[PART I — FINANCIAL INFORMATION](index=3&type=section&id=PART%20I%20%E2%80%94%20FINANCIAL%20INFORMATION) [Financial Statements](index=6&type=section&id=ITEM%201.%20FINANCIAL%20STATEMENTS) Zevra Therapeutics achieved a net income of **$71.6 million** for the six months ended June 30, 2025, a significant turnaround driven by a **$148.3 million** gain from a PRV sale and increased revenue from MIPLYFFA, despite an OLPRUVA impairment. [Condensed Consolidated Balance Sheets](index=7&type=section&id=UNAUDITED%20CONDENSED%20CONSOLIDATED%20BALANCE%20SHEETS) As of June 30, 2025, total assets increased to **$256.3 million** from **$178.1 million** at year-end 2024, primarily due to higher cash and marketable securities, while total stockholders' equity grew substantially to **$117.2 million**. Balance Sheet Summary | Balance Sheet Item | June 30, 2025 ($ in thousands) | December 31, 2024 ($ in thousands) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | 47,712 | 33,785 | | Securities at fair value | 169,988 | 41,721 | | Total current assets | 226,337 | 86,027 | | Intangible assets, net | 7,053 | 68,993 | | **Total assets** | **256,277** | **178,127** | | **Liabilities & Equity** | | | | Total current liabilities | 28,832 | 34,065 | | Long-term debt | 60,692 | 59,504 | | **Total liabilities** | **139,047** | **138,461** | | **Total stockholders' equity** | **117,230** | **39,666** | - The significant decrease in net intangible assets from **$69.0 million** to **$7.1 million** is primarily due to a **$58.7 million** impairment charge related to OLPRUVA recorded in Q2 2025[17](index=17&type=chunk)[113](index=113&type=chunk) [Condensed Consolidated Statements of Operations](index=8&type=section&id=UNAUDITED%20CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20OPERATIONS) The company reported a net income of **$74.7 million** for Q2 2025 and **$71.6 million** for the first six months, a significant improvement from prior-year losses, driven by a **$148.3 million** gain on a PRV sale and increased net revenue, partially offset by a **$58.7 million** OLPRUVA impairment. Statements of Operations Summary | Metric ($ in thousands, except EPS) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Revenue, net | 25,881 | 4,449 | 46,282 | 7,874 | | Impairment of intangible assets | 58,710 | — | 58,710 | — | | Loss from operations | (71,039) | (23,795) | (76,401) | (44,281) | | Gain on sale of PRV | 148,325 | — | 148,325 | — | | **Net income (loss)** | **74,707** | **(19,925)** | **71,608** | **(36,547)** | | Diluted EPS | $1.21 | $(0.48) | $1.16 | $(0.87) | [Condensed Consolidated Statements of Cash Flows](index=12&type=section&id=UNAUDITED%20CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20CASH%20FLOWS) For the six months ended June 30, 2025, net cash used in operating activities decreased to **$11.8 million**, while investing activities provided **$22.5 million**, primarily from **$150.0 million** in PRV sale proceeds, partially offset by investment purchases. Cash Flow Activity Summary | Cash Flow Activity ($ in thousands) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | (11,823) | (35,274) | | Net cash provided by investing activities | 22,476 | 14,664 | | Net cash provided by financing activities | 2,988 | 16,547 | | **Net increase (decrease) in cash** | **13,927** | **(3,789)** | - The primary driver of positive cash flow from investing activities was the **$150.0 million** received from the sale of the Priority Review Voucher (PRV)[27](index=27&type=chunk) [Notes to Financial Statements](index=14&type=section&id=NOTES%20TO%20UNAUDITED%20CONDENSED%20CONSOLIDATED%20FINANCIAL%20STATEMENTS) The notes detail significant events including FDA approval and commercialization of MIPLYFFA, the **$150.0 million** PRV sale, a **$58.7 million** OLPRUVA intangible asset impairment, revenue breakdown, debt structure, and ongoing litigation with Commave. - The company sold a transferable rare priority review voucher (PRV) received with the FDA approval of MIPLYFFA for **$150.0 million**, resulting in a net gain of **$148.3 million** in Q2 2025[41](index=41&type=chunk) - A **$58.7 million** intangible asset impairment charge was recorded in Q2 2025 for OLPRUVA after a triggering event indicated its carrying amount was not recoverable[113](index=113&type=chunk) - For the six months ended June 30, 2025, the company recognized an inventory write-down charge of approximately **$11.7 million** for unsaleable inventory, compared to **$3.2 million** in the same period of 2024[54](index=54&type=chunk) Revenue by Source | Revenue Source ($ in millions) | Six Months Ended June 30, 2025 | | :--- | :--- | | MIPLYFFA Sales | 38.6 | | Arimoclomol French AC | 5.0 | | AZSTARYS License Agreement | 2.1 | | OLPRUVA Sales | 0.4 | | **Total Revenue** | **46.3** | [Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A)](index=39&type=section&id=ITEM%202.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) Management discusses the company's transformation into a commercial-stage rare disease company, highlighting the successful launch of MIPLYFFA, the **$148.3 million** PRV gain, and the **$58.7 million** OLPRUVA impairment, with **$217.7 million** in cash and investments providing sufficient funding for the next twelve months. [Overview](index=39&type=section&id=Overview) Zevra has transformed into a commercial-stage rare disease company with approved products MIPLYFFA and OLPRUVA, driven by the FDA approval of MIPLYFFA, the **$148.3 million** PRV sale, and the acquisition of Acer Therapeutics, focusing on late-stage development. - The company's five-year strategic plan focuses on transforming Zevra into a leading rare-disease company by prioritizing late-stage clinical development and commercial opportunities over in-house drug discovery[127](index=127&type=chunk) - MIPLYFFA was approved by the FDA on September 20, 2024, for Niemann-Pick disease type C (NPC), and the associated Priority Review Voucher (PRV) was sold for net proceeds of **$148.3 million** on April 1, 2025[129](index=129&type=chunk) - The acquisition of Acer Therapeutics was completed on November 17, 2023, adding the FDA-approved product OLPRUVA and the investigational candidate celiprolol to Zevra's portfolio[130](index=130&type=chunk) [Results of Operations](index=49&type=section&id=Results%20of%20Operations) For the six months ended June 30, 2025, net income was **$71.6 million**, a significant improvement driven by a **$38.4 million** increase in revenue from MIPLYFFA sales and a **$148.3 million** PRV gain, partially offset by a **$58.7 million** OLPRUVA impairment. Operations Metrics | Metric ($ in thousands) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | | Revenue, net | 46,282 | 7,874 | 38,408 | | Impairment of intangible assets | 58,710 | — | 58,710 | | Research and development | 6,691 | 22,798 | (16,107) | | Selling, general and administrative | 40,327 | 22,535 | 17,792 | | Gain on sale of PRV | 148,325 | — | 148,325 | | **Net income (loss)** | **71,608** | **(36,547)** | **108,155** | - The increase in net income was primarily attributable to the **$148.3 million** gain on the sale of the PRV and a **$38.4 million** increase in revenue, mainly from MIPLYFFA sales[185](index=185&type=chunk)[186](index=186&type=chunk) - R&D expenses decreased by **$16.1 million**, primarily due to reduced spending on the Phase 2 clinical study for KP1077[190](index=190&type=chunk) - SG&A expenses increased by **$17.8 million**, reflecting higher personnel costs, professional fees, and other expenses related to building the commercial organization[191](index=191&type=chunk) [Liquidity and Capital Resources](index=53&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2025, the company held **$217.7 million** in cash, cash equivalents, and investments, with liquidity bolstered by product sales and **$148.3 million** net proceeds from a PRV sale, and management believes current capital is sufficient for at least the next twelve months. - As of June 30, 2025, the company had cash, cash equivalents and investments of **$217.7 million**[193](index=193&type=chunk) - In August 2024, the company completed an equity offering, raising net proceeds of approximately **$64.5 million** to support commercialization and development activities[199](index=199&type=chunk) - The company has a senior secured loan facility for up to **$100.0 million**, with **$60.0 million** funded as of the Term Loans Closing Date on April 5, 2024[205](index=205&type=chunk) - Management believes available cash and future operating cash flow are sufficient to fund capital requirements for at least the next twelve months[214](index=214&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=59&type=section&id=ITEM%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) This section is not applicable for the reporting period. - Not applicable[222](index=222&type=chunk) [Controls and Procedures](index=59&type=section&id=ITEM%204.%20CONTROLS%20AND%20PROCEDURES) Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were effective as of June 30, 2025, with no material changes to internal control over financial reporting during the quarter. - The CEO and CFO concluded that as of June 30, 2025, the company's disclosure controls and procedures were effective at a reasonable assurance level[224](index=224&type=chunk) - No material changes to internal control over financial reporting occurred during the fiscal quarter ended June 30, 2025[225](index=225&type=chunk) [PART II — OTHER INFORMATION](index=60&type=section&id=PART%20II%20%E2%80%94%20OTHER%20INFORMATION) [Legal Proceedings](index=60&type=section&id=ITEM%201.%20LEGAL%20PROCEEDINGS) The company is engaged in a legal dispute with Commave Therapeutics SA regarding the AZSTARYS License Agreement, with the case currently in the discovery phase, and Zevra intends to vigorously defend itself against the alleged breach of contract. - A legal dispute has arisen with Commave Therapeutics SA concerning the AZSTARYS License Agreement, with Commave filing a complaint in the Court of Chancery of the State of Delaware in September 2024[227](index=227&type=chunk)[228](index=228&type=chunk) - The case is currently in the discovery phase after the company's motion to dismiss was denied in February 2025, and the company believes the lawsuit is without merit but cannot predict the outcome[229](index=229&type=chunk)[230](index=230&type=chunk) [Risk Factors](index=60&type=section&id=ITEM%201A.%20RISK%20FACTORS) The company highlights risks including potential impairment of remaining assets after a **$58.7 million** OLPRUVA charge, the impact of healthcare reforms like the Inflation Reduction Act, challenges in retaining key personnel, and geopolitical disputes affecting supply chains. - Following a **$58.7 million** impairment charge on definite-lived intangible assets in Q2 2025, there is a risk that remaining assets could become impaired in the future, which would negatively affect operating results[239](index=239&type=chunk) - Healthcare reform, including the Inflation Reduction Act of 2022 (IRA) and the fictional One Big Beautiful Bill Act of July 2025, could increase costs, create pricing pressure, and negatively impact the business through measures like Medicare price negotiations and Medicaid funding reductions[240](index=240&type=chunk)[244](index=244&type=chunk)[245](index=245&type=chunk) - The company's success is highly dependent on its ability to retain key executives and qualified scientific, clinical, and commercial personnel in a competitive hiring environment[236](index=236&type=chunk)[238](index=238&type=chunk) - The company faces risks from significant political, trade, and regulatory developments, such as tariffs and sanctions, which could impair development and commercialization efforts by affecting supply chains and increasing costs[235](index=235&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=68&type=section&id=ITEM%202.%20UNREGISTERED%20SALES%20OF%20EQUITY%20SECURITIES%20AND%20USE%20OF%20PROCEEDS) There were no unregistered sales of equity securities or purchases of equity securities by the issuer during the reporting period. - None[262](index=262&type=chunk) [Other Information](index=68&type=section&id=ITEM%205.%20OTHER%20INFORMATION) During the three months ended June 30, 2025, no director or officer of the company adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement. - No director or officer adopted or terminated a Rule 10b5-1 trading arrangement during the quarter[267](index=267&type=chunk) [Exhibits](index=70&type=section&id=ITEM%206.%20EXHIBITS) This section lists the exhibits filed as part of the Form 10-Q, including certifications from the Principal Executive Officer and Principal Financial Officer. - The exhibits filed with this report include Certifications of the Principal Executive Officer and Principal Financial Officer pursuant to SEC rules[268](index=268&type=chunk)

Exhibit 99.1 Zevra Reports Second Quarter 2025 Financial Results and Corporate Update Q2 2025 net revenue of $25.9 million, driven by product net revenue of $21.8 million Completed sale of PRV for $150.0 million, bolstering the balance sheet as the Company executes on its commercial launches and development programs Submitted a Marketing Authorisation Application to the European Medicines Agency for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C Company to host conference cal ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Organization) (I.R.S. Employer Identification No.) 1180 Celebration Boulevard, Suite 103, Celebration, FL 34747 (Address of Principal Executive Offices) (Zip Code) For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period ...

Exhibit 99.1 Zevra Reports First Quarter 2025 Financial Results and Corporate Update Q1 2025 net revenue of $20.4 million, driven by product net revenue of $17.2 million Completed sale of PRV for gross proceeds of $150 million, positioning balance sheet to drive the Company's commercial launches and development programs Company to host conference call and webcast today, May 13, 2025, at 4:30 p.m. ET CELEBRATION, Fla., May. 13, 2025 -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a comm ...