ZEVRA THERAPEUTICS, INC.

FORM 10-K

SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS

This Annual Report on Form 10-K, including the section entitled “Management’s Discussion and Analysis of Financial Condition and Results of Operations,” contains forward-looking statements regarding future events and our future results that are subject to the safe harbors created under the Securities Act of 1933, as amended (the “Securities Act”), and the Securities Exchange Act of 1934, as amended (the “Exchange Act”). Forward-looking statements relate to future events or our future financial performance. We generally identify forward-looking statements by terminology such as “may,” “will,” “would,” “should,” “expects,” “plans,” “anticipates,” “could,” “intends,” “target,” “projects,” “contemplates,” “believes,” “estimates,” “predicts,” “assume,” “potential,” “continue” or other similar words or the negative of these terms. We have based these forward-looking statements largely on our current expectations about future events and financial trends that we believe may affect our business, financial condition and results of operations. The outcomes of the events described in these forward-looking statements are subject to risks, uncertainties and other factors described in “Risk Factors” and elsewhere in this report. Accordingly, you should not place undue reliance upon these forward-looking statements. We cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur, and the timing of events and circumstances and actual results could differ materially from those anticipated in the forward-looking statements. Forward-looking statements contained in this report include, but are not limited to, statements about:

•our ability to commercialize and the timing of commercializing our products and product candidates, if approved;

•the potential therapeutic benefits and effectiveness of our products and product candidates;

•the progress of, timing of and expected amount of expenses associated with our commercialization, research, and development activities;

•the size and characteristics of the markets that may be addressed by our products and product candidates;

•the expected timing of clinical trials for our product candidates and the availability of data and results of those trials;

•the progress of, outcome of, and timing of any regulatory approval for any of our product candidates;

•our expectations regarding federal, state and foreign tax, legal and regulatory requirements;

•our intention to seek to establish, and the potential benefits to us from, any strategic collaborations or partnerships for the development or sale of our products and product candidates, if approved;

•our expectations as to future financial performance, expense levels and liquidity sources;

•the sufficiency of our cash resources to fund our operating expenses and capital investment requirements for any period;

•our ability to raise additional funds if needed on commercially reasonable terms, or at all, in order to support our continued operations;

•senior leadership and board member transitions and refreshments; and

•other factors discussed elsewhere in this report.

The forward-looking statements made in this report relate only to events as of the date on which the statements are made. We have included or made reference to important factors in the cautionary statements included in this report, particularly in the section entitled “Risk Factors” that we believe could cause actual results or events to differ materially from the forward-looking statements that we make. Our forward-looking statements do not reflect the potential impact of any future developments, including acquisitions, mergers, dispositions, joint ventures or investments we may make. Except as required by law, we do not assume any intent to update any forward-looking statements after the date on which the statement is made, whether as a result of new information, future events or circumstances or otherwise.

RISK FACTORS SUMMARY

The risk factors summarized below could materially harm our business, operating results, and/or financial condition, impair our future prospects, and/or cause the price of our common stock to decline. These risks are discussed more fully in the section titled“Risk Factors”. Material risks that may affect our business, financial condition, results of operations, and trading price of our common stock include the following:

Risks Related to the Commercialization of Our Approved Products and Product Candidates

•If we are unable to establish effective sales, marketing and distribution capabilities for our products, or if our products fail to achieve expected market acceptance, we may not be successful in commercializing such products.

•The target patient population for our rare disease products is small, so we must achieve significant market share and obtain relatively high per-patient prices to be successful.

•We may be subject to enforcement action if we engage in improper marketing or promotion of our products.

•Product liability claims could cause us to incur substantial liabilities and adversely affect commercialization of our products.

•Significant political, trade or regulatory developments, such as increased tariffs or other measures that can restrict international trade can negatively effect development and commercialization of our products and product candidates.

•We are subject to a number of risks associated with commercializing our products internationally.

•Arimoclomol is currently available to Niemann-Pick disease type C patients in select foreign jurisdictions through a global expanded access program (“EAP”), and if the EAP is terminated prior to regulatory approval of arimoclomol and commercialization in any such foreign jurisdiction, our business would be harmed.

Risks Related to Our Business and Our Financial Position

•We are highly dependent on MIPLYFFA (arimoclomol) for substantially all of our revenues, and any loss of revenue from this product could materially harm our business.

•We have had recurring negative net operating cash flows throughout our operating history, and we cannot guarantee or predict when we may begin to consistently generate positive net cash flows from operations, or if at all.

Risks Related to Our Dependence on Third Parties

•We rely on a limited number of suppliers, in some cases sole-source suppliers, and the inability of such sole-source suppliers to meet our supply requirements would materially harm our business.

•The facilities used by our third-party contract manufacturers are subject to regulatory requirements and inspections, and any failure by such third-party contract manufacturers to comply with regulatory requirements could materially harm our business.

•We rely on and expect to continue to rely on third parties to conduct our clinical trials for our product candidates, and those third parties may not perform satisfactorily.

•We have entered into a collaboration with Commave, to develop, manufacture and commercialize AZSTARYS worldwide, a party with whom we are currently in dispute, and if this collaboration is not successful, we may not be able to capitalize on the market potential of AZSTARYS.

Risks Related to Our Intellectual Property

•If we are unable to obtain and maintain adequate intellectual property protection for our technology and products, our competitors could develop and commercialize technology and products similar or identical to ours, and our ability to successfully commercialize our products would be impaired.

•We may become involved in lawsuits to protect or enforce our patents or other intellectual property against infringing third parties, which could be expensive, time consuming and unsuccessful.

•Third parties may initiate legal proceedings against us alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could significantly harm our business.

Risks Related to Regulatory Approval of Our Product Candidates and Other Legal Compliance Matters

•If we are not able to obtain required regulatory approvals for any of our product candidates, we will not be able to commercialize them and our ability to generate revenue or profits could be limited.

•If we experience delays or difficulties in the enrollment of subjects in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.

•Our approved products are, and any of our product candidates for which we obtain marketing approval will be, subject to significant post-marketing regulatory requirements and oversight.

•Current and future healthcare reform legislation or regulation in the United States and foreign jurisdictions may increase the difficulty and cost for us to obtain marketing approval of our product candidates and increase the cost to commercialize our approved products.

•We may not be able to obtain or maintain the benefits associated with orphan drug designation, including the potential for market exclusivity.

•Our relationships with healthcare professionals, principal investigators, consultants, customers and third-party payors in the United States and elsewhere may be subject to applicable anti-kickback, fraud and abuse, false claims, physician payment transparency, and other healthcare laws and regulations, which could expose us to penalties.

•If we fail to comply with our reporting and payment obligations under the Medicaid Drug Rebate Program or other governmental pricing programs in which we participate, we could be subject to additional reimbursement requirements, penalties, sanctions and fines.

Risks Related to Cybersecurity and Data Privacy

•Cybersecurity breaches, loss of data and other disruptions could compromise sensitive information related to our business or prevent us from accessing critical information and expose us to liability, which could adversely affect our business and our reputation.

•We are currently subject to, and may in the future become subject to additional, U.S. federal and state and international laws and regulations imposing obligations on how we collect, store and process personal information.

Risks Related to Ownership of Our Common Stock

•The trading price of the shares of our common stock is likely to be volatile, and purchasers of our common stock could incur substantial losses.

•Anti-takeover provisions in our certificate of incorporation and bylaws, as well as provisions of Delaware law and the terms of some or our contracts, might discourage, delay or prevent a change in control of the company or changes in our board of directors or management and, therefore, depress the price of our common stock.

•We could be negatively affected as a result of the actions of activist stockholders, which could be disruptive and costly and may conflict with or disrupt the strategic direction of our business.

NOTE REGARDING COMPANY REFERENCE

Unless the context otherwise requires, we use the terms “Zevra,” “Company,” “we,” “us” and “our” in this Annual Report on Form 10-K to refer to Zevra Therapeutics, Inc. and its subsidiaries. We have proprietary rights to a number of trademarks and service marks used in this Annual Report on Form 10-K that are important to our business, including MIPLYFFA® and its related logo, OLPRUVA® and its related logo, LAT®, and the Zevra companies' logos. In addition, Zevra Therapeutics® and Zevra®are both registered trademarks of the Company. All other trademarks, trade names and service marks appearing in this Annual Report on Form 10-K are the property of their respective owners. Solely for convenience, the trademarks and trade names in this Annual Report on Form 10-K are referred to without the® and ™ symbols, but such references should not be construed as any indicator that their respective owners will not assert, to the fullest extent under applicable law, their rights thereto.

NOTE REGARDING MARKET AND INDUSTRY DATA

This Annual Report on Form 10-K contains statistical data, estimates and forecasts that are based on independent industry publications or other publicly available information, as well as other information based on our internal sources. While we believe the industry and market data included in this Annual Report on Form 10-K is reliable and is based on reasonable assumptions, this data involves many assumptions and limitations, and you are cautioned not to give undue weight to these estimates. We have not independently verified the accuracy or completeness of the data contained in these industry publications and other publicly available information. The industry in which we operate is subject to a high degree of uncertainty and risk due to a variety of factors, including those described in the section titled “Risk Factors” and “Special Note Regarding Forward-Looking Statements” included in this Annual Report on Form 10-K.