CELEBRATION, Fla., July 16, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, announced the publication of “Long-term Efficacy and Safety of Arimoclomol in Niemann-Pick Disease Type C: Final Results of the Phase 2/3 NPC-002 48-month Open-label Extension Trial” in the peer-reviewed journal, Molecular Genetics and Metabolism (https://doi.org/10.1016/j.ymgme.2025.109189). MIP ...

Core Viewpoint - Zevra Therapeutics (ZVRA) has shown a significant price increase of 40.2% over the past four weeks, with a mean price target of $23.86 indicating an upside potential of 86.6% from the current price of $12.79 [1] Price Targets and Analyst Estimates - The mean estimate consists of seven short-term price targets with a standard deviation of $3.39, where the lowest estimate is $19.00 (48.6% increase) and the highest is $29.00 (126.7% increase) [2] - A low standard deviation among price targets suggests a strong agreement among analysts regarding the stock's price movement [9] Earnings Estimates and Analyst Optimism - Analysts have shown growing optimism regarding ZVRA's earnings prospects, as indicated by a positive trend in earnings estimate revisions, which correlates with potential stock price movements [11] - Over the last 30 days, two earnings estimates for ZVRA have increased, leading to a Zacks Consensus Estimate increase of 145.2% [12] Zacks Rank and Investment Potential - ZVRA holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors, indicating strong potential for upside [13] - While consensus price targets may not be entirely reliable, the direction they imply appears to be a useful guide for potential price movements [14]

Core Viewpoint - The article emphasizes the importance of identifying sustainable trends in stock prices for short-term investing, highlighting that while price movements can be profitable, they can also reverse quickly, leading to potential losses [1][2]. Group 1: Stock Performance - Zevra Therapeutics (ZVRA) has shown a significant price increase of 86.2% over the past 12 weeks, indicating strong investor interest and potential upside [4]. - The stock has also experienced a price increase of 40.2% over the last four weeks, suggesting that the upward trend is still intact [5]. - ZVRA is currently trading at 98.5% of its 52-week high-low range, indicating it may be on the verge of a 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 has an Average Broker Recommendation of 1 (Strong Buy), reflecting high optimism from the brokerage community regarding its near-term performance [7]. Group 3: Investment Strategy - The article suggests that investors can utilize the "Recent Price Strength" screen to identify stocks like ZVRA that are on an upward trend supported by strong fundamentals [3][8]. - It also mentions that there are over 45 Zacks Premium Screens available for investors to find stocks that align with their investment strategies [8].

Core Insights - Zevra Therapeutics, Inc. announced presentations on MIPLYFFA (arimoclomol) at the NNPDF Conference, highlighting its role as the first FDA-approved treatment for Niemann-Pick disease type C (NPC) [1][2] - MIPLYFFA is indicated for use in combination with miglustat for treating neurological manifestations of NPC in patients aged 2 years and older [7][6] Presentation and Research Findings - Dr. Barbara K. Burton presented an overview of MIPLYFFA, emphasizing its unique mechanism that improves lysosomal function and halts disease progression at 12 months in a pivotal study [2][3] - The pivotal trial demonstrated that arimoclomol combined with miglustat halted disease progression compared to placebo, confirmed by a 48-month open-label extension study [4][6] - Two posters were presented: one detailing long-term effectiveness and safety of arimoclomol, and another providing mechanistic evidence of arimoclomol's action through the CLEAR network [4][5] Drug Mechanism and Clinical Significance - MIPLYFFA enhances the activation of transcription factors TFEB and TFE3, leading to the upregulation of CLEAR genes and improved lysosomal function [6][17] - The drug has shown to reduce unesterified cholesterol in NPC fibroblasts, although the clinical significance of this finding remains to be fully understood [6] Company Overview - Zevra Therapeutics focuses on developing therapies for rare diseases with limited treatment options, aiming to create transformational therapies through data-driven strategies [18]

Company Overview - Zevra Therapeutics (ZVRA) shares increased by 5.1% to close at $9.41, with trading volume significantly higher than usual [1] - The stock has shown a 0.9% gain over the past four weeks [1] Product Performance - The rise in stock price is linked to positive investor sentiment regarding the initial uptake of Miplyffa (arimoclomol), which received FDA approval for Niemann-Pick disease type C in September 2024 [2] - Miplyffa generated sales of $17.1 million in Q1 2025 [2] Earnings Expectations - Zevra Therapeutics is projected to report quarterly earnings of $0.65 per share, reflecting a year-over-year increase of 235.4% [3] - Expected revenues for the upcoming quarter are $71.83 million, representing a 1514.2% increase from the same quarter last year [3] - The consensus EPS estimate has remained unchanged over the last 30 days, indicating a potential lack of momentum in earnings estimate revisions [4] Industry Context - Zevra Therapeutics is categorized under the Zacks Medical - Drugs industry, which includes other companies like Xeris Biopharma (XERS) [5] - Xeris Biopharma's stock closed 1.5% higher at $4.73, with a slight return of 0.2% over the past month [5] - Xeris Biopharma's consensus EPS estimate has also remained unchanged, with a year-over-year change of 70% [6]

Company Overview - Zevra Therapeutics is focused on bringing life-changing therapeutics to the rare disease community[9] - The company has two commercial-stage rare disease products and an advanced clinical development pipeline[8,82] - Zevra has a strong financial position with $217 million in cash, cash equivalents, and investments as of April 1, 2025, including $687 million as of March 31, 2025, plus $1483 million net proceeds from a PRV sale[8,81] Product Portfolio - MIPLYFFA (arimoclomol) is FDA-approved for Niemann-Pick Disease Type C (NPC) and launched in November 2024[20] - Approximately 900 individuals in the U S live with NPC, with 300-350 diagnosed or treated[38] - OLPRUVA (sodium phenylbutyrate) for oral suspension is FDA-approved for Urea Cycle Disorders (UCD) in patients weighing 20 kg or greater and with a body surface area (BSA) of 12 m2 or greater[20,50] - Approximately 1,100 individuals are diagnosed with UCDs in the U S [59] Pipeline Development - The company is targeting an EMA MAA submission for arimoclomol in Europe in the second half of 2025[49] - Celiprolol is in Phase 3 development for Vascular Ehlers-Danlos Syndrome (VEDS), with IP through 2038[20] - Approximately 7,500 individuals in the U S are diagnosed with VEDS[74]

Core Points - Zevra Therapeutics, Inc. announced the re-election of Wendy L. Dixon, Ph.D. and Tamara A. Favorito to its Board of Directors, serving until the 2028 Annual Meeting [1][2] - The Board expressed gratitude for stockholder participation and support, viewing the election results as a validation of their strategic direction and progress over the past two years [2][4] - Daniel J. Mangless attempted to nominate alternative candidates but failed to meet SEC proxy rules and the Company's bylaws, resulting in his nominations being disregarded [2][3] Company Strategy and Performance - The Board and management reaffirmed their commitment to driving growth and executing their strategic plan, focusing on creating long-term value for stockholders [2][4] - The election results indicated strong stockholder support for the Board's industry expertise and commitment to delivering value [4] Financial Oversight - Stockholders approved the appointment of Ernst & Young LLP as the independent auditors for the fiscal year ending December 31, 2025 [5] Company Overview - Zevra Therapeutics is a commercial-stage company dedicated to developing therapies for rare diseases, utilizing data-driven strategies to overcome drug development challenges [6]

Core Viewpoint - All three leading proxy advisory firms, ISS, Glass Lewis, and Egan-Jones, have recommended that stockholders vote "FOR" Wendy L. Dixon, Ph.D. and Tamara A. Favorito, while advising to "WITHHOLD" votes for dissident nominees Travis Mickle and Arthur Regan, indicating a lack of compelling case for change from the dissident [1][2][4]. Proxy Advisory Recommendations - The Zevra Board of Directors expressed satisfaction with the unanimous endorsement from the proxy advisors, reinforcing their commitment to stockholder value creation under current leadership [2]. - Proxy advisors concluded that the dissident, Mangless, failed to provide sufficient arguments for electing his nominees, lacking a clear strategy to enhance stockholder value [2][5]. Shareholder Returns - Proxy advisors highlighted significant total shareholder returns under Zevra's current management, with ISS noting that the company has outperformed the broader biotech market during McFarlane's tenure as CEO [3][5]. - Egan-Jones emphasized that the past two years have yielded exceptional returns, surpassing both the total market and the S&P Biotech ETF [5]. Concerns About Dissident Nominees - ISS raised concerns about the potential disruption that electing the dissident nominees could cause, particularly highlighting the risks associated with having a former CEO on the board [5]. - Glass Lewis pointed out that Mangless did not present a meaningful alternative strategy for Zevra, lacking a clear plan to improve operational efficacy [5][13]. - Egan-Jones questioned the relevant expertise of Mangless' nominees, suggesting that their backgrounds do not offer meaningful value to Zevra's board [13].

Financial Data and Key Metrics Changes - The company reported a strong cash position with $217 million in the bank, allowing it to operate independently of capital markets [47] - The enrollment forms for MyPlifa have reached 122 since its launch, indicating a successful initial uptake [8][12] Business Line Data and Key Metrics Changes - MyPlifa, the recently launched product for Niemann Pick disease type C, is in its launch phase and has shown promising enrollment numbers in the U.S. market [5][8] - Olpruva, the other commercial product, is targeting specific urea cycle disorders and has seen five enrollments in line with its revised strategy [42] Market Data and Key Metrics Changes - In the U.S., there are approximately 900 patients diagnosed with Niemann Pick disease type C, with 300 to 350 diagnosed patients currently identified [7][8] - In Europe, the market is more mature with about 1,100 patients, but many are already on treatment, presenting a challenge for new product uptake [9] Company Strategy and Development Direction - The company is focused on executing the launch of MyPlifa and expanding its European filing for the product in the second half of the year [37][40] - There is an emphasis on increasing disease state awareness and product awareness to drive patient diagnosis and treatment [18][36] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the future, citing the successful launch of MyPlifa and the potential for increased patient identification through awareness efforts [18][36] - The company is committed to supporting patients through the reimbursement landscape and ensuring access to therapies [27][32] Other Important Information - The company is exploring various avenues for its go-to-market strategy in Europe, considering both direct and partnership approaches [40] - The enrollment in the saliprolol program for vascular Ehlers Danlos syndrome has reached 32 patients, with ongoing efforts to increase participation [44] Q&A Session Summary Question: Can you provide an overview of Zebra and its products? - The company is a commercial stage rare disease company with two products in the U.S. market and a pipeline in various stages of development [4] Question: How is the launch of MyPlifa progressing? - The launch is exceeding expectations, with significant patient enrollment already achieved [8][12] Question: What are the challenges in diagnosing Niemann Pick disease type C? - The heterogeneous nature of the disease complicates diagnosis, leading to many undiagnosed patients [14][15] Question: How does the company plan to penetrate the market further? - The company is leveraging successful strategies from Europe to increase awareness and diagnosis in the U.S. [18][33] Question: What is the current status of reimbursement levels? - The company is pleased with the current reimbursement efforts and the willingness of physicians and patients to navigate the process [26][28] Question: How does the company support patients transitioning from EAP to commercial therapy? - The company has a robust support system in place to assist patients through the transition [29][32] Question: What is the strategy for the European market? - The company is focused on the MAA filing and is exploring various market entry strategies [37][40]

Financial Performance - Zevra Therapeutics reported a quarterly loss of $0.06 per share, significantly better than the Zacks Consensus Estimate of a loss of $0.21, and an improvement from a loss of $0.40 per share a year ago, representing an earnings surprise of 71.43% [1] - The company posted revenues of $20.4 million for the quarter ended March 2025, exceeding the Zacks Consensus Estimate by 29.12%, compared to revenues of $3.43 million in the same quarter last year [2] Stock Performance - Zevra Therapeutics shares have declined approximately 7.1% since the beginning of the year, while the S&P 500 has seen a smaller decline of 0.6% [3] - The current Zacks Rank for Zevra Therapeutics is 4 (Sell), indicating expectations of underperformance in the near future [6] Earnings Outlook - The consensus EPS estimate for the upcoming quarter is -$0.11 on revenues of $20.67 million, and for the current fiscal year, it is -$0.34 on revenues of $88.1 million [7] - The trend for estimate revisions ahead of the earnings release has been unfavorable, which may impact future stock movements [6] Industry Context - The Medical - Drugs industry, to which Zevra Therapeutics belongs, is currently ranked in the top 29% of over 250 Zacks industries, suggesting a favorable environment for stocks in this sector [8]