• Alexion Pharmaceuticals, but for a different portfolio of rare diseases.
• Vertex Pharmaceuticals, but for a broader array of rare diseases.
• Amgen, but exclusively focused on rare disease therapies.

• Azstarys: A commercialized central nervous system stimulant medication for Attention-Deficit/Hyperactivity Disorder (ADHD), for which Zevra receives royalties from its licensee.
• ZV-107: An investigational drug candidate in development for rare neurological sleep disorders, including Idiopathic Hypersomnia and Narcolepsy.
• Arimoclomol: An investigational oral therapy being developed for the treatment of Niemann-Pick type C (NPC) disease, a rare and fatal neurodegenerative disorder.
• KP879: An investigational prodrug candidate designed to treat Stimulant Use Disorder (SUD).

Zevra Therapeutics (ZVRA) primarily sells its pharmaceutical products to other companies, specifically major wholesale drug distributors who then supply pharmacies, hospitals, and other healthcare providers. As a pharmaceutical company, Zevra does not typically sell directly to individual consumers.

The major customer companies for Zevra Therapeutics are expected to include the largest pharmaceutical wholesalers in the United States:

• McKesson Corporation (Symbol: MCK)
• Cencora, Inc. (formerly AmerisourceBergen Corporation) (Symbol: COR)
• Cardinal Health, Inc. (Symbol: CAH)

• Merck KGaA (MRKGY)
• Takeda Pharmaceutical Company Limited (TAK)
• Catalent Pharma Solutions, Inc. (CTLT)

Neil F. McFarlane, President & Chief Executive Officer

Mr. McFarlane has served as President and Chief Executive Officer of Zevra Therapeutics since October 2023. He brings over 25 years of global biopharmaceutical and life sciences experience. Prior to joining Zevra, he was the Chief Executive Officer and a member of the Board of Directors of Adamas Pharmaceuticals, Inc. until its acquisition by Supernus Pharmaceuticals, Inc. in November 2021. He previously served as Chief Operating Officer of Retrophin, Inc. (now Travere Therapeutics, Inc.) from 2016 to 2019. Mr. McFarlane also held positions of increasing responsibility at UCB, Inc., Genzyme Corporation (now Sanofi), and Sangstat Medical Corporation, which was acquired by Genzyme. He has served on the board of Collegium Pharmaceutical, Inc.

R. LaDuane Clifton, MBA, CPA, Chief Financial Officer and Treasurer

As Chief Financial Officer and Treasurer, Mr. Clifton is responsible for Zevra's financial, operating, strategy, and business development activities. He possesses significant public company and global operations leadership experience, along with expertise in managed care contracting, compliance, investor relations, and business development. Mr. Clifton joined Zevra in 2015. Before Zevra, he served as Chief Financial Officer, Secretary, and Treasurer of The LGL Group, Inc., a publicly held global producer of highly engineered electronic components and services. He was also previously the Chief Financial Officer of a21, Inc., a publicly held holding company. His earlier career included various finance, medical cost analysis, and managed care provider contracting roles with Aetna, Inc., and he was an auditor with KPMG, LLP.

Adrian Quartel, M.D., FFPM, Chief Medical Officer

Dr. Quartel joined Zevra Therapeutics as Chief Medical Officer in 2024, bringing over 20 years of experience in the international pharmaceutical industry with senior roles in clinical development, pharmacovigilance, and medical affairs. He previously served as Chief Medical Officer of Acer Therapeutics, Inc., which was acquired by Zevra, where he guided clinical development, medical affairs, and regulatory compliance. Prior to Acer, he was Chief Medical Officer at Adamas Pharmaceuticals from 2020 to 2022, a company focused on drug development for neurological diseases. Dr. Quartel also served as Group Vice President of Global Medical Affairs at BioMarin Pharmaceutical Inc., where he led the launch of six treatments for rare diseases or genetic disorders. His experience also includes senior medical leadership positions at Astellas, Chiltern, and ICON Clinical Research.

Rahsaan W. Thompson, Chief Legal Officer, Secretary and Compliance Officer

Mr. Thompson was appointed Chief Legal Officer, Secretary, and Compliance Officer in June 2024. He brings more than 25 years of experience as a business leader and attorney within the biotech industry. Before joining Zevra, Mr. Thompson was the Chief Legal Officer at Lyell Immunopharma, a publicly traded and clinical-stage biotech corporation. His extensive experience encompasses corporate governance, clinical studies, intellectual property, licensing arrangements, litigation, government affairs, SEC compliance, and commercial management.

The key risks to Zevra Therapeutics' business include:

1. Clinical Trial and Regulatory Success: As a biotechnology company focused on developing therapies for rare diseases, Zevra Therapeutics' financial performance and future growth are heavily dependent on the successful outcome of its research and development (R&D) efforts, particularly its clinical trials, and subsequent regulatory approvals. The inherent unpredictability of drug development means that clinical trials can fail, or regulatory agencies may delay or deny approval for pipeline candidates like KP1077 (for idiopathic hypersomnia and narcolepsy) and Celiprolol (for vascular Ehlers Danlos syndrome).
2. Product Concentration and Commercialization Challenges in Rare Diseases: Zevra Therapeutics currently relies significantly on its approved therapy, MIPLYFFA, for a substantial portion of its revenue. Any setbacks in MIPLYFFA's adoption or unexpected market challenges could materially impact the company's financial performance. Furthermore, the commercialization of therapies for rare diseases presents unique challenges, including navigating narrow patient pools, slow diagnosis rates, limited physician awareness, intense competition, and resistance from payers regarding reimbursement. Diversifying its product portfolio is crucial for long-term stability.
3. Achieving and Sustaining Profitability: Despite experiencing impressive revenue growth, Zevra Therapeutics has faced challenges in consistently converting revenue into profit, indicated by a negative earnings per share (EPS) and the absence of a trailing price-to-earnings (P/E) ratio. The company also recorded a significant intangible asset impairment of $58.7 million related to OLPRUVA and an inventory write-down in the second quarter of 2025, suggesting difficulties in realizing expected post-acquisition synergies and asset value. While recent strategic capital events have strengthened its balance sheet, the inherent risks associated with unprofitable biotech firms, including high operating and R&D expenses, persist.

Addressable Markets for Zevra Therapeutics' Main Products

Zevra Therapeutics (NASDAQ: ZVRA) focuses on developing and commercializing therapies for rare diseases. The addressable markets for their main products are as follows:

• MIPLYFFA (arimoclomol) for Niemann-Pick disease type C (NPC):
  • In the U.S., the addressable market consists of approximately 300-350 diagnosed patients.
  • In Europe, the estimated patient population is around 1,100 individuals. This market has a potential annual revenue of $500–700 million if 70% market penetration is achieved, assuming a U.S.-style pricing model of approximately $250,000 per year.
• Celiprolol for Vascular Ehlers-Danlos syndrome (VEDS):
  • The global market for Vascular Ehlers-Danlos Syndrome (VEDS) is projected to reach $35.69 billion by 2034, growing at a compound annual growth rate (CAGR) of 3.42%.
• KP1077 for Idiopathic Hypersomnia (IH):
  • The addressable market in the U.S. includes approximately 37,000 currently diagnosed patients.

No specific addressable market size information in terms of patient numbers or revenue was found for OLPRUVA (Urea Cycle Disorders) or for AZSTARYS (Attention Deficit Hyperactivity Disorder) that directly relates to Zevra's market share or revenue beyond royalty income. No specific market size was found for KP1077 in Narcolepsy.

1. Continued U.S. Market Penetration of MIPLYFFA: Zevra's lead product, MIPLYFFA (arimoclomol), approved for Niemann-Pick disease type C (NPC), has demonstrated strong early performance with consistent growth in patient enrollment forms since its commercial launch in November 2024. The company's focus on increasing NPC diagnosis rates and treatment access beyond specialized centers is expected to further drive its adoption and revenue within the U.S. market.
2. Geographic Expansion of MIPLYFFA into Europe: Zevra Therapeutics has submitted or is on track to submit a Marketing Authorization Application (MAA) for MIPLYFFA with the European Medicines Agency (EMA) in the second half of 2025. This regulatory milestone could open up a significant new market for the product, positioning MIPLYFFA as a cornerstone treatment for NPC globally and contributing substantially to future revenue growth.
3. Potential Launch of Celiprolol for Vascular Ehlers-Danlos Syndrome (VEDS): The company is advancing Celiprolol, a late-stage pipeline asset currently in a Phase 3 trial (DiSCOVER trial) for the treatment of Vascular Ehlers-Danlos Syndrome (VEDS). A successful outcome of this trial and subsequent regulatory approval and launch would introduce a new product to Zevra's portfolio, offering a significant new revenue stream in an unmet medical need area.
4. Increased Commercial Momentum and Adoption of OLPRUVA: While adoption has been slower than anticipated, Zevra is implementing targeted commercial strategies for OLPRUVA (sodium phenylbutyrate), approved for certain Urea Cycle Disorders (UCDs). Efforts to engage with payers have already increased market access to 78% of covered lives, and ongoing commercial initiatives, particularly targeting adult UCD patients, are aimed at improving its market performance and contributing to revenue growth.

Share Repurchases

• KemPharm (now Zevra Therapeutics) authorized a $50 million share repurchase program on December 20, 2021, valid through December 31, 2023.
• Approximately $2.4 million in share repurchases were settled during Q4 2021.

Share Issuance

• In January 2021, KemPharm priced an underwritten public offering of 7,692,307 shares of common stock and accompanying warrants, with aggregate gross proceeds expected to total $50.0 million.
• In August 2024, Zevra Therapeutics priced an underwritten public offering of 9,230,770 shares of its common stock at $6.50 per share, for total gross proceeds of approximately $60.0 million.

Inbound Investments

• Zevra Therapeutics received $148.3 million in net proceeds from the sale of its Rare Pediatric Disease Priority Review Voucher in April 2025.
• In April 2024, Zevra refinanced existing debt with a new credit facility providing up to $100 million in committed capital, with an initial draw of $60 million used to retire approximately $43.1 million of existing debt.

Outbound Investments

• Zevra completed the acquisition of Acer Therapeutics Inc. in November 2023, which added OLPRUVA and Acer's pipeline of investigational product candidates to Zevra's portfolio.

Capital Expenditures

• Capital expenditures were $0.09 million in 2022, $0.30 million in 2023, and $0.00 million in 2024.
• The capital expenditures forecast median consensus for the next fiscal year (2025) is $1.0 million.