Company Overview - Zevra Therapeutics is focused on bringing life-changing therapeutics to the rare disease community[9] - The company has two commercial-stage rare disease products and an advanced clinical development pipeline[8,82] - Zevra has a strong financial position with $217 million in cash, cash equivalents, and investments as of April 1, 2025, including $687 million as of March 31, 2025, plus $1483 million net proceeds from a PRV sale[8,81] Product Portfolio - MIPLYFFA (arimoclomol) is FDA-approved for Niemann-Pick Disease Type C (NPC) and launched in November 2024[20] - Approximately 900 individuals in the U S live with NPC, with 300-350 diagnosed or treated[38] - OLPRUVA (sodium phenylbutyrate) for oral suspension is FDA-approved for Urea Cycle Disorders (UCD) in patients weighing 20 kg or greater and with a body surface area (BSA) of 12 m2 or greater[20,50] - Approximately 1,100 individuals are diagnosed with UCDs in the U S [59] Pipeline Development - The company is targeting an EMA MAA submission for arimoclomol in Europe in the second half of 2025[49] - Celiprolol is in Phase 3 development for Vascular Ehlers-Danlos Syndrome (VEDS), with IP through 2038[20] - Approximately 7,500 individuals in the U S are diagnosed with VEDS[74]

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].

Financial Data and Key Metrics Changes - In Q4 2024, the company reported net revenue of $12 million, which included $10.1 million from MIPLYFFA, $0.1 million from OLPRUVA, and $1.1 million from net reimbursements from the French EAP for arimoclomol [41][44] - The full year 2024 net revenue was $23.6 million, with a net loss of $105.5 million or $2.28 per share, compared to a net loss of $46 million or $1.30 per share in 2023 [44][46] - Operating expenses for Q4 were $24.5 million, with R&D expenses decreasing to $8.4 million, while SG&A expenses increased to $16.1 million [43][45] Business Line Data and Key Metrics Changes - MIPLYFFA's launch exceeded expectations, with 109 patient enrollment forms received in Q4, reflecting strong demand in the NPC community [10][23] - OLPRUVA saw four new enrollments in Q4, an increase from three in Q3, with market access remaining consistent at 76% of covered lives [12][35] Market Data and Key Metrics Changes - The company estimates approximately 900 people with NPC in the U.S., with 300 to 350 diagnosed, indicating a significant market opportunity for MIPLYFFA [24][28] - The European market is expected to have around 1,100 patients living with NPC, with plans to submit a marketing authorization application in the second half of 2025 [11][53] Company Strategy and Development Direction - The company aims to expand MIPLYFFA's availability outside the U.S. and is focused on regulatory submissions in Europe [11][20] - The strategic plan includes four pillars: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation [7][19] - The company is rationalizing its in-house discovery efforts and outsourcing activities to focus investments where there is the greatest potential for impact [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the early success of MIPLYFFA and the potential for significant market penetration in both the U.S. and Europe [20][28] - The company is focused on building financial strength and disciplined capital allocation to support long-term transformation [19][47] Other Important Information - The company refinanced existing debt and completed a secondary offering, providing financial stability [19] - A priority review voucher was monetized for $150 million, expected to provide non-dilutive capital for future growth [20][46] Q&A Session Summary Question: Can you provide guidance on the European filing timeline? - Management indicated plans to file in the second half of 2025 and is confident in the acceptance of the filing based on improved long-term data [53][56] Question: How do you expect the European market dynamics to affect patient identification? - The European market is more mature, with more patients diagnosed and treated due to the long-standing presence of miglustat [53] Question: Can you provide more quantitative details on MIPLYFFA sales during the quarter? - MIPLYFFA revenue of $10.1 million included initial patient dispenses and refills, with inventory levels maintained at target levels [65] Question: What is the expected long-term net patient addition rate? - The focus will be on finding diagnosed but untreated patients, with expectations for steady growth in patient enrollments [67] Question: How many patients are currently treated at centers of excellence? - Most diagnosed patients have connections to centers of excellence, with ongoing efforts to expand treatment access [78] Question: What is the average time from enrollment to reimbursement? - It is too early to provide specifics, but initial experiences indicate a quick turnaround for some patients [104] Question: Have there been any patient denials for enrollment? - A few patients have faced initial denials, but the company has robust support systems in place to assist them [108]