the treatment paradigm. And we believe that its ability to address disease progression is an important differentiator that will lead to long term success. We are incredibly proud of the work our team is doing to support the healthcare community and eligible patients with an intense sense of urgency. We’ve received positive feedback from both patients and physicians, which is reflected in the strong performance since lunch with a total of 120 prescription forms through the end of Q2, of which seven were enrolled in the quarter. Strong Through rapid uptake in the early stages of launch, we have been able to reach over one third of those diagnosed with MPC in The United States.

This group primarily included patients in our Expanded Access Program or EAP, who have been treated with Myplica for up to seven years with the majority of patients remaining on therapy. Following the success of our early launch penetration, our commercialization efforts are focused on increasing both diagnosis and treatment access. Second segment of patients that we’re focused on are those who have received an NPC diagnosis, but may or may not be currently receiving treatment. These patients typically were diagnosed at centers of excellence and managing coordination with a local provider for ongoing care. We’ve mapped these referral patterns beyond the centers of excellence and are engaging the broader group of prescribers.

The last segment of our patients are those living with NPC, but who have not been diagnosed. Part of our contribution and responsibility to the patient community is to increase awareness and shorten the time to diagnosis for this devastating disease. We are demonstrating leadership through our disease state awareness campaign to help drive early diagnosis. The treatment of NPC is multifaceted and our program offers education and genetic testing options for individuals with suspected lysosomal storage disorders. With growing awareness of MyPlica, we look forward to extending our reach to serve additional patients.

Our work with advocacy organizations is very important and our support has been well received. Josh will provide more details on our strong presence at recent conferences where Exevra and our specialty pharmacy engage with caregivers and people living with NPC. These are some of the ways that we’re building our reputation as a reliable partner. Another exciting opportunity for MyPlifer is in Europe, where we estimate approximately eleven hundred people are living with NPC. One of our priorities is to secure European approval and determine the optimal go to market strategy to ensure access for a greater number of patients.

Just a few weeks ago, we announced the submission of the Aramchol MAA to the European Medicines Agency, delivering on the early side of our 2025 guidance. This is another significant and timely milestone for Zevra. To support the submission, we received guidance from EU regulators and assembled a robust data package, including new mechanistic and long term clinical data generated since our FDA submission. We have demonstrated a synergistic effect with nimblastat, which is approved in Europe for MPC and is the standard of care in this more mature market, where we have an established EAP program, which increased to eighty nine patients enrolled at the end of the second quarter. And to further our efforts in the scientific and medical community, our data from the open label extension study was recently published in the Journal of Molecular Genetics and Metabolism.

The paper presents the impressive long term efficacy and safety outcomes for NPC patients treated with MyPlipa in the forty eight month open label extension phase following the end of the twelve month pivotal trial. Additionally, we published data showing expression of genes belonging to the Coordinated Lysosomal Expression and Regulation Network or the CLEAR network, targeting the underlying pathophysiology of NPC to improve autophagy, reduce cholesterol accumulation and prevent cell death. As the NPC marketplace evolves, shaping treatment guidelines will become more important for the NPC community. We believe our success in establishing MyPlaza’s benefit to the largest dataset of NPC clinical trial patients and having these data published in peer reviewed journals will help inform treatment decisions. Turning now to Olpruva, which is a treatment for certain urea cycle disorders.

We’ve previously discussed the limited pull through we experienced in this launch, which continued in the second quarter with one prescription in a woman form. Our refined marketing efforts over the last several months have focused on identifying the patients for whom Opruva is best suited, strengthening our patient support services and addressing reimbursement However, adoption continues to be slow and we remain cautious. Josh will provide more detail on the product performance and LeDuane will provide financial details on the non cash charge related to the prevailing trend later in the call. Moving on to our pipeline, we’re advancing ciliprolol as a potential treatment of vascular Ehlers Danlos syndrome or VEDS in the Phase three DISCOVER trial. VEDS is a severe genetic connective tissue disorder characterized by a risk of dissection and rupture of the arteries, gastrointestinal tract and vascular wall of large arteries and hollow organs.

In the second quarter, we enrolled seven patients in the DISCOVER trial, bringing the total to 39 patients enrolled out of the 150 patients required for complete enrollment of the trial. The genetic testing initiative to identify patients who have the COL3A1 gene mutation, which causes the disease is ramping up in collaboration with patient advocates, KOLs, treating physicians and clinics, clinics, providing increased visibility and has the potential to accelerate future enrollment. We are making progress in advancing our vision of becoming a leading rare disease company through focusing on the patients we serve. Our strategy balances near term operational excellence with the agility and financial flexibility to support initiatives that build sustainable value for patients and shareholders alike. Let me turn the call over to Josh, who will provide more details on our commercial products.

Josh?

Josh Schafer, Chief Commercial Officer, Zevra Therapeutics: Thank you, Neil, and good afternoon. Let’s begin with Myclifa for NPC. Myclifa in combination with miglustat is the only treatment shown to halt disease progression twelve months in a pivotal placebo controlled study using the NPC clinical severity scale, which is the only validated measurement of NPC progression assessing clinically meaningful markers such as impairment of mobility, cognition, speech and swallowing. In the second quarter, we received seven prescription enrollment forms and we have increased our product net revenue by 26% quarter over quarter, a reflection of new patient demand as well as access and retention. A new enrollment is defined as a prescription submitted to our specialty pharmacy, which begins the benefits investigation process to determine reimbursement eligibility.

In the second quarter, our coverage reached fifty two percent of all covered lives. It’s consistent with our expectations at this stage of the launch. But as other patients who are not immediately covered, we’ve been able to achieve reimbursement through medical exception pathways. We remain actively engaged in discussions with payers to facilitate reimbursement. And our current focus is on demonstrating the clinical value of Myclypha to payers, especially leveraging our long term open label extension data, which showed durable clinical benefit for up to five years.

Our field team, including case managers, continues to play a critical role on the front lines, working with patients and healthcare providers to navigate the reimbursement landscape. Their efforts have been instrumental in helping to overcome access barriers and allowing patients to receive timely support to secure coverage. Additionally, our Amplify Assist program, which is a centralized resource that assists clinicians’ offices and patients in navigating the reimbursement challenges and supporting coverage for all of our commercialized products has been well received. Further bolster our penetration into the second segment of patients, those NTC patients who have been diagnosed and are either on other treatments or not being treated, we have a number of initiatives underway. We are emphasizing the robustness of our data with its strong presence at scientific, medical and advocacy related conferences where physicians and patients can learn more about MycWiFA’s therapeutic impact.

Last month at the National Neumann Tick Disease Foundation Conference, Doctor. Barbara Burton, a key opinion leader and professor at Northwestern University’s Feinberg School of Medicine, presented an overview of MyPlaza and our unprecedented long term data. We had a similarly strong presence at the Southeastern Regional Genetics Group Conference with four poster presentations. As Neil mentioned, the recent publication of our open label extension study is our long term forty eight month data to the forefront for physicians, highlighting the duration of clinical benefit. Specifically, an improvement in disease progression was seen at the first evaluated time point at twelve weeks and then continued for more than five years in a heterogeneous population of NPC patients with no new safety concerns.

These results align with our pivotal trial data, which showed that Myplica in combination with progression compared to placebo over a one year study duration. Presentation of key data and insights like those provided in these recent publications are an important part of our strategy to raise awareness of the clinically meaningful impact that MyPlaza can have for patients. And we will continue to execute our publication strategy to support prescribing decisions. The third segment of patients, those that are undiagnosed, are an important population for us. It is critical to shorten the time to diagnosis, to halt the progression of disease sooner.

Our disease state awareness campaign, Learn NPC, Read Between the Signs, is designed to provide educational and genetic testing resources to support the diagnosis of new NPC patients. This program is helping to drive awareness of the disease as well as identifying patients who were previously undiagnosed and may be candidates for MyPlayFlo. Turning to Alpruva, UCDs are rare inherited metabolic disorders characterized by an excess accumulation of ammonia, which can be neurotoxic and lead to neurocognitive damage or even death. Although current treatments are effective, over twenty five percent of hyperammonemia crises are caused by poor adherence to treatment. Alprovo was launched based on established efficacy and safety profile, but with an innovative formulation that offers a powerful option in convenient pre measured single dose envelopes for ease of use and ammonia control on the go.

We believe in the benefits that I’ll prove offers people with UCD. However, we have seen slower than expected uptake. The mature UCD market and patient satisfaction with existing treatments resulted in one prescription enrollment form in the second quarter. An authorized generic to the market leader is anticipated, essentially creating a shift in the competitive dynamics. We believe our Pruvus profile as well as our patient support activities will position us to compete in this changing landscape.

We have been actively engaged in strategic negotiations with payers to facilitate reimbursement. In the second quarter, we saw our overall coverage increased to 79%. In summary, our commercial organization has reached critical mass and the strength of our capabilities are being leveraged across our portfolio with the primary focus on MyPlifo. We are prioritizing and executing on key strategies to deliver value patients living with rare diseases. I will now pass the call to Lou Duane, who will present the financial results for the 2025.

LeDuane Clifton, Chief Financial Officer, Zevra Therapeutics: Thank you, Josh, and good afternoon, everyone. In addition to the financial details included in today’s call, we encourage you to refer to Zebra’s quarterly report on Form 10 Q for more detailed information, which we intend to file later today. In the 2025, we reported net revenue of $25,900,000 which includes 21,500,000.0 from MyPlifer, 300,000 from Alprova, 2,600,000.0 in net reimbursements from the French EAP for Imakamol, 1,200,000.0 from royalties and other reimbursements under the Astaris license and an upfront payment of $300,000 from the out license of dextropen during the period. For our commercial products MyPlaza and Vopruva, we referenced revenue when shipments are received at the specialty pharmacy. Cost of product revenue for Q2 twenty twenty five was $14,000,000 which includes $1,600,000 non cash intangible asset amortization.

We conduct quarterly assessments of the recoverability of certain intangible assets and inventory in accordance with U. S. GAAP. Based on the prevailing trends for OLPUVA, including enrollments, paid authorizations, as well as market and competitive landscape dynamics, we have recognized a non cash charge of $58,700,000 for impairment of intangible assets and an inventory write down of $11,700,000 as of 06/30/2025. Operating expense for the second quarter was $24,200,000 which was an increase of 1,100,000 compared to the same quarter a year ago.

R and D expenses were $3,400,000 for Q2 twenty twenty five, which was a decrease of 7,100,000.0 compared to Q2 twenty twenty four due to a decrease in third party and personnel related costs following the completion of the ten seventy seven Phase two trial. SG and A expenses were $20,800,000 for Q2 twenty twenty five, which was an increase of 8.2 due to professional fees incurred related to the proxy contest earlier this year as well as other expenses associated with our commercial activities. During Q2 twenty twenty five, we also recognized $147,900,000 in other income, which includes $148,300,000 in net proceeds from the sale of the PRV asset, which was completed on 04/01/2025. This one time transaction has provided significant non dilutive capital for the company and has further strengthened our financial position. Net income for the quarter was $74,700,000 or $1.24 per basic share and $1.21 per diluted share.

Excluding the one time PRV sale, the one time non cash impairment charge and the inventory obsolescence charge recognized during the quarter, adjusted net loss was $3,200,000 or $06 adjusted net loss per basic and diluted share for Q2 twenty twenty five. For the same quarter in 2024, we reported a GAAP net loss of $19,900,000 or $0.48 per basic and diluted share. We are pleased with the early stages of the MyPlaza launch and our continued progress in building our corporate foundation through solid execution. Our focus remains on executing the launch of our commercial products and on the development of our pipeline, which includes supporting both the Aramcholam MAA in Europe and the ongoing Phase three trial for ciliprolol. As of 06/30/2025, total cash, cash equivalents and investments were $217,700,000 compared to $68,700,000 at the end of the prior quarter.

Total debt was approximately $60,700,000 We believe that our existing capital resources continue to be sufficient to allow us to execute on our strategic priorities independent of the capital markets. Now I will turn the call back to Neil for his closing remarks.

Neil McFarlane, President and CEO, Zevra Therapeutics: Thank you, LeDuane. We are fortunate to be in a unique position with commercial therapies, late stage development program and a solid balance sheet to execute on our priorities. We attribute our success and momentum to staying true to our mission and the dedication of our team committed to making an impact on the lives of people living with rare diseases. Our focus moving forward remains firmly on executing across our four strategic pillars to accelerate our trajectory for transformative growth and long term value creation. Thank you.

We’ll now open the call for questions. Operator?

Conference Operator: C. Wainwright.

Brandon, Analyst, C. Wainwright: Hi, thanks for taking my questions and congratulations on all the progress. Maybe just starting in The U. S. And my the most new patient starts in 2Q or sort of just any or I guess, an enrollment. And any trend you’re starting to see in the new in The U.

S. In terms of enrollments, in terms of where they may be coming from?

Neil McFarlane, President and CEO, Zevra Therapeutics: Brandon, you cut out a little bit in the first part of your comment. It sounded like you were asking about U. S. MyPLAFA and any trends. But can I ask you to repeat the question?

Yes,

Brandon, Analyst, C. Wainwright: absolutely. Just any specialties contributing most to new patient enrollment in 2Q and since launch as well? Just any trends you see in The U. S. In terms of patient enrollments who weren’t in the EAP program?

Neil McFarlane, President and CEO, Zevra Therapeutics: I’ll ask Josh to comment on the specifics around specialties, but maybe I’ll take the opportunity to be able to talk a little bit about the strong performance in Q1 to Q2 overall. In Q1, we had MyPiper revenue of about $17,100,000 and a 26% growth from Q1 to Q2 with $21,500,000 And that’s really a lot to do with the pull through of patients from the patient enrollment forms on our patient services group and field reimbursement, walking through to help to drive patients through that funnel, through the reimbursement hurdles to get them onto commercial product. Tremendous job by the team in pulling that through. And if you look at our total enrollments from Q1 to Q2, we’re at 122 total enrollments and then ending Q2 at 129. That’s coming from a fairly small number, but it’s still a third of the patient population today that’s been diagnosed, the three hundred to three fifty.

I’ll pass it off to Joss a little bit to talk a little bit about the specialties and trends. But recall, these are small numbers in terms of the total number of patients in The U. S.

Josh Schafer, Chief Commercial Officer, Zevra Therapeutics: Yeah, thanks for the question. We’re seeing initially, as we expected, that the early patients were coming from those clinicians who were part of our largely neurologists and pediatricians. Now keep in mind that while many of these clinicians might have a have done training as a pediatrician, they are seeing both children and adult patients with NPC. We’re also seeing medical geneticists treating these patients. And we’re starting to see an emerging small group of psychiatrists who are seeing these patients as well.

So this is very consistent with what we expected, and we’re very pleased with what we’re seeing so far.

Brandon, Analyst, C. Wainwright: Great. Thanks very much. And then if I may just shift gears to Europe. You’ve obviously done a tremendous job in The U. S.

In terms of EAP conversion. Can you help us just think through the pushes and pulls of converting the EAP patients in Europe? Should you be approved there versus sort of what we saw in that sort of really quick and very good execution in The U. S?

Neil McFarlane, President and CEO, Zevra Therapeutics: Thanks, Brandon. So you’re absolutely right. If you’re taking note, we’ve gone from this time last year, maybe 70 to 80 patients in our what we call as our global EAP, which is in Europe primarily in a small number of markets. And each quarter, we continue to give you a number that’s a little higher. We ended with 89 patients at the end of Q2, and that’s from a small number of European markets that we have this expanded access program.

These patients, if we think about the durability and how long they’ve been on therapy and then the ability for us at a market by market level after approval, reimbursement in Europe is going to be by country. So it’s variable in regards to making that happen, but I do think our continued growth in the EAP program in Europe along with the durability of patients in the long that have been in the program for a long period of time bodes well for us to be able to pull those patients through once we get reimbursement in those countries.

Brandon, Analyst, C. Wainwright: Great. Thank you very much and congratulations on all of the progress.

Conference Operator: Thanks. We’ll go next to Kristen Kluska at Cantor.

Kristen Kluska, Analyst, Cantor: Hi, everyone. Congrats on another good quarter. First, I wanted to ask, you talked about the different patient segments out there, one of them being patients that are diagnosed but not on any therapies yet. Can you walk us through what that means? Are these newly diagnosed and they’re taking the steps?

Are they interested in therapies, hoping to learn more? And then I have a follow-up.

Neil McFarlane, President and CEO, Zevra Therapeutics: All right, Kristen. I’m going try and quarterback this year. But that one, I think, goes directly to Josh to be able to talk a little bit about our diagnosed patient population and how we plan to get the rest of those patients exposed to MyPolypa. And then while we’re continuing to unblock the newly diagnosed patients as well, which we’re starting to see as well. Scott?

Josh Schafer, Chief Commercial Officer, Zevra Therapeutics: Yes. So specifically, your question around those patients who are diagnosed but not receiving treatment, Those are typically patients who maybe were diagnosed a year ago prior to any therapies being on the market. And at that time, maybe their symptoms weren’t severe enough for them to receive any treatment. We’re finding that those patients are now coming back into the offices, either their local clinicians who are now being made aware of treatment options or going back to those COEs. And so we’re finding those patients now that we’ve been on the market for nine months or so, and we’re building that awareness and being able to bring those patients who had not previously received treatment to be eligible for MyPlifil.

Neil McFarlane, President and CEO, Zevra Therapeutics: Yeah. And Kristen, one of the things I might just follow-up on, Josh, and maybe from the prior question as well. When we think about these centers of excellence and we think about the local physicians who are working in concert with the diagnosing center of excellence. As we’ve talked about in the prepared remarks, we’ve mapped those physicians that are not in the actual centers of excellence. And our field organization is optimized to be able to go out and make sure that we can educate and offer MyPlypa to those physicians in the event that they had more than one, right?

A lot of these physicians have only ever seen one patient and only have one patient and rely on that center of excellence. Now that they’ve got experience with MyPlica, we have the opportunity to be able to make sure that we’re providing them with the disease state awareness campaign and everything else to know that if they see it once, maybe they can see it twice.

Kristen Kluska, Analyst, Cantor: Okay, thanks. And then can you provide us a sense of what percent of patients are on paid MiFlyfa? And then our check support that those patients that are on combination that reimbursement has gone well, but curious what you’re seeing from year end? Thanks so much again.

Neil McFarlane, President and CEO, Zevra Therapeutics: I might start that one off. Kristen, we give metrics that are around the prescription enrollment forms coming in the top that allows for us to show kind of the top of the funnel, if you will. And then we provide that covered lives. And we provide also the reimbursement sorry, the revenue number. When we talk about those patients that are on paid drug, you’ll see that you go from $17,100,000 in Q1 to $21,500,000 in Q2.

That’s a 26% increase quarter over quarter in paid drug. But going from 122 enrollment forms to 129, it’s obviously not 26%. So we’re hopeful that the guidance we’re giving you around revenue, which is what comes out of the bottom, we’ll be able to provide that takeaway on performance. Yeah. And I

Josh Schafer, Chief Commercial Officer, Zevra Therapeutics: would just add to that part of the question, Kristen, that our patient services team has done a phenomenal job of being able to convert enrollments to paid patients and make sure that these patients are able to receive monoclipal. Also importantly, making sure that they continue to get their refills. And so the retention rate of these patients is also really quite high. I think you had a second question, which was really around combination and what are we seeing in terms of patients receiving combination of therapy. Specifically from reimbursement, sorry.

Reimbursement, yeah. Well, would remind you that by label, patients who are receiving MyPlifer are also receiving miglustat. And so that by definition is a combination of those two therapies. We’re not seeing any pushback or very limited pushback from a reimbursement standpoint. As we noted in the prepared remarks, we currently have 52% coverage.

And for those who are not immediately covered, we’re able to get reimbursement through medical exception pathways.

Kristen Kluska, Analyst, Cantor: Thanks again.

Neil McFarlane, President and CEO, Zevra Therapeutics: Thanks, Kristen.

Conference Operator: We will go next to Jason Butler with Citizens.

Jason Butler, Analyst, Citizens: Thanks for taking the questions and congrats on the quarter. Two questions that I understand that you’re still early in the launch and you may need to run time to get complete clarity on. But can you do you have a sense now that you’re a couple of quarters in of what the average time is taking from enrollment form to getting a patient on reimbursed drug? And then in terms of retention, do you have enough data yet to speak to whether the retention rate for patients that were not in the EAP is consistent with the retention rate that you saw in the EAP? Thanks.

Neil McFarlane, President and CEO, Zevra Therapeutics: Yes. Jason, I appreciate the call. I’ll ask Josh to be able to talk a little bit about the time to from enrollment form to getting those patients through the system and then into commercial drug. It’s improving for sure. I’ll have Josh talk about that.

In regards to our retention rate of patients that are newly on product, we’re only in the second full quarter of launch. It’s really hard for us to be able to tell you about persistency rates and or adherence rates to prescriptions at this point, only the second quarter. Some patients have only received one commercial fill, others have received seven. So I think it’s a little early for us to go there. And usually, we need about six quarters to be able to actually get a true persistency and adherence rate.

So it’ll take a little bit more time for us to get to that. But one thing I can say is that of our EAP patients who converted to commercial product, they have been the majority of those patients have been consistent on product. And we’ve had limited, if any, of folks come off products. And I

Josh Schafer, Chief Commercial Officer, Zevra Therapeutics: would just add to that point that we’re really not seeing any differentiation from a payer perspective as to whether a patient was on the EAP or not. And so we’re looking at retention rates more holistically and haven’t seen any differences really materially between a patient who was enrolled in the EAP and those new patients who have come to MyPlaypha since then who were not on the EAP. So our retention remains quite high across all of those patients. In terms of the time to from enrollment to paid, again, varies widely based on the type of plan that a patient might be on, whether that’s commercial or Medicaid. And then within commercial, there’s a high degree of variability.

But what I can tell you is that that timeframe across all payers is being reduced dramatically. So much that in some instances, we’re seeing patients getting covered within seventy two hours. Now, not all of them, of course, but we are seeing some patients with very rapid conversion from enrollment to pay. And we continue to see that improve quarter over quarter.

Jason Butler, Analyst, Citizens: Great. And then just one more for me Olprova. Obviously, you’re thinking carefully about the strategy here. Can you speak to what the magnitude of investment in commercializing the product that doesn’t overlap directly with MyPlica? Just trying to get a sense of essentially how much you’re investing in the product and how you’re thinking about continuing that investment?

Neil McFarlane, President and CEO, Zevra Therapeutics: Thanks, Jason. So hopefully we made it really clear in our prepared remarks and I’ll reiterate it here. We are today got a commercial infrastructure that is actually running really well. When we talk about handling our enrollment forms as well as pulling those patients through our field reimbursement support, all the things that are working for us to be able to deliver this strong performance in Q2 for MyPlica, it’s synergistic with Oprove all the way. This question you’re asking around if we took out I think is what you’re asking, if we took out MyPlica out of this, what would it be costing you to be able to run Oprova?

And the reality is that it would be fairly fairly similar to what you see in our s g and a today because of the high level of overlap and our ability to be able to to truly execute on what our business plan is. Having these centers of excellence or so across the country that we can provide value across the chain from commercial to medical to disease state awareness and really help this rare disease community. So it’s hard for me to break it out. It’s very limited in regards to one or the other, but the synergies are huge for us. That provides us with a great opportunity to be able to do this really well, earn the right to go do it again someday.

Jason Butler, Analyst, Citizens: Great. Thank you. Thanks for taking the questions.

Conference Operator: We’ll now go next to Sumant Kulkarni with Concord.

Sumant Kulkarni, Analyst, Concord: Good afternoon. Thanks for taking my question. You said the seven additional patient enrollment forms and my profile takes you up to 129 in total, is in line with your expectations at the stage of the launch. How should we think about growth in patient enrollment forms going forward? And how do you think the availability of ACNURA, which is also approved for linenpiq type C is impacting patient enrollment?

Neil McFarlane, President and CEO, Zevra Therapeutics: Yes. Thanks, Sumant, and appreciate the question. And we’re looking forward to spending time with you at your conference tomorrow. You kind of set up the question nicely. When we think about three hundred to three fifty patients diagnosed in The U.

S, having one hundred and twenty nine of those patients enrolled into our program is a quite frankly, just I’m incredibly proud of this organization in delivering that. When we think about moving forward and the variability of enrollment on a quarter to quarter basis or a month to month basis, Unlocking this opportunity between the three hundred and three fifty to the 900 prevalent in The United States, we want to be more like Europe in this regard. A decade ago when meglustat was approved in Europe, there were a small number of patients who actually have been diagnosed. And with a prevalence of eleven hundred in Europe, a majority of those patients are now being have been diagnosed and on some type of treatment. So I’ll ask Josh to talk a little bit about some of the areas and things that he’s doing to be able to ensure that we can continue to drive the performance in The U.

S. But we’ve got a great comp in Europe that shows us that as we continue to invest, we can be much more like this mature market having a product approved for a while in Europe.

Josh Schafer, Chief Commercial Officer, Zevra Therapeutics: Yes. We’re really focusing on these two patient segments, those that are diagnosed and perhaps not yet receiving treatment. And that’s really around building awareness and reemphasizing the clinical differentiation of MyPlica and all the benefits that it brings in terms of it is the only drug that has demonstrated that it holds the progression of the disease at twelve months. We’ve been able to demonstrate at the first clinical time point, which was twelve weeks that we have an impact on disease. We’ve recently published new data around the mechanism of action, which really draws a strong connection between MyPlaza and its ability to affect the underlying pathology of the disease.

And then importantly, recent open label extension data that shows that the durability of that effect lasts up to five years. And we’re really emphasizing that data and using every opportunity that we can to reinforce that. For the undiagnosed patients, we have a number of different tactics to try and bring as many of those patients to diagnosis so that they can receive treatment as quickly as possible and ultimately halt the progression of disease. We have a disease awareness campaign read between the signs. Connected to that is a genetic testing capability to allow physicians to test those patients that they might have suspicion around whether or not they have NPC.

And then we’re using other really sort of sophisticated machine learning to be able to identify signs and symptoms of patients who have not yet been diagnosed based on a profile of an NPC patient, which will allow us to help continue to educate clinicians around how to identify these patients. So we’re really excited about those things. And as Neil mentioned, we see Europe as a model that can help us sort of determine or look forward to how we can see that diagnosis rate increase over time.

Neil McFarlane, President and CEO, Zevra Therapeutics: Yes. Sumay, maybe I’ll just add one more thing. I sorry, go ahead. Add another question.

Sumant Kulkarni, Analyst, Concord: Yes, did. On the pipeline, so you enrolled seven patients in the DISCOVER trial for saliprolol for vascular Ehlers Danlos syndrome. How happy are you with the pace of enrollment? And at what point will you be able to let us know when we might expect top line data on that?

Neil McFarlane, President and CEO, Zevra Therapeutics: Adrian is here with us. I’ll kind of kick it off and hand it off to him. When we think about the fact that it was at the end of Q3, early start of Q4 that we reestablished the enrollment of this trial and started to invest in the tactics to screen the outstanding patients that had been in the queue, but also some of the tactics we’ve been executing on that I’ll ask Adrian to talk more about, to actually get a higher quality COL3A1 genetically identified already patient that could then go into the trial and be and have the opportunity to be screened and then enrolled. I think we’re starting to see some traction here. But Adrian, why don’t you give an update here?

Adrian Cortell, Chief Medical Officer, Zevra Therapeutics: Yes, Smond. We started a genetic testing initiative a couple of months ago. We’ve also connected with most of the centers where those patients are actually seen and treated. And I mean the core Tier one positive SATS patients. And we’ve got a serious amount of physicians that are interested in referring the patients into the trial and significant patient interest.

It takes a bit of time to see that come to fruition because it takes quite a bit of time to get those patients to the screening process. So we’re very hopeful to report positive outcomes of that in the next quarter.

Neil McFarlane, President and CEO, Zevra Therapeutics: Suman, what I was going to say earlier on to your question, when we think about Niemantixi in The U. S, it sometimes goes in the face of what we’ve been looking at that we’re launching this product in MyPlayFib with the largest clinical data set of NPC patients that have ever been put together. In addition to that, we’re not just launching a product with twelve months’ worth of data, we’re now launching a product that now has got five years’ worth of clinical data reinforcing the durability of treatment effect in the open label extension study that was published in this last quarter. We’re now also being able to elucidate the mechanism of action work that we’ve talked about as well. And I think this is just the beginning.

We are in a very fortunate position. Usually, you’re not launching a product for the first time with five years of data. So when Josh is talking about the pull through, the work his team is doing on driving the awareness with payers with the strength of our data is what’s delivering the strong performance or part of what’s delivering the strong performance in what we’re doing. The ability for us to be able to have the MAA filed as a new MAA with this new very robust data sets, it puts us in a wonderful position to be able to bring MyPlifer to Europe and to the rest of the globe and take care of NPC patients outside of The United States. So we’re very fortunate that we’re able to be able to really jump on the back of a lot of folks that are out there and have been working in this disease area for a long time and now execute for patients with NPC.

Sumant Kulkarni, Analyst, Concord: Thanks. I have a lot more questions for you tomorrow.

Brandon, Analyst, C. Wainwright: We’ll move

Conference Operator: next to Sami Corwin with William Blair.

Nicole Osher, VP of Investor Relations and Corporate Communications, Zevra Therapeutics: Congrats on the progress this quarter and thanks for taking our questions. I was curious how many unique prescribers there are now and how that’s run over the last quarter and if you are approaching prescriber saturation? And then you expanded the MyPipe the lives significantly compared to last quarter. How much further do you think you can expand the extent of covered lives? And are you aware of any policies that are still being finalized?

Thank you.

Neil McFarlane, President and CEO, Zevra Therapeutics: Thanks, Sami. I will actually pass both of those over to Josh, the unique prescribers as well as if there’s any prescriber fatigue, which definitely there is not.

Josh Schafer, Chief Commercial Officer, Zevra Therapeutics: Yes. So we have seen a pretty significant growth in the number of prescribers. When we first launched and we were dealing with the EAP patients, those were patients who were under the care of just a handful of EAP investigators. And you might have seen clinicians with anywhere from 12 to eight patients. Now as we move past that into this next cohort of patients, we’re seeing clinicians who are treating three, four, maybe one or two patients.

And so naturally, the number of prescribers, unique prescribers has grown pretty significantly. And we expect that to continue as this really is an ultra rare disease where now we’re getting into those clinicians who are seeing one or two patients over the course of a year. And so that prescriber base will continue to grow. And we expect that the number of lives and patients will grow as well. I think you asked the question around reimbursement and covered lives.

And as noted, we’re at fifty two percent today, which is very consistent with what we’d expect for this stage of a launch. And that’s really more a reflection of plans who just haven’t yet put MyPlayFe in front of their P and T committee and some plans wait anywhere from nine to twelve months after product has been launched before they make those decisions. And so we expect that number to increase over the course of our launch. And a good analog is looking at Elprovo, which has been out on the market for a little bit longer in a more mature market. And as we noted, that’s at 79%.

So you could see us absolutely growing from the 52 of covered lives to where we are today.

Conference Operator: That’s very helpful. Thank you. We’ll go next to Eddie Hickman with Guggenheim Securities.

Nicole Osher, VP of Investor Relations and Corporate Communications, Zevra Therapeutics0: Hi, good afternoon, everyone. Thanks for taking my questions. Congrats on all the progress. I wanted to follow-up on a previous question just to sort of double click on this penetration. If we think specifically about the sort of two thirds of diagnosed patients that you know have MPC and that you’ve yet to penetrate, should we expect sort of a similar cadence of new patient enrollment forms throughout the next couple of quarters?

Or do you reach a point where it starts to get sort of difficult to find those patients? This is sort of beyond finding new patients, but just within those sort of known diagnosed patients. And then just sort of just a logistical question, can patients start meglustat and MyPlyFa at the same time or the payers ever asked for or doctors ever asked for a delay in getting a patient sort of stably on MyPlisa before trying to get coverage for MyPlisa? Appreciate it.

Neil McFarlane, President and CEO, Zevra Therapeutics: Yes. Thanks, Eddie. I think those questions I’ll hand over to Josh to handle.

Josh Schafer, Chief Commercial Officer, Zevra Therapeutics: Yes. So patients can absolutely start megastat and Myclyfa at the same time. And we’ve seen some new patients new to therapy starting both at the same time. So that is absolutely possible. Your other question was really around the cadence of enrollments as what do we expect in future quarters.

I would just have to say it’s really early in the launch to be able to give you any sense of that and be able to give you a sense of trends. I think as you look at where we are with enrollments, that is a good reflection of the demand that we’re seeing. I think more importantly, looking at the performance from a net sales perspective of 26% increase really speaks to our ability to convert those patients who have come in and be able to convert them to paid and then retain those patients. And then as we really begin to pull through the initiatives that we’ve been talking about with new patient identification, building awareness around clinical differentiation, we expect the enrollments to come in over the next couple of quarters. But again, probably too early to give you any sense of trends at this point.

Got it. Thank

Conference Operator: And it does not appear we have any other questions holding. This concludes the Q and A portion of today’s call.

Nicole Osher, VP of Investor Relations and Corporate Communications, Zevra Therapeutics0: I will now turn the

Conference Operator: program back to our presenters for any additional or closing remarks.

Neil McFarlane, President and CEO, Zevra Therapeutics: Yes. Thank you, operator, and thanks, everybody, for joining the call today. We look forward to keeping you appraised on future progress. Have a great week.

Conference Operator: Thank you. This does conclude today’s program. Thank you for your participation. You may disconnect at any time.

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