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CELEBRATION, Fla., April 17, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announces the publication of “Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease” in Molecular Genetics and Metabolism. “This elucidation of MIPLYFFA’s® highly differentiated mechanism of action marks a critical step in understanding its interactions with Niemann-Pick disease type C (NPC) at a cellular level,” said Adrian Quartel, MD, FFPM, Zevra’s Chief Medical Officer. “These insights further confirm that MIPLYFFA addresses the underlying pathology of NPC and supports the long-term treatment benefit observed in our clinical trials. Our findings may inform more effective treatment strategies for patients.” The publication presents data demonstrating that arimoclomol enters the cell and increases the translocation of translation factors EB and E3 (FTEB & TFE3) from the cytosol to the nucleus, a key initial step for triggering a cascade of downstream events. Specifically, TFEB & TFE3 upregulate the coordinated lysosomal expression and regulation (CLEAR) genes including NPC1, which is essential for the regulation of lysosomal function. Increased CLEAR gene expression then causes higher NPC1 protein levels in the lysosomes, leading to greater correction of aberrant cholesterol trafficking. Of note, in animal NPC models, greater correction of aberrant cholesterol trafficking was shown to correlate with the improvement of specific neurological behaviors, such as rearing and gait. About MIPLYFFA® (arimoclomol) MIPLYFFA (arimoclomol) is Zevra’s approved therapy for use in combination with miglustat 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. 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. Call your doctor for medical advice about side effects. You may report side effects to Zevra at 1-844-600-2237, or to the 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 Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage rare disease company combining science, data, and patient need to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. Expanded access programs are made available by Zevra Therapeutics, Inc. and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy, as published on its website. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion. 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 sale of the PRV and anticipated proceeds therefrom; promise and potential impact of our preclinical or clinical trial data; the initiation, timing and results of any clinical trials or readouts, the content, information used for, timing or results of any NDA submissions or resubmissions for any products or product candidates for any specific disease indication or at any dosage; the potential benefits of any of our products or product candidates for any specific disease or at any dosage; future research and development activities; our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company; the potential benefits of our debt facility; our financial position, including our cash balance and anticipated cash runway; potential revenues from MIPLYFFA sales; potential revenues from our arimoclomol expanded access program in France; the potential for royalty and milestone contributions, the presentation of data at conferences; and the timing of any of the foregoing. 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, including that the PRV sale is subject to conditions and may not close in the timeframe expected or at all. 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, 2024, 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. Zevra Contact Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com Russo Partners Contact David Schull+1 (858) 717-2310david.schull@russopartnersllc.com

MIPLYFFA, the first FDA-approved treatment for Niemann-Pick disease type C, is available at Zevra’s specialty pharmacy for dispense AmplifyAssist™, Zevra’s comprehensive patient support program, in place to address access barriers Nov. 21, 2024 -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that MIPLYFFA™ (MY-PLY-FAH) (arimoclomol), the first treatment approved by the U.S. Food and Drug Administration (FDA) for the treatment of Nieman Pick disease type C (NPC), is now commercially available for dispense. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. “Early adoption of MIPLYFFA has exceeded our expectations, and we are pleased to report that product is now available for shipment to NPC patients. This is in line with the shorter timeframe of our guidance of eight to 12 weeks post-approval,” said Josh Schafer, Chief Commercial Officer and Executive Vice President of Business Development, Zevra Therapeutics. “We continue to receive prescription enrollments through our patient services program, AmplifyAssist, and are supporting patients as they navigate the benefits verification process to obtain MIPLYFFA.” AmplifyAssist is Zevra’s comprehensive patient support program designed to support the individual needs of eligible patients and those who care for them. Available resources include personalized insurance coverage education and support, copay and alternate funding identification assistance for eligible patients’ product needs, disease state information and therapy management counseling, and ongoing interactions to address barriers while facilitating timely prescription refills. Information about the program is available at MIPLYFFA.com or via telephone. The AmplifyAssist team can be reached toll-free at (888) 668-4198 from 8 a.m. CT to 6 p.m. CT Monday through Friday. Patients and caregivers should speak with their physician to get started with MIPLYFFA. Physicians can fill out an enrollment form at MIPLYFFA.com, order the prescription, and when approved, the medication will be mailed to their patient’s home. Healthcare providers and patients/caregivers should refer to the Full Prescribing Information and Instructions for Use for information on the proper administration of MIPLYFFA. Zevra is committed to assisting those whose lives are affected by NPC to overcome the barriers and challenges that may impact their treatment journey. AmplifyAssist is Zevra’s comprehensive patient support program. The mission of the program is to support the individual needs of eligible patients and those who care for them. Available resources include personalized insurance coverage education and support, copay and alternate funding identification assistance for eligible patients’ product needs, disease state information and therapy management counseling, and ongoing interactions to address barriers while facilitating timely prescription refills. Information about the program is available at MIPLYFFA.com or via telephone. The AmplifyAssist team can be reached toll-free at (888) 668-4198 from 8 a.m. CT to 6 p.m. CT Monday through Friday. Healthcare providers who want to submit prescriptions can visit MIPLYFFA.com to complete the prescription enrollment form that initiates the process for accessing the treatment. 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 was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation by the FDA for the treatment of NPC. MIPLYFFA was further granted Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various cell types, including neurons. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making the NPC1 and NPC2 lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC can lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease diagnosis can often take years, with disease progression being irreversible and often leading to early mortality. Zevra Therapeutics, Inc. is a commercial-stage rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. Expanded access programs are made available by Zevra Therapeutics, Inc. and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy, as published on its website. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion. 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FDA approval of MIPLYFFA™ and product launch cap third quarter filled with multiple commercial and clinical development milestones Zevra received rare pediatric disease Priority Review Voucher Company ends quarter with cash, cash equivalents and investments of $95.5 million Company to host conference call and webcast today, Nov. 12, 2024, 4:30 p.m. ET CELEBRATION, Fla., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today reported its financial results for the third quarter ended Sept. 30, 2024, and provided corporate updates. “The third quarter was one of the most exciting and transformational periods in Zevra’s journey,” said Neil F. McFarlane, Zevra’s President and Chief Executive Officer. “After years of tireless effort, our team has achieved a major milestone with FDA approval of MIPLYFFA™ (arimoclomol), and we’re celebrating with the Niemann-Pick disease type C (NPC) community. We intend to achieve our goals with a high-performing team committed to execute, focus and innovate to drive our continued growth and long-term transformation.” Program Highlights On Sept. 20, the U.S. Food and Drug Administration (FDA) approved MIPLYFFA capsules as an orally delivered treatment for NPC. MIPLYFFA, the first NPC drug approved by the FDA, is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. With the approval of MIPLYFFA, Zevra received a rare pediatric disease Priority Review Voucher (PRV) which the Company could monetize to help fund its growth. As of Oct. 31, Zevra has received 90 prescription enrollment forms for MIPLYFFA of which 30% are approved for reimbursement and are ready for fulfillment upon drug availability, consistent with the Company’s prior guidance of eight to 12 weeks post launch. Zevra has launched AmplifyAssist™, a comprehensive support program for caregivers and those eligible living with NPC or UCDs. OLPRUVA® had three new patient enrollments in the third quarter, and reimbursement coverage is at approximately 76% of covered lives. Improving OLPRUVA’s formulary position is a primary objective to make it the preferred treatment for UCDs. Zevra is refining its commercial strategy for OLPRUVA to focus on specific patient segments where there are fewer access barriers, and, together with The National Urea Cycle Disorders Foundation, launched a targeted patient education campaign highlighting the importance of prompt and accurate testing of blood ammonia levels, which, when elevated, may be a sign of a UCD. Campaign materials are available at www.checkammonia.com. The Company recently completed an End-of-Phase 2 meeting with the FDA for KP1077. The Agency indicated that a single pivotal study with appropriate confirmatory evidence will be sufficient to submit a new drug application (NDA). Zevra is evaluating strategic alternatives to advance clinical development and later, commercialization. The Phase 3 DiSCOVER trial of celiprolol for the treatment of Vascular Ehler-Danlos Syndrome (VEDS) is underway, and the Company has achieved its objective to restart enrollment and to begin dosing new patients, reporting that a total of 19 patients are now in the trial as of the end of the third quarter. Zevra is continuing its efforts to expand its partnerships with patient advocacy groups, treatment centers of excellence, and medical key opinion leaders. Corporate Highlights and Upcoming Milestones In August, Zevra completed an underwritten public offering of 10,615,385 shares of its common stock at an offering price of $6.50 per share for total net proceeds of approximately $64.5 million, after deducting underwriting discounts and commissions and offering expenses. Cantor and William Blair were joint book-running managers for the offering. Citizens JMP and H.C. Wainwright & Co. were co-managers. The Company completed a thorough portfolio assessment and developed a strategic plan for 2025 that builds from its 2024 accomplishments and furthers Zevra’s transformation towards becoming a leading rare disease company with a five-year vision to create value for patients and shareholders by organizing our priorities on four key pillars: Commercial Excellence, Pipeline and Innovation, Talent and Culture, and Corporate Foundation. Now focused on late-stage clinical development and commercial opportunities, Zevra has discontinued its in-house drug discovery activities and is closing its laboratory facilities in Iowa and Virginia. Future early research and development activities will be outsourced. Q3 2024 Financial Highlights Revenue, Net: $3.7 million, comprised of $2.6 million in net reimbursements from the French expanded access program (EAP) for arimoclomol, $1.1 million of royalties and other reimbursements under the AZSTARYS® license agreement, and de minimis OLPRUVA revenue. Cost of Goods Sold: $2.3 million, which includes recognition of $2.0 million in inventory obsolescence reserve expense related to OLPRUVA inventory that is nearing expiration. R&D Expenses: $10.9 million, which represents a decrease of $1.4 million compared to Q3 2023 due to the completion of the KP1077 phase 2 trial. SG&A Expenses: $16.2 million for Q3 2024, compared to $5.8 million in Q3 2023, which reflects the commercial team fully in place and actively engaged in commercial launch activities and patient services initiatives. This includes non-cash stock compensation of $6.1 million during the quarter, of which $2.5 million was performance-based upon the approval of MIPLYFFA. Net Loss: ($33.2) million, or ($0.69) per basic and diluted share for Q3 2024, compared to a net loss of ($10.4) million, or ($0.30) per basic and diluted share in Q3 2023. Cash Position: Cash, cash equivalents and investments were $95.5 million as of Sept. 30, 2024. Cash Runway Forecast: Based on the Company’s current operating plan, available cash, cash equivalents and investments are expected to extend Zevra’s cash runway into 2027, subject to continuing compliance with debt covenants. Cash runway forecast includes anticipated revenue from MIPLYFFA sales, reimbursements from the French EAP for arimoclomol, royalties under the AZSTARYS license agreement, and continued investments into our development pipeline programs. It does not include potential proceeds from a PRV sale. Common and Fully Diluted Shares O/S: As of Sept. 30, 2024, total shares of common stock outstanding were 53,227,364, and fully diluted common shares were 67,698,898, which included 5,483,537 shares issuable upon exercise of warrants. Upcoming Events Guggenheim Securities Healthcare Innovation Conference, with fireside chat on Wednesday, Nov. 13, 2024, at 4:00 p.m. ET. Management will be available for one-on-one meetings with registered attendees. Conference Call Information - UPDATED 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 third quarter of 2024. The audio webcast will be accessible via the Investor Relations section of the Company’s website, http://investors.zevra.com/. An archive of the audio webcast will be available for ninety (90) days beginning at approximately 5:30 p.m. ET on Nov. 12, 2024. Additionally, interested participants and investors may access the conference call by dialing either: (785) 838-9345 (United States) -- updated dial-in +1 (203) 518-9814 (International)Conference ID: ZVRAQ324 About MIPLYFFA 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. MIPLYFFA was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation by the FDA for the treatment of NPC. MIPLYFFA was further granted Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. 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. Before prescribing MIPLYFFA, please read the full Prescribing Information, including Instructions for Use. 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) was approved for the treatment of certain UCDs in December 2022 and has recently been marketed under the brand name, OLPRUVA. 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 FDA at 1-800-FDA-1088. About KP1077 Serdexmethylphenidate (SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and is the sole active pharmaceutical ingredient (API) of KP1077. KP1077 has been granted Orphan Drug designation by the FDA, and by the European Commission, for the treatment of Idiopathic Hypersomnia, or IH. The U.S. Drug Enforcement Agency (DEA) has classified SDX as a Schedule IV controlled substance based on evidence suggesting SDX has a lower potential for abuse when compared to d-MPH, a Schedule II controlled substance. In addition, KP1077 has intellectual property protection through 2037. About Celiprolol Celiprolol is an investigational clinical candidate for the treatment of Vascular Ehlers-Danlos Syndrome (VEDS). Celiprolol has been granted Orphan Drug and Breakthrough Therapy designations by the FDA. Zevra recently restarted enrollment in the Phase 3 trial, known as the DiSCOVER trial being conducted under a Special Protocol Assessment (SPA) agreement with the 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 rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. Expanded access programs are made available by Zevra Therapeutics, Inc. and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy, as published on its website. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion. 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 promise and potential impact of our preclinical or clinical trial data; the initiation, timing and results of any clinical trials or readouts, the content, information used for, timing or results of any NDA submissions or resubmissions for any products or product candidates for any specific disease indication or at any dosage; the potential benefits of any of our products or product candidates for any specific disease or at any dosage; the impact of meetings or communications with the FDA or any advisory committee; statements or decisions by the FDA or any other entity for any products or product candidates; our ability to monetize any PRV; future research and development activities; our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company; the potential benefits of our debt facility; our financial position, including our cash balance; our corporate governance objectives; potential revenues from our arimoclomol expanded access program in France; the potential for royalty and milestone contributions, the presentation of data at conferences; and the timing of any of the foregoing. 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 Dec. 31, 2023, Zevra’s Quarterly Report on Form 10-Q for the three months ended Sept. 30, 2024, 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. Zevra Contact Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com Russo Partners Contact David Schull+1 (858) 717-2310david.schull@russopartnersllc.com ZEVRA THERAPEUTICS, INC. UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(in thousands, except share and per share amounts) Three months ended September 30, Nine months ended September 30, 2024 2023 2024 2023 Revenue, net $3,695 $2,895 $11,569 $14,541 Cost of product revenue (excluding $1,545 and $4,619 in intangible asset amortization for the three and nine months ended September 30, 2024, respectively shown separately below) 2,303 144 6,051 946 Intangible asset amortization 1,545 — 4,619 — Operating expenses: Research and development 10,945 12,297 33,743 28,385 Selling, general and administrative 16,208 5,818 38,743 19,657 Total operating expenses 27,153 18,115 72,486 48,042 Loss from operations (27,306) (15,364) (71,587) (34,447)Other (expense) income: Interest expense (2,312) (366) (5,157) (745)Fair value adjustment related to warrant and CVR liability (4,746) 3,678 4,660 4,253 Fair value adjustment related to investments 90 124 64 451 Interest and other income (expense), net 1,049 1,738 2,248 4,331 Total other (expense) income (5,919) 5,174 1,815 8,290 Loss before income taxes (33,225) (10,190) (69,772) (26,157)Income tax expense — (177) — — Net loss $(33,225) $(10,367) $(69,772) $(26,157) Basic and diluted net loss per share of common stock: Net loss $(0.69) $(0.30) $(1.59) $(0.76) Weighted average number of shares of common stock outstanding: Basic and diluted 47,808,817 34,724,614 43,843,851 34,364,075 ZEVRA THERAPEUTICS, INC. UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands, except share and par value amounts) September 30, December 31, 2024 2023 Assets Current assets: Cash and cash equivalents $54,039 $43,049 Securities at fair value, current 35,337 24,688 Accounts and other receivables 7,790 17,377 Prepaid expenses and other current assets 2,276 1,824 Total current assets 99,442 86,938 Inventories 8,756 9,841 Securities at fair value, long-term 6,105 — Property and equipment, net 607 736 Operating lease right-of-use assets 820 790 Goodwill 4,701 4,701 Intangible assets, net 70,608 69,227 Other long-term assets 512 94 Total assets $191,551 $172,327 Liabilities and stockholders' equity Current liabilities: Accounts payable and accrued expenses $24,464 $28,403 Line of credit payable — 37,700 Current portion of operating lease liabilities 540 543 Current portion of discount and rebate liabilities 8,547 4,550 Other current liabilities 930 2,524 Total current liabilities 34,481 73,720 Long-term debt 58,904 5,066 Warrant liability 13,902 16,100 Operating lease liabilities, less current portion 483 456 Discount and rebate liabilities, less current portion 8,490 7,663 Other long-term liabilities 5,521 7,458 Total liabilities 121,781 110,463 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 September 30, 2024, or December 31, 2023 — — Common stock, $0.0001 par value, 250,000,000 shares authorized, 54,803,056 shares issued and 53,227,364 shares outstanding as of September 30, 2024; 43,110,360 shares issued and 41,534,668 shares outstanding as of December 31, 2023 5 4 Additional paid-in capital 550,413 472,664 Treasury stock, at cost (10,983) (10,983)Accumulated deficit (469,550) (399,778)Accumulated other comprehensive income (loss) (115) (43)Total stockholders' equity 69,770 61,864 Total liabilities and stockholders' equity $191,551 $172,327