Core Viewpoint - Zevra Therapeutics (ZVRA) has seen a 16.8% increase in share price over the past four weeks, closing at $10.99, with analysts suggesting a potential upside of 117.1% based on a mean price target of $23.86 [1][4]. Price Targets and Analyst Estimates - The mean price target is derived from seven short-term estimates with a standard deviation of $3.39, indicating a range of potential outcomes; the lowest estimate is $19.00 (72.9% increase), while the highest is $29.00 (163.9% increase) [2][9]. - A low standard deviation among price targets suggests a strong consensus among analysts regarding the stock's price direction, although it does not guarantee that the stock will reach the average target [9][10]. Earnings Estimates and Market Sentiment - Analysts are optimistic about ZVRA's earnings prospects, as indicated by a significant increase in earnings estimate revisions, with a 337.1% rise in the Zacks Consensus Estimate for the current year [11][12]. - ZVRA holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors, which supports the stock's potential for near-term upside [13]. Conclusion on Price Movement - While consensus price targets may not be entirely reliable, the positive direction indicated by earnings estimate revisions appears to be a valid guide for potential stock price movements [14].

CELEBRATION, Fla., July 28, 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 diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare, neurological disease caused by genetic mutations that result in lipid ...

Core Insights - Zevra Therapeutics is actively participating in the 42nd Annual Meeting of the Southeastern Regional Genetics Group (SERGG), showcasing its therapies MIPLYFFA and OLPRUVA, which are approved for treating rare diseases [1][2] Group 1: MIPLYFFA (arimoclomol) - MIPLYFFA is approved in the U.S. for treating Niemann-Pick disease type C (NPC) and has shown disease-modifying activity leading to sustained disease stabilization in patients over multiple years [2][7] - Data from an Expanded Access Program (EAP) indicates that patients treated with MIPLYFFA experienced stable disease over up to 3 years, contrasting with the average progression of 1.0–2.0 points per year on the Niemann-Pick type C Clinical Severity Scale [3] - A 48-month open-label trial demonstrated that patients treated with MIPLYFFA experienced sustained reduction in disease progression for at least 5 years [3] - MIPLYFFA targets the pathophysiology of NPC through two pathways, enhancing the expression of genes in the Coordinated Lysosomal Expression and Regulation (CLEAR) network [3] Group 2: OLPRUVA (sodium phenylbutyrate) - OLPRUVA is approved for treating certain patients with urea cycle disorders (UCDs) and can be administered via a gastrostomy tube, which is significant for managing UCD in some patients [2][4][18] - Studies confirm that OLPRUVA meets administration criteria through gastrostomy tubes at evaluated doses and volumes [4] - OLPRUVA is indicated for long-term management of UCDs in adults and children weighing 44 pounds (20 kg) or greater, but is not used for acute hyperammonemia [18] Group 3: Company Overview - Zevra Therapeutics focuses on developing therapies for rare diseases, aiming to provide life-changing treatments for patients with limited or no options [23] - The company employs unique, data-driven strategies to overcome challenges in drug development and commercialization [23]

Company Overview - Zevra Therapeutics (ZVRA) currently holds a Momentum Style Score of A, indicating strong momentum characteristics [2] - The company has a Zacks Rank of 2 (Buy), suggesting a favorable outlook for investors [3] Price Performance - ZVRA shares have increased by 28.64% over the past week, significantly outperforming the Zacks Medical - Drugs industry, which rose by 1.75% during the same period [5] - Over the past month, ZVRA's price change is 45.18%, compared to the industry's 1.25% [5] - In the last quarter, ZVRA shares have risen by 74.97%, and over the past year, they have gained 95.57%, while the S&P 500 has only moved 16.04% and 12.11%, respectively [6] Trading Volume - ZVRA's average 20-day trading volume is 1,122,698 shares, which serves as a bullish indicator when combined with rising stock prices [7] Earnings Estimates - In the past two months, three earnings estimates for ZVRA have been revised upwards, with no downward revisions, leading to an increase in the consensus estimate from $0.31 to $1.35 [9] - For the next fiscal year, two estimates have also moved upwards, with no downward revisions during the same period [9] Conclusion - Given the strong momentum indicators and positive earnings outlook, ZVRA is positioned as a solid momentum pick for investors [11]

Industry Overview - The uncertainty surrounding tariffs and trade measures has impacted economic growth, with President Trump threatening to impose tariffs as high as 200% on pharmaceutical imports to encourage domestic production [1] - Despite these threats, the biotech sector has remained stable, with many global pharmaceutical companies already investing in domestic manufacturing [1] Current Trends - The Zacks Medical-Drugs industry is showing promising trends due to positive pipeline and regulatory developments [2] - Key areas of innovation include rare diseases, next-generation oncology treatments, obesity, immunology, and neuroscience, attracting significant investor interest [3] M&A Activity - M&A activity in the sector remains healthy, indicating growth potential for companies like Catalyst Pharmaceuticals, Zevra Therapeutics, Theravance Biopharma, Aldeyra Therapeutics, and Larimar Therapeutics [3] Industry Characteristics - The Zacks Medical-Drugs industry consists of small to medium-sized drug companies that produce medicines for human and veterinary use, often relying on collaboration payments for revenue [4] Factors Influencing Future Growth - The success of key pipeline candidates in clinical studies can significantly impact stock prices, with successful innovations acting as catalysts [5] - Strong collaboration with larger drugmakers is a positive indicator for small pharma companies, especially when equity investments are involved [6] - Investment in technology and personalized medicine is crucial for smaller companies to thrive in a changing healthcare landscape [7] Performance Metrics - The Zacks Medical-Drugs industry currently holds a Zacks Industry Rank of 96, placing it in the top 39% of 245 Zacks industries, indicating strong prospects [9] - Year-to-date, the industry has risen 5.6%, outperforming the Zacks Medical sector, which has decreased by 2.9%, while slightly underperforming the S&P 500, which has risen by 6.9% [11] Valuation - The industry is currently trading at a trailing 12-month price-to-sales ratio of 2.32, compared to the S&P 500's 5.66 and the Zacks Medical sector's 2.37 [12] Company Highlights - **Catalyst Pharmaceuticals**: Lead drug Firdapse shows strong demand; stock has risen 0.4% this year; earnings estimate for 2025 increased from $2.23 to $2.25 [16][17] - **Theravance Biopharma**: Strong sales from Yupelri; stock has risen 22.6% this year; earnings estimate for 2025 declined from 3 cents to 1 cent [20][21] - **Larimar Therapeutics**: Developing nomlabofusp for Friedreich's ataxia; stock has declined 18.3% this year; loss estimate for 2025 narrowed from $1.90 to $1.89 [24][25] - **Zevra Therapeutics**: Early adoption of Miplyffa exceeds expectations; stock has risen 53.4% this year; earnings estimate for 2025 increased from 31 cents to 76 cents [28][29] - **Aldeyra Therapeutics**: Focused on RASP modulators; stock has declined 0.4% this year; loss estimate for 2025 narrowed from $1.03 to 90 cents [31][32]

Core Insights - Zevra Therapeutics announced the publication of long-term efficacy and safety results for MIPLYFFA (arimoclomol) in treating Niemann-Pick Disease Type C (NPC), demonstrating sustained disease progression halting over multiple years [1][2] Company Overview - Zevra Therapeutics is a commercial-stage company focused on therapies for rare diseases, aiming to provide transformational treatments for conditions with limited options [14] Product Information - MIPLYFFA is an approved therapy for NPC, indicated for use in combination with miglustat for patients aged 2 years and older [4] - The drug received FDA approval on September 20, 2024, and has shown the ability to activate transcription factors that regulate lysosomal function [3] Clinical Trial Results - The Phase 2/3 trial demonstrated that MIPLYFFA halted disease progression compared to placebo over a one-year duration, with long-term data showing sustained efficacy for at least five years in over 270 patients [2][3] - The open-label extension study confirmed no new safety concerns, aligning with previous trial results [2] Safety Information - Common adverse reactions reported include upper respiratory tract infection, diarrhea, and decreased weight, with hypersensitivity reactions noted in some patients [5][9][10] - MIPLYFFA may cause increases in serum creatinine without affecting glomerular function, primarily occurring in the first month of treatment [8]

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]