Core Insights - Zevra Therapeutics reported Q2 2025 net revenue of $25.9 million, significantly up from $4.4 million in Q2 2024, driven primarily by product net revenue of $21.8 million from MIPLYFFA [1][8] - The company completed the sale of its Pediatric Rare Disease Priority Review Voucher (PRV) for $150 million, enhancing its balance sheet as it advances commercial launches and development programs [1][5] - Zevra submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency for arimoclomol, aimed at treating Niemann-Pick Disease Type C [1][7] Commercial Highlights - MIPLYFFA achieved 7 prescription enrollments in Q2 2025, totaling 129 since its launch, with market access reaching 52% of covered lives [7] - OLPRUVA saw 1 prescription enrollment in Q2 2025, bringing the total to 29, with market access increasing to 79% of covered lives [7] Financial Highlights - Total revenue for Q2 2025 was $25.9 million, including $21.5 million from MIPLYFFA, $0.3 million from OLPRUVA, and $2.6 million in net reimbursements for arimoclomol [8] - Operating expenses were $24.2 million, with R&D expenses decreasing to $3.4 million, while SG&A expenses increased to $20.8 million [14] - The company reported a net income of $74.7 million for Q2 2025, compared to a net loss of $19.9 million in Q2 2024 [10][14] Pipeline and Innovation Highlights - Arimoclomol has been designated as an Orphan Medicinal Product by the EMA, and the company is on track with its guidance for submission in the second half of 2025 [7][17] - Zevra enrolled 7 patients in the Phase 3 DiSCOVER trial of celiprolol for Vascular Ehlers-Danlos Syndrome during Q2 2025, totaling 39 enrolled patients [7] Corporate Highlights - As of June 30, 2025, Zevra's cash, cash equivalents, and securities totaled $217.7 million, providing sufficient resources for strategic priorities [14] - The company has granted inducement equity awards to 9 individuals totaling 54,875 shares of restricted stock units and 49,750 options to purchase common stock [15][16]

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]

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]