Zevra Therapeutics, Inc. Common Stock Canaccord Genuity's 46th Annual Growth Conference

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.

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 3 to 5 years, these three prongs on the stool that I discussed about starts 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.

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 regards 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.

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.