Zevra Therapeutics, Inc. Common Stock Morgan Stanley 24th Annual Global Healthcare Conference

Yeah. First of all, thank you for having us. We've had a great day today. We really appreciate the support of the Morgan Stanley. I will be making forward-looking statements, so I'd ask for everybody to take a look at our most recent SEC filings to ensure that you get the most up-to-date information. On behalf of Justin and the rest of the team, I really appreciate answering this question, because what we do in the rare disease world is clearly a challenging part of pharmaceutical development and bringing medicines to people who otherwise have no other opportunity to get medicines. Rare disease is not as unique as it once was 20 or 30 years ago, but what we do hasn't changed much. We're trying to develop medicines and redefine how we get them to patients to then impact their lives.

For us, we're doing that in a commercial sense today, primarily with commercial programs in Niemann-Pick disease type C, ultra-orphan, along with UCD. But in addition to that, we're also expanding globally to patients outside of the U.S., and we have a phase III program also going in vascular Ehlers-Danlos syndrome. For us, it's ultra-orphan diseases. We're talking about prevalence rates of less than 1,000 for some of them and less than 7,500 for others. But I'm looking forward to getting into that more with you as we move forward. But it's a real privilege for us to be able to be working in the rare disease space and thinking differently about how we impact the lives of patients living with rare diseases.

Yeah. We have taken a little bit of a different tact. We are a commercial stage organization that has been a commercial stage organization for just a few years. Today we have a U.S. opportunity in MIPLYFFA in the Niemann-Pick disease type C space. About a 900 patient prevalent population in the U.S., about 300 to 350 of those patients. This is a commercial opportunity for us. We have been now at it for about six quarters in terms of the launch. We will get into some more of this detail later on. But in addition to that is one leg on the stool. The other leg on the stool for us is our global expansion. We are trying to be able to get MIPLYFFA into, or arimoclomol, I should say, into the Europe through an MAA process that we will talk about a little bit more as well.

Yeah. We are globally expanding into regions and territories where we can leverage our U.S. label into other markets.

Then we have our third leg on the stool, which is our pipeline, our phase III development program for vascular Ehlers-Danlos syndrome, which has enrolled about 66 patients out of 150 today, and we will talk a little bit more about that later on. But that is in a place for us today that we can really go to the agency and start to have some conversations around how do we accelerate the development. Zevra today is three legs on a stool. It is a U.S. opportunity that has a lot of untapped potential. It is a global expansion opportunity in our second leg on the stool, and thirdly, it is around celiprolol in the pipeline.

Yeah. Most excited about the learnings we are having. As I mentioned, 900 patient prevalent in the U.S. About 300 to 350 of those patients were diagnosed. Now the opportunity for us six quarters in, at the end of Q2, we had 184 prescription enrollment forms so far. That is an amazing impact that our team has been able to drive so far. With only 350 patients, getting 184 of those in six quarters is pretty good. That is primarily those patients. We also are even more excited today than we were a year ago because we have been learning. We have been learning, number one, that it is not only a pediatric disease anymore.

We've about 50% of our patients are adult patients. That learning is now informing how we try to get newly diagnosed patients into the mix. I'll give you an example. We have had patients that have been misdiagnosed with multiple sclerosis, adult patients that then have progressed with other symptoms that are more attuned to Niemann-Pick disease type C, get them genetic tested, and then they've eventually gotten on the product based on the fact that they had NPC, not MS. We've had a Wilson disease patient that has been recently published, actually about a quarter or two ago, that was misdiagnosed.

With this, we're learning, and in any rare disease, the more you learn about the journey of patients that are getting them to the ultimate diagnosis and then on treatment, it's better off that we can then tailor our patient-finding programs to being able to then get more newly diagnosed patients. If I look back at the last six quarters, a year ago today, I would not have told you that 50% of our patients would be adult patients. They used to coin Niemann-Pick disease type C as childhood Alzheimer's, and today we're understanding more, we're learning more, and now those newly diagnosed patients are giving us a lot more confidence that the market is somewhere between that 350 currently diagnosed patients and the newly diagnosed patients that quarter over quarter, we're seeing more of those now that we're learning.

That's been probably the biggest surprise for us, that there is real opportunity for growth beyond the 350. And that's very similar to Europe. In Europe today, there are about 1,100 patient prevalence, and the majority of those patients have been diagnosed and on some type of therapy. The reason for that is miglustat was originally approved well over a decade ago. Previous company went out and invested in disease state awareness and diagnostic modalities in publishing the suspicion index, and that market has been more mature over the last decade of identifying and diagnosing patients closer to the 1,100 patient population in Europe. We have a lot of confidence that if we can continue to invest in disease state awareness, continue to get diagnosis earlier, that we too will be able to continue to build newly diagnosed patients- Yep to get closer to 900 than 350.