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]

[PART I — FINANCIAL INFORMATION](index=3&type=section&id=PART%20I%20%E2%80%94%20FINANCIAL%20INFORMATION) [ITEM 1. Unaudited Condensed Consolidated Financial Statements](index=6&type=section&id=ITEM%201.%20UNAUDITED%20CONDENSED%20CONSOLIDATED%20FINANCIAL%20STATEMENTS) This section presents the unaudited condensed consolidated financial statements for Zevra Therapeutics, Inc. as of March 31, 2025, and for the three-month periods ending March 31, 2025, and 2024, including balance sheets, statements of operations, comprehensive loss, stockholders' equity, cash flows, and accompanying notes Key Financial Statement Data (Q1 2025 vs Q1 2024) | Financial Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | **Revenue, net** | $20,401,000 | $3,425,000 | | **Loss from operations** | $(5,362,000) | $(20,486,000) | | **Net loss** | $(3,099,000) | $(16,622,000) | | **Net loss per share (basic and diluted)** | $(0.06) | $(0.40) | | **Net cash used in operating activities** | $(8,222,000) | $(16,165,000) | Condensed Balance Sheet Highlights | Balance Sheet Item | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Cash and cash equivalents** | $37,340,000 | $33,785,000 | | **Total assets** | $172,708,000 | $178,127,000 | | **Total liabilities** | $131,685,000 | $138,461,000 | | **Total stockholders' equity** | $41,023,000 | $39,666,000 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) The notes provide critical context to the financial statements, detailing the company's business, significant accounting policies, and specific financial items, including FDA approval of MIPLYFFA®, PRV sale, revenue breakdowns, debt obligations, and a legal dispute - On September 20, 2024, the FDA approved the New Drug Application (NDA) for **MIPLYFFA®** for Niemann-Pick disease type C (NPC), granting the company a transferable rare pediatric disease **priority review voucher (PRV)**[27](index=27&type=chunk) - Subsequent to the quarter end, on April 1, 2025, the company sold the PRV for **$150.0 million**, resulting in net proceeds of **$148.3 million**, which is not reflected in the March 31, 2025 financial statements[28](index=28&type=chunk)[132](index=132&type=chunk) Revenue Breakdown (Three months ended March 31, 2025) | Revenue Source | Amount (in millions) | | :--- | :--- | | MIPLYFFA® Sales | $17.1 | | French AC (arimoclomol) | $2.3 | | OLPRUVA® Sales | $0.1 | | AZSTARYS® License Agreement | $0.9 | | **Total Revenue** | **$20.4** | - The company is engaged in a legal dispute with Commave Therapeutics SA regarding the **AZSTARYS® License Agreement**, with litigation in the discovery phase and no contingent liability accrued as of March 31, 2025[130](index=130&type=chunk) - The company has a **$100.0 million** senior secured loan facility, of which **$60.0 million** was drawn as of the closing date, bearing interest at 3-Month Term SOFR plus 7.00% per annum, with a floor of 4.00%[78](index=78&type=chunk)[79](index=79&type=chunk) [ITEM 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=37&type=section&id=ITEM%202.%20MANAGEMENT%27S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) Management discusses the company's financial performance and condition, highlighting its transformation into a leading rare-disease company, covering the commercial launch of MIPLYFFA®, sales of OLPRUVA®, pipeline status, operational results comparison, liquidity, capital resources, and the significant cash infusion from the post-quarter PRV sale [Overview and Product Portfolio](index=37&type=section&id=Overview%20and%20Product%20Portfolio) Zevra is a commercial-stage company focused on rare diseases, with a portfolio including approved products MIPLYFFA® and OLPRUVA®, partnered product AZSTARYS®, and a clinical pipeline featuring celiprolol and KP1077, with the recent FDA approval of MIPLYFFA® and subsequent PRV sale strengthening its financial position - The company's strategic plan focuses on becoming a **leading rare-disease company**, supported by the commercialization of **OLPRUVA®** and the recent approval and launch of **MIPLYFFA®**[136](index=136&type=chunk) Active Commercial and Development Assets | Product / Candidate | Indication | Development Status | | :--- | :--- | :--- | | **MIPLYFFA®** | Niemann-Pick disease type C (NPC) | FDA Approved / European EAP | | **OLPRUVA®** | Urea Cycle Disorders (UCD) | FDA Approved | | **Celiprolol** | Vascular Ehlers-Danlos Syndrome (VEDS) | Clinical - Phase 3 Ongoing | | **KP1077** | Idiopathic Hypersomnia (IH) / Narcolepsy | Clinical - Phase 2 / Phase 1/2 | | **AZSTARYS®** | ADHD | FDA Approved and Partnered | - As of March 31, 2025, there were **122 enrollments** to receive **MIPLYFFA®**, including conversions from the U.S. Expanded Access Program (EAP)[146](index=146&type=chunk) - The **Phase 3 DiSCOVER trial** for **celiprolol** in VEDS has **32 enrolled participants** as of March 31, 2025[163](index=163&type=chunk) [Results of Operations](index=47&type=section&id=Results%20of%20Operations) For the three months ended March 31, 2025, Zevra reported a net loss of $3.1 million, a significant improvement from a $16.6 million net loss in the same period of 2024, driven by a $17.0 million revenue increase primarily from MIPLYFFA® sales, partially offset by increased SG&A expenses and decreased R&D expenses Comparison of Operations (Three months ended March 31) | Line Item (in thousands) | 2025 | 2024 | Change | | :--- | :--- | :--- | :--- | | **Revenue, net** | $20,401 | $3,425 | $16,976 | | **Research and development** | $3,258 | $12,277 | $(9,019) | | **Selling, general and administrative** | $19,545 | $9,931 | $9,614 | | **Loss from operations** | $(5,362) | $(20,486) | $15,124 | | **Net loss** | $(3,099) | $(16,622) | $13,523 | - The **$17.0 million revenue increase** was primarily due to **$17.1 million** in product sales of **MIPLYFFA®**, which was approved in September 2024[187](index=187&type=chunk) - **R&D expenses decreased by $9.0 million**, mainly due to reduced spending for the Phase 2 clinical study of KP1077 and lower personnel costs[190](index=190&type=chunk) - **SG&A expenses increased by $9.6 million**, primarily from higher personnel-related costs and professional fees to build out the commercial organization[191](index=191&type=chunk) [Liquidity and Capital Resources](index=49&type=section&id=Liquidity%20and%20Capital%20Resources) As of March 31, 2025, the company had $68.7 million in cash, cash equivalents, and investments, significantly bolstered by the post-quarter PRV sale for $148.3 million, with management believing these resources, along with access to a $100 million debt facility, are sufficient to fund operations for at least the next twelve months - As of March 31, 2025, the company held cash, cash equivalents, and investments totaling **$68.7 million**[193](index=193&type=chunk) - On April 1, 2025, the company received net proceeds of **$148.3 million** from the sale of its PRV, significantly bolstering its cash position[194](index=194&type=chunk) Cash Flow Summary (Three months ended March 31) | Cash Flow (in thousands) | 2025 | 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(8,222) | $(16,165) | | Net cash provided by investing activities | $10,542 | $14,793 | | Net cash provided by financing activities | $1,607 | $1,217 | - The company believes its available cash, operating cash flow, and access to borrowings are **sufficient to fund capital requirements for at least the next twelve months**[213](index=213&type=chunk) [ITEM 3. Quantitative and Qualitative Disclosures About Market Risk](index=55&type=section&id=ITEM%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) This item is marked as 'Not applicable' in the report - The company has indicated that this section is **not applicable**[221](index=221&type=chunk) [ITEM 4. Controls and Procedures](index=55&type=section&id=ITEM%204.%20CONTROLS%20AND%20PROCEDURES) Management, including the CEO and CFO, evaluated the company's disclosure controls and procedures as of March 31, 2025, concluding they were effective at a reasonable assurance level, with no material changes to internal control over financial reporting during the quarter - As of March 31, 2025, the CEO and CFO concluded that the company's disclosure controls and procedures were **effective**[223](index=223&type=chunk) - **No changes in internal control over financial reporting** occurred during the quarter that materially affected, or are reasonably likely to materially affect, internal controls[224](index=224&type=chunk) [PART II — OTHER INFORMATION](index=56&type=section&id=PART%20II%20%E2%80%94%20OTHER%20INFORMATION) [ITEM 1. Legal Proceedings](index=56&type=section&id=ITEM%201.%20LEGAL%20PROCEEDINGS) The company is involved in a legal dispute with Commave Therapeutics SA regarding the interpretation of the AZSTARYS® License Agreement, with Commave alleging breach of contract, and while the company's motion to dismiss was denied, it believes the lawsuit is without merit and has not recorded any contingent liability - A **dispute** has arisen with Commave Therapeutics SA over the **AZSTARYS® License Agreement**, with Commave filing a complaint on September 4, 2024, alleging breach of contract[227](index=227&type=chunk) - The company's **motion to dismiss was denied** on February 12, 2025, and the case is now in the discovery phase, with the company believing the lawsuit is **without merit**[228](index=228&type=chunk) [ITEM 1A. Risk Factors](index=56&type=section&id=ITEM%201A.%20RISK%20FACTORS) This section states there have been no material changes to the risk factors disclosed in the Annual Report on Form 10-K, but highlights specific risks, including potential adverse effects from political, trade, and regulatory developments, and the company's dependency on retaining key executives and qualified personnel - The report notes **no material changes** from the risk factors in the Annual Report on Form 10-K, but highlights specific risks[233](index=233&type=chunk) - A highlighted risk is that significant **political, trade, and regulatory developments**, such as tariffs or sanctions, could impair development or commercialization efforts[234](index=234&type=chunk) - The company is **highly dependent on its ability to retain key executives and attract qualified personnel** in a competitive market[235](index=235&type=chunk)[238](index=238&type=chunk) [ITEM 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=59&type=section&id=ITEM%202.%20UNREGISTERED%20SALES%20OF%20EQUITY%20SECU%20RITIES%20AND%20USE%20OF%20PROCEEDS) The company reported no unregistered sales of equity securities and no purchases of equity securities by the issuer or affiliated purchasers during the period - There were **no unregistered sales of equity securities** during the reporting period[240](index=240&type=chunk) [ITEM 5. Other Information](index=59&type=section&id=ITEM%205.%20OTHER%20INFORMATION) This section discloses that several of the company's executive officers adopted Rule 10b5-1 trading arrangements in March 2025, intended to satisfy the affirmative defense of Rule 10b5-1(c) for the sale of a specified number of common stock shares over a defined period - In March 2025, several executive officers, including the CEO, CFO, CMO, CCO, and CLO, adopted **Rule 10b5-1 trading arrangements** for the future sale of company stock[248](index=248&type=chunk)[249](index=249&type=chunk)[250](index=250&type=chunk) [ITEM 6. Exhibits](index=60&type=section&id=ITEM%206.%20EXHIBITS) This section lists the exhibits filed with the Form 10-Q, including the Asset Purchase Agreement for the PRV, the Tenth Amended and Restated Non-Employee Director Compensation Policy, and required officer certifications

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]

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.

Company will host conference call at 4:30 p.m. ET on Tuesday, May 13, 2025CELEBRATION, Fla., May 06, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced it will report corporate and financial results for the first quarter 2025 on Tuesday, May 13, 2025. The Company will issue a news release after the market closes and host a conference call/audio webcast at 4: ...

Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on developing therapies for rare diseases with limited or no treatment options, aiming to create transformational therapies that address patient needs [3]. Upcoming Events - Members of Zevra's executive leadership team will participate in a fireside chat at the Citizens Life Science Conference in New York on May 7, 2025, at 11:30 a.m. ET [1]. - Management will also be available for one-on-one meetings with registered attendees at the conference [2]. Investor Relations - Live webcasts of the events will be accessible via the "Events & Presentations" section on Zevra's Investor Relations website [2].

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 published significant findings regarding the mechanism of action of its therapy MIPLYFFA (arimoclomol) for Niemann-Pick disease type C (NPC), highlighting its potential to address the underlying pathology of the disease [1][2][3] Group 1: Mechanism of Action - The publication details how arimoclomol enhances the translocation of translation factors EB and E3 (TFEB & TFE3) from the cytosol to the nucleus, initiating a cascade that upregulates coordinated lysosomal expression and regulation (CLEAR) genes, including NPC1 [2] - Increased expression of CLEAR genes leads to higher NPC1 protein levels in lysosomes, improving cholesterol trafficking and correlating with better neurological behaviors in animal models [2] Group 2: Clinical Significance - MIPLYFFA, approved by the U.S. FDA on September 20, 2024, is indicated for use in combination with miglustat for treating neurological manifestations of NPC in patients aged 2 years and older [3][4] - In a pivotal phase 3 trial, MIPLYFFA demonstrated the ability to halt disease progression compared to placebo, as measured by the NPC Clinical Severity Scale [3] Group 3: Safety Information - Common adverse reactions reported in clinical trials include upper respiratory tract infection, diarrhea, and decreased weight, with hypersensitivity reactions such as urticaria and angioedema noted in some patients [5][8][9] - MIPLYFFA may cause increased serum creatinine levels without affecting glomerular function, primarily occurring within the first month of treatment [7] Group 4: Company Overview - Zevra Therapeutics focuses on developing therapies for rare diseases, aiming to create transformational treatments for conditions with limited or no options [13] - The company employs data-driven strategies to navigate complex drug development challenges, striving to make new therapies accessible to the rare disease community [13]

Financial Transactions - Zevra Therapeutics completed the sale of its Rare Pediatric Disease Priority Review Voucher for gross proceeds of $150.0 million on April 1, 2025[4][5]. - The company received the Priority Review Voucher when MIPLYFFA (arimoclomol) was approved by the FDA for treating neurological manifestations of Niemann-Pick disease type C[4]. - The Asset Sale was executed under a PRV Transfer Agreement dated February 26, 2025[5]. - The press release related to the Asset Sale was issued on April 7, 2025[6][11]. Financial Position - As of March 31, 2025, the company's cash, cash equivalents, and investments totaled approximately $68.7 million[6][8]. - The cash and investment figures are based on preliminary unaudited information and management estimates for the quarter ended March 31, 2025[9]. - The company has not yet completed its financial closing procedures for the quarter ended March 31, 2025, and the figures are subject to change[9]. - The independent registered public accounting firm has not reviewed the preliminary estimates provided[9]. Forward-Looking Statements - Forward-looking statements regarding the company's financial position are subject to various risks and uncertainties[10]. - The company may update forward-looking statements in the future but disclaims any obligation to do so unless required by law[10].