Financial Data and Key Metrics Changes - The company reported net revenue of $20.4 million for Q1 2025, with $17.1 million from MyPlifa, $0.1 million from Olpruva, and $2.3 million in net reimbursements from the French EAP for Aramcholamol [29] - Operating expenses decreased to $22.8 million, down $0.6 million compared to the same quarter last year [30] - 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, for the same quarter a year ago [30] Business Line Data and Key Metrics Changes - MyPlifa's launch has exceeded expectations, with 122 prescription enrollment forms received since launch, indicating that approximately one-third of diagnosed NPC patients in the U.S. have been enrolled [19][20] - Olpruva received a total of 28 prescription enrollment forms since its initial availability, with five forms in Q1 2025 [12][26] Market Data and Key Metrics Changes - The company achieved 38% of covered lives for MyPlifa by the end of Q1 2025, which aligns with expectations for this stage of the launch [20][64] - The European market for MyPlifa is being targeted, with an estimated 1,100 people living with NPC, and regulatory submissions are planned for the second half of the year [11] Company Strategy and Development Direction - The company is focused on commercial excellence, pipeline innovation, talent and culture, and corporate foundation as its four strategic pillars [5][6] - The recent monetization of the Pediatric Rare Disease Priority Review Voucher added $148.3 million of non-dilutive capital to the balance sheet, enhancing financial strength [8][30] - The company aims to establish MyPlifa as a cornerstone treatment for NPC globally and maximize commercial opportunities with Olpruva [16][32] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the early success of MyPlifa and the refined marketing strategy for Olpruva, indicating a strong position for future growth [33] - The company is well-positioned to support strategic priorities independent from capital markets, with ample resources for execution [32] Other Important Information - The company has made a strategic decision to withdraw the NDA for Apodas, reducing regulatory costs [16] - The ongoing Phase III trial for saliprolol is being actively managed to drive enrollment, with 32 patients currently enrolled [14][72] Q&A Session Summary Question: Details on the number of patients on MyPlifa and reimbursement status - Management indicated that the majority of the 122 enrollments are on the drug, with some undergoing benefits investigation [36] Question: Top reasons for reimbursement denials for MyPlifa - Initial denials are common, primarily due to prior authorization requirements, but the company has successfully secured coverage for most patients [39] Question: Visibility on patient sources and enrollment cadence - Approximately 600 undiagnosed patients exist, and efforts are ongoing to raise awareness and enroll both diagnosed and undiagnosed patients [48] Question: Inventory levels at the end of the quarter - The company is managing inventory closely and maintaining target levels as they support patient needs [51] Question: Qualitative metrics on refill rates and patient retention - Most patients receiving active drug have continued with refills, reflecting the clinical benefit of MyPlifa [55] Question: Coverage details and impact on patient access - 38% of covered lives means that many plans have reviewed MyPlifa, with successful reimbursement through medical exception pathways [64][66]

Financial Data and Key Metrics Changes - In Q1 2025, the company reported net revenue of $20.4 million, with $17.1 million from MyPlifa, $0.1 million from Olpruva, and $2.3 million in net reimbursements from the French EAP for Aramcholamol [29][30] - Operating expenses for Q1 were $22.8 million, a decrease of $0.6 million compared to the same quarter last year [30] - The net loss for Q1 2025 was $3.1 million, compared to a net loss of $16.6 million in the same quarter a year ago [30][31] - Total cash, cash equivalents, and investments as of March 31, 2025, were $68.7 million, a decrease of $6.8 million from December 31, 2024 [30][31] Business Line Data and Key Metrics Changes - MyPlifa's launch has exceeded expectations, with 122 prescription enrollment forms received since launch, indicating that approximately one-third of diagnosed NPC patients in the U.S. have been enrolled [20][21] - Olpruva received a total of 28 prescription enrollment forms since its initial availability, with five forms received in Q1 2025 [12][27] - The company has achieved 38% of covered lives for MyPlifa, which is in line with expectations at this stage of the launch [21][22] Market Data and Key Metrics Changes - The company is focusing on expanding MyPlifa's availability outside the U.S., with plans for a marketing authorization application in Europe in the second half of the year [11] - The European market for NPC is well established, with approximately 1,100 people estimated to be living with the disease [11] Company Strategy and Development Direction - The company is executing on four strategic pillars: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation [5][8] - The focus is on bringing innovative therapies to patients with rare diseases, with a strong emphasis on MyPlifa as a cornerstone treatment for NPC [17][33] - The company is also assessing strategic alternatives for its Phase III ready asset KP1077 for rare sleep disorders [6] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the early success of MyPlifa and the refined marketing strategy for Olpruva, indicating a strong position for future growth [33] - The company believes it is well positioned to support its strategic priorities and execute on its long-term vision, independent from capital markets [31][32] Other Important Information - The company has successfully monetized its Pediatric Rare Disease Priority Review Voucher, adding $148.3 million of non-dilutive capital to its balance sheet [8][31] - The company is committed to patient access and comprehensive patient services through its in-house program, Amplify Assist [27] Q&A Session Summary Question: Details on the number of patients on MyPlifa and reimbursement status - The company received 122 enrollments since launch, with the majority of patients actively on the drug, while some are in the benefits investigation process [36] Question: Reasons for reimbursement denials for MyPlifa - Initial denials are common for rare disease products, primarily due to prior authorization requirements, but the company has been successful in securing coverage through medical exception processes [39][40] Question: Visibility on patient cohorts and enrollment cadence - The company estimates around 600 undiagnosed patients and 300 diagnosed patients, with ongoing efforts to raise awareness and facilitate treatment [47][59] Question: Inventory levels at the end of the quarter - The company is managing inventory closely and maintaining target levels as it supports patient needs during the early launch phase [50] Question: Qualitative metrics on refill rates and patient retention - The majority of patients receiving active drug have continued to refill, reflecting the clinical benefit and durability of MyPlifa [54] Question: Coverage details and impact on patient access - 38% of covered lives have access to MyPlifa, with ongoing efforts to increase this percentage as more plans evaluate the product [63][64]

Summary of Zevra Therapeutics (ZVRA) Conference Call Company Overview - **Company**: Zevra Therapeutics - **Focus**: Developing and commercializing therapies for rare diseases - **Key Product**: MyPlifa for Niemann Pick type C, launched in late 2024 [1][2] Core Points and Arguments Product Launch and Performance - **MyPlifa**: First FDA-approved therapy for Niemann Pick disease type C, launched in September 2024 [6][10] - **Clinical Profile**: Demonstrated a two-point difference in disease progression compared to Miglustat alone, with a strong safety profile [6][7] - **Patient Population**: Approximately 900 patients in the U.S., with 300-350 diagnosed [10] - **Expanded Access Program (EAP)**: Transitioned 70-80 patients from EAP to commercial therapy, supporting long-term data on efficacy and safety [11][12] Physician Feedback and Education - **Physician Insights**: Initial hesitance from physicians regarding prescribing due to the need for more data; however, long-term data has built confidence [14][15] - **Education Efforts**: Focus on educating physicians and patients about the disease and available therapies, including media campaigns to raise awareness [19][20] Market Dynamics and Opportunities - **Diagnosis Rate**: Low diagnosis rate in the U.S. compared to Europe, where a higher prevalence of diagnosed patients exists due to prior approved therapies [21][22] - **Market Potential**: Total addressable market (TAM) estimated to be similar in the U.S. and Europe, with expectations for faster penetration in Europe due to existing familiarity with Miglustat [30] Financial Position and Strategy - **Cash Position**: Strong cash balance of $217 million post-PRV sale, enabling continued support for MyPlifa launch and other programs [32][33] - **Strategic Focus**: Emphasis on executing current commercial strategies and exploring additional assets for future growth [34][36] Other Products and Pipeline - **Opruva**: Relaunched with a focus on clinical differentiation; ongoing efforts to improve physician engagement and product awareness [39][40] - **Soliprolol**: Phase three program for Vascular Ehlers Danlos Syndrome (VEDS) with a focus on rapid enrollment due to unmet medical needs [41][42] Additional Important Content - **Patient Engagement**: The company has seen a significant level of engagement from patients and families, which is crucial for transitioning to commercial therapy [12][18] - **Regulatory Plans**: Plans to file for marketing authorization in Europe in the second half of the year, leveraging existing data and EAP experiences [29][30] This summary encapsulates the key points discussed during the conference call, highlighting the company's strategic direction, product performance, market opportunities, and financial health.