Zevra Therapeutics, Inc.

Stand by, your program is about to begin. If you need audio assistance during today's program, please press star zero. Good afternoon and thank you for joining Zevra's Therapeutics fourth quarter and full year 2024 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 of Investor Relations and Corporate Communications.

Thank you, and welcome to those who are joining us. Today, we will provide an overview of our accomplishments in the fourth quarter and full year of 2024, followed by a review of financial results. I encourage you to read our financial results news release, which we distributed this afternoon and is available in the investor section of our website. Before we begin, 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 several 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. These forward-looking statements should be evaluated together with the cautionary statements contained in the risk factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K, and our other filings with the SEC. I'm pleased to welcome Zevra's management team members participating in today's call. Neal McFarling, Zevra's president and chief executive officer, LaDwayne Clifton, our chief financial officer, and Josh Schaefer, our chief commercial officer and EVP of business development. Our chief medical officer, Adrian Cortell, will also be available for today's question and answer session. Now, it's my pleasure to hand the call over to Neal.

Thank you, Nicole, and thank you for joining us this afternoon. As Nicole mentioned, today we'll provide our fourth quarter and fiscal year 2024 financial results and share progress on the execution of our priorities for 2025. Our priorities are guided by Zevra's strategic plan, unveiled during our third quarter call and categorized under four actionable pillars, commercial excellence, pipeline and innovation, talent and culture, and corporate foundation. 2024 was a transformational year for Zebra, and bringing my plight for the people living with Niemann-Pick disease type C, or NPC, was a crowning achievement. In the past year, we became a commercial stage company that had the opportunity to positively impact the lives of people living with rare diseases. The approval of myPIPA last year was an outcome of our long-standing collaboration with the NPC community and included contributions from more than 270 Niemann-Pick disease type C patients who participated globally in our pivotal studies, open-label extension studies, and expanded access programs. The early days of the MyPlypa launch have exceeded our expectations. In the fourth quarter, we received 109 patient enrollment forms, which we believe reflected the anticipation and unmet need in the MPC community. Additionally, by the end of Q4, all active U.S. EAP participants submitted an enrollment form, and our entire organization has been activated to ensure smooth transition to MyPlypa commercial product. As previously reported, we anticipate that the U.S. EAP will close by the end of Q2. Going forward, our goal is to expand availability of MyPlypha outside of the U.S. to provide access to as many people living with NPC as possible. With this goal in mind, we are progressing regulatory submissions. With the focus on submitting a marketing authorization application in Europe during the second half of 2025, where we estimate 1,100 people are living with NPC. Additionally, we will maintain our global expanded access program that currently supports 70 to 80 NPC patients, and we look forward to providing updates in the coming quarters as we seek to expand access to MyPlypha. We're leveraging our infrastructure for both urea cycle disorders, or UCDs, and NPC, leading to efficiencies that drive commercial excellence. The launch of Olpruva in early 2024 facilitated key learnings as we built our commercial capabilities. During the fourth quarter, we received four Olpruva enrollments. As announced in our Q3 call, we refined our strategy to reach the adult onset population for whom Olpruva's portability and ease of administration may provide a benefit with a focus on patients receiving payer pushback. us will provide more details related to the performance of our commercial products later in the call. Moving to pipeline and innovation, which refers to our ambition to develop products that address significant unmet needs. Oliparol is our phase three investigational clinical candidate for the treatment of vascular Ehlers-Danlos syndrome, or VETS, which is an inherited connective tissue disorder caused by the COL3A1 gene mutation. that can result in spontaneous arterial aneurysms and hollow organ ruptures. There are currently no treatments approved in the U.S. where there are approximately 7,500 people living with this disease, and the prevailing treatment paradigm is only reactive surgical interventions. Although Soliparol is not approved for VEDS in any country, it is utilized as a standard of care in several European countries. In Q4, we enrolled eight patients in our Phase III DISCOVER trial, bringing the total number of enrolled patients to 27. As an event-driven trial, the rate of participant enrollment is integral to the program's success. As a result, we are investing in tactics to accelerate enrollment among genetically confirmed patients with providers and clinics that treat COL3A1-positive patients. In addition, we have support from the Vets Foundation Professional Advisory Board on our path forward. Moving on to KP 1077. As outlined on our Q3 call and following a successful end of Phase II meeting with the FDA, we are exploring strategic alternatives to advance clinical development and future commercialization. We're focused on maximizing the value of this Phase III-ready asset that has regulatory feedback for the potential development in both idiopathic hypersomnia and narcolepsy indications. In 2024, we completed a comprehensive review of our IP portfolio to identify areas for optimization. This review led to a decision to rationalize our in-house discovery efforts and outsource those activities to focus our investments where we have the greatest potential for impact. To execute our strategic plan and deliver on our key priorities, it's paramount that we develop a high-performing culture and attract and retain talent. In the second quarter of 2024, we expanded our executive leadership team with the appointment of Rahsaan Thompson as Chief Legal Officer, Secretary, and Compliance Officer, and Allison Peters as Chief People Officer. Additionally, during the fourth quarter, we consolidated our development and scientific functions under Adrian Cortell, our Chief Medical Officer. We believe these changes optimize Zebra for success with cognitive leadership and the appropriate span of control to achieve our objectives. An important element of executing against our strategy is to build and maintain our position of financial strength and disciplined capital allocation that we refer to as our corporate foundation. This underpins all other pillars and allows us to responsibly invest in our long-term transformation. in 2024 we refinanced existing debt with a new credit facility and completed a modest secondary offering which provided financial stability recently we entered into an agreement to monetize our priority review voucher for 150 million dollars which upon closing will provide non-diluted capital to support our future growth building on the momentum from 2024 We are pleased to start 2025 from a position of strength across our four strategic pillars, execute on the tremendous opportunity we have to serve people living with rare diseases. I'll now turn the call over to Josh, who will provide an update on our commercial products.