Company Overview - Zevra Therapeutics (ZVRA) shares increased by 5.1% to close at $9.41, with trading volume significantly higher than usual [1] - The stock has shown a 0.9% gain over the past four weeks [1] Product Performance - The rise in stock price is linked to positive investor sentiment regarding the initial uptake of Miplyffa (arimoclomol), which received FDA approval for Niemann-Pick disease type C in September 2024 [2] - Miplyffa generated sales of $17.1 million in Q1 2025 [2] Earnings Expectations - Zevra Therapeutics is projected to report quarterly earnings of $0.65 per share, reflecting a year-over-year increase of 235.4% [3] - Expected revenues for the upcoming quarter are $71.83 million, representing a 1514.2% increase from the same quarter last year [3] - The consensus EPS estimate has remained unchanged over the last 30 days, indicating a potential lack of momentum in earnings estimate revisions [4] Industry Context - Zevra Therapeutics is categorized under the Zacks Medical - Drugs industry, which includes other companies like Xeris Biopharma (XERS) [5] - Xeris Biopharma's stock closed 1.5% higher at $4.73, with a slight return of 0.2% over the past month [5] - Xeris Biopharma's consensus EPS estimate has also remained unchanged, with a year-over-year change of 70% [6]

June 2025 NasdaqGS: ZVRA 1 A Rare Approach to Therapeutics Cautionary Note Regarding Forward-Looking Statements Presentation may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements relate to future events or our future financial performance. We generally identify forward-looking statements by terminology such as "may," "will," "would," "should," "expects," "plans," "anticipates," "could," "intends," "target," "projects," " ...

Core Points - Zevra Therapeutics, Inc. announced the re-election of Wendy L. Dixon, Ph.D. and Tamara A. Favorito to its Board of Directors, serving until the 2028 Annual Meeting [1][2] - The Board expressed gratitude for stockholder participation and support, viewing the election results as a validation of their strategic direction and progress over the past two years [2][4] - Daniel J. Mangless attempted to nominate alternative candidates but failed to meet SEC proxy rules and the Company's bylaws, resulting in his nominations being disregarded [2][3] Company Strategy and Performance - The Board and management reaffirmed their commitment to driving growth and executing their strategic plan, focusing on creating long-term value for stockholders [2][4] - The election results indicated strong stockholder support for the Board's industry expertise and commitment to delivering value [4] Financial Oversight - Stockholders approved the appointment of Ernst & Young LLP as the independent auditors for the fiscal year ending December 31, 2025 [5] Company Overview - Zevra Therapeutics is a commercial-stage company dedicated to developing therapies for rare diseases, utilizing data-driven strategies to overcome drug development challenges [6]

Core Viewpoint - All three leading proxy advisory firms, ISS, Glass Lewis, and Egan-Jones, have recommended that stockholders vote "FOR" Wendy L. Dixon, Ph.D. and Tamara A. Favorito, while advising to "WITHHOLD" votes for dissident nominees Travis Mickle and Arthur Regan, indicating a lack of compelling case for change from the dissident [1][2][4]. Proxy Advisory Recommendations - The Zevra Board of Directors expressed satisfaction with the unanimous endorsement from the proxy advisors, reinforcing their commitment to stockholder value creation under current leadership [2]. - Proxy advisors concluded that the dissident, Mangless, failed to provide sufficient arguments for electing his nominees, lacking a clear strategy to enhance stockholder value [2][5]. Shareholder Returns - Proxy advisors highlighted significant total shareholder returns under Zevra's current management, with ISS noting that the company has outperformed the broader biotech market during McFarlane's tenure as CEO [3][5]. - Egan-Jones emphasized that the past two years have yielded exceptional returns, surpassing both the total market and the S&P Biotech ETF [5]. Concerns About Dissident Nominees - ISS raised concerns about the potential disruption that electing the dissident nominees could cause, particularly highlighting the risks associated with having a former CEO on the board [5]. - Glass Lewis pointed out that Mangless did not present a meaningful alternative strategy for Zevra, lacking a clear plan to improve operational efficacy [5][13]. - Egan-Jones questioned the relevant expertise of Mangless' nominees, suggesting that their backgrounds do not offer meaningful value to Zevra's board [13].

Zevra Therapeutics (ZVRA) FY 2025 Conference May 20, 2025 12:30 PM ET Speaker0 Alright. Good afternoon, everyone. My name is Brandon Folks. I'm one of the biopharma analysts here at H. C. Wainwright. And joining me for a fireside chat from Zebra Therapeutics is CEO, Neil McFarlane. Neil, thank you very much. Speaker1 Yeah. Thank you for having us. Speaker0 So maybe just to start, do you wanna just spend a few minutes giving people a quick overview of Zebra and the products that you currently have? Speaker1 ...

Financial Performance - Zevra Therapeutics reported a quarterly loss of $0.06 per share, significantly better than the Zacks Consensus Estimate of a loss of $0.21, and an improvement from a loss of $0.40 per share a year ago, representing an earnings surprise of 71.43% [1] - The company posted revenues of $20.4 million for the quarter ended March 2025, exceeding the Zacks Consensus Estimate by 29.12%, compared to revenues of $3.43 million in the same quarter last year [2] Stock Performance - Zevra Therapeutics shares have declined approximately 7.1% since the beginning of the year, while the S&P 500 has seen a smaller decline of 0.6% [3] - The current Zacks Rank for Zevra Therapeutics is 4 (Sell), indicating expectations of underperformance in the near future [6] Earnings Outlook - The consensus EPS estimate for the upcoming quarter is -$0.11 on revenues of $20.67 million, and for the current fiscal year, it is -$0.34 on revenues of $88.1 million [7] - The trend for estimate revisions ahead of the earnings release has been unfavorable, which may impact future stock movements [6] Industry Context - The Medical - Drugs industry, to which Zevra Therapeutics belongs, is currently ranked in the top 29% of over 250 Zacks industries, suggesting a favorable environment for stocks in this sector [8]

Zevra Therapeutics (ZVRA) Q1 2025 Earnings Call May 13, 2025 04:30 PM ET Company Participants Nichol Ochsner - Vice President of Investor Relations & Corporate CommunicationsNeil McFarlane - President & CEOJoshua Schafer - CCOR. LaDuane Clifton - CFO, Secretary & TreasurerSumant Kulkarni - Managing DirectorJason Butler - Managing Director & Biotechnology Equity ResearchEddie Hickman - Vice President Conference Call Participants Lachlan Hanbury-Brown - Biotech Equity Research Analyst Operator Good afternoon ...

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]