Good morning, everyone. My name is Brandon Folkes. I'm one of the equity research analysts here at H.C. Wainwright, and thank you very much for joining us at our global investment conference. Next up, we have a fireside discussion with Zevra Therapeutics, and joining me from Zevra is CEO Neil McFarlane and CFO Justin Renz. Neil, Justin, thank you very much. Good morning. Good morning. Neil, maybe you can just set the scene for us about Zevra. There's obviously been tremendous progress over the last 12 months. Can you just update us on that progress over the last 12 months, where Zevra is today, and where the company could be in maybe 24 months from here? Yeah. That's great. Thank you for having us. We really look forward to a productive day today. I will be making some forward-looking statements, so I'd ask you to please take a look at our most recent SEC filings for the most up-to-date information. Yeah, we've had a tremendous transformation over the last number of years, as you mentioned. Today we're a commercial stage organization that is commercializing products for ultra-rare diseases in the U.S. We have a global expansion ongoing with our expansion of MIPLYFFA, or arimoclomol, I should say, in Europe and expansion throughout the rest of the world as well. Then we have a phase III program ongoing with celiprolol. I know we'll get into some more of this for vascular Ehlers-Danlos syndrome. But these, we call it three legs on the stool, but it's really right now being focused our efforts into the U.S. business, and the upside when it comes to Europe as well as celiprolol in the U.S. Fantastic. I'm going to start with MIPLYFFA in the U.S., just given the success you've had there. What have you learned during the launch, and what have you learned during the launch that gives you confidence that you are expanding that diagnosed population? Yeah. When you think about the ultra rare disease space, we are learning every day, and every patient's journey is a journey to getting a diagnosis, much less than understanding what your treatment options are, and then how you're able to be able to impact the disease course. For us, in six quarters since launch, what we've learned is twofold. One is every patient is its own patient in terms of the diagnosis and the journey they go through. The other is that we've learned that this disease, Niemann-Pick disease type C, was originally kind of thought of as childhood Alzheimer's. Today we're diagnosing de novo patients in their 20s, 30s, 40s, and it's also an adult disease. So about 50% of the patients that we've got, and as of the end of Q2 this year, we had 184 prescription enrollment forms. Just to remind you, we're talking about 300 to 350 patients in the United States that were diagnosed at the time of our approval, and then about 900 from a prevalence perspective. What we've learned in the last six quarters is, number one, that this is an equal weighting disease so far. We're learning more about that. Adults that have been misdiagnosed or quite frankly not yet diagnosed. The other is that we're learning that we're actually able to increase the diagnosis rates of newly diagnosed patients on top of those that were there. Our confidence from when we started the launch of this program to today around what the ultimate TAM is a lot higher today than it was when we started. That 350 to the 900 TAM, we believe is absolutely the sweet spot. It's not anything like 200 patients or whatever we had heard when we first launched the program. A lot of confidence there. Fantastic. We've touched on patient identification, right? That's really just the first step in any ultra-orphan disease, right? Can you just talk about what you've learned and also the progress in terms of identifying a suspected patient and ultimately getting that patient onto paid therapy of MIPLYFFA. How has that evolved through the launch? Right. With any launch of ultra therapeutic, and high value therapeutic, you have to do a lot of education. Most physicians and most payers have never heard of Niemann-Pick disease type C. As we have launched the program, what we've also had the benefit of is long-term data supportive of what we saw in our clinical program. We've now published, since launch, our four-year open label extension data, our four-year EAP data. In addition to that, some of the pediatric data that we had been working on in our pediatric sub-study. That is providing a lot of opportunity for us to educate both the payer community who has never heard of NPC, and provide them not just with a package insert or your clinical trial data that you did, but also four-year longitudinal data that supports the efforts that we saw on a clinical benefit and safety of the program. That has been really helpful. I would also say that in six quarters, what we have also learned is, once you get a payer up to speed, the next time they see a patient, it is not as daunting of a task for them to get up to speed on what NPC is, what the payer landscape, what the benefit is and so on and so forth. That we are able to get some of those patients into CoverMyMeds and there you get a diagnosis, you get a prescription enrollment form, and that thing can ship in 48 hours. On the other side, you had a payer who has never heard of NPC, and you are educating, you are going through the prior authorizations, you are going through the medical exception process, and our medical folks are in there with our payer teams, educating that payer for the first time. We expect that we are going to have to continue that effort over the next through the launch period. As new payers see NPC for the first time, we are going to have to double down and do it again. It is a little bit of the learning of how to do it more efficiently, but also the learning of once you shared the robustness of the data, and now we package that with clinical guidelines that were updated that came in Q2, that showed genetically test early. It shows get patients onto therapy as early as you can. Utilize combination therapy, which is what we have been saying, multiple modality to allow for you to have decreased the progression of the disease as early as you can. That is the kind of education that we are going to have to continue doing with the payer community and with patients and physicians. Fantastic. I would be remiss not to ask about enrollment forms, right? I know it has been a big focus. That said, whilst the investor community focuses a lot on enrollment forms, what are some of the metrics you focus on internally that gives you confidence that the team is doing a great job to get that enrollment form, right? Because it is a lot of work that goes into getting an enrollment form. Well, it's a great question. We publicly talk about enrollment forms because that's meaningful, right? Once a physician takes that education to then write the prescription form, our team's done a great job, but it doesn't stop there. It's about pull-through of that to make sure that the patient then gets on therapy. And it's the white glove service that we utilize when it comes to our patient services, that we have to do to have patients continue the refill rates, which are very high that we see, and they don't discontinue because of something we could do. We have very low discontinuation rates. So that investment continues in that regard. Internally, I think we look at a lot of different things. We want to be able to make sure that we're continuing to invest in ways to get patients that have never had a diagnosis, diagnosed. We're investing in earlier testing. We're investing in disease state awareness to be able to accelerate the education across the physician network. We're also investing in peer-to-peer activity. You can imagine that with such a ultra-rare disease, that a physician who might actually get to a genetic test that is NPC may never have actually ever seen an NPC patient. We see that time and time again. Then we have what we've invested in called the Expert Connect, where a physician where we've identified and they've put an NPC patient that's got diagnosed, we get them in connected with a physician who's very familiar at one of the centers of excellence or somebody who's had multiple patients that they've treated for NPC. Because you can imagine you can become an expert with one patient that you've treated and taken care of for a long period of time. We're trying to get those folks access to experts that then allows them to put good treatment plans together for these patients. Internally, we really want to be able to see the tide continue to rise to get the newly diagnosed patients. That will allow us to be able to get that TAM closer to the 900, or should I say penetrate closer to the 900 than the 350 that we know are available today. Fantastic. You talked about the success you had growing the market and the undiagnosed population as well as the success you've had in adult population where many of us thought this was a childhood disease. If we think about getting from where we are today in terms of diagnosed to the 900, 950 patient population, do you have a sense of how much of the undiagnosed ahead is going to be driven by the adults coming on therapy versus pediatrics? What a great question. I do not know the answer to that question. Again, this gets back to an organization that is learning every day. We are trying to make sure that we pilot certain activities and certain investments that we put thresholds of what success looks like, and if it works, we will double down. If it does not work, we will kill it. I think that kind of continuous learning and curiosity that our team and the organization is doing is allowing us to learn about things like the adult patient population. I will give you an example. We are also trying to do this disease suspicion index work of NPC that was published a while ago, many years ago, along with claims data that you see the ICD-9 or ICD-10 NPC codes. Then we are bringing in EMR data, along with that, and we are trying to understand what are the common patterns. Then also where could there be specific connections, and we are doing some machine learning to then figure out how do we then get our reps in a place where if we are focused on trying to bring through a NPC patient in territory X or in city Y, they can go see physicians who have patients that could potentially look like NPC patients based on the suspicion index of NPC to educate those physicians. We have seen some success with that because it is not boiling the ocean and trying to get your reps to go do everything, but it is focusing our reps and our medical affairs professionals to go into these areas and be more directed at where we suspect there may be NPC patients based on this overlapping, in a specific zip code or at a specific physician's office that may have a patient like that. For us, those investments will allow for the tide to rise and for us to be able to think about how we can execute the undiagnosed patient and get them diagnosed. The other thing we are doing is, in the adult population, which is new, and again, I am pretty confident that the 50 + patients we published in one of our recent papers is the largest cohort of adults ever published. We are trying to understand that learning too. We have done some of this work also to say, Okay, what are some of the commonalities of these patients? Because they have all individual journeys, but then how do we then put them, and overlap them to see? One of the things we saw recently was a large portion of the newly diagnosed adult patients also have a cataplexy ICD-10 code. Now, we have to then figure out, well, what does that mean? Is there more to this story that we want to understand? It has clearly been defined in the suspicion index. You can have cataplexy, but of the adult patients that we see that. Now, we have got to go figure out how we can attack that. The team is again, learning every day. To me, that is how we are going to create a great environment to continue to diagnose newly diagnosed patients. As a company, you've done a lot of really good work helping patients get identified and get on treatment for NPC. As we think about other entrants coming into the NPC market, other therapies, how does that help you and help the patients, especially just given the guidelines reinforce combination therapy? I firmly believe that having more options for patients at varying stages of their development, whether it be infantile or childhood or adult, that it will rise the opportunity in a very small number of patients in the U.S. The more voices we can have to raise that awareness, the more investment we can have to be able to have genetic testing early and decrease the barriers to that is, the better off the community will be. The more we will diagnose patients closer to the 900 than the 350. So it's a win-win. Fantastic. I do want to shift focus to Europe quickly. You've obviously got the re-examination coming up there. Can you just walk us through what's different about this re-examination and what gives you confidence of a positive opinion? Yeah. The data and the guidelines and what we've just talked about around the strength and robustness of our data is what remains to give us the confidence in a successful outcome. That being said, we are in the same regulatory process, and now we are in this re-examination phase of the process to actually address the outstanding regulatory issues. We have gotten two new rapporteurs. They will be independent in their analysis of the issue that's at the table today. We will present our dossier to them around the specific issues to bring to life the data that we have around these issues. For us, we don't know what the outcome is going to be, but we remain confident that the data and the strength of the data, along with the clinical guidelines, both of our randomized control trial as well as the expanded access open label extension in our pediatric study, that there's a real benefit for patients in Europe. I think the other thing that gives us maybe not a sign of success with the regulators, but gives us a lot of confidence that we have a product that is meaningful, is we continue to get inbounds from the European countries that we have expanded access in today, even post the opinion from the CHMP. We have a lot of strength in numbers in that regard. Fantastic. I do want to also just touch on celiprolol. Another sort of very interesting opportunity. Can you just talk about what you learned from your initial FDA interaction and how we should think about that program going forward and the options on the table to bring that to market? Yeah. We are developing celiprolol for vascular Ehlers-Danlos syndrome. We have 66 patients enrolled out of 150 patients at the end of Q2 this year. We started to disclose event rates back a couple of quarters ago. We started with one, we have two, now we have three at the end of Q2. That's not enough event rates at a pace that we felt comfortable. So we went to the agency the first part of the year and said, Hey, here's where we are. Here's all the information that we have, and we'd like to explore solutions to accelerate the clinical development and/or regulatory pathways to be able to get the product to patients sooner. That's the conversation we had. I can't really get into a lot of details at this point in the game, but it was a productive conversation and instructive for us to go back and do some homework. In the second half of this year, we're on target to reengage with that homework and understand if there is a path to be able to accelerate the development and get this product to patients in the U.S. Fantastic. Justin, I do want to bring you in here. You obviously have a very strong balance sheet. It is a testament to a lot of things, including the commercial execution. With all these opportunities you and I have discussed, how do we think about capital allocation, both internally and potentially externally, just given that strength? Yeah. Thank you. In the moment, we are really focused on the three legs of the stool that Neil and you have spoken on, the U.S. execution, the global opportunity, and celiprolol. Part of our balance sheet has been solidified by work Neil and the team did earlier this year where we monetized some non-core assets and turned that around and used those proceeds to repay some debt. That really, again, further strengthened our balance sheet and got rid of some double-digit interest rate debt. Now we are generating some cash while looking at how we can deploy it in the future. So we always look at various opportunities. We are very focused on running a lean, mean, rare disease machine, and we are making great progress. So lots to look forward to later this year. Fantastic. Neil. Maybe I will just expand on that a little bit. I know we have 2 minutes left here. It sounds like $260 million is a lot of resources. We are also looking at the opportunity on these three areas we are focused on where if we see investment opportunity to accelerate our U.S. diagnosis or the opportunity to get to 900 much sooner than what we saw in Europe based on the work that was done over a decade plus of investing. There is an 1,100 patient population addressable prevalence in Europe. The majority of those patients were diagnosed. They were diagnosed from investment and a product that was available. If we find ways to be able to do it faster, smarter in the U.S., we have the capital to invest. Likewise, with success in Europe, we have the opportunity to make the right call as to whether we do it ourselves or we partner. Our go-to-market strategy is open today for the reason that we need to understand what the label is and where we go. There is also outside of Europe markets that we are penetrating today, too. We have to have the capital to do that, to be able to invest in those markets and understand how we can then make smart calls and c eliprolol. First world problem is for us to be able to have celiprolol with success, we would be behind because we have been very disciplined on when and how we invest in that program. Success means that we have got to put quite a lot of resources to work to be able to execute on that opportunity. We are being very disciplined, understanding that there are trade-offs to the decisions we make, but under Justin's leadership and I think the team that is focused on investing with return on that investment, we have got that discipline that is necessary to create value for shareholders. Fantastic. One more question from me. Obviously, we focus so much on the commercial end result of the U.S., but I think one thing that goes unnoticed is you put out a lot of good data on MIPLYFFA over the past year, a lot of incrementally very, I think, meaningful data. Is that something we should continue to keep an eye on and see more of it, just given your sort of length of therapy these patients have been on and your wealth of data you could generate from it? Yeah, no, we are absolutely continuing to learn and then applying those investments into data that will allow both physicians, payers, and the NPC community to understand how best to use MIPLYFFA moving forward. So yeah, no, you should absolutely expect us to continue to invest. That comes on the back of the Orange Book listing that took place, that gets our intellectual property out to 2041 now. This is exactly the kind of investment that a leading rare disease company will do, but also somebody who has a keen interest in making sure that we can continue to advance science and patient care for NPC. Fantastic. Yeah, the NPC clinical guidelines were just recently updated earlier this year. They hadn't been updated in years, plural, and really, they encouraged all to treat as early and with many common different modalities as possible. It's great for the community, and we want to be part of that community to really raise awareness and treat as many patients as we can. Yeah. No, I think you're leading the community, so thank you for what you do for patients, but also thank you for joining me today. Thank you. Thank you very much.