Core Insights - Zevra Therapeutics reported a quarterly earnings of $1.21 per share, which was below the Zacks Consensus Estimate of $2.19, resulting in an earnings surprise of -44.75% [1] - The company posted revenues of $25.88 million for the quarter ended June 2025, exceeding the Zacks Consensus Estimate by 14.35%, and showing significant growth from $4.45 million in the same quarter last year [2] - The stock has increased approximately 40.3% year-to-date, outperforming the S&P 500's gain of 8.4% [3] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.06 on revenues of $24.48 million, while for the current fiscal year, the estimate is $1.36 on revenues of $94.1 million [7] - The estimate revisions trend for Zevra Therapeutics was mixed prior to the earnings release, resulting in a Zacks Rank 3 (Hold) for the stock, indicating expected performance in line with the market [6] Industry Context - The Medical - Drugs industry, to which Zevra Therapeutics belongs, is currently ranked in the top 35% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5]

Summary of Zebra Therapeutics Conference Call Company Overview - **Company**: Zebra Therapeutics - **Focus**: Rare diseases, specifically ultra-rare diseases - **Products**: - MyPlifa (adimoclomol) for Niemann Pick type C - Alpruva for certain urea cycle disorders - Pipeline includes a program for vascular Ehlers Danlos syndrome (in phase three) and KP1077 for idiopathic hypersomnia (phase two) [1][7][6] Key Points on MyPlifa - **Patient Enrollment**: - 129 patient enrollment forms submitted, with 7 added in the latest quarter - Represents approximately 40% of the diagnosed patient population in the U.S. (300-350 diagnosed out of 900 total prevalence) [10][11][12] - **Market Share Growth**: - Achieved a 2% market share growth with the addition of 7 patients in a quarter [12] - Revenue growth of 26% quarter-over-quarter from Q1 to Q2 [13][15] - **Treatment Efficacy**: - MyPlifa is the only drug shown to halt disease progression for up to five years [14] - **Reimbursement Trends**: - 52% of covered lives have MyPlifa on formulary, with expectations for this number to increase [30][32] - High success rate in securing coverage for patients despite initial denials [30][15] Competitive Landscape - **Miglustat**: - Off-label standard of care for Niemann Pick type C, with 80-90% of diagnosed patients having been on it [19][20][21] - Combination therapy with MyPlifa shows synergistic effects [41] - **Alpruva**: - Launched in a mature market with competition from Revicti and an upcoming authorized generic [58] - Recognized an impairment charge due to enrollment trends and market dynamics [60] Pipeline and Future Prospects - **Vascular Ehlers Danlos Syndrome**: - Ongoing phase three trial with an interim analysis planned [63][64] - **Cash Position**: - Cash balance of $2.17 billion after selling a priority review voucher, with neutral cash burn during the quarter [66] Additional Insights - **Market Awareness**: - Increased awareness of MyPlifa among community physicians, leading to more undiagnosed patients being identified [34] - **Regulatory Strategy**: - Filed for MAA in Europe ahead of schedule, with a robust data package to support approval [48][49] - **Intellectual Property**: - Orphan drug exclusivity and ongoing efforts to extend patent life [43] This summary encapsulates the key discussions and insights from the Zebra Therapeutics conference call, highlighting the company's strategic focus, product performance, and market dynamics.

Financial Data and Key Metrics Changes - Net revenue for Q2 2025 reached $25.9 million, reflecting a 26% increase from Q1 2025, which had revenue of $17.1 million [6][30] - Adjusted net loss for Q2 2025 was $3.2 million, compared to a GAAP net loss of $19.9 million in Q2 2024 [30] - Net income for the quarter was $74.7 million, or $1.24 per basic share and $1.21 per diluted share, largely due to a one-time sale of a PRV asset [30] Business Line Data and Key Metrics Changes - Myclypha generated $21.5 million in revenue for Q2 2025, with a 26% quarter-over-quarter growth [18][30] - Alprova contributed $0.3 million in revenue during the same period, indicating slow uptake [30] - The company recognized a non-cash impairment charge of $58.7 million related to Alprova due to market dynamics [28] Market Data and Key Metrics Changes - Coverage for Myclypha reached 52% of all covered lives in the U.S. as of Q2 2025 [19] - In Europe, the company ended Q2 2025 with 89 patients enrolled in its expanded access program, showing growth from previous quarters [41] Company Strategy and Development Direction - The company aims to build a leading life sciences company focused on rare diseases, driven by four strategic pillars: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation [5] - The submission of the marketing authorization application for Aramchol in Europe marks a significant step in geographic expansion efforts [13] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the early stages of Myclypha's launch and its potential to drive long-term success [30] - The company is focused on increasing awareness and shortening the time to diagnosis for NPC, which is critical for patient treatment [11][70] Other Important Information - The company has a solid balance sheet with total cash and investments of $217.7 million as of June 30, 2025, compared to $68.7 million at the end of the prior quarter [30] - Total debt was approximately $60.7 million, indicating a strong financial position to support strategic priorities [31] Q&A Session Summary Question: Trends in new patient enrollment for Myclypha in the U.S. - Management noted a strong performance with 129 total enrollments by the end of Q2, representing about one-third of diagnosed patients [36][66] Question: Conversion of EAP patients in Europe - Management indicated that the growth in the EAP program in Europe bodes well for future patient conversions post-approval [42] Question: Patients diagnosed but not receiving treatment - Management explained that these patients are typically those diagnosed before therapies were available and are now returning for treatment [46] Question: Average time from enrollment to getting a patient on reimbursed drug - Management stated that the time frame is improving, with some patients getting covered within 72 hours [61] Question: Investment in commercializing Alprova - Management highlighted that the commercial infrastructure for Alprova is synergistic with Myclypha, making it difficult to separate the costs [62] Question: Growth in patient enrollment forms going forward - Management expressed optimism about continued growth in patient enrollments, leveraging awareness and clinical differentiation strategies [68]

PART I — FINANCIAL INFORMATION [Item 1. Financial Statements](index=6&type=section&id=ITEM%201.%20FINANCIAL%20STATEMENTS) The company's unaudited financial statements show a significant net income increase due to a PRV sale [Unaudited Condensed Consolidated Balance Sheets](index=6&type=section&id=UNAUDITED%20CONDENSED%20CONSOLIDATED%20BALANCE%20SHEETS) Condensed Consolidated Balance Sheets (in thousands) | | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Total current assets** | $226,337 | $86,027 | | **Total assets** | **$256,277** | **$178,127** | | **Total current liabilities** | $28,832 | $34,065 | | **Total liabilities** | $139,047 | $138,461 | | **Total stockholders' equity** | **$117,230** | **$39,666** | - Total assets increased to **$256.3 million** as of June 30, 2025, from $178.1 million at the end of 2024, driven by higher cash and securities[17](index=17&type=chunk) - Total stockholders' equity saw a substantial increase to **$117.2 million** from $39.7 million[17](index=17&type=chunk) [Unaudited Condensed Consolidated Statements of Operations](index=8&type=section&id=UNAUDITED%20CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20OPERATIONS) Condensed Consolidated Statements of Operations | (in thousands) | 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 net income (loss) per share** | $1.21 | $(0.48) | $1.16 | $(0.87) | - The company reported net income of **$74.7 million** for Q2 2025, a significant turnaround from a net loss of $19.9 million in Q2 2024[18](index=18&type=chunk) - This improvement was driven by a **$148.3 million gain** on the sale of a Priority Review Voucher (PRV), offsetting a $58.7 million impairment charge[18](index=18&type=chunk) [Unaudited Condensed Consolidated Statements of Cash Flows](index=12&type=section&id=UNAUDITED%20CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20CASH%20FLOWS) Condensed Consolidated Statements of Cash Flows | (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 and cash equivalents** | $13,927 | $(3,789) | | **Cash and cash equivalents, end of period** | $47,712 | $39,260 | - For the first six months of 2025, net cash used in operating activities improved to **$11.8 million** from $35.3 million in the prior year period[27](index=27&type=chunk) - Investing activities provided **$22.5 million** in cash, primarily from the $150.0 million proceeds from the PRV sale[27](index=27&type=chunk) [Notes to Unaudited Condensed Consolidated Financial Statements](index=14&type=section&id=NOTES%20TO%20UNAUDITED%20CONDENSED%20CONSOLIDATED%20FINANCIAL%20STATEMENTS) Notes detail the $150.0 million PRV sale, a $58.7 million asset impairment, and revenue recognition policies - The company sold a transferable rare priority review voucher (PRV) for aggregate proceeds of **$150.0 million**, resulting in a net gain of $148.3 million[41](index=41&type=chunk) - In Q2 2025, the company recorded an intangible asset impairment charge of **$58.7 million** related to OLPRUVA due to decreased future cash flow expectations[113](index=113&type=chunk) - As of June 30, 2025, the company had **$60.7 million** in long-term debt outstanding from a credit agreement, maturing in 2029[73](index=73&type=chunk) Revenue Breakdown (Six Months Ended June 30, 2025) | Revenue Source | Amount (in millions) | | :--- | :--- | | MIPLYFFA Sales | $38.6 | | Arimoclomol French AC | $5.0 | | AZSTARYS License Agreement | $2.1 | | OLPRUVA Sales | $0.4 | | **Total Revenue** | **$46.3** | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=39&type=section&id=ITEM%202.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) Management discusses its rare-disease strategy, financial results, and confirms sufficient liquidity for operations - The company's five-year strategic plan focuses on transforming Zevra into a **leading rare-disease company**[127](index=127&type=chunk) - Following FDA approval, MIPLYFFA became commercially available, and the company sold the associated PRV for net proceeds of **$148.3 million**[129](index=129&type=chunk)[138](index=138&type=chunk) - Due to revised commercial expectations for OLPRUVA, the company recorded a significant intangible asset impairment charge of **$58.7 million**[146](index=146&type=chunk) - As of June 30, 2025, the company had cash, cash equivalents, and investments totaling **$217.7 million**, sufficient to fund operations for at least twelve months[193](index=193&type=chunk)[214](index=214&type=chunk) Results of Operations | (in thousands) | Three months ended June 30, 2025 | Three months ended June 30, 2024 | | :--- | :--- | :--- | | **Revenue, net** | $25,881 | $4,449 | | **Impairment of intangible assets** | $58,710 | $— | | **Gain on sale of PRV** | $148,325 | $— | | **Net income (loss)** | $74,707 | $(19,925) | [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=59&type=section&id=ITEM%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) The company, as a smaller reporting company, is not required to provide this information - The company has determined that this section is **not applicable**[222](index=222&type=chunk) [Item 4. Controls and Procedures](index=59&type=section&id=ITEM%204.%20CONTROLS%20AND%20PROCEDURES) Management concluded that disclosure controls and internal financial reporting controls were effective - Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were **effective** as of June 30, 2025[224](index=224&type=chunk) - There were **no material changes** in the company's internal control over financial reporting during the fiscal quarter ended June 30, 2025[225](index=225&type=chunk) PART II — OTHER INFORMATION [Item 1. Legal Proceedings](index=60&type=section&id=ITEM%201.%20LEGAL%20PROCEEDINGS) The company is defending a breach of contract lawsuit from Commave Therapeutics SA regarding the AZSTARYS agreement - A legal dispute has arisen with Commave Therapeutics SA concerning the interpretation of the **AZSTARYS License Agreement**[227](index=227&type=chunk) - Commave filed a complaint against Zevra in September 2024 alleging breach of contract, and the case is now in the **discovery phase**[228](index=228&type=chunk)[229](index=229&type=chunk) - Zevra believes the lawsuit is **without merit** and has not recorded any accrual for contingent liability, but expects to incur significant legal expenses[230](index=230&type=chunk)[232](index=232&type=chunk) [Item 1A. Risk Factors](index=60&type=section&id=ITEM%201A.%20RISK%20FACTORS) Key risks include geopolitical issues, asset impairment charges, and impacts from healthcare reform legislation - **Geopolitical disputes and trade policies**, such as tariffs and sanctions, could materially adversely affect the business by impacting manufacturing and costs[235](index=235&type=chunk) - The company recognized a **$58.7 million impairment charge** on definite-lived intangible assets during Q2 2025 and warns of potential future impairments[239](index=239&type=chunk) - **Healthcare reform legislation**, including the Inflation Reduction Act, could increase costs and create downward pressure on product pricing[240](index=240&type=chunk)[244](index=244&type=chunk)[245](index=245&type=chunk) - Failure to comply with complex **governmental pricing programs** like MDRP and the 340B program could lead to significant penalties and fines[255](index=255&type=chunk)[257](index=257&type=chunk) [Item 2. 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) The company reports no unregistered sales or repurchases of its equity securities during the period - There were **no unregistered sales** of equity securities during the reporting period[262](index=262&type=chunk) - There were **no purchases** of equity securities by the company or its affiliates[263](index=263&type=chunk) [Item 3. Defaults Upon Senior Securities](index=68&type=section&id=ITEM%203.%20DEFAULTS%20UPON%20SENIOR%20SECURITIES) The company reports this item is not applicable - The company reports this item is **not applicable**[264](index=264&type=chunk) [Item 4. Mine Safety Disclosures](index=68&type=section&id=ITEM%204.%20MINE%20SAFETY%20DISCLOSURES) The company reports this item is not applicable - The company reports this item is **not applicable**[265](index=265&type=chunk) [Item 5. Other Information](index=68&type=section&id=ITEM%205.%20OTHER%20INFORMATION) No material undisclosed events or changes to director nomination procedures occurred during the quarter - During the three months ended June 30, 2025, no director or officer adopted or terminated a **Rule 10b5-1 trading arrangement**[267](index=267&type=chunk) [Item 6. Exhibits](index=70&type=section&id=ITEM%206.%20EXHIBITS) This section lists all exhibits filed with the Form 10-Q, including officer certifications and XBRL data - This section provides a list of exhibits filed as part of the Form 10-Q, including **officer certifications** and Inline XBRL documents[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 ...

Core Insights - Zevra Therapeutics reported Q2 2025 net revenue of $25.9 million, significantly up from $4.4 million in Q2 2024, driven primarily by product net revenue of $21.8 million from MIPLYFFA [1][8] - The company completed the sale of its Pediatric Rare Disease Priority Review Voucher (PRV) for $150 million, enhancing its balance sheet as it advances commercial launches and development programs [1][5] - Zevra submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency for arimoclomol, aimed at treating Niemann-Pick Disease Type C [1][7] Commercial Highlights - MIPLYFFA achieved 7 prescription enrollments in Q2 2025, totaling 129 since its launch, with market access reaching 52% of covered lives [7] - OLPRUVA saw 1 prescription enrollment in Q2 2025, bringing the total to 29, with market access increasing to 79% of covered lives [7] Financial Highlights - Total revenue for Q2 2025 was $25.9 million, including $21.5 million from MIPLYFFA, $0.3 million from OLPRUVA, and $2.6 million in net reimbursements for arimoclomol [8] - Operating expenses were $24.2 million, with R&D expenses decreasing to $3.4 million, while SG&A expenses increased to $20.8 million [14] - The company reported a net income of $74.7 million for Q2 2025, compared to a net loss of $19.9 million in Q2 2024 [10][14] Pipeline and Innovation Highlights - Arimoclomol has been designated as an Orphan Medicinal Product by the EMA, and the company is on track with its guidance for submission in the second half of 2025 [7][17] - Zevra enrolled 7 patients in the Phase 3 DiSCOVER trial of celiprolol for Vascular Ehlers-Danlos Syndrome during Q2 2025, totaling 39 enrolled patients [7] Corporate Highlights - As of June 30, 2025, Zevra's cash, cash equivalents, and securities totaled $217.7 million, providing sufficient resources for strategic priorities [14] - The company has granted inducement equity awards to 9 individuals totaling 54,875 shares of restricted stock units and 49,750 options to purchase common stock [15][16]

Core Viewpoint - The article emphasizes the importance of confirming the sustainability of stock trends for successful short-term investing, highlighting the utility of a specific screening strategy to identify stocks with strong fundamentals and positive price momentum [1][2]. Group 1: Stock Screening Strategy - The "Recent Price Strength" screen is designed to identify stocks with sufficient fundamental strength to maintain their upward price trends, focusing on those trading in the upper portion of their 52-week high-low range, indicating bullishness [3]. - Zevra Therapeutics (ZVRA) is highlighted as a strong candidate for trend investing, having increased by 43.9% over the past 12 weeks, reflecting investor confidence in its potential [4]. - ZVRA has maintained a price increase of 0.9% over the last four weeks, indicating that the upward trend is still intact, and it is currently trading at 85.2% of its 52-week high-low range, suggesting a potential breakout [5]. Group 2: Fundamental Strength - ZVRA holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate revisions and EPS surprises, which are critical for near-term price movements [6]. - The stock also has an Average Broker Recommendation of 1 (Strong Buy), reflecting high optimism from the brokerage community regarding its near-term price performance [7]. - The article suggests that the price trend for ZVRA is unlikely to reverse soon, indicating a favorable outlook for investors [8].

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 provide life-changing therapeutics to affected individuals [3]. Upcoming Events - Members of Zevra's executive leadership team will participate in a fireside chat at the Canaccord Genuity 45th Annual Growth Conference in Boston, MA, on August 13, 2025, at 1:00 p.m. ET [1]. - Management will also be available for one-on-one meetings with registered conference attendees [2]. Investor Relations - A live webcast of the fireside chat will be accessible via the "Events & Presentations" section on Zevra's Investor Relations website [2].

The consensus EPS estimate for the quarter has been revised 10.53% higher over the last 30 days to the current level. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period. Investors should keep in mind that an aggregate change may not always reflect the direction of estimate revisions by each of the covering analysts. Price, Consensus and EPS Surprise While the sustainability of the immediate price change and future earnings expe ...

Core Points - Zevra Therapeutics, Inc. will report its corporate and financial results for Q2 2025 on August 12, 2025, after market close, followed by a conference call at 4:30 p.m. ET [1] - The audio webcast of the conference call will be available on Zevra's Investor Relations website, with a replay accessible for 90 days [2] - Zevra is a commercial-stage company focused on developing therapies for rare diseases, aiming to provide life-changing treatments where options are limited or nonexistent [3] Company Overview - Zevra Therapeutics combines science, data, and patient needs to create transformational therapies for rare diseases [3] - The company employs unique, data-driven strategies for drug development and commercialization to address complex challenges in making new therapies available [3]