Zevra Therapeutics, Inc.

Good afternoon, and thank you for joining Debra's fourth quarter and full year 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, Debra's vice president of investor relations and corporate communications.

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 our fourth quarter and full year 2025 financial results. I encourage you to read the financial results news release, which was distributed this afternoon and is available in the investors 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 forward-looking statements due to risks and uncertainties associated with Debra's business. Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties, and other important factors that may lead to actual results differing materially from the projections made. and should be evaluated together with the risk factor section in our most recent quarterly report on Form 10Q, our annual report on Form 10K, and other filings with the SEC. I'm pleased to welcome ZEVRA's management team members participating in today's call. Neal McFarlane, ZEVRA's President and Chief Executive Officer, and Josh Schaefer, our Chief Commercial Officer, Our Chief Medical Officer, Adrian Cortell, and our Senior Vice President of Finance and Corporate Controller, Timothy Sangiovanni, will be available for today's question and answer session. Now, it is my pleasure to hand the call over to Neil.

Thank you, Nicole, and welcome to everyone joining our quarterly call this afternoon. I want to start off by welcoming our newly appointed Chief Financial Officer, Justin Renz, whose official start date is today. Justin brings more than 25 years of biopharmaceutical financial leadership experience with a strong track record supporting global commercial operations along with extensive experience in capital markets and investor relations. He will share a few brief remarks at the end of the call. However, he will not be taking questions on today's call. At Zebra, we're on a mission to redefine what's possible in delivering life-changing therapies to people living with rare diseases. As a commercial stage company with a late stage pipeline, we have a unique opportunity to make meaningful impact for patients. Our vision comes to life through our strategic plan and our core values of patient centricity, integrity, accountability, innovation, and courage. Guided by the execution of our plan and demonstrating our values, 2025 was a banner year with net revenue of $106.5 million. driven by the performance of MyPlypha, which delivered $87.4 million in revenue. Specifically in the fourth quarter, we generated $34.1 million in total net revenue, which included $26.4 million from MyPlypha sales. This momentum set the stage for multiple growth drivers in 2026. Our success was reflected in the strong commercial performance of MyPlypha, with 24 prescription enrollment forms received in the fourth quarter, bringing the total in the first full year of commercialization to 52 enrollment forms, and a total of 161 enrollments since launch. MyPipe is our foundational treatment for Niemann-Pick disease type C, or NPC, and this increasing penetration means that more patients are gaining access to the therapy they need. Importantly, This growth is being driven by previously diagnosed patients and increasingly by newly diagnosed patients. This trend reinforces our confidence in the estimated U.S. prevalence of approximately 900 affected individuals, of whom 300 to 350 are currently diagnosed. Later on the call, Josh will review some of the initiatives that are fueling our traction in the identification of newly diagnosed patients. As a reminder, MyPlypha has exclusivity in the U.S. through 2031 under the orphan drug designation, and we are diligently pursuing a patent term extension, which is currently under review by the U.S. Patent Office. It's important to note that additional exclusivity would represent positive upside to our existing runway. We're expanding access to MyPlypha, or Arimoclomol, beyond the U.S. through our Global Expanded Access Program, or EAP. which had 113 patients enrolled at the end of 2025. Over the past year, we've built a strong reputation across Europe, in particular through our global EAP, enabling clinicians to gain first-hand experience with Aramoclomol. In the fourth quarter, we established a new distribution agreement to extend the global EAP to select territories outside of Europe and initiated shipments of named patient supply prior to year end. As is common with these types of distribution agreements, we anticipate variability in ordering patterns and the rate of new enrollments in the first few years until the patient base has stabilized. We're pleased with the rapid execution by our partner to ensure swift delivery to patients. Importantly, in late July of last year, we submitted our marketing authorization application to the European Medicines Agency, supported by a robust data package including our pivotal phase 2-3 trial, open-label extension study, expanded access programs, and our pediatric sub-study. As a part of the standard review process, we received a 120-day list of questions from the EU regulators at the end of 2025. At this time, we are fully prepared to respond to these questions within the regulatory 90-day clock stop period and look forward to our continued engagement with the agency to make Aramacamol available to the EU NPC community. In Europe, NPC prevalence is estimated at approximately 1,100 individuals, and our current EAP is supporting nearly 10% of this population. Diagnosis rates are significantly higher than in the US, due in large part to the European approval of Miglistat for the treatment of NPC, which provided a clear treatment pathway and has led to stronger position awareness and higher patient identification. As we continue expanding access and strengthening relationships with key opinion leaders, we are well positioned to maximize our commercial impact in Europe through strategic partnerships or independently. Beyond MyPlypha, we're advancing Serpol for the treatment of vascular Ehlers-Danlos syndrome, or VETS, a rare inherited connective tissue disorder caused by COL3A1 gene mutations. These mutations lead to defects in type 3 pro-collagen in the walls of blood vessels and hollow organs, resulting in arterial dissections and organ ruptures. In the U.S., approximately 7,500 individuals have been diagnosed with VEDS. Soliperol acts as a selective adrenoseptor modulator, reducing mechanical stress on collagen fibers. Data on soliperol generated outside of the U.S., including results from the B-BEST study, and two long-term European cohorts demonstrated a reduction in major vascular events and improved overall survival among patients receiving treatment. Our ongoing phase three DISCOVER study designed to confirm the clinical benefit observed in prior studies. We enrolled eight patients in the fourth quarter, bringing the total number of enrolled patients at year end to 52 of the planned 150 patients to be enrolled in the study. and one confirmed event out of the 28 events required to trigger the interim analysis. We are continuing to implement strategies to boost our enrollment, including building a network of genetic testing centers to help diagnose individuals with VEDS, as well as connecting with specialists that care for these patients, such as the vascular surgeons who intervene when a patient experiences an event. In parallel, we recently engaged the FDA in a Type C meeting to discuss our regulatory options to accelerate the development program. While discussions are in their early stages, we appreciate the agency's responsiveness to our request and look forward to further engagement in the second half of the year. Finally, from a corporate standpoint, we've moved our headquarters to Boston, a strategic step that strengthens our foundation for long-term growth and positions us at the center of one of the world's most dynamic biotech ecosystems. This move enhances our ability to collaborate with industry leaders and access a deep pool of highly specialized talent, ensuring we remain well equipped to deliver on our strategic plan. Before turning the call over to Josh, I would like to recognize that February was Rare Disease Awareness Month, and to coincide with this, we were invited to ring the opening bell at NASDAQ. It was a powerful tribute to the patients who inspire our mission and drive us forward every day. It's especially important to connect with the community. And during the month, we also held an all-hands meeting where we had the privilege of hearing from a mother of two children with NPC. Her story reinforced the importance of expanding access to diagnosis, treatment, and support for the community. And it was a moving reminder of why we do what we do for people living with rare diseases. Josh?