Good morning, everyone. I'm Sumant Kulkarni, a senior biotechnology analyst here at Canaccord Genuity, and it's my pleasure to have Zevra Therapeutics with us here today. Zevra, as you might know, has a product that's approved and on the market already for an ultra-rare disease called Niemann-Pick disease type C. The product is called MIPLYFFA, and it's been launched for some time now. We've had some interesting things back and forth with Europe as well, the European Medicines Agency there. We have a bunch of questions for you guys, and thanks for making it. For Zevra, we have CEO Neil McFarlane and CFO Justin Renz. Out there in the audience, we have Nichol Ochsner, who knows everything about the company as well. We'll keep this interactive. We do have a mic going around, so please feel free to raise your hands, and we'll get the mic across to you in case you have any questions. With that, I'll kick it off. Neil, can you set the stage a little bit on exactly what's happening with MIPLYFFA, the unmet need that that product serves, the Niemann-Pick disease type C community, and how it's received that, and what you've done so far with the product? Yeah. Thank you, Sumant, and thanks for having us. We'll be making some forward-looking statements, so take a look at our most recent SEC filings for the most up-to-date information. Yes, we had our earnings last week, and we ended up announcing a very good quarter. Again, we are three prongs on a stool here. We have MIPLYFFA in the U.S. that it remains our core focus on driving value for patients in combination with miglustat. We are also working through our geographic expansion strategy. We'll talk a little bit more about that in a moment, not only with our MAA process in Europe, which is going through a re-examination right now, but also in our market expansions that we're working on through named reimbursement and expanding access there. Lastly, our celiprolol program, where we are driving with engaging with the FDA in the second half of this year, after earlier this year engagement on how we can accelerate the development of this program for patients with Vascular Ehlers-Danlos Syndrome. Very proud of the execution in the first half of the year, and looking forward to taking some questions and having a conversation today. Thanks for that. You mentioned prongs on the stool. I am going to ask you to pick the highest priority prong right now. Is it MIPLYFFA in the U.S.? Is it the MIPLYFFA pending application or the resubmission that you have in to the EMA or something else entirely? Yeah, I am not sure. Clearly, our focus is on the U.S. MIPLYFFA business, but I do not know if there is one that is more important than the other today. All of them, I think, provide the opportunity for us to access meaningful therapeutics and potentially meaningful therapeutics to patients with rare diseases. From a standpoint of how we see the evolution of our business and the growth of our business in the coming three to five years, these three prongs on the stool that I discussed about start with executing in the U.S. for MIPLYFFA. The team has done a remarkable job so far. As you know, we had 184 prescription enrollment forms from launch to date in this marketplace, where I think we are starting to generate not only patients who were currently diagnosed but also continuing to unlock those newly diagnosed patients, which gives us that confidence that the market, in just a short period of time, is greater than the 300 to 350 patients that are currently diagnosed and more between that 350 and the 900 patients of a prevalent marketplace. We are pleased with what we are doing, but we are not stopping here. We are going to continue to lean into the future. Got it. This is a bit of a question related to that. Now that MIPLYFFA has been on the U.S. market for some time, what can you say is working in your efforts on the commercial side versus where you think the company could do better? Has the Niemann-Pick type C market surprised you in any way? Let me start with the last question, because I do think that the Niemann-Pick disease type C market has surprised us in one way, and it is in a good way. We for years have thought of Niemann-Pick disease type C as a childhood disease. We have always known that it had kind of four different phases, from infantile to an adult version of the disease. I think the biggest surprise has been the number of adult patients that we are seeing in our enrollments, as we have become more, getting more real-world data. Today when we. Actually, when we launched the product, our EAP was comprised of about 50% adults and 50% kids. Today, we have surpassed that number by well over 100, and we are continuing to see this adult population and child population continue to remain about 50/50. That is a big surprise. Number one, it is a big surprise because patients have been either misdiagnosed for a long period of time with something else, which we have seen. We talked about this on our earnings call. We have had some patients who had multiple sclerosis for many years, who then continued to progress in symptomatology that was not necessarily defined in multiple sclerosis patients, and they have been genetically tested and then had NPC. We have had some patients who had autism spectrum disorder but then progressed in other ways that you would normally not see in autism, that they then got genetic testing and came on board. Just recently, actually, there was an article published last month, or actually no, we are in August, June, and it talked about a case study around a patient who had Wilson disease for many years, who then had copper chelation and so on and so forth, but yet still progressed. They did genetic testing and came up with Niemann-Pick disease type C, an adult. This learning is a big surprise for us in regard to what we are seeing in the marketplace. Additionally, last week, we got our expanded access data published. That expanded access data had a very large cohort of adult patients for the first time, with some of them out to four years. That is a big surprise, but that also then reinforces the market and the potential for expansion of the TAM between that 350 to 900. The other tactics that we are doing, I cannot say there is one tactic that is the one thing that has been driving all the early new enrollments or one thing that has been really key to us educating physicians of patients that they have. It is really a multitude of factors. We are doing the disease state awareness. We are offering genetic testing. We are driving our publication efforts to make sure that that is out there. The clinical guidelines have recently been published, which really gives a lot of strength to diagnosing early, utilizing combination therapy, and those things. All of the pieces of the puzzle is really an integrated strategy that I think has been rising the tide and lifting the diagnosis and treatment of patients. Got it. Patient enrollment forms: this is a metric that investors are keenly focused on every quarter and maybe overly keenly. What can you do there that might make those more predictable for you and for us in the sense that you might start giving us what to expect on that? It is really challenging with the law of small numbers. We are talking about 900 patients in the U.S. from a prevalence perspective. I wish I had an answer that I could tell you that we are going to find X amount per quarter or per month. But in rare disease and in ultra-rare disease, once you know one patient, you know one patient. I think the diagnostic odyssey that patients have, the journey that they have in their disease, everyone is different. So it is not something where I could say, if I do X, I am going to see Y. We are doing a multitude of activities across the, I would like to say, the rare disease playbook. We will scale some up that are working and scale some down that may not be. But I think that the playbook of supporting patients and physicians for a new disease, we are starting to see those efforts pay off in the newly diagnosed patients and the expansion of the market. Got it. You mentioned roughly 180 or so cumulative enrollment forms. How representative is that of true patient share within 300 to 350 diagnosed and treated patients? 184 patients from [inaudible] to date. The majority of that represents patients who were already diagnosed in that 300 to 350. However, as I mentioned last year this time, we were starting to see newly diagnosed patients, kind of early in the launch, but newly diagnosed patients. We have seen newly diagnosed patients every quarter since then. That is what has given us the confidence that the market and the TAM is expanding beyond the 300 to 350. Actually, that is something that we have had communicated back to us with those folks who have got access to claims data. They are also seeing it; even with the lagging claims data, they are seeing the marketplace rise based on the activity. It is good. Now with guidelines and the efforts of our team and our medical team and everything else, it is really picking up. Just to clarify, the 180 or so, it corresponds to one new patient every time. 184 prescription enrollment forms comprises the majority of those comprised patients who were previously diagnosed. A smaller part of that is the newly diagnosed patients. Right. The 300 to 350: the majority of our 184 prescription enrollment forms are in that 300 to 350, with the newly diagnosed patients coming. Sure. It is 184 distinct patients, though. It is 184 distinct. There is no refill component or anything like that to an enrollment form. That is one prescription for one patient. Okay. Got it. At what point would you be willing to share what fraction would be newly diagnosed patients in the patient enrollment form number that you have? I think it's really too early for us to provide any guidance in that regard. I think it's important also to note that we have a very small population of a high impact product, but also a high value therapeutic that me guiding you with one patient difference is a significant and meaningful change in our numbers. So it is really hard for me to give you that right now until we get to a steady state. I don't know when that steady state is because our growth rate both in the U.S. on a quarter-over-quarter basis, as we were having this conversation last year this time, it was certain quarters were single-digit enrollments, certain quarters were double-digit enrollments, and very hard to navigate. But on a full year, we did about 52 enrollments last year. To date this year, although we had a single-digit enrollment, a double-digit enrollment, what we have now is 23 patients. So it's very challenging, I think, to go quarter by quarter, and the variability will continue as we continue to move forward. But the key here is the total market, addressable market. We're gaining confidence that it's bigger in our second year of launch. Got it. So on that gaining confidence note, I am going to press, but this is going to be a qualitative question. You can answer quantitatively if you would like, but I think you will not, so I will keep it qualitative. So now that you know that you have a certain number of newly diagnosed patients within those patient enrollment forms, do you feel better about the number of years it might take you to expand that market from 350 to 900 prevalent patients, or are you the same or worse compared to where you were last quarter? We are expanding that market now. Every newly enrolled patient above what was previously the running average of new enrollments, which we are exceeding today on an annual basis year-over-year means we are expanding that market. So we are expanding it now. In terms of quantitatively, I cannot answer that question. I tried. Oh, next time we should get chairs with cup holders. Yeah. No, it is okay. I guess you have these kind of green shoots that you see on newly diagnosed patients getting into the fold. What needs to happen from a larger or a wider viewpoint, I guess, to get more of those patients in? I know you have genetic testing going on. You have these AI models that you're using. Can you give us a flavor for what those might entail and what exactly needs to happen to get to that number? Well, like I said, it's an integrated strategy here. There's no home run. The opportunity to enroll patients earlier and get them diagnosed earlier and on treatment that allows for the halting and progression of this progressive and devastating disease—that's where we're focused on. So some of our efforts are to establishing the diagnostic earlier by educating the physicians and patient groups and everybody else. The other part of it is, as you mentioned, our bespoke AI model that we're utilizing EMR data along with claims data through the Suspicion Index of NPC to be able to hone in on our representatives who have got some of this data that now allows them to be more efficient, when they're in a call of a patient or of a doctor that they know has an NPC patient, can they start to expand in those areas? Our goal is to drive additional patients who are not yet diagnosed, and then have it in the back end, lower the barrier for genetic testing for physicians to be able to then have a test available to them at no cost if they need to outside of their own health system. So, I think it's through the journey, both the diagnostic journey and the journey of the patient with the disease to us to be able to look at this more integrated approach among all these things to help to rise the tide. I don't think that, like I said, I don't think there's one thing, but there is an element of this that is a playbook that we're following, and this is an important component. Miglustat was approved in Europe for NPC for well over a decade, and in Europe you have a prevalent number that's about 1,100 patients in Europe. And they have more population than the U.S., so you'd expect a little more. 900 in the U.S., 1,100 in Europe. But the disease state, the education, and the other things that have happened in Europe have led to a much more mature patient base that is closer to the prevalent number than where we are today in the U.S. So we're utilizing that playbook to get our numbers closer to the prevalent number. And we're seeing that in the market today. We're seeing that in the numbers. On that note, do you think your current spending on the level of education on NPC in the U.S. is appropriate given what your plans might be for the longer term? This may be a great opportunity for me to engage our CFO, who is next to me here. We have a regular push and pull with our Chief Commercial Officer, Josh, to find the best use of our treasure. We take shareholder money very seriously. We have regular dialogue on sizing of the team, efforts to invest. We are in the growth stage, when we have $260 million on our balance sheet as of June 30 with no debt. We have the ability to invest. To your point, we want to be very disciplined in that. We try to demonstrate that in our quarterly results. It is a regular source of discussion. We want to make sure that these investments that we are making in building out, because again, we are still in the second year of launch, so there is a lot of room to grow, and I think we are on the right track, but we are always open-minded. All right. So you have given us the number of diagnosed and treated patients. You have given us what the prevalent population is in the U.S. At what point will you be in a position to maybe provide us peak sales potential for the product? Yeah, I think that goes back to your guidance question around can we smooth the lumpiness of our quarterly enrollment. If we are able to get to a place where we could do that, I think it would behoove us to be able to provide that level of guidance. Today, I just can't. I would go back to the peak opportunity for us in the U.S. goes back to the patient numbers. That's that 350 - 900. A year ago, I didn't have as much confidence in where we were because we were one year into launch, and we were doing well and exceeding expectations. Today, I can firmly say and confirm that in the numbers, and you can fact-check this, that the growth of the market in the newly diagnosed patients is growing. That is what's given us confidence that the peak opportunity is now between that 350 and 900. Give us some more time and we'll let you know. We'll continue to execute on that. I just don't know how to answer that question in such a lumpy and myriad of approaches in such small numbers and rare disease. Right. The good news is the patients that come on MIPLYFFA stay on MIPLYFFA. You have a product here that targets clearly a really high unmet need in NPC. You mentioned to my first question that, or second question, I guess, you were pleasantly surprised about some of the older patients are coming into the fold now diagnosed. Your product is a weight-based dosing. For us financial types, what does that mean in terms of pricing on a weighted average, no pun intended, basis? Yeah. We provided some guidance on our initial call post-launch that of our expanded access program patients, which were about 83 patients at the time; the mix of those patients were 50% adults and 50% kids. That guided us, even though we have multiple strengths and it's weight-based dosing to be able to provide the $85,000 WAC per month. Per month. Per patient. That has remained, plus or minus a few hundred dollars here or there. That has remained consistent. When it comes to our current patient base, which is again, we've brought on board as many children as adults. That is consistent. I think until we see a significant switch between adults and children, which we have not seen, and I have to say we anticipated more children than adults at this point, then we'll provide different guidance at that time. Got it. In the U.S., the label for MIPLYFFA includes a combo therapy with miglustat, which is the off-label standard of care. Are there any initiatives underway at the company to potentially use MIPLYFFA as a monotherapy? The answer to your question is, you are correct. The label is in combination with miglustat. We are very happy with our label, and it is becoming more apparent that our label is part of the strength of the disease-modifying therapies that physicians have at their fingertips now. It became even more apparent with the clinical guidelines that came out last quarter, which basically state, diagnose as early as possible, treat as early as possible, and use combination therapy and disease-modifying combination therapy. In those guidelines, we are the only product that has combination therapy in the label with randomized clinical trial data that shows plus or minus MIPLYFFA with miglustat. That data along with what we are seeing in the real world, where physicians have been embracing combination therapy, we think that the future of the Niemann-Pick disease treatment paradigm is going to follow the guidelines. Which is treat with as many disease-modifying products as you can for the specific patients and utilize the different mechanisms of action to be able to clear cholesterol as best you can. That gives people the best chance for lacking of the progression of the disease. That has played out pre-launch. It has played out now in the real-world evidence we see, and now it has played out in the clinical guidelines. We see the strength in our label, actually, that is in combination because that is where the future is, and we are the only product that has randomized controlled data to show that. Right. In the competitive landscape, it is you, there is combination use with miglustat, and there is a product from IntraBio, AQNEURSA, and then there is a couple of players who are either near approval or have phase III data that is coming. That is Beren and Rafael/Cyclo. How do you expect this to play out? Do you expect to compete for payer dollars? Are all these complementary? How is the Niemann-Pick type C market going to play out from a competitive standpoint? Well, one, as a company that is focused on patients and driving best impact for patients, the more therapies in the NPC space, the better. Right. And as the guidelines state, utilizing multiple mechanisms, I think, is great. We've also seen that the investment that's come from these other players have also helped to rise the tide, right? More investment in disease state awareness, more investment in genetic testing, more investment in physician awareness. It's been great, and we hope that more products can come in. The other part of this is that our understanding with some of these that you mentioned are they are actually going after a part of our label we don't have. It's the less than two years old. And it would be wonderful if we could have patients who can have access to therapy earlier, and hopefully halt or slow the progression of this disease, and give those patients an opportunity to have multiple modalities like a MIPLYFFA and miglustat in their later lives to help them move forward. We see this as beneficial for patients. I think the guidelines have clearly stated that utilize multiple modalities and for the best outcomes. Got it. Moving on to the application in Europe. You got the negative opinion from the European Medicines Agency. You've submitted a request for re-examination. We've had a recent case where a product for a rare disease, trofinetide for Rett syndrome, there was a reversal of a vote. Clearly circumstances are different, but what gives you confidence that the EMA will reverse course on your pending application now? Yeah. Our confidence has remained consistent, and it has to do with the unmet need, and recognized unmet need, as well as the totality of the clinical evidence that we have. I stressed on our earnings call, and thank you for the questions on the earnings call. I stressed on the earnings call, and I'll do it again here today. What we have in our global EAP patients and the continuous request for MIPLYFFA, and the majority of those EAP patients are in Europe, continues to show the pull for an unmet need for a product that's not even registered, right? We're utilizing that in Europe, mostly compassionate use outside of France, which has a specific program, and that gives us a lot of confidence, right? That we are trying to fulfill a need. The clinical guidelines have also kind of reinforced that in Europe as well. The other part of this is that we have a clinically significant and statistically significant clinical trial. That's now been bolstered with both real-world evidence on the open-label extension data going out four years. Last week, the publication of our EAP data going out four years. The Pediatric Investigational Plan data, that's also now been published. We feel like that robust totality all points in the same direction, which is halting the progression of this disease. Between those two, we're hopeful now as we go and have requested our re-examination, we will now have a higher emphasis on those specific areas of concern that the agency has come up with now that allows for the voices of experts in those areas to come to the table and to bring our story to life. Right. That's what we're doing here with the re-examination process. As you also saw in that opinion, we've checked a lot of the boxes that were not checked in the previous application. We've moved the needle. Now we have to continue to lean in on the totality of our evidence and demonstrate the unmet need. Well, we have a couple of minutes here, so I'll be remiss if I don't ask you any questions on celiprolol. I've pointed that out as flying under the radar. I don't want to contribute to that myself. So celiprolol for Vascular Ehlers-Danlos Syndrome. It's the only product in phase III that we know of. There's no product approved specifically for vEDS in the U.S. How are you thinking about what you can do to get that product to patients sooner, given the nature of that trial? Well, we are doing everything. We have invested significantly in the acceleration of the enrollment, and just last month, we were at a regional meeting in Texas and driving through that as well. We're investing in all of the clinical operations and trial enhancement activities you can think of. We also, though, took the opportunity in the first half of this year to go to the agency and to talk to them about the fact that we have lower event rates and the enrollment, it's not like the screening is not there. We're screening, but the enrollment's and the conversion of that, lower than we'd like. But more importantly, how can we also work with them to find ways to accelerate the clinical development of the program through new regulatory mechanisms? We had an informative discussion. We have now gone back out based on that, and they gave us some homework. We've been doing the homework, and we're on track to be able to reengage with the agency in the second half of this year to be able to have further dialogue around those two areas, accelerate enrollment and/or accelerating the development of the program. The last one, I'm going to squeeze this in for Justin. You're one of those rare biopharma companies that don't really need to raise capital, but you may want to. Given that luxury, how are you thinking about business development? What kinds of ultra-rare disease assets are there? What might fit, what might not? Those kinds of things. Yeah, great question. We have really built the infrastructure around MIPLYFFA in the U.S., and of course, modest investments in Europe, but very targeted and specific and be milestone driven. We very much, with our business development team, look for complementary assets. We'd look for an ultra-rare disease, either late stage or commercial, that could complement the MIPLYFFA team because we have the infrastructure where we could truly add a product to our offerings at these centers of excellence, calling on the same doctors, and so we are very specific in our look. It has to be the right price, the right drug, the right fit, and so that is absolutely part of our inorganic growth strategy that we look to. Again, we have the flexibility with our capital structure to look, but again, our focus is on MIPLYFFA in the U.S., Europe, and celiprolol 1.2.3 with the inorganic growth strategy on top of that. Okay. Thank you. With all our time, thank you everyone for coming.