Core Insights - Zevra Therapeutics, Inc. has granted options to purchase a total of 38,000 shares of common stock to two new employees as part of its 2023 Employment Inducement Award Plan [1][2] - Each Inducement Award vests over four years, with 25% vesting on the first anniversary of the employee's start date, and the remaining 75% vesting in three equal annual installments [1] - The awards were approved by the Compensation Committee of the Board of Directors and are intended as an inducement for new employees [2] Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on developing therapies for rare diseases that have limited or no treatment options [3] - The company's mission is to provide life-changing therapeutics to individuals living with rare diseases through innovative, data-driven development and commercialization strategies [3]

Company Overview - Zevra Therapeutics (ZVRA) shares increased by 10.4% to close at $8.95, following a period of 13.8% loss over the past four weeks, indicating a significant recovery in investor sentiment [1][2] - The surge in share price is attributed to positive investor expectations regarding Zevra's growth prospects, particularly related to its drug Miplyffa, which treats Niemann-Pick disease type C [2] Financial Performance - Zevra is expected to report a quarterly loss of $0.04 per share, reflecting a year-over-year change of +94.2%, while revenues are projected to be $26.53 million, up 619.1% from the previous year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, suggesting stability in earnings expectations [4] Industry Context - Zevra Therapeutics operates within the Zacks Medical - Drugs industry, which includes other companies like Nektar Therapeutics (NKTR), that recently saw a 1.2% increase in share price [4] - Nektar's consensus EPS estimate has decreased by 2.2% over the past month, indicating potential challenges within the industry [5]

Core Insights - Zevra Therapeutics, Inc. (NASDAQ:ZVRA) is highlighted as a promising investment opportunity in the biotech sector, particularly due to its recent positive data regarding MIPLYFFA® (arimoclomol) for Niemann-Pick Disease Type C [1][2] - The company focuses on developing therapies for rare diseases, specifically targeting conditions with limited treatment options, including Idiopathic Hypersomnia and Urea Cycle Disorders [2] Group 1 - On September 4, Zevra Therapeutics announced new data supporting the effectiveness of MIPLYFFA® in treating Niemann-Pick Disease Type C at the International Congress of Inborn Errors of Metabolism [1] - A pre-specified analysis indicated that patients on concomitant miglustat who switched from placebo to MIPLYFFA experienced a decline in annual disease progression [2] - Zevra Therapeutics is a commercial-stage company with a product portfolio that emphasizes rare neurological and sleep conditions [2]

Core Insights - Zevra Therapeutics presented new positive data on MIPLYFFA for Niemann-Pick disease type C (NPC) at the International Congress of Inborn Errors of Metabolism (ICIEM) [1][2] - MIPLYFFA has shown efficacy in reducing annual disease progression in patients who switched from placebo while on concomitant miglustat treatment [4] - The company received a nomination for Best Poster Award for its presentation on MIPLYFFA's mechanism of action [2] Data Highlights - MIPLYFFA activates transcription factors that enhance NPC1 protein levels, addressing NPC etiology through multiple pathways [3] - In a pediatric substudy, MIPLYFFA was well tolerated in children aged 6 to under 24 months, with no new safety signals observed [5] - Long-term outcomes from an expanded access program confirmed the effectiveness and safety of MIPLYFFA, consistent with results from the pivotal Phase 3 study [6] Product Information - MIPLYFFA (arimoclomol) is approved for treating neurological manifestations of NPC in patients aged 2 years and older, in combination with miglustat [9] - The drug received FDA approval on September 20, 2024, and has Orphan Medicinal Product designation from the European Medicines Agency [7][8] - MIPLYFFA has been administered to over 270 NPC patients globally, making it part of the most extensive clinical development program for NPC to date [7][8] Company Overview - Zevra Therapeutics focuses on developing therapies for rare diseases with limited treatment options, aiming to provide life-changing therapeutics [19] - The company employs unique, data-driven strategies to overcome drug development challenges in the rare disease community [19]

Summary of Zevra Therapeutics (ZVRA) 2025 Conference Call Company Overview - Zevra Therapeutics is a commercial stage company focused on rare diseases with two current commercial products: one for Niemann Pick disease type C and another for urea cycle disorders with a nitrogen scavenger called Opruva [6][7] - The company is developing a late-stage program for vascular Ehlers Danlos syndrome (VEDS) and has out-licensed a program for ADHD, receiving royalties and milestones [7] Key Products and Market Insights - **Niemann Pick Disease Type C (NPC)**: - Approximately 900 patients in the U.S. and 1,100 in Europe are affected by NPC [9] - The disease is characterized by cholesterol buildup leading to cell death and various neurological symptoms [11] - The company has published data showing disease modification with their product, demonstrating halting of disease progression over 12 months and up to five years [13] - **Opruva for Urea Cycle Disorders (UCDs)**: - The product has been slower to gain traction than anticipated, but the company is focusing on active patients who would benefit from its advantages [41] - **Vascular Ehlers Danlos Syndrome (VEDS)**: - There are about 7,500 patients in the U.S. with VEDS, and the company is conducting a trial to potentially become the first approved treatment in the U.S. [43] Financial Position - The company has a strong balance sheet with approximately $217 million in cash [7] - Metrics shared indicate a successful launch, with 129 patients enrolled out of 300-350 diagnosed NPC patients in the U.S. [21] - Coverage metrics show 52% of lives covered by payers, with a target of reaching 78-79% similar to Opruva [22] Regulatory and Intellectual Property Developments - The company is in the process of seeking patent term extensions and has filed for a Marketing Authorization Application (MAA) in Europe, which is currently under review [14][32] - The FDA has provided feedback on the patent term extension filing, and the company anticipates a public review process [18][15] Market Dynamics and Competitive Landscape - The company views its products as complementary to others in the market, with a focus on educating physicians and patients about the disease and treatment options [19] - The presence of two approved therapies in the U.S. is seen as beneficial for overall market education and patient access [18] Future Focus and Strategy - The company aims to unlock the undiagnosed patient population and continue to educate physicians through initiatives like Continuing Medical Education (CME) programs [24] - Plans to ramp up the VEDS program and expand the European market presence are in place, with ongoing investments in genetic centers to identify appropriate patients for trials [46][52] Conclusion - Zevra Therapeutics is positioned well in the rare disease market with a strong financial foundation and promising product pipeline. The focus on education, regulatory advancements, and market penetration strategies will be critical for future growth and success in both the U.S. and European markets [48][49]

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.

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]