Zevra Therapeutics, Inc. Q3 FY2025 Earnings Call

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.

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.

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 toward 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. 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.

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 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.

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.

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.

Our first question will come from Kristen Kluska with Cantor.

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?

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.

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.

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.

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?

Yes, thanks, Kristen. We go through an annual planning cycle with our Board where we discuss capital allocation and explore how we can best position ourselves to move forward. I'll have LaDuane address this, but it's all about our commitment to executing on our existing resources, doing our best for both the commercial products and development programs, and then earning the opportunity to pursue additional initiatives later. LaDuane?

Thank you, Neil. Kristen, as we proceed with the MIPLYFFA launch, our operating results are beginning to show positive outcomes, which we appreciate. However, while we generated some cash this quarter, there is still variability in our results due to fluctuating working capital needs related to accounts receivable and accounts payable timing. We haven't completely turned the corner yet, but progress is being made. As Neil mentioned, we are closely following our strategic plan. Currently, our focus is on the MIPLYFFA launch in the U.S., potential expansion and approval in Europe, exploring opportunities outside the U.S., and supporting Celiprolol. These are the opportunities ahead of us, and as we move forward, we will maintain discipline and consider how to best allocate capital across these various prospects.

Our next question will come from Sami Corwin with William Blair.

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?

Great. So Sami, I want to clarify something. We didn't say that most of the new patients coming in or the new patients filling prescription forms are undiagnosed or newly diagnosed. Some of them are, but saying the majority would be an exaggeration. This isn't the first quarter we've seen new patients; we had new patients last quarter as well. I just wanted to make that clear. Regarding how we view the additional patients out there, I'll ask Josh to address that. He'll also cover the topic of refills after one year.

Yes. Thank you, Neil, for clarifying the majority comment. I was going to mention the same point. Most of the patients submitting enrollment forms are those who have previously been diagnosed and are either currently on treatment or have been diagnosed but not treated. We continue to identify more patients like this daily. As we have mentioned in previous calls, we know where most of these patients are, and we keep working with clinicians in those offices to help get them onto MIPLYFFA. Additionally, we are pleased to see undiagnosed patients now coming forward to fill out enrollment forms as well. Regarding the percentage of refills, I want to emphasize that we are very pleased that most incoming patients have been able to find a way to secure reimbursement. Currently, we have 66% of covered lives, and even for those not covered by formulary, we can obtain paid coverage through a medical exception pathway. Almost all of these patients are receiving refills on a monthly basis.

Got it. And one more question, if I may. What is the average age or weight of the patients currently on therapy? Has that differed for those in the Expanded Access Program compared to those who have started the drug afterwards?

Yes, I'll address that question, Sami. Our clinical trial program focused mainly on children, but our Expanded Access Program ended up including more adults, resulting in an approximately equal mix of children and adults. As we transition to real-world applications, this 50-50 split has remained consistent. Regarding weight, when we announced our pricing, we based it on an average weight derived from the 83 patients in our Expanded Access Program. While we don't provide specific guidance on weight, I believe it still holds at a 50-50 ratio today.

Our next question will come from Eddie Hickman with Guggenheim Securities.

Congratulations on the progress this quarter. I want to highlight the impressive increase in coverage, which has grown from 52% to 66%. What have been the key drivers behind such a significant increase this quarter? How fast and how high do you expect that coverage level to rise? Now that you have a year's experience, could you discuss the patient enrollment split between the centers of excellence and the non-centers of excellence?

Yes. I'll ask Josh to take both of those. It’s right up his alley.

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.

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?

So Eddie, we are currently in the post-validation phase, which is a 120-day period that will give us our first insights into our progress. It's important to remember that we are coming off an FDA approval, an advisory committee vote, and a strong launch in the U.S. market. We have the largest and most impressive data package in NPC. Additionally, we have included new data beyond what was submitted to the FDA, which gives us confidence in the MAA submission. Once we receive the information after the 120 days, we will have a clearer understanding. However, it may take closer to 150 days for us to effectively discuss our findings. Regarding the French EAP conversion, we are optimistic about the potential, but it will depend on reimbursement rates in Europe. After we receive product approval, the reimbursement process will be handled on a country-by-country basis, with quicker approvals leading to faster conversions. Currently, we have 92 patients involved in our global EAP, which is mainly in Europe, and those patients are from a limited number of countries. We believe we can continue to fulfill patients in the EAP and expect to convert them at a similar rate once reimbursement is in place in those countries.

Our next question will come from Sumant Kulkarni with Canaccord.

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?

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.

On MIPLYFFA in Europe, what are your initial thoughts on the pricing compared to the U.S. or the EAP in France? Additionally, the sleep market seems to be gaining momentum with buyouts related to orexin data. What are your current thoughts on KP1077 and the strategic options available?

Yes. Let me address a couple of those points. Regarding Europe and the pricing situation, we've been conducting extensive research on market dynamics as we move forward with our marketing authorization application. As you mentioned about our expanded access program in France, we are generating pre-commercial revenue from that, which has been factored into our assessment. We won't have clarity on the final pricing in Europe until we have a better understanding of the product labeling. One key aspect we recognize in Europe is that out of 1,100 patients, a significant portion has already been diagnosed and treated with miglustat for over a decade, which provides us with an opportunity to penetrate the European market more rapidly than what we have seen in the U.S., where diagnosis rates have been lower. Therefore, we are optimistic about our market penetration. However, whether the pricing will be equivalent to that in the U.S. remains to be seen. 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.

Our next question will come from Brandon Folkes with H.C. Wainwright.

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?

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?

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 what works, what doesn't work and pivot as we need to because it really is an absolute priority for us.

Our next question will come from Jonathan Aschoff with ROTH Capital.

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?

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. 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.

Yes. 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.

Lastly, Neil, just remind me how many of those 92 patients are French or in France?

Yes. Approximately 30 patients is what has been consistent over a number of years in the French EAP program. It's remained the same.

This concludes the Q&A portion of today's call. I'll now turn it back over to Neil for closing remarks.

Thank you all for joining us today. Sorry, operator, are you still there? It looks like we have one more person in the queue.

We'll next go to Jason Butler with Citizens.

Could you provide some insights on the number of patients who have submitted an enrollment form but have not yet received the reimbursed drug? Specifically, has that number remained stable on a quarter-over-quarter basis, or have you noticed a decrease? Additionally, is the timeframe from submitting an enrollment form to receiving the reimbursed drug stable, or have there been any changes in that regard?

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, the team is doing an outstanding job. So Josh?

Yes, Jason, thank you for that question. We're very proud of what we're seeing. The number of patients receiving a paid drug is very high. Some patients may start with a period of free drug while we assess their benefits, which might last for about 30 days. During this time, they may not transition to paid patients immediately. Therefore, at any moment, we could have patients in the free goods program who are then moved to paid status in the following month. It's challenging to provide precise numbers, but I can assure you that the majority do end up getting paid. Regarding your second question about the time from enrollment to receiving a paid dispense, we've seen that period decrease significantly since the launch. This improvement is largely due to our team's execution and payers recognizing the strength of MIPLYFFA data and its benefits for patients. Consequently, the timeframe has decreased from months to weeks, and we have even seen some patients get processed within 24 hours. We're really pleased with this progress.

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?

Yes, the specific adjustment was due to the redesign of Medicare Part D that arose from recent administrative changes, including the Inflation Reduction Act. This is something we can consider resolved now. It's important to note that this change involved adjustments to rebates for catastrophic coverage. MIPLYFFA, being a high-cost rare disease drug, qualifies for catastrophic coverage under Medicare more quickly. Two key adjustments were made: the threshold to reach catastrophic coverage was lowered, and the rebate increased from 9% to 20%. This adjustment is now incorporated into our future estimates. However, regarding gross to net, we typically do not delve into details unless they significantly impact a quarter, but other adjustments may occur going forward. The payer mix can shift, especially early in the launch, and we anticipate ongoing changes in various dynamics. Therefore, changes to gross to net are likely to be a regular aspect of our operations as a commercial entity. For now, this specific item has been finalized and is reflected in our calculations.

I am showing no further questions at this time.

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.

Thank you. This brings us to the end of today's meeting. We appreciate your time and patience. You may now disconnect. Thank you.