ZVRA

Interested in Zevra Therapeutics, Inc.? Here are five stocks we like better. Revenue grew strongly: Zevra’s Q2 2026 net revenue rose 53% year over year to $39.7 million, led by MIPLYFFA U.S. revenue of $30.2 million. Adjusted net income was $15.2 million, excluding a $6.4 million non-cash warrant-related charge. MIPLYFFA adoption continued to expand: The company had received 184 prescription enrollment forms since launch, while payer coverage held at 69% of covered lives and management reported high refill and persistence rates. Pipeline and financial flexibility remain priorities: Zevra expects a fourth-quarter decision on the European re-examination of arimoclomol, is advancing celiprolol’s Phase III trial, and ended the quarter with $260.2 million in cash and investments. MIPLYFFA also received U.S. patent protection through 2041. Zevra Therapeutics (NASDAQ:ZVRA) reported second-quarter 2026 net revenue of $39.7 million, up 53% from $25.9 million a year earlier, driven primarily by U.S. sales of its Niemann-Pick disease type C treatment MIPLYFFA. Net U.S. revenue for MIPLYFFA totaled $30.2 million during the quarter, increasing 23% from the first quarter and 40% from the prior-year period. Chief Executive Officer Neil McFarlane said the company’s commercial momentum reflects continued adoption of MIPLYFFA in combination with miglustat as a foundational treatment for NPC, a rare genetic disease. → 3 Drone Stocks That Should Soar After the Summer Slump The company reported net income of $8.8 million, or $0.14 per basic and diluted share, compared with net income of $74.7 million, or $1.24 per basic share and $1.21 per diluted share, in the second quarter of 2025. The latest quarter included a $6.4 million non-cash mark-to-market charge related to outstanding common-stock warrants. Excluding that item, Zevra said adjusted net income would have been $15.2 million, or $0.25 per share. As of June 30, Zevra had received 184 prescription enrollment forms for MIPLYFFA since launch, including 14 during the second quarter and 23 during the first half of 2026. The second-quarter enrollments included patients in Puerto Rico, according to the company. → Meta’s Earnings Drop Shows Wall Street Wants More Than Ad Growth Josh Schafer, Zevra’s chief commercial officer, said enrollment forms represent prescriptions submitted to the company’s specialty pharmacy, which then begins the benefits investigation process. He said enrollments have come from both centers of excellence and community practices, and include newly diagnosed and previously diagnosed patients as well as pediatric and adult patients. McFarlane said Zevra is seeing an approximately equal mix of adult and pediatric patients among the 184 enrollment forms. The company estimates that 900 people in the U.S. may be living with NPC, of whom 300 to 350 are diagnosed. → Jersey Mike's Serves Fresh Gains After IPO Stumble Schafer said Zevra’s disease-awareness efforts, genetic-testing collaborations, AI-driven physician targeting and Expert Connect program are intended to help identify patients and support diagnosis. The company’s payer coverage remained at 69% of covered lives during the quarter. Management said refill, compliance and persistence rates have been high among patients receiving MIPLYFFA. Schafer said that, among patients who have stopped treatment, the vast majority had died as a result of the severity of the disease. Zevra is pursuing a re-examination of its Marketing Authorization Application for arimoclomol in the European Union after the Committee for Medicinal Products for Human Use issued a negative opinion following the company’s oral explanation. McFarlane said the re-examination will enable Zevra to address the grounds cited in the CHMP’s refusal and have its evidence package reviewed by a new rapporteur and co-rapporteur. The process typically takes 120 days from submission to a CHMP opinion, with an outcome expected in the fourth quarter. The company has also requested a Scientific Advisory Group meeting as part of the process. McFarlane said Zevra’s confidence is based on the totality of its evidence, including its pivotal study, a four-year open-label extension study, early-access program data, a pediatric substudy and a broader safety database. Meanwhile, Zevra will continue providing arimoclomol access for eligible patients through its global expanded-access program, which includes compassionate-use and reimbursed named-patient programs. The program had enrolled 132 patients as of June 30, including 10 added in the second quarter. McFarlane said most of those patients are in Europe. Net reimbursements totaled $9 million in the quarter. McFarlane said the French expanded-access program has been consistent at approximately $2.5 million in net revenue per quarter, or about $10 million annually, while reimbursement and ordering patterns in other territories remain unpredictable. Zevra said it secured an Orange Book listing for MIPLYFFA providing U.S. patent protection through 2041. The company is still awaiting a decision from the U.S. Patent Office on a separately requested patent-term extension. McFarlane said the longer intellectual-property protection gives Zevra a basis to consider further investments in the MIPLYFFA franchise, including potential global expansion. The company is also advancing celiprolol in its Phase III DISCOVER trial for vascular Ehlers-Danlos syndrome. The event-driven study has enrolled 66 patients, including four during the second quarter, out of a planned 150 patients. It requires 28 events to trigger an interim analysis, and Zevra has recorded three confirmed events to date. McFarlane said the company is working to accelerate enrollment by expanding its network of genetic-testing centers and is exploring regulatory pathways with the Food and Drug Administration. Following a Type C meeting in the first quarter, Zevra expects to reengage with the FDA during the second half of 2026. Operating expenses were $21 million in the second quarter, down $3.2 million from a year earlier. Research and development expense increased $1.1 million to $4.5 million, primarily due to ongoing arimoclomol efforts, while selling, general and administrative expense declined $4.2 million to $16.6 million, primarily because of lower professional fees and third-party spending. Zevra ended the quarter with $260.2 million in cash equivalents and investments, up approximately $23.4 million from March 31. Chief Financial Officer Justin Renz said the company has the financial capacity to pursue MIPLYFFA growth, global opportunities, celiprolol development and selected external opportunities while maintaining disciplined capital allocation. Renz said share repurchases are not part of Zevra’s current capital-allocation strategy, as management remains focused on growth investments and evaluating potential value-creating opportunities. Zevra Therapeutics, Inc discovers and develops various proprietary prodrugs to treat serious medical conditions in the United States. The company develops its products through Ligand Activated Therapy platform. Its lead product candidate is KP1077, consisting of KP1077IH, which is under Phase 2 clinical trial for the treatment of idiopathic hypersomnia, and KP1077N, which is under Phase ½ clinical trial to treat narcolepsy. The company is also developing Celiprolol, a prodrug product candidate that is under Phase 1/2 clinical trial for the treatment of vascular Ehlers Danlos syndrome. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. The article "Zevra Therapeutics Q2 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for August 2026.

Zevra Therapeutics (ZVRA) came out with quarterly earnings of $0.25 per share, beating the Zacks Consensus Estimate of $0.08 per share. This compares to earnings of $1.21 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of +212.50%. A quarter ago, it was expected that this specialty pharmaceutical company would post earnings of $0.06 per share when it actually produced earnings of $0.18, delivering a surprise of +200%. Over the last four quarters, the company has surpassed consensus EPS estimates four times. Zevra Therapeutics, which belongs to the Zacks Medical - Drugs industry, posted revenues of $39.66 million for the quarter ended June 2026, surpassing the Zacks Consensus Estimate by 22.76%. This compares to year-ago revenues of $25.88 million. The company has topped consensus revenue estimates three times over the last four quarters. The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call. Zevra Therapeutics shares have added about 17.4% since the beginning of the year versus the S&P 500's gain of 13%. While Zevra Therapeutics has outperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock? There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately. Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions. Ahead of this earnings release, the estimate revisions trend for Zevra Therapeutics was unfavorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #4 (Sell) for the stock. So, the shares are expected to underperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is $0.12 on $34.89 million in revenues for the coming quarter and $0.95 on $142.15 million in revenues for the current fiscal year. Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Drugs is currently in the bottom 39% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1. One other stock from the same industry, Xeris Biopharma (XERS), is yet to report results for the quarter ended June 2026. The results are expected to be released on August 6. This company is expected to post quarterly earnings of $0.02 per share in its upcoming report, which represents a year-over-year change of +300%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days. Xeris Biopharma's revenues are expected to be $90.73 million, up 26.8% from the year-ago quarter. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Zevra Therapeutics, Inc. (ZVRA) : Free Stock Analysis Report Xeris Biopharma Holdings, Inc. (XERS) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

This article first appeared on GuruFocus. Total Net Revenue: $39.7 million in Q2 2026, a 53% increase year-over-year. MIPLYFFA Net Sales (US): $30.2 million in Q2 2026, up 23% sequentially and 40% year-over-year. Arimoclomol Global EAP Net Reimbursements: $9.0 million in Q2 2026. OLPRUVA Revenue: $0.2 million in Q2 2026. Pass-Through Royalty Revenue (Commave): $0.3 million in Q2 2026. Operating Expenses: $21.0 million in Q2 2026, a decrease of $3.2 million year-over-year. R&D Expense: $4.5 million in Q2 2026, an increase of $1.1 million year-over-year. SG&A Expense: $16.6 million in Q2 2026, a decrease of $4.2 million year-over-year. Net Income: $8.8 million, or $0.14 per basic and diluted share, in Q2 2026. Adjusted Net Income (excl. warrant mark-to-market charge): $15.2 million, or $0.25 per share, in Q2 2026. Cash, Cash Equivalents, and Investments: $260.2 million as of June 30, 2026, an increase of $23.4 million from March 31, 2026. Prescription Enrollment Forms (MIPLYFFA): 184 total received since launch through June 30, 2026, with 14 received in Q2 2026. Global EAP Enrollment (Arimoclomol): 132 patients enrolled, with 10 added in Q2 2026. Warning! GuruFocus has detected 7 Warning Signs with ZVRA. Is ZVRA fairly valued? Test your thesis with our free DCF calculator. Release Date: August 05, 2026 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Zevra Therapeutics Inc (NASDAQ:ZVRA) reported a 53% year-over-year increase in total net revenue for Q2 2026, reaching $39.7 million, driven by strong US sales of MIPLYFFA. The company strengthened its intellectual property position for MIPLYFFA with an Orange Book listing providing protection through 2041, supporting long-term commercial opportunity. Zevra Therapeutics Inc (NASDAQ:ZVRA) maintains a robust financial foundation with over $260 million in cash, cash equivalents, and investments, providing flexibility for strategic investments. The company's commercial strategy is showing progress, with 184 prescription enrollment forms for MIPLYFFA received since launch, including a diverse mix of newly diagnosed, previously diagnosed, adult, and pediatric patients. Zevra Therapeutics Inc (NASDAQ:ZVRA) continues to advance its pipeline, including the Phase III DISCOVER trial for celiprolol, and is exploring regulatory pathways to accelerate development. The company achieved net income of $8.8 million in Q2 2026, demonstrating improved profitability compared to the prior year period. Zevra Therapeutics Inc (NASDAQ:ZVRA) is expanding access to MIPLYFFA through global expanded access programs in response to strong international interest. The CHMP issued a negative opinion regarding the arimoclomol Marketing Authorization Application in the EU, creating regulatory uncertainty for the product's approval in Europe. Reimbursements beyond the French EAP for arimoclomol are expected to be highly variable and unpredictable in the near term, impacting revenue stability. The company incurred a $6.4 million non-cash mark-to-market charge related to outstanding common stock warrants, which negatively impacted reported net income. The celiprolol Phase III DISCOVER trial is progressing slowly, with only 3 confirmed events out of the 28 required to trigger the interim analysis, indicating a lengthy timeline. Zevra Therapeutics Inc (NASDAQ:ZVRA) faces ongoing challenges in patient identification and diagnosis for NPC, with an estimated 300-350 diagnosed patients out of a potential 900, limiting near-term market growth. The company's reliance on a single commercial product, MIPLYFFA, for US revenue growth exposes it to concentration risk. The re-examination process for arimoclomol with the CHMP is expected to take approximately 120 days, with the outcome uncertain and potentially unfavorable. Q: What gives you confidence that you can be successful in the re-examination of the arimoclomol MAA with the CHMP, especially with a new rapporteur and co-rapporteur reviewing the evidence? A: Neil McFarlane (President and CEO): Our confidence is grounded in the totality of the evidence and the clear unmet patient need. The re-examination process allows us to focus specifically on the grounds cited for refusal and bring new expert voices into the discussion. We have a positive, clinically meaningful, and statistically significant pivotal study as the foundation, bolstered by long-term clinical experience from our global EAP (132 patients, with the majority in the EU), multi-year safety data with no new concerns, and published pediatric sub-study data. We believe the benefit-risk profile is strongly in favor of arimoclomol for EU patients. Q: Can you provide more color on the commercial momentum for MIPLYFFA, specifically regarding the mix of newly diagnosed vs. previously diagnosed patients and the age distribution? A: Joshua Schafer (Chief Commercial Officer) and Neil McFarlane (CEO): We are seeing a diverse mix of enrollments, including both newly diagnosed and previously diagnosed patients, from centers of excellence and community practices. The age distribution is roughly 50/50 between adults and children. For example, we recently enrolled a newly diagnosed 2-year-old and a patient in his mid-20s who was misdiagnosed with epilepsy for years. This diversity supports our confidence in the total addressable market of 350 to 900 diagnosed patients in the US. Q: How should we model the ex-US EAP revenue, given the variability in ordering patterns outside of France? A: Neil McFarlane (CEO): The French EAP program remains very consistent at approximately $2.5 million net per quarter, or $10 million net per year, with about 30 stable patients. However, ordering patterns from other reimbursed named patient programs are unpredictable, as a single patient may order a full year's supply or order on a monthly or quarterly basis. It is driven by timing, order volume, program type, and geography, making it difficult to provide specific guidance for future quarters. Q: With a strong balance sheet of $260.2 million in cash, how are you thinking about capital allocation and potential share buybacks? A: Justin Renz (CFO): We are in growth mode, and our priorities remain unchanged: driving MIPLYFFA growth, pursuing global opportunities, advancing celiprolol, and evaluating aligned external opportunities. We maintain a disciplined capital allocation strategy and are open-minded to value-creating opportunities. While the board discusses these topics, share buybacks are not currently part of our capital allocation strategy. Q: What is your latest competitive intelligence on the US market, particularly regarding patients on other approved products like IntraBio's arimoclomol, and how does this affect MIPLYFFA's positioning? A: Neil McFarlane (CEO) and Joshua Schafer (CCO): We believe the treatments are complementary, and the updated clinical practice guidelines reinforce that combination therapy should be considered for all patients with confirmed NPC. MIPLYFFA is the only FDA-approved treatment indicated for use in combination with miglustat and is recognized as a disease-modifying therapy. We do not see this as an either/or market; rather, we see it as an opportunity to grow our share of diagnosed patients and, more importantly, reach the undiagnosed population. Q: Can you provide details on patient attrition and persistence rates for MIPLYFFA, and how did channel inventory compare at the end of Q2 versus Q1? A: Joshua Schafer (CCO) and Justin Renz (CFO): Refill and persistence rates are quite high. The vast majority of patients who discontinue do so due to the severity of the disease, often passing away. Channel inventory, which fell below the target range at the end of Q1 due to shipping timing, returned to normal target levels at the end of Q2. Q: What is the status of the celiprolol Phase III DISCOVER trial, and what did you learn from the Type C meeting with the FDA? A: Neil McFarlane (CEO): The Type C meeting was informative, and we are working on parallel tracks to accelerate the program: exploring regulatory pathways to speed development and driving enrollment to reach the required events for the interim analysis. We have 66 patients enrolled (out of 150) and 3 confirmed events (out of 28 required). We plan to reengage with the FDA in the second half of this year to discuss acceleration strategies. Q: How are you leveraging your strong cash position to invest in the MIPLYFFA franchise, especially given the strengthened IP position with the Orange Book listing through 2041? A: Neil McFarlane (CEO): The longer duration of IP protection gives us a lens to further invest capital into the US business, as well as European and global expansion and the celiprolol program. We are looking to maximize the long-term value of MIPLYFFA, though we don't have a specific action to announce regarding the current extension. Q: What role will a Scientific Advisory Group (SAG) meeting and KOL testimony play in the re-examination process? A: Neil McFarlane (CEO): We have requested a SAG meeting as part of the re-examination process. This is a key part of bringing new expert voices into the procedural discussions and will allow us to further educate the CHMP on the comprehensive evidence package. Q: Can you elaborate on the drivers of the 14 new prescription enrollment forms in Q2 and the overall progress toward the 184 total forms? A: Joshua Schafer (CCO) and Neil McFarlane (CEO): The enrollments are driven by an integrated strategy, not a single tactic. This includes our disease awareness campaign (Learn NPC), genetic testing collaborations, AI-driven targeting models, and the Expert Connect program. We are seeing a mix of newly diagnosed and previously diagnosed patients, which gives us confidence in the addressable market. The 23 enrollments in the first half of 2026 are tracking well against the 52 total enrollments in 2025. For the complete transcript of the earnings call, please refer to the full earnings call transcript.

$39.7 million net revenue for Q2 2026, driven by $30.2 million in revenue from MIPLYFFA 53% increase in total net revenue over Q2 2025 Operational execution fueled strong cash position of $260.2 million Company to host conference call and webcast TODAY, August 5, 2026, at 4:30 p.m. ET BOSTON, Aug. 05, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, today reported its financial results for the second quarter ended June 30, 2026. “Zevra delivered strong MIPLYFFA performance and further established the therapy as an important treatment option for people living with Niemann-Pick disease type C,” said Neil F. McFarlane, Zevra's President and Chief Executive Officer. “These results reflect the strength of our U.S. commercial execution. Additionally, we are pursuing access to arimoclomol for people living with NPC in Europe, and we have submitted a request for the re-examination of our Marketing Authorisation Application. Our global Expanded Access Program remains active for eligible patients, reflecting our steadfast commitment to patients and families.” MIPLYFFA® (arimoclomol) Highlights U.S.: Received 14 MIPLYFFA prescription enrollment forms for Niemann-Pick disease type C (NPC) during Q2 2026, bringing the total to 184 since product launch. Market access remains stable at 69% of covered lives. EU: The Company has requested a re-examination of its Marketing Authorisation Application seeking approval of arimoclomol for the treatment of NPC. Global Expanded Access Program (EAP): As of June 30, 2026, 132 patients were enrolled in the global EAP. Strengthened our intellectual property position for MIPLYFFA with an Orange Book listing through 2041. New data published in Molecular Genetics and Metabolism Reports show MIPLYFFA was well tolerated in pediatric patients with no new safety signals observed. Published the long-term real-world safety and effectiveness data of MIPLYFFA from the U.S. Early Access Program in Molecular Genetics and Metabolism. Pipeline and Innovation Highlights Enrolled four patients in the event-driven Phase 3 DiSCOVER trial of celiprolol for the treatment of Vascular Ehlers-Danlos Syndrome during Q2 2026, bringing the total number of enrolled patients to 66, with a total of three confirmed events. The Company expects to continue engagement with the U.S. Food and Drug Administration (FDA) in the second half of this year to explore pathways to accelerate clinical development. Q2 2026 Financial Highlights Revenue, Net: $39.7 million for Q2 2026, which includes $30.2 million of MIPLYFFA net revenue, $0.2 million of OLPRUVA net revenue, $9.0 million in net reimbursements from our global EAP, and $0.3 million in past due royalties and other reimbursements under the AZSTARYS® license agreement. This was an increase in total net revenue of $13.8 million compared to $25.9 million in Q2 2025. Cost of Product Revenue: $1.5 million for Q2 2026, excluding non-cash intangible asset amortization. Cost of product revenue for Q2 2025 was $12.4 million, excluding non-cash intangible asset amortization. Operating Expenses: $21.0 million for Q2 2026, which includes non-cash stock compensation expense of $3.0 million. Total operating expenses for Q2 2025 were $24.2 million. Net income: Net income of $8.8 million, or $0.14 per basic and diluted share for Q2 2026, compared to a net income of $74.7 million, or $1.24 per basic and $1.21 diluted share for Q2 2025. Cash Position: Cash, cash equivalents and investments were $260.2 million as of June 30, 2026. Based on its current operating forecast, the Company believes available financial resources are sufficient to execute on its strategic priorities independent from the capital markets. Common and Fully Diluted Shares O/S: As of June 30, 2026, total shares of common stock outstanding were 59,341,906, and fully diluted common shares were 69,298,105, which included 7,426,820 shares issuable from outstanding awards under equity incentive plans, and 2,529,379 shares issuable upon exercise of warrants.1 Adjusted net income and adjusted net income per share are non-GAAP financial measures. Management believes that adjusted net income and adjusted net income per share provide useful information for investors, and management uses these supplemental measures to assess the Company’s operating performance. Adjusted net income and adjusted net income per share have limitations as analytical tools because they do not reflect all of the amounts associated with our results of operations as determined in accordance with U.S. GAAP. Additionally, they may not be comparable to similarly titled measures of other companies, including in our industry, limiting the usefulness of those measures for comparative purposes. Because of these limitations, these non-GAAP financial measures should be considered along with other operating and financial performance measures presented in accordance with U.S. GAAP. The presentation of these non-GAAP financial measures are not intended to be considered in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with U.S. GAAP. Conference Call Information Zevra will host a conference call and audio webcast TODAY at 4:30 p.m. ET to discuss its corporate update and financial results for the second quarter 2026. A link to the audio webcast is accessible on the “Events & Presentations” page in the Investor Relations section of the Zevra's website at investors.zevra.com. A replay of the webcast will be available for 90 days beginning at approximately 5:30 p.m. ET on August 5, 2026. Additionally, interested participants and investors may access the conference call by dialing either: (800) 274-8461 (United States) +1 (203) -518-9814 (International) Conference ID: ZVRAQ226 About MIPLYFFA® (arimoclomol) MIPLYFFA (arimoclomol) is Zevra’s approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. The extensive data generated for MIPLYFFA has shown long-term, meaningful clinical outcomes with more than 5 years of patient experience across more than 270 NPC patients worldwide through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. Zevra has submitted a Marketing Authorization Application to the European Medicines Agency for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C. INDICATIONS AND USAGE MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions: Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve. Embryofetal Toxicity: MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential. Increased Creatinine without Affecting Glomerular Function: Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function. During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation. The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight. Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema. To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. toll-free at 1-844-600-2237 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Drug Interaction(s): Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate. Use in Females and Males of Reproductive Potential: Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights. Renal Impairment: The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function. MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg. For more information, please see the full Prescribing Information, including Instructions for Use. About OLPRUVA® OLPRUVA (sodium phenylbutyrate) is Zevra’s approved treatment for the treatment of certain UCDs. OLPRUVA (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapies, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). OLPRUVA is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. For more information, please visit www.OLPRUVA.com. Important Safety Information Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with OLPRUVA. Tell your doctor about all the medicines you or your child take, especially if you or your child take corticosteroids, valproic acid, haloperidol, and/or probenecid. OLPRUVA can cause serious side effects, including: 1) nervous system problems (neurotoxicity). Symptoms include sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, confusion, 2) low potassium levels in your blood (hypokalemia) and 3) conditions related to swelling (edema). OLPRUVA contains salt (sodium), which can cause swelling from salt and water retention. Tell your doctor right away if you or your child get any of these symptoms. Your doctor may do certain blood tests to check for side effects during treatment with OLPRUVA. If you have certain medical conditions such as heart, liver or kidney problems, are pregnant/planning to get pregnant or breast-feeding, your doctor will decide if OLPRUVA is right for you. The most common side effects of OLPRUVA include absent or irregular menstrual periods, decreased appetite, body odor, bad taste or avoiding foods you ate prior to getting sick (taste aversion). These are not all of the possible side effects of OLPRUVA. Call your doctor for medical advice about side effects. You may report side effects to U.S. FDA at 1-800-FDA-1088. About Celiprolol Celiprolol is Zevra’s investigational clinical candidate for the treatment of Vascular Ehlers-Danlos Syndrome (VEDS). Celiprolol has been granted Orphan Drug and Breakthrough Therapy designations by the U.S. FDA. Zevra recently restarted enrollment in the DiSCOVER trial, a Phase 3 trial being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. FDA. Celiprolol’s mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. The Company is focused on broadening access through geographic expansion opportunities, progressing its pipeline toward key milestones, and delivering meaningful therapeutics. The commercialization of its lead product, marketed in the U.S. for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disease, provides a strong corporate foundation and validates its ability to advance therapies from development to market. Zevra's vision is realized through disciplined execution of its strategic plan and core values — patient centricity, integrity, accountability, innovation, and courage — which guide its efforts to deliver long-term value. For more information, please visit www.zevra.com or follow us on X and LinkedIn. Cautionary Note Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding our request for a re-examination of the MAA for arimoclomol; the continued availability of our global EAP; the potential to accelerate development of the Company's treatment for Vascular Ehlers-Danlos Syndrome and continued engagement with FDA; and the sufficiency of the Company’s available financial resources to execute on its strategic priorities. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2025, filed on March 9, 2026, and Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Investor Contact Nichol Ochsner+1 (732) [email protected] Media Contact Julie Downs+1 (508) [email protected]

BOSTON, Mass., July 22, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, today announced it will report corporate and financial results for the second quarter on Wednesday, August 5, 2026, via a news release after the market close, and will host a conference call/audio webcast at 4:30 p.m. ET that day. A link to the audio webcast will be accessible on the “Events & Presentations” page in the Investor Relations section of Zevra’s website at investors.zevra.com. To join via telephone, please use the following dial-in information: (800) 274-8461 (United States) +1 (203) 518-9814 (International) Conference ID: ZVRAQ226 A replay of the webcast will be available for 90 days beginning at approximately 5:30 p.m. ET. The replay will be accessible on the “Events & Presentations” page in the Investor Relations section of Zevra’s website at investors.zevra.com. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. The Company is focused on broadening access through geographic expansion opportunities, progressing its pipeline toward key milestones, and delivering meaningful therapeutics. The commercialization of its lead product, marketed in the U.S. for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disease, provides a strong corporate foundation and validates its ability to advance therapies from development to market. Zevra's vision is realized through disciplined execution of its strategic plan and core values — patient centricity, integrity, accountability, innovation, and courage — which guide its efforts to deliver long-term value. For more information, please visit www.zevra.com or follow us on X and LinkedIn. Caution Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the Securities and Exchange Commission on March 9, 2026, and Zevra's other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Investor Contact Nichol Ochsner +1 (732) 754-2545 [email protected] Media Contact Julie Downs+1 (508) 246-3230 [email protected]

Even though Zevra Therapeutics, Inc. (NASDAQ:ZVRA) posted strong earnings recently, the stock hasn't reacted in a large way. We looked deeper into the numbers and found that shareholders might be concerned with some underlying weaknesses. This technology could replace computers: discover the 20 stocks are working to make quantum computing a reality. Many investors haven't heard of the accrual ratio from cashflow, but it is actually a useful measure of how well a company's profit is backed up by free cash flow (FCF) during a given period. In plain english, this ratio subtracts FCF from net profit, and divides that number by the company's average operating assets over that period. This ratio tells us how much of a company's profit is not backed by free cashflow. That means a negative accrual ratio is a good thing, because it shows that the company is bringing in more free cash flow than its profit would suggest. While it's not a problem to have a positive accrual ratio, indicating a certain level of non-cash profits, a high accrual ratio is arguably a bad thing, because it indicates paper profits are not matched by cash flow. That's because some academic studies have suggested that high accruals ratios tend to lead to lower profit or less profit growth. Over the twelve months to March 2026, Zevra Therapeutics recorded an accrual ratio of 4.81. Ergo, its free cash flow is significantly weaker than its profit. Statistically speaking, that's a real negative for future earnings. In fact, it had free cash flow of US$12m in the last year, which was a lot less than its statutory profit of US$117.0m. Given that Zevra Therapeutics had negative free cash flow in the prior corresponding period, the trailing twelve month resul of US$12m would seem to be a step in the right direction. Having said that, there is more to consider. We must also consider the impact of unusual items on statutory profit (and thus the accrual ratio), as well as note the ramifications of the company issuing new shares. The good news for shareholders is that Zevra Therapeutics' accrual ratio was much better last year, so this year's poor reading might simply be a case of a short term mismatch between profit and FCF. As a result, some shareholders may be looking for stronger cash conversion in the current year. View our latest analysis for Zevra Therapeutics That might leave you wondering what analysts are forecasting in terms of future profitability. Luckily, you can click here to see an interactive graph depicting future profitability, based on their estimates. To understand the value of a company's earnings growth, it is imperative to consider any dilution of shareholders' interests. Zevra Therapeutics expanded the number of shares on issue by 8.1% over the last year. As a result, its net income is now split between a greater number of shares. To talk about net income, without noticing earnings per share, is to be distracted by the big numbers while ignoring the smaller numbers that talk to per share value. Check out Zevra Therapeutics' historical EPS growth by clicking on this link. Three years ago, Zevra Therapeutics lost money. And even focusing only on the last twelve months, we don't have a meaningful growth rate because it made a loss a year ago, too. But mathematics aside, it is always good to see when a formerly unprofitable business come good (though we accept profit would have been higher if dilution had not been required). Therefore, the dilution is having a noteworthy influence on shareholder returns. In the long term, if Zevra Therapeutics' earnings per share can increase, then the share price should too. But on the other hand, we'd be far less excited to learn profit (but not EPS) was improving. For that reason, you could say that EPS is more important that net income in the long run, assuming the goal is to assess whether a company's share price might grow. The fact that the company had unusual items boosting profit by US$123m, in the last year, probably goes some way to explain why its accrual ratio was so weak. We can't deny that higher profits generally leave us optimistic, but we'd prefer it if the profit were to be sustainable. When we crunched the numbers on thousands of publicly listed companies, we found that a boost from unusual items in a given year is often not repeated the next year. Which is hardly surprising, given the name. Zevra Therapeutics had a rather significant contribution from unusual items relative to its profit to March 2026. All else being equal, this would likely have the effect of making the statutory profit a poor guide to underlying earnings power. Zevra Therapeutics didn't back up its earnings with free cashflow, but this isn't too surprising given profits were inflated by unusual items. The dilution means the results are weaker when viewed from a per-share perspective. On reflection, the above-mentioned factors give us the strong impression that Zevra Therapeutics'underlying earnings power is not as good as it might seem, based on the statutory profit numbers. In light of this, if you'd like to do more analysis on the company, it's vital to be informed of the risks involved. At Simply Wall St, we found 1 warning sign for Zevra Therapeutics and we think they deserve your attention. Our examination of Zevra Therapeutics has focussed on certain factors that can make its earnings look better than they are. And, on that basis, we are somewhat skeptical. But there are plenty of other ways to inform your opinion of a company. Some people consider a high return on equity to be a good sign of a quality business. So you may wish to see this free collection of companies boasting high return on equity, or this list of stocks with high insider ownership. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) simplywallst.com.This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.

A week ago, Zevra Therapeutics, Inc. (NASDAQ:ZVRA) came out with a strong set of quarterly numbers that could potentially lead to a re-rate of the stock. It was a solid earnings report, with revenues and statutory earnings per share (EPS) both coming in strong. Revenues were 13% higher than the analysts had forecast, at US$36m, while EPS were US$0.60 beating analyst models by 567%. The analysts typically update their forecasts at each earnings report, and we can judge from their estimates whether their view of the company has changed or if there are any new concerns to be aware of. Readers will be glad to know we've aggregated the latest statutory forecasts to see whether the analysts have changed their mind on Zevra Therapeutics after the latest results. This technology could replace computers: discover the 20 stocks are working to make quantum computing a reality. Taking into account the latest results, the consensus forecast from Zevra Therapeutics' nine analysts is for revenues of US$144.9m in 2026. This reflects a meaningful 18% improvement in revenue compared to the last 12 months. Statutory earnings per share are forecast to nosedive 73% to US$0.54 in the same period. In the lead-up to this report, the analysts had been modelling revenues of US$143.3m and earnings per share (EPS) of US$0.52 in 2026. The analysts seems to have become more bullish on the business, judging by their new earnings per share estimates. Check out our latest analysis for Zevra Therapeutics There's been no major changes to the consensus price target of US$23.00, suggesting that the improved earnings per share outlook is not enough to have a long-term positive impact on the stock's valuation. There's another way to think about price targets though, and that's to look at the range of price targets put forward by analysts, because a wide range of estimates could suggest a diverse view on possible outcomes for the business. Currently, the most bullish analyst values Zevra Therapeutics at US$26.00 per share, while the most bearish prices it at US$18.00. These price targets show that analysts do have some differing views on the business, but the estimates do not vary enough to suggest to us that some are betting on wild success or utter failure. One way to get more context on these forecasts is to look at how they compare to both past performance, and how other companies in the same industry are performing. It's pretty clear that there is an expectation that Zevra Therapeutics' revenue growth will slow down substantially, with revenues to the end of 2026 expected to display 25% growth on an annualised basis. This is compared to a historical growth rate of 40% over the past five years. By way of comparison, the other companies in this industry with analyst coverage are forecast to grow their revenue at 8.7% annually. So it's pretty clear that, while Zevra Therapeutics' revenue growth is expected to slow, it's still expected to grow faster than the industry itself. The most important thing here is that the analysts upgraded their earnings per share estimates, suggesting that there has been a clear increase in optimism towards Zevra Therapeutics following these results. Happily, there were no major changes to revenue forecasts, with the business still expected to grow faster than the wider industry. The consensus price target held steady at US$23.00, with the latest estimates not enough to have an impact on their price targets. Keeping that in mind, we still think that the longer term trajectory of the business is much more important for investors to consider. At Simply Wall St, we have a full range of analyst estimates for Zevra Therapeutics going out to 2028, and you can see them free on our platform here.. And what about risks? Every company has them, and we've spotted 1 warning sign for Zevra Therapeutics you should know about. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) simplywallst.com.This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.

Q1 2026 net revenue of $36.2 million, a 78% increase over Q1 2025 Completed $50.0 million sale of SDX portfolio to Commave Therapeutics Operational execution fueled strong cash position of $236.8 million Company to host conference call and webcast TODAY, May 6, 2026, at 4:30 p.m. ET BOSTON, May 06, 2026 (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, today reported its financial results for the first quarter ended March 31, 2026. “We made meaningful progress across the business in the first quarter, led by continued performance for MIPLYFFA, underscoring its role in addressing the needs of patients with Niemann-Pick disease type C,” said Neil F. McFarlane, Zevra's President and Chief Executive Officer. “We also completed the $50.0 million divestiture of the SDX portfolio, and we repaid our term loan debt, sharpening our strategic focus and strengthening our financial position as we execute on our 2026 priorities.” MIPLYFFA® (arimoclomol) Highlights U.S.: Received nine MIPLYFFA prescription enrollment forms for Niemann-Pick disease type C (NPC) during Q1 2026, bringing the total to 170 since product launch. Market access remains stable at 69% of covered lives. EU: A Marketing Authorisation Application for the evaluation of arimoclomol for the treatment of NPC is under review by the European Medicines Agency (EMA). The Company submitted its response to the EMA’s 120-day list of questions within the 90-day clock stop period, advancing the application along the standard review process. Arimoclomol has been designated an Orphan Medicinal Product by the EMA. Global Expanded Access Program (EAP): As of March 31, 2026, 122 patients were enrolled in the global EAP. In the Journal of Inherited Metabolic Disease, MIPLYFFA was included in the newly updated Clinical Practice Guidelines for the treatment and management of NPC. Pipeline and Innovation Highlights Enrolled 10 patients in the event-driven Phase 3 DiSCOVER trial for the treatment of Vascular Ehlers-Danlos Syndrome during Q1 2026, bringing the total number of enrolled patients to 62, with a total of two confirmed events. The Company expects to hold a follow-up meeting with the Food and Drug Administration (FDA) in the second half of this year to explore pathways to accelerate clinical development. Corporate Highlights Executed $50.0 million SDX portfolio sale to Commave Therapeutics, strengthening the balance sheet and supporting strategic priorities. Prepaid the principal balance on its $63.1 million term loan in full, resulting in a strong, debt-free balance sheet and enhanced financial and strategic flexibility. Q1 2026 Financial Highlights Revenue, Net: $36.2 million for Q1 2026, which includes $24.6 million of MIPLYFFA net revenue, $0.3 million of OLPRUVA net revenue, $10.2 million in net reimbursements from our EAP, and $1.1 million in royalties and other reimbursements under the AZSTARYS® license agreement. This was an increase in total net revenue of $15.8 million compared to $20.4 million in Q1 2025. Cost of Product Revenue: $1.9 million for Q1 2026, excluding non-cash intangible asset amortization. Cost of product revenue for Q1 2025 was $1.3 million. Operating Expenses: $25.2 million for Q1 2026, which includes non-cash stock compensation expense of $3.1 million. Total operating expenses for Q1 2025 were $22.8 million. R&D expense was $4.4 million for Q1 2026, which was an increase of $1.1 million compared to $3.3 million for Q1 2025 due primarily to an increase in third-party costs incurred and professional fees. SG&A expense was $20.8 million for Q1 2026, which was an increase of $1.2 million compared to $19.5 million for Q1 2025, due primarily to an increase in professional fees, partially offset by a decrease in third party spending. Net income (loss): Net income of $37.9 million, or $0.62 per basic and $0.60 diluted share for Q1 2026, compared to a net loss of $(3.1) million, or $(0.06) per basic and diluted share, in Q1 2025. In Q1 2026, the Company received $40.5 million of the $45.0 million in net proceeds from the sale of the SDX portfolio. Excluding $43.3 million from the one-time gain on the sale of the SDX portfolio, one-time charges of $2.8 million in loss on extinguishment of debt and $7.2 million in loss on derivative liability and payoff premium related to the prepayment of the term loan in full, and $6.9 million in income tax expense related to the transaction, estimated adjusted quarterly net income would be $11.5 million, or $0.18 per diluted share.1 Cash Position: Cash, cash equivalents and securities were $236.8 million as of March 31, 2026. Based on its current operating forecast, the Company believes available financial resources are sufficient to execute on its strategic priorities independent from the capital markets. Common and Fully Diluted Shares O/S: As of March 31, 2026, total shares of common stock outstanding were 59,114,850, and fully diluted common shares were 68,946,838, which included 7,302,609 issuable from outstanding awards under equity incentive plans, and 2,529,379 shares issuable upon exercise of warrants. 1 Adjusted net income and adjusted net income per share are non-GAAP financial measures. Management believes that adjusted net income and adjusted net income per share provide useful information for investors, and management uses these supplemental measures to assess the Company’s operating performance. Adjusted net income and adjusted net income per share have limitations as analytical tools because they do not reflect all of the amounts associated with our results of operations as determined in accordance with U.S. GAAP. Additionally, they may not be comparable to similarly titled measures of other companies, including in our industry, limiting the usefulness of those measures for comparative purposes. Because of these limitations, these non-GAAP financial measures should be considered along with other operating and financial performance measures presented in accordance with U.S. GAAP. The presentation of these non-GAAP financial measures are not intended to be considered in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with U.S. GAAP. Conference Call Information Zevra will host a conference call and audio webcast TODAY at 4:30 p.m. ET to discuss its corporate update and financial results for the first quarter 2026. A link to the audio webcast is accessible on the “Events & Presentations” page in the Investor Relations section of the Zevra's website at investors.zevra.com. A replay of the webcast will be available for 90 days beginning at approximately 5:30 p.m. ET on May 6, 2026. Additionally, interested participants and investors may access the conference call by dialing either: (800) 245-3047 (United States) (203) 518-9765 (International) Conference ID: ZVRAQ126 About MIPLYFFA® (arimoclomol) MIPLYFFA (arimoclomol) is Zevra’s approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. The extensive data generated for MIPLYFFA has shown long-term, meaningful clinical outcomes with 5 and in some patients 7 years of patient experience across more than 270 NPC patients worldwide through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. Zevra has submitted a Marketing Authorization Application to the European Medicines Agency for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C. INDICATIONS AND USAGE MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions: Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve. Embryofetal Toxicity: MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential. Increased Creatinine without Affecting Glomerular Function: Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function. During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation. The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight. Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema. To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch. Drug Interaction(s): Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate. Use in Females and Males of Reproductive Potential: Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights. Renal Impairment: The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function. MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg. About OLPRUVA® OLPRUVA (sodium phenylbutyrate) is Zevra’s approved treatment for the treatment of certain UCDs. OLPRUVA (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapies, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). OLPRUVA is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. For more information, please visit www.OLPRUVA.com. Important Safety Information Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with OLPRUVA. Tell your doctor about all the medicines you or your child take, especially if you or your child take corticosteroids, valproic acid, haloperidol, and/or probenecid. OLPRUVA can cause serious side effects, including: 1) nervous system problems (neurotoxicity). Symptoms include sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, confusion, 2) low potassium levels in your blood (hypokalemia) and 3) conditions related to swelling (edema). OLPRUVA contains salt (sodium), which can cause swelling from salt and water retention. Tell your doctor right away if you or your child get any of these symptoms. Your doctor may do certain blood tests to check for side effects during treatment with OLPRUVA. If you have certain medical conditions such as heart, liver or kidney problems, are pregnant/planning to get pregnant or breast-feeding, your doctor will decide if OLPRUVA is right for you. The most common side effects of OLPRUVA include absent or irregular menstrual periods, decreased appetite, body odor, bad taste or avoiding foods you ate prior to getting sick (taste aversion). These are not all of the possible side effects of OLPRUVA. Call your doctor for medical advice about side effects. You may report side effects to U.S. FDA at 1-800-FDA-1088. About Celiprolol Celiprolol is Zevra’s investigational clinical candidate for the treatment of Vascular Ehlers-Danlos Syndrome (VEDS). Celiprolol has been granted Orphan Drug and Breakthrough Therapy designations by the U.S. FDA. Zevra recently restarted enrollment in the DiSCOVER trial, a Phase 3 trial being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. FDA. Celiprolol’s mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. The Company is focused on broadening access through geographic expansion opportunities, progressing its pipeline toward key milestones, and delivering meaningful therapeutics. The commercialization of its lead product, marketed in the U.S. for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disease, provides a strong corporate foundation and validates its ability to advance therapies from development to market. Zevra's vision is realized through disciplined execution of its strategic plan and core values — patient centricity, integrity, accountability, innovation, and courage — which guide its efforts to deliver long-term value. For more information, please visit www.zevra.com or follow us on X and LinkedIn. Cautionary Note Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the expected timing of EMA review of our MAA for arimoclomol; the potential to accelerate development of the Company's treatment for Vascular Ehlers-Danlos Syndrome and the timing of a follow-up meeting with FDA; and the sufficiency of the Company’s available financial resources to execute on its strategic priorities. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2025, filed on March 9, 2026, Quarterly Report on Form 10-Q for the three months ended March 31, 2026, to be filed with the SEC, as well as and Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Investor Contact Nichol Ochsner +1 (732) 754-2545 [email protected] Media Contact Julie Downs +1 (508) 246-3230 [email protected]

Interested in Zevra Therapeutics, Inc.? Here are five stocks we like better. Strong Q1 financials: Net revenue rose to $36.2 million (up 78% YoY) driven by MIPLYFFA sales and Global EAP reimbursements, and a one-time ~$43.3M gain from the SDX portfolio sale helped Zevra retire its debt — leaving it debt-free with $236.8M in cash and investments. MIPLYFFA commercial momentum: The NPC therapy has generated 170 prescription enrollment forms through March 31, is now included in updated NPC clinical guidelines, holds U.S. orphan exclusivity through 2031 (patent-extension pending), and is under review by the EMA. Late-stage pipeline progress: The Phase III DiSCOVER trial of celiprolol in vEDS has enrolled 62 patients with 2 of the 28 events required for an interim analysis, and Zevra reported constructive FDA Type C discussions with a follow-up meeting planned. Zevra Therapeutics (NASDAQ:ZVRA) reported first-quarter 2026 net revenue of $36.2 million, up 78% from the prior-year period, as the company highlighted continued adoption of its Niemann-Pick disease type C (NPC) therapy MIPLYFFA in the U.S., growth in its global expanded access programs, and progress in its late-stage celiprolol program for vascular Ehlers-Danlos syndrome (vEDS). President and CEO Neil McFarlane said the company is “building a durable rare disease company grounded in disciplined execution, financial strength, and a commitment to patients,” pointing to commercial execution for MIPLYFFA and efforts to expand access outside the U.S. → Berkshire Hathaway’s Record Cash Hoard: Why and What's Next? MIPLYFFA has generated 170 prescription enrollment forms from launch through March 31, including nine received in the first quarter. McFarlane noted estimated NPC prevalence of about 900 patients in the U.S., with roughly 300–350 currently diagnosed, and said the company has “successfully reached roughly half of this patient population.” On the intellectual property and exclusivity front, McFarlane said MIPLYFFA received orphan drug designation in the U.S., enabling marketing exclusivity through 2031. He added that Zevra is pursuing a patent term extension through the U.S. Patent Office, which “could provide coverage beyond 2031,” and said the company is awaiting a decision. → A Prada Payday: Is AMC Back in Style? Outside the U.S., McFarlane highlighted the company’s Global Expanded Access Program (EAP) for arimoclomol, stating that 122 patients were enrolled across geographies as of quarter-end. He cautioned that EAP enrollment and reimbursement can vary by country and program type—including compassionate use and named patient reimbursement—particularly early on, citing Zevra’s prior experience in France where the patient base has stabilized. Zevra also provided an update on its European regulatory process. McFarlane said the company’s marketing authorization application is under review at the European Medicines Agency (EMA), and that Zevra responded to the EMA’s 120-day list of questions within the 90-day clock-stop period. In response to an analyst question, McFarlane said the review engagement has been “fairly standard” and that, since submitting a new application, the company has not seen new questions beyond those encountered in prior submissions. → Insider Sales: Top AST SpaceMobile Insider Cuts Postion Over 30% Chief Commercial Officer Josh Schafer reviewed NPC disease background and emphasized the clinical dataset supporting MIPLYFFA, including “more than five years of data across more than 270 NPC patients worldwide” from clinical studies, open-label extension, global EAP, and a pediatric sub-study. Schafer said MIPLYFFA in combination with miglustat is “the first and only disease-modifying therapy shown to halt disease progression at 12 months in a randomized controlled trial” using the validated NPC Clinical Severity Scale, with benefit observed at 12 weeks and treatment effects sustained for over five years. During the quarter, Zevra said MIPLYFFA was added to NPC clinical practice guidelines published in the Journal of Inherited Metabolic Disease, described as the first guideline update since 2018. Schafer and McFarlane said the guideline update reinforces the importance of early detection, genetic testing, and the NPC Clinical Severity Scale, and supports consideration of combination therapy. In Q&A, Schafer said the guidelines represent the views of key opinion leaders and can help build consistency for healthcare providers less familiar with NPC. Zevra’s commercial strategy, Schafer said, focuses on three priorities: Accelerating time to diagnosis and treatment Driving demand Facilitating access To support earlier diagnosis, Schafer cited conference engagement, a disease awareness campaign (“Learn NPC, Read Between the Signs”), an AI-driven targeting model intended to identify likely NPC patients, and collaborations with genetic testing providers. He said Zevra is seeing increased demand outside traditional centers of excellence and an expanding prescriber base that includes community-based physicians. Schafer also discussed an “Expert Connect” initiative to link providers unfamiliar with NPC to experts who can answer questions about the disease and treatment options. He said the company’s treated patient mix is roughly evenly split between adults and children. From an access standpoint, Schafer said coverage is stable at 69% of covered lives, with reimbursement achieved through medical exception pathways for other patients. In response to a question about differences between formulary coverage and medical exceptions, McFarlane said the company is not contracting today and that net price has not materially changed, though quarter-to-quarter gross-to-net variability can occur. Schafer said formulary placement can reduce administrative burden and time to therapy, while Zevra’s patient services help offices navigate access processes. McFarlane also updated investors on the company’s late-stage celiprolol program. Zevra’s Phase III DiSCOVER trial is evaluating celiprolol in vEDS, a rare inherited connective tissue disorder driven by COL3A1 mutations. McFarlane said there are no approved therapies for vEDS and estimated roughly 7,500 individuals in the U.S. living with the condition. The company enrolled 62 patients in the DiSCOVER study as of quarter-end, including 10 enrolled in the first quarter. McFarlane said the trial is event-driven, with two confirmed events toward the 28 events required to trigger an interim analysis. He said the company is working to drive enrollment by building a network of genetic testing centers to improve diagnosis and strengthening connections with specialists who manage these patients. On regulatory strategy, McFarlane said Zevra held an FDA Type C meeting in the first quarter and is preparing for a follow-up meeting in the second half of 2026 to explore potential pathways to accelerate development. He characterized the FDA discussions as “constructive” and “informative,” but said it was “too early” to detail specific strategies, adding that the company is pursuing both enrollment initiatives and “additional pathways with additional data.” Chief Financial Officer Justin Renz reported first-quarter 2026 net revenue of $36.2 million versus $20.4 million a year earlier. Revenue components included: $24.6 million from MIPLYFFA U.S. net sales $0.3 million from OLPRUVA $10.2 million in net reimbursements from the Global EAP for arimoclomol $1.1 million in royalty revenue Renz noted the quarter had “one less shipment week” for MIPLYFFA in the U.S. due to the delivery calendar, contributing to channel inventory falling below the low end of Zevra’s targeted range. In Q&A, Renz said Zevra expects inventory to return within its targeted range by the end of the second quarter. Renz also detailed a March transaction in which Zevra agreed to sell its SDX portfolio to Commave Therapeutics for $50 million. He said assets were monetized because they were “not central to our core investment thesis,” and that Aquestive Therapeutics received $5 million, or 10% of gross proceeds, under contractual obligations. Zevra received $40.5 million of $45 million net proceeds in the first quarter and a final $4.5 million payment in April. Renz said Zevra retired its debt early in connection with the transaction, which he said would save “on average approximately $8 million a year” in future interest expense. The company is now debt-free. The quarter included a one-time gain of about $43.3 million, partially offset by an approximately $10 million expense tied to early debt extinguishment. Operating expenses were $25.2 million, up $2.4 million year over year. R&D expense rose to $4.4 million, and SG&A increased to $20.8 million. Renz said the company has utilized “the vast majority” of usable net operating loss carryforwards, and recorded an estimated tax provision of $6.9 million due to one-time transactions. Net income for the quarter was $37.9 million, or $0.60 per diluted share, compared with a net loss of $3.1 million in the prior-year quarter. Excluding one-time transactions and the related tax provision, Renz said estimated quarterly net income would be $11.5 million, or $0.18 per diluted share. As of March 31, 2026, cash, cash equivalents, and investments totaled $236.8 million, with no outstanding debt. McFarlane said the company’s strong balance sheet provides financial flexibility to drive growth. In closing remarks, McFarlane said Zevra has evolved its corporate profile by executing on strategic priorities, monetizing non-core assets, and strengthening leadership. He also said the company relocated its corporate headquarters to Boston. Looking ahead, McFarlane said Zevra is “anchored by a clinically meaningful commercial product with multiple opportunities for global growth, a late-stage pipeline, and a strong financial position,” adding that the team is focused on expanding its impact in rare diseases. Zevra Therapeutics, Inc discovers and develops various proprietary prodrugs to treat serious medical conditions in the United States. The company develops its products through Ligand Activated Therapy platform. Its lead product candidate is KP1077, consisting of KP1077IH, which is under Phase 2 clinical trial for the treatment of idiopathic hypersomnia, and KP1077N, which is under Phase ½ clinical trial to treat narcolepsy. The company is also developing Celiprolol, a prodrug product candidate that is under Phase 1/2 clinical trial for the treatment of vascular Ehlers Danlos syndrome. The article "Zevra Therapeutics Q1 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for May 2026.