Zevra Therapeutics, Inc.ZVRANASDAQ
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Operator: Good afternoon, and thank you for joining Zevra's Third Quarter 2025 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 Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications. Please go ahead.
Nichol Ochsner: 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 third 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 in any forward-looking statements due to risks and uncertainties associated with Zevra's business. Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other important factors that may lead to actual results differing materially from the projections made and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K and other filings with the SEC. I'm pleased to welcome Zevra's management team members participating in today's call. Neil McFarlane, Zevra's President and Chief Executive Officer; LaDuane Clifton, our Chief Financial Officer; and Josh Schafer, our Chief Commercial Officer. Our Chief Medical Officer, Adrian Quartel, will also be available today for the question-and-answer session. Now, it's my pleasure to hand the call over to Neil.
Neil McFarlane: Thank you, Nichol, and welcome to everyone joining our quarterly call this afternoon. Zevra is building on a foundation for success, guided by our strategic priorities and fueled by the strong performance of MIPLYFFA, which is delivering meaningful benefits to patients with Niemann-Pick disease type C or NPC. This impact is translating into revenue generation and leading us towards operating stability that enables us to invest strategically to build a leading rare disease company. MIPLYFFA is a foundational advancement in the management of NPC and the first treatment approved in the United States for this indication. NPC is a progressive and fatal disorder caused by mutations in the NPC1 or NPC2 gene, which disrupts intracellular lipid transportation. This leads to cholesterol and lipid buildup in cells, causing widespread dysfunction. NPC affects both children and adults with symptoms, including cognitive decline, speech and swallowing difficulties, motor impairments and premature mortality. NPC is considered to be an ultra-rare disease with an estimated 900 individuals affected in the United States. And of those, only 300 to 350 have been diagnosed. We're excited to deliver on the promise of strong scientific data, which demonstrates that MIPLYFFA in combination with miglustat is the only disease-modifying therapy that halts the progression of NPC at 12 months and demonstrated a rapid onset of effect within 12 weeks of treatment initiation. Long-term data showed that MIPLYFFA was both safe and effective in halting the progression of the disease for more than 5 years. This compelling data is resonating with the community and driving continued strong performance. I'm pleased to share that our third quarter net revenue reached $26.1 million, driven by $22.4 million in net revenue from MIPLYFFA. Additionally, there were 8 new prescription enrollment forms received in the quarter, bringing the total to 137 since MIPLYFFA became commercially available just a year ago. Building on the success of our launch, our commercialization strategy is focused on identifying diagnosed patients who may benefit from MIPLYFFA and on implementing targeted initiatives to improve detection and diagnosis for the many people who remain unaware they are living with this life-threatening disease. We have seen traction in both of these cohorts, and Josh will further elaborate later in the call. A significant near-term growth milestone is to expand into the European market, where we estimate approximately 1,100 people are living with NPC. In Europe, there is an established precedent for treatment with miglustat. And with the existing clinical network, there is a favorable environment for a foundational treatment such as MIPLYFFA. As a reminder, we filed the Marketing Authorization Application or MAA at the end of July. That application has been validated and is under review by the European Medicines Agency. We are highly confident in our submission, which incorporates an expanded data set beyond what served as the basis for the FDA filing and U.S.A. approval. As a reminder, our filing submission included data on more than 270 NPC patients that have been treated with MIPLYFFA through the pivotal trial, open-label extension study, expanded access programs and pediatric substudy. Our go-to-market strategy for Europe is under active development and will inform our decision to launch independently or through a strategic collaboration. Additionally, our Expanded Access Program in Europe and the U.K. is playing a critical role in building product experience and fostering strong relationships with physicians and clinics with 92 patients enrolled at the end of the third quarter. These engagements not only support awareness and education, but also lay the groundwork for a potential commercial launch. We've established a solid patent position for MIPLYFFA, and we've also requested a patent term extension with the U.S. Patent and Trademark Office, which could provide coverage beyond the term granted through our orphan drug exclusivity. For OLPRUVA, there was one new prescription enrollment form in Q3 and covered lives reached 81%. Notwithstanding our best commercial efforts, OLPRUVA’s clinical differentiation has not penetrated a mature and well-served UCD market. As a data-driven organization committed to making judicious investments that drive meaningful benefit for patients, we have made the decision to scale back our sales and marketing efforts for OLPRUVA. While we evaluate the options for OLPRUVA, we will maintain market access and product availability for patients and continue providing support through our patient services program, AmplifyAssist. Going forward, we will provide updates if there's anything material to report. Turning to our development pipeline. We are actively recruiting patients in the DiSCOVER trial, our ongoing Phase III trial to evaluate Celiprolol as a treatment for Vascular Ehlers-Danlos Syndrome, or VEDS. VEDS is a rare inherited disorder caused by mutations in the COL3A1 gene, which impairs Type 3 collagen production, essential for vessel and organ strength, leading to fragile tissues that put patients at high risk of ruptures in the arteries, intestines and uterus. In Q3, we enrolled an additional 5 patients in the DiSCOVER trial, bringing the total to 44 of 150 patients required for full enrollment. Our efforts are focused on bolstering our relationships with clinics involved in the management of these patients' care while also expanding the network of genetic testing centers collaborating with us to identify individuals carrying the gene mutation. In parallel, we are conducting educational outreach to vascular specialists who frequently encounter these patients following rupture-related surgical interventions. As an event-driven study under a special protocol assessment with the FDA, we have an interim analysis built in after 28 qualifying events, and there has been one confirmed event through the end of Q3. In summary, we continue to have strong momentum in Q3 and are excited about the many future growth opportunities for MIPLYFFA, including continued performance in the U.S., potential for patent term extension, significant upside potential through geographic expansion and the late-stage development asset with Celiprolol. I'll now turn the call over to Josh for a deeper dive into our commercial progress with MIPLYFFA.
Joshua Schafer: Thank you, Neil, and good afternoon. Physicians have expressed enthusiasm and interest in MIPLYFFA as the foundational treatment for NPC. As Neil mentioned, in the third quarter, we received 8 new prescription enrollment forms. This quarter marks the 1-year anniversary of the launch. And as we reflect on our progress, we are pleased with the overall trends. We have received enrollment forms from the leading NPC centers of excellence that our rare disease specialists have been targeting, demonstrating the impact of our field teams. In addition, our sales efforts have expanded beyond the centers of excellence into community practices where many NPC patients are treated after initial diagnosis and treatment recommendations. Our sales and medical teams have been informing and educating clinicians about our differentiated mechanism of action, demonstrated clinical benefit as well as the recently published long-term data from our open-label extension trial. These compelling data provide real-world evidence that resonates deeply with physicians and patients alike. We recently conducted independent market research with clinicians and NPC patients, and I'm pleased to share that MIPLYFFA was recognized as the preferred disease-modifying therapy for NPC. Clinicians reported confidence in prescribing MIPLYFFA for long-term use based on its efficacy and tolerability and patients and caregivers described noticing improvements in balance and swallowing, fewer falls and changes in cognition and speech. We continue to focus on raising awareness of not only MIPLYFFA, but also of NPC to support the diagnosis and treatment of new patients. Our early investments in disease awareness initiatives, including our Learn NPC, Read Between the Signs campaign and our genetic testing resources are identifying patients who were previously undiagnosed. Through this testing program, physicians can order a genetic test for individuals suspected of having NPC. Anecdotally, we are hearing of physicians previously unfamiliar with NPC who have identified suspected patients, confirmed diagnosis through our resources and are now prescribing MIPLYFFA. We have built bespoke AI-driven models to analyze electronic medical records and claims data to identify both formally diagnosed but untreated patients and those who have not yet been -- who have not yet received a diagnosis. These examples highlight the tangible impact of increased disease awareness, and they underscore the importance of continued education and outreach in ensuring patients receive timely diagnosis and treatment. In the third quarter, we also maintained an impressive presence at local, regional and national scientific conferences and patient events. These included an oral presentation at the International Niemann-Pick Disease Alliance face-to-face meeting, several poster presentations at the Child Neurology Society Annual Meeting and 4 poster presentations at the International Congress of Inborn Errors of Metabolism. The presentations included new data demonstrating that patients who were randomized to receive miglustat only in our pivotal trial and then added MIPLYFFA in the open-label extension phase experienced a decline in annual disease progression. We continue to generate, publish and present peer-reviewed data to reinforce MIPLYFFA's use as a foundational treatment for NPC. Finally, growing market access via reimbursement coverage remains top of mind for us. And in the third quarter, we increased the percent of covered lives to 66%, which is in line with what we would expect 1 year into the launch. We're achieving high overall reimbursement rates for MIPLYFFA, either through direct formulary coverage or through medical exception pathways. We seek to provide access to as many patients as possible. And to date, there has been broad utilization of our co-pay assistance and patient services. Importantly, MIPLYFFA patients are showing continued adherence and staying on treatment. Patients conveniently receive their monthly refills through our AmplifyAssist program, which aims to deliver a first-class experience for patients and has helped to establish Zevra as a preferred partner within the NPC community. In short, our strong third quarter performance reflects MIPLYFFA’s differentiation and our focused commercial execution, and we are encouraged by the sustained momentum heading into 2026. Now I will turn the call over to LaDuane.
R. Clifton: Thank you, Josh, and good afternoon, everyone. In addition to the financial details included in today's call, we encourage you to refer to Zevra's quarterly report on Form 10-Q for more detailed information, which we intend to file later today. In the third quarter of 2025, we reported net revenue of $26.1 million, which includes $22.4 million from MIPLYFFA, $2.4 million in net reimbursements from the French EAP for arimoclomol, $1.2 million from royalties and other reimbursements under the AZSTARYS license and $100,000 from OLPRUVA. For our commercial products, we recognize revenue when shipments are received by the specialty pharmacy. Q3 MIPLYFFA net revenue was impacted by the redesign of Medicare Part D rebates, which led to recognition of a gross to net true-up from prior quarters of $1.2 million. The updated rates will become an ongoing component of gross to net estimates going forward. Cost of product revenue for the third quarter, excluding noncash intangible asset amortization, was $1.2 million. Operating expense for the third quarter was $20.4 million, which was a decrease of $6.8 million compared to the same quarter a year ago. R&D expense was $3.4 million for Q3 2025, which was a decrease of $7.5 million compared to Q3 2024, primarily due to reduced third-party costs following the completion of the KP1077 Phase II trial, combined with a decrease in personnel-related costs. SG&A expense was approximately $16.9 million for Q3 2025, which was an increase of $700,000 compared to Q3 2024 due to additional investments in our commercial, medical and launch activities. Looking ahead to the impact of scaling back on promotional activities for OLPRUVA, these resources are expected to be reallocated to MIPLYFFA and to our patient services activities. Therefore, a meaningful change in selling expenses is not anticipated. Net loss for Q3 2025 was $500,000, which led to loss per basic and diluted share of $0.01. This includes noncash stock compensation expense of $2.8 million, noncash fair value adjustment related to warrant and CVR liabilities of $5.5 million during Q3 and noncash intangible amortization expense of $300,000. For the same quarter in 2024, we reported a net loss of $33.2 million or $0.69 per share. As of September 30, 2025, total cash, cash equivalents and investments were $230.4 million, which was an increase of $12.7 million compared to the end of Q2 2025. The increase in cash included $6.6 million in nonoperating cash received in exchange for the exercise of warrants and options during Q3 and $2.3 million in cash from interest income. Total debt was approximately [ $61 million ]. Based on the company's current operating forecast, we believe available financial resources are sufficient to execute on our strategic priorities independent from the capital markets. While we continue to anticipate variability in our working capital requirements, which is largely dependent on the timing of receivables and payables, our disciplined approach to operating investments and capital allocation has significantly strengthened our financial position. Our commitment to maintaining cost efficiency and rigorous operational accountability has cultivated the flexibility and capacity to strategically invest in high-impact opportunities such as the MIPLYFFA U.S. launch and the Phase III study of Celiprolol while also preparing for the potential launch of MIPLYFFA in Europe. We are well positioned to pursue the opportunities we have to drive long-term value creation for our stakeholders and to accelerate our growth initiatives with confidence. Now, I will turn the call back to Neil for his closing remarks.
Neil McFarlane: Thank you, LaDuane. We're pleased by the continued success of MIPLYFFA and deeply encouraged by the support from both patients and physicians. Looking ahead, we believe we have a significant growth opportunity on the horizon with the potential approval and commercialization in Europe. We're committed to aligning our capital allocation with our strategic plan and to invest in opportunities that meaningfully impact patients while maintaining a sharp focus on creating sustainable growth. Thank you. We'll now open the call for questions. Operator?
Operator: [Operator Instructions] Our first question will come from Kristen Kluska with Cantor.
Kristen Kluska: The first one I have is, as we think about the last 2 quarters, there's been 15 new start forms. Curious if any of these have been patients that are newly diagnosed through perhaps some of your efforts? And also, as we think about the next few quarters up ahead, how you're thinking about the potential of finding additional patients that currently aren't diagnosed today?
Neil McFarlane: Kristen, thanks for the questions. I'll get us kicked off a little bit here. You're absolutely right. The last couple of quarters, we've had about 13 new start forms and really pleased with where the team has been continuing to move forward. And actually, as I'm thinking about this, I think it's not 13, but 15, we had 8 and 7. So I just want to make sure we clarify that on the call. The reality, though, is that our team continues to move forward with the investments that we're moving. I'll ask Josh to talk a little bit more around the newly diagnosed components of where we are.
Joshua Schafer: Kristen, thanks for the question. And yes, we're really excited not only with the performance we're seeing in terms of the number of enrollments coming in. But as you noted, many of them are coming in as newly diagnosed patients, and that number has increased from last quarter to this quarter. And that is largely through the efforts that we've articulated in the prepared remarks. But specifically, these are coming from the disease awareness campaign and our partnership with the genetic testing collaborators to be able to identify these new patients and make us aware of these newly diagnosed patients so then we're able to go into those offices and work with the physicians and clinicians to get them onto MIPLYFFA. We've also done a lot in terms of helping to build disease awareness through our medical teams and building the evidence through conferences and publications. And we've also done quite a bit in terms of using advanced analytics and machine learning to be able to look into electronic medical records and claims data to find these undiagnosed patients. And so we are seeing a lot of that success.
Neil McFarlane: And Kristen, let me follow up on that just a little bit. When we think about our 1 year in the market, which we're celebrating here right now, the total number is 137 prescription enrollment forms. That's approximately 40% of the diagnosed patient population that we've actually seen. And that's really a testament not just to the team and what they're doing, but also the strength of the long-term data that we've been able to execute on educating physicians and getting that awareness out there with patients and the like. So really pleased with where we are 12 months into the launch.
Kristen Kluska: Okay. Appreciate that. And then when I think about your balance sheet, it's very robust. And this quarter, you were pretty much on the edge of breaking even. So how are you thinking about perhaps in the near to midterm, how you might take advantage of your robust balance sheet?
Neil McFarlane: Yes. Thanks, Kristen. So we go through an annual cycle of planning for strategic session with our Board. And part of that, we thought -- we talk about capital allocation and understanding where is it that we can best be positioned to be able to move forward. I'll ask LaDuane to touch base on it, but this is all about us leaning in, executing on what we have, doing the best we can for both the commercial products as well as the development programs and then earning the right to do something else later on. LaDuane?
R. Clifton: Yes. Thank you, Neil. And Kristen, as we execute against the MIPLYFFA launch, we certainly see that our operating results are beginning to reflect that success. And so we're appreciative and thankful for how that's coming through. Although I will tell you, although we did generate cash -- some cash this quarter, there remains some variability in our results as we continue to have dynamic working capital requirements between accounts receivable timing and accounts payable timing. So we haven't turned the corner yet, but definitely, things are pointing in the right direction. And as Neil said, we are very much guided by our strategic plan. And I think executing right now on what's in front of us, the MIPLYFFA launch in the U.S., the expansion and potential approval in Europe, other ex-U.S. opportunities that we're exploring and then maintaining and continuing to support Celiprolol. These are the opportunities we have in front of us. And as we execute on those, we can continue to apply discipline and think about how we deploy capital across all the opportunities that may present themselves.
Operator: Our next question will come from Sami Corwin with William Blair.
Samantha Corwin: Congrats on the progress. I guess since the majority of patients that are submitting the new enrollment forms or newly diagnosed patients, how are you kind of thinking about reaching the additional 200 or so patients that have already been identified? I guess when I'm kind of thinking about strategy, that kind of seems like it'd be low-hanging fruit. So how are you thinking about that? And then given you're a year into the launch, what percentage of patients are you seeing that are getting refills?
Neil McFarlane: Great. So Sami, I want to make sure that I clarify. We didn't say the majority of the new patients that are coming in or hope we didn't say that the majority of the new patients that are coming into prescription forms are undiagnosed patients that are newly diagnosed patients. There are a component of them that are, but majority would be an overstatement. We have actually -- this is not the first quarter we've gotten new patients, though. We've actually had new patients in our last quarter as well. So that -- I just want to clarify that. In regards to how we're thinking about the additional patients that are out there, I'll actually ask Josh to hit on that. And then 1 year in terms of refills, I'll ask Josh to hit on that as well.
Joshua Schafer: Yes. So Neil, thank you for clarifying that majority comment. I was going to make the same. The majority of patients who are getting -- submitting enrollment forms are, in fact, patients who have previously been diagnosed and either on a current treatment or have been diagnosed and not received treatment. And we're continuing to find more and more patients like that every day. As we've noted on prior calls, we know where the majority of these patients are, and we continue to go into those offices and work with the clinicians to get those patients on to MIPLYFFA. In addition, we are very pleased that there are undiagnosed or previously undiagnosed patients who are now coming into the enrollment or now receiving enrollment forms as well. With regards to the percent of refills, I would note that we are really, really pleased that the majority of patients are actually who are coming in, we're able to find a pathway to getting reimbursement. We have 66% of covered lives today. And even those who are not covered through formulary, we're able to get paid coverage through a medical exception pathway. And almost all of those patients are getting refills on a month-to-month basis.
Samantha Corwin: Got it. And one more, if I may. What is the average age or weight of the patients currently on therapy? And has that kind of differed with the -- or differed compared to the patients who are on the Expanded Access Program versus those that have come on to drug after?
Neil McFarlane: Yes. So I'll take that one, Sami. So when -- if you recall, our clinical trial program was primarily skewed towards children. Our Expanded Access Program actually brought in more adults, and it was about a 50-50 mix between children and adults. As we now actually get into the real world, the real world experience has continued to actually allow us to actually have that [ 50-50 percent ] split in regards to kids and adults. In regards to the weight, what we did when we actually announced our pricing for the program is we used an average weight that was based on our 83 Expanded Access Program patients that were in there as well. So we don't provide the guidance specific to weight, but I do believe that it's still at a 50-50 today.
Operator: Our next question will come from Eddie Hickman with Guggenheim Securities.
Eddie Hickman: Congrats on the progress this quarter. Just a few for me. It's really nice impressive increase in coverage, I think, growing from 52% to 66%. So what have been the key drivers to see that much increase this quarter? And how fast and how high do you anticipate that coverage level going? And then when you -- now that you have a year under your belt, can you talk about how the patient enrollment form split is between the centers of excellence and the non-centers of excellence?
Neil McFarlane: Yes. I'll ask Josh to take both of those. It’s right up his alley. .
Joshua Schafer: Yes. So we're really pleased with the progress that we're making in terms of the percent of covered lives. I think it really speaks to the strength of the data and the fact that MIPLYFFA is the only disease-modifying treatment out there with the data that shows that it halts the progression of disease at 12 months. And that data has been really compelling to payers. We're continuing to see and anticipate that, that coverage will increase. And we feel like we are right where we should be 12 months into launch, and we're going to continue to work to increase that coverage and to ensure as many patients as possible can receive MIPLYFFA. We're also really pleased that we've been able to target and receive enrollment forms from almost all of the centers of excellence that our sales representatives or rare disease specialists are targeting. But more importantly, what we're seeing now is that the new enrollments coming in are coming from community-based physicians as well. And the dynamic is maybe a patient will go to one of these centers of excellence, get an initial diagnosis and a referral and then go back to his or her local GP or internal medicine specialists who is then managing the care going forward. So what we're seeing is a real breadth and increase in the overall number of prescribers as well, and we're really pleased with that.
Eddie Hickman: Appreciate that color. And then one final one, if I may. For Europe, when do we expect the next update on that? And when just thinking about maybe just the French EAP, like should we assume a similar conversion as the U.S. EAP once that's approved there? Or help us think about the dynamic of how that EAP will factor into paid drug if you got approved there?
Neil McFarlane: So Eddie, we are in the valid -- as we talked about in the prepared remarks, we are right now in the post-validation phase, 120-day clock that will allow us to be able to get our first inclination as to where we are. But I think it is important to remind everybody that we are coming off of the back of an FDA approval and an AdCom and a positive vote and a very solid launch into the U.S. market. It's the largest, most compelling data package in NPC. We've added, as we talked about in our prepared remarks, the additional data on top of what we submitted to the FDA. So we feel really good and confident about the MAA submission. Once we get that 120-day information, it will provide us with an opportunity to know a lot more. I don't know when we will then come back to you and the Street to understand what that looks like because it's a list of questions. It's not an approval. For us to be able to understand how to be able to then talk about that more broadly, I think it will be more closer to the 150 days. So give us some time on that and then see if we come through. You talked about the French EAP conversion. We do feel really good about the opportunity, but it will be based on reimbursement on the conversion rates within Europe. Once we get the product approved, each country will go country by country around how we reimburse -- navigate reimbursement within those countries. And those countries that come quickest will convert fastest. And today, as I mentioned on the call, we have 92 patients now in our -- it's really, we call it our global EAP. It's primarily Europe now. And those patients are a very small number of countries. So we feel like we've got the ability to continue to drive patients in the EAP -- well, not drive, fulfill patients in the EAP that we'll be able to convert them at the same rate once we get reimbursement in the countries.
Operator: Our next question will come from Sumant Kulkarni with Canaccord.
Sumant Kulkarni: On MIPLYFFA in the U.S., we appreciate the 40% share that you achieved pretty quickly, but do you think we are at steady state with respect to addition of patient enrollment forms for a quarter? And what are the key variables that might cause a step change in that number, especially given that the sales force will only have MIPLYFFA to focus on given the scale back on OLPRUVA?
Neil McFarlane: Yes. So we've seen 13 in the first quarter, 7 in the second quarter, 8 now in this third quarter. I think it's way too early for us to call this a steady state. We are seeing the results of our efforts to find new patients and to diagnose new patients. And as mentioned, several of the new enrollments that we got in the quarter were from newly diagnosed patients. So I expect that we're going to continue to see more of that taking place and the efforts of our sales team to continue to identify currently diagnosed patients, to also find these undiagnosed patients. I think all of that is really telling a good story looking forward. I'd also mention that we completed recently some market research, which we completed with payers, clinicians as well as patients. And clinicians stated that they felt very confident in prescribing MIPLYFFA for the long term based on its safety and its efficacy, and they recognized it as really the preferred disease-modifying therapy for NPC. And patients stated that they saw median improvements in their balance, their swallowing, fewer falls and change in cognition. And I think all of that really speaks well for what future quarters might bring.
Sumant Kulkarni: Got it. On MIPLYFFA in Europe, what are your initial thoughts on what price might be like relative to the U.S. or the EAP in France? And finally, there's been -- I guess, the sleep market is waking up with buyouts orexin data. What are your latest thoughts on KP1077 and the strategic options there?
Neil McFarlane: Yes. Let me take a couple of those. In regards to Europe -- in terms of European pricing, Sumant, we've done in parallel to our marketing authorization application, a lot of work in regards to understanding the market dynamics in Europe, what the pricing potential is in Europe. As you just stated around our EAP in France, we do get pre-commercial revenue from that, which has also been part of the calculation. We won't know based on -- until we understand what the label looks like, what a final price in Europe is. What we do really appreciate around Europe is that of the 1,100 patients from a prevalence perspective, there are the majority of those patients with miglustat being approved for well over a decade that have actually been diagnosed and have been on some type of a therapy, that provides us with an opportunity to be able to impact the European market at a faster rate than that of what we even have seen in the U.S. because the diagnosis rates in the U.S. have been lower than that of in Europe. So that's something that, for us, we believe the penetration will be good. Will it be the same price as the U.S.? That's to be determined at this point.
Sumant Kulkarni: Got it.
Neil McFarlane: You also asked another question, Sumant, around 1077. Yes. No. So to comment on 1077, we remain open to strategic alternatives. We are having conversations, but I can't really comment more on that right now.
Operator: [Operator Instructions] Our next question will come from Brandon Folkes with H.C. Wainwright.
Brandon Folkes: Congratulations on the quarter. Maybe just following on from a few questions before. How are you thinking about additional commercial investments behind MIPLYFFA in the U.S. in 2026 versus the current commercial infrastructure? And given you're seeing some success, right, in the disease awareness campaign driving previously undiagnosed, right? I think it's the success to have any of those patients kind of being enrolled at this stage. But how are you weighing up maybe potential additional investments behind disease awareness and increased diagnosis in 2026?
Neil McFarlane: So Brandon, I'm going to ask Josh to comment on some of the specifics around this. But if you recall, we have been investing now into a commercial infrastructure to be able to not only invest in one product, but in multiple products in these centers of excellence and providing value to these centers that we can then provide the education, the patient services, the medical support, the clinical data that's necessary to be able to pull products through and provide patients with the best experience that they can once they get prescriptions. So I think that our investment is rightsized to be able to move forward and execute on this product. And as we have mentioned in the past, getting OLPRUVA up to speed too in terms of the awareness and clinical differentiation. Josh, do you want to talk a little bit about 2026?
Joshua Schafer: Yes, sure. And just to kind of underscore what Neil said, our top priority from a commercial and medical standpoint is to identify patients who can benefit from MIPLYFFA. So there really is no single tactic that we're talking about. It is a cross-functional across the entire organization. This is a top priority for us. And so we continue to invest in new publication strategies and new data generation, continuing to look at ways that we can identify patients through claims data and electronic health records. So we're continuing to evaluate that, and our budget is really based on that as a priority. We've been doing that in 2025. We'll continue to do that in '26 and onward. So I don't see any market change in our investments to do that. We're going to continue to evaluate the -- what works, what doesn't work and pivot as we need to because it really is an absolute priority for us.
Operator: Our next question will come from Jonathan Aschoff with ROTH Capital.
Jonathan Aschoff: Congrats on the quarter. And I was just curious, what's the rough cost you expect to have to sell out to launch in the EU? And is there anything noteworthy to say about MIPLYFFA inventory levels end of the second quarter versus end of the third?
Neil McFarlane: Yes. Thanks, Jonathan. I'll take that in regards to the European opportunity. We've laid out a number of different approaches in terms of the research we've done, the pricing we've done, the approaches we may take. We've been having capabilities presentations from local distributors to some type of a hybrid approach that we might take or to just put it in the hands of folks who can actually deliver for patients better than we could. I mentioned previously that we had 92 patients already in Europe in our Expanded Access Program and continuing to grow that on a quarter-over-quarter basis. Our goal is to be able to either execute on the opportunity in Europe ourselves or put it in the hands of somebody who can do it better, [Technical Difficulty] shareholders as well. What that cost is, is to really depend upon the approach that we take. So I can't give you a number today. But what I can tell you is that we've done all the work that's necessary for either going it alone or put it in the hands of another party that can service patients better and with better economics as well as the ramp that it would take to be able to get MIPLYFFA in the hands of patients in Europe. So stay tuned on what that might look like as we get through our labeling discussions and the MAA. I'll ask LaDuane to talk about your inventory question on a quarter-over-quarter basis.
R. Clifton: Yes. Thank you. The MIPLYFFA inventory, we monitor carefully, and we utilize internal metrics like days on hand to make sure that what's in the channel is consistent and is sufficient to service patients. So there is no large fluctuations quarter-to-quarter since we use those metrics to guide us and make sure that, that's not driving any kind of trends. One other detail I want to make sure is clear, though. When we think about revenue, we also have the impact of our gross to net. And while we don't normally give the details of that, we did note that there was a true-up adjustment from the recent changes redesigned to the Medicare Part D rebates. And that did have some impact as we noted. About $1.2 million was recognized in Q3, therefore, reducing net sales by that amount, which was from prior periods. So just calling out that there's 2 dynamics there to be clear. Inventory in the channel is consistent at the levels appropriate that we've maintained throughout the launch period. And then this gross to net had a unique impact in Q3, and we've adjusted our rates based on that to go forward from there.
Jonathan Aschoff: Lastly, Neil, just remind me how many of those 92 patients are French or in France?
Neil McFarlane: Yes. Approximately 30 patients is what has been consistent over a number of years in the French EAP program. It's remained the same.
Operator: This concludes the Q&A portion of today's call. I'll now turn it back over to Neil for closing remarks.
Neil McFarlane: Thank you all for joining us today. Sorry, operator, are you still there? It looks like we have one more person in the queue.
Operator: We'll next go to Jason Butler with Citizens.
Jason Butler: I guess just wondering if you could comment qualitatively on the number of patients that have received -- have an enrollment form but haven't received reimbursed drug yet. And I guess what I'm asking is directionally quarter-over-quarter, has that remained stable? Or are you seeing that number decrease or any comment there? And then also the time from receiving an enrollment form to getting reimbursed drug. Is that now stable? Or is that changing at all?
Neil McFarlane: Yes. We are still in the active phase of launch, Jason, but it's a really good question that I'll ask Josh to hone in on. The pull-through of patients to paid drug, I am incredibly impressed with the team. And then also in regards to as we get new enrollments in and then are able to move through that reimbursement process, team is doing an outstanding job. So Josh?
Joshua Schafer: Yes. Jason, thanks for that question. And again, it is something that we're incredibly proud of what we're seeing. The number of patients who are able to receive a paid drug is exceptionally high. We do have patients who are going through some period of free drug as we're investigating their benefits. They might go on for a 30-day period. Those patients are oftentimes unable to get into paid patients. So at any given time, we might have some patients in the free goods program who then become paid in the following month. So it's a little bit difficult to give you any precise numbers there. But what I can tell you is the vast majority of them do end up getting paid. And we are seeing -- your second question was around the time from an enrollment coming in to then a paid dispense. And we have seen that time shrunk dramatically since launch. And I think that's due to, again, the execution of our team. It's also due to the payers really beginning to appreciate the strength of MIPLYFFA data, and the benefit that it brings to patients. And so that time has really gone from what was, in some cases, months, now down to weeks. And we have, in fact, seen some patients get turned around in 24 hours. And so we're really pleased with that.
Jason Butler: Got it. And then just a quick follow-up for LaDuane on the gross to net point. Is this reestimation or true-up something that we should expect to happen on a seasonal basis, an annual basis? Or just how should we think about the stability of the gross to net moving forward?
R. Clifton: Yes. So this specific adjustment came from the redesign of the Medicare Part D, which came out of recent sort of administration changes, Inflation Reduction Act, et cetera. And so this one is sort of, I think, behind us. But -- and let's be clear, this was a change in the rebate in terms of catastrophic coverage. So MIPLYFFA being one of the higher price as a rare disease drug, it does get through to catastrophic coverage under Medicare sooner. So 2 changes. They made that threshold to get to catastrophic sooner, and they also increased that rebate from 9% up to 20%. So that's a change now that's in the system, and we've adjusted our estimates going forward. But the other detail about gross to net and one of the reasons we don't always dig into the details, except for in this case where it had a meaningful impact in this quarter is there could be other adjustments to gross to net going forward. Payer mix is something that early days in the launch. We know where we are today, but that could shift. We could continue to see changes in other dynamics within that. And so gross to net changes probably are something we will live with as a commercial company. But for right now, I think this specific item is now trued up and would be in the computations.
Operator: I am showing no further questions at this time.
Neil McFarlane: Thank you, operator, and thanks, everybody, for joining us today. We look forward to discussing our year-end and Q4 data with you in the coming year.
Operator: Thank you. This brings us to the end of today's meeting. We appreciate your time and patience. You may now disconnect. Thank you.