Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on providing therapies for rare diseases, aiming to address unmet medical needs and deliver life-changing therapeutics to affected individuals [3]. Board of Directors Update - Alicia Secor has been appointed to the Board of Directors, bringing over thirty years of experience in the life sciences industry, including leadership roles at Atalanta Therapeutics and Juniper Pharmaceuticals [2]. - Wendy Dixon, PhD, has retired from the Board, effective immediately, after providing strategic counsel during her tenure [2][3]. Leadership Insights - The Chair of the Board, Tamara A. Favorito, expressed confidence that Alicia Secor's expertise will support the company's strategic execution and enhance shareholder value [2]. - CEO Neil F. McFarlane highlighted Alicia's experience in rare diseases as a valuable addition to the team and thanked Wendy for her leadership [3].

Core Points - Zevra Therapeutics, Inc. announced the resignation of its Chief Financial Officer, R. LaDuane Clifton, effective December 31, 2025, to pursue other professional opportunities [1][2] - Clifton has been with the company since 2015, providing leadership and guidance during its transition from a development stage to a commercial-stage company focused on rare diseases [2] - The company is currently searching for a new Chief Financial Officer to continue its growth trajectory [2] Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company dedicated to addressing unmet needs in the treatment of rare diseases [3] - The company's mission is to deliver life-changing therapeutics to individuals living with rare diseases through innovative, data-driven development and commercialization strategies [3] Expanded Access Programs - Zevra Therapeutics offers expanded access programs, which are subject to the company's policy and the laws of each jurisdiction [4]

Summary of Zevra Therapeutics FY Conference Call Company Overview - **Company**: Zevra Therapeutics (NasdaqGS: ZVRA) - **Industry**: Biopharmaceuticals focusing on rare diseases - **Products**: Two commercial products for urea cycle disorders and Niemann Pick disease type C (NPC) [3][4] Key Points Product Launch and Market Penetration - Zevra has successfully launched its product for Niemann Pick disease type C in the U.S., achieving 137 prescription enrollment forms, representing approximately 40% of the diagnosed patient population of 300 to 350 patients [5][14] - The company is actively working to expand its market presence in Europe, with a patient population of about 1,100 for NPC [5][41] - The marketing authorization application for the European market has been validated, with a response expected by the end of the year [5][41] Patient Diagnosis and Education - The company is focusing on educating physicians about NPC to increase awareness and diagnosis rates, particularly in community settings [13][26] - There is a notable increase in newly diagnosed patients due to efforts in disease state awareness and genetic testing [14][15] - The patient journey for NPC varies significantly, with different age groups presenting unique challenges in diagnosis and treatment [9][10] Financial Health - Zevra has a robust balance sheet with approximately $230 million in capital [4] - The company reported a pre-commercial revenue of about $10 million per year through its expanded access program in Europe [51] Clinical Data and Market Research - Recent market research indicates that patients are experiencing improvements in gait and swallowing, and physicians prefer Zevra's product based on long-term data [32][33] - The company has published five-year data reinforcing the efficacy of its treatment, which is crucial for payer acceptance [33][38] Challenges and Future Guidance - The company faces challenges in converting patient enrollment forms to paid prescriptions, but adherence and persistency rates are high [36][37] - Future guidance on patient enrollment and revenue will depend on market dynamics and the ongoing efforts in disease awareness [40] European Market Strategy - Zevra is considering various strategies for its European launch, including potential partnerships to expedite patient access [46][49] - The company has conducted pricing corridor work to prepare for market entry, with different reimbursement processes expected across countries [48][51] Additional Insights - The company is learning from its U.S. launch experience to inform its European strategy, aiming for broad availability across markets [52] - Anecdotal evidence suggests that community physicians are becoming more aware of NPC, leading to increased diagnoses and treatment opportunities [30][32] This summary encapsulates the key points discussed during the Zevra Therapeutics FY Conference Call, highlighting the company's strategic focus, market opportunities, and ongoing challenges in the rare disease sector.

Financial Data and Key Metrics Changes - In Q3 2025, the company reported net revenue of $26.1 million, with $22.4 million attributed to Miplyf, $2.4 million from the French Expanded Access Program, $1.2 million from royalties, and $100,000 from Olpruva [6][18][20] - Operating expenses decreased by 6.8% year-over-year to $20.4 million, while R&D expenses decreased by 7.5% to $3.4 million [19] - The net loss for Q3 2025 was $500,000, significantly improved from a net loss of $33.2 million in Q3 2024 [20] Business Line Data and Key Metrics Changes - Miplyf's performance remains strong, with 137 total prescription enrollment forms received since its launch, indicating approximately 40% of the diagnosed patient population [6][29] - The company has seen a total of eight new prescription enrollment forms in Q3 2025, reflecting ongoing efforts in disease awareness and patient identification [12][29] - Apruva's market penetration remains limited, leading to a decision to scale back sales and marketing efforts for this product [9] Market Data and Key Metrics Changes - The company is preparing to expand Miplyf into the European market, where an estimated 1,100 individuals are living with Niemann-Pick Disease Type C [6][7] - The Marketing Authorization Application for Miplyf in Europe has been validated and is under review, with confidence in the submission based on an expanded data set [7][8] - Coverage for Miplyf has increased to 66%, up from 52%, indicating improved access for patients [16][45] Company Strategy and Development Direction - The company aims to build a leading rare disease company, focusing on Miplyf as a foundational treatment for Niemann-Pick Disease Type C [4][11] - Strategic priorities include expanding into Europe and maintaining a disciplined approach to capital allocation while investing in high-impact opportunities [22][23] - The company is actively recruiting patients for the Discover Trial, evaluating salicylol for vascular Ehlers-Danlos syndrome [9][10] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the continued success of Miplyf and the potential for geographic expansion and patent term extension [11][23] - The company is focused on maintaining cost efficiency and operational accountability to support long-term value creation [22] - Management highlighted the importance of ongoing disease awareness initiatives to identify undiagnosed patients and improve treatment access [14][15] Other Important Information - The company has established a solid patent position for Miplyf and requested a patent term extension with the U.S. Patent and Trademark Office [8] - The Expanded Access Program in Europe is critical for building relationships with physicians and clinics, with 92 patients enrolled by the end of Q3 [8][9] Q&A Session Summary Question: Have any of the new start forms been from newly diagnosed patients? - Management confirmed that many new enrollment forms are from newly diagnosed patients, largely due to disease awareness campaigns and partnerships with genetic testing collaborators [25][27] Question: How is the company thinking about its robust balance sheet? - Management indicated that they are focused on executing the Miplyf launch and exploring opportunities for expansion while maintaining a disciplined approach to capital allocation [30][32] Question: What percentage of patients are getting refills? - Management reported that the majority of patients are receiving refills, with 66% of covered lives today, and emphasized the effectiveness of their reimbursement processes [39] Question: What are the key drivers for the increase in coverage? - The increase in coverage is attributed to the strength of Miplyf's clinical data, which has resonated with payers, and the targeting of Centers of Excellence and community-based physicians [43][45] Question: What are the expectations for the European market? - Management is confident in the MAA submission for Europe and anticipates a favorable reception based on the comprehensive data package [48][49] Question: What is the status of the KP1077 program? - Management remains open to strategic alternatives for KP1077 and is currently in discussions, but no specific updates were provided [58]

Financial Performance - Revenue for the three months ended September 30, 2025, was $26,063,000, a significant increase from $3,695,000 in the same period of 2024, marking a growth of 605%[19] - For the nine months ended September 30, 2025, total revenues reached $72.3 million, compared to $11.6 million for the same period in 2024, indicating a growth of about 522%[86] - Net income for the nine months ended September 30, 2025, was $71,064,000, a significant improvement compared to a net loss of $69,772,000 for the same period in 2024[27] - The company reported a gain on the sale of PRV amounting to $148,325,000 for the nine months ended September 30, 2025[19] - Other income for the nine months ended September 30, 2025, was $146.3 million, compared to $1.8 million in the same period of 2024, mainly due to a gain on the sale of the PRV[205] Assets and Liabilities - Total current assets increased to $225,115,000 as of September 30, 2025, compared to $86,027,000 as of December 31, 2024, representing a growth of 162%[18] - Total liabilities decreased slightly to $136,951,000 as of September 30, 2025, from $138,461,000 as of December 31, 2024[18] - The company’s total assets increased to $270,116,000 as of September 30, 2025, from $178,127,000 as of December 31, 2024, reflecting a growth of 51%[18] - As of September 30, 2025, the Company held investment securities with an aggregate fair value of $175.9 million, up from $41.7 million as of December 31, 2024, reflecting a significant increase in investment holdings[38] - Long-term debt as of September 30, 2025, was $61.3 million, an increase from $59.5 million as of December 31, 2024[85] Equity and Stockholder Information - Stockholders' equity increased significantly to $133,165,000 as of September 30, 2025, compared to $39,666,000 as of December 31, 2024, reflecting an increase of 235%[18] - The balance of stockholders' equity as of September 30, 2024, was $69,770,000, reflecting a decrease from previous periods due to net losses[24] - The fair value of the warrant liability was approximately $13.8 million as of September 30, 2025, down from $17.8 million as of December 31, 2024[106] - The balance of common stock warrant liabilities decreased to $13,767,000 as of September 30, 2025, from $17,804,000 as of December 31, 2024, reflecting a decline of 22.8%[118] Expenses - Operating expenses for the nine months ended September 30, 2025, totaled $67,385,000, a decrease from $72,486,000 in the same period of 2024, showing a reduction of approximately 7%[19] - Research and development expenses directly identified to programs for the three months ended September 30, 2025, were $1.8 million, down from $4.9 million in 2024, a decrease of approximately 64%[77] - Selling, general and administrative expenses directly identified to programs for the three months ended September 30, 2025, were $5.1 million, an increase from $4.9 million in 2024, reflecting a growth of about 4%[77] - Research and development expenses for the nine months ended September 30, 2025, were $3,322,000, down from $4,696,000 in 2024, reflecting a decrease of 29.3%[112] - Selling, general and administrative expenses increased by approximately $18.5 million, from $38.7 million in 2024 to $57.3 million in 2025, driven by higher personnel-related costs and professional fees[204] Cash Flow - Cash flows from operating activities resulted in a net cash used of $7,114,000 for the nine months ended September 30, 2025, compared to $53,415,000 in 2024[27] - The company reported a net cash provided by financing activities of $9,518,000 for the nine months ended September 30, 2025, compared to $81,312,000 in 2024[27] - As of September 30, 2025, the company had cash, cash equivalents, and investments totaling $230.4 million[206] Product Development and Regulatory Approvals - On September 20, 2024, the FDA approved MIPLYFFA (arimoclomol) for Niemann-Pick disease type C, marking it as the first FDA-approved treatment for this condition[29] - MIPLYFFA generated sales of $22.4 million and $61.0 million for the three and nine months ended September 30, 2025, respectively[88] - The company is focusing on obtaining regulatory approval for MIPLYFFA in Europe, having submitted a Marketing Authorization Application (MAA) in July 2025[155] - Celiprolol is being advanced as an investigational product for Vascular Ehlers-Danlos Syndrome (VEDS) and has received orphan drug designation from the FDA[165] - The DiSCOVER trial for celiprolol aims to enroll 150 VEDS patients, with 44 participants enrolled as of September 30, 2025[168] Impairments and Write-downs - The company recognized an impairment of intangible assets amounting to $58,710,000 in 2025, while there was no such charge in 2024[27] - The company recorded an intangible asset impairment charge of $58.7 million for the nine months ended September 30, 2025, related to the OLPRUVA asset group, which was determined to be not fully recoverable[128] - The Company recognized charges of approximately $11.7 million for unsaleable inventory for the nine months ended September 30, 2025, compared to $5.2 million for the same period in 2024[59] Market and Sales Information - The estimated incidence of NPC is one in 100,000 to 130,000 live births, with approximately 2,000 individuals affected in the U.S. and Europe combined, including about 900 in the U.S.[150] - OLPRUVA, approved in the U.S. for chronic management of Urea Cycle Disorders (UCDs), began generating revenue in Q4 2023[160] - The company has generated revenue from product sales of MIPLYFFA and OLPRUVA, as well as reimbursements under the French expanded access program, with royalties and milestone payments contributing to revenue streams[60]

Financial Performance - Q3 2025 net revenue was $26.1 million, with MIPLYFFA® contributing $22.4 million, compared to $3.7 million in Q3 2024[7] - Zevra Therapeutics reported net revenue of $26,063,000 for the three months ended September 30, 2025, compared to $3,695,000 for the same period in 2024, representing a significant increase of 605%[31] - The company reported a net income of $71,064,000 for the nine months ended September 30, 2025, compared to a net loss of $69,772,000 for the same period in 2024[31] Operating Expenses - Operating expenses decreased to $20.4 million in Q3 2025 from $27.2 million in Q3 2024, with R&D expenses down to $3.4 million[7] - The company’s total operating expenses for the nine months ended September 30, 2025, were $67,385,000, a decrease of 7% from $72,486,000 in the same period of 2024[31] - Zevra's research and development expenses for the three months ended September 30, 2025, were $3,432,000, a decrease of 69.7% from $10,945,000 in the same period of 2024[31] - The company’s selling, general and administrative expenses for the nine months ended September 30, 2025, were $57,262,000, an increase of 47.5% from $38,743,000 in the same period of 2024[31] Loss and Cash Position - Net loss for Q3 2025 was $(0.5) million, or $(0.01) per share, significantly improved from a net loss of $(33.2) million, or $(0.69) per share, in Q3 2024[7] - Cash position stood at $230.4 million as of September 30, 2025, providing sufficient resources for strategic priorities[7] - Zevra's cash and cash equivalents increased to $54,439,000 as of September 30, 2025, up from $33,785,000 at the end of 2024, indicating improved liquidity[33] - The company’s accumulated deficit improved to $(434,225,000) as of September 30, 2025, compared to $(505,289,000) at the end of 2024[33] Market Access and Product Development - MIPLYFFA achieved market access for 66% of covered lives, while OLPRUVA reached 81%[5] - A Marketing Authorization Application for arimoclomol is under review by the European Medicines Agency[5] - MIPLYFFA has been designated as an Orphan Medicinal Product by the EMA, highlighting its significance in treating Niemann-Pick disease type C[12] - The company plans to scale back sales and marketing efforts for OLPRUVA while maintaining product availability[5] Clinical Trials - Five patients were enrolled in the Phase 3 DiSCOVER trial for Celiprolol, totaling 44 enrolled patients[5] - MIPLYFFA's clinical data showed a decline in annual disease progression for patients switching from placebo[7] Assets and Liabilities - The total assets of Zevra Therapeutics rose to $270,116,000 as of September 30, 2025, compared to $178,127,000 at the end of 2024, reflecting a growth of 51.7%[33] - Zevra's total liabilities decreased slightly to $136,951,000 as of September 30, 2025, from $138,461,000 at the end of 2024[33] Shareholder Information - The weighted-average shares of common stock outstanding increased to 55,951,572 for the three months ended September 30, 2025, compared to 47,808,817 for the same period in 2024[31]

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, Inc. is participating in Guggenheim's 2nd Annual Healthcare Innovation Conference on November 12, 2025, at 11:30 a.m. ET [1] - The company's executive leadership team will engage in a fireside discussion and will also be available for one-on-one meetings with registered attendees [2] - Zevra focuses on developing therapies for rare diseases with limited or no treatment options, aiming to provide life-changing therapeutics [3] Company Overview - Zevra Therapeutics is a commercial-stage company that combines science, data, and patient needs to create transformational therapies for rare diseases [3] - The company employs unique, data-driven development and commercialization strategies to address complex drug development challenges [3]

Core Viewpoint - Zevra Therapeutics, Inc. will report its third quarter 2025 corporate and financial results on November 5, 2025, followed by a conference call at 4:30 p.m. ET [1] Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on developing therapies for rare diseases that have limited or no treatment options [3] - The company's mission is to provide life-changing therapeutics to individuals living with rare diseases through innovative, data-driven development and commercialization strategies [3] Upcoming Events - The third quarter 2025 results will be released via a news release after market close on November 5, 2025 [1] - An audio webcast of the conference call will be available on the "Events & Presentations" page of Zevra's Investor Relations website [2] - A replay of the webcast will be accessible for 90 days starting approximately at 5:30 p.m. ET on the same day [2]

Core Insights - Zevra Therapeutics (ZVRA) shares increased by 12.3% to close at $11.78, with a notable trading volume, and a total gain of 44.5% over the past four weeks [1][2] Company Performance - The rise in stock price is linked to positive investor sentiment regarding Zevra's growth prospects, particularly due to its drug Miplyffa, which received FDA approval for treating Niemann-Pick disease type C in September 2024, generating $38.6 million in sales during the first half of 2025 [2] - The company is expected to report a quarterly loss of $0.04 per share, reflecting a year-over-year change of +94.2%, with revenues projected at $26.53 million, a significant increase of 619.1% from the previous year [3] - The consensus EPS estimate for the upcoming quarter has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [4] Industry Context - Zevra Therapeutics is categorized under the Zacks Medical - Drugs industry, which includes other companies like OmniAb, Inc. (OABI), that also experienced a stock increase of 6.2% to $1.71 in the last trading session [5] - OmniAb's consensus EPS estimate for the upcoming report has remained unchanged at -$0.15, representing a year-over-year change of +6.3% [6]