Zevra Therapeutics, Inc.

Good afternoon and thank you for joining Zevra's second 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 of investor relations and corporate communications. 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 our second quarter financial results. I encourage you to read our financial news release, which was distributed this afternoon and is available in the investor relations 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 Devereux'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 to differ materially from the projections made. and should be evaluated together with the risk factor section in our most recently filed quarterly report on Form 10-Q, annual report on Form 10-K, and other filings with the SEC. In addition, we will disclose certain non-GAAP information on today's call, specifically adjusted net loss and adjusted net loss per share. Reconciling information to GAAP can be found in our financial results news release. I'm pleased to welcome Zebra's management team members participating in today's call. Neal McFarlane, Zebra's President and Chief Executive Officer, Liz Wayne Clifton, our Chief Financial Officer, and Josh Schaefer, our Chief Commercial Officer. Our Chief Medical Officer, Adrian Cortell, will 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 to everyone who's joining our update call this afternoon. At Zevra, our vision is to build a leading life sciences company whose core mission is to serve patients by bringing life-changing therapeutics to people living with rare diseases. We are demonstrating our ability to execute in a complex regulatory, clinical development, and commercial environment to advance promising therapies with an approach that balances science with patient needs. As a reminder, this vision is driven by the four pillars of our corporate strategy, commercial excellence, pipeline and innovation, talent and culture, and corporate foundation. The execution of our strategy delivered remarkable performance in the second quarter and set the strong foundation for continued momentum in Q3. On today's call, we'll highlight several key achievements. Q2 net revenue reached $25.9 million. reflecting robust demand and effective operational execution. We completed the sale of our PRV for $150 million, a strategic transaction that strengthened our balance sheet. We also submitted our Marketing Authorization Application, or MAA, for our Markle Modeling Europe, marking an important milestone in our geographic expansion efforts. Let's dive in with an update on the U.S. launch of MyKleifa. the first approved treatment for people diagnosed with Niemann-Pick disease type C, or NPC, and the only treatment that has been shown to halt disease progression by addressing its underlying pathology. As a quick reminder, NPC is an ultra-rare, relentlessly progressive neurodegenerative disease caused by inherited lysosomal storage disorder and leads to premature mortality. In the U.S., we estimate that roughly 300 to 350 patients have been diagnosed with MPC out of the estimated 900 people living with the disease. The approval of myClypha marked a fundamental shift in the treatment paradigm, and we believe that its ability to address disease progression is an important differentiator that will lead to long-term success. We are incredibly proud of the work our team is doing to support the healthcare community and eligible patients with an intense sense of urgency. We've received positive feedback from both patients and physicians, which is reflected in the strong performance since launch, with a total of 129 prescription forms through the end of Q2, of which seven were enrolled in the quarter. Through rapid uptake in the early stages of launch, we have been able to reach over one-third of those diagnosed with MPC in the United States. This group primarily included patients in our Expanded Access Program, or EAP, who have been treated with MIPLIFER for up to seven years, with the majority of patients remaining on therapy. Following the success of our early launch penetration, our commercialization efforts are focused on increasing both diagnosis and treatment access. The second segment of patients that we're focused on are those who have received an NPC diagnosis but may or may not be currently receiving treatment. These patients typically were diagnosed at centers of excellence and managing coordination with a local provider for ongoing care. We've mapped these referral patterns beyond the centers of excellence and are engaging the broader group of prescribers. The last segment of our patients are those living with NPC but who have not been diagnosed. Part of our contribution and responsibility to the patient community is to increase awareness and shorten the time to diagnosis for this devastating disease. We are demonstrating leadership through our disease state awareness campaign to help drive early diagnosis. The treatment of NPC is multifaceted and our program offers education and genetic testing options for individuals with suspected lysosomal storage disorders. With growing awareness of MyPlytha, we look forward to extending our reach to serve additional patients. Our work with advocacy organizations is very important, and our support has been well received. We also provide more details on our strong presence at recent conferences where Xevra and our specialty pharmacy engage with caregivers and people living with NPC. These are some of the ways that we're building our reputation as a reliable partner. Another exciting opportunity for MyPlyfor is in Europe, where we estimate approximately 1,100 people are living with NPC. One of our priorities is to secure European approval and determine the optimal go-to-market strategy to ensure access for a greater number of patients. Just a few weeks ago, we announced the submission of the Aramocomal MAA to the European Medicines Agency, delivering on the early side of our second half of 2025 guidance. This is another significant and timely milestone for Zebra. To support the submission, we received guidance from EU regulators and assembled a robust data package, including new mechanistic and long-term clinical data generated since our FDA submission. We have demonstrated a synergistic effect with Miglestat, which is approved in Europe for MPC and is the standard of care in this more mature market. where we have an established EAP program, which increased to 89 patients enrolled at the end of the second quarter. And to further our efforts in the scientific and medical community, our data from the Open Label Extension Study was recently published in the Journal of Molecular Genetics and Metabolism. The paper presents the impressive long-term efficacy and safety outcomes for NPC patients treated with MyPlypha in the 48-month open-label extension phase following the end of the 12-month pivotal trial. Additionally, we published data showing that MyPlypha upregulates expression of genes, belonging to the Coordinated Lysosomal Expression and Regulation Network, or the CLEAR network, targeting the underlying pathophysiology of NPC to improve autophagy, reduce cholesterol accumulation, and prevent cell death. As the NPC marketplace evolves, shaping treatment guidelines will become more important for the NPC community. We believe our success in establishing myPlypus benefit through the largest data set of NPC clinical trial patients and having these data published in peer-reviewed journals will help inform treatment decisions. Turning now to Opruva, which is a treatment for certain urea cycle disorders. We've previously discussed the limited pull-through we experienced in this launch, which continued in the second quarter, with one prescription in woman form. Our refined marketing efforts over the last several months have focused on identifying the patients for whom OPruva is best suited, strengthening our patient support services, and addressing reimbursement challenges. However, adoption continues to be slow, and we remain cautious. Josh will provide more detail on the product performance, and LeBwayne will provide financial details on the non-cash charge related to the prevailing trend later in the fall. Moving on to our pipeline, we're advancing Solipilol as a potential treatment of vascular Ehlers-Danlos Syndrome, or VEDS, in the Phase III DISCOVER trial. VEDS is a severe genetic connective tissue disorder characterized by a risk of dissection and rupture of the arteries, gastrointestinal tract, and uterus. Solipilol is an adenoceptor modulator believed to decrease mechanical stress on the vascular wall of large arteries and hollow organs. In the second quarter, we enrolled seven patients in the DISCOVER trial, bringing the total to 39 patients enrolled out of the 150 patients required for complete enrollment of the trial. The genetic testing initiative to identify patients who have the COL3A1 gene mutation, which causes the disease, is ramping up in collaboration with patient advocates, KOLs, treating physicians, and clinics, providing increased visibility and has the potential to accelerate future enrollment. We are making progress in advancing our vision of becoming a leading rare disease company through focusing on the patients we serve. Our strategy balances near-term operational excellence with the agility and financial flexibility to support initiatives that build sustainable value for patients and shareholders alike. Let me turn the call over to Josh, who will provide more details on our commercial products. Josh?