Zevra Therapeutics, Inc. Common Stock H.C. Wainwright 28th Annual Global Investment Conference

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 3 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.

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 6 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.

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.