Zevra Therapeutics, Inc. (NASDAQ:ZVRA) is one of the best affordable biotech stocks to invest in now. On September 4, Zevra Therapeutics, Inc. (NASDAQ:ZVRA) announced positive new data supporting the foundational role of MIPLYFFA® (arimoclomol) for the treatment of Niemann-Pick Disease Type C at the International Congress of Inborn Errors of Metabolism (ICIEM). The new data from pre-specified analysis showed that “patients on concomitant miglustat who switched from placebo to MIPLYFFA experienced a decli ...

New data from pre-specified analysis shows patients on concomitant miglustat who switched from placebo to MIPLYFFA experienced a decline in annual disease progression Nomination for Best Poster Award received for poster highlighting MIPLYFFA’s differentiated mechanism of action targets underlying pathology of NPC CELEBRATION, Fla., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living ...

Zevra Therapeutics (ZVRA) 2025 Conference September 03, 2025 11:30 AM ET Company ParticipantsNeil McFarlane - President, CEO & DirectorConference Call ParticipantsKristen Kluska - Equity Research AnalystKristen KluskaHi, good morning everybody. I'm Kristen Kluska at Cantor, very happy to be hosting Neal McFarlane at Zevra Therapeutics. Thank you for being here today.Neil McFarlaneThanks for having us.Kristen KluskaThis is our first fireside together since I launched coverage earlier in the year so it's been ...

Core Insights - Zevra Therapeutics, Inc. announced the presentation of four posters on MIPLYFFA® (arimoclomol) at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan, from September 2-6, 2025 [1][2] Product Overview - MIPLYFFA is approved in the U.S. for treating Niemann-Pick disease type C (NPC) and has shown a unique mechanism of action targeting the underlying pathophysiology of NPC [2][6] - The drug received FDA approval on September 20, 2024, and has demonstrated the ability to halt disease progression over a one-year trial compared to placebo, as measured by the NPC Clinical Severity Scale [6] Clinical Data Presentation - A poster detailing the safety and efficacy of arimoclomol in a pediatric substudy for patients aged 6 to <24 months will be presented on September 3, 2025 [3] - A poster selected for a Best Poster award will present long-term outcomes from an expanded access program in the U.S. on September 4, 2025 [4] - Additional presentations will include efficacy results from a 12-month double-blind randomized trial and an open-label extension phase for patients treated with miglustat [4] Regulatory and Market Position - MIPLYFFA has received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for NPC treatment, and a Marketing Authorization Application is currently under review by the EMA [6] - The extensive clinical development program for MIPLYFFA includes data from over 270 NPC patients worldwide, showcasing long-term clinical outcomes [6] Company Mission and Strategy - Zevra Therapeutics focuses on developing therapies for rare diseases with limited treatment options, aiming to bring life-changing therapeutics to affected individuals [17]

Core Viewpoint - Zevra Therapeutics, Inc. is actively engaging in upcoming industry events to showcase its commitment to developing therapies for rare diseases [1][2]. Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on creating transformational therapies for rare diseases that have limited or no treatment options [2]. - The company's mission is to provide life-changing therapeutics to individuals living with rare diseases, utilizing unique, data-driven development and commercialization strategies [2]. Upcoming Events - Zevra's executive leadership team will participate in the Cantor Global Healthcare Conference on September 3, 2025, at 11:30 a.m. ET [5]. - The company will also be present at the H.C. Wainwright 27th Annual Global Investment Conference on September 8, 2025, at 10:00 a.m. ET [5]. - Management will be available for one-on-one meetings with registered conference attendees, and live webcasts will be accessible on the Investor Relations section of Zevra's website [1].

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.

Zevra Therapeutics (ZVRA) Q2 2025 Earnings Call August 12, 2025 04:30 PM ET Speaker0Good afternoon and thank you for joining Zebra's Second Quarter twenty twenty five Financial Results and Corporate Update Conference Call. Today's call is being recorded and will be available via the Investor Relations section of the company's website later today. The host for today's call is Nicole Osher, Zebra's Vice President of Investor Relations and Corporate Communications. Please go ahead.Speaker1Thank you, and welcom ...

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 ...