Sodium Phenylbutyrate

$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 . R&D expense was $4.5 million for Q2 2026, which was an increase of $1.1 million compared to $3.4 million for Q2 2025, due primarily to an increase in spending for ongoing arimoclomol efforts. SG&A expense was $16.6 million for Q2 2026, which was a decrease of $4.2 million compared to $20.8 million for Q2 2025, due primarily to decreases in professional fees and third party spending, partially offset by an increase in personnel related costs. 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. In Q2 2026, the Company recognized $6.4 million in noncash fair value adjustments related to existing warrant liabilities and contingent value rights (CVR) compared to $0.8 million in Q2 2025. R&D expense was $4.5 million for Q2 2026, which was an increase of $1.1 million compared to $3.4 million for Q2 2025, due primarily to an increase in spending for ongoing arimoclomol efforts. SG&A expense was $16.6 million for Q2 2026, which was a decrease of $4.2 million compared to $20.8 million for Q2 2025, due primarily to decreases in professional fees and third party spending, partially offset by an increase in personnel related costs. In Q2 2026, the Company recognized $6.4 million in noncash fair value adjustments related to existing warrant liabilities and contingent value rights (CVR) compared to $0.8 million in Q2 2025. In addition, in Q2 2025, the Company recognized a one-time gain on the sale of a priority review voucher of $148.3 million , offset by a one time charge of $58.7 million related to impairment of intangible assets and an $11.7 million charge related to inventory obsolescence. Excluding these one-time charges of $6.4 million in Q2 2026 and the net $77.1 million income in Q2 2025 related to the Priority Review Voucher sale, OLPRUVA impairment and warrant and CVR fair value adjustment, the Company would have recognized estimated adjusted quarterly net income (loss) of $15.2 million , or $0.25 per diluted share in Q2 2026 and ($2.4) million , or ( $0.04 ) per diluted share in Q2 2025.1 In addition, in Q2 2025, the Company recognized a one-time gain on the sale of a priority review voucher of $148.3 million , offset by a one time charge of $58.7 million related to impairment of intangible assets and an $11.7 million charge related to inventory obsolescence. Excluding these one-time charges of $6.4 million in Q2 2026 and the net $77.1 million income in Q2 2025 related to the Priority Review Voucher sale, OLPRUVA impairment and warrant and CVR fair value adjustment, the Company would have recognized estimated adjusted quarterly net income (loss) of $15.2 million , or $0.25 per diluted share in Q2 2026 and ($2.4) million , or ( $0.04 ) per diluted share in Q2 2025.1 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) 754-2545nochsner@zevra.com Media Contact Julie Downs+1 (508) 246-3230jdowns@zevra.com Source: Zevra Therapeutics

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced that Week 24 and Week 48 results from the Phase 2 open-label HELIOS clinical trial of AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol, in adults living with Wolfram syndrome have been published in The Journal of Clinical Investigation, a peer-reviewed medical journal. The publication reports Week 24 and Week 48 results from the Phase 2 open‑label HELIOS trial previously presented in part at medical meetings. Consistent with earlier disclosures, the findings indicate continued improvements observed in pancreatic beta cell function through Week 48, as measured by C‑peptide response to a mixed‑meal tolerance test. Additionally, secondary measures of glycemic control, including HbA1c and time in target glucose range (70–180 mg/dL), demonstrated improvement from baseline at Weeks 24 and 48, while best-corrected visual acuity trended toward stabilization over the 48‑week period. Participant and Clinician Global Impression of Change assessments also classified participants with available data as responders, defined by improvement or disease stabilization. “Wolfram syndrome is a rare genetic disease that presents significant challenges for patients and their families. It typically begins in childhood, with insulin-requiring diabetes and progressive optic nerve changes affecting vision, and over time can involve broader neurological symptoms that increasingly affect daily life, with no disease-modifying treatments approved today,” said Fumihiko Urano, MD, PhD, Principal Investigator of the HELIOS trial and the Samuel E. Schechter Professor of Medicine in the Division of Endocrinology, Metabolism & Lipid Research at Washington University School of Medicine in St. Louis. “In this context, the publication of the HELIOS results at Weeks 24 and 48 is highly encouraging. The observed objective improvement in pancreatic function and visual acuity stabilization over 48 weeks, combined with a favorable safety profile, reinforces AMX0035’s potential to meaningfully alter the trajectory of this disorder.” “We are excited to share the peer-reviewed publication of these results, which details the clinical findings of AMX0035 at Week 24 and 48 across both the endocrine and neurodegenerative manifestations of Wolfram syndrome. These findings are encouraging given that all trial participants were at least 17 years old, and their disease course had advanced considerably by the time of enrollment,” said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. “We extend our deepest gratitude to Dr. Urano, the clinical trial site staff, and most importantly, the trial participants and their families. Your unwavering dedication makes this research possible as we continue to work with the FDA on a Phase 3 clinical trial in Wolfram syndrome.” The peer-reviewed publication also details qualitative interviews that reinforced the clinical relevance of these objective outcomes, with participants indicating meaningful improvements in diabetes and vision problems. Furthermore, the published safety findings remained consistent with prior clinical experience with AMX0035, with reported adverse events being mild or moderate and no serious adverse events related to treatment. HELIOS is a single-site, single-arm, open-label, Phase 2 clinical trial investigating AMX0035 in 12 adults living with Wolfram syndrome for up to 208 weeks followed by a four-week safety follow-up. The Company expects to present additional longer-term data from HELIOS at an upcoming scientific meeting. About AMX0035 AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the U.S.). AMX0035 was designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum (ER) stress and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration. Amylyx believes that its proprietary combination of PB and TURSO and their complementary mechanisms of action will allow us to synergistically target abnormal cell death to better prevent neurodegeneration than treatment targeted at either mechanism of action alone. The FDA and the European Commission granted Orphan Drug Designation to AMX0035 for the treatment of Wolfram syndrome in November 2020 and August 2024, respectively. About Wolfram Syndrome Wolfram syndrome is a rare, monogenic, progressive neurodegenerative disorder that progressively impacts multiple organs and systems. Wolfram syndrome is characterized by childhood-onset diabetes mellitus, optic nerve atrophy, and neurodegeneration. Common manifestations of Wolfram syndrome include diabetes mellitus and diabetes insipidus, gradual vision loss leading to blindness, hearing loss, neurogenic bladder, difficulties with balance and coordination, and difficulty breathing that can lead to respiratory failure and premature mortality. Wolfram syndrome is most commonly caused by pathogenic variants in Wolfram syndrome type 1 gene (WFS1). Because of the clear link between WFS1 mutations and endoplasmic reticulum (ER) stress, Wolfram syndrome is considered a prototypical ER stress disorder. Wolfram syndrome affects approximately 3,000 people living in the U.S., and there are currently no FDA-approved treatment options. About the HELIOS Trial HELIOS (NCT05676034) is a 12-participant, single-site, single-arm, open-label, Phase 2 trial designed to evaluate the safety and tolerability of AMX0035, as well as its effects on various measures of endocrinological, neurological, and ophthalmologic function in adult participants living with Wolfram syndrome. Participants in HELIOS receive AMX0035 for up to 208 weeks followed by a four-week safety follow-up. Primary and secondary outcomes are assessed at Week 24 and at longer-term time points. About Amylyx Pharmaceuticals At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on four investigational therapies across several endocrine conditions and neurodegenerative diseases in which we believe can make the greatest impact. For more information, visit amylyx.com and follow us on LinkedIn and X. For investors, please visit investors.amylyx.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx’s expectations regarding: the potential of AMX0035 (sodium phenylbutyrate and taurursodiol) as a treatment for Wolfram syndrome; and plans for a Phase 3 trial of AMX0035 in Wolfram syndrome. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’s program development activities; Amylyx’s ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; the risk that early-stage results may not reflect later-stage results; Amylyx’s ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx’s operations, as well as the risks and uncertainties set forth in Amylyx’s United States Securities and Exchange Commission (SEC) filings, including Amylyx’s Annual Report on Form 10-K for the year ended December 31, 2025, and subsequent filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.