Acute hyperammonemia

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

New clinical efficacy and safety data for arimoclomol as a possible treatment for Niemann-Pick disease type C, including from long-term and real-world settings, demonstrate clinically meaningful reduction in disease progression Arimoclomol was well tolerated during the Open Label Extension trial and Early Access Program with no safety signals identified PK modeling studies showed that administration of OLPRUVA while fasting vs fed results in higher drug exposure which may allow for lower effective dosages when taken without food CELEBRATION, Fla., Sept. 06, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced the presentation of five posters at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium. Four posters focused on data from multiple studies showing the efficacy and safety of arimoclomol as a treatment for people living with Niemann-Pick disease type C (NPC), and one poster highlighted data from pharmacokinetic modeling studies of OLPRUVA®, a therapy for the long-term management of certain adult and pediatric patients with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinic acid synthetase (AS). “The data collected during the Phase 2/3 study of arimoclomol, including long-term data from Open Label Extension (OLE) and Early Access Program (EAP) participants, add to the large body of evidence that demonstrates arimoclomol’s clinical efficacy and safety as a treatment for people living with NPC,” said Adrian Quartel, M.D., FFPM, Chief Medical Officer of Zevra. “Additionally, we presented PK modeling data from OLPRUVA in both adult and pediatric virtual patients, showing an increase in drug exposure under fasting conditions.” Presentation Details The data for arimoclomol presented at SSIEM is summarized below: Poster Number: 21260 Title:Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for Treatment of Niemann Pick Disease Type C – presenting an improved 4-Domain NPC Clinical Severity Scale Authors:Marc Patterson, Sven Guenther and Christine i Dali Summary:The treatment effect of arimoclomol was evaluated in a 12-month, double-blind, placebo-controlled clinical trial (NCT02612129) using the original 5-domain Niemann pick type C clinical severity scale (5DNPCCSS) and the modified 4-domain Niemann Pick type C clinical severity scale (4DNPCCSS). A statistically significant treatment effect was shown using the modified 4DNPCCSS and the prespecified 5DNPCCSS primary endpoint in the 12-month clinical trial, representing a clinically meaningful reduction in disease progression with arimoclomol treatment compared to placebo. Poster Number: 21271 Title:Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann Pick Type C – Data from 48 Months Open Label Trial Authors:Marc Patterson, Eugene Mengel, Sven Guenther and Christine i Dali Summary:The long-term safety and efficacy of arimoclomol in the 12-month double-blind, and 48-month open-label extension (OLE) portion of the clinical trial (NCT02612129) were presented using the 4-Domain Niemann Pick Type C Clinical Severity scale (4DNPCCSS) which evaluates ambulation, speech, swallowing and fine motor skills. For those patients transitioning from placebo to arimoclomol at the start of the open-label extension period, the mean annual rate of disease progression reduced from an annual rate of change of 1.9 points during the double-blind phase, to a rate of 0.3 in the first 12 months of treatment, remained numerically smaller for the rest of the trial, and was comparable between the double-blind phase of the trial and the open-label extension phase of the trial. Additionally, arimoclomol was well tolerated with no new safety signals observed. Poster Number: PO-212 Title:Arimoclomol for the Treatment of NPC in a Real-World Setting: Long-term Outcomes from an Expanded Access Program in the USA Authors:Walla Al-Hertani, Elizabeth M. Berry-Kravis, Raymond Wang, Marc Patterson, Can Ficicioglu, Loren Pena, Kristina Julich, Damara Ortiz, Paula Schleifer, Caroline Hastings, Paul Hillman, Ronan O'Reilly, Christine Dali and Daniel Gallo Summary:The long-term safety and efficacy of arimoclomol in a real-world setting were evaluated in a total of 56 adult and pediatric patients in the U.S. arimoclomol expanded access program (EAP) trial (NCT04316637). Data presented included over 3 years of U.S. EAP clinical outcomes and demonstrated that adult and pediatric patients treated with arimoclomol, including those with and without miglustat as a component of their routine clinical care, experienced relatively stable disease as measured by the 5DNPCCSS and 4DNPCCSS and show that arimoclomol was well tolerated. Poster Number: 20950 Title:Arimoclomol safety profile in the treatment of NPC in a Real-World setting: Long-term data from an Expanded Access program in the USA Authors:Can Ficicioglu, Elizabeth M. Berry-Kravis, Walla Al-Hertani, Raymond Wang, Marc Patterson, Loren Pena, Kristina Julich, Damara Ortiz, Paula Schleifer, Paul Hillman, Caroline Hastings, Ronan O'Reilly, Christine Dali and Daniel Gallo Summary:Safety data from 94 adult and pediatric participants in the arimoclomol US EAP were presented. Safety outcomes reported from exposure periods spanning as much as 46 months of treatment, demonstrated a safety profile consistent with the published clinical trial experience of arimoclomol in NPC. The data for OLPRUVA presented at SSIEM is summarized below: Poster Number: PO-609 Title:Modeling the Pharmacokinetics of Phenylbutyrate in Fed and Fasted States Authors:Steiner, Rebecca Baillie, Tongli Zhang, Christina Friedrich, Meredith Hart, Mike Reed Summary:Pharmacokinetic modeling evaluating whether sodium phenylbutyrate (NaPBA) could be safely and effectively administered while fasting showed greater absorption and bioavailability with increased drug exposure in fasted administration of NaPBA compared to fed administration of NaPBA and glycerol phenylbutyrate (GPB) in both adult and child virtual patients, which is predicted to increase efficacy in proportion to increased drug exposure, theoretically allowing for a 30% dose decrease compared to fed conditions. About the SSIEM 2024 Annual Symposium The Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium took place September 3-6, 2024, in Porto, Portugal. The annual symposium is intended to foster the study of inherited metabolic disorders and promote the exchange of ideas between professionals in different disciplines who are researching inborn errors of metabolism (IEM). About Niemann-Pick Disease Type C (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 tissue areas, including brain tissue. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease progression is irreversible and can be fatal within months or take years to be diagnosed and advance in severity. About Arimoclomol Arimoclomol, Zevra’s orally-delivered, investigational drug product candidate for the treatment of NPC, has been granted Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation by the FDA, and Orphan Medicinal Product designation for the treatment of NPC by the European Medicines Agency (EMA). The FDA has accepted the resubmission of the NDA for arimoclomol and has set a user fee action date (PDUFA date) of September 21, 2024. About Urea Cycle Disorders UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments if ammonia levels are not controlled. Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels. 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 Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a 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 and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy as published on its website at www.zevra.com. 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 (formerly Twitter) 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 zpotential 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 arimoclomol or any other 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; our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company; potential revenues from our arimoclomol expanded access program; 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 December 31, 2023, Zevra’s Quarterly Report on Form 10-Q for the three months ended June 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 Contacts Adanna G. Alexander, Ph.D.+1 (646) 942-5603adanna.alexander@russopartnersllc.com Ignacio Guerrero-Ros, Ph.D.+1 (646) 942-5604ignacio.guerrero-ros@russopartnersllc.com

FDA advisory committee voted favorably that the data support arimoclomol as effective treatment for patients with NPC; PDUFA date of September 21, 2024 Pro forma June 30, 2024, cash, cash equivalents, investments and net proceeds from underwritten public offering total $113.8 million following the closing on August 12, 2024 Conference call scheduled for today, August 13, 2024, at 4:30 p.m. ET CELEBRATION, Fla., Aug. 13, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today provided corporate updates and reported its financial results for the second quarter ended June 30, 2024. “During the second quarter, we made steady progress executing on our strategic objectives,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra. “We are encouraged by favorable vote from the FDA’s Genetic Metabolic Diseases Advisory Committee, that data presented support that arimoclomol is effective in the treatment in patients with Niemann Pick disease type C. While the vote is non-binding, we believe it is an important factor as the FDA completes its consideration for approval. Additionally, we continue the launch of OLPRUVA with a keen focus on driving patient awareness and intend to use the same commercial team to launch arimoclomol, if approved. Additionally, we expect to meet with the FDA to discuss the design of a pivotal Phase 3 trial to study KP1077 in idiopathic hypersomnia at the end of third quarter.” “We continue to be prudent in our capital allocation as we focus on creating long-term value for stockholders,” said R. LaDuane Clifton, Zevra’s Chief Financial Officer and Treasurer. “Our recent underwritten offering provided net proceeds to the Company of $64.5 million, along with attracting a cadre of institutional investors well known as long term supporters of innovation and building momentum as we lean into our near-term catalysts while also extending our cash runway.” Q2 2024 Corporate Highlights: Arimoclomol On August 2, 2024, the U.S. Food and Drug Administration’s (FDA) newly formed Genetic Metabolic Diseases Advisory Committee (the “GeMDAC”) convened for the first time and discussed the benefits and risks of arimoclomol.The GeMDAC reviewed comments received from independent experts, NPC patients, and patient advocacy group representatives, and voted favorably that arimoclomol is effective in the treatment of NPC.On Friday, August 9, 2024, the Company received the first round of labeling comments, and is working closely with the FDA. OLPRUVA On June 18, 2024, announced transition of specialty pharmacy partner to Orsini.Increased OLPRUVA reimbursement coverage to 75% of covered lives and improved preferred status on formulary plans. Added nine (9) new patient enrollments during Q2 2024, which is defined as a prescription for a patient on the Quick Start program or receiving a paid dispense of OLPRUVA. KP1077 On June 3, 2024, presented positive topline data from its Phase 2 study in patients with idiopathic hypersomnia (IH) at the SLEEP 2024 Annual Meeting. The Company submitted a briefing book to the FDA for an end-of-Phase 2 meeting to be held at the end of Q3 2024. Celiprolol Restarted recruitment for the Phase 3 DiSCOVER trial of celiprolol in patients with Vascular Ehler-Danlos Syndrome (VEDS). Corporate On April 5, 2024, the Company entered into a $100 million credit facility with leading biotech investors, Perceptive Advisors and Healthcare Royalty Partners.On June 25, 2024, the Company announced the appointment of Rahsaan Thompson as Chief Legal Officer, Secretary and Compliance Officer, and Alison Peters as Chief People Officer, bringing expertise that will support the next phase of the Company’s growth. Overview of Q2 2024 Financial Results: Net revenue for Q2 2024 was $4.4 million, compared to net revenue of $8.5 million in Q2 2023. The components of revenue during the second quarter included $3.1 million in net reimbursements from the French EAP for arimoclomol, $1.3 million of royalties and other reimbursements under the AZSTARYS® License Agreement, and de minimis OLPRUVA revenue via sales to the new specialty pharmacy, Orsini, were offset by returns from the prior pharmacy in Q2 2024. In addition, cost of goods sold was inflated during the quarter due to recognition of a $3.2 million obsolescence reserve against OLPRUVA inventory which is nearing expiration. This excess inventory was ordered prior to our acquisition of Acer, and the delayed launch impacted the rate of usage, leading to the need for this reserve to be recognized in the quarter. Research and development (R&D) expenses were $10.5 million for Q2 2024, compared to $7.4 million in Q2 2023. The increase in R&D expenses was primarily driven by an increase in spending for the KP1077 Phase 2 clinical trial and an increase in personnel-related costs, partially offset by a decrease in third-party costs related to arimoclomol. Selling, general and administrative (SG&A) expenses were $12.6 million for Q2 2024, compared to $6.6 million in Q2 2023. The period-over-period increase reflects the commercial team in place for the entire quarter and actively engaged in activities to build awareness and provide patient services related to OLPRUVA, leading to an increase in personnel costs due to the additional headcount and an increase in other expenses related primarily to the launch of OLPRUVA. Net loss for Q2 2024 was ($19.9) million, or ($0.48) per basic and diluted share, compared to a net loss of ($2.6) million, or ($0.08) per basic and diluted share for Q2 2023. As of June 30, 2024, total cash, cash equivalents, and investments were $49.3 million, a decrease of $3.4 million compared to $52.7 million as of March 31, 2024. The decrease was driven, in part, by increased third-party R&D costs related to the KP1077 clinical development program and increased SG&A expenses during the period as the Company invested in its commercial infrastructure. As of June 30, 2024, total shares of common stock outstanding were 41,991,464, and fully diluted common shares were 57,324,496, which included 5,483,537 shares issuable upon exercise of warrants. On April 5, 2024, the Company announced the refinancing of its existing debt with up to $100 million in committed capital, which strengthened its balance sheet, simplified its debt structure, and provided non-dilutive capital flexibility. The refinancing was led by Perceptive Advisors and HealthCare Royalty Partners, premier biotech investors. From the initial draw of $60 million at closing, the Company refinanced its existing debt of $43 million and added an incremental $14 million in net cash proceeds to the cash balance after fees and discounts. A second tranche of up to $20 million is available at the Company’s discretion until October 5, 2025, and a third tranche of up to $20 million will become available upon approval of arimoclomol, in each case subject to certain terms and conditions. On August 12, 2024, the Company completed an underwritten public offering, further strengthening its balance sheet. The underwriters fully exercised their overallotment option, resulting in the issuance of approximately 10.6 million shares at a price of $6.50 per share, raising net proceeds of $64.5 million after fees and expenses. Combining the net proceeds from this offering with our existing resources, pro forma June 30, 2024 total cash, cash equivalents and investments was $113.8 million. Following the closing of the underwritten public offering on August 12, 2024, pro forma June 30, 2024, common shares and fully diluted shares outstanding were 52,606,849 and 67,939,881, respectively. No warrants were issued as part of the underwritten public offering. Based on our current operating plan, available cash, cash equivalents, and investments, including proceeds from the underwritten offering closed in August, are expected to extend our cash runway into the first quarter of 2027, subject to continuing compliance with our debt covenants. Cash runway forecast includes: revenue from the expected sales of OLPRUVA, ongoing reimbursements from the French EAP for arimoclomol, ongoing royalties under the AZSTARYS license agreement, and investments into the incremental commercial activities needed to support the launch of arimoclomol, if approved. Cash runway forecast does not include commercial revenue from arimoclomol which could follow a potential FDA approval or the potential sale of the Priority Review Voucher which would be received upon approval. Conference Call Information Zevra will host a conference call and audio webcast today at 4:30 p.m. ET, to discuss its corporate and financial results for Q2 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 August 13, 2024. Additionally, interested participants and investors may access the conference call by dialing either: (800) 225-9448 (United States)+1 (203) 518- 9708 (International)Conference ID: ZVRAQ224 About Urea Cycle Disorders UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments if ammonia levels are not controlled.i Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels. 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 Niemann-Pick Disease Type C (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 tissue areas, including brain tissue. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease progression is irreversible and can be fatal within months or take years to be diagnosed and advance in severity. About Arimoclomol Arimoclomol, Zevra’s orally-delivered, investigational drug product candidate for the treatment of NPC, has been granted Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation by the FDA, and Orphan Medicinal Product designation for the treatment of NPC by the European Medicines Agency (EMA). The FDA has accepted the resubmission of the NDA for arimoclomol and has set a user fee action date (PDUFA date) of September 21, 2024. About Idiopathic Hypersomnia (IH) Idiopathic hypersomnia (IH) is a rare sleep disorder characterized by excessive daytime sleepiness (EDS). Patients with IH experience daytime lapses into sleep, or an irrepressible need to sleep that persists even with adequate or prolonged nighttime sleep. Additionally, those with IH have extreme difficulty waking, otherwise known as sleep inertia, severe brain fog, and often fall asleep unintentionally or at inappropriate times. These symptoms of IH often lead to further, even more debilitating problems such as memory lapses, difficulty maintaining focus, and depression. It is estimated, based on claims data, that approximately 37,000 patients in the United States are currently diagnosed with IH, although the total patient population may be much larger due to some patients who have not yet been diagnosed, have been misdiagnosed, or are not currently seeking treatment. About KP1077 KP1077 (serdexmethylphenidate, or SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and its sole active pharmaceutical ingredient (API). KP1077 has been granted Orphan Drug Designation by the FDA, and by the European Commission, for the treatment of IH. The U.S. Drug Enforcement Agency (DEA) has classified SDX, the sole API in KP1077, 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 though 2037 and potentially beyond. 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 Vascular Ehlers-Danlos Syndrome Vascular Ehlers-Danlos syndrome is a rare genetic cardiovascular disorder which impairs collagen 3 rich connective tissue and leads to vascular and hollow organ ruptures. It is estimated that approximately 7,500 patients in the United States are currently diagnosed patients with VEDS. There remains an unmet need with no approved treatment in the United States. Celiprolol is currently the standard of care in Europe. About Zevra Therapeutics Zevra Therapeutics is a 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. 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 arimoclomol or any other 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; decisions by the FDA or any other entity for arimoclomol or any other product candidates; 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 our financial position, including our cash balance: our corporate governance objectives; potential revenues from our arimoclomol expanded access program; 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 December 31, 2023, Zevra’s Quarterly Report on Form 10-Q for the three months ended June 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. ________________________i Ah Mew N, et al. Urea cycle disorders overview [updated June 22, 2017]. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews® [Internet]. University of Washington; 1993-2022. Accessed March 20, 2022. Zevra Contact Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com Russo Partners Contacts Adanna G. Alexander, Ph.D.+1 (646) 942-5603adanna.alexander@russopartnersllc.com ZEVRA THERAPEUTICS, INC. UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except share and per share amounts) Three months endedJune 30, Six months endedJune 30, 2024 2023 2024 2023Revenue, net $4,449 $8,470 $7,874 $11,646 Cost of product revenue (excluding $1,546 and $3,074 in intangible asset amortization for the three and six months ended June 30, 2024, respectively shown separately below) 3,573 677 3,748 802 Intangible asset amortization 1,546 — 3,074 — Operating expenses: Research and development 10,521 7,433 22,798 16,088 Selling, general and administrative 12,604 6,612 22,535 13,839 Total operating expenses 23,125 14,045 45,333 29,927 Loss from operations (23,795) (6,252) (44,281) (19,083)Other income (expense): Interest expense (2,110) (197) (2,845) (379)Fair value adjustment related to warrant and CVR liability 5,779 2,118 9,406 575 Fair value adjustment related to investments 1 131 (26) 327 Interest and other income (expense), net 270 1,553 1,199 2,593 Total other income 3,940 3,605 7,734 3,116 Loss before income taxes (19,855) (2,647) (36,547) (15,967)Income tax benefit (70) 74 — 177 Net loss $(19,925) $(2,573) $(36,547) $(15,790) Basic and diluted net loss per share of common stock: Net loss $(0.48) $(0.08) $(0.87) $(0.46)Weighted average number of shares of common stock outstanding: Basic and diluted 41,899,087 33,898,233 41,839,582 34,180,818 ZEVRA THERAPEUTICS, INC. UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands, except share and par value amounts) June 30, December 31, 2024 2023Assets Current assets: Cash and cash equivalents $39,260 $43,049 Securities at fair value 9,998 24,688 Accounts and other receivables 8,947 17,377 Prepaid expenses and other current assets 2,686 1,824 Total current assets 60,891 86,938 Inventories 10,198 9,841 Property and equipment, net 678 736 Operating lease right-of-use assets 911 790 Goodwill 4,701 4,701 Intangible assets, net 66,154 69,227 Other long-term assets 875 94 Total assets $144,408 $172,327 Liabilities and stockholders' equity Current liabilities: Accounts payable and accrued expenses $20,452 $28,403 Line of credit payable — 37,700 Current portion of operating lease liabilities 596 543 Current portion of discount and rebate liabilities 6,768 4,550 Other current liabilities 2,633 2,524 Total current liabilities 30,449 73,720 Long-term debt 58,328 5,066 Warrant liability 7,856 16,100 Operating lease liabilities, less current portion 544 456 Discount and rebate liabilities, less current portion 8,115 7,663 Other long-term liabilities 6,638 7,458 Total liabilities 111,930 110,463 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 June 30, 2024, or December 31, 2023 — — Common stock, $0.0001 par value, 250,000,000 shares authorized, 43,567,156 shares issued and 41,991,464 shares outstanding as of June 30, 2024; 43,110,360 shares issued and 41,534,668 shares outstanding as of December 31, 2023 4 4 Additional paid-in capital 479,361 472,664 Treasury stock, at cost (10,983) (10,983)Accumulated deficit (436,325) (399,778)Accumulated other comprehensive income (loss) 421 (43)Total stockholders' equity 32,478 61,864 Total liabilities and stockholders' equity $144,408 $172,327