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 605.5%[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] - For the nine months ended September 30, 2025, Zevra reported a net income of $71,064,000, a significant improvement compared to a net loss of $69,772,000 for the same period in 2024[27] - Other income for the nine months ended September 30, 2025, was $146.3 million, a substantial increase from $1.8 million in the same period of 2024, mainly due to the gain on 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 162.5%[18] - Total liabilities decreased slightly to $136,951,000 as of September 30, 2025, from $138,461,000 as of December 31, 2024, a decline of 1.1%[18] - 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 235.5%[18] - As of September 30, 2025, total receivables amounted to $16.8 million, an increase from $10.5 million as of December 31, 2024[99] Cash Flow and Financing - Cash and cash equivalents rose to $54,439,000 as of September 30, 2025, up from $33,785,000 as of December 31, 2024, an increase of 61.1%[18] - 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 the same period of 2024[27] - 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 the same period of 2024[27] - As of September 30, 2025, the company had cash, cash equivalents, and investments totaling $230.4 million, primarily funded through debt, equity issuance, and product sales[206] 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, representing a reduction of 7.5%[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] - 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] Stock and Equity - Basic net loss per share improved to $(0.01) for the three months ended September 30, 2025, compared to $(0.69) for the same period in 2024[19] - Total stock-based compensation expense for the three months ended September 30, 2025, was $2,771,000, a decrease of 54.8% compared to $6,137,000 for the same period in 2024[112] - Stock-based compensation expense for the nine months ended September 30, 2025, was $8,350,000, down from $10,888,000 in the prior year[27] - The balance of common stock warrant liabilities as of September 30, 2025, was $13,767,000, down from $17,804,000 as of December 31, 2024, reflecting a decrease of 22.8%[118] Regulatory and Product Development - The FDA approved MIPLYFFA (arimoclomol) on September 20, 2024, as the first treatment for Niemann-Pick disease type C, marking a significant milestone for the company[29] - The company is focusing on obtaining regulatory approval for MIPLYFFA in Europe, having submitted a Marketing Authorization Application (MAA) in July 2025[155] - MIPLYFFA generated sales of $22.4 million and $61.0 million for the three and nine months ended September 30, 2025, respectively[88] - OLPRUVA, approved in the U.S. for chronic management of Urea Cycle Disorders (UCDs), began generating revenue in Q4 2023[160] Acquisitions and Partnerships - The Company completed the acquisition of Acer Therapeutics Inc. on November 17, 2023, consolidating Acer's assets and liabilities as a variable interest entity (VIE) under its control[44] - The company licensed certain intellectual property related to a pre-clinical stage prodrug of dextrorphan in April 2025 for an upfront payment of $250,000, with potential future regulatory milestones of up to $8.45 million[142] - Commave is responsible for commercialization activities for AZSTARYS in the U.S., with milestone payments potentially totaling up to $63.0 million upon specified regulatory milestones[181] 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] - For the three and nine months ended September 30, 2025, the Company recognized charges of approximately $11.7 million and $0 related to write-downs for unsaleable inventory, respectively[59] Market and Incidence - 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] - As of September 30, 2025, there were 137 enrollments for MIPLYFFA, focusing on raising awareness among undiagnosed and untreated NPC patients[153]

Zevra Reports Third Quarter 2025 Financial Results and Corporate Update 2025 EPS of $(0.01) Q3 2025 net revenue of $26.1 million, driven by MIPLYFFA® net revenue of $22.4 million Company to host conference call and webcast TODAY, November 5, 2025, at 4:30 p.m. ET CELEBRATION, Fla., November 5, 2025 -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today reported its financial results for the th ...

[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 ...

[Q1 2025 Executive Summary](index=1&type=section&id=Q1%202025%20Executive%20Summary) Zevra reported Q1 2025 net revenue of $20.4 million, bolstered by a $150 million PRV sale, and progressed its rare disease pipeline Q1 2025 Key Financial Overview | Metric | Q1 2025 | | :----- | :------ | | Net Revenue | $20.4 million | | Product Net Revenue | $17.2 million | - Completed sale of Pediatric Rare Disease Priority Review Voucher (PRV) for gross proceeds of **$150 million**, significantly bolstering the balance sheet for commercial launches and development programs[1](index=1&type=chunk)[2](index=2&type=chunk)[5](index=5&type=chunk) - Company is transforming into a patient-centric, commercial-stage rare disease therapeutics company, focusing on establishing MIPLYFFA® globally, maximizing OLPRUVA®'s commercial opportunity, and supporting growth through its development pipeline[2](index=2&type=chunk) [Commercial and Pipeline Progress](index=1&type=section&id=Commercial%20and%20Pipeline%20Progress) Zevra advanced commercial products MIPLYFFA® and OLPRUVA® with new enrollments and expanded market access, and progressed its celiprolol pipeline [Commercial Highlights](index=1&type=section&id=Commercial%20Highlights) - All active patients in the U.S. Expanded Access Program (EAP) for arimoclomol have been enrolled to receive MIPLYFFA, and the EAP has since closed[6](index=6&type=chunk) MIPLYFFA® Commercial Metrics (Q1 2025) | Metric | Q1 2025 | Total (End of Q1 2025) | Market Access | | :----- | :------ | :--------------------- | :------------ | | New Prescription Enrollment Forms | 13 | 122 | 38% of covered lives | OLPRUVA® Commercial Metrics (Q1 2025) | Metric | Q1 2025 | Total (Since July 2023) | Market Access | | :----- | :------ | :---------------------- | :------------ | | New Patient Enrollment Forms | 5 | 28 | 78% of covered lives | [Pipeline and Innovation Highlights](index=1&type=section&id=Pipeline%20and%20Innovation%20Highlights) - Filing of the MIPLYFFA MAA with the European Medicines Agency remains on track for the second half of 2025[6](index=6&type=chunk) - Enrolled **5 additional patients** in the Phase 3 DiSCOVER trial of celiprolol for Vascular Ehlers-Danlos Syndrome, bringing the total to **32 patients**[6](index=6&type=chunk) - Out-licensed IP related to a pre-clinical prodrug, entitling Zevra to a nominal upfront cash payment, potential future regulatory milestones, and mid-single-digit royalties on net sales[6](index=6&type=chunk) [Corporate Milestones and Upcoming Events](index=1&type=section&id=Corporate%20Highlights%20and%20Upcoming%20Milestones) - On April 7, 2025, Zevra announced closing of the sale of its Pediatric Rare Disease Priority Review Voucher ('PRV') for gross proceeds of **$150 million**[5](index=5&type=chunk) - Zevra will participate in a fireside chat at the H.C. Wainwright 3rd Annual Bioconnect Investor Conference on Tuesday, May 20, 2025[8](index=8&type=chunk) [Detailed Financial Results](index=2&type=section&id=Detailed%20Financial%20Results) Zevra reported Q1 2025 net revenue of $20.4 million, a reduced net loss of ($3.1) million, and a cash position of $68.7 million, bolstered by a PRV sale [Q1 2025 Financial Highlights (Summary)](index=2&type=section&id=Q1%202025%20Financial%20Highlights) Q1 2025 Key Financial Highlights | Metric | Q1 2025 | Q1 2024 | Change (YoY) | | :-------------------------------- | :-------- | :-------- | :----------- | | Revenue, Net | $20.4 million | $3.4 million | +$17.0 million | | - MIPLYFFA net revenue | $17.1 million | N/A | N/A | | - OLPRUVA net revenue | $0.1 million | N/A | N/A | | - French EAP reimbursements | $2.3 million | N/A | N/A | | - Royalties (AZSTARYS®) | $0.9 million | N/A | N/A | | Cost of Goods Sold | $3.0 million | N/A | N/A | | Operating Expenses | $22.8 million | $22.2 million | +$0.6 million | | R&D expense | $3.3 million | $12.3 million | -$9.0 million | | SG&A expense | $19.5 million | $9.9 million | +$9.6 million | | Net Loss | ($3.1) million | ($16.6) million | -$13.5 million | | Net Loss per Share (Basic & Diluted) | ($0.06) | ($0.40) | -$0.34 | - R&D expense decreased by **$9.0 million** compared to Q1 2024, primarily due to decreased third-party costs upon completion of the KP1077 Phase 2 trial and reduced personnel-related costs[10](index=10&type=chunk) - SG&A expense increased by **$9.6 million** compared to Q1 2024, mainly due to increased personnel-related costs, professional fees, and other expenses associated with commercial, medical, and launch activities[10](index=10&type=chunk) Cash Position and Shares Outstanding | Metric | As of Mar. 31, 2025 | Pro Forma (incl. PRV sale) | As of Dec. 31, 2024 | | :-------------------------------- | :------------------ | :------------------------- | :------------------ | | Cash, cash equivalents and investments | $68.7 million | $217.0 million | $69.5 million (Cash & Securities) | | Common Stock Outstanding | 54,679,363 | N/A | 53,670,709 | | Fully Diluted Common Shares | 67,897,631 | N/A | N/A | [Unaudited Condensed Consolidated Statements of Operations](index=6&type=section&id=UNAUDITED%20CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20OPERATIONS) Unaudited Condensed Consolidated Statements of Operations (Q1 2025 vs Q1 2024) | Metric (in thousands) | Three months ended March 31, 2025 | Three months ended March 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Revenue, net | $20,401 | $3,425 | | Cost of product revenue (excl. amortization) | $1,345 | $175 | | Intangible asset amortization | $1,615 | $1,528 | | Research and development | $3,258 | $12,277 | | Selling, general and administrative | $19,545 | $9,931 | | Total operating expenses | $22,803 | $22,208 | | Loss from operations | ($5,362) | ($20,486) | | Total other income | $3,445 | $3,794 | | Income (loss) before income taxes | ($1,917) | ($16,692) | | Income tax (expense) benefit | ($1,182) | $70 | | Net loss | ($3,099) | ($16,622) | | Basic and diluted net loss per share | ($0.06) | ($0.40) | | Weighted average shares outstanding | 54,095,543 | 41,778,774 | [Unaudited Condensed Consolidated Balance Sheets](index=7&type=section&id=UNAUDITED%20CONDENSED%20CONSOLIDATED%20BALANCE%20SHEETS) Unaudited Condensed Consolidated Balance Sheets (Mar 31, 2025 vs Dec 31, 2024) | Metric (in thousands) | March 31, 2025 | December 31, 2024 | | :-------------------------------- | :------------- | :---------------- | | **Assets** | | | | Cash and cash equivalents | $37,340 | $33,785 | | Securities at fair value, current | $25,291 | $35,711 | | Total current assets | $81,573 | $86,027 | | Total assets | $172,708 | $178,127 | | **Liabilities** | | | | Accounts payable and accrued expenses | $17,819 | $25,456 | | Total current liabilities | $26,976 | $34,065 | | Long-term debt | $60,090 | $59,504 | | Warrant liability | $13,030 | $17,804 | | Total liabilities | $131,685 | $138,461 | | **Stockholders' Equity** | | | | Total stockholders' equity | $41,023 | $39,666 | [Product Information](index=3&type=section&id=Product%20Information) This section details Zevra's key products: MIPLYFFA® for NPC, OLPRUVA® for UCDs, and celiprolol for VEDS, including mechanisms, indications, and safety [About MIPLYFFA® (arimoclomol)](index=3&type=section&id=About%20MIPLYFFA%C2%AE%20(arimoclomol)) - MIPLYFFA® (arimoclomol) is Zevra's FDA-approved therapy for Niemann-Pick disease type C (NPC), approved on September 20, 2024[12](index=12&