Zevra Therapeutics, Inc.

Good afternoon and thank you for joining Zevra's first quarter 2025 financial results and corporate update conference call. Today's call is being recorded and will be available via the investor relations section of the company's website later today. The host for today's call is Nicole Oshner, Zevra's vice president. Please go ahead.

Thank you, and welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by a review of first quarter financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the investor section of our website. Before we begin the call, please note that certain information shared today will include forward-looking statements. actual results may differ materially from those stated or implied by any forward-looking statements due to risks and uncertainties associated with sever's business forward-looking statements are not promises or guarantees and are inherently subject to risks uncertainties and other important factors that may lead actual results differing materially from projections made These forward-looking statements should be evaluated together with the cautionary statements contained in the risk factors section in our most recently quarterly report on Form 10-Q, annual report on Form 10-K, and our other filings with the SEC. I am pleased to welcome ZEVR's management team members participating in today's call. Neal McFarlane, Zevers President and Chief Executive Officer, LaDwayne Clifton, our Chief Financial Officer, and Josh Schaefer, our Chief Commercial Officer. Our Chief Medical Officer, Adrian Cortell, will also be available for today's question and answer session. Now, it's my pleasure to turn the call over to Neal.

Thank you, Nicole, and thank you for joining us this afternoon to review our strong progress and the ongoing execution of our four strategic pillars. Let me start with commercial excellence. We are delivering on our two commercial launches to bring innovative therapies to people living with rare diseases. We have exceeded our internal expectations for the MyPipa launch and have enrolled over one-third of those diagnosed with Niemann-Pick disease type C, or NPC, in the United States. With Olpruva for this treatment of certain urea cycle disorders, or UCDs, we refined our marketing efforts to further increase awareness and access for patients and providers. Moving on to pipeline and innovation, we are focused on where our capabilities have the greatest impact for patients as we progress our development programs. Our Phase III DISCOVER trial of soliprolol Our late-stage asset for the potential treatment of vascular Ehlers-Danlos syndrome, or VEDS, which has received both orphan drug and breakthrough therapy designations, continues to progress with patient enrollments. Additionally, we continue assessing value-enhancing strategic alternatives for our Phase III Ready asset, KP-1077, for rare sleep disorders. These clinical-stage assets provide optionality and diversification to our portfolio. Turn it to talent and culture. We have a passionate, highly motivated, experienced, and mission-driven team focused on our vision to bring life-changing therapies to people living with rare diseases. Our team's strong execution is being acknowledged. For example, we earned a spot on Fast Company's top 10 most innovative companies list in the medicines, therapeutics, and pharmaceuticals category. Finally, corporate foundation. This underpins all other pillars and allows us to responsibly invest in our long-term transformation through disciplined capital allocation while maintaining our position of financial strength. With the recent monetization of our Pediatric Rare Disease Priority Review Voucher, or PRV, we added $148.3 million of non-dilutive capital to our balance sheet, bolstering our financial strength. With this, we have ample resources and financial flexibility to execute on our strategic priorities independent from the capital markets. Driving a little deeper into our commercial programs, we are pleased with the early success in the MyPlatform launch for the treatment of NPC, which is an ultra-rare neurodegenerative progressive and fatal disease. The launch is characterized by three unique patient cohorts. those who are participants of our Expanded Access Program, or EAP, those who are diagnosed and may or may not be receiving treatment, and finally, those who are undiagnosed and living with NPC for whom we seek to accelerate diagnosis and treatment. We received a total of 122 prescription enrollment forms from launch through March 31st, with 13 in the first quarter of 2025. Even in these early days of the launch, we have received prescription enrollment forms from all three patient cohorts, affirming the early impact of our commercial strategy. Notably, as we have previously reported, all active U.S. EAP participants have been enrolled to receive MyPlypha, and we have now closed the program, including locking the database and deactivating study centers ahead of our previously stated guidance. We are focused on providing MyPlypha to people living with NPC and have been educating prescribers and payers on the clinical benefit which demonstrates, in combination with Miglistat, a halting of disease progression through 12 months of treatment. Beyond the pivotal trial, MyPlypha's safety profile and the long-term treatment effect have been evaluated in open-label extension studies and through our multi-center EAP, with patients having as many as five to seven years of experience on treatment, which significantly differentiates MyPlypa and positions it as the cornerstone therapy. Josh will provide additional details related to our launch metrics later in today's call. Beyond the successful U.S. launch, our goal is to provide access to as many people living with NPC as possible by expanding the availability of MyPlypa outside of the U.S., We are diligently working towards expanding our regulatory submissions with an immediate focus on a marketing authorization application, or MAA, in Europe in the second half of this year, where we estimate approximately 1,100 people are living with MPC. As a reminder, the European market is already well established, with access to a marketed treatment for more than a decade. Further, we're expanding our global EAP, which has recently increased to approximately 85 NPC patients compared to the previously reported 70 to 80 patients. Our continued investment in MyPlyfa's global EAP in combination with a more established market could accelerate commercialization upon approval in Europe. Turning to Opruba. From initial product availability in July of 2023, and including Zevra's promotion of Olpruva in late January of 2024, through the end of the first quarter, 2025, we've received a total of 28 prescription enrollment forms, with five prescription enrollment forms during the first quarter of this year. In November, we outlined a plan to refine our commercial strategy towards the adult and OTC-deficient populations for whom Olpruva's portability and ease of administration may provide the greatest benefit. The enrollments received in the first quarter were all within this patient segment, suggesting early signs that this strategy is gaining traction. Before turning the call over to Josh, I'll share a few more updates on our clinical stage assets. Through our Phase III DISCOVER trial, saliprolol is being evaluated as a potential treatment of VEDS, a devastating connective tissue disorder caused by the COL3A1 gene mutation and characterized by spontaneous arterial aneurysms, hollow organ ruptures, and aortic dissections. In the U.S., approximately 7,500 people are living with VEDS, where the standard of care relies on reactive and invasive surgical interventions with no approved treatments. Tolipilol is also currently used off-label as a standard of care for VEDS in certain European countries. During the first quarter, we enrolled five additional subjects in the DISCOVER trial, bringing the total to 32. This is an event-driven trial, and the rate of enrollment is important. And we're implementing various strategies, including increasing our efforts to focus on providers and clinics specializing in genetically confirmed COL3A1 positive patients. During the first quarter, we began receiving leads from a recently initiated genetic testing program, providing our team with a new group of confirmed VEDS patients for outreach. Importantly, we are optimistic in this strategy to drive enrollment and expect to see an increase in the enrollment rate in the upcoming quarters. There are also several updates related to the review of our extensive IP portfolio as we seek to extract value by determining where we continue to invest as well as where we choose to stop investing based on our strategic plan. I'm pleased to report that earlier this month, we've outlicensed intellectual property related to a preclinical prodrug. to an undisclosed party for a nominal upfront payment, potential future milestones, and mid-single-digit royalties on net sales. We will continue to optimize and curate our IP portfolio through a variety of avenues to realize value for the company and our shareholders. Additionally, regarding APIDAS, which was previously approved for the treatment of pain and has not been commercially available since 2023, we have made a strategic decision to formally withdraw the NDA. By eliminating the regulatory activities associated with maintaining approval, we are further reducing costs. We are continuing Zevra's transformation into a patient-centric, commercial-stage rare disease therapeutics company. With our bolstered cash balance, we are well-positioned to support our stated strategic priorities and execute on our long-term vision for the company. By establishing myPIPA as a cornerstone treatment for NPD globally, maximizing our commercial opportunity with opruva and supporting our growth through our development pipeline including the ongoing phase three trial for solipol continue to build on our track record of execution positioning us to make future investments that are in alignment with our strategic plan let me turn the call over to josh who will give an update on our commercial products thank you neil and good afternoon