Zevra Therapeutics, Inc. Common Stock 12th Annual Cantor Fitzgerald Global Healthcare Conference

Good morning. Thanks for having us.

Great. Thank you. I will be making some forward-looking statements today, so please take a look at our most recent SEC filings for the most up-to-date information. Thank you for having us here right after Labor Day. It's a great way to kick off the fall. Zevra Therapeutics is a commercial-stage company that is really trying to find ways to redefine what's possible in bringing medicines to the rare disease community. We today have a commercial product in the U.S. for Niemann-Pick disease type C. We also have another product in the U.S. that we don't promote, called OLPRUVA, for urea cycle disorders. Then we have our European and global expansion opportunities that we're looking at for Niemann-Pick disease type C within a regulatory phase right now, along with geographic expansion to new markets to be able to take care of NPC patients.

We have a rare disease program in vascular Ehlers-Danlos syndrome. That is in phase III in the U.S. as well. All of that being said, we are well-capitalized to be able to execute on those three priorities, we call it the three legs on the stool, as we are moving forward, and looking forward to the rest of the conversation today. Tell you all about it.

Yeah, it has been a really interesting ride. The opportunity to come to a company that was leaning in to becoming a commercial-stage rare disease company, with my track record, having done that a few times. The unmet need for patients in NPC in the U.S. versus Europe, and I will talk a little bit about that in a moment, not having an approved product in the U.S. versus having an approved product in Europe, a much more mature market in Europe versus the U.S., led me to believe that, number one, the clinical data was fantastic, both being clinically meaningful and statistical significance in bringing a product to market, but also the unmet need was huge.

The opportunity then for us to be able to take a look at this market and say, okay, there is about 900 patients from a prevalence perspective, but the 300 to 350 patients that we understand have been diagnosed through the ICD-9 and 10 codes and have a code is actually quite smaller than that of Europe, where you see about 1,100 patients in Europe from a prevalence perspective. But the majority of those patients, or a large majority of those patients, have been diagnosed, found, and on therapy because there was an approved therapy in the market in Europe for a decade plus. When we think about the unmet need, the clinical significance, and the opportunity to bring a product in, for me, three years ago, it was a dream.

How could we come in and penetrate the market and really do all the things that I hope we'll talk about some more today that we've been doing commercially to be able to identify patients early, to be able to offer a therapeutic product that has the potential to halt the progression of a devastating disease, and now based on long-term data that we've now published over the last few years, solidifying ourselves as a foundational therapy that we can then move and continue to penetrate the market.

Yeah. Three years ago today, in our second year of launch, could I have imagined that of the 300 to 350 identified patients that we'd have as of the end of Q2, 184 patient enrollment forms and penetrated that percentage of the market? I would say no. I would say that would've been a dream. But today, what we've seen in our efforts around earlier diagnosis, what we've seen in our efforts to getting newly diagnosed patients added really is now allowing us to push the envelope to say, this market is somewhere between the 350 and 900 patients because it's early. We launched the product in the market in the end of November 2024. We're not even two years into the market and have really done a wonderful job of making the product available. I think there's also an element of this that we are learning as we're going.

We did not recognize the fact, and I don't think that anybody recognized the fact that there were so many adults. We're identifying patients in their 20s, 30s, 40s, 50s, even a 60-year-old that's a newly diagnosed patient. That is a market understanding that we didn't know. So we thought of this as a childhood disease, and now we see about 50% of our patients that are coming in are adults versus kids. So that learning, we continue to learn, and now we got to figure out how to be able to then take that learning and expand the market to get closer to the 900 and the 350.

Yeah. As I mentioned, the learnings that we've had over the last two years, and we're learning every day. Every prescription enrollment form that comes in, we're actually trying to get as much of an understanding of the patient's journey, not just to therapy, but to diagnosis. That learning then allows us to be able to put into perspective what it is that we would like to be able to invest in to get more diagnosis. When I think about what we're learning, the wins that we're having off of what we're learning, I would tell you a minimum and maximum. I think the minimum today is that 350. I think the maximum is probably the 900. At this phase, the newly diagnosed patients and the adult patient expansion has given us belief that it's definitely more than the 350.