Zevra Therapeutics (ZVRA) risk factors, FY2025 10-K

What changed since the prior 10-K

New

• NewRisks Related to the Commercialization of Our Approved Products and Product Candidates

  Failure to obtain or maintain coverage and adequate reimbursement for our products could limit our ability to market those products and decrease our ability to generate revenue

  Insurance coverage and reimbursement limitations from third-party payors could reduce product sales and revenue.

• NewRisks Related to the Commercialization of Our Approved Products and Product Candidates

  Significant political, trade, regulatory developments, and other circumstances beyond our control could delay, prevent or impair our development or commercialization efforts

  Geopolitical disputes, international trade policies, and tariffs could disrupt our supply chains and commercialization efforts.

• NewRisks Related to the Commercialization of Our Approved Products and Product Candidates

  Any side effects or adverse events caused by approved products following regulatory approval, could result in a number of potentially significant negative consequences

  Undesirable side effects discovered post-approval could lead to regulatory restrictions, warning labels, fines, or product withdrawal.

• NewRisks Related to Our Business and Our Financial Position

  We are highly dependent on one product for substantially all of our revenues, and any loss of revenue from this product could materially harm our business

  We derived 82% of our 2025 revenues from MIPLYFFA and any loss of sales for this rare disease drug would severely harm us.

• NewRisks Related to Our Business and Our Financial Position

  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

  We have an accumulated deficit of $422.1 million and a history of negative cash flows despite a 2025 net income boost from selling a PRV.

• NewRisks Related to Our Business and Our Financial Position

  We recognized an impairment charge related to intangible assets. If our remaining assets become impaired in the future, we would incur additional impairment charges, which would negatively affect our operating results

  We recorded a $58.7 million impairment charge on intangible assets in mid-2025 and risk further impairment charges.

• NewRisks Related to Our Dependence on Third Parties

  We rely on a limited number of suppliers, in some cases sole-source suppliers

  We rely on sole-source third-party suppliers with no redundant manufacturing arrangements for our approved products and drug substances.

• NewRisks Related to Our Dependence on Third Parties

  The facilities used by our third-party contract manufacturers to manufacture our approved products and any of our product candidates are subject to review by the FDA pursuant to inspections, and such inspections could result in findings that lead to failure to obtain FDA approval of such marketing applications

  FDA inspections of our third-party contract manufacturing facilities could result in negative findings that block product approvals.

• NewRisks Related to Our Dependence on Third Parties

  We rely on a limited number of third‑party distribution partners to commercialize MIPLYFFA, and the loss or inadequate performance of any of these partners could materially and adversely affect our ability to supply MIPLYFFA in key territories

  We rely on a single specialty pharmacy in the U.S. and single distributors in other territories to commercialize MIPLYFFA.

• NewRisks Related to Our Dependence on Third Parties

  We have entered into a collaboration with Commave, to develop, manufacture and commercialize AZSTARYS worldwide. If this collaboration is not successful, we may not be able to capitalize on the market potential of AZSTARYS

  Our collaboration with Commave and Corium for AZSTARYS gives us limited control and they may terminate the agreement.

• NewRisks Related to Our Dependence on Third Parties

  We may seek to establish collaborations for certain product candidates, which may not be available on acceptable terms, or at all

  We may fail to establish or negotiate new pharmaceutical collaborations on acceptable terms or in a timely manner.

• NewRisks Related to Our Intellectual Property

  Intellectual property litigation could cause us to spend substantial resources and distract our personnel from their normal responsibilities

  Intellectual property litigation could drain our financial resources and distract management from normal operations.

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

  Our approved products are, and if marketing approval of any of our product candidates is granted, such product candidates may be, subject to limitations on the indicated uses for which the product may be marketed

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

  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

• NewRisks 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

• NewRisks Related to Cybersecurity and Data Privacy

  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

Dropped

• Dropped

  Soon after approval of MIPLYFFA, the FDA approved a second therapy for NPC, AQNEURSA, which is approved for symptomatic treatment of ataxia in NPC patients, and is marketed by IntraBio, Inc. ("IntraBio")

  FDA approval of competing NPC therapy AQNEURSA and commercial launch details for MIPLYFFA and OLPRUVA.

• Dropped

  Extensive clinical experience with favorable safety data. No significant safety findings have been reported with more than 600 patients treated in various clinical trials conducted for multiple indications and through our EAPs

  Clinical safety data, orphan drug designation, and the PRV Transfer Agreement.

• Dropped

  OLPRUVA is differentiated from currently available forms of phenylbutyrate. OLPRUVA is formulated to improve palatability while providing patients with a portable and discrete pre-measured dose

  OLPRUVA product differentiation and the acquisition of celiprolol for VEDS.

• Dropped

  Unique pharmacological profile. Mechanism of action in VEDS patients is thought to be through vascular dilatation and smooth muscle relaxation, the effect of which is to reduce the mechanical stress on collagen fibers in the arterial wall, and thereby potentially less incidence of vascular ruptures

  Celiprolol pharmacological profile, clinical trial data, and regulatory status.

• Dropped

  Most AEs occurred during the titration period, were mild, and did not lead to early discontinuation

  KP1077 titration period adverse events and clinical improvements in EDS.

• Dropped

  Mean total ESS scores decreased by approximately 9 points after 5 weeks of OL treatment

  Mean total ESS score decreases and other specific clinical trial score metrics for KP1077.

• Dropped

  No drug-to-drug interactions observed to date. We have not observed drug-to-drug interactions in clinical drug-drug interaction studies

  Lack of drug-to-drug interactions and Schedule IV controlled substance status for SDX.

• Dropped

  AZSTARYS (Partnered product)

  Partnership details for AZSTARYS and d-MPH prodrug composition.

• Dropped

  Aquestive Termination Agreement

  Royalty obligations under the March 2012 Aquestive Termination Agreement.

• Dropped

  Relief Exclusive License Agreement

  Exclusive license agreement with Relief Therapeutics for OLPRUVA in Europe and other regions.

• Dropped

  License Agreements Related to MIPLYFFA (arimoclomol)

  Prior license agreements related to MIPLYFFA with XOMA, KU, and UCLB.

• Dropped

  Our Intellectual Property

  Intellectual property strategy protecting our LAT platform technology.

• Dropped

  OLPRUVA (sodium phenylbutyrate)

• Dropped

  We also expect to benefit from potential commercial exclusivity afforded to the first drug approved after obtaining orphan drug designation for the treatment of MSUD. Orphan drug designation for OLPRUVA for the treatment of MSUD was granted by the FDA in August 2014

• Dropped

  AZSTARYS and Serdexmethylphenidate (SDX)

• Dropped

  In September 2024, the FDA approved the NDA for MIPLYFFA™ (arimoclomol), an orally-delivered treatment for NPC. MIPLYFFA has also been granted orphan designation for the treatment of NPC by the European Commission

• Dropped

  Third-Party Payor Coverage and Reimbursement

• Dropped

  In general, for purposes of NDA approval, human clinical trials are typically conducted in three sequential phases that may overlap

• Dropped

  NDA Submission and Review by the FDA

• Dropped

  Advertising and Promotion

• Dropped

  Post-Approval Requirements

• Dropped

  Orphan Drug Designation and Exclusivity

• Dropped

  Rare Pediatric Disease Priority Review Voucher Program

• Dropped

  abuse of the drug may lead to severe psychological or physical dependence

• Dropped

  Other Healthcare Regulatory Frameworks

• Dropped

  Data Privacy and Security Laws

• Dropped

  Healthcare Reform Measures

• Dropped

  The Foreign Corrupt Practices Act

• Dropped

  Similarly to the United States, the various phases of non-clinical and clinical research in the EU are subject to significant regulatory controls

• Dropped

  Data and Marketing Exclusivity

• Dropped

  Orphan Medicinal Products

• Dropped

  The aforementioned EU rules are generally applicable in the European Economic Area (“EEA”) which consists of the 27 EU member states plus Norway, Liechtenstein and Iceland

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  Sales of a substantial number of shares of our common stock in the public market could cause the market price of our common stock to drop significantly, even if our business is doing well

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  We may be subject to securities litigation, class action and derivative lawsuits, which could result in substantial costs and could divert management attention away from other business concerns

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  If equity research analysts do not publish research or reports, or publish unfavorable research or reports, about us, our business or our market, our stock price and trading volume could decline

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  Adverse developments affecting the financial services industry, including events or concerns involving liquidity, defaults, or non-performance by financial institutions, could adversely affect our business, financial conditions or results of operations

• DroppedRisks Related to the Commercialization of Our Approved Products and Product Candidates

  costs associated with maintaining compliance with the FDA’s marketing and promotional requirements, including ongoing training and monitoring, as well as unforeseen costs and expenses associated with creating an independent sales and marketing organization

• DroppedRisks Related to the Commercialization of Our Approved Products and Product Candidates

  We may not be able to obtain either three-year FDA regulatory exclusivity or five-year FDA regulatory exclusivity as a new chemical entity

• DroppedRisks Related to the Commercialization of Our Approved Products and Product Candidates

  Even if we or our collaborators are able to commercialize our approved products, or any of our product candidates, if approved, they may be subject to unfavorable pricing regulations, third-party coverage and reimbursement policies

• DroppedRisks Related to the Commercialization of Our Approved Products and Product Candidates

  the inability to successfully commercialize any of our approved products, or any of our product candidates that might be approved in the future

• DroppedRisks Related to the Commercialization of Our Approved Products and Product Candidates

  If commercialization of our approved products, or any of our product candidates, if approved, are not successful, or we experience significant delays in commercialization, our business will be harmed

• DroppedRisks Related to the Commercialization of Our Approved Products and Product Candidates

  maintaining a continued acceptable safety and efficacy profile of any of our products following approval

• DroppedRisks Related to the Commercialization of Our Approved Products and Product Candidates

  It is possible that none of our product candidates in clinical development or any of our future product candidates will ever obtain regulatory approval, even if we expend substantial time and resources seeking such approval

• DroppedRisks Related to the Development of Our Product Candidates

  even if we obtain marketing approval in one or more countries, future safety or other issues could result in the suspension or withdrawal of regulatory approval in such countries

• DroppedRisks Related to the Development of Our Product Candidates

  Any product candidates we develop may prove to have undesirable or unintended side effects, toxicities or other characteristics that may preclude our obtaining regulatory approval or prevent or limit commercial use with respect to one or all intended indications

• DroppedRisks Related to the Development of Our Product Candidates

  Clinical drug development involves a lengthy and expensive process, with an uncertain outcome. We may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development and commercialization of our product candidates

• DroppedRisks Related to the Development of Our Product Candidates

  our product candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators, regulators or IRBs to suspend or terminate the trials

• DroppedRisks Related to the Development of Our Product Candidates

  have the product removed from the market after obtaining marketing approval

• DroppedRisks Related to the Development of Our Product Candidates

  Certain of our approved products and product candidates contain controlled substances, the manufacture, use, sale, importation, exportation, prescribing and distribution of which are subject to regulation by the DEA and other regulatory agencies

• DroppedRisks Related to the Development of Our Product Candidates

  We may expend our limited resources to pursue a particular product candidate or indication and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success

• DroppedRisks Related to Our Business, Our Financial Position and Our Capital Needs

  Our future results will suffer if we do not effectively manage our expanded operations

• DroppedRisks Related to Our Business, Our Financial Position and Our Capital Needs

  A variety of risks associated with international operations could materially adversely affect our business

• DroppedRisks Related to Our Business, Our Financial Position and Our Capital Needs

  We may not be able to retain suppliers or distributors, or suppliers or distributors may seek to modify contractual relationships with us, which could have an adverse effect on our business and operations. Third parties may terminate or alter existing contracts or relationships with us

• DroppedRisks Related to Our Business, Our Financial Position and Our Capital Needs

  If we fail to maintain effective internal control over financial reporting and disclosure controls and procedures, we may not be able to accurately report our financial results or report them in a timely manner, which may adversely affect investor confidence in us and, as a result, the value of our common stock

• DroppedRisks Related to Our Business, Our Financial Position and Our Capital Needs

  We have incurred significant recurring negative net operating cash flows. We cannot predict if we will incur positive or negative net operating cash flows over the next several years, which may impact our ability to continue as a going concern, and we may never achieve or maintain profitability

• DroppedRisks Related to Our Business, Our Financial Position and Our Capital Needs

  add operational systems and personnel, if needed, to support any future commercialization efforts

• DroppedRisks Related to Our Business, Our Financial Position and Our Capital Needs

  We may need additional funding to pursue our business objectives. If we are unable to raise capital when needed, we could be forced to delay, reduce or altogether cease our development programs or commercialization efforts or cease operations altogether

• DroppedRisks Related to Our Dependence on Third Parties

  carrier disruptions or increased costs that are beyond our control

• DroppedRisks Related to Our Dependence on Third Parties

  Our approved products that we are commercializing, and product candidates that we are currently developing, or may develop in the future, may compete with other product candidates and drugs for access to manufacturing facilities, and we may be unable to obtain access to these facilities on favorable terms

• DroppedRisks Related to Our Dependence on Third Parties

  If we enter into any future collaborations we will face similar risks with any future collaborators as well

• DroppedRisks Related to Our Dependence on Third Parties

  If we are not able to establish collaborations for our product candidates, we may have to alter our development and commercialization plans

• DroppedRisks Related to Our Dependence on Third Parties

  Collaborations are complex and time-consuming to negotiate and document. In addition, there have been a significant number of recent business combinations among large pharmaceutical companies that have resulted in a reduced number of potential future collaborators

• DroppedRisks Related to Our Intellectual Property

  we may not develop additional proprietary technologies that are patentable

• DroppedRisks Related to Our Intellectual Property

  Any of these factors could hurt our ability to gain full patent protection for our products. Registered trademarks and trademark applications in the United States and other countries are subject to similar risks as described above for patents and patent applications, in addition to the risks described below

• DroppedRisks Related to Our Intellectual Property

  Changes in patent law in the United States and other jurisdictions could alter or diminish the value of patents in general, thereby impairing our ability to protect our products and technologies

• DroppedRisks Related to Our Intellectual Property

  Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses and could distract our technical and management personnel from their normal responsibilities

• DroppedRisks Related to Our Intellectual Property

  We may need to license intellectual property from third parties, and such licenses may not be available or may not be available on commercially reasonable terms

• DroppedRisks Related to Our Intellectual Property

  If we or our third-party licensors fail to comply with our obligations in our intellectual property licenses and funding arrangements with third parties, we could lose rights that are important to our business

• DroppedRisks Related to Our Intellectual Property

  The scope of our intellectual property may be reduced or may need to be reduced due to third-party intellectual property claims

• DroppedRisks Related to Our Intellectual Property

  We may be subject to claims by third parties asserting that we or our employees have misappropriated their intellectual property or claiming ownership of what we regard as our own intellectual property

• DroppedRisks Related to Our Intellectual Property

  Our decision to seek approval of our product candidates under certain regulatory pathways, such as the 505(b)(2) NDA pathway for the FDA, may increase the risk that patent infringement suits are filed against us, which would delay FDA or comparable foreign regulatory authorities' approval of such product candidates

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

  Failure to obtain marketing approval in international jurisdictions would prevent our approved products, and any of our other product candidates from being marketed abroad

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

  injunctions or the imposition of civil or criminal penalties, including False Claims Act liability

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

  Our employees, independent contractors, principal investigators, CROs, consultants, commercial collaborators, contract manufacturers, service providers and other vendors may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements

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

  These laws may affect our and our commercial partners' sales, marketing, and other promotional activities by imposing administrative and compliance burdens on us and our commercial partners

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

  a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research

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

  Governments outside the United States tend to impose strict price controls, which may affect our revenue, if any

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

  If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could harm our business

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

  We are subject to complex and changing laws and regulations, which exposes us to potential liabilities, increased costs and other adverse effects on our business

• DroppedRisks Related to Employee Matters and Managing Our Growth

  Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified personnel

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  Future sales and issuances of equity and debt could result in additional dilution to our stockholders

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  The holders of any of the warrants described elsewhere in this Annual Report on Form 10-K may elect to exercise such warrants and receive shares of common stock

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  providing for a supermajority requirement to amend our amended and restated bylaws

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  If appropriate opportunities become available, we may seek to acquire businesses or assets to enhance our business. In connection with any acquisitions, we could issue additional equity securities, which would dilute our stockholders, incur substantial debt to fund the acquisitions or assume significant liabilities

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  Securities litigation against us could result in substantial costs and divert management’s attention from other business concerns, which could seriously harm our business

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  Changes in tax laws or regulations that are applied adversely to us may have a material adverse effect on our business, cash flow, financial condition or results of operations

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  An active trading market for our common stock may not be sustained and you may not be able to resell your shares of our common stock for a profit, if at all

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  The increasing focus on environmental sustainability and social initiatives could increase our costs, harm our reputation and adversely impact our financial results

• DroppedRisks Related to Ownership of Our Common Stock and Our Status as a Public Company

  Adverse developments affecting the financial services industry, including events or concerns involving liquidity, defaults, or non-performance by financial institutions, could adversely affect our business, financial condition or results of operations

Reworded

• 100% rewrittenRisks Related to Ownership of Our Common Stock

  Many of the factors described above are not within our control and we cannot guarantee that future instances of these influencing factors will not have effects on the trading price of our common stock

  Swapped a discussion of past stockholder class action lawsuits for details on anti-takeover provisions and Delaware law.

• 93% rewrittenRisks Related to Regulatory Approval of Our Product Candidates and Other Legal Compliance Matters

  Current and future healthcare reform legislation or regulation 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

  Added a specific reference to the 2010 Affordable Care Act as a major legislative healthcare initiative.

  Was: Recently enacted and future legislation 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, and any of our product candidates that may be approved in the future and affect the prices thereof

• 89% rewrittenRisks Related to Ownership of Our Common Stock

  Because we do not anticipate paying any cash dividends on our common stock in the foreseeable future, capital appreciation, if any, will be your sole source of gains and you may never receive a return on your investment

  Changed the dividend restriction reference from general future debt agreements to our current credit agreement.

• 85% rewrittenRisks Related to the Commercialization of Our Approved Products and Product Candidates

  Our product candidates may be associated with serious adverse events, undesirable side effects or have other properties that could halt their clinical development, prevent their regulatory approval, limit their commercial potential or result in significant negative consequences

  Removed introductory sentences regarding patient reporting of illnesses and tightened language on clinical trial suspensions.

  Was: Our products and product candidates may be associated with serious adverse events, undesirable side effects or have other properties that could halt their clinical development, prevent their regulatory approval, limit their commercial potential or result in significant negative consequences

• 84% rewrittenRisks Related to Regulatory Approval of Our Product Candidates and Other Legal Compliance Matters

  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

  Updated phrasing on Corium's responsibility for AZSTARYS pediatric studies from future-looking to active present tense.

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

• 80% rewrittenRisks 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

  Minor phrasing adjustments regarding FDA regulatory oversight and new drug application requirements.

  Was: If we are not able to obtain required regulatory approvals for any of our product candidates, or the approved labels are not sufficiently differentiated from other competing products, we will not be able to commercialize them and our ability to generate revenue or profits or to raise future capital could be limited

• 77% rewrittenRisks Related to the Commercialization of Our Approved Products and Product Candidates

  We face substantial competition, which may result in others developing or commercializing products before or more successfully than we do

  Minor punctuation and cross-reference wording updates regarding competitor information.

• 77% rewrittenRisks Related to Our Dependence on Third Parties

  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

  Streamlined CRO reliance text to explicitly incorporate good clinical practices and regulatory inspection standards.

  Was: 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, including failing to meet deadlines for the completion of such trials

• 74% rewrittenRisks Related to the Commercialization of Our Approved Products and Product Candidates

  Product liability lawsuits against us could cause us to incur substantial liabilities and to limit commercialization of our approved products, or any of our product candidates that we may develop

• 71% rewrittenRisks Related to Regulatory Approval of Our Product Candidates and Other Legal Compliance Matters

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

  Was: We may not be able to obtain or maintain orphan drug designations which we pursue for any of our product candidates, and we may be unable to maintain the benefits associated with orphan drug designation, including the potential for market exclusivity

• 66% rewrittenRisks 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 approved products, including any of our product candidates that may receive marketing approval, we may not be successful in commercializing such products in the United States or any other jurisdictions

  Was: If we are unable to establish effective sales, marketing and distribution capabilities for our approved products or product candidates, if approved, we may not be successful in commercializing any approved product candidate in the United States or any other jurisdictions

• 65% rewrittenRisks Related to Our Intellectual Property

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

• 60% rewrittenRisks Related to Our Intellectual Property

  If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed

• 59% rewrittenRisks Related to Regulatory Approval of Our Product Candidates and Other Legal Compliance Matters

  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

• 58% rewrittenRisks Related to the Commercialization of Our Approved Products and Product Candidates

  If our Global EAP program is terminated prior to regulatory approval and commercialization of arimoclomol in applicable foreign jurisdictions, it will have a material adverse effect on our business, results of operations, cash flows, financial condition and/or prospects

  Was: If the EAP is terminated prior to commercialization of arimoclomol in the EU, if approved, it will have a material adverse effect on our business, results of operations, cash flows, financial condition and/or prospects

• 55% rewrittenRisks Related to Our Intellectual Property

  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

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

• 55% rewrittenRisks Related to the Commercialization of Our Approved Products and Product Candidates

  Our approved products, as well as any of our product candidates that may receive marketing approval, may fail to achieve the degree of market acceptance by physicians, patients, third-party payors and others in the medical community necessary for commercial success

  Was: Our approved products, or any of our product candidates that may receive marketing approval, may fail to achieve the degree of market acceptance by physicians, patients, third-party payors and others in the medical community necessary for commercial success

• 50% rewrittenRisks Related to Ownership 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

• 45% rewrittenRisks Related to Regulatory Approval of Our Product Candidates and Other Legal Compliance Matters

  Interim, topline, or preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data

• 43% rewrittenRisks Related to Our Business and Our Financial Position

  We might not be able to utilize a significant portion of our net operating loss carryforwards, which could adversely affect our profitability

• 42% rewrittenRisks Related to the Commercialization of Our Approved Products and Product Candidates

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

• 39% rewrittenRisks Related to the Commercialization of Our Approved Products and Product Candidates

  A variety of risks associated with marketing our approved products and any of our product candidates, if approved, internationally, could affect our business

• 36% rewrittenRisks Related to Our Intellectual Property

  Any trademarks we may obtain may be infringed or successfully challenged, resulting in harm to our business

• 34% rewrittenRisks 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

• 27% rewrittenRisks Related to Our Intellectual Property

  We may not be able to protect our intellectual property rights throughout the world

All 42 risk factors

Headings as the filing states them, in filing order.

Other Zevra Therapeutics 10-Ks

• FY2024 10-K risk factors

  115 risks

  Zevra relies heavily on the commercial success of its rare disease products MIPLYFFA and OLPRUVA. Market adoption depends on physician acceptance, third-party payor reimbursement, and competing therapies like AQNEURSA. Strict regulatory compliance, including FDA post-approval requirements and controlled substance laws, governs product manufacturing and distribution.

  Filed Mar 12, 2025

About this page

The headings are the filing's own. The one-line readings and the overview are automated and can be wrong: read Item 1A of the 10-K for the exact wording.