Serdexmethylphenidate/Dexmethylphenidate

– Adds Highly Complementary and Differentiated Medicine with Significant Growth Potential to Collegium’s Existing ADHD Portfolio – – Extends Collegium’s Long-Term Revenue Outlook; AZSTARYS has Expected Patent Protection Through 2037 – – Collegium Raises 2026 Financial Guidance to Reflect Expected Immediate Accretion from Acquisition – – 2026 Total Product Revenues, Net Expected in the Range of $865 to $895 Million and Adjusted EBITDA in the Range of $475 to $500 Million – STOUGHTON, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- Collegium Pharmaceutical, Inc. (Nasdaq: COLL), today announced that it has completed the acquisition of AZSTARYS (serdexmethylphenidate and dexmethylphenidate), a central nervous system (CNS) stimulant prescription medicine used for the treatment of Attention Deficit Hyperactivity Disorder (ADHD) in people 6 years of age and older. Collegium also raised its 2026 financial guidance to include the anticipated impact of the AZSTARYS acquisition. “We are pleased to complete the acquisition of AZSTARYS, a highly strategic addition to our portfolio that strengthens our position in ADHD and further reinforces our long-standing commitment to improving patient care and delivering shareholder value,” said Vikram Karnani, President and Chief Executive Officer. “AZSTARYS is a complementary and differentiated therapy that expands the treatment options we can offer patients and prescribers, and we look forward to rapidly integrating it into our existing commercial infrastructure. This transaction aligns with our disciplined capital deployment approach, and we expect it to be immediately accretive, enabling us to raise our 2026 financial guidance.” Strategic Rationale Strategically aligns with Collegium’s mission of building a leading, diversified biopharmaceutical company committed to improving the lives of people living with serious medical conditions by expanding its position in ADHD and further diversifying its commercial portfolio beyond responsible pain management. Leverages Collegium’s established ADHD commercial infrastructure and expertise to accelerate AZSTARYS’ growth trajectory and drive operational efficiencies, further strengthening Collegium’s financial position. Annual run rate synergies expected to be in excess of $50 million within twelve months. Strengthens Collegium’s position in ADHD. AZSTARYS generated more than 760,000 prescriptions in 2025 and adds a complementary medicine to Collegium’s ADHD portfolio. AZSTARYS is expected to generate $60 to $70 million of net revenue during the remainder of 2026. Expected to extend the longevity of Collegium’s revenue base as AZSTARYS is supported by six Orange Book-listed patents, most of which do not expire until December 2037. Further strengthens Collegium’s financial position by increasing revenues, supporting margin expansion, and enhancing future cash flow generation. For additional background on the acquisition, please read the announcement press release here and view Collegium’s investor presentation here . Additional Transaction Details Under the terms of the agreement, Collegium acquired the AZSTARYS business from Corium Therapeutics for approximately $650 million in cash (subject to customary adjustments for net working capital, indebtedness, cash, and transaction expenses), which was funded by approximately $350 million of Collegium’s existing cash on hand and $300 million from a delayed draw term loan which is part of the syndicated credit facility announced by Collegium in December 2025. Collegium may also pay Corium Therapeutics up to $135 million in additional consideration if AZSTARYS achieves certain future commercial and manufacturing milestones. Financial Guidance for 2026 Collegium raises its full-year 2026 financial guidance for Product Revenues, Net, and Adjusted EBITDA, which now includes the anticipated impact of the acquisition of AZSTARYS. Prior Updated Product Revenues, Net $805 to $825 million $865 to $895 million JORNAY PM Revenue, Net $190 to $200 million Unchanged AZSTARYS Revenue, Net N/A $60 to $70 million Adjusted EBITDA $455 to $475 million $475 to $500 million Leadership Updates The Company also announced two leadership updates. Scott Dreyer will depart from his role as Chief Commercial Officer effective at the end of August 2026 and will remain with the Company through that time to support a smooth transition. In addition, Thomas Smith, M.D., will depart from his role as Chief Medical Officer following a transition period during which the Company will conduct a search for his successor. “I want to thank Scott and Tom for their leadership and meaningful contributions to Collegium,” said Vikram Karnani. “Scott has played an important role in building and scaling our commercial organization, and Tom has helped shape our medical and scientific foundation. We are grateful for their commitment and impact.” About Collegium Pharmaceutical, Inc. Collegium Pharmaceutical is a dynamic, biopharmaceutical company delivering medicines with formulation and delivery innovation for people living with complex central nervous system and pain conditions. Collegium has spent more than a decade proving that responsible stewardship and bold, science-backed approaches can redefine what treatment looks like in categories too often shaped by complexity and misconceptions. With a portfolio of differentiated ADHD medications, anchored by JORNAY PM® (methylphenidate HCl) and AZSTARYS® (serdexmethylphenidate and dexmethylphenidate), and an established leadership position in responsible pain management, Collegium leads with the scientific rigor and commercial expertise to deliver treatment options around how people live their lives. For more information, please visit  collegiumpharma.com or find us on LinkedIn . Non-GAAP Financial Measures To supplement our financial results presented on a GAAP basis, we have included information about certain non-GAAP financial measures. We believe the presentation of these non-GAAP financial measures, when viewed with our results under GAAP and the accompanying reconciliations, provide analysts, investors, lenders, and other third parties with insights into how we evaluate normal operational activities, including our ability to generate cash from operations, on a comparable year-over-year basis and manage our budgeting and forecasting. In addition, certain non-GAAP financial measures, primarily adjusted EBITDA, are used to measure performance when determining components of annual compensation for substantially all non-sales force employees, including senior management. In this press release we discuss the following financial measures that are not calculated in accordance with GAAP. Adjusted EBITDA Adjusted EBITDA is a non-GAAP financial measure that represents GAAP net income or loss adjusted to exclude interest expense, interest income, the benefit from or provision for income taxes, depreciation, amortization, stock-based compensation, and other adjustments to reflect changes that occur in our business but do not represent ongoing operations. Adjusted EBITDA, as used by us, may be calculated differently from, and therefore may not be comparable to, similarly titled measures used by other companies. There are several limitations related to the use of adjusted EBITDA rather than net income or loss, which is the nearest GAAP equivalent, such as: adjusted EBITDA excludes depreciation and amortization, and, although these are non-cash expenses, the assets being depreciated or amortized may have to be replaced in the future, the cash requirements for which are not reflected in adjusted EBITDA; adjusted EBITDA does not reflect changes in, or cash requirements for, working capital needs; adjusted EBITDA does not reflect the benefit from or provision for income taxes or the cash requirements to pay taxes; adjusted EBITDA does not reflect historical cash expenditures or future requirements for capital expenditures or contractual commitments; we exclude stock-based compensation expense from adjusted EBITDA although: (i) it has been, and will continue to be for the foreseeable future, a significant recurring expense for our business and an important part of our compensation strategy; and (ii) if we did not pay out a portion of our compensation in the form of stock-based compensation, the cash salary expense included in operating expenses would be higher, which would affect our cash position; we exclude impairment expenses from adjusted EBITDA and, although these are non-cash expenses, the asset(s) being impaired may have to be replaced in the future, the cash requirements for which are not reflected in adjusted EBITDA; we exclude restructuring expenses from adjusted EBITDA. Restructuring expenses primarily include employee severance and contract termination costs that are not related to acquisitions. The amount and/or frequency of these restructuring expenses are not part of our underlying business; we exclude litigation settlements and contingencies that are subject to recovery from adjusted EBITDA, as well as any applicable income items, credit adjustments, or recoveries due to subsequent changes in estimates. This does not include our legal fees to defend claims, which are expensed as incurred; we exclude acquisition related expenses as the amount and/or frequency of these expenses are not part of our underlying business. Acquisition related expenses include transaction costs, which primarily consisted of financial advisory, banking, legal, and regulatory fees, and other consulting fees, incurred to complete the acquisition, employee-related expenses (severance cost and benefits) for terminated employees after the acquisition, legal defense expenses for specific acquired claims that relate to acts that occurred prior to our acquisition, and miscellaneous other acquisition related expenses incurred; we exclude recognition of the step-up basis in inventory from acquisitions (i.e., the adjustment to record inventory from historic cost to fair value at acquisition) as the adjustment does not reflect the ongoing expense associated with sale of our products as part of our underlying business; we exclude changes in the fair value of contingent consideration, which are non‑cash, acquisition‑related items that are not part of our underlying business; we exclude losses on extinguishments of debt as these expenses are episodic in nature and do not directly correlate to the cost of operating our business on an ongoing basis; we exclude executive transition expenses from adjusted EBITDA as the amount and/or frequency of these expenses are episodic in nature and do not directly correlate to the cost of operating our business on an ongoing basis; and we exclude other expenses, from time to time, that are episodic in nature and do not directly correlate to the cost of operating our business on an ongoing basis. The Company has not provided a reconciliation of its full-year 2026 guidance for adjusted EBITDA to the most directly comparable forward-looking GAAP measures, in reliance on the unreasonable efforts exception provided under Item 10(e)(1)(i)(B) of Regulation S-K, because the Company is unable to predict, without unreasonable efforts, the timing and amount of items that would be included in such a reconciliation, including, but not limited to, stock-based compensation expense, acquisition related expenses, amortization of acquired intangible assets, and changes in fair value of contingent consideration. These items are uncertain and depend on various factors that are outside of the Company’s control or cannot be reasonably predicted. While the Company is unable to address the probable significance of these items, they could have a material impact on GAAP net income and operating expenses for the guidance period. A reconciliation of adjusted EBITDA would imply a degree of precision and certainty as to these future items that does not exist and could be confusing to investors. Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as "predicts," "forecasts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Examples of forward-looking statements contained in this press release include, among others, projected financial performance, including expected revenue and adjusted EBITDA; statements related to the anticipated benefits of the acquisition of AZSTARYS, including its impact on Collegium’s ADHD portfolio and commercial strategy; statements related to current and future market opportunities for our products and our assumptions related thereto and other statements that are not historic facts. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results, performance, or achievements to differ materially from the company's current expectations, including risks relating to, among others: our ability to realize the anticipated benefits of the AZSTARYS acquisition, including the possibility that the expected benefits from the acquisition will not be realized or will not be realized within the expected time period; the risk that the businesses will not be integrated successfully; disruption from the transaction making it more difficult to maintain business and operational relationships; negative effects of this announcement or the consummation of the acquisition on the market price of our common stock and/or operating results; significant transaction costs or the acquisition of unknown liabilities; potential litigation related to the acquisition; future opportunities and plans for AZSTARYS, including uncertainty of the expected financial performance of AZSTARYS; future opportunities and plans for our products, including uncertainty of the expected financial performance of such products; our ability to commercialize and grow sales of our products; our ability to manage our relationships with licensors; the success of competing products that are or become available; our ability to maintain regulatory approval of our products, and any related restrictions, limitations, and/or warnings in the label of our products; the size of the markets for our products, and our ability to service those markets; our ability to obtain reimbursement and third-party payor contracts for our products; the rate and degree of market acceptance of our products; the costs of commercialization activities, including marketing, sales and distribution; changing market conditions for our products; the outcome of any patent infringement or other litigation that may be brought by or against us; the outcome of any governmental investigation related to our business; our ability to secure adequate supplies of active pharmaceutical ingredient for each of our products and manufacture adequate supplies of commercially saleable inventory; our ability to obtain funding for our operations and business development; regulatory developments in the U.S.; our expectations regarding our ability to obtain and maintain sufficient intellectual property protection for our products; our ability to comply with stringent U.S. and foreign government regulation in the manufacture of pharmaceutical products, including U.S. Drug Enforcement Agency compliance; our customer concentration; and the accuracy of our estimates regarding expenses, revenues, capital requirements and need for additional financing. These and other risks are described under the heading "Risk Factors" in our Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q and other filings with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release. About AZSTARYS ® AZSTARYS is a central nervous system (CNS) stimulant indicated for the treatment of Attention Deficit Hyperactivity Disorder (ADHD) in patients aged 6 years and older. The use of AZSTARYS is not recommended in pediatric patients younger than 6 years of age because they had higher plasma exposure and a higher incidence of adverse reactions (e.g., weight loss) than patients 6 years and older at the same dosage. IMPORTANT SAFETY INFORMATION BOXED WARNING: ABUSE, MISUSE, AND ADDICTION See full prescribing information for complete boxed warning. AZSTARYS has a high potential for abuse, misuse, which can lead to the development of a substance use disorder, including addiction. Misuse and abuse of CNS stimulants, including AZSTARYS, can result in overdose and death. Before prescribing, assess each patient’s risk for abuse, misuse, and addiction. Educate patients and families about risks, proper storage, and proper disposal of any unused drug. Frequently monitor for signs and symptoms of abuse, misuse, and addiction throughout treatment. CONTRAINDICATIONS Known hypersensitivity to serdexmethylphenidate, methylphenidate, or product components. Concurrent treatment with a monoamine oxidase inhibitor (MAOI), or use of an MAOI within the preceding 14 days. WARNINGS AND PRECAUTIONS Risks to patients with serious cardiac disease: Avoid use in patients with known structural cardiac abnormalities, cardiomyopathy, serious cardiac arrhythmias, coronary artery disease, or other serious cardiac disease. Increased blood pressure and heart rate: Monitor blood pressure and pulse. Psychiatric adverse reactions: Prior to initiating AZSTARYS, screen patients for risk factors for developing a manic episode. If new psychotic or manic symptoms occur, consider discontinuing AZSTARYS. Priapism: If abnormally sustained or frequent and painful erections occur, patients should seek immediate medical attention. Peripheral vasculopathy, including Raynaud's Phenomenon: Careful observation for digital changes is necessary during AZSTARYS treatment with ADHD stimulants. Further clinical evaluation (e.g., rheumatology referral) may be appropriate for patients who develop signs or symptoms of peripheral vasculopathy (5.6) Long-term suppression of growth in pediatric patients: Monitor height and weight at appropriate intervals in pediatric patients. Acute angle closure glaucoma: AZSTARYS-treated patients considered at risk for acute angle closure glaucoma (e.g., patients with significant hyperopia) should be evaluated by an ophthalmologist. Increased intraocular pressure (IOP) and glaucoma: Prescribe AZSTARYS to patients with open-angle glaucoma or abnormally increased IOP only if the benefit of treatment is considered to outweigh the risk. Closely monitor patients with a history of increased IOP or open angle glaucoma. Motor and verbal tics, and worsening of Tourette’s syndrome: Before initiating AZSTARYS, assess the family history and clinically evaluate patients for tics or Tourette’s syndrome. Regularly monitor patients for the emergence or worsening of tics or Tourette’s syndrome. Discontinue treatment if clinically appropriate. ADVERSE REACTIONS The most common adverse reactions (≥5% and twice the rate of placebo) include decreased appetite, decreased weight, nausea, abdominal pain, dyspepsia, vomiting, insomnia, anxiety, affect lability, irritability, dizziness, increased blood pressure, and tachycardia. DRUG INTERACTIONS Antihypertensive Drugs: Monitor blood pressure. Adjust dosage of antihypertensive drug as needed Please visit for additional important safety information and the Full Prescribing Information, including Boxed Warning, for AZSTARYS. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. Investor Contacts: Ian Karp Head of Investor Relations ir@collegiumpharma.com Danielle Jesse Director, Investor Relations ir@collegiumpharma.com Media Contact: Jessica Cotrone Senior Vice President, Communications & Corporate Affairs communications@collegiumpharma.com

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 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 m 2 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 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 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 nochsner@zevra.com Media Contact Julie Downs +1 (508) 246-3230 jdowns@zevra.com ZEVRA THERAPEUTICS, INC. UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except share and per share amounts) Three months ended March 31, 2026 2025 Revenue, net $ 36,220 $ 20,401 Cost of product revenue (excluding $316 and $1,615 in intangible asset amortization for the three months ended March 31, 2026, and 2025, respectively, shown separately below) 1,897 1,345 Intangible asset amortization 316 1,615 Gain on sale of future royalties, intellectual property, and other assets, net 43,314 — Operating expenses: Research and development 4,392 3,258 Selling, general and administrative 20,783 19,545 Total operating expenses 25,175 22,803 Income (loss) from operations 52,146 (5,362 ) Other (expense) income: Loss on extinguishment of debt (2,756 ) — Loss on derivative liability (7,216 ) — Interest expense (1,711 ) (1,969 ) Fair value adjustment related to warrant and CVR liability 968 4,874 Fair value adjustment related to investments (216 ) (3 ) Interest and other income, net 3,589 543 Total other (expense) income (7,342 ) 3,445 Income (loss) before income taxes 44,804 (1,917 ) Income tax expense (6,914 ) (1,182 ) Net income (loss) $ 37,890 $ (3,099 ) Net income (loss) per share of common stock: Basic $ 0.62 $ (0.06 ) Diluted $ 0.60 $ (0.06 ) Weighted-average shares of common stock outstanding: Basic 58,405,955 54,095,543 Diluted 60,230,490 54,095,543 ZEVRA THERAPEUTICS, INC. UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands, except share and par value amounts) March 31, 2026 December 31, 2025 Assets Current assets: Cash and cash equivalents $ 95,595 $ 62,406 Investments, current 105,021 128,605 Accounts and other receivables 22,098 23,258 Prepaid expenses and other current assets 4,944 6,998 Inventories, current 2,310 1,740 Total current assets 229,968 223,007 Investments, noncurrent 36,145 47,879 Inventories, noncurrent — 879 Property and equipment, net 430 489 Operating lease right-of-use assets 1,087 1,212 Goodwill 4,701 4,701 Intangible assets, net 6,105 6,421 Other long-term assets 143 143 Total assets $ 278,579 $ 284,731 Liabilities and stockholders' equity Current liabilities: Accounts payable and accrued expenses $ 11,688 $ 11,598 Current portion of operating lease liabilities 406 419 Current portion of discount and rebate liabilities 13,172 12,188 Current portion of income tax payable 20,354 13,710 Other current liabilities 1,408 1,362 Total current liabilities 47,028 39,277 Long-term debt — 61,928 Warrant liability 6,797 9,575 Income tax payable 6,871 7,029 Operating lease liabilities, less current portion 741 859 Discount and rebate liabilities, less current portion 9,479 9,693 Other long-term liabilities 1,859 1,713 Total liabilities 72,775 130,074 Commitments and contingencies Stockholders’ equity: Preferred stock: Undesignated preferred stock, $0.0001 par value, 10,000,000 shares authorized, no shares issued or outstanding as of March 31, 2026, or December 31, 2025 — — Common stock, $0.0001 par value, 250,000,000 shares authorized; 60,690,542 shares issued and 59,114,850 shares outstanding as of March 31, 2026; 58,338,319 shares issued and 56,854,781 shares outstanding as of December 31, 2025 6 6 Additional paid-in capital 602,748 588,458 Treasury stock, at cost (10,983 ) (10,983 ) Accumulated deficit (384,170 ) (422,060 ) Accumulated other comprehensive loss (1,797 ) (764 ) Total stockholders' equity 205,804 154,657 Total liabilities and stockholders' equity $ 278,579 $ 284,731