Zevra Therapeutics Q1 2025 Earnings Call Transcript

Quartr
Provided by Quartr
May 13, 2025
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Operator

Good afternoon and thank you for joining Zebra's First 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 Ochsner, SEBRA's Vice President. Please go ahead.

Nichol Ochsner
Nichol Ochsner
Vice President of Investor Relations & Corporate Communications at 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 first quarter financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the Investors 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 Sevres business.

Nichol Ochsner
Nichol Ochsner
Vice President of Investor Relations & Corporate Communications at Zevra Therapeutics

Forward looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other important factors that may lead actual results differing materially from projections made. These forward looking statements should be evaluated together with the cautionary statements contained in the Risk Factors section in our most recently quarterly report on Form 10 Q, annual report on Form 10 ks and our other filings with the SEC. I am pleased to welcome Zevra's management team members participating in today's call. Neil McFarlane, Zevra's President and Chief Executive Officer LeDuane Clifton, our Chief Financial Officer and Josh Schafer, our Chief Commercial Officer. Our Chief Medical Officer, Adrian Cortell, will also be available for today's question and answer session.

Nichol Ochsner
Nichol Ochsner
Vice President of Investor Relations & Corporate Communications at Zevra Therapeutics

Now it's my pleasure to turn the call over to Neil.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Thank you, Nicole, and thank you for joining us this afternoon to review our strong progress and the ongoing execution of our four strategic pillars. Let me start with commercial excellence. We are delivering on our two commercial launches to bring innovative therapies to people living with rare diseases. We have exceeded our internal expectations for the MyPIPA launch and have enrolled over one third of those diagnosed with Neiman Pick disease type C or NPC in The United States. With Olpruva for this treatment of certain urea cycle disorders or UCDs, we refined our marketing efforts to further increase awareness and access for patients and providers.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Moving on to pipeline and innovation. We are focused on where our capabilities have the greatest impact for patients as we progress our development programs. Our Phase III DISCOVER trial of soliprolol, our late stage asset for the potential treatment of vascular Ehlers Danlos syndrome or VEDS, which has received both orphan drug and breakthrough therapy designations, continues to progress with patient enrollments. Additionally, we continue assessing value enhancing strategic alternatives for our Phase III ready asset KP1077 for rare sleep disorders. These clinical stage assets provide optionality and diversification to our portfolio.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Turning to talent and culture. We have a passionate, highly motivated, experienced and mission driven team focused on our vision to bring life changing therapies to people living with rare diseases. Our team's strong execution is being acknowledged. For example, we earned a spot on Fast Company's top 10 most innovative companies list in the medicines, therapeutics and pharmaceuticals category. Finally, corporate foundation.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

This underpins all other pillars and allows us to responsibly invest in our long term transformation through disciplined capital allocation while maintaining our position of financial strength. With the recent monetization of our Pediatric Rare Disease Priority Review Voucher or PRV, we added $148,300,000 of non dilutive capital to our balance sheet, bolstering our financial strength. With this, we have ample resources and financial flexibility to execute on our strategic priorities independent from the capital markets. Driving a little deeper into our commercial programs, we are pleased with the early success in the MyPlatform launch for the treatment of NPC, which is an ultra rare neurodegenerative progressive and fatal disease. The lung cancer the cohorts, those who are participants of our Expanded Access Program or EAP and may or may not be receiving treatment.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

And finally, those who are undiagnosed and living with NPC for whom we seek to accelerate diagnosis and treatment. We received a total of 122 prescription enrollment forms from launch through March 31, with 13 in the first quarter of twenty twenty five. Even in these early days of the launch, we have received prescription enrollment forms from all three patient cohorts affirming the early impact of our commercial strategy. Notably, as we have previously reported, all active U. S.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

EAP participants have been enrolled to receive MyPlifer. And we have now closed the program, including locking the database and deactivating study centers ahead of our previously stated guidance. We are focused on providing Mycplifa to people living with NPC and have been educating prescribers and payers on the clinical benefit, which demonstrates in combination with miglustat a halting of disease progression through twelve months of treatment. Beyond the pivotal trial, Mypipa safety profile and the long term treatment effect have been evaluated in open label extension studies and through our multicenter EAP with patients having as many as five to seven years of experience on treatment, which significantly differentiates MyPlipa and positions it as the cornerstone therapy. Josh will provide additional details related to our launch metrics later in today's call.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Beyond the successful U. S. Launch, our goal is to provide access to as many people living with NPC as possible by expanding the availability of MyPlipa outside of The U. S. We are diligently working towards expanding our regulatory submissions with an immediate focus on our marketing authorization application or MAA in Europe in the second half of this year, where we estimate approximately eleven hundred people are living with NPC.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

As a reminder, the European market is already well established with access to a marketed treatment for more than a decade. Further, we're expanding our global EAP, which has recently increased to approximately eighty five NPC patients compared to the previously reported seventy to eighty patients. Our continued investment in MyPlifer's global EAP in combination with a more established market could accelerate commercialization upon approval in Europe. Turning to Oprova. From initial product availability in July of twenty twenty three and including ZEVRIS promotion of OLPUVA in late January of twenty twenty four, through the end of the first quarter twenty twenty five, we've received a total of 28 prescription enrollment forms with five prescription enrollment forms during the first quarter of this year.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

In November, we outlined a plan to refine our commercial strategy towards the adult and OTC deficient populations for whom OLPURVIS portability and ease of administration may provide the greatest benefit. The enrollments received in the first quarter were all within this patient segment, suggesting early signs that this strategy is gaining traction. Before turning the call over to Josh, I'll share a few more updates on our clinical stage assets. Through our Phase III DISCOVER trial, saliprolol is being evaluated as a potential treatment of VEDS, a devastating connective tissue disorder caused by the COL3A1 gene mutation and characterized by spontaneous arterial aneurysms, hollow organ ruptures and aortic dissections. In The U.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

S, approximately seven thousand five hundred people are living with VEDS, where the standard of care relies on reactive and invasive surgical interventions with no approved treatments. Talipolol is also currently used off label as a standard of care for vets in certain European countries. During the first quarter, we enrolled five additional subjects in the DISCOVER trial, bringing the total to thirty two. This is an event driven trial and the rate of enrollment is important. And we're implementing various strategies, including increasing our efforts to focus on providers and clinics specializing in genetically confirmed COL3A1 positive patients.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

During the first quarter, we began receiving leads from a recently initiated genetic testing program, providing our team with a new group of confirmed VEDS patients for outreach. Importantly, we are optimistic in this strategy to drive enrollment and expect to see an increase in the enrollment rate in the upcoming quarters. There are also several updates related to the review of our extensive IP portfolio as we seek to extract value by determining where we continue to invest as well as where we choose to stop investing based on our strategic plan. I'm pleased to report that earlier this month, we've out licensed intellectual property related to a preclinical prodrug for an undisclosed party for a nominal upfront payment, potential future milestones and mid single digit royalties on net sales. We will continue to optimize and curate our IP portfolio through a variety of avenues to realize value for the company and our shareholders.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Additionally, regarding Apodas, which was previously approved for the treatment of pain and has not been commercially available since 2023, we have made a strategic decision to formally withdraw the NDA. By eliminating the regulatory activities associated with maintaining approval, we are further reducing costs. We are continuing ZEVRA's transformation into a patient centric commercial stage rare disease therapeutics company. With our bolstered cash balance, we are well positioned to support our stated strategic priorities and execute on our long term vision for the company by establishing Mypiper as a cornerstone treatment for NPD globally, maximizing our commercial opportunity with Olpruva and supporting our growth through our development pipeline, including the ongoing Phase III trial for salipolol. Continue to build on our track record of execution, positioning us to make future investments that are in alignment with our strategic plan.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Let me turn the call over to Josh, who will give an update on our commercial products.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

Thank you, Neil, and good afternoon. Today, while discussing our commercial products, we will provide the following metrics: prescription enrollment forms for the quarter, the percent of covered lives and net revenue. Beginning with MyPlifa, as Neil stated, the launch is progressing well and exceeding our expectations. With our EAP cohort fully enrolled, we are focused on reaching the diagnosed and undiagnosed cohorts. We've seen steady growth in the first quarter with 13 prescription enrollment forms, all representing individuals new to MyPlayfa, which indicates a broadening of both the patient and prescriber base.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

This is a result of our ongoing efforts to raise awareness of NPC and to educate prescribers about MyPlaypha's clinically differentiated profile. We estimate that there are approximately nine hundred people living with NPC in The U. S, of which only three hundred to three fifty have been diagnosed. Since launch through March 31, we have received a total of 122 prescription enrollment forms, meaning that roughly one third of the estimated individuals diagnosed with NPC in The U. S.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

Have been enrolled to receive MyPlifa. As a reminder, a prescription enrollment form is a prescription submitted to our specialty pharmacy initiating the benefits investigation process to determine reimbursement and can lead to a thirty day paid dispense of MyPlayfa. Regarding market access, many payers have not yet formalized their formulary coverage or reimbursement policies, and our team has been actively engaging with payers to secure reimbursement. Through the end of the first quarter, we have achieved 38% of covered lives, which is in line with our expectations at this stage of launch. We have been able to secure reimbursement authorization for many of our patients through direct formulary coverage or via the medical exception process.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

While initial denials are commonplace among rare disease products, we are very pleased with our team's ability to swiftly address payer challenges by presenting MyPlife as robust and differentiated clinical data. NPC is a lysosomal storage disorder that is caused by progressive lipid buildup leading to cell death and ultimately organ dysfunction in the spleen, liver and brain. The difficulty in diagnosing NPC patients centers around variability in age of disease onset and the heterogeneity of symptoms. As a result, the disease progression is measured by the only clinically validated tool, the four domain NPC Clinical Severity Scale, which evaluates four key domains deemed by NPC experts to be the most important, including ambulation, fine motor skills, speech and importantly, swallow abilities. MyPlifa is the only product approved by the FDA based on the NPC Clinical Severity Scale.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

And the data in our label demonstrate that MyPlifa in combination with miglustat halts disease progression through twelve months of treatment, as shown by a greater than two point improvement in patients receiving MyPlifa and miglustat compared to those receiving miglustat alone. It is important to note that only a one point improvement is needed to demonstrate a clinically meaningful difference with a well tolerated safety profile. Additionally, MyPllyfa is the only FDA approved product for NPC with more than five years of clinical data in its label and with more than two seventy NPC patients who have participated in our pivotal trial, our open label extension study or our EAP. In our view, these data, in addition to its well tolerated safety profile, establishes MyPlifa as the cornerstone of therapy for NPC. Building on our strong body of evidence, we recently issued a publication discussing mechanistic insights into mediated effects on lysosome function and NPC in the Journal of Molecular Genetics and Metabolism.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

The elucidation of differentiated mechanism of action marks a critical step in understanding its interactions with NPC at a cellular level. These insights substantiate how MyPlifer addresses the underlying pathology of MPC and supports the long term benefit observed in our clinical trials. During our fourth quarter call, we unveiled numerous initiatives to reach the MPC patient cohorts, namely those who are diagnosed and may or may not be receiving treatment as well as the undiagnosed population. We continue to analyze claims data to identify existing patients, and we are employing targeting techniques to identify new and undiagnosed patients based on related symptoms and conditions. In addition, our targeted media campaign to build awareness and educate about early signs and symptoms of NPC is proving to be successful.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

We've expanded to a national scale reaching our team is amplifying the reach of these new segments through branded geo targeting efforts in the corresponding regions. And these new segments are increasing awareness of NPC and the availability of treatment options and are primarily designed to resonate with individuals who have been diagnosed, but are not yet receiving treatment. We also launched our disease state awareness campaign, Learn NPC Read Between the Signs on rare disease day this past February, and it's driving disease recognition and early diagnosis. Treatment of NPC is multifaceted, and our program provides education and genetic testing options for individuals with suspected lysosomal storage disorders. We have already seen the impact of this initiative with new patients who were not previously diagnosed with NPC being identified, enabling us to facilitate earlier diagnosis and offer MyPlifa as a treatment option.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

In summary, we are encouraged by the early results of our efforts to engage the diverse patient cohorts, and we're looking forward to sharing our ongoing progress and future success with the program. Now let's turn to Alprovo, our commercial product for the treatment of certain UCDs. UCDs are a group of rare inherited metabolic disorders resulting from a defect in one of the six enzymes or two transporters in the urea cycle causing an accumulation of ammonia known as hyperanalemia, which can be toxic and lead to neurocognitive damage or even death. As we've discussed in prior quarters, we have moderated our expectations for the pace of the launch, given the unique dynamics of the UCD commercial landscape. From initial product availability in July of twenty twenty three and including Zevra's promotion of OLPUVA from January 2024 through the end of this first quarter, '20 '8 total prescription enrollment forms for OLPUVA have been received, including five in this first quarter of twenty twenty five.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

In the case of Alpruva, our prescription enrollment forms may also include our thirty day free trial program. As Neil highlighted, we have refined our marketing strategy to more specifically target the adult patients seeking more independence as well as the OTC deficient and carrier population who may receive greater benefit from the portability and palatability offered by Iopruva while encountering fewer reimbursement obstacles. All enrollment forms in the first quarter align with our targeted patient segment, suggesting that this positioning resonates with prescribers. Given that patients with this disease typically only see their physician one to two times a year, we anticipate that the impact of our refined marketing strategy will require additional quarters to gain further traction. For both our commercial products, we remain committed to patient access and focused on delivering comprehensive patient services through our in house program Amplify Assist.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

Our field case managers who support both MyPlayfa and OLPUVA are demonstrating early impact through their ability to assist with reimbursement hurdles. And currently, our market access team continues to engage with payers to identify opportunities for Alpruva to improve its formulary position. We maintain regular communication with payers and have increased covered lives to 78%. I look forward to sharing additional updates in the coming quarters. I will now pass the call to Ludwain, who will present the financial results for the first quarter of twenty twenty five.

R. LaDuane Clifton
R. LaDuane Clifton
CFO, Secretary & Treasurer at 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 Zevro's quarterly report on Form 10 Q for more detailed information, which we intend to file shortly. In the first quarter of twenty twenty five, we reported net revenue of $20,400,000 comprised of 17,100,000.0 from MyPlayfa revenue, dollars 100,000.0 from Opruva revenue, dollars 2,300,000.0 in net reimbursements from the French EAP for Aramcholamol and 0.9 from royalties and other reimbursements under the Astaris license. For our commercial products, Myplifa and Olpruva, we recognize revenue when shipments are received by the specialty pharmacy. Our operating expense for the first quarter was $22,800,000 which was a decrease of $600,000 compared to the same quarter a year ago.

R. LaDuane Clifton
R. LaDuane Clifton
CFO, Secretary & Treasurer at Zevra Therapeutics

R and D expenses were 3,300,000.0 for Q1 twenty twenty five, which was a decrease of 9,000,000 compared to Q1 twenty twenty four, due primarily to a decrease in third party cost upon completion of the KP1077 Phase II trial combined with a decrease in personnel related costs. SG and A expenses were 19,500,000.0 for Q1 twenty twenty five, which was an increase of $9,600,000 Period over period increase in SG and A expenses was primarily related to an increase in personnel related costs, professional fees and other expenses associated with our commercial, medical and launch activities. Net loss for the first quarter of twenty twenty five was $3,100,000 or $06 per basic and diluted share compared to $16,600,000 or $0.40 per basic and diluted share for the same quarter a year ago. As of 03/31/2025, total cash, cash equivalents and investments were $68,700,000 which was a decrease of $6,800,000 compared to 12/31/2024. Combined with the net proceeds of 148,300,000.0 from the sale of the PRV received just after the end of the quarter on April 1, cash, cash equivalents and investments would be $217,000,000 Total debt was approximately 60,000,000 As mentioned earlier, adding the non dilutive capital from the PRV sale has further enhanced our financial flexibility to support our strategic priorities, which includes executing on the commercial launches of Myplifa and Opruva and supporting our ongoing Phase three trial for saliprolol.

R. LaDuane Clifton
R. LaDuane Clifton
CFO, Secretary & Treasurer at Zevra Therapeutics

We currently have ample resources to execute on our strategic priorities independent from the capital markets. Our financial results for Q1 twenty twenty five reflect the solid momentum in building a leading rare disease therapeutics company. And we are pleased with the opportunities we have in 2025 and beyond to drive value creation through disciplined investments where we can win. Now I'll turn the call back to Neil for his closing remarks.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

2025 is off to a terrific start for Zebra. The launch of MyPlifa is exceeding expectations. Our course corrections and marketing strategy for OLPUVA are gaining traction, and we're advancing our development pipeline. Today, driven by our sustained execution, we believe we're well positioned for future growth and see Zevra as a promising commercial stage rare disease therapeutics company with a clear plan to drive shareholder value. Our vision for the future is bright and we have many opportunities to serve the needs of patients and caregivers as we execute on our mission.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Thank you for joining the call and we'll now open the call for questions. Operator?

Operator

Thank you. We will go first to Sumant Kulkarni at Canaccord.

Sumant Kulkarni
Managing Director at Canaccord Genuity Inc

Good afternoon. It's nice to see the progress and thanks for taking our questions. I have a couple. So first, relative to the 122 patient enrollment forms, could you give us any details on the number of patients that are actually on MyPlaza right now and what the split of reimbursed patients might be?

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Thanks, Sumat. And just before I answer that question, I want to let everybody know that we're taking the call from multiple locations. So that may necessitate a slight delay in responding as we get the folks who are going to be answering the question. I'll hand that question directly off to Josh in regards to the strong performance we've seen with MyPlipa so far.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

Yes. Thanks, Sumant. So as noted, we have seen 122 or received 122 enrollments since the launch through the end of the first quarter. We do not report on how many of those are actively on drug, but I can say that the majority of them are on drug. And those who are not yet are going through a process of benefits investigation and are in some stage of pending.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

Some might be receiving what we call quick start, which is free drug while we investigate the benefits and then intend to convert those patients to paid. And then there may be others that are just in some various stage of benefits investigation.

Sumant Kulkarni
Managing Director at Canaccord Genuity Inc

Got it. And one more before I hop into the queue. You mentioned denials of reimbursement at early stages in rare disease launches start to be expected. That's understandable. But could you give us any sense of what the top reason might be for the denial of MyPlaza?

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

Yes, absolutely. And those are two different data points. The 38% of covered lives is looking at the total number of out there excuse me, the total number of people out there with insurance. 38% of those total covered lives who are receiving commercial insurance, federal insurance, VA are covered currently.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

And then

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

any challenges that we're finding right now, we do we haven't seen some initial denials, and that's totally to be expected with the rare disease product. And that is typical of products that have to go through some sort of prior authorization before they can get approved. We've been able to get coverage for almost all of our patients either through direct formulary either direct formulary status or through some sort of medical exception process, which might require a physician writing a letter of medical necessity. But we've had great success in getting that covered. And it's largely due to the demonstrated differentiation MyPlifer has with it being the only drug that's demonstrated the halting of disease progression through twelve months.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

It's got five to seven years of new patients treated. And we have the only clinically validated endpoint for NPC, which is the Neiman Tick clinical severity scale.

Sumant Kulkarni
Managing Director at Canaccord Genuity Inc

Thank you.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Sumay, maybe I'll just I'll bring to the forefront. Josh laid it out beautifully. Our differentiation in the clinical profile is what's really driving the strong performance. The feedback from physicians and so far how we've been able to address the market access challenges that come up with every product that's in the specialty space is really demonstrating the strength of the data. That's really what's driving a lot of our performance today.

Sumant Kulkarni
Managing Director at Canaccord Genuity Inc

Thanks.

Operator

We'll go next to Jason Butler with Citizens.

Jason Butler
Managing Director & Biotechnology Equity Research at Citizens JMP

Hi, thanks for taking the questions and congrats on the quarter. A couple for me on MyPlay. First of all, how much visibility do you have into whether patients are coming from that undiagnosed bucket versus diagnosed, but not on a therapy right now? And just how should we think about bringing those patients on board, the cadence you can bring those patients on board throughout the year? And then second question, just qualitatively, can you speak to how inventory levels ended the quarter versus the end

Jason Butler
Managing Director & Biotechnology Equity Research at Citizens JMP

Thank you.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Jason, thanks for the question. I'll try to quarterback this a little bit. I'll ask Josh to talk a little bit specifically in regards to the three cohorts that we talked about and the visibility. And then I'll ask him to hand it off to LaDuane, which again may take a quick second to talk about inventory levels and our targets and where we are today.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

Jason, as we've mentioned, there are three distinct cohorts that we think about. The total prevalence for NPC is roughly nine hundred patients, six hundred of which or so that are undiagnosed living with the disease and don't even yet know And there are about three hundred to three fifty that are diagnosed and either receiving some sort of treatment or just sort of waiting and watching. Of that subset, we had eighty three patients who were in our expanded access program. And those patients have all been enrolled to receive MyPlypha. We reported at the end of the fourth quarter '1 hundred and '9 patients, and that included those EAP patients plus a combination of diagnosed and undiagnosed.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

We continue to see a number of the second the two cohorts, the diagnosed and undiagnosed patients coming in as a direct response to the initiatives that we've rolled out to bring more awareness to the disease and also to MyPlayFe as a treatment option.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

So Blaine, if you can help Yes.

R. LaDuane Clifton
R. LaDuane Clifton
CFO, Secretary & Treasurer at Zevra Therapeutics

Sure. Thank you. And so Jason, with regard to inventory, these are the early days of the launch and we seek to support patients and remain very nimble. So we're managing that closely using a target days on hand. When we reported out in Q4, we explained that we had reached it and stocked at that target level, and we are maintaining that as we come into the end of Q1 as well.

Jason Butler
Managing Director & Biotechnology Equity Research at Citizens JMP

Great. Thanks for taking the questions.

Operator

We'll go next to Eddie Hickman with Guggenheim.

Eddie Hickman
Vice President at Guggenheim Partners

Hey, good afternoon. Thanks for taking my questions and congrats on this progress so far. Any qualitative metrics on refill rates or patient retention or average net price that you can give us that might be helpful for modeling the durability and treatment effects or trajectory going into the second half of the year? Should we expect this cadence of de novo patients of maybe around three to four per month to continue throughout 2025? And what are the challenges in continuing to penetrate those non EAP patients?

Eddie Hickman
Vice President at Guggenheim Partners

Thanks.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

Yes. So I can give you a little bit of color to say that are those patients that have come in and for whom we've received enrollments and who are receiving active drug, the vast majority of them have received refills and are continuing to stay on drug. Certainly, as we continue through the launch, we would expect to see that maybe diminish a little bit. But it really if you reflect back to our EAP, we had patients who stayed on drug for as long as five to seven years. And so that really speaks to the clinical benefit of MyPlifer as well as the durability of response.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

And we would expect to see that commercially as well. Your other questions were around net price. And again, what we're seeing in commercial is something very similar to the distribution of doses that we saw in the EAP with the vast majority of patients receiving one or two of the higher doses. So that can give you kind of a sense of what the WAC price is and give you a sense of how that's trending as well.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Yes. Eddie, before maybe you have a follow-up question here. But before we move forward, when we actually launched the program back in September, we talked about the fact that we were utilizing the average dosing of our EAP patients, which the clinical trial leaned more to younger lighter weight patients. And then our expanded access program really brought more adult patients in too. So what our real world experience has been so far and the average pricing that we had reported out based on the weight distribution of our 83 patients has really kind of continued the same.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

So that guidance that we provided is guidance that I would continue to have you move forward with.

Eddie Hickman
Vice President at Guggenheim Partners

Thanks. And then I just was curious sort of about the cadence of those new patient starts throughout the year. Should we continue to sort of think about 12 to 13 per quarter or will that diminish or grow throughout the year?

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Thanks again for the question. I think it's difficult for us and we're not going to provide guidance. We're going to continue to be disciplined in what we do around the metrics that Josh talked about in new patient enrollment forms as well as market access and then revenue to be able to pull through. What I would say to you though is that as we went through the three cohorts and accelerating the expanded access programs and continuing to get them really rapidly enrolled, which we had originally think would take us twelve months. We got it done in just a quarter and a little bit.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

We're now in the process of getting to these two other cohorts, the diagnosed patients who have not been on therapy as well as those patients who are not yet been diagnosed. We've seen all three of those cohorts in Q1, and we expect that we'll continue to see all three of those cohorts well, no, not the EAP, those are done. But the other two cohorts, those patients who are diagnosed and undiagnosed continue as the strength of the product and the benefits lead to getting patients on therapy. So I can't give you guidance in terms of where the cadence is going to be, but I can tell you that the strong performance today is based on our data.

Eddie Hickman
Vice President at Guggenheim Partners

Got it, right. Thank you so much.

Operator

We will go next to Lachlan Hanbury Brown with William Blair.

Lachlan Hanbury-Brown
Biotech Equity Research Analyst at William Blair

Hey, guys. Thanks for taking the questions and congrats on the progress. I wanted to ask on the coverage. You said thirty eight percent of lives are covered. Can you just elaborate on what that coverage looks like?

Lachlan Hanbury-Brown
Biotech Equity Research Analyst at William Blair

What the requirements are? Did you contract for that? And maybe how much of an impact does having that coverage make in terms of the time to get a patient from sort of prescription enrollment form to actually on drug because I assume it still requires a prior authorization? And then if I could just clarify as well, on the EAP patients, you said that they'd all been enrolled. Just to clarify, is that are they all receiving drug through the commercial channel now?

Lachlan Hanbury-Brown
Biotech Equity Research Analyst at William Blair

Or are some of them still sort of in the benefits investigation stage?

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

Great. Well, I'll take your first question around what the 38% of covered lives means. So again, most commercial plans and federal plans will evaluate a drug based on its clinical merits. Some plans will not by policy evaluate a product until it's been on the market for six months and some it might be as long as twelve months. And so the 38% of covered lives means that those plans, roughly 75 to 100 plans out there covering several hundreds of millions of lives, have reviewed MyPlisa and have made it available to their patients.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

And that represents about thirty eight percent of the total lives out there that have insurance. That does not mean that only thirty eight percent of our patients will receive drug or be able to get drug reimbursed. That just means that there's a direct route through a formulary status or position. All plans have a medical exception pathway, and we've been very successful in getting our patients reimbursed through a medical exception pathway. We expect that thirty eight percent to increase as more plans evaluate MyPlaza going forward.

Joshua Schafer
Joshua Schafer
CCO at Zevra Therapeutics

And we will continue to see that number increase and we will continue to have patients reimbursed either through direct formulary status or through the medical exception pathway.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Just a follow-up on an answer that on your question, you asked if all of the patient enrollment forms have been put on to commercial product. And that's a process that we work this medical exception pathway as well as others to get patients through and convert them from an enrollment form to a paid prescription, if you will, or commercial product. That timeline is not immediate. Some of them are within twenty four hours, they can get through the process. And others take time to work through this peer to peer medical exception, so on and so forth, process of getting specialty pharmacy products through.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

What I'm very proud of is the team has actually worked diligently to be able to ensure that when a prescription form comes in, we do everything that we can to make sure that patient and their family receive the product as soon as possible while we're going through the benefits investigation process. And they're actually rapidly working through this really large number, again, 122 enrollment forms, a dramatic number of forms and really strong performance. And getting through that process, some are going to take thirty days, some are going to take twenty four hours, some are going to take thirty days, some are going to take much longer than that. So I would say that right now, our team is doing a great job and you see that based on the revenue numbers that we've got in just our first full quarter post launch of converting those patients.

Lachlan Hanbury-Brown
Biotech Equity Research Analyst at William Blair

Thanks.

Operator

We'll return now to Simant Kulkarni at Canaccord.

Sumant Kulkarni
Managing Director at Canaccord Genuity Inc

Thanks for the follow-up. This is on celioprolol, which seems to be flying under the radar a little bit. You said you have 32 patients enrolled. It's an event driven trial where you're increasing efforts to drive enrollment. If I remember right, this is a 150 patient trial.

Sumant Kulkarni
Managing Director at Canaccord Genuity Inc

It looks like the last update on clinicaltrials.gov came about a year ago. Given that the Phase III trial for a competitor, enzostatin, has been suspended for some time now, what does that mean in terms of your ability to give us a timeline on when enrollment might be complete?

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

So your numbers are absolutely accurate, Sumant. You talked about N of one hundred and fifty patients in the two:one randomization. I think it's important to just take a quick step back. We inherited this trial after it actually paused enrollment for some time with a large number, as we've said publicly previously, of screenings that were ready to go into the trial. We kicked that off in Q3 with our first patients being dosed re enrolled and re dosed in the trial.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

And then we've been actually investing in getting through the screening of the previous patients, but also now targeting how we're going to be able to drive future enrollment. We talked about our ability to go to COL3A1 patient centers and physicians to be able to find more readily diagnosed patients who are more keen to be able to go into the trial. And also talked about some genetic testing that we're also doing as part of our screening. So we get more patients who are already diagnosed have the genetic defect and we can then offer them the opportunity to be in the trial. Those activities we feel very confident will continue to drive and now accelerate enrollment of the trial.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

But it's really only been two quarters since we got the trial up and going. The learnings as we've walked through and now we've got these five additional patients, 32 patients enrolled to date. And there's a high unmet need and no approved therapy as you know, but we're working through with these clinics and testing centers now to really ramp it. So in a nutshell, I can't tell you exactly when we will finalize it, but the strategies are starting to pay off in terms of our commitment to moving forward.

Sumant Kulkarni
Managing Director at Canaccord Genuity Inc

Got it.

Sumant Kulkarni
Managing Director at Canaccord Genuity Inc

Thanks.

Operator

And that will conclude the Q and A session. I will now turn the program back over to Neil McFarlane for any additional or closing remarks.

Neil McFarlane
Neil McFarlane
President & CEO at Zevra Therapeutics

Thanks, operator, and thanks, everybody, for joining our call today. We look forward to keeping you appraised of future progress and look forward and look to see you in Q2.

Operator

Thank you, sir. This does conclude today's program. We thank you for your participation. You may disconnect at any time.

Executives
    • Nichol Ochsner
      Nichol Ochsner
      Vice President of Investor Relations & Corporate Communications
    • Neil McFarlane
      Neil McFarlane
      President & CEO
    • Joshua Schafer
      Joshua Schafer
      CCO
    • R. LaDuane Clifton
      R. LaDuane Clifton
      CFO, Secretary & Treasurer
Analysts
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Key Takeaways

  • Strong MyPlifa launch with 122 prescription enrollment forms through March 31, enrolling over one third of diagnosed NPC patients in the U.S. and drawing from all three targeted cohorts.
  • Refined Olpruva commercial strategy toward adult and OTC‐deficient UCD patients, with all five Q1 prescription enrollment forms coming from this focused segment.
  • Phase III DISCOVER trial for soliprolol in VEDS reached 32 enrolled subjects, backed by new genetic testing programs and specialist outreach to accelerate the event‐driven enrollment.
  • Q1 2025 net revenue was $20.4 million (MyPlifa $17.1 M, Olpruva $0.1 M), operating expenses of $22.8 M, and net loss of $3.1 M versus $16.6 M a year ago, reflecting improved cost control and revenue build-up.
  • Sale of a Pediatric Rare Disease Priority Review Voucher generated $148.3 million of non-dilutive capital, boosting pro forma cash to ~$217 million and ensuring financial flexibility to execute on strategic priorities.
AI Generated. May Contain Errors.
Earnings Conference Call
Zevra Therapeutics Q1 2025
00:00 / 00:00

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