Zevra Therapeutics(ZVRA) - 2023 Q4 - Annual Report

Explanatory Note Restatement of Consolidated Financial Results The company restated its 2022 and 2023 interim financial statements due to inappropriate accounting for common stock warrants as liabilities - On March 25, 2024, the Audit Committee determined that financial statements for FY 2021, FY 2022, and interim periods in 2022 and 2023 were unreliable[22][23] - The core issue was the incorrect accounting of certain common stock warrants as equity instead of liabilities, leading to understatements of derivative and warrant liability and fluctuations in fair value adjustments[22] - This Form 10-K includes the restated financial data for the affected periods, superseding previously filed reports[24][25] PART I Business Zevra Therapeutics develops and commercializes rare disease therapies, including OLPRUVA® and AZSTARYS®, leveraging internal development and strategic acquisitions - The company focuses on rare diseases, leveraging its expertise in navigating complex regulatory challenges. This is reflected in its name change to Zevra, the Greek word for zebra, the symbol for rare diseases[26][27][28] - Strategic growth is driven by internal development and acquisitions, including the May 2022 purchase of arimoclomol assets and the November 2023 merger with Acer Therapeutics, adding OLPRUVA® and celiprolol[30][31] - The company's recurring operating losses and negative cash flows raise substantial doubt about its ability to continue as a going concern, necessitating additional financing[32] Our Product Candidates and Approved Products Zevra's portfolio includes approved products OLPRUVA® and AZSTARYS®, alongside clinical candidates arimoclomol, celiprolol, and KP1077 Active Commercial and Development Assets | Product / Candidate | Indication | Development Status | Next Milestone(s) | | :--- | :--- | :--- | :--- | | OLPRUVA | Urea Cycle Disorders (UCD) | FDA Approved | Tracking Commercial Progress | | Arimoclomol | Niemann Pick disease type C (NPC) | Pending FDA Review | PDUFA target date September 21, 2024 | | Celiprolol | Vascular Ehlers Danlos Syndrome (vEDS) | Clinical - Phase 3 ongoing | - | | KP1077IH | Idiopathic Hypersomnia (IH) | Clinical - Phase 2 | Evaluation of potential Phase 3 Trial | | KP1077N | Narcolepsy | Clinical - Phase 1/2 | Evaluation of potential Phase 3 Trial | | AZSTARYS | Attention Deficit and Hyperactivity Disorder (ADHD) | FDA Approved and Partnered | Collecting royalties and milestones | - OLPRUVA® was acquired through the Acer merger and its full commercial launch began on January 29, 2024, supported by an in-house rare disease commercial team[39][40] - The NDA for arimoclomol was resubmitted in December 2023 with a new PDUFA date of September 21, 2024, potentially qualifying for a Rare Pediatric Disease Priority Review Voucher[41][46][47] - The partnered product, AZSTARYS®, achieved over $50 million in annual net sales in 2023, triggering a $10.0 million milestone payment to Zevra[78] Our Intellectual Property Zevra protects its assets with 62 U.S. and 241 foreign patents, expiring between 2029 and 2038, and trade secrets for its LAT® platform - As of year-end 2023, the company maintains 62 active U.S. patents and 241 active foreign patents, with patent terms extending between 2029 and 2042[83] - Key patent expirations for major assets include: arimoclomol (2029), OLPRUVA (2036), and celiprolol (2038)[84][85][93] - The company also relies on regulatory exclusivities, such as New Chemical Entity (NCE) for celiprolol and Orphan Drug designation for multiple candidates[89][92] Commercialization Zevra launched OLPRUVA® in the U.S. with an in-house team, while partner Corium commercializes AZSTARYS® domestically and in Greater China - The full U.S. commercial launch of OLPRUVA® for UCDs began on January 29, 2024, supported by a newly built, in-house rare disease commercial team[95] - AZSTARYS® for ADHD is commercialized in the U.S. by partner Corium, Inc. and has been sublicensed for Greater China[96] Competition Zevra faces intense competition for OLPRUVA® (RAVICTI®), arimoclomol (ZAVESCA®), KP1077 (XYWAV®), and AZSTARYS® in the crowded ADHD market - OLPRUVA® competes with Amgen's RAVICTI® and Medunik's PHEBURANE®, with potential for RAVICTI® generics as early as July 2025[100] - Arimoclomol for NPC faces competition from off-label use of miglustat (ZAVESCA®) and a pending NDA from IntraBio for its candidate IB1001[101] - KP1077 for IH will compete with the approved product XYWAV® from Jazz Pharmaceuticals[103] - AZSTARYS® competes in a highly saturated ADHD market against numerous branded products like CONCERTA® and RITALIN®, as well as generics[104] Government Regulation Zevra's operations are subject to extensive FDA regulation, including clinical trials, NDA approval, cGMP, and post-market rules, alongside DEA oversight for controlled substances and healthcare reform impacts - The company must navigate a multi-stage drug approval process, including preclinical studies and three phases of human clinical trials, before submitting an NDA to the FDA[118][121][124] - Zevra utilizes the 505(b)(2) NDA pathway, allowing reliance on existing data for previously approved drugs to potentially streamline development, as was done for AZSTARYS[125][148] - Products like AZSTARYS (Schedule II) and product candidates containing SDX (Schedule IV) are regulated as controlled substances by the DEA, imposing strict manufacturing, distribution, and prescribing requirements[155][156][157] - The business is subject to healthcare reform measures, including the Affordable Care Act (ACA) and the Inflation Reduction Act (IRA), which can impact drug pricing, reimbursement, and overall profitability[172][175] Risk Factors The company faces significant risks including commercialization failures, financial instability, integration challenges, restatement issues, internal control weaknesses, reliance on third parties, intense competition, IP challenges, and stringent government regulation - The company's recurring operating losses and negative cash flows raise substantial doubt about its ability to continue as a going concern[15][278][284] - A material weakness in internal control over financial reporting was recently identified, related to the accounting for common stock warrants, which led to a restatement of prior financial statements[15][274][275] - Successful commercialization of approved products like OLPRUVA and partnered products like AZSTARYS is not guaranteed and is critical to future revenue[15][203] - The company faces risks in integrating the acquired Acer business and realizing anticipated synergies[13][267] - Clinical development is lengthy, expensive, and uncertain, with a high risk of failure for product candidates like arimoclomol, celiprolol, and KP1077[15][225] - The company's products contain controlled substances (e.g., AZSTARYS is Schedule II), making them subject to strict DEA regulations, including production quotas, which could limit supply and commercial potential[247][249][251] Unresolved Staff Comments The company reports no unresolved staff comments - There are no unresolved staff comments[496] Cybersecurity Zevra's cybersecurity program, based on NIST CSF, is overseen by the Audit Committee, with no material incidents reported to date - The company's cybersecurity program is designed and assessed based on the National Institute of Standards and Technology Cybersecurity Framework (NIST CSF)[498] - Oversight is provided by the Audit Committee of the Board of Directors, with operational responsibility held by a management team led by the CFO[501][503] - The company reports that no known cybersecurity threats or incidents have had a material effect on its operations, business strategy, or financial condition[499] Properties Zevra's headquarters is leased in Celebration, Florida, with additional leased office and lab facilities in Massachusetts, Iowa, Virginia, and Denmark - The company's principal executive offices are leased in Celebration, Florida[505] - Additional leased facilities include office and lab spaces in Massachusetts, Iowa, Virginia, and Denmark[505] Legal Proceedings The company is involved in routine legal proceedings, including stockholder litigation from the Acer merger, but management expects no material adverse effect - The company is party to stockholder litigation inherited from the Acer merger, which alleged material omissions in proxy statements related to the transaction[506][789][790][791] - Management believes no pending litigation is reasonably expected to have a material adverse effect on the company[506] Mine Safety Disclosures This item is not applicable to the company - This item is not applicable to the company[507] PART II Market for Registrant's Common Equity, Related Stockholder Matters, and Issuer Purchases of Equity Securities The company's common stock trades on Nasdaq under 'ZVRA', with no history of cash dividends, as earnings are retained for business growth - The company's common stock trades on The Nasdaq Global Select Market under the ticker 'ZVRA'[527] - The company has never declared or paid cash dividends and intends to retain future earnings for business operations and expansion[529] Management's Discussion and Analysis of Financial Condition and Results of Operations Zevra reported a $46.0 million net loss in 2023 due to increased R&D and SG&A, despite revenue growing to $27.5 million, with $67.7 million cash on hand, raising going concern doubts Results of Operations In 2023, Zevra's net loss increased to $46.0 million due to higher R&D ($20.0 million) and SG&A ($19.3 million) expenses, despite revenue growing to $27.5 million from AZSTARYS® milestones Comparison of the Years Ended December 31, 2023, and 2022 (in thousands) | | 2023 | 2022 (As Restated) | Period-to-Period Change | | :--- | :--- | :--- | :--- | | Revenue, net | $27,461 | $10,161 | $17,300 | | Research and development | $39,806 | $19,803 | $20,003 | | Selling, general and administrative | $34,314 | $15,038 | $19,276 | | Acquired in-process research and development | $0 | $17,663 | ($17,663) | | Loss from operations | ($49,604) | ($42,565) ($7,039) | | Net loss | ($46,049) | ($26,772) | ($19,277) | - Revenue growth in 2023 was primarily driven by an $18.5 million increase from the AZSTARYS License Agreement, including $15.0 million in net sales milestones[576][728] - R&D expenses rose by $20.0 million due to increased third-party costs ($14.5 million) and personnel costs ($2.6 million) related to advancing the KP1077 and arimoclomol programs[578] - SG&A expenses increased by $19.3 million, driven by higher professional fees ($8.7 million, including $2.2 million in Acer transaction costs) and personnel-related costs ($9.7 million)[579] Liquidity and Capital Resources As of December 31, 2023, Zevra had $67.7 million in cash, with $33.5 million negative operating cash flow in 2023, raising going concern doubts, and expects funds to last into 2026 - The company ended 2023 with $67.7 million in cash, cash equivalents, and investments[582] - Recurring operating losses and negative cash flows raise substantial doubt about the company's ability to continue as a going concern. Existing resources are projected to last into, but not through, 2026[586][609] - As of December 31, 2023, $37.7 million was outstanding under a margin account agreement with Wells Fargo, collateralized by the company's investments[599] Cash Flow Summary (in thousands) | | Year Ended Dec 31, 2023 | Year Ended Dec 31, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($33,535) | ($18,717) | | Net cash used in investing activities | ($17,390) | ($36,719) | | Net cash provided by financing activities | $28,464 | $8,352 | Critical Accounting Estimates Critical accounting estimates include valuation of intangible assets from acquisitions, accrued R&D expenses, fair value of stock-based compensation, and warrant accounting, which led to a financial restatement - Valuation of intangible assets from acquisitions (e.g., arimoclomol IPR&D, OLPRUVA) is a critical estimate, using income approaches with significant assumptions about future revenue and success rates[616][617] - Accruing expenses for R&D services from third-party vendors requires estimating the level of service performed before invoices are received[620] - Stock-based compensation is valued using the Black-Scholes model, which requires subjective assumptions for volatility, expected term, and interest rates[622][623] - Certain common stock warrants are accounted for as liabilities and re-measured to fair value each period, with changes recognized in the statement of operations. An error in this accounting led to the financial restatement[629][630][631] Quantitative and Qualitative Disclosures About Market Risk This item is not applicable to the company - This item is not applicable to the company[632] Financial Statements and Supplementary Data This section presents Zevra's audited financial statements, with the auditor's report noting the 2022 restatement, going concern doubt, and a critical audit matter regarding Acer acquisition valuation - The independent auditor's report includes an explanatory paragraph expressing substantial doubt about the Company's ability to continue as a going concern due to recurring losses and negative cash flows[662] - The auditor's report also notes that the 2022 financial statements have been restated to correct misstatements related to the accounting for certain warrants[661] - A Critical Audit Matter was identified related to the valuation of contingent consideration and the OLPRUVA intangible asset acquired in the Acer business combination, due to the significant estimation and subjective assumptions involved[667] Changes in and Disagreements With Accountants on Accounting and Financial Disclosures The company reports no changes in or disagreements with its accountants on accounting and financial disclosure - There were no disagreements with accountants on accounting and financial disclosures[634] Controls and Procedures Management concluded that disclosure controls were ineffective as of December 31, 2023, due to a material weakness in warrant accounting, leading to financial restatement, with remediation efforts underway - A material weakness was identified in internal controls over financial reporting concerning the accounting for warrants to purchase the company's common stock[636][639] - As a result of this material weakness, management concluded that disclosure controls and procedures were not effective as of December 31, 2023[637] - Remediation efforts are underway, including reviewing all existing warrant accounting and developing enhanced control procedures[641][642] Other Information No directors or officers adopted, modified, or terminated Rule 10b5-1 trading plans during the fourth quarter of 2023 - No directors or officers adopted, modified, or terminated any Rule 10b5-1 trading arrangements during the three months ended December 31, 2023[645] PART III Directors, Executive Officers and Corporate Governance Information on directors, executive officers, and corporate governance is incorporated by reference from the company's 2024 proxy statement - The information required for this item is incorporated by reference from the company's 2024 proxy statement[649] Executive Compensation Information on executive and director compensation is incorporated by reference from the company's 2024 proxy statement - The information required for this item is incorporated by reference from the company's 2024 proxy statement[651] Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters Information on security ownership and equity compensation plans is incorporated by reference from the company's 2024 proxy statement - The information required for this item is incorporated by reference from the company's 2024 proxy statement[652] Certain Relationships and Related Transactions, and Director Independence Information on related party transactions and director independence is incorporated by reference from the company's 2024 proxy statement - The information required for this item is incorporated by reference from the company's 2024 proxy statement[653] Principal Accounting Fees and Services Information on principal accounting fees and services is incorporated by reference from the company's 2024 proxy statement - The information required for this item is incorporated by reference from the company's 2024 proxy statement[654] PART IV Exhibits and Financial Statement Schedules This section provides an index to the company's consolidated financial statements and a list of all exhibits filed with the Form 10-K - This section provides an index to the financial statements and lists all exhibits filed with the report[655][656] Form 10-K Summary No Form 10-K summary is provided - No Form 10-K summary is provided[871]