Financial Performance - In Q4 2025, total net revenue was $34.1 million, including $26.4 million from MIPLYFFA sales, $400,000 from OLPRUVA, $5.6 million from global EAP reimbursements, and $1.8 million from royalties under the AZSTARYS license [22][23] - For the full year 2025, net revenue reached $106.5 million, with $87.4 million from MIPLYFFA sales, $800,000 from OLPRUVA, $13 million from global EAP reimbursements, and $5 million from royalties [24] - Operating expenses for Q4 were $23 million, a decrease of $1.5 million year-over-year, while full-year operating expenses were $90.4 million, down $6.6 million from 2024 [22][24] - Net income for Q4 was $12.2 million, compared to a net loss of $35.7 million in Q4 2024, and full-year net income was $83.2 million, compared to a net loss of $105.5 million in 2024 [23][24] Business Line Performance - MIPLYFFA generated $87.4 million in revenue for the full year 2025, with strong commercial performance reflected in 52 prescription enrollment forms received in the first year of commercialization [5][6] - In Q4, 24 new prescription enrollment forms were received, contributing to a total of 161 enrollments since launch [6][14] - The company is expanding access to MIPLYFFA through a global expanded access program, with 113 patients enrolled by the end of 2025 [8] Market Data - The estimated U.S. prevalence of Niemann-Pick disease type C (NPC) is approximately 900 individuals, with 300-350 currently diagnosed [7] - In Europe, NPC prevalence is estimated at around 1,100 individuals, with the current EAP supporting nearly 10% of this population [9] - Diagnosis rates in Europe are higher due to the approval of miglustat, leading to stronger physician awareness and higher patient identification [10] Company Strategy and Industry Competition - The company aims to redefine therapies for rare diseases, focusing on patient-centricity, integrity, accountability, innovation, and courage [5] - MIPLYFFA has exclusivity in the U.S. through 2031 under the Orphan Drug Designation, and the company is pursuing a patent term extension [7] - The company is also advancing celiprolol for the treatment of vascular Ehlers-Danlos syndrome, with ongoing Phase III studies [10][11] Management Commentary on Operating Environment and Future Outlook - Management expressed confidence in the growth potential of MIPLYFFA, driven by both previously diagnosed and newly diagnosed patients [15][16] - The company is focused on expanding access and strengthening relationships with key opinion leaders to maximize commercial impact [10] - Management highlighted the importance of ongoing education and awareness campaigns to facilitate early diagnosis and treatment [16][18] Other Important Information - The company moved its headquarters to Boston to enhance collaboration and access to specialized talent [12][13] - The new CFO, Justin Renz, officially started on the call date and will support the company's long-term value creation [4][28] Q&A Session Summary Question: Can you provide details on the split of new diagnoses versus previously identified patients? - Management noted that there has been meaningful growth in newly diagnosed patients, attributed to clinical data strength and disease awareness campaigns [33][34] Question: What is the expected timeline for diagnosis confirmation? - Management indicated that the timeline for diagnosis can vary significantly due to the nature of rare diseases, but efforts are being made to streamline the process [37][40] Question: Can you discuss persistency rates for patients on therapy? - Management stated that it is too early to provide meaningful data on persistency but expressed encouragement from refill rates observed [45][46] Question: How is the initial experience with the Uniphar collaboration? - Management expressed satisfaction with the collaboration, noting that it has facilitated rapid product delivery to patients [47][48] Question: What is the expected growth of EAP revenue? - Management clarified that EAP revenue is based on individual patient shipments and will have variability quarter-over-quarter [54][55] Question: How is the gross to net evolving in the U.S.? - Management noted that gross to net has evolved and is dynamic, making it difficult to provide specific guidance [58] Question: What is the average age of patients on MIPLYFFA? - Management indicated that approximately half of MIPLYFFA patients are adults, reflecting the patient mix seen in the expanded access program [64][66] Question: What are the next steps for the EMA application? - Management confirmed readiness to respond to the EMA's questions and emphasized the robustness of the data package submitted [69]

Financial Data and Key Metrics Changes - In Q4 2025, total net revenue was $34.1 million, including $26.4 million from MIPLYFFA sales, $400,000 from OLPRUVA, and $5.6 million from net reimbursements under the global EAP [22][23] - For the full year 2025, net revenue reached $106.5 million, with $87.4 million from MIPLYFFA sales, compared to a net loss of $105.5 million in 2024 [25] - Operating expenses for Q4 2025 were $23 million, a decrease of $1.5 million year-over-year, while R&D expenses decreased by $5.8 million to $2.6 million [22][24] Business Line Data and Key Metrics Changes - MIPLYFFA generated $87.4 million in revenue for the full year 2025, with a total of 161 enrollments since launch, including 52 in the first full year of commercialization [5][6][15] - The global expanded access program (EAP) had 113 patients enrolled by the end of 2025, with ongoing efforts to expand access beyond the U.S. [8][9] Market Data and Key Metrics Changes - In the U.S., the estimated prevalence of Niemann-Pick disease type C (NPC) is approximately 900 individuals, with 300-350 currently diagnosed [7][10] - In Europe, the estimated prevalence of NPC is around 1,100 individuals, with the current EAP supporting nearly 10% of this population [9] Company Strategy and Development Direction - The company aims to redefine therapies for rare diseases, focusing on patient-centricity and innovation [4] - A strategic move to Boston is intended to enhance collaboration and access to specialized talent within the biotech ecosystem [12] - The company is pursuing a patent term extension for MIPLYFFA, which could provide additional exclusivity [7] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth potential of MIPLYFFA, driven by strong clinical data and increased awareness among healthcare providers [15][16] - The company is optimistic about the continued identification of newly diagnosed patients and the overall market potential for MIPLYFFA [16][20] Other Important Information - The company has engaged the FDA regarding regulatory options to accelerate the development of celiprolol for vascular Ehlers-Danlos syndrome [12][70] - The company has a strong balance sheet with $238.9 million in cash and equivalents, allowing for independent operation and investment in growth opportunities [90] Q&A Session Summary Question: Can you provide details on the split of new diagnoses versus previously identified patients? - Management noted that there has been meaningful growth in newly diagnosed patients, attributed to clinical data strength and disease awareness campaigns [32][33] Question: What is the expected timeline for diagnosis confirmation? - The average time for test results is estimated to be about three weeks, facilitating quicker clinical decision-making [41] Question: Can you discuss persistency rates for patients on therapy? - Management indicated that it is too early to provide meaningful data on persistency but noted encouraging refill rates [46][47] Question: How is the initial experience with the Uniphar collaboration? - The company is pleased with the collaboration and expects continued growth in patient access through this partnership [48][49] Question: What is the total addressable market for diagnosed NPC patients? - Management believes they have engaged approximately 40%-50% of the diagnosed patients currently enrolled to receive MIPLYFFA [80]

Core Insights - Zevra Therapeutics reported Q1 2025 net revenue of $20.4 million, a significant increase from $3.4 million in Q1 2024, primarily driven by product net revenue of $17.2 million from MIPLYFFA [8][6][30] - The company completed the sale of its Pediatric Rare Disease Priority Review Voucher (PRV) for gross proceeds of $150 million, enhancing its financial position to support commercial launches and development programs [5][2] - Zevra aims to establish MIPLYFFA as a cornerstone treatment for Niemann-Pick disease type C (NPC) and maximize commercial opportunities with OLPRUVA [2][30] Financial Highlights - Total net revenue for Q1 2025 was $20.4 million, including $17.1 million from MIPLYFFA, $0.1 million from OLPRUVA, and $2.3 million in reimbursements from the French Expanded Access Program for arimoclomol [8][6] - Operating expenses for Q1 2025 were $22.8 million, with R&D expenses at $3.3 million, a decrease from the previous year, while SG&A expenses increased to $19.5 million [13][8] - The net loss for Q1 2025 was $3.1 million, or $0.06 per share, compared to a net loss of $16.6 million, or $0.40 per share in Q1 2024 [13][8] Commercial Highlights - MIPLYFFA had 13 new prescription enrollments in Q1 2025, totaling 122 prescriptions, with market access at 38% of covered lives [7] - OLPRUVA received 5 new patient enrollment forms in Q1 2025, bringing the total to 28, with market access increasing to 78% of covered lives [7] - The company plans to file the MIPLYFFA Marketing Authorization Application (MAA) with the European Medicines Agency in the second half of 2025 [7] Pipeline and Innovation Highlights - Zevra enrolled 5 additional patients in the Phase 3 DiSCOVER trial of celiprolol for Vascular Ehlers-Danlos Syndrome, bringing total enrollment to 32 [7] - The company out-licensed intellectual property related to a deprioritized pre-clinical prodrug, which may yield future regulatory milestones and royalties [7] Upcoming Events - Zevra will participate in the H.C. Wainwright 3rd Annual Bioconnect Investor Conference on May 20, 2025, and will host a conference call to discuss Q1 2025 results [9][10]

Core Viewpoint - Zevra Therapeutics is urging stockholders to support its current board and management team in the upcoming election, emphasizing their successful track record and strategic direction, while opposing the influence of a minority stockholder seeking to replace qualified directors without a clear plan [1][3][30]. Group 1: Company Performance and Strategy - Zevra has achieved total stockholder returns of 54.8% since the appointment of CEO Neil McFarlane in October 2023, outperforming the biotech sector and the Russell 2000 [8][42]. - The company has launched two rare disease therapies, MIPLYFFA and OLPRUVA, and has expanded its commercial capabilities significantly [20][24]. - A comprehensive five-year strategic plan has been established to drive long-term growth, focusing on commercial excellence, pipeline innovation, talent development, and corporate foundation [7][16]. Group 2: Board and Management Recommendations - The board recommends stockholders vote "FOR" the re-election of independent directors Wendy L. Dixon, Ph.D., and Tamara A. Favorito, who bring extensive experience and have contributed to the company's strategic goals [2][11][29]. - The board emphasizes that seven of the eight current directors have joined since 2023, reflecting a proactive refreshment aimed at enhancing governance and oversight [4][27]. Group 3: Opposition to Minority Stockholder Influence - Daniel J. Mangless, a minority stockholder with 2.8% ownership, is attempting to replace two directors without providing a clear strategy for improving the company [4][19][30]. - The board expresses concerns that Mangless' nominees lack the necessary expertise and could disrupt the company's ongoing progress and strategic execution [12][36]. - The board highlights the potential risks associated with electing Mangless' nominees, citing their previous records of stockholder value destruction [9][41].

Core Insights - Zevra Therapeutics, Inc. has successfully closed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million, enhancing its financial flexibility for strategic priorities [1][2] Financial Performance - As of March 31, 2025, Zevra reported available unaudited cash, cash equivalents, and investments totaling $68.7 million. After the sale of the PRV, net cash proceeds of $148.3 million will increase total cash, cash equivalents, and investments to $217.0 million [3] Company Overview - Zevra Therapeutics is a commercial-stage company focused on developing therapies for rare diseases with limited or no treatment options, aiming to bring life-changing therapeutics to patients [4]