📜 What This Document Is 🗳️

This document is Zevra Therapeutics’ Proxy Statement (a detailed legal filing, or Proxy Statement). Think of it as the company's annual "report card" and a voting ballot rolled into one. It is designed to provide shareholders with all the necessary information before the Annual Meeting of Stockholders to vote on major corporate governance issues.

Since the Annual Meeting is happening on June 4, 2026, this statement covers both the company's financial performance in 2025 and the decisions that need to be made by shareholders for the future.

👉 Key Takeaway: This document isn't just about money; it's primarily about the company's rules, who runs the company, and the major strategic decisions the Board is asking you, the shareholder, to approve.

🏥 Zevra's Mission and Focus 🌟

In simple terms, Zevra is a commercial-stage biotechnology company focused entirely on developing life-changing therapies for people living with rare diseases. The core of their business model is advancing novel, specialized treatments that fill unmet medical needs.

Their commercial success is currently anchored by MIPLYFFA® (arimoclomol), a treatment for Niemann-Pick disease type C (NPC)—a rare, progressive neurodegenerative disease. Zevra aims to achieve long-term value by advancing its pipeline and expanding access globally.

👉 Why It Matters: Because the company operates in the highly specialized rare disease space, its entire value hinges on successful clinical trials and gaining regulatory approval in multiple international markets.

💰 2025 Financial Performance Highlights 💲

The filing summarized Zevra’s financial performance for the year ending December 31, 2025. The company reported strong revenues, with MIPLYFFA serving as the primary revenue generator.

• Total Net Revenue: Zevra generated $106.5 million in net revenue for the full year.
  • What this means: This metric shows the total amount of money the company brought in from sales of its products, proving the commercial traction of its lead drug.
• MIPLYFFA Revenue: Of that total, $87.4 million came specifically from MIPLYFFA®.
  • What this means: This confirms that MIPLYFFA is the dominant revenue driver, showcasing the commercial adoption of the drug in the U.S. market.
• GAAP Diluted EPS: The GAAP diluted Earnings Per Share (EPS) was $1.35.
  • What this means: EPS measures the company's profitability relative to the number of shares outstanding, giving a core metric of financial efficiency.

📈 Operational and Pipeline Progress 🚀

Beyond revenues, the company detailed significant progress across both its existing product and its developmental pipeline assets. This signals that the company is actively growing its global footprint and scientific portfolio.

• MIPLYFFA Commercial Success: The company reached "more than 40% of diagnosed patients" in its first full year of U.S. commercialization. They also recorded 161 MIPLYFFA enrollment forms, showing solid adoption among patients.
  • What this means: High adoption numbers and positive patient feedback translate to market validation and increasing long-term revenue potential.
• Global Expansion & Pipeline Advancement:
  • EU Application: Zevra submitted the European Marketing Authorization Application (MAA) for arimoclomol in July of 2025.
  • Access Program: They continued through their global Expanded Access Program (EAP), providing therapy to 113 patients.
  • Celiprolol: The late-stage development asset, celiprolol, advanced its Phase 3 DiSCOVER trial, with approximately one-third of patients enrolled as of year-end 2025.
  • Key Strategic Decision: The company sold its Rare Pediatric Disease Priority Review Voucher (PRV) in 2025, generating $150 million.
    • What this means: Selling the PRV is a major financial lift for the company, providing significant capital to fund future research and operations.
• Resource Management: They made the decision to scale back promotional investment for OLPRUVA, directing resources instead toward programs with the greatest long-term impact.

💼 Corporate Strategy and Management Enhancements ✨

Zevra outlined several organizational and financial moves designed to strengthen its foundation and position it for future growth.

• Leadership Appointments: In 2026, the company strengthened its leadership by appointing Justin Renz as Chief Financial Officer (CFO).
  • What this means: Hiring a dedicated CFO is crucial for financial scaling and signals a commitment to operational rigor and financial management.
• Corporate Footprint: The company relocated its headquarters to Boston, positioning itself in a leading global biotech ecosystem.
  • What this means: This move is strategic, giving them closer access to specialized talent and necessary industry partnerships.
• Balance Sheet Strengthening: The company was able to repay its term loan, which strengthens its balance sheet and enhances its financial flexibility.

🏛️ Annual Meeting and Governance Votes 🗳️

Since this is a proxy statement, a major portion of the content is focused on corporate governance—the "rules" of the company. The Annual Meeting is scheduled for June 4, 2026, and will be held virtually.

🧬 Proposals for Shareholder Approval

Three specific proposals are before the shareholders for voting:

1. Election of Directors: Electing two new Class II directors (Douglas W. Calder and Corey Watton) to serve until the 2029 annual meeting.
   • Recommendation: The Board recommends voting "FOR" these nominees.
2. Accounting Firm Ratification: Ratifying the appointment of Ernst & Young LLP as the independent registered public accounting firm for the fiscal year ending December 31, 2026.
   • Recommendation: The Board recommends voting "FOR" this proposal.
3. Board Declassification: Approving an amendment to the Restated Certificate of Incorporation to phase out the classified Board structure. If approved, all directors elected at or after the 2027 annual meeting will be elected on an annual basis.
   • What this means: This changes the fundamental rules of how directors are elected, making the structure more transparent and responsive to shareholders.

👥 Board Leadership and Governance Structure

The Board is comprised of multiple experienced leaders and has a detailed structure designed to ensure accountability.

• Current Board Composition: The Board includes experienced members like Tamara A. Favorito (Board Chair), Neil F. McFarlane (CEO), and Alicia Secor.
• Director Independence: The Board determined that seven directors are independent under Nasdaq listing standards, ensuring a majority of the Board is free from company ties.
  • Role Clarification: The CEO (Neil F. McFarlane) is noted as not an independent director by virtue of his employment.
• Board Committees: The Board maintains three standing committees:
  • Audit Committee: Chaired by Tamara A. Favorito. Its principal duties include overseeing the annual audit, ensuring the accounting firm's independence, and reviewing financial risks.
  • Compensation Committee: Chaired by Thomas D. Anderson. It is responsible for setting and recommending compensation levels for the CEO and other executives.
  • Nominating and Corporate Governance Committee: This committee oversees all corporate governance functions, including recommending new directors and ensuring the Board maintains a mix of necessary skills.
• Risk Oversight: The Board maintains overall direct oversight of risk management, including key areas like cybersecurity and financial risks.

📅 Important Logistics & Next Steps ℹ️

This section contains all the practical information necessary for shareholders to participate in the meeting and vote.

• Annual Meeting Details:
  • Date: June 4, 2026
  • Time: 9:00 a.m. Eastern Time
  • Location: Held virtually on the Internet at meetnow.global/M4K7FSC
• Key Dates:
  • Record Date: April 6, 2026 (Only stockholders of record on this date can vote).
  • Proposal Submission Deadline: For proposals for the 2027 meeting, the deadline is December 21, 2026.
• Voting Mechanism: Shareholders can vote online, by telephone, or by proxy card. The instructions require careful attention to how shares are held (whether registered in their name or by a broker/bank).

🧠 The Analogy 🧩

Governing a biotech company like Zevra is like managing a complex, high-stakes research lab and startup all at once. The scientific research (MIPLYFFA and celiprolol) is the exciting, unpredictable discovery phase. The financial and corporate governance parts (the proxy statement) are the required machinery—the reliable systems, quality checks, and rules (the Board structure, the committees, the auditors) that ensure the lab can function year after year, even when a major breakthrough or setback happens.

🧩 Final Takeaway 🏆

Zevra is leveraging strong commercial traction from MIPLYFFA and a significant capital infusion from the PRV sale to fund its next growth phases. Shareholders are voting on both core governance reforms (like annual director elections) and endorsing the operational foundation required to drive the next chapter of its rare disease pipeline.