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  Title	Q4 2024 Zevra Therapeutics Inc Earnings Call
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  Published Date	March 12, 2025, 11:31 a.m.
  Summary	Unlock stock picks and a broker-level newsfeed that powers Wall Street. Upgrade Now Q4 2024 Zevra Therapeutics Inc Earnings Call Thomson Reuters StreetEvents Wed, Mar 12, 2025, 12:31 PM 37 min read In
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  Content	Unlock stock picks and a broker-level newsfeed that powers Wall Street. Upgrade Now Q4 2024 Zevra Therapeutics Inc Earnings Call Thomson Reuters StreetEvents Wed, Mar 12, 2025, 12:31 PM 37 min read In This Article: ZVRA -1.86% Participants Nichol Ochsner; Vice President of Investor Relations and Corporate Communications; Zevra Therapeutics Inc Neil Mcfarlane; President, Chief Executive Officer, Director; Zevra Therapeutics Inc Joshua Schafer; Chief Commercial Officer and Executive Vice President, Business Development; Zevra Therapeutics Inc R. LaDuane Clifton; Chief Financial Officer, Treasurer, Secretary; Zevra Therapeutics Inc Adrian Quartel; Chief Medical Officer; Zevra Therapeutics Inc Kristen Kluska; Analyst; Cantor Fitzgerald LP Jason Butler; Analyst; Citizens JMP Securities LLC Sumant Kulkarni; Analyst; Canaccord Genuity Group Inc Oren Livnat; Analyst; H.C. Wainwright & Co LLC Sami Corwin; Analyst; William Blair & Co LLC Eddie Hickman; Analyst; Guggenheim Securities LLC Presentation Operator Good afternoon, and thank you for joining Zevra Therapeutics fourth quarter and full year 2024 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. Nichol Ochsner Thank you, and welcome to those who are joining us. Today, we will provide an overview of our accomplishments in the fourth quarter and full year of 2024, followed by a review of financial results. I encourage you to read our financial results news release, which we distributed this afternoon and is available in the Investors section of our website. Before we begin, 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 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. These forward-looking statements should be evaluated together with the cautionary statements contained in the Risk Factors section in our most recent quarterly report on Form 10-Q, our Annual Report on Form 10-K and our 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 and EVP of Business Development. Our Chief Medical Officer, Adrian Quartel, will also be available for today's question-and-answer session. Now it's my pleasure to hand the call over to Neil. Story Continues Neil Mcfarlane Thank you, Nichol, and thank you for joining us this afternoon. As Nichol mentioned, today, we'll provide our fourth quarter and fiscal year 2024 financial results and share progress on the execution of our priorities for 2025. Our priorities are guided by Zevra's strategic plan unveiled during our third quarter call and categorized under four actionable pillars, commercial excellence, pipeline and innovation, talent and culture and corporate foundation. 2024 was a transformational year for Zevra and bringing MIPLYFFA to people living with Niemann-Pick Disease Type C, or NPC, was a crowning achievement. In the past year, we became a commercial stage company that had the opportunity to positively impact the lives of people living with rare diseases. The approval of MIPLYFFA last year was an outcome of our long-standing collaboration with the NPC community and included contributions from more than 270 Niemann-Pick Disease Type C patients who participated globally in our pivotal studies, open-label extension studies and Expanded Access Programs. The early days of the MIPLYFFA launch have exceeded our expectations. In the fourth quarter, we received 109 patient enrollment forms, which we believe reflected the anticipation and unmet need in the NPC community. Additionally, by the end of Q4, all active US EAP participants submitted an enrollment form, and our entire organization has been activated to ensure smooth transition to MIPLYFFA commercial product. As previously reported, we anticipate that the US EAP will close by the end of Q2. Going forward, our goal is to expand availability of MIPLYFFA outside of the US to provide access to as many people living with NPC as possible. With this goal in mind, we are progressing regulatory submissions with the focus on submitting a marketing authorization application in Europe during the second half of 2025, where we estimate 1,100 people are living with NPC. Additionally, we will maintain our global Expanded Access Program that currently supports 70 to 80 NPC patients, and we look forward to providing updates in the coming quarters as we seek to expand access to MIPLYFFA. We're leveraging our infrastructure for both urea cycle disorders or UCDs, and NPC, leading to efficiencies that drive commercial excellence. The launch of OLPRUVA in early 2024 facilitated key learnings as we built our commercial capabilities. During the fourth quarter, we received four OLPRUVA enrollments. As announced in our Q3 call, we refined our strategy to reach the adult-onset population for whom OLPRUVA's portability and ease of administration may provide a benefit with a focus on patients receiving payer pushback. Josh will provide more details related to the performance of our commercial products later in the call. Moving to pipeline and innovation, which refers to our ambition to develop products that address significant unmet need. Celiprolol is our Phase 3 investigational clinical candidate for the treatment of Vascular Ehlers-Danlos Syndrome, or VES, which is an inherited connective tissue disorder caused by the COL3A1 gene mutation that can result in spontaneous arterial aneurysms and hollow organ ruptures. There are currently no treatments approved in the US, where there are approximately 7,500 people living with this disease and the prevailing treatment paradigm is only reactive surgical interventions. Although celiprolol is not approved for VES in any country, it is utilized as a standard of care in several European countries. In Q4, we enrolled eight patients in our Phase 3 DiSCOVER trial, bringing the total number of enrolled patients to 27. As an event-driven trial, the rate of participant enrollment is integral to the program's success. As a result, we are investing in tactics to accelerate enrollment among genetically confirmed patients with providers and clinics that treat COL3A1 positive patients. In addition, we have support from the Vets Foundation Professional Advisory Board on our path forward. Moving on to KP1077; as outlined on our Q3 call and following a successful end of Phase 2 meeting with the FDA, we are exploring strategic alternatives to advance clinical development and future commercialization. We are focused on maximizing the value of this Phase 3-ready asset that has regulatory feedback for the potential development in both idiopathic hypersomnia and narcolepsy indications. In 2024, we completed a comprehensive review of our IP portfolio to identify areas for optimization. This review led to a decision to rationalize our in-house discovery efforts and outsource those activities to focus our investments where we have the greatest potential for impact. To execute our strategic plan and deliver on our key priorities, it's paramount that we develop a high-performing culture and attract and retain talent. In the second quarter of 2024, we expanded our executive leadership team with the appointment of Rahsaan Thompson as Chief Legal Officer, Secretary and Compliance Officer; and Alison Peters as Chief People Officer. Additionally, during the fourth quarter, we consolidated our development and scientific functions under Adrian Quartel, our Chief Medical Officer. We believe these changes optimize Zevra for success with continuity of leadership and the appropriate span of control to achieve our objectives. An important element of executing against our strategy is to build and maintain our position of financial strength and disciplined capital allocation that we refer to as our corporate foundation. This underpins all other pillars and allows us to responsibly invest in our long-term transformation. In 2024, we refinanced existing debt with a new credit facility and completed a modest secondary offering, which provided financial stability. Recently, we entered into an agreement to monetize our Priority Review Voucher for $150 million, which upon closing will provide non-dilutive capital to support our future growth. Building on the momentum from 2024, we are pleased to start 2025 from a position of strength across our four strategic pillars, execute on the tremendous opportunity we have to serve people living with rare diseases. I'll now turn the call over to Josh, who will provide an update on our commercial products. Joshua Schafer Thank you, Neil. 2024 was a year of significant milestones for Zevra and the rare disease community. In particular, the FDA's approval of MIPLYFFA marked the first product in the US for the treatment of NPC. At that time, we immediately began receiving patient enrollment forms and initiated the benefits verification process. During the fourth quarter, product was available in the last five weeks of the year. As a reminder, today and on future calls, while discussing our commercial products, we will provide patient enrollments, the percent of covered lives and net revenue for the quarter. As Neil noted, the launch of MIPLYFFA has gone exceptionally well and carries significant momentum into 2025. From the time of approval through December 31, we received 109 total prescription enrollment forms. And as of the end of the year, we had enrolled all of the active US EAP patients. As a reminder, an enrollment is a prescription submitted to our specialty pharmacy, initiating the benefits investigation process, leading to a decision on reimbursement and paid dispense, which is a 30-day supply of MIPLYFFA. In the fourth quarter, early enrollees may have received both an initial dispense and their first refill depending on the time of their enrollment. We estimate that there are approximately 900 people with NPC in the US, of whom an estimated 300 to 350 have been diagnosed. Put this in perspective, our fourth quarter performance resulted in approximately one-third of diagnosed patients submitted an enrollment to receive MIPLYFFA. NPC is a neurodegenerative progressive and fatal lysosomal storage disorder caused by lipid buildup leading to cell death and ultimately organ dysfunction in the spleen, liver and brain. Because symptom presentation and age of onset are heterogeneous, disease progression is primarily assessed using the only clinically validated endpoint, the Niemann-Pick Disease Type C Clinical Severity Score, including measurements of ambulation, fine motor, speech and swallow abilities. The data included on our label shows MIPLYFFA in combination with miglustat halted disease progression through 12 months, demonstrated by more than a 2-point improvement in patients receiving this combination compared to those receiving miglustat alone, where only a 1-point improvement is needed to demonstrate a clinically meaningful difference. In a disease like NPC, access to data covering a 12-month duration is critical to establishing treatments as disease modifying. Beyond the pivotal trial, MIPLYFFA's safety profile and long-term treatment effect have been evaluated in an open-label extension study and through multiple center EAPs with some patients having as many as five to seven years of experience on therapy. We are pleased with MIPLYFFA's data and its indication for use in combination with miglustat, and our market intelligence suggests that healthcare providers prefer a multi-therapeutic approach to treating this heterogeneous disease. Based on early market experience and feedback, we believe providers will use a disease-modifying treatment such as MIPLYFFA plus miglustat as the cornerstone of therapy and then potentially add therapies or supplements to provide symptomatic treatment if needed. Many commercial plans have not yet formalized reimbursement coverage, though it is too early in the launch to provide a meaningful market access and reimbursement update in terms of the percentage of covered lives. However, our team is actively engaged with payers, and we have been addressing coverage hurdles to gain reimbursement through either direct formulary coverage or via the medical exception process thus far. We will continue meeting with payers and presenting clinical data to support MIPLYFFA's use as the cornerstone of therapy for NPC, and we'll report progress in future calls. As we look forward to 2025, our focus will be to grow enrollments of currently diagnosed and untreated patients. We recognize the impact of prompt diagnosis and treatment to halt the progression of disease for people living with NPC and have invested in educational resources and tactics to drive patient identification and early intervention. As an example, we commenced a media campaign working with local outlets and key opinion leaders to educate about early signs and symptoms of NPC. This program led to the identification of three new patients who had not been previously treated. We're also using advanced analytics to understand where recently diagnosed NPC patients are treated and to ensure new patients have an opportunity to receive treatment. Our rare disease sales and reimbursement specialists are actively engaged in raising awareness of NPC diagnosis and available treatments with MIPLYFFA. Our efforts to increase disease awareness are multifaceted and on February 28 of this year, timed with Rare Disease Day, we launched a disease state awareness campaign entitled Learn NPC, Read Between the Science to educate health care professionals unfamiliar with NPC to support the identification and diagnosis of people living with NPC. In addition, we entered into an agreement with a leading diagnostic company to provide genetic testing options for individuals with suspected lysosomal storage disorders including NPC. We believe this program will lead to the identification of new patients and will allow us to support earlier diagnosis and treatment. Our sales and reimbursement teams regularly meet with prescribers to provide education on the strength of MIPLYFFA data and to support the reimbursement processes. Concurrently, under the direction of Adrian Quartel, our Chief Medical Officer, our medical liaisons are engaging with key opinion leaders and patient advocacy groups to build the body of evidence demonstrating the need to treat patients early to delayed progression and to position MIPLYFFA as the cornerstone of treatment. Additionally, our medical affairs team is prioritizing our publication strategy to further drive awareness of the need to treat NPC patients. For example, during the 21st Annual WORLD Symposium, Zevra presented eight posters, including new data assessing the safety and tolerability of MIPLYFFA in NPC patients 6 months to 24 months as evaluated in the pediatric substudy of the pivotal Phase 3 trial. In conclusion for MIPLYFFA, we're encouraged by the initial signs of the launch and are excited to provide future updates on our progress in reaching patients who can benefit the most. Now let's turn to OLPRUVA, our commercial product for the treatment of certain UCDs. UCDs are a group of rare inherited metabolic disorders caused by defect in one of the six enzymes or two transporters in the urea cycle, leading to an accumulation of ammonia. Elevated ammonia or hyperammonemia can be toxic leading to neurocognitive damage or even death. OLPRUVA is a nitrogen scavenger that removes excess ammonia, thus reducing harmful accumulation. The OLPRUVA launch has progressed slower than we would like for this differentiated product. During the fourth quarter, we saw new enrollments increase to four as compared to three in the third quarter, and our market access for OLPRUVA remains consistent at 76% of covered lives. Zevra launched OLPRUVA a little over a year ago into a mature UCD market. At that time, we were able to build initial demand with clinicians and patients looking for alternative therapies that could improve adherence and better control ammonia levels. However, limited patient pull-through and reimbursement support experienced early in the launch necessitated a change to improve the patient experience, and we transitioned to a new specialty pharmacy. We also refined our strategy to target specific patient segments who will receive the greatest benefit from OLPRUVA and who may face fewer reimbursement hurdles. We believe that the adult female OTC deficient carrier population as well as those UCD patients who may seek additional lifestyle independence would receive the greatest benefit from the ammonia control on-the-go that OLPRUVA provides. Another resource we have added includes field reimbursement managers who work with physician teams to assist with reimbursement challenges. We've also identified payer plans that have moved other therapies to the exclusion list and are working with payers to provide a smoother reimbursement process for OLPRUVA. These efforts are in early stage, and we look forward to reporting our progress in future calls. Finally, we are proud to highlight our sponsorship of the National Urea Cycle Disorders Foundation, Check Ammonia campaign to raise awareness of the critical importance of recognizing the signs and symptoms of hyperammonemia anemia and the need to treat early and persistently. Patients remain at the center of what we do and supporting the rare disease community through patient advocacy organizations like NUCDF is one of our highest priorities. I will now turn the call over to LaDuane to discuss the fourth quarter and fiscal year 2024 financial results. R. LaDuane 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 Annual Report on Form 10-K for more detailed information, which we intend to file within the next few days. In the fourth quarter of 2024, we reported net revenue of $12 million, which includes $10.1 million in MIPLYFFA revenue, $0.1 million in OLPRUVA revenue, $1.1 million in net reimbursements from the French EAP for arimoclomol and $0.7 million of royalties and other reimbursements under the AZSTARYS license. This was a record level of quarterly product revenue for Zevra. For our commercial products, MIPLYFFA and OLPRUVA, we recognize revenue when shipments are received by the specialty pharmacy. For MIPLYFFA, Q4 was a partial period and revenue included initial patient dispenses, refills for some early enrollees and target supply levels at the specialty pharmacy. French EAP reimbursements were reduced during Q4 due to a true-up in program access fees, and we continue to expect net reimbursements will continue to be approximately $2.1 million per quarter. Our operating expenses for the fourth quarter were $24.5 million, which includes noncash stock compensation expense of $4 million, of which $2.1 million was accelerated vesting expense related to severance arrangements and severance expenses of $1.6 million recognized within R&D expenses. R&D expenses for Q4 2024 were $8.4 million, which was a decrease of $3 million compared to Q4 2023 due primarily to a decrease in third-party costs upon completion of the KP1077 Phase 2 trial, offset in part by an increase in personnel-related costs. SG&A expenses were $16.1 million for Q4 2024, which was an increase of $1.4 million compared to $14.7 million for Q4 2023, due primarily to an increase in personnel-related costs as our full team was in place and actively engaged in commercial launch activities for the entire quarter. Net loss for the fourth quarter of 2024 was $35.7 million or $0.67 per basic and diluted share compared to $19.6 million or $0.51 per basic and diluted share for the same quarter a year ago. For our full year 2024 results, net revenue was $23.6 million, which included $10.1 million of MIPLYFFA net revenue, $0.1 million of OLPRUVA net revenue, $9.1 million in net reimbursements from the French EAP for arimoclomol and $4.3 million in royalties and other reimbursements under the AZSTARYS license agreement. Our full year operating expenses were $97 million, which includes $14.9 million of noncash stock compensation expense. R&D expenses were $42.1 million, which was an increase of $2.3 million compared to full year 2023 due to an increase of $6.3 million in personnel-related costs, offset by a decrease of $3.6 million in third-party costs, primarily as a result of the completion of the KP1077 Phase 2 trial. SG&A expenses were $54.9 million for 2024 compared to $34.3 million for 2023, and this reflects the commercial team fully in place and actively engaged in our launch activities. For fiscal year 2024, net loss was $105.5 million or $2.28 per basic and diluted share compared to net loss of $46 million or $1.30 per basic and diluted share for 2023. As of December 31, 2024, total cash, cash equivalents and investments were $75.5 million, which was a decrease of $20 million, compared to the end of Q3 2024. Total long-term debt was approximately $60 million. And as announced on February 27, we have entered into an agreement to sell the Priority Review Voucher we received with the MIPLYFFA approval for $150 million. Upon closing, we expect to receive net proceeds of approximately $148.3 million net of fees, and our pro forma balance of cash, cash equivalents and investments will be approximately $223.8 million. This non-dilutive capital adds a significant strength to execute on our strategic priorities with our immediate focus on executing the commercial launches of MIPLYFFA and OLPRUVA, investments in celiprolol and our other development programs and building our corporate foundation while we earn the right to make disciplined growth investments in the future. Based on our execution to-date, our Form 10-K will not include a Going Concern disclosure and our current operating forecast, combined with existing resources, extends our cash runway into 2029. Our cash run rate guidance does not include the potential proceeds from the sale of the PRV, but it does include anticipated net revenue from MIPLYFFA and OLPRUVA sales, net reimbursements from the French EAP for arimoclomol, royalties under the AZSTARYS license agreement and continued investments into our development pipeline programs. Our financial results for 2024 reflect the solid execution of our strategic plan and demonstrates our commitment to our mission to serve patients and generate meaningful value for shareholders. We are pleased with the opportunities we have in 2025 and beyond to drive value creation through disciplined investments where we can win. Now, let's open the call to questions. Operator? Question and Answer Session Operator (Operator Instructions) Kristen Kluska, Cantor. Kristen Kluska Congrats on a really strong start out of the gate, especially around the holidays, I think, speaks to how much these patients really wanted that therapy. So congratulations. A few questions from me. I think this is the first time that you've provided us more specific guidance on the European filing timelines. So can you give us a sense of the last dialogue that you've had with the agency there and the plan? And then when we think about the European market, which is more mature partially due to the fact that miglustat has been there for over a decade. How do you think that drug becoming available and use for patients and how that identified more patients over time can translate to some of the efforts in the US now that you have the approval there? Neil Mcfarlane Thanks, Kristen, and thanks for the comments. It's been a great 2024 and transformational for sure. But as we mentioned in the remarks, the prepared remarks, bringing MIPLYFFA to patients with Niemann-Pick C is definitely a crowning achievement. Let me quickly touch base on a little bit of the market dynamics for Europe. And then I'll ask Adrian to talk a little bit about -- briefly about our regulatory interactions. In Europe, we see about 1,100 patients in Europe based on a prevalence number and miglustat has been approved there for well over a decade. So our current label in the US, and we are still working through what this filing would look like for our MAA in Europe, as you know, have MIPLYFFA and miglustat in combination in order to be able to halt the progression of the disease versus miglustat alone having an approximate 2-point progression over 12 months based on our clinical study. So based on the fact that there has been miglustat in the market, we believe the European market is more mature. There are more patients diagnosed and treated because of decades' worth of work that has been done. And now bringing a product that has the potential in Europe under the MAA filing to actually halt the progression of the disease based on the label that we have and the data we had in our Phase 3 program, we feel like this is a great opportunity to go after in Europe. In regards to the European filing, probably not ready to talk too much about all of the intricacies of the European filing, but to be able to state that the second half of 2025, we believe that we're going to be able to file. Adrian, do you want to talk a little bit about our last interactions? Adrian Quartel Yeah. So the last interaction with the EMA obviously happened via Orphazyme about four years ago when we review the application based upon the complete response letter that we received from the FDA and some of the comments that we received from the EMA in regards to the filing that we had in Europe. We're now obviously in a much better position with long-term data available. We are currently consulting with couple of consultancy group to how to specifically address some of the (technical difficulty) that they had during the original filing. And as I said, we are planning to file in the second half of this year, and we're confident that the filing will be accepted by the EMA. Kristen Kluska Okay. And then thanks for providing the cash runway guidance, which I understand excludes the PRV for now. But can you just maybe high level, give us a sense of like how conservative are you being with launch projections into this? Does this include if you do get an approval in Europe, adding that territory there? Just any high level would be really helpful. Neil Mcfarlane Yeah, of course. Yes, we did not include the PRV proceeds to yourself. And I would just say we have been conservative as we looked at that cash runway. We have to continue executing and continue into '25 with the plans that Josh and the team have got in place. And so that's definitely where our focus is. But yeah, it is safe to call that a conservative -- appropriate conservative estimate. Operator Jason Butler, Citizens JMP. Jason Butler Let me add my congrats on the quarter as well. Great start to the MIPLYFFA launch. Can you maybe give us any more quantitative color on the MIPLYFFA sales during the quarter in terms of the supply amounts to the specialty pharmacies or what proportion of patients got a refill? And then my second question is, how should we think about the launch from here, in terms of net patient adds? You've obviously successfully converted the EAP patients, but should we expect consistent steady net patient adds, or could it be more volatile from month-to-month? Neil Mcfarlane Yeah. Jason, I'm going to see if I can parse that to LaDuane to be able to answer the original questions around inventory, and we're not going to get into specifics, but he'll be able to answer that. And then we'll hand it off to Josh to be able to talk a little bit about this bolus of patients that we've seen and what the future may look like. LaDuane? R. LaDuane Clifton Yeah. So with MIPLYFFA revenue, again, we reported $10.1 million in Q4. That would have included product to handle the initial patient dispenses. Some early enrollees may have received a second refill late in December. And then we did allow for an appropriate level of stock to continue to service patients. It's actually at the target level that we would expect to maintain at this point. And so Josh, I'll turn it to you. Joshua Schafer Yeah, Jason, your question about new patients going forward. I think it's really important for us to kind of reflect on how quickly we were able to get to 109 patients and how quickly we were able specifically to convert those patients who were in our Expanded Access Program to enroll into MIPLYFFA. And so we had all of those patients plus some patients who had previously not been exposed to MIPLYFFA all within the fourth quarter. And so we were really pleased with how quickly we were able to do that. I think it speaks to the demand that there is in the marketplace. I think it also speaks to the execution of the team. And as we look forward into 2025, our focus now is really to be able to find those patients who have been diagnosed but aren't currently treated, as well as those patients who have not yet been diagnosed. And so that's really going to be the focus as we continue into this year. Operator Sumant Kulkarni, Canaccord. Sumant Kulkarni My first one is a bit of a follow-up on the last question. In terms of your sequential delta and patient enrollment forms from MIPLYFFA, that was 90. What are the key variables that we should keep in mind that might influence this trajectory as we look to the next quarter versus this one? Joshua Schafer So you're absolutely right. We went from 90 at the end of October to 109 at the end of December. Again, this really reflects many of these patients were really sort of queued up ready-to-go from our Expanded Access Program. As we move forward into this quarter, as I just mentioned, we're really focused on trying to expand beyond those Expanded Access sites to where we know that these patients are being treated, have been diagnosed. And we're going to continue to deploy our sales teams into those offices. As we continue to on each of these quarters, we'll be providing enrollment numbers, percent of covered lives and net revenue. And you can expect that we'll continue to do that as we move forward into subsequent quarters. Neil Mcfarlane Sumant, it's Neil here. I think it's important as we try to answer this question with a follow-up also to the previous analyst questions as well. When we talked about the transition of our EAP patients, traditionally, we would see that over about a 12-month period. And the team, based on all of the work they did prior to launch -- into launch and then a combination have been able to exceed every expectation we had internally on the conversion of those EAP patients, not over a 12-month period, but actually over a one-quarter period. And I think that's a true testament, as we mentioned previously, to the built-up demand and as well as the unmet need in this patient population. So as we go through this trajectory, and it has been a rocket ship trajectory, we expect that over the next phase of launch for us to be able to then take this momentum into Q1, into Q2, executing on all of the tactics and the phased approach that Josh had mentioned in his prepared remarks. So I think there's an important perspective here for us to be able to show that this has been an outstanding performance on the conversion and on the launch trajectory. We now are going to take this into executing Phase 2. Sumant Kulkarni That's a point well made and taken. So on the 300 to 350 or so diagnosed and treated patients with NPC, roughly how many are treated at Centers of Excellence versus not? Joshua Schafer Yeah. Many of these patients have some diagnosis that takes place at these Centers of Excellence. But keep in mind, there had previously been no treatments approved for Niemann-Pick. So many of these patients would be diagnosed and then go back to their local neurologists to continue being monitored. We're finding now that those patients are coming back to these Centers of Excellence as they're made aware of MIPLYFFA being approved. And so the vast majority of them have some connection, either being treated at the Centers of Excellence or some sort of referral to the Centers of Excellence. Sumant Kulkarni And last one before I hop into the queue back. On OLPRUVA, roughly how many quarters are you giving yourselves with the new patient targeting strategy to see a potential inflection? Joshua Schafer Yeah. We haven't really put any timeframe on that. We know that OLPRUVA can provide benefit to patients. We have revised our strategy to be able to bring that to specific patients who will benefit from the portability and the personalized dose, and these are the adult patients. And in particular, those where there's some reimbursement challenges. So we're continuing to monitor this. It's early in kind of assessing the impact of that. But we also know that there's great synergy within our sales team, within our broader commercial team and having both of these products. It's opening a lot of doors for MIPLYFFA. It's allowing us to foster relationships across the entire commercial spectrum. So we're watching this very closely, and we're allowing some time to see the impact of this change in our strategy. Operator Oren Livnat, H.C. Wainwright. Oren Livnat I have a couple on MIPLYFFA. Can you just characterize that initial bolus of 90 patient referrals that came in really fast in that first month? Did you get the lion's share of NPC patients in a concentrated group of centers. And so now you're more gradually expanding to a broader set of treaters and centers to penetrate those or was that 90 including, I guess, just a fraction of the patients at a broader set of sites, if you understand my question. Just trying to figure out like what the heavy lifting is here going forward? And also, I think on the last call, you had mentioned that 30% of those initial 90 had already been approved, which is really fast in the first month, either I assume via some formal coverage or more likely medical exemptions. Do you have any updates on those percentages for the initial wave, continuing approval rates or the cumulative approval rate for this entire 109 through December? And I do have follow-up. Joshua Schafer Yeah. So I'll take your question about the 90. So as we reported in our last call, of those 90, 69 of them were from the Expanded Access Program and 21 were from other sites that were naive to MIPLYFFA or had not previously received MIPLYFFA. Majority of those patients were still treated within one of those Centers of Excellence. As we've moved forward now to 109, we've gotten the remainder of those EAP patients. We have also expanded beyond the Centers of Excellence to find other prescribers and patients, and we're going to continue to do that as we move into this next phase of launch. Your second question was around the percent of patients that have received authorization for payment. And at the time of our last call, it was 30%. We're continuing to see improvement in that. And what we're seeing is that many commercial payers have not yet made decisions around policy, but the vast majority of our patients are able to get reimbursed either through direct formulary or through a medical exception process. Our team is out in front of these payers talking about the clinical benefits of MIPLYFFA, sharing with them the new data that's been generated since the approval. And so we expect that's going to continue to improve as we move down the launch. Oren Livnat And as you have these reimbursement conversations, can you just maybe give us more color on how that's going, if there's any pushback, it's obviously ultra rare. So I would assume that's somewhat limited. But obviously, we're talking about both two expensive drugs in combination. So just maybe a little more color there. And are you able to give us a sense of what your, I guess, average net dollar per patient per year expectation is going forward? Joshua Schafer So in terms of the conversations that we're having with payers right now, we are really getting in there and we're talking about the benefits that MIPLYFFA offers to these patients. We're talking about the halting of progression of disease after 12 months. We are the only product that has been approved with -- based on a clinically meaningful endpoint and the $2.1 million improvement in that endpoint. And so that's really where the conversations are taking us, and we're continuing to drive that message home. And as I mentioned, most of these patients are getting covered either through formulary or through some sort of medical exception process, and that's really continuing. So that's really the tone. A lot of additional doors are being opened for continued clinical conversations. As it relates to the net pricing, I'm going to turn it over to LaDuane here. R. LaDuane Clifton Yeah. Oren, I would just say we don't generally discuss or comment too much on the gross to net details. But at this point in time, I would say our strategy is still underway in terms of how we get the data story in front of payers. It's not clear yet if we need to even be that aggressive in that concept. So I think it's early days there, but I expect that gross to net is going to be we're going to have -- I'm sorry, gross to net is going to be in a solid place, and I don't expect we'll be very aggressive there at this point. Operator Sami Corwin, William Blair. Sami Corwin Congrats on the great quarter. I guess I was curious what the average time you're kind of seeing from the start enrollment form to the approval of reimbursement. And then as you're kind of thinking about expanding your targeted population and going beyond the 300 to 350 patients that are currently seeking treatment, I guess, to what extent do you think you can further expand that and penetrate into the estimated 900 prevalent population? Neil Mcfarlane Hey Sami, it's Neil. I'm going to kick this one off because I think we're really pleased so far with what we've seen, not just in those patients who are diagnosed, but in our prepared remarks, we talked about the fact that one of the areas we've been investing has been media. And those three patients that we talked about were actually patients who were diagnosed but not treated. And this gets back down to this expanding the market population. And we believe that as we continue to get a treatment in the market and multiple treatments in the market for that matter, physicians that actually have NPC or have other diseases that will start to think about NPC in their diagnostic cascade in these rare diseases that will really allow us to be able to then increase that number from the 300 to 350 into 900. And then if Europe as a marketplace of the 1,100 patients from a prevalence perspective and the miglustat growth that's happened over a decade plus, we think it's a great information -- it's great information for us to inform what our market possibility is in the US. So Doc, you want to talk a little bit about enrollment time to reimbursement and those things? Adrian Quartel Sure. And I was just going to add to this that as we mentioned in our prepared remarks, we've employed a number of other tactics to really identify these patients, including some collaboration with genetic testing, education around the Niemann-Pick, all of which is going to lead to some market expansion. In terms of the average time from enrollment to reimbursement, it's too early into the launch to be able to really give you specifics on that. But I would mention again that in the fourth quarter in those five weeks from the time that we shipped drug until the end of the year, we did have a number of patients who, not only received their first fill, but also refill in that period of time. So I think that really speaks to how quickly our team was able to get some of these patients reimbursed and reauthorized. And I anticipate that that's just going to continue as we work our way through those patients who are pending approval now. Sami Corwin Great. That's very helpful. And a quick follow-up. Have you had any patients that have submitting an enrollment form, but have been ultimately denied? And do you have any clarity as to what the rationale behind any ultimate denial is? Adrian Quartel Yeah. So of course, in any rare disease, there are some initial denials, and we work through those with patients to make sure that we can help them navigate and work through those denials through appeals and other parts of pathways through the medical exception pathway. There are -- we've only had a couple of patients who have been denied. But again, we continue to support those patients. We're continuing to help them work through the reimbursement challenges. And any patient who is experienced in those challenges, we have a very robust patient services plan and patient assistance plan so that no patient would go without drug. Operator Eddie Hickman, Guggenheim Securities. Eddie Hickman I'll add my congratulations on a really nice quarter. What can you tell us about how we should model discontinuation rate so far? And then going forward, do you expect the rate to meaningfully change as we get further outside of the EAP? And then of this sort of current bolus of patients, like how many of them do you expect to retain sort of in the long term? Joshua Schafer Yeah. Thanks for that question. I think it's way too early in the launch to be able to address or answer that question. Neil Mcfarlane Yeah. Eddie, maybe I'll give you a comp though that you can actually think about. As we look at our EAP data and we think about the patients and the continuity, we had patients that remained in our EAP five years and patients went through OLE to EAP now on the commercial product five, six, seven years. You heard that also in the open public forum during our advisory committee. So what we've seen and continue to see also in our European Expanded Access Program is a really high level of continuity of care once patients are on MIPLYFFA and miglustat. Again, we're just a few months in. We're reporting on five weeks of patient shipments in Q4. So it's going to be a little early to talk about discontinuation rates. But if history repeats itself, once patients are on with the side effect profile that MIPLYFFA and miglustat have, we see longevity. Operator I'd now like to turn the call back over to Neil McFarlane for any closing remarks, please. Neil Mcfarlane Thank you, operator. Thanks for joining us today to review what was a transformational 2024. We're pleased to start 2025 from a position of strength and look forward to executing on our mission to serve people living with rare diseases. Have a great day. Operator Thank you. And this does conclude today's program. We thank you for your participation. You may disconnect at any time. View Comments Terms and Privacy Policy Privacy Dashboard More Info Recommended Stories
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