Zevra Therapeutics, Inc. Q3 FY2024 Earnings Call

Good afternoon, and thank you for joining Zevra Therapeutics Third Quarter 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, please go ahead.

Thank you, and welcome to those who are joining us. Today, we will provide an overview of our accomplishments in the third quarter and recent weeks, 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 the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied by any forward-looking statements due to risks and uncertainties associated with 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 projections made. These forward-looking statements should be evaluated together with the cautionary statements contained in the Risk Factors section of our most recent quarterly report on Form 10-Q, our annual report on Form 10-K and other filings with the SEC. I am pleased to welcome Zevra's management team members participating in today's call. Neil McFarlane, Zevra's President and Chief Executive Officer; LaDuane Clifton, our Chief Financial Officer; and Josh Schafer, our Chief Commercial Officer and EVP of Business Development. Also joining us today for the question-and-answer session is Adrian Quartel, our Chief Medical Officer. Now it's my pleasure to hand the call over to Neil.

Thank you, Nichol, and thank you for joining us this afternoon. On the call today, we will provide an update on the company's progress towards achieving our 2024 goals, review the milestones achieved in the third quarter and share with you our priorities going forward. I would like to start by reflecting on the progress we made over the last few quarters with a statement I made in the annual shareholder letter earlier this year. I mentioned how we must execute as one Zevra, focus our resources on high-impact opportunities and drive innovation by investing in programs that clearly address unmet medical needs. Thus far, the pace of Zevra's transformation has been tremendous. And while we celebrate our success, we know there is more to do to achieve our ultimate goals. Today, I'm pleased to report on our progress in the third quarter, which was rich with milestones. Our crowning achievement was the approval and launch of MIPLYFFA, the first FDA-approved therapy for the treatment of Niemann-Pick disease Type C or NPC. This accomplishment was an outcome of our close collaboration with the NPC community and we are delighted to celebrate this victory with them. The launch of MIPLYFFA has exceeded our expectations with 90 prescription enrollment forms submitted as of October 31. This is largely due to the extensive prelaunch preparations and the deep relationships we have built with NPC prescribers. Additionally, we expect to have MIPLYFFA available for patient shipment in line with our previous guidance. We also announced the receipt of a rare pediatric disease priority review voucher, or PRV, with the approval of MIPLYFFA, and we intend to monetize the voucher to support our future growth with non-dilutive capital. Let's turn to OLPRUVA, our commercial product for the treatment of certain urea cycle disorders or UCDs. Our launch has been impacted by low patient awareness and reimbursement hurdles for new products in the UCD market. With only three prescription enrollment forms received in the third quarter, the launch is underperforming and we are taking actions to remedy this. To better understand the challenges we face, we conducted a comprehensive external market and brand assessment that provided us with feedback from the UCD community. Based on this, we adjusted our focus to patient segments that we believe have the greatest potential to benefit from OLPRUVA and to revitalize the launch. Turning briefly to our pipeline. We achieved several clinical milestones in the quarter as well. We conducted an end of Phase II meeting with the FDA at the end of September and have clear direction for a Phase III path forward regarding 1077 in idiopathic hypersomnia, or IH. In addition, we met our goal of dosing new patients in the recently reinitiated Phase III program for celiprolol. I will talk more about these programs after Josh's update on the commercial launches. As we approach the end of the year, I can report that we have completed our portfolio assessment and developed our strategic plan for 2025. This plan builds on our many accomplishments while taking into consideration external market factors and our internal capabilities, along with an exhaustive evaluation of scenarios on how best to drive stakeholder value. Looking to the future, we have a plan to drive continued transformation and have organized our priorities around four key pillars that you'll hear me refer to today and on future calls: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation. To start, we must demonstrate commercial excellence through strong execution. Our business model is built to bring new therapies to highly targeted patients and healthcare professional communities. We will seek to maximize the potential of our current and future products by leveraging our expertise and infrastructure investment. One tangible outcome is our sense of urgency to make MIPLYFFA available globally with pursuit of a regulatory submission in Europe as our top priority. Pipeline and innovation refers to ensuring that we develop products that address a significant unmet need within the rare disease community. We conducted a comprehensive review of our pipeline assets and as a result, we've halted some development activities, most notably osanetant. We also made the difficult but necessary decision to discontinue our in-house pro-drug discovery activities and close our facilities in Iowa and Virginia, with the intent to outsource lab efforts for programs that move forward. Additionally, we are actively seeking to extract value from our legacy intellectual property portfolio through active outreach. With long-term success in mind, we will reprioritize those resources to late-stage programs that maximize our research and development capabilities, allowing us to expand our portfolio with complementary assets. Achieving great things for patients requires Zevra to develop great talent and culture. As the needs of our business and portfolio evolve, we will look to expand our internal capabilities relative to future growth. We can only achieve the ambitions of our strategic plan by shaping and engaging a high-performing team and living an unapologetically patient-centric culture. Our corporate foundation refers to financial strength and disciplined capital allocation to execute our key priorities. This underpins all other pillars, facilitates growth, and allows us to responsibly invest in our long-term transformation. Part of our future growth will be inorganic, and we plan to establish Zevra as a partner of choice for companies with late-stage development or commercial rare disease assets. Now I'll turn the call over to Josh, who will provide an update on our commercial launches for MIPLYFFA and OLPRUVA.

Thanks, Neil. We believe the FDA approval of MIPLYFFA was of monumental importance to those living with NPC, along with their family members, patient advocacy groups, and clinicians. It also marked an extraordinary milestone for Zevra as we advance our commercial efforts to bring new treatments for rare diseases. As Neil mentioned, we received 90 prescription enrollment forms as of October 31, exceeding our internal expectations. These enrollment forms came from both the patients who are in our U.S. expanded access program, or EAP, as well as patients who were not previously on MIPLYFFA. As a reminder, an enrollment is a prescription submitted to our specialty pharmacy that initiates the benefits investigation process, ultimately leading to a decision on reimbursement and paid dispenses. As I mentioned in our September call, a top launch priority is to convert EAP patients to MIPLYFFA. At the time of FDA approval, the program had grown to 83 patients, and as of the end of October, 69 of those patients had submitted an enrollment form. Given the speed of this transition, we anticipate closing the EAP program in the second quarter of next year, well ahead of the expected 12-month post-launch timeframe we previously reported, with our intent to leave no patient behind. Our patient services team has been working through the benefits investigation process for each submitted enrollment. As of the end of October, approximately 30% of the enrollment forms received have been approved for reimbursement and are ready for fulfillment upon drug availability in the channel, which will be within our previous guidance of eight to twelve weeks post-launch. We are pleased with the early progress regarding enrollments, and on future calls, we intend to provide metrics on market access and revenue. To recap the prevalence and market opportunity for NPC, estimates show that approximately 900 people in the U.S. are living with the disease. However, only about a third of this population, or between 300 and 350 patients, have been diagnosed and treated. Data show that of those treated, approximately 80% are currently receiving miglustat. And as a reminder, MIPLYFFA is approved and indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients two years of age and older. The robust demand that we've seen for MIPLYFFA is a direct response to the clinical benefits physicians and patients have experienced through our long-term EAP and open-label extension studies. We are confident that MIPLYFFA in combination with miglustat has the potential to become the cornerstone treatment for NPC. Our position is bolstered through physician feedback on the clinical data that supported the FDA approval of MIPLYFFA. That data showed that in our pivotal clinical trial, MIPLYFFA in combination with miglustat halted disease progression over 12 months. This was demonstrated by more than a 2-point improvement in patients receiving MIPLYFFA and miglustat compared to those receiving miglustat alone. These data were assessed using the rescored four-domain NPC clinical severity scale, which is the clinically relevant endpoint for evaluating treatment effects on NPC disease progression. Additionally, 41 patients from the pivotal trial were then enrolled in the open-label extension study, where 34 of the 41 were treated with MIPLYFFA for more than two years and 17 of those continued to be treated with MIPLYFFA for more than five years, demonstrating its long-term value. Adrian stated on our September conference call that a 1-point difference on this scale could mean the difference in one's ability to feed oneself versus the need for a caregiver, or it could mean the difference between walking on one's own versus the need for a wheelchair. To reiterate, in our study, the MIPLYFFA and miglustat combination saw a more than 2-point difference through the 12-month pivotal trial. It also showed that MIPLYFFA was well tolerated compared to placebo, and adverse events were generally of mild to moderate severity with very few leading to withdrawal of treatment. Wrapping up with MIPLYFFA, we are pleased with the early indicators of launch progress and look forward to sharing more details in future calls. Turning to OLPRUVA, as Neil mentioned, we have refined our strategy to raise patient awareness and improve payer pull-through. Although we have patients continuing on OLPRUVA, we saw new enrollments decrease to three in the third quarter from nine in the second quarter and revenue was de minimis. We know that certain patients struggle to remain compliant on therapy and there is a need to raise awareness of the symptoms of UCD and to treat this indication persistently. Another dynamic of the UCD market is that most patients are on nitrogen scavengers and receive annual paid authorizations. Those prior authorizations are being reviewed by payers as we approach the end of the year and the market leader is increasingly being moved to the exclusion list on many commercial insurance plans. Our market access for OLPRUVA is currently 76% of covered lives. And over the quarter, we've improved our formulary position on a number of accounts to preferred as payers look for a differentiated lower-cost treatment. Also in the quarter, we reassessed the market opportunity, considering OLPRUVA's position relative to other approved products. The outcome was a data-driven shift to target specific patient segments that will receive the greatest benefit from OLPRUVA and face fewer reimbursement hurdles. We focused our resources and promotional efforts in the third quarter to drive patient identification and to improve pull-through with payers. Specifically, we are targeting patients seeking greater lifestyle independence who could benefit from the ammonia control on the go that OLPRUVA offers. Our field team has been working with clinicians to identify appropriate patients across the various centers of excellence that we call on. We have also teamed with the National Urea Cycle Disorders Foundation to highlight the critical importance of recognizing the signs and symptoms of hyperammonemia and the need to treat early and persistently. As a reminder, there is a high overlap in clinicians who treat UCD and NPC. Our targeted efforts for OLPRUVA should allow us to maintain a share of voice in the UCD market while leveraging our field teams and other resources across our commercial portfolio to establish Zevra as a committed partner for those living with rare diseases. I will now turn the call back over to Neil to discuss our pipeline assets.

Thanks, Josh. Starting with KP1077, serdexmethylphenidate for the treatment of idiopathic hypersomnia, I'd like to remind you of the need and opportunity for new treatments for IH. This rare sleep disorder affects approximately 37,000 people in the U.S. and is characterized by excessive daytime sleepiness and difficulty waking, also known as sleep inertia, which is still an unmet need. Currently, there is only one FDA-approved treatment, and we believe the profile of KP1077 uniquely positions the product candidate to address the unmet need. At the end of the third quarter, we completed our end of Phase II meeting in which the FDA agreed that our proposed Phase III design could proceed and indicated that a single pivotal study with appropriate confirmatory evidence will be sufficient to submit an NDA. In parallel, we completed the business case evaluation for KP1077 within the context of our strategic plan. Based on the analysis, we have concluded that the appropriate path forward to maximize the value of KP1077 is to explore strategic alternatives to advance clinical development and future commercialization. Furthermore, while the KP1077 narcolepsy program was previously deprioritized to focus on IH, regulatory flexibility remains to develop the program for narcolepsy concurrently with IH. Turning to celiprolol, which is in late-stage clinical development for the treatment of Vascular Ehlers-Danlos Syndrome, or VEDS. There are approximately 7,500 people in the U.S. where there is no approved treatment. While celiprolol is approved in Europe for hypertension, it is commonly used off-label for VEDS. As previously reported in the second quarter, our decision to restart recruitment in the DISCOVER trial has been welcomed by people suffering from VEDS. Since we restarted recruitment in the second quarter, we met our goal of dosing patients in Q3, bringing the total number of patients enrolled at the end of the third quarter to 19. We continue to have significant interest from patients and physicians to participate in this decentralized trial, and in the future, we intend to report total patient enrollment. Additionally, we continue to build on our existing partnerships with patient advocacy groups as well as with major treatment centers and key opinion leaders. With this progress, we are refining the business case to inform the value proposition, and we intend to provide an update on our next call in early 2025. In summary, I'm proud of the collaborative effort of team Zevra that delivered the accomplishments in the third quarter and throughout 2024. Still, there's more work for us to do to realize our mission of becoming a leading rare disease company. I'm confident that we can get there with our strong commitment to execute, focus, and innovate. I'll now hand the call over to LaDuane, who will provide an update on our financial results.

Thank you, Neil, and good afternoon. While I will provide a detailed financial overview during today's call, I encourage you to review Zevra's quarterly report on Form 10-Q for more detailed information, which we intend to file post-market tomorrow, November 13, 2024. In the third quarter, we reported net revenue of $3.7 million, which includes $2.6 million in net reimbursements from the French EAP for arimoclomol and $1.1 million of royalties and other reimbursements under the AZSTARYS license. As a reminder, for our commercial products, MIPLYFFA and OLPRUVA, we recognize commercial product revenue when shipments are received by the specialty pharmacy. In the third quarter, net revenue for OLPRUVA was de minimis, and we expect to have our initial shipment for MIPLYFFA in Q4. Our R&D expenses for the third quarter were $10.9 million, which was an increase of 4% compared to the second quarter of this year. Selling, general, and administrative expenses were $16.2 million during the third quarter, representing an increase of 28.6% from the second quarter of this year. This includes a non-cash stock compensation expense of $6.1 million during the quarter, of which $2.5 million was performance-based vesting upon the approval of MIPLYFFA. Overall, this increase is in line with our projections as we transition to full commercial stage operations. The net loss for the third quarter was $33.2 million, or $0.69 per basic and diluted share, compared to $10.4 million, or $0.30 per basic and diluted share in the same quarter a year ago. In the third quarter, we completed an underwritten public offering of more than 10.6 million shares of our common stock at a price of $6.50 per share. The total net proceeds were approximately $64.5 million after deducting underwriting discounts, commissions and offering expenses. The proceeds from the offering support our commercial and clinical development operations with an emphasis on the launch activities for our commercial products and pipeline. As of September 30, total cash, cash equivalents, and investments were $95.5 million, which was an increase of $46.2 million compared to June 30 of this year. Use of cash during the period was $18.2 million. Total long-term debt was $58.9 million. Based on our current operating forecast, existing resources extend our cash runway into 2027, subject to continued compliance with debt covenants. Our cash runway guidance is based on our current operating plan, available cash, cash equivalents, and investments and includes revenue from MIPLYFFA sales, reimbursements from the French EAP, royalties under the AZSTARYS license agreement, and continued investments into our development pipeline programs. It does not include potential proceeds from a PRV sale. As we wind down the year, we are pleased with our strong financial footing and our continued ability to be capital efficient with our operations and deliver value to shareholders. As Neil said earlier, we are well on our way to becoming a leading patient-focused rare disease therapeutics company. Now we will open the call to questions.

Thank you, Mr. Clifton. We'll go first this afternoon to Jason Butler of Citizens JMP.

Hi. Thank you for the opportunity to ask questions and congratulations on all the progress made this quarter. I have a couple of questions regarding MIPLYFFA. Could you provide an overview of the remaining steps required for patients who have received reimbursement approval to have their medications shipped? Is everything in place from a manufacturing perspective? Are you prepared to distribute the drug? What are the next steps involved? Additionally, I understand it's early, but it appears you've been able to secure reimbursement approvals quickly. As more new patients enter the process who are not part of the EAP, how should we anticipate the average time from submitting the prescription form to receiving reimbursement approval? Thank you.

Thank you for the question, Jason. I will address the first part regarding manufacturing and our drug and channel timeframe, which we estimated as eight to twelve weeks post-approval. We are progressing well in terms of getting the drug into the channel and believe the eight to twelve week timeframe is still accurate. We are approaching the 8-week mark by the end of the month and we are prepared to move forward with the product. There are no additional obstacles to getting the drug into the channel, aside from logistics. Now, I'll let Josh discuss the next steps for the 30% of the 90 patients enrolled as we move ahead.

Jason, just to remind you, we currently have 90 enrollments. Your question was specifically about the reimbursement process for patients who are not in the EAP program. Out of those 90, only 69 are from the EAP program, which means there are some enrolled patients who are not part of the EAP program and have not previously been exposed to MIPLYFFA. We are addressing each of those enrollments regardless of whether the patient was in the EAP program or naïve to MIPLYFFA. We do not see any additional challenges in obtaining reimbursement, whether the patient was involved with our EAP or not.

Okay. Great. Thanks for taking the questions and congrats again on all the progress.

Thank you. We go next now to Louise Chen of Cantor.

Hi. Congratulations on the progress, and thanks for taking my questions here. So just an additional question on MIPLYFFA. How do we think about modeling the sales in the fourth quarter of '24? Are all 27 of those patients actually getting a product, or is that going to be staged into the following quarter? And then for the EU, you talked about expanding the product there. What are your next steps and timing here? And then the last question here is on your PRV. Any sense of the timing and value of what that's worth? Thank you.

Thank you, Louise. I will look into getting these started and see if LaDuane has anything to add. Regarding our sales projections for Q4, as mentioned earlier, we anticipate our first shipment of the drug to our specialty pharmacy, which will generate revenue in Q4. To answer Jason's previous question, we expect to ship product to patients soon after the drug reaches the specialty pharmacy, based on the 30% of benefits investigations approved as of October 31. These approvals are finalized, and we expect this number to increase as we continue the benefits investigation process for the remaining 90 enrolled patients and additional enrollments in the coming month. Concerning the EU timing, it is a priority for us, and we have conducted an extensive portfolio review. We have made tough decisions to reallocate resources toward areas that can deliver value for both patients and shareholders. The EU remains a key project; however, I cannot provide exact timing as we are in the midst of compiling the comprehensive data we received from the FDA and are working on discussions with the EMEA. We hope to share more on the timing early next year. Regarding the PRV, your question is quite relevant. We've observed that several PRVs have recently sold for significantly higher prices than the previous approximate $100 million, with two recent transactions in the $150 million range. We are actively monitoring the PRV market and aim to monetize the PRV to bring in non-dilutive capital to our balance sheet. LaDuane, do you have anything to add?

I would just say that the recent offering that we completed during this quarter provided the flexibility we have now to watch the market and find the right time to monetize.

Thank you.

Thank you. We go next now to Sami Corwin at William Blair.

Hey, there. Congrats on the progress this quarter, and thank you for taking our question. I was wondering if you could provide more details on the status of the remaining 60 patients that have started the enrollment forms. Are you just still working through those forms? Have there been any denials? And then could you provide any stats as to how many of those 90 patients who submitted to start enrollment forms that had already been on miglustat? Thank you.

I'll ask Josh to talk about it, but I think it's an important perspective, Sami, that the 90 enrollments were as of October 31. So that's not a final number for the quarter as of today. That's as of October 31, where we put a cutoff. I'll ask Josh to talk about the specifics.

Yeah. And to that point, as of October 31, we had been able to process and complete the benefits investigation process for a third or 30 of those 90. We continue to just work through those enrollments and we'll continue to do so. Our patient services team is working very diligently to ensure that all of those patients have the opportunity to hopefully get an approved shipment of MIPLYFFA. Your other question was how many of those are on miglustat. We know that the majority of the patients on our EAP, about 80% to 85% of them, are on miglustat. As they are going through the enrollment form, we're assuming that the same number of those patients are on miglustat.

Great. Thank you. And to clarify, you haven't seen any denials yet?

Sami, I'll take that question. You see denials and you also see the benefits investigation process in everything we do. That being said, we've been able to process 30% or approximately 30 patients so far and getting them to approval status. Some of those patients were denied originally and then gotten through to approval status. So we're working through the same specialty pharmacy benefits investigation that any specialty pharmacy product would go through. We have a high level of confidence, though, that 30% in a very short period of time of those patients have been approved that we’ll be successful in the remaining 90. And again, that was through October 31. We’ve continued to see enrollments through, but we wanted to be able to provide an early number for the Street at this point through October.

Got it. Appreciate the clarity.

Thank you. We'll go next now to Eddie Hickman at Guggenheim Securities.

Hey, good afternoon, and thanks for taking my questions. Congrats on the nice progress so far. Can you talk about miglustat use in the U.S. and what steps physicians currently have to take to get an NPC patient on that product and reimbursed and maybe compare that to what's required for an approved drug now like MIPLYFFA? What are the main hurdles to broadening that coverage? And then given your label, is increasing miglustat use a leading indicator for MIPLYFFA use? Are we seeing any spikes in prescriptions or payer claims that coincide with your ongoing launch? Thanks.

Thanks, Eddie. I think you're asking me to answer some questions that we don't have very good answers to. Your first question was in regards to the miglustat approval process; if I understood it correctly. I can't really opine on how physicians get miglustat. What I can tell you, though, is that those patients, so far in our EAP, about 80% of those patients were on miglustat. As you can see, of the 83 patients that left the EAP when it was closed, 69 of those patients have already had an enrollment form. Our enrollment form states, 'Here's the dose of MIPLYFFA. Have you been on miglustat or on miglustat and then were you in our EAP program?' Those patients who have had miglustat approved so far have also gotten MIPLYFFA approved in our early experience in the 30% of those enrollment forms that have processed so far. So I can't comment on specifically what the process is for miglustat, but I can tell you that those patients who were on or have been on miglustat are also getting approved through the benefits investigation process, and we've seen success so far. And this is very early, and we're pretty excited about the success we've had so far. The next question you asked me was in regards to if there is an increasing use of miglustat use and if that's a leading indicator. Again, miglustat is not approved in the U.S. for NPC and its use is approved for Gaucher's disease Type 1. It's highly genericized. There are a number of generic payers out there. So I think it's hard for us to say whether or not the total miglustat utilization is actually representative of a Niemann-Pick C disease type patient. So I hope I tried my best to answer your questions there. But tell me if you've got a follow-up that I can...

And I have a follow-up. Yeah. I guess a simpler way to ask maybe is, of the 20% of patients that are not on miglustat, how many of them aren't on it because they can't be on it or how many are on it because they just aren't on it yet? I'm wondering if we'll see that number increase at all now that the label includes that language.

Feedback from physicians and the experience that clinicians have had is most patients have been on miglustat at one point in their disease journey. What we've seen is that it really is dependent on side effects as well as payer approvals. So access for miglustat, again, has been about 80% of those patients we've had in our EAP that have been able to get it. But tolerability is also another one of those areas. So I hope that helps to answer your question.

Yeah. Appreciate it. Thank you.

Thank you. We now turn to Sumant Kulkarni at Canaccord.

Good afternoon. Thanks for taking my questions. On MIPLYFFA, you said that 69 of the patients that have prescription enrollment forms are in the expanded access program. But could you provide that ratio for the 30% of the 90 prescriptions that are approved for reimbursement? And what are you seeing in terms of competition from the IntraBio product that's also approved for NPC?

Thanks, Sumant. We have not really parsed the paid authorizations. We are going through them one by one. Of course we're very excited to see as many EAP patients come through as we have with nearly 70 of the 83. But we are not having any sort of differential processing of those, whether they come through the EAP or whether they're new to MIPLYFFA. So we're not really in a position to give those numbers. In regards to your question around what we're seeing with IntraBio, again, we're not commenting on what is going on with IntraBio. I think what is important is to really reiterate the differentiated data around MIPLYFFA and the fact that we are the only product out there with data that has demonstrated the halting of disease progression through 12 months. We've demonstrated a 2-point improvement on a score which is used to measure disease modification, the rescored four-domain Niemann-Pick severity score. We've got long-term data that shows that patients have seen clinical benefits up to five years through our EAP and open-label study. So that's really what we're focused on, is our data and the great success in what we're seeing already with the enrollments that we've seen to date.

Got it. And on 1077, is there a preference for the types of strategic alternatives you might be contemplating?

Sumant, I'll address that question. We examined our potential to create value with a single pivotal trial, while also exploring a narcolepsy indication. The data from our Phase II study has informed our business case significantly. It provides insight into what it would take to successfully commercialize this program and the necessary infrastructure to expand our reach effectively with a larger organization and a unique product that addresses an unmet need. This is quite different from our current focus. I can't specify which strategic alternatives we might pursue, but we recognize that partnering could help us determine the best fit for this opportunity.

Got it. And then my last question is on celiprolol. Given there aren't really too many alternatives or even products in trials for VEDS, how would you characterize the demand from a patient perspective there?

So, Sumant, this has been one of the big surprises in 2024 for us. The excitement around the ability to screen patients and what we heard prior to reestablishing and restarting the trial, we couldn't really tell whether or not that would translate into enrollments. What we've seen is that with no approved treatments for VEDS in the U.S. and that prevalence, we see in about 7,500 patients, for us, this is an opportunity to reestablish enrollment and see if we can actually now target what enrollment time frames we might be looking at to be able to then think about our interim analysis and our final analysis and trying to deliver then that commercially. This is an important data point for us in regards to restarting the trial, getting our first few patients enrolled in Q3. Now it's really up for us in Q4 to understand what that ramp looks like and then early next year, to be able to get back to the market to let them know what we believe the time frame is and how we move towards unlocking the value. But our early indicators are strong that we’ve seen these patients and the desire to get into the trial is quite high. Now we’re working through that to refine the case.

Thank you.

Thank you. We take our next question now from Oren Livnat at H.C. Wainwright.

Thanks. I appreciate that you're open to various sources for my clinical patients outside of the EAP program. I'm really interested in your perspective on the level of awareness and enthusiasm surrounding the treatment outside of EAP, as that represents the majority of patients receiving treatment. If that awareness is already significant, it would be a very encouraging sign. Can you share any insights on that? Additionally, regarding the ramp-up, I understand you're not providing guidance at this time. However, given your visibility into the limited and concentrated patient population, do you have any plans to offer guidance for next year's sales in early 2025? I also have a follow-up question.

Thanks, Oren. We are not planning to provide guidance during the early stages of our launch, but we believe it's significant to highlight our expectations for the product in the upcoming quarters. We estimate there are around 900 patients in the U.S., with approximately 300 of these patients identified and diagnosed. The current ramp we are experiencing is a positive indicator for our ability to pursue not only those who have been diagnosed but also the additional patients needed to reach the 900-patient target. Often in rare diseases, once a therapy is available, it tends to become more prominent in the minds of physicians as they consider their differential diagnoses. This will help us in our educational efforts and in raising awareness for NPC, ultimately attracting the additional 300 to 350 patients who could benefit from MIPLYFFA. I'll have Josh elaborate on the ramp and the market awareness he has been observing.

Yeah. So as you know, we've been engaged with the patient advocacy community for some time. I think that long-standing relationship is really helping to heighten the awareness of MIPLYFFA. In addition, our sales team has been out in the market interacting with many of these prescribers through their connections and calling on them for OLPRUVA. One of the benefits and strategic rationale for having these two products is that there is a high degree of overlap in the call points and the prescribing physicians. Awareness is quite high with patient advocacy and our MSL activities with thought leaders as well as all the work that we're doing in the offices to really build awareness not only for OLPRUVA, but also just around disease awareness to help identify new patients who might be diagnosed with NPC.

All right. And did I hear or did I understand you correctly that this increase in SG&A already this quarter, is that a pretty good indication of a fully loaded rightsized investment rate for this launch or is that going to keep ramping up into the fourth quarter and beyond with this launch?

LaDuane, do you want to answer that one?

Yeah. Thank you, Oren. I think that it does reflect our full commercial activities and patient services being in place. But it is important to note that it included non-cash stock compensation of $6.1 million, which included a one-time vesting of about $2.5 million, which was performance-based on the approval of MIPLYFFA. So that inflated the number just a little bit there. So with that adjustment, it does reflect our full infrastructure in place.

Okay. Lastly, regarding OLPRUVA, I appreciate your transparency. Can you tell us how many patients are currently receiving the therapy, regardless of whether they are paying for it? Are we not seeing any revenue because some patients are not reimbursed, or are you possibly dealing with inherited inventory? To keep it straightforward, many of us are focused on MIPLYFFA. Should we assume that revenue from OLPRUVA will be minimal until further notice?

Oren, can you ask that last part of the question; and I'm going to pass it off to Joshua. Can you ask the last part of the question? We lost that last one.

In response to your first question, we have several patients who are still receiving active treatment, but we cannot disclose those numbers at this time. I want to remind you that we recognize revenue when shipments are received at the specialty pharmacy, which doesn't always directly correlate with the number of patients undergoing ongoing therapy due to variability. Our commitment to OLPRUVA remains strong. As mentioned in our prepared remarks, we have refined our strategy to target patients who will benefit the most while also working diligently to enhance our formulary positions with various commercial plans to drive revenue and demand, ensuring that as many patients as possible can benefit. This is our current approach, and we will provide updates in future quarters.

Yeah. All right. I appreciate it. Good luck with the launch.

Thank you. I’d like to thank all of our stakeholders for helping us get to this point and for your continued commitment. We’re at the beginning of the next phase of growth, and our team is ready to execute, focus, and innovate to create value for patients and shareholders. We’re looking forward to updating you on our next call. Thank you very much. Have a good day.

Thank you. And again, everyone, that does conclude today's conference call. Again, thanks so much for joining us, and we wish you all a great remainder of your day. Goodbye.