Company Overview - Zevra Therapeutics is a commercial-stage company focused on impacting individuals with rare diseases [2] - The company is currently in a growth phase, with the operational capacity to pursue its commercial and development goals [2] Product and Revenue - Zevra is commercializing its lead product, MIPLYFFA, in the U.S. for Niemann-Pick disease type C and has an MAA under review by the EMA [3] - The company is expanding access through geographic opportunities and has a pipeline of rare disease programs [3] - In the first nine months of the previous year, Zevra generated net revenues of $72.3 million and ended Q3 with a strong cash position of $230.4 million [3]

Summary of Zevra Therapeutics FY Conference Call Company Overview - **Company**: Zevra Therapeutics (NasdaqGS: ZVRA) - **Industry**: Rare Disease Therapeutics - **Lead Product**: MIPLYFFA for Niemann-Pick disease type C (NPC) - **Financial Position**: Net revenue of $72.3 million in the first nine months of 2025, with a cash position of $230.4 million at the end of Q3 2025 [2][5][30] Core Points and Arguments Company Mission and Vision - Zevra aims to redefine possibilities in rare disease therapies, focusing on patient-centricity and accountability to stakeholders [4][5] - The company is in growth mode with a late-stage pipeline and geographic expansion plans [5][10] Product and Market Performance - MIPLYFFA received FDA approval in September 2024 and has orphan drug exclusivity until 2031 [5][6] - The product has achieved approximately 40% penetration of the diagnosed patient population within the first 12 months post-launch, a notable achievement in the rare disease space [10][19] - The company is expanding access to MIPLYFFA in Europe, with an MAA under review by the EMA [5][11] Pipeline and Future Growth - Zevra has a diversified portfolio including OLPRUVA for urea cycle disorders and AZSTARYS, with various products under development [6][28] - Celiprolol is in a pivotal phase 3 trial for vascular Ehlers-Danlos syndrome (VEDS) [28] - The company is leveraging AI for patient identification and diagnosis, enhancing its market reach [22][39] Financial Discipline and Strategy - Strong balance sheet with $230.4 million in cash and $61 million in debt [30] - The company monetized a pediatric priority review voucher for $150 million, adding non-dilutive capital [13] - Focus on sustainable growth through disciplined investments and prioritization of high-value opportunities [9][30] Important but Overlooked Content - The company is actively working on expanding its geographic reach beyond the U.S. and Europe, with distribution agreements in place for markets outside these regions [25][50] - The potential for patent term extension could significantly impact the company's valuation, with a ruling expected in 2026 [52][53] - The total addressable market (TAM) for NPC is estimated between 300 to 900 patients in the U.S., with ongoing efforts to identify undiagnosed patients [21][23] Conclusion Zevra Therapeutics is positioned for significant growth in the rare disease therapeutics market, driven by its innovative product MIPLYFFA, a strong financial foundation, and strategic expansion efforts. The company is focused on enhancing patient access and leveraging its pipeline to solidify its market presence.

Financial Performance & Cash Position - Zevra exited Q3 2025 with a strong cash position of $230.4 million[8], and total debt of $61.3 million[44] - The company generated net revenue of $72.3 million in the 9-month period[8], and $26.1 million for Q3 2025[43] - Q3 2025 net loss was $(0.5) million, or $(0.01) per basic and diluted share[43] Product Portfolio & Pipeline - MIPLYFFA adoption achieved in approximately 40% of diagnosed Niemann-Pick Disease Type C (NPC) patients within the first year of launch[13] - Payor coverage for MIPLYFFA reached 66% of covered lives as of Q3 2025[24] - Approximately 80% of patients who participated in the Phase 2/3 clinical trial for MIPLYFFA took miglustat[20] - Celiprolol Phase 3 DiSCOVER trial is ongoing for Vascular Ehlers-Danlos Syndrome (VEDS), with 44 of 150 patients enrolled as of Q3 2025[40] - For VEDS patients on celiprolol, the annual major vascular event rate is approximately 5% compared to approximately 12% in untreated patients[40] Market & Regulatory - Approximately 900 individuals in the U S live with NPC, of which 300-350 are diagnosed or treated[27] - Approximately 1,100 individuals are living with NPC in Europe[27] - MAA for Arimoclomol is under review by the EMA[8, 13]

Core Insights - Zevra Therapeutics (ZVRA) reported Q3 FY25 earnings on November 5th, 2025, with net revenues of $26.1 million, of which $22.4 million was generated from their primary revenue driver, MIPLYFFA, indicating a revenue increase of 605% compared to the same quarter last year [1] Company Performance - The company achieved a significant revenue growth of 605% year-over-year, highlighting its strong performance in the market [1] - MIPLYFFA remains the primary revenue driver, contributing a substantial portion of the total revenues [1]

Core Insights - Zevra Therapeutics reported a net revenue of $26.1 million for Q3 2025, significantly up from $3.7 million in Q3 2024, primarily driven by MIPLYFFA net revenue of $22.4 million [2][6][31] - The company is focused on expanding its presence in the rare disease market, particularly through the strong performance of MIPLYFFA for Niemann-Pick disease type C [3][28] Financial Highlights - Q3 2025 net revenue was $26.1 million, including $22.4 million from MIPLYFFA, $0.1 million from OLPRUVA, and $2.4 million in net reimbursements from the French EAP for arimoclomol [6][31] - Operating expenses for Q3 2025 were $20.4 million, down from $27.2 million in Q3 2024, with R&D expenses decreasing to $3.4 million from $10.9 million [11][31] - The net loss for Q3 2025 was $(0.5) million, or $(0.01) per share, compared to a net loss of $(33.2) million, or $(0.69) per share, in Q3 2024 [11][31] Product Performance - MIPLYFFA has achieved a market access rate of 66% of covered lives, with 137 prescription enrollment forms submitted since its launch [7] - OLPRUVA has reached an 81% market access rate, with 30 prescription enrollment forms submitted [7] - The company is evaluating options for OLPRUVA while maintaining product availability and support services for patients [7] Pipeline and Innovation - A Marketing Authorization Application for arimoclomol for Niemann-Pick disease type C has been validated and is under review by the European Medicines Agency [7] - Celiprolol, an investigational candidate for Vascular Ehlers-Danlos Syndrome, has restarted enrollment in the Phase 3 DiSCOVER trial [27] Cash Position - As of September 30, 2025, the company had cash, cash equivalents, and securities totaling $230.4 million, indicating sufficient financial resources to execute strategic priorities [11][31]

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]

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]